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
Recruiting now · Phase 4
Conditions studied: Muscular Atrophy, Spinal
In brief
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.
Key facts
- Study ID
- NCT05861999
- Run by
- Hoffmann-La Roche
- People needed
- 28
- Starts
- 2024-08-14
- Expected to finish
- 2029-03-31
- Last updated by the study team
- 2026-07-09
Who can join
Age: 0 and older, up to 2. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- <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.
You may not qualify if…
- 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
- 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.
Where it is running
- University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States (enrolling)
- Valley Children's Hospital — Madera, California, United States (enrolling)
- Stanford Univ Medical Center — Palo Alto, California, United States (enrolling)
- Children's Hospital of Colorado — Aurora, Colorado, United States (enrolling)
- University of Florida Pediatrics — Gainesville, Florida, United States (enrolling)
- Children's Healthcare of Atlanta Center for Advanced Pediatrics — Atlanta, Georgia, United States (enrolling)
- Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids, Michigan, United States (enrolling)
- Children'S Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
- University of Texas Southwestern Medical Center — Dallas, Texas, United States (enrolling)
- Great Ormond Street Hospital For Children — London, United Kingdom (enrolling)
- Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie — Berlin, Germany (enrolling)
- UKGM Standort Gießen — Giessen, Germany (enrolling)
- Soroka Medical Center — Beersheba, Israel (enrolling)
- Schneider Children's Medical Center of Israel — Petah Tikva, Israel (enrolling)
- Sourasky MC, Dana-Dwek Children's Hospital — Tel Aviv, Israel (enrolling)
- Uniwersyteckie Centrum Kliniczne — Gda?sk, Poland (enrolling)
- Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw, Poland (enrolling)
- Sidra Medicine — Al Rayyan, Qatar (enrolling)
- Children's Hospital of the King's Daughter — Norfolk, Virginia, United States
Full record on ClinicalTrials.gov
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