- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05861986
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy (HINALEA 1)
March 29, 2024 updated by: Hoffmann-La Roche
A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
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.
Study Overview
Study Type
Interventional
Enrollment (Estimated)
28
Phase
- Phase 4
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Reference Study ID Number: BN44620 https://forpatients.roche.com/
- Phone Number: 888-662-6728 (U.S. Only)
- Email: global-roche-genentech-trials@gene.com
Study Locations
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Arkansas
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Little Rock, Arkansas, United States, 72103
- Recruiting
- University of Arkansas for Medical Sciences
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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.
Eligibility Criteria
Ages Eligible for Study
- Child
Accepts Healthy Volunteers
No
Description
Inclusion 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, but not more than 7 months, prior to enrollment
- Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration
Exclusion Criteria:
- 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
- Requiring awake non-invasive ventilation or with awake hypoxemia (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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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.
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Participants will receive risdiplam orally at the currently approved dose.
The dose should be adapted for weight and age.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
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Baseline, Week 72
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Percentage of Participants With Adverse Events
Time Frame: Up to 120 weeks
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Up to 120 weeks
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Percentage of Participants With Serious Adverse Events
Time Frame: Up to 120 weeks
|
Up to 120 weeks
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Percentage of Participants With Treatment Discontinuation Due to Adverse Events
Time Frame: Up to 120 weeks
|
Up to 120 weeks
|
Other Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Change in Swallowing Function Assessment as Measured by the Pediatric Functional Oral Intake Scale (p-FOIS) at 72 Weeks of Risdiplam Treatment and Over Time
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Percentage of Participants With a Gross Motor Index Between 80-109 as Measured by the Peabody Developmental Motor Scale, Third Edition (PDMS-3) at 72 Weeks of Risdiplam Treatment and Over Time
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Percentage of Participants With a Fine Motor Index Between 80-109 as Measured by the PDMS-3 at 72 Weeks of Risdiplam Treatment and Over Time
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Change in World Health Organization (WHO) Motor Milestone Achievement at 72 Weeks of Risdiplam Treatment and Over Time
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Percentage of Participants With Improvement or No Change in Respiratory Illness as Assessed by Clinical Global Impression of Change (CGI-C)
Time Frame: As per respiratory event on Day 10 and Day 20 post-event (up to Week 120)
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As per respiratory event on Day 10 and Day 20 post-event (up to Week 120)
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Percentage of Participants Within 3rd Percentile of Normal Range for Weight-to-Age at 72 Weeks of Risdiplam Treatment and Over Time
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Percentage of Participants Within 3rd Percentile of Normal Range for Length/Height-to-Age at 72 Weeks of Risdiplam Treatment and Over Time
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Percentage of Participants Within 3rd Percentile of Normal Range for Weight-to-Length/Height at 72 Weeks of Risdiplam Treatment and Over Time
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Number of Respiratory-Related Hospitalizations During the 72-Week Risdiplam Treatment and Over Time
Time Frame: Up to 120 weeks
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Up to 120 weeks
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Change from Baseline in the Raw Score of BSID-III Gross Motor Score Over Time Under Risdiplam Treatment
Time Frame: From baseline up to Week 120
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From baseline up to Week 120
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Clinical Trials, Hoffmann-La Roche
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Estimated)
April 29, 2024
Primary Completion (Estimated)
January 31, 2027
Study Completion (Estimated)
March 31, 2028
Study Registration Dates
First Submitted
May 8, 2023
First Submitted That Met QC Criteria
May 8, 2023
First Posted (Actual)
May 17, 2023
Study Record Updates
Last Update Posted (Actual)
April 1, 2024
Last Update Submitted That Met QC Criteria
March 29, 2024
Last Verified
March 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neuromuscular Manifestations
- Pathological Conditions, Anatomical
- Spinal Cord Diseases
- Motor Neuron Disease
- Muscular Atrophy
- Atrophy
- Muscular Atrophy, Spinal
- Physiological Effects of Drugs
- Peripheral Nervous System Agents
- Neuromuscular Agents
- Risdiplam
Other Study ID Numbers
- BN44620
- 2023-504508-26-00 (Registry Identifier: EU CT number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
IPD Plan Description
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.
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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