Spinal Muscular Atrophy Trial, Recruiting (NCT04292574)

Spinal Muscular Atrophy Trial, Recruiting NCT04292574 Sponsor: Newcastle University Condition: Spinal Muscular Atrophy

Printed from Voxsanity (voxsanity.com.au) — NCT04292574 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Spinal Muscular Atrophy

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:

Anyone who has been diagnosed with SMA (Spinal Muscular Atrophy), or is currently waiting for a diagnosis to be confirmed, may be eligible.
Diagnosis of SMA needs to be confirmed through genetic testing results (or may still be pending at the time of joining).

Who may not be able to join:

There are no specific reasons listed that would prevent someone from joining this registry.

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: Chiara Marini-Bettolo, MD, PhD, Newcastle University

Phone: 0191 2418640

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: NCT04292574

Sponsor: Newcastle University

Phase: Not Applicable

Status: Recruiting

Condition: Spinal Muscular Atrophy

Trial contact: 0191 2418640

More information: https://clinicaltrials.gov/study/NCT04292574

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:

Anyone who has been diagnosed with SMA (Spinal Muscular Atrophy), or is currently waiting for a diagnosis to be confirmed, may be eligible.
Diagnosis of SMA needs to be confirmed through genetic testing results (or may still be pending at the time of joining).

Who may not be able to join:

There are no specific reasons listed that would prevent someone from joining this registry.

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

Newcastle University

Registry

ClinicalTrials.gov

Start date

13 July 2008

Est. completion

31 May 2025

Where this trial is recruiting

🇬🇧 United Kingdom

Primary endpoints

Patient questionnaire

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