Is NCT04086329 recruiting in Philadelphia, PA?

Yes, NCT04086329 is currently recruiting participants at its Philadelphia, PA location. This mitochondrial myopathies clinical trial is actively enrolling qualified participants.

How do I join the mitochondrial myopathies clinical trial in Philadelphia?

To join this clinical trial in Philadelphia, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this Philadelphia clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Mitochondrial Myopathies Clinical Trial in Philadelphia, PA

About This Mitochondrial Myopathies Study in Philadelphia

Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomarkers in mitochondrial myopathy (MM). There is currently a growing number of potential new treatments to be tested through MM clinical intervention trials, which has created a pressing need for quantitative biomarkers that reliably reflect MM disease severity, progression, and therapeutic response. The purpose of the study is to measure the efficacy of an electrochemical oxygen nanosensor to measure in vivo mitochondrial function in human muscle tissue, and its ability to discriminate MM patients from healthy volunteers. The data and results from this nanosensor study may contribute to current and future research, including improved diagnostic and therapeutic approaches for patients with mitochondrial disease.

Sponsor: Children's Hospital of Philadelphia

Who Can Participate

Inclusion Criteria

for Healthy Controls

Males and females, between the ages of 18 and 65 years, inclusive

Provide informed consent for study participation; able to understand and complete the protocol

Able to ambulate independently

Able to perform bicycle ergometry Inclusion Criteria for MM Cases

Males and females, between the ages of 18 and 65 years, inclusive

Provide informed consent for study participation; able to understand and complete the protocol

Genetically-confirmed MM as defined by a diagnosis of primary mitochondrial disease (PMD) with predominant symptoms of myopathy as expressed by exercise intolerance and muscle weakness and fatigue.

Previously enrolled (or will enroll) in Children's Hospital of Philadelphia (CHOP) Institutional Review Board (IRB) study #08-006177 (Falk, PI) or CHOP IRB #16-013364 (Zolkipli, PI)

Able to ambulate independently

Able to perform bicycle ergometry

Exclusion Criteria

for All Participants Subjects will be excluded if any of the following apply:

Unable to provide informed consent and complete all study procedures, including ergometry

Non-ambulatory or unable to ambulate independently

Within 1 month of a recent hospital admission due to acute illness

Have severe cardiac disease as defined by an ejection fraction of less than 35% and New York Heart Association Functional Classification Class III; or severe pulmonary disease as defined by the need for supplemental O2 therapy or daytime ventilatory support

Have a tracheostomy

Have a known bleeding disorder and/or family history (first-degree relative) with a known bleeding disorder

Daily intake of aspirin or any other anti-platelet therapy which cannot be temporarily discontinued for medical reasons

a) Have known or suspected congenital or acquired immune deficiency; b) concurrent use of immunosuppressive drugs, including corticosteroids; c) past history of recurrent (more than 6 times per year) severe (required hospitalization) skin or soft tissue infections; d) history of infection or delayed wound healing after surgery or biopsy; e) known history of neutropenia with absolute neutrophil count less than 500/mm3

Undergo chronic steroid treatment as defined by daily oral intake (for more than 1 month) or have existing untreated endocrinopathies, such as hypothyroidism that caused acquired myopathy

Prone to hypertrophic scars and keloids

Have any other known inherited myopathy, such as Duchenne muscular dystrophy or congenital myopathy

Known allergy to lidocaine

Have a cognitive impairment that may prevent the ability to complete study procedures

Unable to comply with the requirements of the study protocol and/or unsuitable for the study for any reason, in the opinion of the principal investigator

Individuals from vulnerable populations (e.g., prisoners/detainees)

Participants who are unable to speak and/or read English (as participants will be required to be proficient to complete study procedures)

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 (NCT04086329) 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.

Mitochondrial Myopathies Treatment Options in Philadelphia, PA

If you're searching for mitochondrial myopathies treatment options in Philadelphia, PA, this clinical trial (NCT04086329) 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 phase 1 clinical trial. You'll receive care from experienced mitochondrial myopathies 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 mitochondrial myopathies clinical trials near you to find additional studies recruiting in your area.

Mitochondrial Myopathies Clinical Trial in Philadelphia, PA

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IRB Approved

No-Cost Care

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