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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)

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ClinicalTrials.gov Identifier: NCT05861999
Recruitment Status : Not yet recruiting
First Posted : May 17, 2023
Last Update Posted : February 9, 2024
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

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.

Condition or disease Intervention/treatment Phase
Muscular Atrophy, Spinal Drug: risdiplam Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 28 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Study Start Date : March 12, 2024
Estimated Primary Completion Date : January 31, 2027
Estimated Study Completion Date : March 31, 2028


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.
Drug: risdiplam
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
Other Name: RO7034067




Primary Outcome Measures :
  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 [ Time Frame: Baseline, Week 72 ]

Other Outcome Measures:
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. 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 ]
  5. 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 ]
  6. 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 postevent (up to Week 120) ]
  7. 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 ]
  8. 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 ]
  9. 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 ]
  10. Number of Respiratory-Related Hospitalizations During the 72-Week Risdiplam Treatment and Over Time [ Time Frame: Up to 120 weeks ]
  11. Percentage of Participants With Adverse Events [ Time Frame: Up to 120 weeks ]
  12. Percentage of Participants With Serious Adverse Events [ Time Frame: Up to 120 weeks ]
  13. Percentage of Participants With Treatment Discontinuation Due to Adverse Events [ Time Frame: Up to 120 weeks ]


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Ages Eligible for Study:   3 Months to 24 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 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.

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
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05861999


Contacts
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Contact: Reference Study ID Number: BN44621 https://forpatients.roche.com/ 888-662-6728 (U.S. Only) global-roche-genentech-trials@gene.com

Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT05861999    
Other Study ID Numbers: BN44621
2023-505161-81-00 ( Registry Identifier: EU CT number )
First Posted: May 17, 2023    Key Record Dates
Last Update Posted: February 9, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
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.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Risdiplam
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs