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

PHASE4RecruitingINTERVENTIONAL
Enrollment

28

Participants

Timeline

Start Date

August 14, 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 (19)

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

72103

RECRUITING

University of Arkansas for Medical Sciences, Little Rock

75390

RECRUITING

University of Texas Southwestern Medical Center, Dallas

80045

RECRUITING

Children's Hospital of Colorado, Aurora

93636

RECRUITING

Valley Children's Hospital, Madera

94304

RECRUITING

Stanford Univ Medical Center, Palo Alto

4920235

RECRUITING

Schneider Children's Medical Center of Israel, Petah Tikva

6423906

RECRUITING

Sourasky MC, Dana-Dwek Children's Hospital, Tel Aviv

8410101

RECRUITING

Soroka Medical Center, Beersheba

30329-2309

RECRUITING

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

80-952

RECRUITING

Uniwersyteckie Centrum Kliniczne, Gdansk

04-730

RECRUITING

Instytut Pomnik Centrum Zdrowia Dziecka, Warsaw

Unknown

RECRUITING

Sidra Medicine, Al Rayyan

WC1N 3JH

RECRUITING

Great Ormond Street Hospital For Children, London

All Listed Sponsors
lead

Hoffmann-La Roche

INDUSTRY