Drugs / Deferiprone
Deferiprone
also known as
DFP · Ferriprox · 1,2 dimethyl-3-hydroxy-pyrid-4-one · CP20 · CRMD001 · 1,2-dimethyl-3-hydroxypyridin-4-one · deferiprone 50 mg/kg/day brand name GPO-L-ONE · DFX · +1 more
Small molecule targets DOHH via inhibition
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansions (3) | US (FDA) | FERRIPROX | — | 2018-04-20 – 2021-04-30 | fda.gov ↗ |
| Approved | US (FDA) | FERRIPROX | — | 2011-10-14 | fda.gov ↗ |
| Approved | EU (EMA) | Ferriprox | — | 1999-08-25 | europa.eu ↗ |
Trials 42
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT04184453 | Dec 2019 → Oct 2022 | aceruloplasminemia | First Affiliated Hospital of Fujian Medical University | Unknown | No outcome recorded |
| Phase 16 trials | ||||||
| Phase 1 | NCT02465489 | Jun → Aug 2015 | — | ApoPharma | Completed | No outcome recorded |
| Phase 1 | NCT02456558 | Jun 2015 → Mar 2016 | — | ApoPharma | Completed | No outcome recorded |
| Phase 1 | NCT02442310 | May → Jul 2015 | — | ApoPharma | Completed | No outcome recorded |
| Phase 1 | NCT02189941 | May → Jun 2014 | — | ApoPharma | Completed | No outcome recorded |
| Phase 1 | NCT01989455 | Nov 2013 → Mar 2014 | — | ApoPharma | Completed | No outcome recorded |
| Phase 1 | NCT02191657 | Nov 2006 → May 2008 | HIV infectious disease | ApoPharma | Completed | No outcome recorded |
| Phase 1/23 trials | ||||||
| Phase 1/2 | NCT03754725 | Oct 2020 → Jun 2026 overdue | acquired aneurysmal subarachnoid hemorrhage, dementia | Duke University | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT01709032 | Sep 2012 → Aug 2015 | beta-thalassemia major | Children's Hospital of Philadelphia | Completed | No outcome recorded |
| Phase 1/2 | NCT00530127 | Apr 2008 → Jul 2009 | Friedreich ataxia | ApoPharma | Completed | No outcome recorded |
| Phase 213 trials · 3 missed | ||||||
| Phase 2 | NCT07023666 | Oct 2025 → Jun 2027 expected | sickle cell disease | Inova Health Care Services | Recruiting | No outcome recorded |
| Phase 2 | NCT05111821 | Jun 2022 → Apr 2025 | stroke disorder | University Hospital, Bordeaux | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT03802916 | Mar → Dec 2019 | hemosiderosis | ApoPharma | Completed | No outcome recorded |
| Phase 2 | NCT03234686 | Jan 2018 → Jan 2023 | Alzheimer disease, cognitive impairment with or without cerebellar ataxia | Neuroscience Trials Australia | Completed | Missed primary |
| Phase 2 | NCT02728843 | Oct 2016 → Aug 2019 | Parkinson disease | ApoPharma | Completed | Missed primary |
| Phase 2 | NCT02477631 | Feb 2016 → May 2017 | hemosiderosis, myelodysplastic syndrome | Sheba Medical Center | Completed | No outcome recorded |
| Phase 2 | NCT02164253 | Sep 2013 → Dec 2016 | amyotrophic lateral sclerosis, disorder of iron metabolism and transport | University Hospital, Lille | Completed | No outcome recorded |
| Phase 2 | NCT01740713 | Dec 2012 → Dec 2013 | disorder of iron metabolism and transport | Consorzio per Valutazioni Biologiche e Farmacologiche | Completed | No outcome recorded |
| Phase 2 | NCT01146925 | Jun 2010 → Jun 2011 | acute kidney injury | CorMedix | Completed | No outcome recorded |
| Phase 2 | NCT00897221 | Jun 2009 → Mar 2011 | Friedreich ataxia | ApoPharma | Completed | No outcome recorded |
| Phase 2 | NCT00907283 | Nov 2008 → Dec 2024 | disorder of iron metabolism and transport, neurodegenerative disease | Ente Ospedaliero Ospedali Galliera | Unknown | No outcome recorded |
| Phase 2 | NCT00115349 | Jun 2005 → Jul 2008 | beta thalassemia, heart disorder | Carelon Research | Terminated | Missed primary Stop: Enrollment |
| Phase 2 | NCT00349453 | Mar 2005 → May 2011 | disorder of iron metabolism and transport | Lipomed | Completed | No outcome recorded |
| Phase 2/32 trials | ||||||
| Phase 2/3 | NCT03293069 | Jan 2019 → Nov 2023 | amyotrophic lateral sclerosis | University Hospital, Lille | Completed | No outcome recorded |
| Phase 2/3 | NCT02882477 | Dec 2016 → May 2018 | diabetes mellitus, iron metabolism disease, optic atrophy, peptic ulcer disease +2 | Hadassah Medical Organization | Unknown | No outcome recorded |
| Phase 36 trials · 1 met primary · 1 missed | ||||||
| Phase 3 | NCT02174848 | Jun 2014 → Mar 2018 | pantothenate kinase-associated neurodegeneration | ApoPharma | Completed | No outcome recorded |
| Phase 3 | NCT01825512 | Mar 2014 → Sep 2017 | disorder of iron metabolism and transport | Consorzio per Valutazioni Biologiche e Farmacologiche | Completed | Met primary |
| Phase 3 | NCT01741532 | Dec 2012 → Oct 2016 | pantothenate kinase-associated neurodegeneration | ApoPharma | Completed | Missed primary |
| Phase 3 | NCT01391520 | Jan 2012 → Mar 2013 | acute kidney injury | CorMedix | Withdrawn | No outcome recorded Stop: Business |
| Phase 3 | NCT00529152 | Aug 2007 → Jul 2008 | disorder of iron metabolism and transport | ApoPharma | Completed | No outcome recorded |
| Phase 3 | NCT00350662 | Jan 2002 → ? | disorder of iron metabolism and transport | Lipomed | Completed | No outcome recorded |
| Phase 48 trials · 2 met primary | ||||||
| Phase 4 | NCT03591575 | Nov 2018 → Sep 2020 | beta-thalassemia major, disorder of iron metabolism and transport | Chiesi Canada Corp | Completed | No outcome recorded |
| Phase 4 | NCT02443545 | May 2015 → Apr 2019 | anemia, disorder of iron metabolism and transport, sickle cell disease | ApoPharma | Terminated | No outcome recorded |
| Phase 4 | NCT02041299 | Apr 2014 → Apr 2019 | anemia, disorder of iron metabolism and transport, sickle cell disease | ApoPharma | Terminated | Met primary Stop: Enrollment |
| Phase 4 | NCT01770652 | Jan → Jul 2013 | impaired renal function disease | ApoPharma | Completed | No outcome recorded |
| Phase 4 | NCT01860703 | Nov → Dec 2012 | — | ApoPharma | Completed | No outcome recorded |
| Phase 4 | NCT00103753 | May 2004 → Jun 2005 | beta thalassemia | Royal Brompton & Harefield NHS Foundation Trust | Unknown | No outcome recorded |
| Phase 4 | NCT00105495 | Dec 2002 → Oct 2004 | beta-thalassemia major, hemosiderosis | ApoPharma | Completed | No outcome recorded |
| Phase 4 | NCT00733811 | Sep 2000 → Jan 2008 | beta-thalassemia major | Azienda Ospedaliera V. Cervello | Completed | Met primary |
| Phase not applicable1 trial | ||||||
| — | NCT02198508 | Jul 2007 → Jul 2008 | beta-thalassemia major | China Medical University Hospital | Completed | No outcome recorded |
| Phase not stated2 trials | ||||||
| — | NCT00293098 | Mar 2006 → ? | disorder of iron metabolism and transport | Children's Hospital of Philadelphia | Approved for marketing | No outcome recorded |
| — | NCT02635841 | — | pantothenate kinase-associated neurodegeneration | Chiesi Canada Corp | No longer available | No outcome recorded |
| Comparator or background therapy13 trials | ||||||
| Phase 0 | NCT02878538 Comparator | Jan 2018 → Apr 2022 | cognitive impairment with or without cerebellar ataxia | The University of Texas Health Science Center at San Antonio | Withdrawn | No outcome recorded |
| Phase 2 | NCT07759895 Comparator | Aug 2026 → Dec 2028 expected | schizoaffective disorder, schizophrenia | Amit Lotan | Recruiting | No outcome recorded |
| Phase 2 | NCT07292259 Background | Jan 2024 → Dec 2025 | beta-thalassemia major | Pakistan Blood and Marrow Transplant (PBMT) Group | Completed | No outcome recorded |
| Phase 2 | NCT05604131 Comparator | Nov 2022 → Mar 2029 expected | acute myocardial infarction | Rohan Dharmakumar | Recruiting | No outcome recorded |
| Phase 2 | NCT02655315 Comparator | Feb 2016 → Sep 2020 | Parkinson disease | University Hospital, Lille | Completed | No outcome recorded |
| Phase 2 | NCT01539837 Comparator | Feb 2012 → Sep 2014 | Parkinson disease | Imperial College London | Completed | No outcome recorded |
| Phase 2/3 | NCT02173951 Comparator | Jul 2014 → Jul 2015 | beta-thalassemia major | Ain Shams University | Unknown | No outcome recorded |
| Phase 2/3 | NCT02065492 Background | Feb 2014 → Oct 2015 | thalassemia | Aga Khan University | Completed | No outcome recorded |
| Phase 2/3 | NCT01511848 Comparator | Feb 2012 → Feb 2013 | beta-thalassemia major, hemosiderosis, sickle cell disease | Ain Shams University | Unknown | No outcome recorded |
| Phase 2/3 | NCT00943748 Comparator | Oct 2009 → Aug 2011 | Parkinson disease | University Hospital, Lille | Completed | No outcome recorded |
| Phase 4 | NCT00800761 Comparator | Dec 2001 → Jun 2006 | cardiomyopathy, disorder of iron metabolism and transport | Ospedale Microcitemico | Completed | No outcome recorded |
| — | NCT02880033 Comparator | Feb 2011 → Oct 2019 | Parkinson disease, amyotrophic lateral sclerosis, disorder of iron metabolism and transport | University Hospital, Lille | Completed | No outcome recorded |
| — | NCT01597765 Background | Jun 2009 → Jun 2011 | thalassemia | Mahidol University | Completed | No outcome recorded |
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 60 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Deferiprone | “Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and...” PMID 25600572 ↗ Mar 201554“This will comprise of standard chelation drugs (Deferasirox or Deferoxamine or Combination of Deferoxamine and Deferiprone).” NCT02065492 ↗ “Patients will also continue the iron chelation therapy (Deferasirox, Deferiprone or Deferoxamine) in case of iron overload.” NCT07292259 ↗ |
| Known as | 1,2 dimethyl-3-hydroxy-pyrid-4-one | “Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxy-pyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987.” NCT00350662 ↗1“Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxypyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987.” NCT00349453 ↗ |
| Known as | 1,2-dimethyl-3-hydroxypyridin-4-one | “We recently demonstrated (for the first time) the feasibility, efficacy and acceptability of the conservative iron chelation approach in pilot translational studies in...” NCT02655315 ↗ |
| Known as | CP20 | “Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxy-pyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987.” NCT00350662 ↗1“Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxypyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987.” NCT00349453 ↗ |
| Known as | CRMD001 | “This trial will evaluate whether treatment with CRMD-001 (unique formulations of the iron chelator, Deferiprone) will reduce the incidence of AKI in subjects with CKD and...” NCT01146925 ↗1“This trial will evaluate whether treatment with CRMD001 (unique formulations of the iron chelator, Deferiprone) will reduce morbidity and mortality” NCT01391520 ↗ |
| Known as | deferiprone 50 mg/kg/day brand name GPO-L-ONE | “deferiprone 50 mg/kg/day brand name GPO-L-ONE” NCT01597765 ↗ |
| Known as | DFP | “Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and...” PMID 25600572 ↗ Mar 20156“Half of participants will receive the deferiprone (DFP) to 15 mg / kg twice daily morning and evening (30mg / kg per day).The treatment lasts nine months.” NCT02655315 ↗ “we assessed the effect of oxidative insults in mice systemically prechelated with deferiprone (DFP) by following motor functions” PMID 24251381 ↗ Feb 2014 “oral deferiprone (DFP) with those of combinations of parenteral desferrioxamine (DFO) with oral DFP.” PMID 18055982 ↗ Dec 2007 “starting dose of Deferiprone (DFP) 50mg⁄ kg ⁄ d, administered orally 3 times daily.” NCT02173951 ↗ “Deferiprone (DFP)” NCT07759895 ↗ “DFP(Deferiprone)” NCT02198508 ↗ |
| Known as | DFX | “Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and...” PMID 25600572 ↗ Mar 2015 |
| Known as | Ferriprox | “Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg in 3 divided doses (better tolerated if started and then built up over 4 weeks).” NCT01511848 ↗4“Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients” NCT00529152 ↗ “who are currently taking deferiprone immediate-release tablets (Ferriprox) three times a day” NCT03802916 ↗ “Ferriprox (deferiprone) 500 mg immediate release tablet formulation” NCT02465489 ↗ “Ferriprox (deferiprone)” NCT00105495 ↗ |
| Known as | Ferrirpox | “long-term chelation through Deferiprone (Ferrirpox®, Chiesi) administered daily from 3-to-5 days following stroke to 6 months” NCT05111821 ↗ |
| Action | Inhibit | “deferiprone produced infection-independent deficiency of hydroxylated hypusyl-eIF5A.” PMID 27191165 ↗ May 2016 |
| Modality | Small molecule | “Deferiprone presents chemical-physical characteristics (low molecular weight, favourable octanol:water partition coefficient, neutral charge)” NCT00907283 ↗ |
| Route | Oral | “Fifteen of these patients received combination therapy with subcutaneous deferoxamine and oral deferiprone with CMR follow-up.” PMID 18298856 ↗ Feb 2008 |
| Target | DOHH | “The uniform deferiprone threshold is in agreement with mapping of, and crystallographic 3D-data on, the active site of deoxyhypusyl hydroxylase (DOHH), the eIF5A-hydroxylating enzyme.” PMID 27191165 ↗ May 2016 |