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A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Purpose

This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.

Condition

Muscular Atrophy, Spinal

Eligibility

Eligible Ages: Under 19 Days
Eligible Sex: All
Accepts Healthy Volunteers: No

Inclusion Criteria

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.

Exclusion Criteria

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 - 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

Study Design

Phase: Phase 2
Study Type: Interventional
Allocation: N/A
Intervention Model: Single Group Assignment
Primary Purpose: Treatment
Masking: None (Open Label)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Risdiplam	Participants will receive risdiplam once daily for 28 days.	Drug: Risdiplam Participants will receive 0.15 mg/kg risdiplam orally once daily for 28 days. Other names: Evrysdi

Recruiting Locations

Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois 60611

University Of Michigan
Ann Arbor, Michigan 48109

Clinic for Special Children.
Gordonville, Pennsylvania 17529

More Details

NCT ID: NCT05808764
Status: Recruiting
Sponsor: Hoffmann-La Roche

Study Contact

Reference Study ID Number: BN44619 https://forpatients.roche.com/
888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.