Philadelphia, PANCT07180355Now EnrollingIRB Ready

Friedreich's Ataxia (FA) Clinical Trial in Philadelphia, PA

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

Sponsored by Solid Biosciences Inc.

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Expert Care in Philadelphia

Access friedreich's ataxia (fa) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related friedreich's ataxia (fa) treatment provided free

Apply for This Philadelphia Location

Check if you qualify for this friedreich's ataxia (fa) 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 Friedreich's Ataxia (FA) Study in Philadelphia

This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA. All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.

Sponsor: Solid Biosciences Inc.

Who Can Participate

Inclusion Criteria

Has history of FA symptom onset ≤25 years of age
Has a clinical and genetic diagnosis of FA
Has a staging score of ≥1 but \<6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score
Is willing to agree to the following rules for use of omaveloxolone (Skyclarys):
For a candidate who is currently taking omaveloxolone, has been on a stable dose for 12 weeks, expects to continue taking omaveloxolone at that dose throughout the study, and is willing to stop taking omaveloxolone at the direction of the Investigator or Sponsor's Medical Monitor if evidence of transaminitis or synthetic liver dysfunction is detected during the study
For a candidate who is not actively taking omaveloxolone, at least 12 weeks have passed since the last dose and the candidate agrees not to resume omaveloxolone during the 18-month period after SGT-212 infusion NOTE: The use of any other approved or investigational medicinal product for the treatment of FA should be discussed with the study team.

Exclusion Criteria

Antibodies against adeno-associated virus serotype 9 (AAV9)
Has a modified FARS (mFARS) score \<20
Has a body weight ≤25 kilogram (kg) or has body mass index (BMI) ≥33 kg/m\^2
Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization
Is unable to undergo cardiac and brain MRI with contrast, including hypersensitivity to gadolinium contrast agent, presence of a non-MRI-compatible cardiac pacemaker, presence of a non-MRI-compatible implantable cardiac defibrillator, or physical condition (e.g., contractures)
Has uncontrolled diabetes as defined by a hemoglobin (Hb) A1c \>9%
Has participated in recent interventional clinical studies or received any investigational therapy administered within 3 months or 5 half-lives (whichever is longer) prior to Screening
Has received gene therapy at any time
Has contraindications to receiving corticosteroids
Has any contraindication to the surgical procedures involved with IDN infusion of SGT-212
Has any known cardiac disease not related to FA including known obstructive coronary artery disease (CAD)
Other Inclusion/Exclusion criteria to be applied as per protocol.

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 (NCT07180355) 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.

Friedreich's Ataxia (FA) Treatment Options in Philadelphia, PA

If you're searching for friedreich's ataxia (fa) treatment options in Philadelphia, PA, this clinical trial (NCT07180355) 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 friedreich's ataxia (fa) 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 (fa) clinical trials near you to find additional studies recruiting in your area.

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