Hydroxyurea

also known as Hydroxycarbamide · Hydrea · Hydroxycarbamid · Droxia · Hidroxicarbamida · Hydroxyurea-Droxia-Hydrea-Hydroxycarbamide · NSC-32065 · ONCOCARBIDE (HYDROXYUREA) · +3 more

Other / unclassified

Developed for: sickle cell disease · HIV infectious disease · beta thalassemia · head and neck cancer · leukemia · stroke disorder · beta-thalassemia intermedia · esophageal cancer
+57 more
· meningioma · pulmonary hypertension · sickle cell-hemoglobin c disease syndrome · spinal muscular atrophy · acquired polycythemia vera · beta-thalassemia major · cancer · central nervous system neoplasm · chronic myeloid leukemia · gastric cancer · glioblastoma · gliosarcoma · myelodysplastic syndrome · refractory malignant neoplasm · acute leukemia of ambiguous lineage · acute lymphoblastic leukemia · acute myeloid leukemia · AIDS · astrocytoma (excluding glioblastoma) · brain glioblastoma · carcinoid tumor · carcinoma of pharynx · central retinal vein occlusion · desmoid tumor · disorder of defective peroxisome oxidative status · disorder of iron metabolism and transport · Epstein-Barr virus-associated lymphoproliferative disorder · extrahepatic bile duct carcinoma · head and neck squamous cell carcinoma · human papillomavirus-related squamous cell carcinoma · laryngeal carcinoma · lung cancer · lymphoma · malignant pancreatic neoplasm · multiple sclerosis · myelodysplastic/myeloproliferative neoplasm · myelofibrosis with myeloid metaplasia · nasal cavity neoplasm · nasopharyngeal type undifferentiated carcinoma · oral cavity cancer · oropharynx squamous cell carcinoma · paranasal sinus neoplasm · post-transplant lymphoproliferative disease · protein-energy malnutrition · sickle cell-beta zero-thalassemia · sickle cell-beta-thalassemia disease syndrome · small intestine cancer · squamous cell carcinoma · thalassemia · urinary bladder cancer · urothelial carcinoma · blood platelet disease · chronic myelomonocytic leukemia · essential thrombocythemia · myelofibrosis · myeloproliferative neoplasm · primary myelofibrosis
Investigated by: National Cancer Institute (NCI) · Children's Hospital Medical Center, Cincinnati · National Heart, Lung, and Blood Institute (NHLBI) · Duke University · National Institute of Allergy and Infectious Diseases (NIAID) · University of Chicago
+135 more
· Bristol-Myers Squibb · Theravia · Makerere University · Mulago Hospital, Uganda · Novartis Pharmaceuticals · St. Jude Children's Research Hospital · Vanderbilt University Medical Center · Aminu Kano Teaching Hospital · Baylor College of Medicine · Children's Hospital of Philadelphia · Columbia University · Doris Duke Charitable Foundation · Indiana University · Ann & Robert H Lurie Children's Hospital of Chicago · Bionoxx Inc. · Boston Children's Hospital · Children's Hospital Karachi · Children's Mercy Hospital Kansas City · Children's National Research Institute · Eastern Cooperative Oncology Group · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti · Medical University of South Carolina · National Institute of Blood and Marrow Transplant (NIBMT), Pakistan · National Institute of Neurological Disorders and Stroke (NINDS) · Northwestern University · Novartis · Stanford University · Triangle Pharmaceuticals · University of Alabama at Birmingham · University of Miami · University of Pittsburgh · Washington University School of Medicine · A.O. Ospedale Papa Giovanni XXIII · Agenzia Italiana del Farmaco · Ain Shams University · Assistance Publique - Hôpitaux de Paris · Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh · Barau Dikko Teaching Hospital · Beni-Suef Health insurance hospital · Beni-Suef University · Boehringer Ingelheim · Brown University · Bugando Medical Centre · Carelon Research · Caribbean Institute for Health Research (CAIHR) · Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts · Children's Healthcare of Atlanta · Children's Hospital of The King's Daughters · Children's Hospitals and Clinics of Minnesota · Consorzio Mario Negri Sud · Dartmouth-Hitchcock Medical Center · Dr.Saqib Hussain Ansari · East Carolina University · Emory University · First Affiliated Hospital of Harbin Medical University · For Drug Consulting · Fred Hutchinson Cancer Center · GERCOR - Multidisciplinary Oncology Cooperative Group · German CML Study Group · Glaxo Wellcome · Global Health Uganda LTD · Greater Cincinnati Foundation · Groupe Francophone des Myelodysplasies · Gustave Roussy, Cancer Campus, Grand Paris · Heidelberg University · Hopital Universitaire Robert-Debre · Hospital Infantil Dr. Robert Reid Cabral · III. Medizinische Klinik Mannheim · Imugene Limited · Indian Institute of Science · Institut National de la Transfusion Sanguine · Janssen-Cilag Ltd. · Jinja Regional Referral Hospital (JRRH), Sickle Cell Clinic, Jinja, Uganda · all 141 on the full record

Regulatory milestones approvals, filings & regulatory actions · 5 recorded

Milestone	Jurisdiction	Brand	Indication	Date	Sentence it was read from
Label expansion	US (FDA)	SIKLOS	—	2021-12-07	fda.gov ↗
Approved	EU (EMA)	Siklos	—	2007-06-29	europa.eu ↗
Label expansions (2)	US (FDA)	DROXIA	—	1998-02-25 – 2003-06-26	fda.gov ↗
Approved	US (FDA)	DROXIA	—	1967-12-07	fda.gov ↗

Trials 130 · started per year

today

US approved 1967

EU approved 2007

196719681969197019711972197319741975197619771978197919801981198219831984198519861987198819891990199119921993199419951996199719981999200020012002200320042005200620072008200920102011201220132014201520162017201820192020202120222023202420252026202720282029

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 02 trials
Phase 0	NCT05662098	Jun 2022 → Dec 2026 expected	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Active not recruiting	No outcome recorded
Phase 0	NCT01950585	Sep 2013 → Oct 2014	pulmonary hypertension	National Heart, Lung, and Blood Institute (NHLBI)	Withdrawn	No outcome recorded
Phase 114 trials
Phase 1	NCT07170592	Jan 2027 → Dec 2028 expected	refractory malignant neoplasm	Bionoxx Inc.	Not yet recruiting	No outcome recorded
Phase 1	NCT07190833	Mar 2026 → Dec 2028 expected	refractory malignant neoplasm	Bionoxx Inc.	Recruiting	No outcome recorded
Phase 1	NCT06005324	Dec 2023 → Jun 2026 overdue	squamous cell carcinoma	University of Chicago	Recruiting	No outcome recorded
Phase 1	NCT06063317	Oct 2023 → Feb 2026	cancer	Imugene Limited	Terminated	No outcome recorded Stop: Business
Phase 1	NCT03463733	Mar 2018 → Jun 2021	glioblastoma	M.E. van Linde	Unknown	No outcome recorded
Phase 1	NCT01521299	Mar 2012 → Dec 2013	cancer	Dartmouth-Hitchcock Medical Center	Withdrawn	No outcome recorded
Phase 1	NCT01506544	Dec 2011 → Dec 2013	sickle cell disease	Children's Mercy Hospital Kansas City	Completed	No outcome recorded
Phase 1	NCT00270478	Dec 2005 → Aug 2009	chronic kidney disease, pulmonary hypertension, sickle cell disease	National Heart, Lung, and Blood Institute (NHLBI)	Completed	No outcome recorded
Phase 1	NCT00387933	Jul 2005 → Jan 2009	central nervous system neoplasm	Duke University	Completed	No outcome recorded
Phase 1	NCT00056433	Mar 2003 → Dec 2006	sickle cell disease	National Institutes of Health Clinical Center (CC)	Completed	No outcome recorded
Phase 1	NCT00001818	Jun 1999 → Nov 2001	HIV infectious disease	National Cancer Institute (NCI)	Completed	No outcome recorded
Phase 1	NCT00002427	May 1999 → ?	HIV infectious disease	Research Institute for Genetic and Human Therapy	Completed	No outcome recorded
Phase 1	NCT00002887	Jul 1995 → ?	lung cancer	Ottawa Regional Cancer Centre	Unknown	No outcome recorded
Phase 1	NCT00001074	? → Jan 2000	HIV infectious disease	National Institute of Allergy and Infectious Diseases (NIAID)	Completed	No outcome recorded

Phase 1/211 trials · 1 met primary · 1 missed
Phase 1/2	NCT07708714	May 2026 → Nov 2030 expected	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Recruiting	No outcome recorded
Phase 1/2	NCT03763656	Jan 2019 → May 2021	sickle cell-beta-thalassemia disease syndrome, sickle cell-hemoglobin c disease syndrome	Nova Laboratories Limited	Completed	No outcome recorded
Phase 1/2	NCT02225132	Aug 2014 → May 2018	sickle cell disease	National Heart, Lung, and Blood Institute (NHLBI)	Completed	No outcome recorded
Phase 1/2	NCT01966731	Jun 2014 → Jul 2018 overdue	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Active not recruiting	Met primary
Phase 1/2	NCT00350844	Jul 2006 → Jun 2008	pulmonary hypertension, sickle cell disease	Ann & Robert H Lurie Children's Hospital of Chicago	Terminated	No outcome recorded Stop: Enrollment
Phase 1/2	NCT02480608	Apr 2004 → Aug 2011	chronic myeloid leukemia	University of Leipzig	Completed	Missed primary
Phase 1/2	NCT00568698	Jan 2004 → Feb 2012	spinal muscular atrophy	Stanford University	Completed	No outcome recorded
Phase 1/2	NCT00568802	Jan 2004 → Mar 2010	spinal muscular atrophy	Stanford University	Completed	No outcome recorded
Phase 1/2	NCT00004901	Oct 1999 → Jul 2004	head and neck cancer	Robert H. Lurie Cancer Center	Completed	No outcome recorded
Phase 1/2	NCT00004492	Oct 1999 → Sep 2001	sickle cell disease	University of North Carolina	Completed	No outcome recorded
Phase 1/2	NCT00008047	Aug 1999 → ?	esophageal cancer	GERCOR - Multidisciplinary Oncology Cooperative Group	Unknown	No outcome recorded

Phase 255 trials · 2 met primary · 2 missed
Phase 2	NCT06578507	Jan 2025 → Feb 2028 expected	sickle cell disease	Theravia	Recruiting	No outcome recorded
Phase 2	NCT07292259	Jan 2024 → Dec 2025	beta-thalassemia major	Pakistan Blood and Marrow Transplant (PBMT) Group	Completed	No outcome recorded
Phase 2	NCT06171217	Oct 2023 → Oct 2027 expected	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Active not recruiting	No outcome recorded
Phase 2	NCT05005182	Feb → Apr 2023	myelodysplastic/myeloproliferative neoplasm	Mayo Clinic	Terminated	No outcome recorded Stop: Enrollment
Phase 2	NCT05470270	Jul → Oct 2022	sickle cell disease	Theravia	Completed	No outcome recorded
Phase 2	NCT05142254	Apr 2022 → Jul 2023	priapism	Vanderbilt University Medical Center	Completed	No outcome recorded
Phase 2	NCT06490601	Apr 2022 → Apr 2024 overdue	beta thalassemia	National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	Active not recruiting	No outcome recorded
Phase 2	NCT06239389	Sep → Dec 2021	beta thalassemia	National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	Completed	No outcome recorded
Phase 2	NCT03634488	Aug 2021 → Oct 2022	protein-energy malnutrition, sickle cell disease	Vanderbilt University Medical Center	Completed	No outcome recorded
Phase 2	NCT03806452	May 2019 → May 2024	sickle cell disease	Theravia	Completed	No outcome recorded
Phase 2	NCT03948867	Apr 2019 → Dec 2026 expected	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Enrolling by invitation	Met primary
Phase 2	NCT03644953	Oct 2018 → Jul 2021	sickle cell disease	Children's National Research Institute	Completed	No outcome recorded
Phase 2	NCT03183375	Aug 2017 → Apr 2018	beta thalassemia	Dr.Saqib Hussain Ansari	Unknown	No outcome recorded
Phase 2	NCT03107182	Jun 2017 → Sep 2024	head and neck squamous cell carcinoma, human papillomavirus-related squamous cell carcinoma	University of Chicago	Completed	No outcome recorded
Phase 2	NCT03020615	May 2017 → Jun 2020	sickle cell disease	St. Jude Children's Research Hospital	Completed	No outcome recorded
Phase 2	NCT02844673	Apr 2017 → Jan 2018	sickle cell disease	Muhimbili University of Health and Allied Sciences	Completed	No outcome recorded
Phase 2	NCT02788201	Mar 2017 → Oct 2019	urinary bladder cancer, urothelial carcinoma	National Cancer Institute (NCI)	Completed	No outcome recorded
Phase 2	NCT03062501	Nov 2016 → Apr 2017	sickle cell disease	Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti	Unknown	No outcome recorded
Phase 2	NCT02957760	Dec 2015 → Aug 2017	central retinal vein occlusion	Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts	Unknown	No outcome recorded
Phase 2	NCT02640573	Oct 2015 → Nov 2017	sickle cell-hemoglobin c disease syndrome	Baylor College of Medicine	Terminated	No outcome recorded Stop: Enrollment
Phase 2	NCT02336373	Dec 2014 → Feb 2017	sickle cell-hemoglobin c disease syndrome	Baylor College of Medicine	Terminated	No outcome recorded Stop: Enrollment
Phase 2	NCT02258659	Sep 2014 → Sep 2024	human papilloma virus infection, oropharynx squamous cell carcinoma	University of Chicago	Completed	Met primary
Phase 2	NCT02556099	Aug 2014 → Jan 2023	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Completed	No outcome recorded
Phase 2	NCT01389024	Aug 2012 → May 2022	sickle cell disease, stroke disorder	Johns Hopkins University	Completed	Missed primary
Phase 2	NCT01005576	Jan 2010 → Apr 2014	thalassemia	Washington University School of Medicine	Completed	No outcome recorded
Phase 2	NCT00978146	Oct 2009 → Jan 2015	desmoid tumor	Children's Hospital of Philadelphia	Withdrawn	No outcome recorded Stop: Enrollment
Phase 2	NCT01964755	Apr 2009 → Jun 2018	Epstein-Barr virus-associated lymphoproliferative disorder, post-transplant lymphoproliferative disease	University of Miami	Terminated	No outcome recorded
Phase 2	NCT00706810	Dec 2007 → Sep 2015	brain cancer, meningioma	University of Utah	Completed	No outcome recorded
Phase 2	NCT00809042	Jun → Nov 2007	beta-thalassemia intermedia	Shiraz University of Medical Sciences	Completed	No outcome recorded
Phase 2	NCT00462735	Feb 2007 → Oct 2010	carcinoma of pharynx, head and neck cancer, laryngeal carcinoma, nasal cavity neoplasm +2	Johnny Kao	Completed	No outcome recorded
Phase 2	NCT00468169	Jul 2006 → Aug 2012	head and neck cancer	University of Chicago	Completed	Missed primary
Phase 2	NCT00290771	Feb 2006 → Aug 2008	brain glioblastoma	Novartis	Terminated	No outcome recorded
Phase 2	NCT00615927	Feb 2006 → Apr 2009	gliosarcoma	Duke University	Completed	No outcome recorded
Phase 2	NCT00354913	May 2005 → Mar 2009	gliosarcoma	Duke University	Completed	No outcome recorded
Phase 2	NCT00402480	Apr 2003 → Mar 2006	stroke disorder	Duke University	Completed	No outcome recorded
Phase 2	NCT01693718	Mar → Sep 2003	head and neck cancer	University of Chicago	Completed	No outcome recorded
Phase 2	NCT00001958	Dec 1999 → Feb 2002	beta-thalassemia intermedia	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Completed	No outcome recorded
Phase 2	NCT00004897	Oct 1999 → Jul 2007	esophageal cancer	Northwestern University	Terminated	No outcome recorded Stop: Enrollment
Phase 2	NCT00004094	Aug 1999 → ?	head and neck cancer	National Cancer Institute (NCI)	Unknown	No outcome recorded
Phase 2	NCT00004089	Aug 1999 → Apr 2002	head and neck cancer	National Cancer Institute (NCI)	Completed	No outcome recorded
Phase 2	NCT00006119	Jul 1999 → Dec 2004	central nervous system neoplasm	UNICANCER	Completed	No outcome recorded
Phase 2	NCT00003590	Nov 1998 → Dec 2008	meningioma	SWOG Cancer Research Network	Completed	No outcome recorded
Phase 2	NCT00019474	Mar 1998 → Aug 2000	carcinoid tumor, extrahepatic bile duct carcinoma, gastric cancer, malignant pancreatic neoplasm +1	Montefiore Medical Center	Completed	No outcome recorded
Phase 2	NCT00003172	Dec 1997 → Sep 2004	gastric cancer	Eastern Cooperative Oncology Group	Completed	No outcome recorded
Phase 2	NCT00002940	Oct 1996 → Mar 2002	lymphoma	St. Jude Children's Research Hospital	Completed	No outcome recorded
Phase 2	NCT00002951	Jun 1996 → Jun 2004	esophageal cancer, head and neck cancer	University of Chicago	Completed	No outcome recorded
Phase 2	NCT00002674	Oct 1994 → Mar 2000	leukemia	Fred Hutchinson Cancer Center	Completed	No outcome recorded
Phase 2	NCT00000602	Apr 1994 → Mar 1997	sickle cell disease	National Heart, Lung, and Blood Institute (NHLBI)	Completed	No outcome recorded
Phase 2	NCT00001197	Feb 1984 → Dec 2003	sickle cell disease	National Heart, Lung, and Blood Institute (NHLBI)	Completed	No outcome recorded
Phase 2	NCT00000916	? → Aug 2004	HIV infectious disease	National Institute of Allergy and Infectious Diseases (NIAID)	Completed	No outcome recorded
Phase 2	NCT00008866	—	HIV infectious disease	National Institute of Allergy and Infectious Diseases (NIAID)	Withdrawn	No outcome recorded
Phase 2	NCT00002418	—	HIV infectious disease	Bristol-Myers Squibb	Terminated	No outcome recorded
Phase 2	NCT00000939	? → Jan 2000	HIV infectious disease	National Institute of Allergy and Infectious Diseases (NIAID)	Completed	No outcome recorded
Phase 2	NCT00006339	—	HIV infectious disease	National Institute of Allergy and Infectious Diseases (NIAID)	Withdrawn	No outcome recorded
Phase 2	NCT00002420	—	HIV infectious disease	Bristol-Myers Squibb	Terminated	No outcome recorded

Phase 2/38 trials · 1 met primary
Phase 2/3	NCT06153784	Jul 2020 → Jul 2023	beta thalassemia	Children's Hospital Karachi	Completed	No outcome recorded
Phase 2/3	NCT05132270	Jan → Dec 2020	beta thalassemia	Children's Hospital Karachi	Completed	Met primary
Phase 2/3	NCT04292314	Nov 2019 → Dec 2020	beta-thalassemia major, disorder of defective peroxisome oxidative status, disorder of iron metabolism and transport	Beni-Suef University	Completed	No outcome recorded
Phase 2/3	NCT03662126	Jan 2019 → Dec 2023	myelofibrosis with myeloid metaplasia	Kartos Therapeutics, Inc.	Unknown	No outcome recorded
Phase 2/3	NCT03793517	Sep 2018 → Oct 2023 overdue	acute myeloid leukemia, disease related to hematopoietic stem cell transplant	Peking University People's Hospital	Recruiting	No outcome recorded
Phase 2/3	NCT01624038	Jun → Dec 2012	beta-thalassemia intermedia	Ain Shams University	Unknown	No outcome recorded
Phase 2/3	NCT01103583	Jul 2011 → Dec 2013	multiple sclerosis	S. Andrea Hospital	Terminated	No outcome recorded
Phase 2/3	NCT00485511	Jun 2007 → Jun 2009	spinal muscular atrophy	Kaohsiung Medical University Chung-Ho Memorial Hospital	Completed	No outcome recorded
Phase 319 trials · 4 met primary · 3 missed
Phase 3	NCT07613450	May 2026 → Jul 2029 expected	meningioma	Shanghai Runshi Pharmaceutical Technology Co., Ltd	Not yet recruiting	No outcome recorded
Phase 3	NCT05285917	Nov 2023 → Sep 2026 overdue	sickle cell disease	Brown University	Recruiting	No outcome recorded
Phase 3	NCT04750707	Mar 2021 → Mar 2025	sickle cell disease	Global Health Uganda LTD	Completed	No outcome recorded
Phase 3	NCT03789591	Jan 2019 → Mar 2025	sickle cell disease	Lifespan	Completed	No outcome recorded
Phase 3	NCT03515018	May 2018 → May 2020	chronic myeloid leukemia	First Affiliated Hospital of Harbin Medical University	Unknown	No outcome recorded
Phase 3	NCT03128515	Jul 2017 → Apr 2019	malaria, sickle cell disease	Indiana University	Completed	Met primary
Phase 3	NCT02214407	Oct 2014 → Jul 2021	myelodysplastic syndrome	Groupe Francophone des Myelodysplasies	Completed	Missed primary
Phase 3	NCT01976416	Sep 2014 → Oct 2016	malaria, sickle cell disease	Indiana University	Completed	Missed primary
Phase 3	NCT01531387	May 2012 → Jan 2014	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Terminated	Missed primary Stop: Enrollment
Phase 3	NCT01425307	Aug 2011 → Mar 2015	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Terminated	Met primary
Phase 3	NCT01645124	May 2008 → Jun 2012	acquired polycythemia vera	Consorzio Mario Negri Sud	Terminated	Met primary Stop: Enrollment
Phase 3	NCT00154375	Oct 2004 → Aug 2008	astrocytoma (excluding glioblastoma), glioblastoma	Novartis Pharmaceuticals	Completed	No outcome recorded
Phase 3	NCT00055874	Jun 2002 → Mar 2012	leukemia	Heidelberg University	Completed	Met primary
Phase 3	NCT00005823	Dec 1998 → Dec 2007	leukemia, myelodysplastic syndrome, myelodysplastic/myeloproliferative disease	Leukemia Research Fund	Completed	No outcome recorded
Phase 3	NCT00025402	Jul 1997 → ?	leukemia	III. Medizinische Klinik Mannheim	Unknown	No outcome recorded
Phase 3	NCT00180973	Feb 1995 → ?	nasopharyngeal type undifferentiated carcinoma	Gustave Roussy, Cancer Campus, Grand Paris	Unknown	No outcome recorded
Phase 3	NCT00002771	Jan 1995 → ?	leukemia	German CML Study Group	Unknown	No outcome recorded
Phase 3	NCT00000586	Jan 1992 → Jun 1994	sickle cell disease	National Heart, Lung, and Blood Institute (NHLBI)	Completed	No outcome recorded
Phase 3	NCT00002230	—	HIV infectious disease	Northwestern University	Completed	No outcome recorded

Phase 48 trials
Phase 4	NCT07177300	Dec 2024 → Dec 2026 expected	sickle cell disease, sickle cell-beta zero-thalassemia	Children's Hospital Medical Center, Cincinnati	Recruiting	No outcome recorded
Phase 4	NCT06526117	Aug 2024 → Dec 2029 expected	sickle cell disease, stroke disorder	Vanderbilt University Medical Center	Recruiting	No outcome recorded
Phase 4	NCT06299670	Nov 2023 → Jan 2026	beta thalassemia	Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh	Completed	No outcome recorded
Phase 4	NCT05853458	Jul 2023 → Feb 2026	acquired polycythemia vera	Novartis Pharmaceuticals	Terminated	No outcome recorded Stop: Business
Phase 4	NCT02522104	Sep 2015 → Nov 2016	impaired renal function disease, sickle cell disease	Theravia	Completed	No outcome recorded
Phase 4	NCT02149537	Dec 2014 → Sep 2017	sickle cell disease	Loyola University	Completed	No outcome recorded
Phase 4	NCT01609192	Apr 2010 → Feb 2013	—	Assistance Publique - Hôpitaux de Paris	Completed	No outcome recorded
Phase 4	NCT00005018	Jul 1999 → Oct 2000	HIV infectious disease	Glaxo Wellcome	Completed	No outcome recorded
Phase not applicable13 trials · 1 met primary
—	NCT07673302	Jun 2026 → Jun 2027 expected	beta thalassemia	Riphah International University	Not yet recruiting	No outcome recorded
—	NCT05434000	Jan 2021 → Jul 2025	sickle cell disease, stroke disorder	Barau Dikko Teaching Hospital	Unknown	No outcome recorded
—	NCT05681598	Aug 2017 → May 2020	sickle cell disease	University of Kinshasa	Completed	No outcome recorded
—	NCT02769845	Mar 2016 → Jul 2019	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Completed	No outcome recorded
—	NCT02551718	Sep 2015 → Jun 2020	acute leukemia of ambiguous lineage, acute lymphoblastic leukemia	University of Washington	Completed	No outcome recorded
—	NCT02286154	Oct 2014 → Dec 2026 expected	sickle cell disease	Children's Hospital Medical Center, Cincinnati	Active not recruiting	Met primary
—	NCT01801423	Apr 2013 → Jan 2019	sickle cell disease, stroke disorder	Vanderbilt University Medical Center	Completed	No outcome recorded
—	NCT00344981	Jun 2003 → Nov 2006	AIDS	University of Maryland, Baltimore	Completed	No outcome recorded
—	NCT00519701	Apr 2002 → Feb 2007	sickle cell disease	Duke University	Completed	No outcome recorded
—	NCT00002177	May 1997 → Mar 2000	HIV infectious disease	Bristol-Myers Squibb	Completed	No outcome recorded
—	NCT00002176	May 1997 → Mar 2000	HIV infectious disease	Bristol-Myers Squibb	Completed	No outcome recorded
—	NCT00004404	Apr 1997 → Sep 2002	sickle cell disease	Boston Children's Hospital	Completed	No outcome recorded
—	NCT00000919	? → Nov 2002	HIV infectious disease	National Institute of Allergy and Infectious Diseases (NIAID)	Completed	No outcome recorded

Comparator or background therapy85 trials
Phase 0	NCT02042222 Comparator	Oct 2013 → Jul 2020	sickle cell disease	Baylor College of Medicine	Completed	No outcome recorded
Phase 1	NCT06871410 Background	Feb 2026 → Apr 2028 expected	acute myeloid leukemia	Roswell Park Cancer Institute	Recruiting	No outcome recorded
Phase 1	NCT05526924 Background	Mar 2023 → Jan 2028 expected	head and neck squamous cell carcinoma, pharynx cancer	University of Chicago	Recruiting	No outcome recorded
Phase 1	NCT02165007 Background	Jan 2015 → Jan 2024	sickle cell-beta-thalassemia disease syndrome, thalassemia	Catherine Bollard	Completed	No outcome recorded
Phase 1	NCT01848925 Comparator	May 2013 → Jul 2014	sickle cell disease	Prolong Pharmaceuticals	Completed	No outcome recorded
Phase 1	NCT01847326 Background	Mar 2013 → Jan 2024	hypopharynx squamous cell carcinoma, larynx verrucous carcinoma, nasal cavity and paranasal sinus squamous cell carcinoma, nasopharyngeal squamous cell carcinoma +4	University of Chicago	Completed	No outcome recorded
Phase 1	NCT00744692 Background	Oct 2008 → Dec 2012	bone marrow failure syndrome, immunodeficiency disease, lysosomal storage disease, sickle cell disease +1	Duke University	Completed	No outcome recorded
Phase 1	NCT00613054 Background	Nov 2007 → Mar 2009	gliosarcoma	Annick Desjardins	Completed	No outcome recorded
Phase 1	NCT00613132 Background	May 2005 → Aug 2008	gliosarcoma	Annick Desjardins	Completed	No outcome recorded
Phase 1	NCT00152113 Background	Apr 2005 → Dec 2008	sickle cell disease	St. Jude Children's Research Hospital	Completed	No outcome recorded
Phase 1	NCT00143572 Background	Nov 2004 → Jan 2008	sickle cell disease	St. Jude Children's Research Hospital	Completed	No outcome recorded
Phase 1	NCT00023959 Background	Jul 2001 → Mar 2010	adenoid cystic carcinoma, esthesioneuroblastoma, hypopharynx squamous cell carcinoma, intraoral basal cell carcinoma +11	National Cancer Institute (NCI)	Completed	No outcome recorded
Phase 1	NCT00019058 Background	Apr 1995 → ?	central nervous system neoplasm	National Cancer Institute (NCI)	Completed	No outcome recorded
Phase 1/2	NCT06199557 Comparator	May 2024 → Nov 2025 overdue	acute myeloid leukemia, myelodysplastic syndrome	Haukeland University Hospital	Recruiting	No outcome recorded
Phase 1/2	NCT05451940 Background	May 2023 → Dec 2024	sickle cell disease	Julia Xu	Completed	No outcome recorded
Phase 1/2	NCT05357482 Background	May 2022 → Jan 2026 overdue	beta thalassemia, sickle cell disease	National Heart, Lung, and Blood Institute (NHLBI)	Active not recruiting	No outcome recorded
Phase 1/2	NCT03653338 Background	Aug 2018 → Aug 2026 overdue	Diamond-Blackfan anemia, beta-thalassemia major, sickle cell disease	Paul Szabolcs	Recruiting	No outcome recorded
Phase 1/2	NCT03500731 Background	Apr 2018 → Dec 2027 expected	chronic obstructive pulmonary disease, idiopathic pulmonary fibrosis	Paul Szabolcs	Recruiting	No outcome recorded
Phase 1/2	NCT03077542 Background	Apr 2017 → Jul 2023 overdue	sickle cell disease	National Heart, Lung, and Blood Institute (NHLBI)	Active not recruiting	No outcome recorded
Phase 1/2	NCT03128996 Background	Mar 2017 → Apr 2028 expected	bone marrow failure syndrome, metabolic disease, sickle cell disease	Washington University School of Medicine	Recruiting	No outcome recorded
Phase 1/2	NCT01711541 Background	Oct 2012 → Feb 2018	head and neck squamous cell carcinoma, oropharyngeal carcinoma	National Cancer Institute (NCI)	Completed	No outcome recorded
Phase 1/2	NCT00920972 Background	Dec 2001 → Dec 2026 expected	inherited hemoglobinopathy, metabolic disease	Washington University School of Medicine	Recruiting	No outcome recorded
Phase 2	NCT06930352 Background	Sep 2026 → Dec 2027 expected	acute myeloid leukemia	Uma Borate	Not yet recruiting	No outcome recorded
Phase 2	NCT07616154 Background	Sep 2026 → Sep 2034 expected	sickle cell disease	St. Jude Children's Research Hospital	Not yet recruiting	No outcome recorded
Phase 2	NCT07507760 Background	Jun 2026 → Apr 2029 expected	acute myeloid leukemia	PETHEMA Foundation	Recruiting	No outcome recorded
Phase 2	NCT07033598 Comparator	Jan 2026 → Jul 2028 expected	chronic myelomonocytic leukemia	Theradex	Recruiting	No outcome recorded
Phase 2	NCT06326190 Comparator	Mar 2025 → Apr 2027 expected	meningioma	European Organisation for Research and Treatment of Cancer - EORTC	Recruiting	No outcome recorded
Phase 2	NCT05953584 Background	Jun 2023 → Aug 2027 expected	sickle cell disease	Forma Therapeutics, Inc.	Recruiting	No outcome recorded
Phase 2	NCT05405114 Comparator	Jul 2022 → Jul 2025	sickle cell disease	Novo Nordisk A/S	Completed	No outcome recorded
Phase 2	NCT05020652 Comparator	Nov 2021 → Oct 2023	myelofibrosis	Chia Tai Tianqing Pharmaceutical Group Co., Ltd.	Completed	No outcome recorded
Phase 2	NCT05062278 Comparator	Jul 2021 → Jul 2025	acute myeloid leukemia	Hospital Universitario Dr. Jose E. Gonzalez	Unknown	No outcome recorded
Phase 2	NCT04687176 Background	Jan 2021 → Dec 2025 overdue	acute promyelocytic leukemia	The University of Hong Kong	Recruiting	No outcome recorded
Phase 2	NCT04362293 Background	Apr 2020 → Jun 2026 overdue	sickle cell disease	St. Jude Children's Research Hospital	Active not recruiting	No outcome recorded
Phase 2	NCT04126681 Comparator	Oct 2019 → Aug 2024	chronic myeloid leukemia	Ascentage Pharma Group Inc.	Completed	Met primary
Phase 2	NCT03944915 Background	Aug 2019 → Nov 2023	head and neck squamous cell carcinoma, human papilloma virus infection, human papillomavirus-related squamous cell carcinoma	University of Chicago	Completed	Met primary
Phase 2	NCT03860844 Background	Aug 2019 → Sep 2022	acute lymphoblastic leukemia, acute myeloid leukemia	Sanofi	Terminated	No outcome recorded Stop: Business
Phase 2	NCT02757885 Background	Jul 2019 → Dec 2021	sickle cell disease	Emory University	Completed	No outcome recorded
Phase 2	NCT03825341 Comparator	Jun 2019 → Jan 2022	sickle cell disease, thalassemia	St. Jude Children's Research Hospital	Terminated	No outcome recorded Stop: Enrollment
Phase 2	NCT03263559 Background	Oct 2017 → Jan 2025	sickle cell disease	Medical College of Wisconsin	Completed	No outcome recorded
Phase 2	NCT02626715 Comparator	Sep 2015 → Apr 2023	acute myeloid leukemia, disease related to hematopoietic stem cell transplant, myelodysplastic syndrome	Randy Windreich	Completed	No outcome recorded
Phase 2	NCT02088541 Comparator	Mar 2014 → Jan 2018	acute myeloid leukemia	Karyopharm Therapeutics Inc	Completed	Missed primary
Phase 2	NCT01962415 Background	Feb 2014 → Nov 2026 expected	bone marrow failure syndrome, hereditary anemia, immunodeficiency disease, inborn errors of metabolism +1	Paul Szabolcs	Recruiting	No outcome recorded
Phase 2	NCT02090296 Comparator	Feb 2014 → Jun 2015	sickle cell disease	Children's National Research Institute	Withdrawn	No outcome recorded
Phase 2	NCT01960413 Comparator	Nov 2013 → Mar 2018	sickle cell disease	Vanderbilt University Medical Center	Completed	Missed primary
Phase 2	NCT01461837 Background	Jan 2012 → Dec 2025 overdue	sickle cell disease	New York Medical College	Active not recruiting	No outcome recorded
Phase 2	NCT01133678 Background	May 2010 → Jan 2015	head and neck squamous cell carcinoma	University of Chicago	Terminated	Missed primary
Phase 2	NCT00904735 Background	Jun 2009 → ?	central nervous system neoplasm	San Raffaele Scientific Institute	Unknown	No outcome recorded
Phase 2	NCT00928707 Background	Jun 2009 → Jul 2011	acquired polycythemia vera	Italfarmaco	Completed	No outcome recorded
Phase 2	NCT00532883 Comparator	Jan 2007 → Mar 2009	sickle cell-hemoglobin c disease syndrome	St. Jude Children's Research Hospital	Terminated	No outcome recorded Stop: Enrollment
Phase 2	NCT00383448 Background	Sep 2006 → Sep 2014	GM1 gangliosidosis, Krabbe disease, Sandhoff disease, Tay-Sachs disease +5	Masonic Cancer Center, University of Minnesota	Completed	No outcome recorded
Phase 2	NCT00083187 Background	Nov 2005 → Jan 2007	leukemia, myelodysplastic syndrome, myelodysplastic/myeloproliferative neoplasm	Vion Pharmaceuticals	Completed	No outcome recorded
Phase 2	NCT00203905 Background	Jan 2004 → Nov 2007	head and neck cancer	University of Chicago	Completed	No outcome recorded
Phase 2	NCT00002412 Background	—	HIV infectious disease	Triangle Pharmaceuticals	Completed	No outcome recorded
Phase 2/3	NCT04301336 Background	Nov 2019 → Nov 2020	sickle cell disease	Beni-Suef University	Completed	No outcome recorded
Phase 2/3	NCT03799224 Background	Dec 2018 → Dec 2021	acute leukemia, disease related to hematopoietic stem cell transplant	Peking University People's Hospital	Unknown	No outcome recorded
Phase 2/3	NCT03651102 Background	Jan 2018 → Nov 2021	thalassemia	Blood Diseases Clinic	Completed	No outcome recorded
Phase 3	NCT06456346 Comparator	Jul 2024 → Sep 2027 expected	essential thrombocythemia	Merck Sharp & Dohme LLC	Active not recruiting	No outcome recorded
Phase 3	NCT06093672 Comparator	Mar 2024 → Jul 2026 overdue	acquired polycythemia vera	Italfarmaco	Recruiting	No outcome recorded
Phase 3	NCT04576156 Comparator	Apr 2021 → Jun 2028 expected	myelofibrosis	Geron Corporation	Active not recruiting	No outcome recorded
Phase 3	NCT04617028 Comparator	Feb 2021 → Oct 2023	myelofibrosis	Suzhou Zelgen Biopharmaceuticals Co.,Ltd	Completed	Met primary
Phase 3	NCT04116502 Comparator	Oct 2019 → Oct 2028 expected	acquired polycythemia vera	University of Birmingham	Recruiting	No outcome recorded
Phase 3	NCT03165734 Comparator	Jun 2017 → Oct 2026 expected	myelofibrosis, primary myelofibrosis	Swedish Orphan Biovitrum	Active not recruiting	No outcome recorded
Phase 3	NCT02675790 Comparator	Jan 2017 → Mar 2020	sickle cell disease, stroke disorder	Vanderbilt University Medical Center	Completed	No outcome recorded
Phase 3	NCT02899169 Background	Sep 2016 → Sep 2022	acute promyelocytic leukemia	First Affiliated Hospital Xi'an Jiaotong University	Unknown	Met primary
Phase 3	NCT02560935 Comparator	Jul 2016 → Mar 2020	sickle cell disease, stroke disorder	Vanderbilt University Medical Center	Completed	No outcome recorded
Phase 3	NCT02611973 Background	Mar 2016 → Nov 2019	essential thrombocythemia	Assistance Publique - Hôpitaux de Paris	Unknown	No outcome recorded
Phase 3	NCT02038036 Background	Mar 2014 → Sep 2015	acquired polycythemia vera	Novartis Pharmaceuticals	Completed	Met primary
Phase 3	NCT01949805 Comparator	Sep 2013 → Jul 2016	acquired polycythemia vera	AOP Orphan Pharmaceuticals AG	Completed	No outcome recorded
Phase 3	NCT01632904 Comparator	Jun 2012 → Mar 2014	acquired polycythemia vera	Incyte Corporation	Completed	Missed primary
Phase 3	NCT01387763 Comparator	Jan 2012 → Jun 2020	acquired polycythemia vera, essential thrombocythemia, primary myelofibrosis	Thomas Stauffer Larsen	Completed	No outcome recorded
Phase 3	NCT01259856 Comparator	Sep 2011 → Jun 2017	acquired polycythemia vera, essential thrombocythemia	Ronald Hoffman	Completed	Missed primary
Phase 3	NCT00122980 Comparator	Oct 2006 → Jun 2010	hereditary hemochromatosis, sickle cell disease, stroke disorder	St. Jude Children's Research Hospital	Terminated	No outcome recorded
Phase 3	NCT00117572 Comparator	Nov 2004 → Dec 2016	larynx cancer, nasal cavity cancer, oral cavity cancer, paranasal sinus neoplasm +1	University of Chicago	Completed	No outcome recorded
Phase 3	NCT01065038 Comparator	Sep 2002 → ?	essential thrombocythemia	AOP Orphan Pharmaceuticals AG	Completed	Met primary
Phase 3	NCT00006400 Comparator	Aug 2000 → Sep 2009	sickle cell disease	National Heart, Lung, and Blood Institute (NHLBI)	Completed	No outcome recorded
Phase 3	NCT00004933 Comparator	Jan 2000 → May 2001	leukemia	Alliance for Clinical Trials in Oncology	Terminated	No outcome recorded Stop: Enrollment
Phase 3	NCT00002868 Background	Nov 1997 → Mar 2004	leukemia	Eastern Cooperative Oncology Group	Completed	No outcome recorded
Phase 3	NCT00002869 Background	Apr 1995 → ?	leukemia	Medical Research Council	Unknown	No outcome recorded
Phase 4	NCT07488520 Background	Jul 2026 → Dec 2029 expected	sickle cell disease	Swiss Tropical & Public Health Institute	Recruiting	No outcome recorded
Phase 4	NCT06606886 Comparator	Sep 2024 → Feb 2026	beta-thalassemia major	Bahria University	Completed	No outcome recorded
Phase 4	NCT05081349 Comparator	Jan 2017 → Jun 2020	sickle cell disease	Assiut University	Completed	No outcome recorded
Phase 4	NCT00202644 Comparator	Jan 2006 → Dec 2015	blood platelet disease	Shire	Completed	No outcome recorded
—	NCT03293641 Background	May 2016 → Jan 2017	disorder of zinc metabolism, infectious disease, sickle cell disease	Lucas Otieno Tina, MD MSc	Completed	No outcome recorded
—	NCT00336362 Comparator	Jul 2006 → Aug 2010	beta-thalassemia major	University of Washington	Completed	No outcome recorded
—	NCT00175838 Comparator	Jul 1997 → Nov 2016	blood platelet disease, myeloproliferative neoplasm	University of Cambridge	Completed	No outcome recorded

News releases announcing trial results or a regulatory action · 1

Date	Issuer	Release
2020-06-18	Novartis Pharmaceuticals	Regulatory Novartis expands Africa Sickle Cell Disease program to Uganda and Tanzania novartis.com ↗ At the same time, Novartis has registered the medicine for the treatment of SCD in Uganda, Tanzania and Kenya.

All press releases naming this drug 1 release

Date	Issuer	Release
2020-06-18	Novartis Pharmaceuticals	Novartis expands Africa Sickle Cell Disease program to Uganda and Tanzania novartis.com ↗

Evidence & citations 15 cited values

Every value below carries the sentence it was read from. 218 sources stand behind the page.

Field	Value	Cited text
Known as	Hydroxy Urea	“Twenty-two children with a median age of 2.8 years, many with severe comorbidities and prior viral infections, were enrolled in a novel RIC protocol consisting of hydroxyurea,...” PMID 24296492 ↗ Dec 2013 203 “In total, 136 patients will be randomized in a 2:1 ratio to [177Lu]Lu-DOTATATE (≤4 doses of 7.4 GBq given every 4 wk) or local standard of care (hydroxyurea, bevacizumab,...” PMID 40774695 ↗ Jan 2026 “Hydroxyurea and azathioprine were administered for a 3 month period prior to the initiation of the transplant procedure in an effort to help prevent rejection of the donor product.” NCT00152113 ↗ “standard administration of antibacterial and antimalarial prophylaxis, vaccinations for pneumococcal and Haemophilus influenzae type b (Hib), hydroxyurea, parental education” NCT07488520 ↗ “which may include but is not limited to hydroxyurea, thalidomide or an analog of thalidomide, interferon, danazol, hypomethylating agents, chemotherapy or radiotherapy.” NCT04576156 ↗ “Best Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation.” NCT02038036 ↗ “In patients not receiving daunorubicin, hydroxyurea if WBC ≥ 5 x 10\^9/L within the first 14 days of induction.” NCT04687176 ↗ “Chemotherapy with paclitaxel, fluorouracil (5FU), and hydroxyurea given in combination with radiation therapy.” NCT06005324 ↗ “Hydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11” NCT01461837 ↗ “Best available therapy options include: 1. hydroxyurea 2. chemotherapy or” NCT03662126 ↗ “The preparatory regimen will consist of Hydroxyurea from Days -50 to -22” NCT02165007 ↗ Show 193 more sources
Known as	Droxia	“Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.” NCT03825341 ↗
Known as	Hidroxicarbamida	ChEMBL registry synonym — accepted as the source's own label CHEMBL467 ↗
Known as	Hydrea	“Hydrea® (hydroxyurea )” NCT01609192 ↗ 1 NCT01387763 ↗
Known as	Hydroxycarbamid	ClinicalTrials.gov intervention name — accepted as the source's own label NCT06578507 ↗ 1 NCT02957760 ↗
Known as	Hydroxycarbamide	“Hypothesis : Hydroxycarbamide (HC) by reducing the erythrocyte adhesion to the endothelium may prevent or delay the onset of retinal capillary non-perfusion in patients with CRVO.” NCT02957760 ↗ 15 “Hydroxyurea is 500mg hydroxycarbamide capsules; initial dose is 1000mg/day, administered in two divided doses (500mg/dose). Dose titrated to effect to achieve a response.” NCT00202644 ↗ “Hydroxyurea (HU/hydroxycarbamide) is a hydroxylated analogue of urea which prevents DNA synthesis by inhibiting the activity of ribonucleotide reductase (RNR).” NCT06199557 ↗ “Hydroxyurea (HU, or hydroxycarbamide) is the only drug approved to date by the American FDA for use in adults with sickle cell disease.” NCT03062501 ↗ “an open-label non-randomised trial of hydroxyurea (hydroxycarbamide) in children with sickle cell anaemia in sub-Saharan Africa.” PMID 38701812 ↗ Apr 2024 “Hydroxyurea (hydroxycarbamide) is an effective treatment for sickle cell anaemia” PMID 34227124 ↗ Jul 2021 “Hydroxyurea or hydroxycarbamide (HU) lies in the category of antimetabolite.” NCT06490601 ↗ “hydroxycarbamide (80 mg·kg-1) orally on day -10” NCT03793517 ↗ “hydroxycarbamide (80 mg·kg-1) orally on day -10” NCT03799224 ↗ NCT03806452 ↗ NCT05020652 ↗ Show 5 more sources
Known as	Hydroxyurea-Droxia-Hydrea-Hydroxycarbamide	“Hydroxyurea-Droxia-Hydrea-Hydroxycarbamide” NCT00613132 ↗
Known as	NSC-32065	ChEMBL registry synonym — accepted as the source's own label CHEMBL467 ↗
Known as	ONCOCARBIDE (HYDROXYUREA)	“ONCOCARBIDE (HYDROXYUREA)” NCT00928707 ↗
Known as	Siklos	ClinicalTrials.gov intervention name — accepted as the source's own label NCT02522104 ↗
Known as	SQ 1089	ChEMBL registry synonym — accepted as the source's own label CHEMBL467 ↗
Known as	Xromi	ChEMBL registry synonym — accepted as the source's own label CHEMBL467 ↗
Action	Inhibit	“blocks DNA synthesis by inhibiting ribonucleotide reductase” NCT00001197 ↗
Modality	Other / unclassified	“FDA approved anti-metabolite, hydroxyurea” NCT03634488 ↗
Route	Oral	“orally administered hydroxyurea” NCT00000586 ↗