Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy (AVANCE1)

March 28, 2024 updated by: Sqy Therapeutics

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • France
      • Garches, France, 92380
        • Recruiting
        • Hopital Raymond Poincare
        • Principal Investigator:
          • Helge Amthor, MD, PhD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

INCLUSION CRITERIA FOR PHASE 1:

  • Boys of ≥6 years of age and ≥ 16 kg body weight.
  • Ambulatory or non-ambulatory status,
  • Patients and, if minor, their legal guardians, who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
  • Diagnosed with Duchenne Muscular Dystrophy (DMD), genotypically confirmed with DMD mutations amenable to exon-51 skipping.
  • Stable hepatic and renal function.
  • Left ventricular ejection fraction (LVEF) at screening ≥40%.
  • If clinically indicated, approved concomitant treatment within standards of care guidelines for DMD, such as antihypertensive, vasodilators, lipid lowering, thyroid replacement, vitamins, mineral substitution, gastric protectors, and nutritional supplements.
  • Non-invasive mechanical ventilation is permissive if < 16 h/day.
  • Being affiliated with a French social security.
  • Informed consent form signed by the patient or, if minor, by the legal guardian(s).

INCLUSION CRITERIA FOR PHASE 2a:

Patients must have completed Phase 1 of the study.

EXCLUSION CRITERIA FOR PHASE 1 AND 2a:

  • Patient with any serious medical/surgical or psychiatric condition/illness/history that in the opinion of the investigator would jeopardize patient's safety or would interfere with the study assessments/results, including insufficient vaccination against infectious diseases as recommended by national guidelines, medical history of infection with Hepatitis B,C and HIV.
  • Patient with any known allergies to products likely to be used in the study (e.g., antiseptics, anesthetics), known hypersensitivity to any of the ingredients, or excipients of the study drug).
  • Patient who participated in other investigational study within the last three months, including those with investigational drugs that aim at restoring dystrophin expression such as other antisense oligomers.
  • Patient that received gene therapy.
  • Patient with intellectual disability or behavioral problem such that they cannot comply with the study procedure.
  • Patient with advanced cardiomyopathy and LVEF < 40%. Patients with dysrhythmias and being treated for dysrhythmias. Patients with non-treated tachycardia.
  • Patient for which orthopedic surgery is planned during the time of the study.
  • Tracheostomized patients and dependent on invasive mechanical ventilation. Non-invasive mechanical ventilation ≥ 16 h/day. Predicted vital forced capacity < 20%. Medical history with more than two respiratory decompensations requiring hospitalization during the previous year. No respiratory decompensation in the four months preceding enrolment.
  • Patients on medications that can restore dystrophin expression, tamoxifen and other drugs without indication for DMD or paediatric population.
  • Abnormal laboratory values in the clinically significant range.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Non-Randomized
  • Interventional Model: Crossover Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: Phase 1, SQY51
SQY51 is administered by intravenous infusion.
Experimental: Phase 2a - Treatment arm (Dose 1)
Non randomized participants will receive by IV dose 1 of SQY51 in 4 blocks of 4-weeks.
Drug: Phase 2a, SQY51 (cohort 1)
SQY51 is administered by intravenous infusion at dose 1
Experimental: Phase 2a - Treatment arm (Dose 2)
Non randomized participants will receive by IV dose 2 of SQY51 in 4 blocks of 4-weeks.
Drug: Phase 2a, SQY51 (cohort 2)
SQY51 is administered by intravenous infusion at dose 2.
Experimental: Phase 2a - Treatment arm (Dose 3)
Non randomized participants will receive by IV dose 3 of SQY51 in 4 blocks of 4-weeks.
Drug: Phase 2a, SQY51 (cohort 3)
SQY51 is administered by intravenous infusion at dose 3.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of AEs in all participants
Time Frame: From baseline up to week 49
From baseline up to week 49

Secondary Outcome Measures

Outcome Measure
Time Frame
Pharmacokinetic plasma concentration of SQY51 (µg/ml)
Time Frame: From baseline up to week 49
From baseline up to week 49
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
Time Frame: From baseline up to week 49
From baseline up to week 49
Changes from baseline in skeletal muscle dystrophin expression
Time Frame: From baseline up to week 49
From baseline up to week 49

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sqy Therapeutics

Collaborators

Biotrial

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 26, 2023

Primary Completion (Estimated)

February 1, 2025

Study Completion (Estimated)

February 1, 2025

Study Registration Dates

First Submitted

February 6, 2023

First Submitted That Met QC Criteria

February 21, 2023

First Posted (Actual)

March 3, 2023

Study Record Updates

Last Update Posted (Actual)

April 1, 2024

Last Update Submitted That Met QC Criteria

March 28, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AVANCE1-1/2a
  • 2022-500703-49-01 (Other Identifier: EMA)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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