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
HINALEA 2
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
2 other identifiers
interventional
28
6 countries
19
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_4
Started Aug 2024
Longer than P75 for phase_4
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 8, 2023
CompletedFirst Posted
Study publicly available on registry
May 17, 2023
CompletedStudy Start
First participant enrolled
August 14, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 31, 2029
April 13, 2026
April 1, 2026
3.6 years
May 8, 2023
April 10, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
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
The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw score indicates improvement.
Baseline, Week 72
Secondary Outcomes (3)
Percentage of Participants With Adverse Events
Up to 120 weeks
Percentage of Participants With Serious Adverse Events
Up to 120 weeks
Percentage of Participants With Treatment Discontinuation Due to Adverse Events
Up to 120 weeks
Other Outcomes (11)
Change from Baseline in Bulbar/Swallowing Function Assessment as Measured by the Oral and Swallowing Abilities Tool (OrSAT) at 72 Weeks of Risdiplam Treatment and Over Time
From baseline up to Week 120
Change in Swallowing Function Assessment as Measured by the Pediatric Functional Oral Intake Scale (p-FOIS) Over Time
From baseline up to Week 120
Change from Baseline in the Raw Score of BSID-III Gross Motor Score Over Time Under Risdiplam Treatment
From baseline up to Week 120
- +8 more other outcomes
Study Arms (1)
Risdiplam
EXPERIMENTALParticipants 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.
Interventions
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
Eligibility Criteria
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (19)
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72103, United States
Valley Children's Hospital
Madera, California, 93636, United States
Stanford Univ Medical Center
Palo Alto, California, 94304, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
University of Florida Pediatrics
Gainesville, Florida, 32610, United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, 30329-2309, United States
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, 49503, United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Children's Hospital of the King's Daughter
Norfolk, Virginia, 23510, United States
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
Berlin, 13353, Germany
UKGM Standort Gießen
Giessen, 35392, Germany
Soroka Medical Center
Beersheba, 8410101, Israel
Schneider Children's Medical Center of Israel
Petah Tikva, 4920235, Israel
Sourasky MC, Dana-Dwek Children's Hospital
Tel Aviv, 6423906, Israel
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952, Poland
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
Sidra Medicine
Al Rayyan, Qatar
Great Ormond Street Hospital For Children
London, WC1N 3JH, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Clinical Trials
Hoffmann-La Roche
Central Study Contacts
Reference Study ID Number: BN44621 https://forpatients.roche.com/
CONTACT
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 8, 2023
First Posted
May 17, 2023
Study Start
August 14, 2024
Primary Completion (Estimated)
March 31, 2028
Study Completion (Estimated)
March 31, 2029
Last Updated
April 13, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will not share
This clinical trial evaluates treatment of a rare genetic disease in a small cohort of participants. No data is planned to be shared in order to protect and maintain participant privacy/confidentiality.