New hope for SMA babies: risdiplam trial launches in newborns

NCT ID NCT05808764

Completed Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is testing the drug risdiplam in newborn babies with spinal muscular atrophy (SMA), a serious muscle-weakening disease. The goal is to see how the drug moves through the body and if it is safe for infants under 20 days old. Ten babies will take part, and researchers will monitor side effects and drug levels in the blood.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for MUSCULAR ATROPHY, SPINAL are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MUSCULAR ATROPHY, SPINAL

Contacts and locations

Locations

Ann and Robert H. Lurie Children Hospital of Chicago

Chicago, Illinois, 60611, United States
CHR Citadelle

Liège, 4000, Belgium
Children'S Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada
Clinic for Special Children.

Gordonville, Pennsylvania, 17529, United States
Fondazione Policlinico Univeristario A. Gemelli

ROMA, Emilia-Romagna, 00168, Italy
Fondazione Serena Onlus - CENTRO CLINICO NEMO

Milano, Emilia-Romagna, 20162, Italy
Hopital Universitaire des Enfants Reine Fabiola

Brussels, 1020, Belgium
Instytut Pomnik - Centrum Zdrowia Dziecka

Warsaw, 04-730, Poland
OUS (Oslo University Hospital), Rikshospitalet

Oslo, 0372, Norway
UMC Utrecht

Utrecht, 3508, Netherlands
Universitatsklinikum Essen

Essen, 45147, Germany
University Of Michigan

Ann Arbor, Michigan, 48109, United States
Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

More trials for these conditions

Other studies related to the condition(s) this trial covers.