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

Phase 1

Recruiting

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: Phase 1, SQY51; Drug: Phase 2a, SQY51 (cohort 3); Drug: Phase 2a, SQY51 (cohort 1); Drug: Phase 2a, SQY51 (cohort 2)

• Registration Number: NCT05753462
• Lead Sponsor: Sqy Therapeutics

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.

Study Timeline

• Study First Submitted: 2023-02-06
• Study First Submitted QC: 2023-02-21
• Study First Posted: 2023-03-03
• Study Start: 2023-04-26
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-28
• Last Update Posted: 2024-04-01
• Primary Completion: 2025-02
• Study Completion: 2025-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 12

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Phase 1	Phase 1, SQY51	Participants will receive single escalating doses of 2, 4, 6, 10, 16 and 25 mg/kg by intravenous infusion of SQY51 every 2 weeks.
Phase 2a - Treatment arm (Dose 3)	Phase 2a, SQY51 (cohort 3)	Non randomized participants will receive by IV dose 3 of SQY51 in 4 blocks of 4-weeks.
Phase 2a - Treatment arm (Dose 1)	Phase 2a, SQY51 (cohort 1)	Non randomized participants will receive by IV dose 1 of SQY51 in 4 blocks of 4-weeks.
Phase 2a - Treatment arm (Dose 2)	Phase 2a, SQY51 (cohort 2)	Non randomized participants will receive by IV dose 2 of SQY51 in 4 blocks of 4-weeks.

Primary Outcome Measures

Name	Time	Method
Incidence of AEs in all participants	From baseline up to week 49

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Hôpital Raymond Poincaré; 🇫🇷 Garches, France