Philadelphia, PANCT06890520Now EnrollingIRB Ready

Primary Mitochondrial Disease Clinical Trial in Philadelphia, PA

Access cutting-edge primary mitochondrial disease treatment through this clinical trial at a research site in Philadelphia. 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 Philadelphia

Access primary mitochondrial disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related primary mitochondrial disease treatment provided free

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Check if you qualify for this primary mitochondrial disease clinical trial in Philadelphia, PA

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

Why Participate?

  • No-Cost Study Care

  • Local to Philadelphia

    Convenient for PA 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 Philadelphia site if eligible
  4. 4Begin participation

About This Primary Mitochondrial Disease Study in Philadelphia

Primary Mitochondrial Disease (PMD) is a genetic neurometabolic disorder, leading to central nervous system degeneration and increased risk of early mortality. There is a strong link between the pathophysiology of mitochondrial disease and biomarkers related to the biochemistry of redox imbalance, involving the levels of glutathione. Investigators will use Magnetic Resonance Imaging and Spectroscopy to non-invasively measure glutathione and other chemicals in the brain to identify redox imbalance in patients with PMD.

Sponsor: Children's Hospital of Philadelphia

Who Can Participate

Inclusion Criteria

Must be between 8 and 75 years, inclusive
Genetically confirmed primary mitochondrial disease
Receiving standard-of-care treatment including mitochondrial supplements that may include N-acetylcysteine (NAC), a precursor of glutathione Inclusion Criteria for Healthy Controls:
Must be between 8 and 75 years, inclusive

Exclusion Criteria

MRI contraindications
In the investigator's opinion, inability to fully comply with research procedures
Active self-reported alcohol and/or substance abuse, including tobacco-use
A pacemaker; any metal-based medical or non-medical devices/implants; any non-removable metal-based object (e.g., body piercings, jewelry, etc.) that cannot be cleared through radiologic evaluation
Any history of intraocular injury or fragment in or around the orbit that cannot be cleared through radiologic evaluation
Any history of bullet, shrapnel, or stabbing wounds that cannot be cleared through radiologic evaluation
Past or current employment involving (or exposure to) a metal grinder (e.g., at a construction worksite)
At the discretion of the principal investigator (PI), any medical condition that will interfere with or prevent the safe completion of the study
Any female participant with childbearing potential who is knowingly pregnant or suspects that she is pregnant will be removed from the study. (Although there are no known risks of MRI on pregnant females or fetuses, there is a possibility of yet undiscovered pregnancy-related risks. Since there is no direct benefit from participating in this protocol for pregnant females, they will be excluded to ensure their long-term safety and that of their unborn fetus.)
To note, for this protocol, participants are instructed to lie still in the MRI scanner; there is no contrast or sedation. Participants who do not possess the cognitive and / or physical abilities to perform these procedures will not be included.

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 Philadelphia?

Yes, this clinical trial (NCT06890520) has an active research site in Philadelphia, PA 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.

Primary Mitochondrial Disease Treatment Options in Philadelphia, PA

If you're searching for primary mitochondrial disease treatment options in Philadelphia, PA, this clinical trial (NCT06890520) 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 Philadelphia research site is actively enrolling participants for this clinical trial. You'll receive care from experienced primary mitochondrial disease 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 primary mitochondrial disease clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Philadelphia, PA