Phase 2 Spinal Muscular Atrophy Trial, Active, Not Recruiting (NCT03779334)

Phase 2 Spinal Muscular Atrophy Trial, Active, Not Recruiting NCT03779334 Sponsor: Hoffmann-La Roche Condition: Spinal Muscular Atrophy

Printed from Voxsanity (voxsanity.com.au) — NCT03779334 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

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Trial results

AI generated results 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

According to the results reported on ClinicalTrials.gov, this trial (NCT03779334) looked at a medicine called risdiplam in infants who had been diagnosed with spinal muscular atrophy (SMA) before they showed any symptoms — meaning they were identified through newborn screening. A total of 26 babies took part, split into three groups based on how many copies of a gene called SMN2 they carried: 8 babies had 2 copies, 13 had 3 copies, and 5 had 4 or more copies. The trial's main question was focused specifically on a smaller subgroup of 5 babies from the 2-copy group who met particular criteria at the start of the study. Notably, the reported data shows that none of the 26 participants completed the study as planned — all are listed as "not completed," though the reasons for this are not detailed in the data provided here. The reported data shows that for the primary outcome — whether babies in that specific 5-person subgroup could sit without any support for at least 5 seconds — 80% of those participants (that is, 4 out of 5 babies) achieved this milestone. This was the only outcome measure for which numbers were reported. For all of the secondary outcomes — including things like whether participants developed SMA symptoms, how many remained alive without needing a breathing machine, and whether babies reached other movement milestones — no numerical results were provided in the data submitted to ClinicalTrials.gov. Because most of the secondary outcomes have no reported figures, it is not possible to describe what those measurements found. The data was simply not reported for those measures. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 40802943) ↗

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

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:

Your baby is a boy or girl who is between 1 day and 42 days old at the time of the first dose (the very first baby enrolled must be at least 7 days old)
Your baby was born between 37 and 42 weeks of pregnancy (if a single birth), or between 34 and 42 weeks (if a twin)
Your baby's weight is at or above the 3rd percentile (a low but acceptable weight range) for their age, based on guidelines for your country
Your baby has been genetically diagnosed with spinal muscular atrophy (SMA) caused by a specific change in the SMN1 gene
Your baby does not yet show clear physical signs or symptoms of SMA at the time of screening or at the start of the study, in the doctor's opinion
Your baby is eating and drinking well enough at the time of screening, in the doctor's opinion
Your baby has recovered from any recent illness and is considered well enough to take part, in the doctor's opinion
You are able and expected to safely travel to the study site for all required visits throughout the whole study, in the doctor's opinion
Your baby can complete all study procedures and visits, and you (as parent or caregiver) have a stable and supportive home situation, in the doctor's opinion
You (as parent or caregiver) are willing to consider a feeding tube (through the nose or stomach) if the doctor recommends it, to keep your baby safely nourished and able to receive treatment
You (as parent or caregiver) are willing to consider non-invasive breathing support for your baby if the doctor recommends it during the study

Who may not be able to join:

Your baby has previously taken part in, or is currently taking part in, any other clinical trial for a drug or medical device
Your baby has previously received or is currently receiving any other SMA treatment, including certain gene therapies or SMA-specific medicines, either in a trial or as regular medical care
Your baby has another significant medical condition or syndrome alongside SMA
Your baby does not have suitable veins or capillaries for the blood tests required in the study, in the doctor's opinion
Your baby currently needs a breathing machine (invasive ventilation), a tracheostomy (a breathing tube in the throat), or breathing support while awake
Your baby has low oxygen levels in the blood while awake, with or without breathing support
Your baby was born with multiple or fixed joint stiffness and/or a hip that is out of place
Your baby has blood pressure or heart rate that the doctor considers a medical concern
Your baby has certain heart rhythm abnormalities detected before the study starts, including a specific measurement on a heart tracing (ECG) that is too high (above 460 ms), or a personal or family history of a heart condition called long QT syndrome that the doctor feels poses a risk (confirm with trial site)
Your baby (or their mother, if breastfeeding) is taking certain medicines that affect how the study drug is processed by the body — including some within the past 2 to 4 weeks (confirm with trial site for the specific medicines involved)
Your baby has significant abnormalities in blood test results
Your baby is known or suspected to be allergic to the study drug (risdiplam) or any of its ingredients
Your baby is taking salbutamol (albuterol) or a similar medicine by mouth for SMA (inhaled versions are allowed)
Your baby was exposed — through the mother during pregnancy or breastfeeding — to medicines known to harm the retina (the back of the eye), or such medicines may be needed during the study
Your baby has been diagnosed with an eye disease

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

Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.

Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Clinical Trials, Hoffmann-La Roche

Australian sites

Sydney Children's Hospital, Randwick, New South Wales

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 found the following past clinical trial relevant to my condition, and wanted to raise it with you. It is no longer recruiting, but I wanted to ask whether similar active trials exist, or whether its results are relevant to my care:

Trial: NCT03779334

Sponsor: Hoffmann-La Roche

Phase: Phase 2

Status: Active, Not Recruiting

Condition: Spinal Muscular Atrophy

More information: https://clinicaltrials.gov/study/NCT03779334

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:

Your baby is a boy or girl who is between 1 day and 42 days old at the time of the first dose (the very first baby enrolled must be at least 7 days old)
Your baby was born between 37 and 42 weeks of pregnancy (if a single birth), or between 34 and 42 weeks (if a twin)
Your baby's weight is at or above the 3rd percentile (a low but acceptable weight range) for their age, based on guidelines for your country
Your baby has been genetically diagnosed with spinal muscular atrophy (SMA) caused by a specific change in the SMN1 gene
Your baby does not yet show clear physical signs or symptoms of SMA at the time of screening or at the start of the study, in the doctor's opinion
Your baby is eating and drinking well enough at the time of screening, in the doctor's opinion
Your baby has recovered from any recent illness and is considered well enough to take part, in the doctor's opinion
You are able and expected to safely travel to the study site for all required visits throughout the whole study, in the doctor's opinion
Your baby can complete all study procedures and visits, and you (as parent or caregiver) have a stable and supportive home situation, in the doctor's opinion
You (as parent or caregiver) are willing to consider a feeding tube (through the nose or stomach) if the doctor recommends it, to keep your baby safely nourished and able to receive treatment
You (as parent or caregiver) are willing to consider non-invasive breathing support for your baby if the doctor recommends it during the study

Who may not be able to join:

Your baby has previously taken part in, or is currently taking part in, any other clinical trial for a drug or medical device
Your baby has previously received or is currently receiving any other SMA treatment, including certain gene therapies or SMA-specific medicines, either in a trial or as regular medical care
Your baby has another significant medical condition or syndrome alongside SMA
Your baby does not have suitable veins or capillaries for the blood tests required in the study, in the doctor's opinion
Your baby currently needs a breathing machine (invasive ventilation), a tracheostomy (a breathing tube in the throat), or breathing support while awake
Your baby has low oxygen levels in the blood while awake, with or without breathing support
Your baby was born with multiple or fixed joint stiffness and/or a hip that is out of place
Your baby has blood pressure or heart rate that the doctor considers a medical concern
Your baby has certain heart rhythm abnormalities detected before the study starts, including a specific measurement on a heart tracing (ECG) that is too high (above 460 ms), or a personal or family history of a heart condition called long QT syndrome that the doctor feels poses a risk (confirm with trial site)
Your baby (or their mother, if breastfeeding) is taking certain medicines that affect how the study drug is processed by the body — including some within the past 2 to 4 weeks (confirm with trial site for the specific medicines involved)
Your baby has significant abnormalities in blood test results
Your baby is known or suspected to be allergic to the study drug (risdiplam) or any of its ingredients
Your baby is taking salbutamol (albuterol) or a similar medicine by mouth for SMA (inhaled versions are allowed)
Your baby was exposed — through the mother during pregnancy or breastfeeding — to medicines known to harm the retina (the back of the eye), or such medicines may be needed during the study
Your baby has been diagnosed with an eye disease

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

Active, Not Recruiting

Phase

Phase 2

Sponsor

Hoffmann-La Roche

Registry

ClinicalTrials.gov

Start date

7 August 2019

Est. completion

20 February 2023

Where this trial is recruiting

🇦🇺 Australia 🇧🇪 Belgium 🇧🇷 Brazil 🇵🇱 Poland 🇷🇺 Russia 🇹🇼 Taiwan 🇺🇸 United States

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

Primary endpoints

Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support

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