NCT03368742

Brief Summary

This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants 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 participants were dosed.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
6mo left

Started Dec 2017

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress95%
Dec 2017Oct 2026

First Submitted

Initial submission to the registry

December 5, 2017

Completed
1 day until next milestone

Study Start

First participant enrolled

December 6, 2017

Completed
5 days until next milestone

First Posted

Study publicly available on registry

December 11, 2017

Completed
8.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 15, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 15, 2026

Last Updated

March 3, 2026

Status Verified

March 1, 2026

Enrollment Period

8.9 years

First QC Date

December 5, 2017

Last Update Submit

March 2, 2026

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

  • Number of Participants with Treatment Emergent Adverse Events (TEAEs)

    Up to 5 years

Secondary Outcomes (14)

  • Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters

    Up to 5 years

  • Number of Participants with Clinically Significant Abnormalities in Vital Signs

    Up to 5 years

  • Number of Participants with Clinically Significant Abnormalities in Physical Examinations

    Up to 5 years

  • Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)

    Up to 5 years

  • Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)

    Baseline, 12 months

  • +9 more secondary outcomes

Study Arms (3)

SGT-001 - Dose Level 1

EXPERIMENTAL

Single IV infusion of SGT-001 at starting dose

Genetic: SGT-001

SGT-001 - Dose Level 2

EXPERIMENTAL

Single IV infusion of SGT-001 at next ascending dose

Genetic: SGT-001

Untreated Control

NO INTERVENTION

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

Interventions

SGT-001GENETIC

AAV9 vector containing muscle-specific promoter and microdystrophin construct

SGT-001 - Dose Level 1SGT-001 - Dose Level 2

Eligibility Criteria

Age4 Years - 17 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
  • Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants)
  • 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 weeks

You may not qualify if:

  • Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Related Publications (1)

  • Boehler JF, Brown KJ, Ricotti V, Morris CA. N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy. Skelet Muscle. 2024 Jan 16;14(1):2. doi: 10.1186/s13395-023-00334-y.

    PMID: 38229112DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Solid Bio Clinical Trials

    Solid Biosciences

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 5, 2017

First Posted

December 11, 2017

Study Start

December 6, 2017

Primary Completion (Estimated)

October 15, 2026

Study Completion (Estimated)

October 15, 2026

Last Updated

March 3, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations

US(2)