New York, NYNCT02316314Now EnrollingIRB Ready

Friedreich's Ataxia Clinical Trial in New York, NY

Access cutting-edge friedreich's ataxia treatment through this clinical trial at a research site in New York. Study-provided care at no cost to qualified participants.

Sponsored by Weill Medical College of Cornell University

Quick Self-Assessment

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Expert Care in New York

Access friedreich's ataxia specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related friedreich's ataxia treatment provided free

Apply for This New York Location

Check if you qualify for this friedreich's ataxia clinical trial in New York, NY

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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 New York

    Convenient for NY 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 New York site if eligible
  4. 4Begin participation

About This Friedreich's Ataxia Study in New York

Friedreich's ataxia (FRDA) is an autosomal recessive disease characterized by loss of coordination and cardiomyopathy. It is the most common form of inherited ataxia with an incidence in 1/50,000 in the Caucasian population. FRDA is associated with progressive damage to the nervous system, resulting in symptoms ranging from gait disturbance to speech problems, as well as diabetes and heart disease. The heart disease manifests as cardiomyopathy, and is responsible for approximately 60% of deaths from FRDA. This study is designed to characterize the cardiac manifestations of the disease using exercise, MRI, ECHO and serum parameters, in the context of the neurological disease. In addition, this study will demonstrate that corneal confocal microscopy (CCM) may also provide a biomarker for FRDA.

Sponsor: Weill Medical College of Cornell University

Who Can Participate

Inclusion Criteria

Males and females, age 12 to 50
Willing and able to provide informed consent (adolescents will need to provide assent and a parent to provide consent)
Definitive diagnosis of FRDA, based on clinical phenotype and genotype
Left ventricle ejection fraction measured by ECHO of \>35% (If results of an ECHO are not available for a potential subject, then an ECHO will first be performed and subjects with an LVEF \<35% will not be required to perform the CPET) Friedreich's Ataxia -

Exclusion Criteria

Signs and symptoms of cardiac failure
Moderate to severe atrial or ventricular arrythmias
History of angina pectoris
Implanted pacemaker and/ or defibrillator or any other device that would preclude MRI assessment
Any form of dialysis; Severe or end-stage CKD (CKD 4 or 5, eGFR \< 30 ml/min/1.73 m2) without dialysis; eGFR 30 to 40 ml/min/1.73 m2 without dialysis; Acute kidney injury (If a recent assessment is not available, then a blood test to assess kidney function will be performed prior to cardiac MRI)
Females who are pregnant
Receipt of an investigational drug within 30 days or 5 half-lives, whichever is longer, prior to screening, or active enrollment in an investigational medication or device study
Unable to undergo cardiac MRI with gadolinium contrast or claustrophobia
Clinical history or evidence of Type 1 or Type 2 Diabetes mellitus
Any condition, disorder, or abnormal laboratory test findings at screening which, in the judgment of the investigator, would interfere with the individual's ability to comply with all study requirements, or would require the administration of treatment during the study that could potentially affect the interpretation of the study data, or would place the individual at an unacceptable risk by his/ her participation in the study Normal controls - Inclusion Criteria:
Males and females, age 12 to 30
Willing and able to provide informed consent (Adolescents will need to provide assent and a parent to provide consent)
Matched age, gender and ethnicity to the FRDA group
Capable of undergoing the various modalities of cardiac assessment
Left ventricle ejection fraction measured by ECHO of \>35% (If results of an ECHO are not available for a potential subject, then an ECHO will first be performed and subjects with an LVEF \<35% will be withdrawn from the study) Normal controls - Exclusion Criteria:
Individuals not deemed in good overall health by the investigator will not be accepted into the study
Signs and symptoms of cardiac failure
Moderate to severe atrial or ventricular arrhythmias
History of angina pectoris
Implanted pacemaker and/ or defibrillator or any other device that would preclude MRI assessment
Any form of dialysis; Severe or end-stage CKD (CKD 4 or 5, eGFR \< 30 ml/min/1.73 m2) without dialysis; eGFR 30 to 40 ml/min/1.73 m2 without dialysis; Acute kidney injury (If a recent assessment is not available, then a blood test to assess kidney function will be performed prior to cardiac MRI)
Females who are pregnant or lactating
Receipt of an investigational drug within 30 days or 5 half-lives, whichever is longer, prior to screening, or active enrollment in an investigational medication or device study
Unable to sit with back support
Unable to undergo cardiac MRI with gadolinium contrast or claustrophobia
Unable to undergo exercise tests
Clinical history or evidence of Type 1 or Type 2 Diabetes mellitus
Any condition, disorder, or abnormal laboratory test findings at screening which, in the judgment of the investigator, would interfere with the individual's ability to comply with all study requirements, or would require the administration of treatment during the study that could potentially affect the interpretation of the study data, or would place the individual at an unacceptable risk by his/ her participation in the study

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 New York?

Yes, this clinical trial (NCT02316314) has an active research site in New York, NY 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.

Friedreich's Ataxia Treatment Options in New York, NY

If you're searching for friedreich's ataxia treatment options in New York, NY, this clinical trial (NCT02316314) 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 New York research site is actively enrolling participants for this clinical trial. You'll receive care from experienced friedreich's ataxia 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 friedreich's ataxia clinical trials near you to find additional studies recruiting in your area.

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