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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)

Primary Purpose

Duchenne Muscular Dystrophy

Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
SGT-001
Sponsored by
Solid Biosciences Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy

Eligibility Criteria

4 Years - 17 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
  • Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory patients)
  • Anti-AAV9 antibodies below protocol-specified thresholds
  • Stable cardiac and pulmonary function
  • Adolescents: non-ambulatory by protocol-specified criteria
  • Children: ambulatory by protocol-specified criteria
  • Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 wks

Exclusion Criteria:

  • Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect subject safety, compromise completion of treatment and follow-up, or impair assessment of study results
  • Abnormal liver function
  • Abnormal renal function
  • Clinically significant coagulation abnormalities
  • Impaired cardiovascular function based on cardiac MRI or ECHO
  • Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support
  • Significant spinal deformity or presence of spinal rods
  • Body mass index ≥ 95th percentile for age
  • Exposure to another investigational drug within 3 months or 5 half-lives prior to screening
  • Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening

Additional inclusion/exclusion criteria may apply. Patients over 30 kg will not be eligible for treatment at this time. A weight limit of ≤ 18 kg will be implemented for the next two patients to be dosed.

Sites / Locations

  • David Geffen School of Medicine at UCLA
  • University of Florida

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

No Intervention

Arm Label

SGT-001 - Dose Level 1

SGT-001 - Dose Level 2

Untreated Control

Arm Description

Single IV infusion of SGT-001 at starting dose

Single IV infusion of SGT-001 at next ascending dose

Untreated control group. After 1 year, treatment-eligible control patients will receive SGT-001 at the selected dose.

Outcomes

Primary Outcome Measures

Primary efficacy endpoint
Change from baseline in microdystrophin protein in muscle biopsies (active treatment group)
Primary safety endpoint
Incidence of adverse events
Primary safety endpoint
Incidence of clinical laboratory abnormalities
Primary safety endpoint
Incidence of abnormalities in vital signs
Primary safety endpoint
Incidence of abnormalities in physical examinations
Primary safety endpoint
Incidence of abnormalities on ECGs

Secondary Outcome Measures

Full Information

First Posted
December 5, 2017
Last Updated
October 12, 2023
Sponsor
Solid Biosciences Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03368742
Brief Title
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
Acronym
IGNITE DMD
Official Title
A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 6, 2017 (Actual)
Primary Completion Date
December 2026 (Anticipated)
Study Completion Date
December 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Solid Biosciences Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Patients will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 subjects were dosed. Subjects currently enrolling are assigned to active treatment. Control subjects enrolled under original protocol will continue through the study per the original protocol.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Active treatment group for all patients enrolled after June 2019. Total of approximately 16 to 32 patients.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
16 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
SGT-001 - Dose Level 1
Arm Type
Experimental
Arm Description
Single IV infusion of SGT-001 at starting dose
Arm Title
SGT-001 - Dose Level 2
Arm Type
Experimental
Arm Description
Single IV infusion of SGT-001 at next ascending dose
Arm Title
Untreated Control
Arm Type
No Intervention
Arm Description
Untreated control group. After 1 year, treatment-eligible control patients will receive SGT-001 at the selected dose.
Intervention Type
Genetic
Intervention Name(s)
SGT-001
Intervention Description
AAV9 vector containing muscle-specific promoter and microdystrophin construct
Primary Outcome Measure Information:
Title
Primary efficacy endpoint
Description
Change from baseline in microdystrophin protein in muscle biopsies (active treatment group)
Time Frame
12 months
Title
Primary safety endpoint
Description
Incidence of adverse events
Time Frame
12 months
Title
Primary safety endpoint
Description
Incidence of clinical laboratory abnormalities
Time Frame
12 months
Title
Primary safety endpoint
Description
Incidence of abnormalities in vital signs
Time Frame
12 months
Title
Primary safety endpoint
Description
Incidence of abnormalities in physical examinations
Time Frame
12 months
Title
Primary safety endpoint
Description
Incidence of abnormalities on ECGs
Time Frame
12 months

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory patients) Anti-AAV9 antibodies below protocol-specified thresholds Stable cardiac and pulmonary function Adolescents: non-ambulatory by protocol-specified criteria Children: ambulatory by protocol-specified criteria Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 wks Exclusion Criteria: Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect subject safety, compromise completion of treatment and follow-up, or impair assessment of study results Abnormal liver function Abnormal renal function Clinically significant coagulation abnormalities Impaired cardiovascular function based on cardiac MRI or ECHO Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support Significant spinal deformity or presence of spinal rods Body mass index ≥ 95th percentile for age Exposure to another investigational drug within 3 months or 5 half-lives prior to screening Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening Additional inclusion/exclusion criteria may apply. Patients over 30 kg will not be eligible for treatment at this time. A weight limit of ≤ 18 kg will be implemented for the next two patients to be dosed.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Roxana Donisa Dreghici, MD
Organizational Affiliation
Solid Biosciences
Official's Role
Study Director
Facility Information:
Facility Name
David Geffen School of Medicine at UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

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