A Phase IV Open-Label 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

Status: Recruiting
Location: See all (19) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
SUMMARY

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3 months
Maximum Age: 2
Healthy Volunteers: f
View:

• \<2 years of age at the time of informed consent

• Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene

• Confirmed presence of two SMN2 gene copies as documented through laboratory testing

• Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically

• Has received onasemnogene abeparvovec for SMA no less than 13 weeks prior to enrollment

• If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration.

• In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 26 weeks or less) documented by 2 individual time points in the functions as follows: swallowing AND one additional function/ability (respiratory, motor function, other) per appropriate expectation.

Locations
United States
Arkansas
University of Arkansas for Medical Sciences
RECRUITING
Little Rock
California
Valley Children's Hospital
RECRUITING
Madera
Stanford Univ Medical Center
RECRUITING
Palo Alto
Colorado
Children's Hospital of Colorado
RECRUITING
Aurora
Florida
University of Florida Pediatrics
RECRUITING
Gainesville
Georgia
Children's Healthcare of Atlanta Center for Advanced Pediatrics
RECRUITING
Atlanta
Michigan
Helen DeVos Children's Hospital at Spectrum Health
RECRUITING
Grand Rapids
Pennsylvania
Children'S Hospital of Philadelphia
RECRUITING
Philadelphia
Texas
University of Texas Southwestern Medical Center
RECRUITING
Dallas
Virginia
Children's Hospital of the King's Daughter
RECRUITING
Norfolk
Other Locations
Germany
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
RECRUITING
Berlin
UKGM Standort Gießen
RECRUITING
Giessen
Israel
Soroka Medical Center
RECRUITING
Beersheba
Schneider Children's Medical Center of Israel
RECRUITING
Petah Tikva
Sourasky MC, Dana-Dwek Children's Hospital
RECRUITING
Tel Aviv
Poland
Uniwersyteckie Centrum Kliniczne
RECRUITING
Gdansk
Instytut Pomnik Centrum Zdrowia Dziecka
RECRUITING
Warsaw
Qatar
Sidra Medicine
RECRUITING
Al Rayyan
United Kingdom
Great Ormond Street Hospital For Children
RECRUITING
London
Contact Information
Primary
Reference Study ID Number: BN44621 https://forpatients.roche.com/
global-roche-genentech-trials@gene.com
888-662-6728 (U.S. Only)
Time Frame
Start Date: 2024-08-14
Estimated Completion Date: 2029-03-31
Participants
Target number of participants: 28
Treatments
Experimental: Risdiplam
Participants will receive risdiplam orally once daily for 72 weeks (Treatment Period). The Treatment Period will be followed by a 1-year Treatment Extension Period for a total study duration of 120 weeks (approximately 2.5 years) for each participant enrolled.
Sponsors
Leads: Hoffmann-La Roche

This content was sourced from clinicaltrials.gov