Spinal Muscular Atrophy Trial, Recruiting (NCT07543003)

Spinal Muscular Atrophy Trial, Recruiting NCT07543003 Sponsor: University of Oxford Condition: Spinal Muscular Atrophy

Printed from Voxsanity (voxsanity.com.au) — NCT07543003 — 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:

For children with SMA (Test group):

Your child has been confirmed through genetic testing to have SMA, and the number of their SMN2 gene copies is known
Your child was identified through newborn screening and is receiving treatment for SMA, OR was diagnosed because a sibling has SMA, or was found another way
Your child has 4 or more copies of the SMN2 gene and is not currently receiving SMA treatment, OR has fewer than 4 copies of the SMN2 gene and is not currently receiving SMA treatment
Your child is between 4 months and 4 years old at the start of the study (children younger than 4 months may also be considered)
A parent or legal guardian is able to give written permission for the child to take part
The child can be a boy or a girl

For children without SMA (Control group):

Your child is typically developing with no SMA diagnosis
Your child is between 6 months and 4 years old at the time of joining
A parent or legal guardian is able to give written permission for the child to take part
The child can be a boy or a girl

Who may not be able to join:

Your child has any ongoing or long-term health condition that the study doctor feels would affect the assessments or their physical development (confirm with trial site)
Your child is currently taking part in another clinical research study involving an experimental treatment

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

Phone: 01865618799

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: NCT07543003

Sponsor: University of Oxford

Phase: Not Applicable

Status: Recruiting

Condition: Spinal Muscular Atrophy

Trial contact: 01865618799

More information: https://clinicaltrials.gov/study/NCT07543003

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:

For children with SMA (Test group):

Your child has been confirmed through genetic testing to have SMA, and the number of their SMN2 gene copies is known
Your child was identified through newborn screening and is receiving treatment for SMA, OR was diagnosed because a sibling has SMA, or was found another way
Your child has 4 or more copies of the SMN2 gene and is not currently receiving SMA treatment, OR has fewer than 4 copies of the SMN2 gene and is not currently receiving SMA treatment
Your child is between 4 months and 4 years old at the start of the study (children younger than 4 months may also be considered)
A parent or legal guardian is able to give written permission for the child to take part
The child can be a boy or a girl

For children without SMA (Control group):

Your child is typically developing with no SMA diagnosis
Your child is between 6 months and 4 years old at the time of joining
A parent or legal guardian is able to give written permission for the child to take part
The child can be a boy or a girl

Who may not be able to join:

Your child has any ongoing or long-term health condition that the study doctor feels would affect the assessments or their physical development (confirm with trial site)
Your child is currently taking part in another clinical research study involving an experimental treatment

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

University of Oxford

Registry

ClinicalTrials.gov

Start date

1 May 2026

Est. completion

1 July 2029

Where this trial is recruiting

🇬🇧 United Kingdom

Primary endpoints

To validate clinical outcome measures in patients with SMA from 4 months of age.

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