Los Angeles, CANCT03047369Now EnrollingIRB Ready

Leukodystrophy Clinical Trial in Los Angeles, CA

Access cutting-edge leukodystrophy treatment through this clinical trial at a research site in Los Angeles. Study-provided care at no cost to qualified participants.

Sponsored by Children's Hospital of Philadelphia

Quick Self-Assessment

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Expert Care in Los Angeles

Access leukodystrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related leukodystrophy treatment provided free

Apply for This Los Angeles Location

Check if you qualify for this leukodystrophy clinical trial in Los Angeles, CA

Secure & Confidential

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

Why Participate?

  • No-Cost Study Care

  • Local to Los Angeles

    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 Los Angeles site if eligible
  4. 4Begin participation

About This Leukodystrophy Study in Los Angeles

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

Sponsor: Children's Hospital of Philadelphia

Who Can Participate

Inclusion Criteria

(Affected Subjects):
Male or female of any age;
Suspected or confirmed diagnosis of leukodystrophy or other disorder affecting the white matter of the brain based primarily on the finding of central nervous system neuroimaging consistent with this diagnosis or on an existing diagnosis of a leukodystrophy or genetic leukoencephalopathy as defined in existing classification systems, or in the presence of variant(s) of uncertain significance or genotype consistent with leukodytrophy;
Documentation of informed consent by the subject, parent, or legal guardian, and, if appropriate, documentation of assent;
Willingness to provide clinical data, participate in standardized assessments, and/or provide biologic samples.

Exclusion Criteria

(Affected Subjects)
Established diagnosis at the time of referral that is not consistent with a genetic disorder of the white matter, such as an acquired demyelinating condition (e.g. multiple sclerosis), or an infectious etiology, with the exception of sequelae of congenital infections such as CMV;
Inability to provide consent. Inclusion Criteria (Healthy Controls)
Male or female of any age;
Individuals with no confirmed or suspected diagnosis of leukodystrophy or other disorder affecting the white matter of the brain (including affected patients' caregivers);
Documentation of informed consent by the subject, parent, or legal guardian, and, if appropriate, documentation of assent. Exclusion Criteria (Healthy Controls) \- Inability to provide consent.

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 Los Angeles?

Yes, this clinical trial (NCT03047369) has an active research site in Los Angeles, 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.

Leukodystrophy Treatment Options in Los Angeles, CA

If you're searching for leukodystrophy treatment options in Los Angeles, CA, this clinical trial (NCT03047369) 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 Los Angeles research site is actively enrolling participants for this clinical trial. You'll receive care from experienced leukodystrophy 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 leukodystrophy clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Los Angeles, CA