Columbus, OHNCT06817382Now EnrollingIRB Ready

Duchenne Muscular Dystrophy Clinical Trial in Columbus, OH

Access cutting-edge duchenne muscular dystrophy treatment through this clinical trial at a research site in Columbus. Study-provided care at no cost to qualified participants.

Sponsored by Insmed Gene Therapy LLC

Quick Self-Assessment

See if you qualify for this Columbus location

Preparing your pre-screening questions…

Expert Care in Columbus

Access duchenne muscular dystrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related duchenne muscular dystrophy treatment provided free

Apply for This Columbus Location

Check if you qualify for this duchenne muscular dystrophy clinical trial in Columbus, OH

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Columbus

    Convenient for OH residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Columbus site if eligible
  4. 4Begin participation

About This Duchenne Muscular Dystrophy Study in Columbus

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.

Sponsor: Insmed Gene Therapy LLC

Who Can Participate

Inclusion Criteria

Participant must be male at birth, 3 to \<5 years of age, inclusive (Part 1) and 2 to \<3 years of age (Part 2), at the time of legally authorized representative (LAR) signing and dating the informed consent form.
Ambulatory -as defined as the ability to walk at least 10 meters unassisted (ie, without personal assistance or use of any assistive devices) Note: children who have not yet developed the ability to walk by the time of screening (for whatever reason) will not be eligible for the study.
Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not have to repeat genetic testing for the diagnosis of DMD if one is already on file. Genetic reports must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein (mutations in exons 1-17 or 59-71 are therefore not permitted).
Able to cooperate with motor assessment testing.
Has received vaccinations recommended for the participant's age and DMD disease according to Centers for Disease Control and Prevention (CDC) Child and Adolescent Immunization Schedule by Age, World Health Organization, or local recommendation incorporating the Advisory Committee on Immunization Practices (ACIP) Vaccine Recommendations and Guidelines for Patients with Altered Immunocompetence. Exception is made for seasonal influenza and coronavirus disease 2019 (COVID-19) vaccines, for which shared decision-making with the participant's physician is encouraged.

Exclusion Criteria

Prior treatment with gene or cell-based therapy at any time.
Oligonucleotide-based exon skipping or small molecule stop codon readthrough-promoting therapies for at least 6 months prior to enrolment.
Has left ventricular ejection fraction \< 50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy.
Has cardiac arrhythmia or significant electrocardiogram (ECG) interval abnormalities.
Major surgery within 3 months prior to Day 1 or planned surgery or procedures that would interfere with the conduct of the study at any time during this study.
The presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic/allergic, behavioural disease, infection, unhealed injury, malignancy, concomitant illness, extenuating circumstance, or requirement for chronic drug treatment that, in the opinion of the Investigator:
Creates unnecessary risks for undergoing gene transfer;
Might compromise the participant's ability to comply with the protocol-required testing or procedures; or
Might compromise the participant's well-being, safety, or clinical interpretability.
Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
Has signs of clinically significant symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
Has contraindications for IT administration of the product or for lumbar puncture, such as anatomical abnormalities, bleeding disorders or other medical conditions (eg, spina bifida, meningitis, or significant clotting abnormalities).
Demonstrates cognitive or developmental delay or impairment that could confound assessment of motor development in the opinion of the Investigator.
Total serum anti-AAV9 antibody titers of \> 1:50 as determined by ELISA within 14 days of Day 1. Note: Other inclusion/exclusion criteria may apply.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Columbus?

Yes, this clinical trial (NCT06817382) has an active research site in Columbus, OH that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Duchenne Muscular Dystrophy Treatment Options in Columbus, OH

If you're searching for duchenne muscular dystrophy treatment options in Columbus, OH, this clinical trial (NCT06817382) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Columbus research site is actively enrolling participants for this clinical trial. You'll receive care from experienced duchenne muscular dystrophy specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all duchenne muscular dystrophy clinical trials near you to find additional studies recruiting in your area.

More Ulcerative Colitis Trials in Columbus, OH

See all ulcerative colitis clinical trials recruiting in Columbus — not just this study.

Browse Ulcerative Colitis Trials in Columbus →

Browse More Trials by Condition

Ulcerative Colitis TrialsPulmonary Hypertension TrialsDuchenne Muscular Dystrophy TrialsAll Conditions →

Ready to Join in Columbus?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Columbus, OH