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

Description

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.

Conditions

Muscular Atrophy, Spinal

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Study Overview

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Study Details

Study 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.

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

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

Condition
Muscular Atrophy, Spinal
Intervention / Treatment

-

Contacts and Locations

Little Rock

University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States, 72103

Madera

Valley Children's Hospital, Madera, California, United States, 93636

Atlanta

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

Dallas

University of Texas Southwestern Medical Center, Dallas, Texas, United States, 75390-0001

Norfolk

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * \<2 years of age at the time of informed consent
  • * Confirmed diagnosis of 5q-autosomal recessive SMA
  • * Confirmed presence of two SMN2 gene copies
  • * Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
  • * Has received onasemnogene abeparvovec for SMA no less than 3 months prior to enrollment
  • * In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 6 months 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.
  • * Treatment with investigational therapy prior to initiation of study treatment
  • * Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information
  • * Concomitant or previous administration of a SMN2-targeting antisense oligonucleotide or SMN2 splicing modifier either in a clinical study or as part of medical care
  • * 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.

Ages Eligible for Study

3 Months to 24 Months

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Hoffmann-La Roche,

Clinical Trials, STUDY_DIRECTOR, Hoffmann-La Roche

Study Record Dates

2028-03-31