Philadelphia, PANCT06953583Now EnrollingIRB Ready

Friedreich's Ataxia Clinical Trial in Philadelphia, PA

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

Sponsored by Biogen

Quick Self-Assessment

See if you qualify for this Philadelphia location

Preparing your pre-screening questions...

Expert Care in Philadelphia

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 Philadelphia Location

Check if you qualify for this friedreich's ataxia clinical trial in Philadelphia, PA

Secure & Confidential

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 Study in Philadelphia

In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15

Sponsor: Biogen

Who Can Participate

Inclusion Criteria

Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point mutations or deletions, or other non-GAA expansion mutations in the other allele.
Symptomatic for FA as reported by the participant and/or the parent/caregiver a. Children 7 to \< 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline Part 1 RCT: Key

Exclusion Criteria

Glycosylated hemoglobin A1C (HbA1c) \> 11%
B-type natriuretic peptide (BNP) \> 200 picograms per milliliter (pg/mL) at screening
Ejection fraction (EF) \< 40% \[based on echocardiogram (ECHO) performed at screening visit\]
Clinically significant cardiac disease except mild to moderate cardiomyopathy Part 2 OLE: Eligibility criteria:
Participants have completed Part 1 RCT of the study and no discontinuation criteria have

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 (NCT06953583) 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 Treatment Options in Philadelphia, PA

If you're searching for friedreich's ataxia treatment options in Philadelphia, PA, this clinical trial (NCT06953583) 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 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.

More Erectile Dysfunction Trials in Philadelphia, PA

See all erectile dysfunction clinical trials recruiting in Philadelphia — not just this study.

Browse Erectile Dysfunction Trials in Philadelphia

Ready to Join in Philadelphia?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Philadelphia, PA