Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

PHASE4Active, not recruitingINTERVENTIONAL
Enrollment

402

Participants

Timeline

Start Date

March 29, 2022

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2026

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

Risdiplam

Risdiplam taken by participants as prescribed by their physician.

DEVICE

SMA-DAT Application and ADAM Sensor

Participants will perform a programmed selection of SMA-DAT tasks while wearing the ADAM Sensor.

Trial Locations (39)

10003

NYU Hospital for Joint Diseases, New York

10032

Columbia University Med Center, New York

17033

Penn State Milton S. Hershey Medical Center, Hershey

19104

Childrens Hospital of Philadelphia, Philadelphia

20007

Medstar Georgetown University Hospital, Washington D.C.

20010

Children's National Hospital, Washington D.C.

22903

University of Virginia Children?s Hospital, Charlottesville

26508

UBC (Remote Coordinating Center, no physical facility), Morgantown

30329

Rare Disease Research, LLC, Atlanta

32789

Advent Health Orlando, Winter Park

32827

Nemour's Children's Hospital, Florida, Orlando

33701

All Children's Research Institute, Inc., St. Petersburg

40202

Norton Children's Hospital, Louisville

40536

University of Kentucky Medical Center, Lexington

45229

Cincinnati Childrens Hospital, Cincinnati

48109

University of Michigan Pediatric Rehabilitation Center, Ann Arbor

49503

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

53201

Childrens Hospital of Wisconsin, Milwaukee

55343

Gillette Children's Specialty Healthcare, Minnetonka

55905

Mayo Clinic, Rochester

63110

Washington University;Wash Uni. Sch. Of Med, St Louis

66160

University of Kansas Medical Center, Kansas City

72103

University of Arkansas for Medical Sciences, Little Rock

76208

Neurology & Neuromuscular Care Center, Denton

78229

Methodist Children's Hospital of South Texas, San Antonio

78681

Central Texas Neurology Consultants, Round Rock

78723

University of Texas at Austin Health sciences, Dell Medical School, Austin

80045

University of Colorado, Aurora

84108

University Of Utah, Salt Lake City

85013

Barrow Neurological Institute, Phoenix

85016

Phoenix Children's Hospital, Phoenix

90010

Children's Hospital Los Angeles, Los Angeles

92354

Loma Linda University Health Care, Loma Linda

92868

University California - Irvine, Orange

93636

Valley Children's Hospital, Madera

95817

University of California Davis Medical Center, Sacramento

98405

MultiCare Health System Institute for Research and Innovation, Tacoma

02114

Massachusetts General Hospital, Boston

00912

Instituto de Rehabilitación del Caribe, Santurce

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Genentech, Inc.

INDUSTRY

NCT05232929 - Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) | Biotech Hunter | Biotech Hunter