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Recruiting NCT05861999

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

Phase IV Interventional Muscular Atrophy, Spinal

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Risdiplam.
Who it may be relevant to
Registry conditions: Muscular Atrophy, Spinal. Basic parameters: 3 months — 24 months · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Germany, Israel, Poland, Qatar +1
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Drug Risdiplam
    Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.

Primary outcome measures

  • Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment [Time frame: Baseline, Week 72]
Secondary outcome measures (3)
  • Percentage of Participants With Adverse Events [Time frame: Up to 120 weeks]
  • Percentage of Participants With Serious Adverse Events [Time frame: Up to 120 weeks]
  • Percentage of Participants With Treatment Discontinuation Due to Adverse Events [Time frame: Up to 120 weeks]

Eligibility criteria

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 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.

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
  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 10 centers
  • University of Arkansas for Medical Sciences — Little Rock
  • Valley Children's Hospital — Madera
  • Stanford Univ Medical Center — Palo Alto
  • Children's Hospital of Colorado — Aurora
  • University of Florida Pediatrics — Gainesville
  • Children's Healthcare of Atlanta Center for Advanced Pediatrics — Atlanta
  • Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids
  • Children'S Hospital of Philadelphia — Philadelphia
  • … and 2 more centers
Israel · 3 centers
  • Soroka Medical Center — Beersheba
  • Schneider Children's Medical Center of Israel — Petah Tikva
  • Sourasky MC, Dana-Dwek Children's Hospital — Tel Aviv
Germany · 2 centers
  • Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie — Berlin
  • UKGM Standort Gießen — Giessen
Poland · 2 centers
  • Uniwersyteckie Centrum Kliniczne — Gda?sk
  • Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw
Qatar · 1 center
  • Sidra Medicine — Al Rayyan
United Kingdom · 1 center
  • Great Ormond Street Hospital For Children — London

Identifiers

NCT: NCT05861999 · BN44621 · 2023-505161-81-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗