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A Study to Investigate the Pharmacokinetics (PK) and Safety of Risdiplam in Infants with Spinal Muscular Atrophy
EU CTIS ID: 2023-505602-42-00
What this study is testing
To characterize the risdiplam PK profile and to evaluate the safety of risdiplam
- 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
- Male or female newborn infant aged <20 days at first dose.
- Newborn infants with genetic diagnosis of 5q-autosomal recessive SMA or newborn infants identified as positive for SMA via newborn screening or via prenatal testing.
- Gestational age equal to or greater than 37 weeks.
- Receiving adequate nutrition and hydration at the time of screening.
- Adequately recovered from any acute illness at baseline and considered well enough to participate in the study.
- Parent/caregiver is willing to consider nasogastric, nasojejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator.
You likely can't join if
- Presence of clinical symptoms or signs consistent with SMA Type 0.
- In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures.
- Systolic blood pressure or diastolic blood pressure or heart rate abnormalities or presence of clinically relevant electrocardiogram (ECG) abnormalities.
- The infant (or the person breastfeeding the infant) taking any of the following: any inhibitor of CYP3A4 taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing, any inducer of CYP3A4 taken within 4 weeks (or within 5 times the elimination half-life, whichever is longer prior to dosing, and/or use of any multidrug and toxin extrusion (MATE) substrates taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing.
- Concurrent or previous administration of nusinersen or onasemnogene abeparvovec.
- Clinically significant abnormalities in laboratory test.
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- United States
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 Canada; United States. 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.