Muscular Atrophy, SpinalPhase 4HINALEA 2NCT05861999
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
A Phase 4 study testing Risdiplam in people with Muscular Atrophy, Spinal, run by Hoffmann-La Roche at 17 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 24 months
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- Risdiplam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 17 sitesin 6 countries
- Study dates
- 14 Aug 2024to 31 Mar 2029
- Phase
- Phase 4
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- <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.
Who cannot join
- 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.
Trial locations and contacts
17 sites in 6 countries.
17 sites in 6 countries
United States8 sites
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, 30329-2309
Children's Hospital of Colorado
Aurora, Colorado, 80045
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
University of Florida Pediatrics
Gainesville, Florida, 32610
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, 49503
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72103
Valley Children's Hospital
Madera, California, 93636
Stanford Univ Medical Center
Palo Alto, California, 94304
Israel3 sites
Soroka Medical Center
Beersheba, 8410101
Schneider Children's Medical Center of Israel
Petah Tikva, 4920235
Sourasky MC, Dana-Dwek Children's Hospital
Tel Aviv, 6423906
Germany2 sites
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
Berlin, 13353
UKGM Standort Gießen
Giessen, 35392
Poland2 sites
Uniwersyteckie Centrum Kliniczne
Gda?sk, 80-952
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730
United Kingdom1 site
Great Ormond Street Hospital For Children
London, WC1N 3JH
About this study
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
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
- Being tested
- Risdiplam
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RisdiplamDrug
Also called RO7034067
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
| Study group | Role | What they receive |
|---|---|---|
| Risdiplam | Experimental | 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. Receives: Drug: Risdiplam |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, Week 72
Secondary outcomes (3)
Percentage of Participants With Adverse Events
Measured: Up to 120 weeks
Percentage of Participants With Serious Adverse Events
Measured: Up to 120 weeks
Percentage of Participants With Treatment Discontinuation Due to Adverse Events
Measured: Up to 120 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2023
- Study start14 Aug 2024
- Primary completion31 Mar 2028 (estimated)
- Study completion31 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05861999
- Sponsor's protocol ID
- BN44621
- Other IDs
- 2023-505161-81-00, 2023
- Acronym
- HINALEA 2
- Status last verified
- Oct 2026
- First submitted
- 8 May 2023
- Last updated
- 22 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.