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

Purpose

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children < 2 years of age genetically diagnosed with SMA.

Condition

  • Muscular Atrophy, Spinal

Eligibility

Eligible Ages
Between 3 Months and 24 Months
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • <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, but not more than months 30 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 - Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration

Exclusion Criteria

  • Previous or current enrolment in investigational study prior to initiation of study treatment - Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information - Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide - Concomitant or previous use of an anti-myostatin agent - Participants requiring invasive ventilation or tracheostomy - Participants requiring awake non-invasive ventilation or with awake hypoxemia (Arterial Oxygen Saturation [SaO2] <95%) with or without ventilator support - Presence of feeding tube and an OrSAT score of 0 - Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening - Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.

Study Design

Phase
Phase 4
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
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.
  • Drug: Risdiplam
    Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
    Other names:
    • RO7034067

Recruiting Locations

University of Arkansas for Medical Sciences
Little Rock, Arkansas 72103

Children's Hospital of Colorado
Aurora, Colorado 80045

University of Florida Pediatrics
Gainesville, Florida 32610

Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia 30329-2309

Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan 49503

Columbia University Medical Center
New York, New York 10032

Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania 19104

The University of Texas Southwestern Medical Center at Dallas
Dallas, Texas 75390

Cook Children's Jane and John Justin Neurosciences Center
Fort Worth, Texas 76104

Children's Hospital of the King's Daughter
Norfolk, Virginia 23510

More Details

Status
Recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Reference Study ID Number: BN44620 https://forpatients.roche.com/
888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com