Hydroxyurea
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
Also used as a comparator or background therapy in 85 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT02042222 Comparator | Oct 2013 → Jul 2020 | sickle cell disease | Baylor College of Medicine | Completed | No outcome recorded |
| Phase 112 trials | ||||||
| Phase 1 | NCT06871410 Background | Mar 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 1/29 trials | ||||||
| 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 231 trials | ||||||
| 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 | Apr 2026 → Apr 2029 expected | acute myeloid leukemia | PETHEMA Foundation | Not yet 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 → Sep 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 → Aug 2026 | sickle cell disease | St. Jude Children's Research Hospital | Suspended | No outcome recorded |
| Phase 2 | NCT04126681 Comparator | Oct 2019 → Aug 2024 | chronic myeloid leukemia | Ascentage Pharma Group Inc. | Completed | No outcome recorded |
| 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 | No outcome recorded |
| Phase 2 | NCT03860844 Background | Aug 2019 → Sep 2022 | acute lymphoblastic leukemia, acute myeloid leukemia | Sanofi | Terminated | No outcome recorded |
| 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 |
| 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 | No outcome recorded |
| 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 | No outcome recorded |
| 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 | No outcome recorded |
| Phase 2 | NCT00928707 Background | Jun 2009 → Jul 2011 | acquired polycythemia vera | Italfarmaco | Completed | No outcome recorded |
| Phase 2 | NCT00904735 Background | Jun 2009 → ? | central nervous system neoplasm | San Raffaele Scientific Institute | Unknown | 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 |
| 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 2/33 trials | ||||||
| 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 322 trials | ||||||
| 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 | No outcome recorded |
| 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 | No outcome recorded |
| 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 | No outcome recorded |
| 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 | No outcome recorded |
| 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 | No outcome recorded |
| 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 | No outcome recorded |
| 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 |
| 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 44 trials | ||||||
| Phase 4 | NCT07488520 Background | Apr 2026 → Dec 2029 expected | sickle cell disease | Swiss Tropical & Public Health Institute | Not yet 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 |
| Phase not applicable3 trials | ||||||
| — | 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 |
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 2013203“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 ↗ “Investigator's Choice Treatment:bevacizumab, temozolomide or hydroxyurea” NCT07613450 ↗ “Folic Acid, Proguanil, Penicillin V, Hydroxyurea over 6 months” NCT03293641 ↗ “corticosteroids, hydroxyurea, danazol, or low-dose ruxolitinib” NCT03165734 ↗ “Droxia (hydroxyurea) - drug class: antimetabolite” NCT02626715 ↗ “ordinary treatment of Hydroxyurea (20 mg/kg/day)” NCT04301336 ↗ “Day -50 to -21: Hydroxyurea 30mg/kg PO q day” NCT00920972 ↗ “Days -50 to -21: Hydroxyurea 30mg/kg po” NCT01005576 ↗ “Gemcitabine, Hydroxyurea, Idarubicin” NCT02788201 ↗ “Gemcitabine HCl Hydroxyurea Imatinib” NCT02551718 ↗ “hydroxyurea 500 mg BID PO (day 1-5)” NCT01133678 ↗ “Patients will get only Hydroxyurea” NCT06299670 ↗ “hydroxyurea (30mg/kg/day po)” NCT03128996 ↗ “Drug: Hydroxy Urea” NCT06299670 ↗ “Drug: Hydroxy Urea” NCT04301336 ↗ |
| 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 ↗ |
| Known as | Hydroxycarbamid | ClinicalTrials.gov intervention name — accepted as the source's own label 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” NCT03799224 ↗ “hydroxycarbamide (80 mg·kg-1) orally on day -10” NCT03793517 ↗ |
| 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 ↗ |