Clinical Trials

Deferiprone is currently being evaluated across numerous clinical trials spanning Early Phase 1 through Phase 4, focusing on conditions characterized by systemic or focal iron accumulation, including transfusion-related iron overload, neurodegenerative disorders, and psychiatric illnesses. Sponsored by pharmaceutical corporations such as ApoPharma and Chiesi Canada Corp alongside academic centers like the University Hospital of Lille, these research initiatives display a broad range of recruitment statuses, encompassing active, prospective, completed, withdrawn, and terminated trials.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05604131 RECRUITING
Acute Myocardial Infarction Type 1
Rohan Dharmakumar
 2022-11-08 PHASE2
NCT07759895 NOT_YET_RECRUITING
Schizoaffecitve Disorder; Schizophrenia and Schizoaffective Disorder; Schizophrenia and Predominant Negative Symptoms
Amit Lotan
 2026-09-01 PHASE2
NCT03754725 ACTIVE_NOT_RECRUITING
SAH; Dementia
Duke University
 2020-10-01 PHASE1; PHASE2
NCT07023666 RECRUITING
Sickle Cell Disease
Inova Health Care Services
 2025-10-07 PHASE2
NCT05111821 TERMINATED
Stroke
University Hospital, Bordeaux
 2022-06-08 PHASE2
NCT00907283 UNKNOWN
Neurodegenerative Disease; Iron Overload
Ente Ospedaliero Ospedali Galliera
 2008-11 PHASE2
NCT03293069 COMPLETED
Amyotrophic Lateral Sclerosis
University Hospital, Lille
 2019-01-30 PHASE2; PHASE3
NCT03234686 COMPLETED
Mild Cognitive Impairment; Prodromal Alzheimer's Disease; Mild Alzheimer's Disease
Neuroscience Trials Australia
 2018-01-19 PHASE2
NCT04184453 UNKNOWN
Aceruloplasminemia
First Affiliated Hospital of Fujian Medical University
 2019-12-23 EARLY_PHASE1
NCT02878538 WITHDRAWN
Mild Cognitive Impairment
The University of Texas Health Science Center at San Antonio
 2018-01 EARLY_PHASE1
NCT03591575 COMPLETED
Beta Thalassemia Major Anemia; Iron Overload
Chiesi Canada Corp
 2018-11-09 PHASE4
NCT02655315 COMPLETED
Parkinson Disease
University Hospital, Lille
 2016-02-09 PHASE2
NCT03802916 COMPLETED
Iron Overload Due to Repeated Red Blood Cell Transfusions
ApoPharma
 2019-03-06 PHASE2
NCT02880033 COMPLETED
Parkinson's Disease; Amyotrophic Lateral Sclerosis; Oxidative Stress; Iron Overload
University Hospital, Lille
 2011-02
NCT02728843 COMPLETED
Parkinson's Disease
ApoPharma
 2016-10-12 PHASE2
NCT02443545 TERMINATED
Iron Overload; Sickle Cell Disease; Other Anemias
ApoPharma
 2015-05-21 PHASE4
NCT02041299 TERMINATED
Iron Overload; Sickle Cell Disease; Other Anemias
ApoPharma
 2014-04-17 PHASE4
NCT02882477 UNKNOWN
Diabetes Mellitus; Iron Metabolism Disorders; Gastroduodenal Ulcer; Optic Atrophy; Sensorineural Hearing Loss; Platelet Dysfunction
Hadassah Medical Organization
 2016-12 PHASE2; PHASE3
NCT02174848 COMPLETED
Pantothenate Kinase-Associated Neurodegeneration
ApoPharma
 2014-06 PHASE3
NCT02878538 Withdrawn
Mild Cognitive Impairment
The University of Texas Health Science Center at San Antonio
 2018-01 Early Phase 1
NCT01825512 COMPLETED
Chronic Iron Overload
Consorzio per Valutazioni Biologiche e Farmacologiche
 2014-03-17 PHASE3
NCT02477631 COMPLETED
Myelodysplastic Syndrome With Low-grade Lesions; Iron Overload Due to Repeated Red Blood Cell Transfusions
Sheba Medical Center
 2016-02 PHASE2
NCT02164253 COMPLETED
ALS (Amyotrophic Lateral Sclerosis); Iron Overload
University Hospital, Lille
 2013-09 PHASE2
NCT01741532 COMPLETED
Pantothenate Kinase-Associated Neurodegeneration
ApoPharma
 2012-12-13 PHASE3
NCT02728843 Completed
Parkinson''s Disease
ApoPharma|Chiesi Canada Corp
 2016-10-12 Phase 2
NCT02980458 COMPLETED
Bioequivalence
SocraTec R&D GmbH
 2016-08 PHASE1
NCT02456558 COMPLETED
Asymptomatic HIV Infection
ApoPharma
 2015-06 PHASE1
NCT02477631 Completed
Myelodysplastic Syndrome With Low-grade Lesions|Iron Overload Due to Repeated Red Blood Cell Transfusions
Sheba Medical Center|Hadassah Medical Organization|Tel Aviv Medical Center|Kaplan Medical Center|Ziv Medical Center
 2016-02 Phase 2
NCT01709032 COMPLETED
Thalassemia Major With Severe Transfusional Iron Overload
Children's Hospital of Philadelphia
 2012-09 PHASE1; PHASE2
NCT02465489 COMPLETED
Healthy
ApoPharma
 2015-06 PHASE1
NCT02173951 UNKNOWN
Beta Thalassemia Major
Ain Shams University
 2014-07 PHASE2; PHASE3
NCT02442310 COMPLETED
Healthy
ApoPharma
 2015-05 PHASE1
NCT01539837 COMPLETED
Parkinson's Disease
Imperial College London
 2012-02 PHASE2
NCT02189941 COMPLETED
Healthy
ApoPharma
 2014-05 PHASE1
NCT02083575 UNKNOWN
Vitamin c; Thalassemia Major; Iron Chelation
Ain Shams University
 2014-04 PHASE2; PHASE3
NCT01989455 COMPLETED
Healthy Volunteers
ApoPharma
 2013-11 PHASE1
NCT01740713 COMPLETED
Chronic Iron Overload
Consorzio per Valutazioni Biologiche e Farmacologiche
 2012-12 PHASE2
NCT01770652 COMPLETED
Renal Impairment
ApoPharma
 2013-01 PHASE4
NCT01391520 WITHDRAWN
Acute Kidney Injury
CorMedix Therapeutics
 2012-01 PHASE3
NCT01511848 UNKNOWN
Beta-thalassemia Major; Sickle Cell Disease; Iron Hemosiderosis
Ain Shams University
 2012-02 PHASE2; PHASE3
NCT01860703 COMPLETED
Prolonged QTc Interval
ApoPharma
 2012-11 PHASE4
NCT00943748 COMPLETED
Parkinson's Disease
University Hospital, Lille
 2009-10 PHASE2; PHASE3
NCT01597765 COMPLETED
Thalassemia
Mahidol University
 2009-06
NCT01146925 COMPLETED
Contrast-Induced Acute Kidney Injury
CorMedix Therapeutics
 2010-06 PHASE2
NCT00349453 COMPLETED
Hemochromatosis
Lipomed
 2005-03 PHASE2
NCT00897221 COMPLETED
Friedreich's Ataxia
ApoPharma
 2009-06 PHASE2
NCT00530127 COMPLETED
Friedreich's Ataxia
ApoPharma
 2008-04 PHASE1; PHASE2
NCT02198508 COMPLETED
Beta-thalassemia Major
China Medical University Hospital
 2007-07
NCT00529152 COMPLETED
Iron Overload
ApoPharma
 2007-08 PHASE3
NCT00115349 TERMINATED
Cardiovascular Diseases; Heart Diseases; Beta-Thalassemia
Carelon Research
 2005-06 PHASE2
NCT02191657 COMPLETED
HIV Infection
ApoPharma
 2006-11 PHASE1
NCT00733811 COMPLETED
Beta-Thalassemia; Thalassemia Major
Azienda Ospedaliera V. Cervello
 2000-09 PHASE4
NCT00800761 COMPLETED
Iron Overload; Cardiomyopathy
Ospedale Microcitemico
 2001-12 PHASE4
NCT00105495 COMPLETED
Thalassemia Major; Hemosiderosis
ApoPharma
 2002-12 PHASE4
NCT00103753 UNKNOWN
Beta-Thalassemia
Royal Brompton & Harefield NHS Foundation Trust
 2004-05 PHASE4
NCT00350662 COMPLETED
Hemochromatosis
Lipomed
 2002-01 PHASE3

(data from https://clinicaltrials.gov, updated on 2026-04-08)

Check the Deferiprone product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Deferiprone selectively binds ferric ions to form stable neutral 3:1 drug-to-iron complexes, thereby sequestering catalytic iron and inhibiting the downstream generation of reactive oxygen species. By reducing toxic labile iron pools and penetrating cellular membranes, this mechanism protects tissues against oxidative stress, demonstrating clinical relevance in managing transfusion-dependent iron overload disorders, thalassemia major, and neurodegenerative diseases like Parkinson's disease.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.