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

PHASE4RecruitingINTERVENTIONAL
Enrollment

28

Participants

Timeline

Start Date

May 30, 2024

Primary Completion Date

March 31, 2028

Study Completion Date

March 31, 2029

Conditions
Muscular Atrophy, Spinal
Interventions
DRUG

Risdiplam

Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.

Trial Locations (16)

10032

RECRUITING

Columbia University Medical Center, New York

13353

RECRUITING

Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie, Berlin

19104

RECRUITING

Children'S Hospital of Philadelphia, Philadelphia

23510

RECRUITING

Children's Hospital of the King's Daughter, Norfolk

32610

RECRUITING

University of Florida Pediatrics, Gainesville

35392

RECRUITING

UKGM Standort Gießen, Giessen

49503

RECRUITING

Helen DeVos Children's Hospital at Spectrum Health, Grand Rapids

60611

NOT_YET_RECRUITING

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

72103

RECRUITING

University of Arkansas for Medical Sciences, Little Rock

75390

RECRUITING

The University of Texas Southwestern Medical Center at Dallas, Dallas

76104

RECRUITING

Cook Children's Jane and John Justin Neurosciences Center, Fort Worth

80045

RECRUITING

Children's Hospital of Colorado, Aurora

30329-2309

RECRUITING

Children's Healthcare of Atlanta Center for Advanced Pediatrics, Atlanta

80-952

RECRUITING

Uniwersyteckie Centrum Kliniczne, Gda?sk

04-730

RECRUITING

Instytut Pomnik Centrum Zdrowia Dziecka, Warsaw

WC1N 3JH

RECRUITING

Great Ormond Street Hospital For Children, London

All Listed Sponsors
lead

Hoffmann-La Roche

INDUSTRY