RecruitingPhase 2NCT05808764

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

Sponsor

Hoffmann-La Roche

Enrollment

10 participants

Start Date

Apr 26, 2024

Study Type

INTERVENTIONAL

Conditions

Muscular Atrophy, Spinal

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

Max Age: 19 Days

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Risdiplam for people with muscular atrophy, spinal. The study is currently recruiting participants at 13 locations. People eligible for this study include up to age 19 Days.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGRisdiplam

Participants will receive 0.15 mg/kg risdiplam orally once daily for 28 days.

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

OUS (Oslo University Hospital), Rikshospitalet

Oslo, Norway

Uniwersyteckie Centrum Kliniczne

Gdansk, Poland

Instytut Pomnik - Centrum Zdrowia Dziecka

Warsaw, Poland

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT05808764

Related Trials

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

NCT0586199919 locations

A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)

NCT0655541919 locations

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

NCT0586198616 locations

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)

NCT072216695 locations

A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

NCT07444450