NCT05753462

Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy

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

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Sqy Therapeutics · Academic / Other
• Sex: Male
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Phase 1, SQY51	SQY51 is administered by intravenous infusion.
DRUG	Phase 2a, SQY51 (cohort 1)	SQY51 is administered by intravenous infusion at dose 1
DRUG	Phase 2a, SQY51 (cohort 2)	SQY51 is administered by intravenous infusion at dose 2.
DRUG	Phase 2a, SQY51 (cohort 3)	SQY51 is administered by intravenous infusion at dose 3.

Timeline

• Start date: 2023-04-26
• Primary completion: 2025-02-01
• Completion: 2025-02-01
• First posted: 2023-03-03
• Last updated: 2024-04-01

Locations

1 site across 1 country: France