THERAPY DOSSIER · th-436383d77703

Deferiprone (with ferrostatin-1 tested separately)

Drug · Unknown

No trauma translation Suggest a correction

This record is classified as “NO TRAUMA TRANSLATION”. Read the indication and notes below for the specific context.

Evidence & regulatory context

Mechanism: Unknown
Target: Not recorded
Injury models: Radiation
Reported indication: Not recorded
First approval: 1999
Development stage: Not recorded
ChEMBL record: CHEMBL70927 ↗
ChEMBL max phase: 4 / 4 (development scale, not regulatory approval)
Synonyms: APO-066; CP-20; Deferiprone; Ferriprox; L1; NSC-758880; PL1
Note: Not recorded

Clinical trials 7

0 linked trials carry an injury-indication flag. These flags support record-level exploration and sit alongside the stringent aggregate counts described in Methods.

NCT02174848 ↗PHASE3completed

Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)

Pantothenate Kinase-Associated Neurodegeneration

Enrolment: 68 · Results not recorded

NCT02882477 ↗PHASE2/PHASE3unknown

Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy

Diabetes Mellitus · Iron Metabolism Disorders · Gastroduodenal Ulcer · Optic Atrophy · Sensorineural Hearing Loss · Platelet Dysfunction

Enrolment: 20 · Results not recorded

NCT00897221 ↗PHASE2completed

An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia

Friedreich's Ataxia

Enrolment: 36 · Results not recorded

NCT01709032 ↗PHASE1/PHASE2completed

Pilot Study of Deferasirox and Deferiprone Combination Oral Chelation for Individuals With Transfusion Dependent Thalassemia and High Iron Burden

Thalassemia Major With Severe Transfusional Iron Overload

Enrolment: 9 · Results not recorded

NCT01989455 ↗PHASE1completed

A Single Center, Phase I, Double-blind, Placebo-controlled Evaluation of the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of Deferiprone Administered by Intravenous Infusion to Healthy Volunteers

Healthy Volunteers

Enrolment: 64 · Results not recorded

NCT02880033 ↗NAcompleted

Modulation of Oxidative Stress and Apoptosis of Energy Metabolism by Deferiprone From the Circulating Lymphocytes of Patients With Parkinson's Disease or Amyotrophic Lateral Sclerosis

Parkinson's Disease · Amyotrophic Lateral Sclerosis · Oxidative Stress · Iron Overload

Enrolment: 90 · Results not recorded

NCT02635841 ↗Phase not recordedno longer available

The Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration

Pantothenate Kinase-Associated Neurodegeneration

Enrolment not recorded · Results not recorded

Preclinical evidence 1

1 studies reported at therapy level; 1 evidence entries itemised.

PMID 32868705 ↗2020Reported benefit

A Potential Role for Excess Tissue Iron in Development of Cardiovascular Delayed Effects of Acute Radiation Exposure

MouseRadiationC57BL/6Male and female (50/50), male only for the time course and ferrostatin experimentsDeferiprone oral in drinking water ad libitum; ferrostatin-1 intraperitonealDeferiprone 2.5 g/litre in acidified drinking water, estima…Both agents started approximately 24 h before irradiation

Model: Single uniform total body gamma irradiation of 8.53 Gy (LD50/30) from a caesium source, haematopoietic acute radiation syndrome model

Serum iron and transferrin saturation peaked at 2 weeks post irradiation and normalised by 4 weeks, myocardial iron appeared by 2 weeks and persisted to 12 weeks, and coronary endothelial cell density fell from 2 weeks and remained reduced to 12 weeks. Deferiprone pretreatment reduced myocardial ir…

Extraction: extract · Supplied extraction confidence: 0.85. Machine-extracted fields require source verification.

Loading the evidence…