Ignite Creation Date:
2024-05-06 @ 8:28 PM
Last Modification Date:
2024-10-26 @ 3:28 PM
Study NCT ID:
NCT06392724
Status:
RECRUITING
Last Update Posted:
2024-07-08
First Post:
2024-04-26
Brief Title:
A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy
Sponsor:
Peking Union Medical College Hospital
Organization:
Peking Union Medical College Hospital
Study Overview
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
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
GEN6050XIIT
Brief Summary:
The study will evaluate the safety and tolerability of GEN6050X gene therapy in Duchenne muscular dystrophy DMD patients amenable to exon 50 skipping
Detailed Description:
GEN6050X is an intravenously administered human DMD exon 50 skipping base editing drug containing dual single-stranded adeno-associated virus serotype 9 ssAAV9 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 51013 vgkg 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 regimenincluding rituximab and sirolimus will be administered prior to treatment in order to mitigate potential immune response
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 51013 vgkg 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 regimenincluding rituximab and sirolimus will be administered prior to treatment in order to mitigate potential immune response
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None