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A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy | Clinical Trials | Clareo Health

RECRUITINGPHASE2

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

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

NCT05808764•Hoffmann-La Roche

View on ClinicalTrials.gov

Summary

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.

Eligibility

Age

0 - 0 years

Sex

ALL

Healthy Volunteers

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

Locations (13)

Ann and Robert H. Lurie Children Hospital of Chicago

Chicago, Illinois, United States

University Of Michigan

Ann Arbor, Michigan, United States

Clinic for Special Children.

Gordonville, Pennsylvania, United States

Hopital Universitaire des Enfants Reine Fabiola

Brussels, Belgium

CHR Citadelle

Liège, Belgium

Children'S Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Universitatsklinikum Essen

Essen, Germany

Fondazione Serena Onlus - CENTRO CLINICO NEMO

Milano, Emilia-Romagna, Italy

Fondazione Policlinico Univeristario A. Gemelli

ROMA, Emilia-Romagna, Italy

UMC Utrecht

Utrecht, Netherlands

Key Dates

Start Date

April 26, 2024

Primary Completion Date

November 30, 2026

Completion Date

November 30, 2026

Last Updated

March 6, 2026

Enrollment

ESTIMATED participants

Conditions

Muscular Atrophy, Spinal

Interventions

Risdiplam

DRUG

Contacts

Reference Study ID Number: BN44619 https://forpatients.roche.com/

CONTACT

888-662-6728 (U.S. Only)global-roche-genentech-trials@gene.com

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Muscular Atrophy, Spinal

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RECRUITING

Natural History of SMA

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ACTIVE_NOT_RECRUITING

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 6, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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

Natural History of SMA

Adult Spinal Muscular Atrophy (SMA) China Registry

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Ultrasound-assisted vs Landmark Based Intrathecal Administration of Nusinersen

Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection