Spinal Muscular Atrophy Trial, Recruiting (NCT04177134)

Spinal Muscular Atrophy Trial, Recruiting NCT04177134 Sponsor: Assistance Publique - Hôpitaux de Paris Condition: Spinal Muscular Atrophy

Printed from Voxsanity (voxsanity.com.au) — NCT04177134 — 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:

You have been diagnosed with Spinal Muscular Atrophy (SMA) caused by a change in the 5q gene (types 1 to 4), confirmed by a genetic test.
You have been treated at a specialist SMA centre in France between September 1, 2016 and August 31, 2024.
For the part of the study looking forward in time: you have signed a consent form agreeing to take part.
For the part of the study looking at past records: you have been given an information form and have not objected to your data being used.
You are covered by health insurance.

Who may not be able to join:

You have a different type of SMA that is not caused by the 5q gene change.
You are currently under a legal guardianship or curatorship arrangement (meaning someone else has been appointed by a court to make decisions for you).
You are not able to understand the French language.

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: Susana Quijano-Roy, MD, PhD, Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, Hôpital Raymond Poincaré, 92380 Garches, FRANCE

Phone: +33147107890

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: NCT04177134

Sponsor: Assistance Publique - Hôpitaux de Paris

Phase: Not Applicable

Status: Recruiting

Condition: Spinal Muscular Atrophy

Trial contact: +33147107890

More information: https://clinicaltrials.gov/study/NCT04177134

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:

You have been diagnosed with Spinal Muscular Atrophy (SMA) caused by a change in the 5q gene (types 1 to 4), confirmed by a genetic test.
You have been treated at a specialist SMA centre in France between September 1, 2016 and August 31, 2024.
For the part of the study looking forward in time: you have signed a consent form agreeing to take part.
For the part of the study looking at past records: you have been given an information form and have not objected to your data being used.
You are covered by health insurance.

Who may not be able to join:

You have a different type of SMA that is not caused by the 5q gene change.
You are currently under a legal guardianship or curatorship arrangement (meaning someone else has been appointed by a court to make decisions for you).
You are not able to understand the French language.

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

Assistance Publique - Hôpitaux de Paris

Registry

ClinicalTrials.gov

Start date

31 January 2020

Est. completion

31 January 2029

Where this trial is recruiting

🇫🇷 France

Primary endpoints

Motor functional development or status; Motor function scores; Yearly changes of morbi-mortality-vital events; Respiratory events; Digestive-nutritional events; Yearly changes of spinal events

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