INS1201 for Duchenne Muscular Dystrophy

(ASCEND Trial)

Recruiting at 8 trial locations

Overseen By Insmed Medical Information

Age: < 18

Sex: Male

Trial Phase: Phase 1

Sponsor: Insmed Gene Therapy LLC

Must not be taking: Gene therapy, Exon skipping

Disqualifiers: Cardiomyopathy, Arrhythmia, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment, INS1201, for boys with Duchenne Muscular Dystrophy (DMD), a condition affecting muscles and movement. The goal is to determine if a single dose of INS1201 is safe and tolerable. The trial includes two groups: boys aged 3 to under 5 years and boys aged 2 to under 3 years. It seeks boys who can walk at least 10 meters independently and have a specific genetic diagnosis of DMD. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, it excludes participants who require chronic drug treatment that might compromise safety or compliance, so it's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that INS1201 is likely to be safe for humans?

Research shows that the treatment INS1201 is undergoing safety testing in boys with Duchenne Muscular Dystrophy (DMD). This marks the first human trial of INS1201, so specific safety information is not yet available. At this early stage, researchers focus primarily on ensuring the treatment's safety for participants. Safety remains their top priority. As more boys receive the treatment, researchers will closely monitor for any side effects to ensure it is well-tolerated. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne Muscular Dystrophy?

INS1201 is unique because it offers a novel approach for treating Duchenne Muscular Dystrophy (DMD) in young children. Unlike standard treatments such as corticosteroids, which aim to reduce inflammation, INS1201 is delivered directly into the spinal fluid through an intrathecal (IT) injection. This method allows the drug to target the nervous system more directly and potentially slow muscle degeneration. Researchers are excited about INS1201 because its targeted delivery could lead to more effective management of DMD symptoms with potentially fewer side effects compared to traditional therapies.

What evidence suggests that INS1201 might be an effective treatment for Duchenne Muscular Dystrophy?

Research suggests that INS1201 could be a promising treatment for Duchenne Muscular Dystrophy (DMD). Early animal studies showed that this treatment reduced DMD symptoms and improved muscle strength. The therapy aims to correct the genetic issue causing DMD, potentially leading to better muscle function. Although human study data remains limited, these early findings encourage those considering joining this trial, where participants will receive different dose levels of INS1201 based on their age group. ^{4 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for ambulatory male children with Duchenne Muscular Dystrophy (DMD). They must be between 2 to less than 5 years old, able to walk at least 10 meters without help, and have a specific genetic mutation in the DMD gene. Participants need to be up-to-date on vaccinations as per CDC guidelines.

Inclusion Criteria

I can walk 10 meters without help or devices.

I am up to date with all vaccinations recommended for my age and condition.

I am a boy aged 3 to <5 years (Part 1) or 2 to <3 years (Part 2), at the time my legal guardian consented.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intrathecal injection of INS1201

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• INS1201

Trial Overview The study is testing the safety of a single intrathecal injection of INS1201 in boys with DMD. The main goal is to see how well participants tolerate this dose and how the drug spreads within their bodies.

How Is the Trial Designed?

Group I: Part 2: Cohort 4 Experimental Treatment 1 Intervention

Participants aged 2 to \<3 years will receive a single dose level 2 of INS1201 by IT injection on Day 1.

Group II: Part 2: Cohort 3 Experimental Treatment 1 Intervention

Participants aged 2 to \<3 years will receive a single dose level 1 of INS1201 by IT injection on Day 1.

Group III: Part 1: Cohort 2 Experimental Treatment 1 Intervention

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

Group IV: Part 1: Cohort 1 Experimental Treatment 1 Intervention

Participants aged 3 to \<5 years will receive a single dose level 1 of INS1201 by IT injection on Day 1.

Who Is Running the Clinical Trial?

Insmed Gene Therapy LLC

Lead Sponsor

Trials

1

Recruited

10+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06817382

NCT06817382 | A Study to Investigate the Safety and ... 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 ...

2. parentprojectmd.org parentprojectmd.org/clinical-trial/a-study-to-investigate-the-safety-and-biodistribution-of-a-single-intrathecal-it-injection-of-ins1201-in-ambulatory-males-with-duchenne-muscular-dystrophy-dmd/

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

3. pharmaceutical-technology.com pharmaceutical-technology.com/analyst-comment/insmed-gene-therapy-dmd-challenge-landscape/

Insmed's gene therapy poised to challenge DMD ... Insmed is set to challenge the Duchenne muscular dystrophy (DMD) treatment space with its innovative gene therapy asset, INS1201.

4. nature.com nature.com/articles/s41434-025-00561-6

AAV microdystrophin gene replacement therapy for ... Further data from the NCT03362502 study reported in March 2021 demonstrated both efficacy and an acceptable safety profile [129]. At that stage, ...

5. bioworld.com bioworld.com/articles/718450-insmed-reports-preclinical-data-on-ins-1201-for-dmd

Insmed reports preclinical data on INS-1201 for DMD INS-1201 treatment also reduced DMD disease pathology and improved forelimb and hindlimb muscle strength and physiology, suggesting that the ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06817382

NCT06817382 | A Study to Investigate the Safety and ... 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 ...

7. ascenddmd.com ascenddmd.com/

ASCEND Study - Insmed | Ascend DMD ASCEND is a first-in-human experimental research study designed to test the safety of INS1201. · The study will include boys aged 2 to <5 years · INS1201 is given ...

8. scge.mcw.edu scge.mcw.edu/platform/data/clinicalTrials/report/NCT06817382

Gene Therapy Trial Report 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 ...