Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, PK and PD of SQY51 in Paediatric and Adult Patients With a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy
Overview
- Phase
- Phase 1
- Intervention
- Phase 1, SQY51
- Conditions
- Duchenne Muscular Dystrophy
- Sponsor
- Sqy Therapeutics
- Enrollment
- 12
- Locations
- 1
- Primary Endpoint
- Incidence of AEs in all participants
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
This is a Phase 1/2a, monocentric, open label study to evaluate the safety, pharmacokinetics, and pharmacodynamics of SQY51 in patients with Duchenne muscular dystrophy
Detailed Description
Avance1 is a Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SQY51 in 12 patients with a genetically confirmed diagnosis of Duchenne muscular dystrophy, This study will include i) 13-week Phase 1 Multiple Dose Escalation Phase, and a ii) 32-week Phase 2a. Twelve (12) patients ≥ 6 years, both ambulant and non-ambulant, will be sequentially enrolled in phase 1 and will receive escalating doses of SQY51 once every two weeks. In phase 2a, patients will be allocated in three cohorts in a non-randomized manner. On the 25th March 2024, SQY Therapeutics received the authorization from the European Medicines Agency (EMA) to initiate the Phase 2a clinical trial. All the patients involved in the Phase 1 will progress to the Phase 2a.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Phase 1
Participants will receive single escalating doses of 2, 4, 6, 10, 16 and 25 mg/kg by intravenous infusion of SQY51 every 2 weeks.
Intervention: Phase 1, SQY51
Phase 2a - Treatment arm (Dose 1)
Non randomized participants will receive by IV dose 1 of SQY51 in 4 blocks of 4-weeks.
Intervention: Phase 2a, SQY51 (cohort 1)
Phase 2a - Treatment arm (Dose 2)
Non randomized participants will receive by IV dose 2 of SQY51 in 4 blocks of 4-weeks.
Intervention: Phase 2a, SQY51 (cohort 2)
Phase 2a - Treatment arm (Dose 3)
Non randomized participants will receive by IV dose 3 of SQY51 in 4 blocks of 4-weeks.
Intervention: Phase 2a, SQY51 (cohort 3)
Outcomes
Primary Outcomes
Incidence of AEs in all participants
Time Frame: From baseline up to week 49
Secondary Outcomes
- Change from baseline in time to rise from floor, time to complete 1-min, 6-min and 10-min walk in ambulant patients as well as MFM and PUL scores in both ambulant and non-ambulant patients(From baseline up to week 49)
- Changes from baseline in skeletal muscle dystrophin expression(From baseline up to week 49)
- Pharmacokinetic plasma concentration of SQY51 (µg/ml)(From baseline up to week 49)