Is NCT05302271 recruiting in New York, NY?

Yes, NCT05302271 is currently recruiting participants at its New York, NY location. This friedreich ataxia clinical trial is actively enrolling qualified participants.

How do I join the friedreich ataxia clinical trial in New York?

To join this clinical trial in New York, 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 New York 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.

Friedreich Ataxia Clinical Trial in New York, NY

About This Friedreich Ataxia Study in New York

The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). AAVrh.10hFXN is a serotype rh.10 adeno-associated virus gene transfer vector coding for Frataxin (FXN). The drug is administered intravenously. This is a phase 1, open label, dose escalation study with a total of 25 participants.

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

Definitive diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on both alleles)

\>600 GAA repeats in intron 1 in at least one allele

FARS and SARA neurologic scores consistent with diagnosis of Friedreich's ataxia

Left ventricle ejection fraction (EF) measured by cardiac MRI of ≥35% to 75%

Evidence of FA-related cardiac disease, must meet the following criteria: must be abnormal in ≥2 of the following parameters, at least one of which is an abnormal cardiac MRI left ventricular mass index or abnormal cardiopulmonary exercise test

Adults: In the absence of other factors known to cause left ventricular hypertrophy, cardiac MRI left ventricular mass index \>2 standard deviations above the normal range (males \>84 gm/m2, females \>69 gm/m2 or Pediatrics: In the absence of other factors known to cause left ventricular hypertrophy, cardiac MRI left ventricular mass index \>95th centile based on normal BSA for their age and gender

Cardiopulmonary arm crank testing with assessment of VO2 max ≤20 mL/kg-min, peak VO2 ≥10 mL/kg-min while maintaining revolutions of ≥40/min. To insure consistency of effort, peak RER ≥1.0

Cardiac MRI stroke volume index \<45 mL/m2

Cardiac MRI global longitudinal left ventricular strain \<20%

Serum high-sensitivity cardiac troponin above the normal range

Fibrosis ≤10% in the left ventricular wall on late gadolinium enhancement cardiac MRI

Resting O2 saturation ≥95%

Serum neutralizing anti-AAVrh.10 titer \<1:125

Hematocrit \>30%

White blood cell levels within normal limits

Normal prothrombin, partial thromboplastin time

Normal liver-related serum parameters (ALT, AST, ALP, bilirubin); normal liver ultrasound and serum alpha fetoprotein

Normal kidney function as assessed by plasma urea and creatinine; estimated GFR \>30 mL/min/1.73m2

No evidence of active infection of any types, including hepatitis virus (A, B or C), human immunodeficiency virus (HIV-1 and HIV-2), or SARS-CoV2

Fertile individuals should utilize barrier birth control measures to prevent pregnancy for up to 6 months after vector administration

Individuals not receiving experimental medications or participating in another experimental protocol for at least 12 wk prior to entry to the study (individuals who are/have received approved therapy will be included).

Capable of undergoing cardiac MRI

No contraindications to receiving corticosteroid immunosuppression

Exclusion Criteria

Individuals receiving corticosteroids or other immunosuppressive medications

Individuals with uncontrolled diabetes (glycated hemoglobin, HbA1c levels \>7%)

Genotype FA missense mutation on one or both alleles

Evidence of infection defined by elevated white blood cell count, temperature \>38.5̊ C, infiltrate on chest x-ray

Decompensated heart failure (NY4A class III-IV at time of baseline clinical assessment)

Hemoglobin \<10 g/dl

Absolute neutrophil count \<1500 cells/mm3

Platelet count \<100,000 cells/mm3

Hemodynamically unstable atrial or ventricular arrhythmias which require medical intervention

Contraindication to cardiac MRI (e.g., non-MRI compatible pacemaker/defibrillator) or gadolinium (known or suspected hypersensitivity, glomerular filtration rate \<30 mL/min/1.73m2)

Any malignancy during the last five years, except basal cell skin cancer

Unrelated clinical condition with life expectancy \<12 months (prohibiting follow-up)

Concomitant conditions (other than FA) known to produce left ventricular hypertrophy, including aortic stenosis, systemic hypertension (BP ≥140/90 on noninvasive blood pressure), or genetically mediated hypertrophic cardiomyopathy

Use of oxygen supplementation

Risk for thromboembolic disease, including history of thromboembolic disease hospitalization within the last 90 days, recent trauma and/or recent surgical procedure. If the history of thromboembolic disease is not definitive, the subject will be excluded if laboratory testing suggests a risk for thromboembolic disease because of mutations in the protein-S, protein C, antithrombin, factor V Leiden or prothrombin gene

Any uncontrolled psychiatric disease

Pregnant or breastfeeding woman

Prior participation in any gene and/or cell therapy

Known obstructive coronary artery disease (as documented by clinical history of myocardial infarction, prior coronary revascularization or angina symptoms (Canadian Cardiovascular Society grade ≥2 at time of baseline clinical assessment), or epicardial obstructive coronary artery disease (≥ 50% left main, ≥ 70% of other major coronary arteries)

Any lung function abnormalities that would affect cardiopulmonary testing

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

If prior infection with SARS-CoV2, any related residual cardiac or pulmonary abnormalities

Alcoholism or drug addiction (see reference 71 for alcoholism, reference 72 for drug addiction)

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 (NCT05302271) 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 Ataxia Treatment Options in New York, NY

If you're searching for friedreich ataxia treatment options in New York, NY, this clinical trial (NCT05302271) 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 phase 1 clinical trial. You'll receive care from experienced friedreich 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 ataxia clinical trials near you to find additional studies recruiting in your area.

Friedreich Ataxia Clinical Trial in New York, NY

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IRB Approved

No-Cost Care

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