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ACTIVE NOT RECRUITING
NCT06128564
PHASE2

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

Sponsor: Hoffmann-La Roche

View on ClinicalTrials.gov

Summary

This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.

Official title: A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy

Key Details

Gender

MALE

Age Range

2 Years - 3 Years

Study Type

INTERVENTIONAL

Enrollment

13

Start Date

2023-11-29

Completion Date

2034-01-30

Last Updated

2026-02-10

Healthy Volunteers

No

Interventions

GENETIC

delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec

Locations (7)

Chr de La Citadelle

Liège, Belgium

Hôpital Necker-Enfants Malades

Paris, France

Universitätsklinikum Essen

Essen, Germany

PU A. Gemelli, Università Cattolica del Sacro Cuore

Rome, Lazio, Italy

Hospital Sant Joan De Deu

Esplugues de Llobregas, Barcelona, Spain

Great Ormond Street Hospital for Children

London, United Kingdom

John Radcliffe Hospital

Oxford, United Kingdom