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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

Status
Recruiting
Phases
Phase 1Phase 2
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-505187-11-00
Acronym
GNT-016-MDYF
Enrollment
20
Registered
2023-06-21
Start date
2021-03-01
Completion date
Unknown
Last updated
2026-01-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Brief summary

NSAA: change from baseline at week 52

Detailed description

- Safety and tolerability, measured by the incidence of adverse event (AE) or serious adverse event (SAE) evaluated by changes in laboratory parameters, vital signs and in the physical examination, PK/PD endpoints including vector shedding quantification in blood, urine, saliva, feces, Clinical efficacy endpoints including NSAA, Time to 10 Meters Walk/Run Test (10MWRT), Time to Rise From Floor (RFF), 6-Minutes Walk Test (6MWT)

Interventions

DRUGRapamune 1 mg/mL oral solution
DRUGrAAV8-hMD1
DRUGPREDNISOLONE
DRUGPlacebo AxMP is a solution intended to be administered by oral route for clinical purposes. Placebo AxMP is a pale yellow to yellow solution. Placebo AxMP composition corresponds to the excipients of the authorized product Rapamune® 1 mg/mL oral solution composed of a mixture of Phosal 50PG and Polysorbate 80.
DRUGSoliris 300 mg concentrate for solution for infusion
DRUGRINGER LACTATE FRESENIUS KABI FRANCE
DRUGsolution pour perfusion
DRUGMETHYLPREDNISOLONE

Sponsors

Genethon
Lead SponsorOTHER

Eligibility

Sex/Gender
Male
Age
0 Years to 17 Years

Design outcomes

Primary

MeasureTime frame
NSAA: change from baseline at week 52—

Secondary

MeasureTime frame
- Safety and tolerability, measured by the incidence of adverse event (AE) or serious adverse event (SAE) evaluated by changes in laboratory parameters, vital signs and in the physical examination, PK/PD endpoints including vector shedding quantification in blood, urine, saliva, feces, Clinical efficacy endpoints including NSAA, Time to 10 Meters Walk/Run Test (10MWRT), Time to Rise From Floor (RFF), 6-Minutes Walk Test (6MWT)—

Countries

France

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026