A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

{ "Risdiplam as an Early Intervention for Spinal Muscular Atrophy After Gene Therapy", "This study is looking at how well and how safely risdiplam works when given early to children with spinal muscular atrophy (SMA) who have already received gene therapy (onasemnogene abeparvovec). Risdiplam is a medicine given by mouth. You might be able to join if you are between 3 and 24 months old, have a confirmed diagnosis of SMA with two copies of the SMN2 gene, and have previously received onasemnogene abeparvovec. The study will measure changes in your gross motor skills (large body movements like sitting and walking) using a special test called the Bayley Scales of Infant and Toddler Development after 72 weeks of risdiplam treatment. The study aims to enroll 28 participants.", "design": "This is an open-label, single-arm study, meaning everyone receives risdiplam and there is no comparison group. It plans to enroll 28 participants.", "commitments": "Participants will receive risdiplam orally at a dose adjusted for their weight and age. Motor skills will be assessed at the beginning of the study and again after 72 weeks of treatment.", "compensation": "Not stated in the trial record.", "follow_up": "Participants will be followed for 72 weeks after starting risdiplam treatment to assess changes in motor skills.", }

Study design: Not specified.
What's involved: Not specified in the trial record.
Compensation: Not stated in the trial record.
Follow-up: Not specified.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Recruiting

PHASE4Ages 3–24InterventionalTreatment

Hoffmann-La Roche

~28 participants

Updated 2026-08-04 on ClinicalTrials.gov

What's tested:Risdiplam

At a glance

Recruiting sites

14 of 16 listed sites are recruiting right now

RecruitingSuspended, closed, or not yet open

What they're measuring

Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment

Measured over Baseline, Week 72

Conditions

Muscular Atrophy, Spinal

Where it's being run

16 sites across 13 states

Texas2

Germany2

Poland2

Arkansas1

Colorado1

Florida1

Georgia1

Illinois1

Study leadership

Clinical Trials · STUDY_DIRECTOR · Hoffmann-La Roche

Who to contact

Reference Study ID Number: BN44620 https://forpatients.roche.com/ No attachments to email below.

[email protected]

888-662-6728 (U.S. Only)

Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime

Eligibility criteria

Inclusion

\<2 years of age at the time of informed consent

Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene

Confirmed presence of two SMN2 gene copies as documented through laboratory testing

Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically

Has received onasemnogene abeparvovec for SMA no less than 13 weeks, but not more than months 30 weeks, prior to enrollment

If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration

Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration

Exclusion

Previous or current enrolment in investigational study prior to initiation of study treatment

Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information

Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide

Concomitant or previous use of an anti-myostatin agent

Participants requiring invasive ventilation or tracheostomy

Participants requiring awake non-invasive ventilation or with awake hypoxemia (Arterial Oxygen Saturation \[SaO2\] \<95%) with or without ventilator support

Presence of feeding tube and an OrSAT score of 0

Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening

Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.

What this trial measures

Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam TreatmentBaseline, Week 72
The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw score indicates improvement.

Trialytics