WHO ICTRP trial
A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene…
EUCTR2020-002093-27-FR · EU Clinical Trials Register · Authorised
Key facts
| Trial ID | EUCTR2020-002093-27-FR |
|---|---|
| Public title | A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1). After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2). In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3). |
| Scientific title | Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys… |
| Primary sponsor | Genethon |
| Registry | EU Clinical Trials Register |
| Recruitment status | Authorised |
| Study type | Interventional clinical trial of medicinal product |
| Phase | Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no |
| Countries | United States;France;Israel;United Kingdom |
| Conditions | Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C]… |
| Interventions | Product Name: rAAV8-hMD1 Product Code: GNT0004 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: To be determined Current Sponsor code: GNT0004 Other descriptive name: Adeno-associated viral vector serotype 8… |
| Registration date | Jun 24, 2020 |
| Target size | 51 |
| Secondary IDs | GNT-016-MDYF |
| Registry page | clinicaltrialsregister.eu |
At a glance
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About WHO ICTRP trial registrations
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Source: World Health Organization (WHO), International Clinical Trials Registry Platform (ICTRP) · Source date: