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A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
EU CTIS ID: 2023-506009-20-00
What this study is testing
1. To evaluate the efficacy of risdiplam in patients 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) amplitude ≥ 1.5mV, as determined by the proportion of patients who are sitting without support after 12 months of treatment
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Males and females aged from birth (1 day) to 6 weeks (42 days) of age at the time of first dose (Day 1); a minimum age of 7 days at first dose is required for the first infant to be enrolled
- 2. Gestational age of 37-42 weeks for singleton births; gestational age of 34-42 weeks for twins
- 3. Body weight ≥ 3rd percentile for age, using appropriate country specific guidelines
- 4. Genetic diagnosis of 5q-autosomal recessive SMA, including confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the SMN1 gene
- 5. Absence of clinical signs or symptoms at screening (Day -42 to Day -2) or at baseline (Day -1) that are, in the opinion of the investigator, strongly suggestive of SMA
- 6. Receiving adequate nutrition and hydration at the time of screening, in the opinion of the investigator
You likely can't join if
- 1. Concomitant or previous participation in any investigational drug or device study at any time
- 2. Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide, SMN2-splicing modifier, or gene therapy either in a clinical study or as part of medical care
- 3. Presence of significant concurrent syndromes or diseases
- 4. In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures
- 5. Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation
- 6. Awake hypoxemia (SaO2 < 95%) with or without ventilator support
The study team makes the final eligibility decision.
Where it's taking place
- Taiwan
- United States
- Brazil
- Australia
- Russian Federation
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Taiwan; United States; Brazil; Australia; Russian Federation. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.