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)

PHASE1RecruitingINTERVENTIONAL
Enrollment

58

Participants

Timeline

Start Date

January 16, 2025

Primary Completion Date

May 15, 2026

Study Completion Date

May 15, 2026

Conditions
Muscular Atrophy, Spinal
Interventions
DRUG

Nusinersen

Administered as specified in the treatment arm.

DEVICE

ThecaFlex DRx System

Implanted as specified in the treatment arm.

Trial Locations (18)

168

NOT_YET_RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Roma

19104

RECRUITING

Children's Hospital of Philadelphia, Philadelphia

20162

RECRUITING

Fondazione Serena Onlus - Centro Clinico Nemo_Milano, Milan

22903

RECRUITING

Stacey Hall Developmental Pediatrics, Charlottesville

23507

RECRUITING

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

28046

RECRUITING

Hospital Universitario La Paz, Madrid

45147

RECRUITING

Universitaetsklinikum Essen, Essen

46026

RECRUITING

Hospital Universitari i Politecnic La Fe_Valencia, Valencia

49503

RECRUITING

Helen DeVos Children's Hospita, Grand Rapids

77030

RECRUITING

Texas Childrens Hospital Houston, Houston

92380

RECRUITING

Hôpital Raymond Poincaré, Garches

92868

RECRUITING

Children's Hospital of Orange County, Orange

California

RECRUITING

"Stanford University Medical Center , Department of Neurology_Palo Alto"

60611-2605

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

02115

RECRUITING

Boston Children's Hospital, Boston

Pennsylvania

RECRUITING

"Milton S. Hershey Medical Center , Pennsylvania State University_Hershey"

31-826

RECRUITING

Szpital Specjalistyczny im. L.Rydygiera w Krakowie, Krakow

93-338

RECRUITING

Instytut Centrum Zdrowia Matki Polki, Lodz

Sponsors

Lead Sponsor

Collaborators (1)

All Listed Sponsors
collaborator

Alcyone Therapeutics, Inc

INDUSTRY

lead

Biogen

INDUSTRY