A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Insmed Gene Therapy LLC
Study ID
NCT06817382
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
MALE
Age
2 Years - 4 Years
Healthy Volunteers
Not accepted

Interventions

  • INS1201 — GENETIC
    Suspension for IT injection.

Study Details

The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.

Key Dates

Start date
Jul 22, 2025
Status verified
Jun 2026
Primary completion
Jan 31, 2028
Completion
Mar 31, 2028

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1: Cohort 1
    Participants aged 3 to \<5 years will receive a single dose level 1 of INS1201 by IT injection on Day 1.
  • Experimental: Part 1: Cohort 2
    Participants aged 3 to \<5 years will receive a single dose level 2 of INS1201 by IT injection on Day 1.
  • Experimental: Part 2: Cohort 3
    Participants aged 2 to \<3 years will receive a single dose level 1 of INS1201 by IT injection on Day 1.
  • Experimental: Part 2: Cohort 4
    Participants aged 2 to \<3 years will receive a single dose level 2 of INS1201 by IT injection on Day 1.

Primary Outcome Measure

Parts 1 and 2: Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to Week 96 ]

Central Contacts

Locations (10)

FacilityCityStateZIPSite coordinators
USA012Little RockArkansas72202
Kendra Stroud
[email protected]
USA010DavisCalifornia95616
Amanda Marie Figueroa Lopez
[email protected]
USA009Los AngelesCalifornia90095
Ummi Qasim
[email protected]
310-825-3284
USA002Palo AltoCalifornia94070
Rabia Farooquee
[email protected]
USA005San DiegoCalifornia93123
Elizabeth (Ellie) Moreno
[email protected]
Rare Disease Research (USA004)AtlantaGeorgia30329
Laura Sutton
[email protected]
USA008RochesterNew York14642
USA006ColumbusOhio43205
Abigail Hanson
[email protected]
USA001MemphisTennessee38105
Colin Quillivan
[email protected]
USA015NorfolkVirginia23507
Erika Paradiso
[email protected]

Find similar trials in Little Rock, AR

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
USA012· Little Rock, ARUSA010· Davis, CAUSA009· Los Angeles, CAUSA002· Palo Alto, CAUSA005· San Diego, CARare Disease Research (USA004)· Atlanta, GA

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