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NCT03642145: PTCEMF
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
Phase 3 trial testing Deflazacort in Duchenne Muscular Dystrophy. Withdrawn.
31 July 2021
Quick facts
| Lead sponsor | PTC Therapeutics |
|---|---|
| Phase | Phase 3 |
| Status | Withdrawn |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Start date | 31 October 2018 |
| Primary completion | 31 July 2021 |
| Estimated completion | 31 July 2021 |
Drugs / interventions tested
- Deflazacort (DEFLAZACORT) — full drug profile →
Conditions studied
- Duchenne Muscular Dystrophy — all drugs for Duchenne Muscular Dystrophy →
Sponsor
PTC Therapeutics — full company profile →
Who can join
Adults 2 to 4, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males with DMD aged greater than or equal to (\>=) 2 to lesser than (\<) 5 years. The study will comprise of 2 periods (Period 1: 52-week safety and pharmacokinetics \[PK\], and Period 2: 52-week extension). Participants will be randomized in a 1:1 ratio to one of 2 treatment arms: 0.9 mg/kg deflazacort, and 0.45 mg/kg of deflazacort. A historic control group (which should match the study population as closely as possible) will be used as a comparator to characterize the safety and tolerability of deflazacort.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT03642145
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Deflazacort
Trials testing the same drug.
- NCT04791345 — Study of the Excretion of Orally Administered Corticosteroids for the Improval of the Detection of Said Substances in An · Phase 1 · unknown
- NCT03783923 — A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) · Phase 3 · terminated
- NCT02592941 — Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy · approved for marketing
Other recruiting trials for Duchenne Muscular Dystrophy
Currently open trials in the same condition.
- NCT07287189 — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients · Phase 2 · recruiting
- NCT06817382 — A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Ma · Phase 1 · recruiting
- NCT06402942 — Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy · NA · recruiting
- NCT06450639 — A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) · Phase 2 · active not recruiting
- NCT06692426 — Trial of Cell Based Therapy for DMD · Phase 1 · recruiting
Other PTC Therapeutics trials
Trials by the same sponsor.
- NCT06302348 — A Study of Sepiapterin in Participants With Phenylketonuria (PKU) · Phase 3 · recruiting
- NCT05515536 — A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia · Phase 3 · active not recruiting
- NCT05485987 — A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia · Phase 2 · completed
- NCT05218655 — A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease · Phase 3 · completed
- NCT05358717 — A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) · Phase 2 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT03642145 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by PTC Therapeutics
- Last refreshed: 21 June 2019
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03642145.
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