Peking Union Medical College Hospital
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The study will evaluate the safety and tolerability of GEN6050X gene therapy in Duchenne muscular dystrophy (DMD) patients amenable to exon 50 skipping.
Duchenne Muscular Dystrophy (DMD)
GEN6050X intravenous injection
Early Phase 1
GEN6050X is an intravenously administered human DMD exon 50 skipping base editing drug containing dual single-stranded adeno-associated virus serotype 9 (ss.AAV9) vectors. The study is a first-in-human, single-arm, open-label, single-center clinical trial to evaluate safety and tolerability of a single intravenous infusion of GEN6050X in ambulatory boys with DMD. Other objectives include pharmacokinetics, pharmacodynamics, and the preliminary clinical efficacy of GEN6050X over 52 weeks. A total of three ambulatory pediatric participants (aged 4 to 9 years old) are expected to enroll, each receiving a dose of 5×10^13 vg/kg. These participants will be dosed in a staggered fashion. Safety assessments will include monitoring of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study duration. In addition, a comprehensive short-term prophylactic immunosuppression regimen(including rituximab and sirolimus) will be administered prior to treatment in order to mitigate potential immune response.
Study Type : | Interventional |
Estimated Enrollment : | 3 participants |
Masking : | None (Open Label) |
Primary Purpose : | Treatment |
Official Title : | A Single-arm, Open-label, Single-center Study to Evaluate the Safety and Tolerability of Intravenous GEN6050X Gene Therapy in Ambulatory Boys With Duchenne Muscular Dystrophy (DMD). |
Actual Study Start Date : | May 2024 |
Estimated Primary Completion Date : | December 2025 |
Estimated Study Completion Date : | December 2027 |
Arm | Intervention/treatment |
---|---|
Experimental: GEN6050X A single IV infusion of GEN6050X at a dose of 5×10^13 vector genome(VG)/kg body weight Interventions: Genetic: GEN6050X |
Genetic: GEN6050X intravenous injection |
Ages Eligible for Study: | 4 Years to 9 Years |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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