Rare Disease Trial, Recruiting (NCT06016946)

Rare Disease Trial, Recruiting NCT06016946 Sponsor: Friedreich's Ataxia Research Alliance Condition: Rare Disease

Printed from Voxsanity (voxsanity.com.au) — NCT06016946 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

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Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

People of any age, whether male or female, may be eligible
People who have been diagnosed with Friedreich's ataxia confirmed by genetic testing
People who are able and willing to sign a consent form agreeing to take part
For participants who are under 18, both the young person and their parent or legal guardian must sign the consent form
For participants who are not legally able to make their own decisions, their legal representative must sign the consent form on their behalf

Who may not be able to join:

People who are unable or unwilling to give their consent to take part in the study
People who have a current or ongoing health condition or other situation that could interfere with the study or its assessments (confirm with trial site)
People who, for any reason, the study doctor believes would be unlikely or unable to follow the study's requirements

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 28 July 2026

This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Principal Investigator: David Lynch, MD, Children's Hospital of Philadelphia

Phone: 16513291892

Australian sites

Murdoch Childrens Research Institute, Parkville, Victoria
61 8341 6374

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT06016946

Sponsor: Friedreich's Ataxia Research Alliance

Phase: Not Applicable

Status: Recruiting

Condition: Rare Disease

Trial contact: 16513291892

More information: https://clinicaltrials.gov/study/NCT06016946

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

People of any age, whether male or female, may be eligible
People who have been diagnosed with Friedreich's ataxia confirmed by genetic testing
People who are able and willing to sign a consent form agreeing to take part
For participants who are under 18, both the young person and their parent or legal guardian must sign the consent form
For participants who are not legally able to make their own decisions, their legal representative must sign the consent form on their behalf

Who may not be able to join:

People who are unable or unwilling to give their consent to take part in the study
People who have a current or ongoing health condition or other situation that could interfere with the study or its assessments (confirm with trial site)
People who, for any reason, the study doctor believes would be unlikely or unable to follow the study's requirements

Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Recruiting

Phase

Not Applicable

Sponsor

Friedreich's Ataxia Research Alliance

Registry

ClinicalTrials.gov

Start date

28 June 2023

Est. completion

28 January 2048

Where this trial is recruiting

🇦🇺 Australia 🇦🇹 Austria 🇧🇪 Belgium 🇧🇷 Brazil 🇨🇦 Canada 🇨🇿 Czechia 🇫🇷 France 🇩🇪 Germany 🇬🇷 Greece 🇮🇳 India 🇮🇪 Ireland 🇮🇹 Italy 🇳🇱 Netherlands 🇳🇿 New Zealand 🇪🇸 Spain 🇬🇧 United Kingdom 🇺🇸 United States

1 site(s) in Australia. Confirm current status and contact details directly with the trial site.

Primary endpoints

Change in modified Friedreich Ataxia Rating Scale (mFARS) Score; Change in Scale for the Assessment and Rating of Ataxia (SARA) Score; Change in FA Activities of Daily Living (ADL) Score; Change in Upright Stability (US) Score

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov