Stanford, CANCT06503367Now EnrollingIRB Ready

LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\) Clinical Trial in Stanford, CA

Access cutting-edge lama2-md \(merosin deficient congenital muscular dystrophy, mdc1a\) treatment through this clinical trial at a research site in Stanford. Study-provided care at no cost to qualified participants.

Sponsored by Nationwide Children's Hospital

Quick Self-Assessment

See if you qualify for this Stanford location

Preparing your pre-screening questions…

Expert Care in Stanford

Access lama2-md \(merosin deficient congenital muscular dystrophy, mdc1a\) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related lama2-md \(merosin deficient congenital muscular dystrophy, mdc1a\) treatment provided free

Apply for This Stanford Location

Check if you qualify for this lama2-md \(merosin deficient congenital muscular dystrophy, mdc1a\) clinical trial in Stanford, CA

Secure & Confidential

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

Why Participate?

  • No-Cost Study Care

  • Local to Stanford

    Convenient for CA 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 Stanford site if eligible
  4. 4Begin participation

About This LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\) Study in Stanford

The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems. Participants will undergo: * Neuromuscular assessments * Blood collections * Swallowing and breathing assessments * Questionnaires

Sponsor: Nationwide Children's Hospital

Who Can Participate

Inclusion Criteria

Signed informed consent by the subject, parent(s) or legally authorized representative (LAR) and/or assent by the subject (when applicable).
Subject must be aged birth to less than 5.0 years of age at time of consent.
A confirmed diagnosis of LAMA2-RD confirmed via: a: Two pathogenic variants in the LAMA2 gene (via a CLIA-approved laboratory) or: b. muscle biopsy with absence of merosin (laminin-211) and at least one pathogenic variant in the LAMA2 gene
Absence of another confirmed genetic disease.
Willingness to maintain current exercise and/or physical therapy regimen for the duration of the clinical study.
Willingness to comply with the study protocol, including but not limited to, all study procedures and visits.

Exclusion Criteria

Acute medical illness or hospitalization within 30 days prior to informed consent.
Participation in a previous trial of any investigational agent for LAMA2-RD within 1 month prior to informed consent, or use of any other investigational therapy (including off-label use of Losartan) within 30 days prior to informed consent, or participation in other clinical studies, within 30 days (or 3 half-lives, whichever is longer) prior to informed consent, which in the opinion of the PI, may potentially confound results from this study.
Other significant medical condition, which in the opinion of the site Principal Investigator may confound interpretation of the clinical course of LAMA2- RD.

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 Stanford?

Yes, this clinical trial (NCT06503367) has an active research site in Stanford, CA 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.

LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\) Treatment Options in Stanford, CA

If you're searching for lama2-md \(merosin deficient congenital muscular dystrophy, mdc1a\) treatment options in Stanford, CA, this clinical trial (NCT06503367) 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 Stanford research site is actively enrolling participants for this clinical trial. You'll receive care from experienced lama2-md \(merosin deficient congenital muscular dystrophy, mdc1a\) 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 lama2-md \(merosin deficient congenital muscular dystrophy, mdc1a\) clinical trials near you to find additional studies recruiting in your area.

More Pulmonary Hypertension Trials in Stanford, CA

See all pulmonary hypertension clinical trials recruiting in Stanford — not just this study.

Browse Pulmonary Hypertension Trials in Stanford →

Browse More Trials by Condition

Pulmonary Hypertension TrialsInterstitial Cystitis TrialsDuchenne Muscular Dystrophy TrialsMyalgic Encephalomyelitis TrialsAll Conditions →

Ready to Join in Stanford?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Stanford, CA