Muscular Atrophy, SpinalPhase 2RainbowfishNCT03779334
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
A Phase 2 study testing Risdiplam in people with Muscular Atrophy, Spinal, run by Hoffmann-La Roche at 7 sites in 7 countries.
Trial summary
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA). Read more about this study
- Who is it for?
- Ages 1 day to 6 weeks
- 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
- 26 participants
- Where
- 7 sitesin 7 countries
- Study dates
- 7 Aug 2019to 28 Feb 2027
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 day to 6 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Males and females aged from birth (1 day) to 6 weeks (42 days) of age at the time of first dose (Day 1); a minimum age of 7 days at first dose is required for the first infant to be enrolled
- Gestational age of 37-42 weeks for singleton births; gestational age of 34-42 weeks for twins
- Body weight >= 3rd percentile for age, using appropriate country-specific guidelines
- Genetic diagnosis of 5q-autosomal recessive SMA, including confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the SMN1 gene
- Absence of clinical signs or symptoms at screening (Day -42 to Day -2) or at baseline (Day -1) that are, in the opinion of the investigator, strongly suggestive of SMA
- Receiving adequate nutrition and hydration at the time of screening, in the opinion of the investigator
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- Adequately recovered from any acute illness at baseline and considered well enough to participate in the study, in the opinion of the investigator
- Able and expected to be able to safely travel to the study site for the entire duration of the study and in accordance to the frequency of required study visits, in the opinion of the investigator
- Able to complete all study procedures, measurements, and visits, and the parent (or caregiver), in the opinion of the investigator, has adequately supportive psychosocial circumstances
- Parent (or caregiver) is willing to consider nasogastric, naso-jejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator
- Parent (or caregiver) is willing to consider the use of non-invasive ventilation during the study, if recommended by the investigator
Who cannot join
- Concomitant or previous participation in any investigational drug or device study at any time
- Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide, SMN2-splicing modifier, or gene therapy either in a clinical study or as part of medical care
- Presence of significant concurrent syndromes or diseases
- In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures
- Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation
- Awake hypoxemia (SaO2 < 95%) with or without ventilator support
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- Multiple or fixed contractures and/or hip subluxation or dislocation at birth
- Systolic blood pressure or diastolic blood pressure or heart rate considered to be clinically significant by the investigator
- Presence of clinically relevant ECG abnormalities before study drug administration; corrected QT interval using Bazett's method > 460 ms; personal or family history (first degree relatives) of congenital long QT syndrome indicating a safety risk for patients as determined by the investigator. First-degree atrioventricular block or isolated right bundle branch block are allowed
- The infant (and the mother, if breastfeeding the infant) taking any inhibitor of CYP3A4 taken within 2 weeks, any inducer of CYP3A4 taken within 4 weeks, any OCT 2 and MATE substrates within 2 weeks and known FMO1 or FMO3 inhibitors or substrates
- Clinically significant abnormalities in laboratory test results
- Ascertained or presumptive hypersensitivity to risdiplam or to the constituents of its formulation
- Treatment with oral salbutamol or another beta-2 adrenergic agonist taken orally for SMA is not allowed. Use of inhaled beta-2 adrenergic agonists is allowed
- Infants exposed to drugs with known retinal toxicity given to mothers during pregnancy (and lactation) should not be enrolled. Anticipated need for drugs known to cause retinal toxicity during the study.
- Diagnosis of ophthalmic diseases
Trial locations and contacts
7 sites in 7 countries.
7 sites in 7 countries
Russia1 site
Russian Children Neuromuscular Center of Veltischev
Moscow, Moscow Oblast, 125412
Taiwan1 site
Kaohsiung Medical University Chung-Ho Hospital
Kaohsiung City, 807
About this study
Official title: An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
Read the detailed description
The study is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants aged from birth to 6 weeks who have been genetically diagnosed with SMA but are not yet presenting with symptoms. There will be a screening, treatment, open-label extension (OLE) and a follow-up. All participants will receive risdiplam orally once daily for 2 years followed by an OLE phase of at least 3 years and a follow-up (if applicable), for a total treatment duration of at least 5 years for each participant enrolled.
- 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 Evrysdi
Risdiplam will be administered orally.
| Study group | Role | What they receive |
|---|---|---|
| Open-label Risdiplam | Experimental | Participants will be enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range. Receives: Drug: Risdiplam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support
The Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. An exact binomial test was performed. If the lower limit of the two-sided 90% CI was above the 5% threshold, the primary objective of the study was considered achieved.
Measured: At Month 12
Secondary outcomes (27)
Percentage of Participants Developing Clinically Manifested SMA
Measured: At Month 12 and 24
Time to Permanent Ventilation and/or Death
Measured: Up to 7 years
Percentage of Participants Who Are Alive Without Permanent Ventilation
Measured: At Month 12 and 24
Percentage of Participants Alive
Measured: At Month 12 and 24
Percentage of Participants Who Achieve the Attainment Level of the Motor Milestones as Assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2)
HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking
Measured: At Month 12 and 24
Percentage of Participants With Two Copies of the SMN2 Gene Sitting Without Support for 5 Seconds (Independent of the CMAP Value at Baseline).
Assessed in Item 22 of the BSID-III Gross Motor Scale. The BSID-III is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method.
Measured: At Month 12
Percentage of Participants Sitting Without Support for 5 Seconds
Assessed with BSID-III Gross Motor Scale
Measured: At Month 24
Percentage of Participants Sitting Without Support for 30 Seconds
Assessed with BSID-III Gross Motor Scale
Measured: At Month 12 and 24
Percentage of Participants Standing for at Least 3 Seconds
Assessed with BSID-III Gross Motor Scale
Measured: At Month 24
Percentage of Participants Walking (Takes at Least 3 Steps)
Assessed with BSID-III Gross Motor Scale
Measured: At Month 24
Percentage of Participants Demonstrating the Ability to Achieve a Scaled Score on BSID-III Gross Motor Subtests Within 1.5 Standard Deviations of Chronological Reference Standard
Assessed through BSID-III Gross Motor Scale
Measured: At Month 24 and 42
Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12
The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. A positive change from baseline indicates an improvement.
Measured: Baseline, Month 12
Percentage of Participants Who Achieve a Score of 40 or Higher, 50 or Higher, and 60 or Higher in the CHOP INTEND Motor Function Scale at Month 12
The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. Data are presented with a two-sided 90% Clopper-Pearson (exact) CI for the proportion.
Measured: At Month 12
Percentage of Participants Who Meet CHOP INTEND Stopping Criteria at Any Point
Measured: Up to Month 24
Change From Baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) Score
Measured: At Month 60
Number and Percentage of Participants Within 3rd Percentile of Normal Range for Weight-for-Age, Length/Height-for-Age and Weight-for-Length/Height
Based on the WHO Child Growth Standards (WHO 2019)
Measured: At Month 12, 24, 36, 48 and 60
Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age
Based on the WHO Child Growth Standards (WHO 2019)
Measured: At Month 12 and 24
Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height
Measured: At Month 12, 24, 36, 48 and 60
Change From Baseline Percentiles for Head Circumference- For-age
Measured: At Month 12 and 24
Change From Baseline in Chest Circumference
Measured: At Month 12 and 24
Ratio Between Chest and Head Circumferences
Measured: At Month 12 and 24
Percentage of Participants With the Ability to Swallow and to Feed Orally
Measured: At Month 12, 24, 36, 48 and 60
Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitude
Measured by CMAP
Measured: At Month 12 and 24
Measurement of Pharmacodynamic Marker Levels in Blood
Measured: Day 1, 56, 196, 364, 728 and at early withdrawal
Percentage of Participants With Adverse Events
Adverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5
Measured: Up to 7 years
Ophthalmological Examination as Appropriate for Age
Measured: Up to 7 years
Plasma Concentration of Risdiplam and Its Metabolites to Characterize the PK Profile
Measured: Up to 7 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2018
- Study start7 Aug 2019
- Primary completion20 Feb 2023
- Study completion28 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label, single arm
- Masking
- None (open label)
- Enrolment
- 26 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
- NCT03779334
- Sponsor's protocol ID
- BN40703
- Other IDs
- 2018-002087-12, 2023-506009-20-00, 2023, 2018
- Acronym
- Rainbowfish
- Status last verified
- Oct 2026
- First submitted
- 17 Dec 2018
- Last updated
- 8 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Publications
Finkel RS, Servais L, Vlodavets D, Zanoteli E, Mazurkiewicz-Beldzinska M, Jong YJ, Navas-Nazario A, Al-Muhaizea M, Araujo APQC, Nelson L, Wang Y, Jaber B, Gorni K, Kletzl H, Palfreeman L, Rabbia M, Summers D, Gaki E, Wagner KR, Fontoura P, Farrar MA, Bertini E; RAINBOWFISH Study Group. Risdiplam in Presymptomatic Spinal Muscular Atrophy. N Engl J Med. 2025 Aug 14;393(7):671-682. doi: 10.1056/NEJMoa2410120.
PubMed 40802943
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT03779334
- Acronym / sponsor ID
- Rainbowfish / BN40703
- Conditions
- Muscular Atrophy, Spinal
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 7 Aug 2019 (actual)
- Primary completion
- 20 Feb 2023 (actual)
- Study completion
- 28 Feb 2027 (estimated)
- First posted
- 19 Dec 2018 (actual)
- Results
- Posted 5 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
Detailed description
The study is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants aged from birth to 6 weeks who have been genetically diagnosed with SMA but are not yet presenting with symptoms. There will be a screening, treatment, open-label extension (OLE) and a follow-up. All participants will receive risdiplam orally once daily for 2 years followed by an OLE phase of at least 3 years and a follow-up (if applicable), for a total treatment duration of at least 5 years for each participant enrolled.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label, single arm
| Arm | Type | Receives |
|---|---|---|
| Open-label Risdiplam | Experimental | Drug: RisdiplamParticipants will be enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range. |
Who may qualify
- Ages
- 1 day to 6 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- Males and females aged from birth (1 day) to 6 weeks (42 days) of age at the time of first dose (Day 1); a minimum age of 7 days at first dose is required for the first infant to be enrolled
- Gestational age of 37-42 weeks for singleton births; gestational age of 34-42 weeks for twins
- Body weight >= 3rd percentile for age, using appropriate country-specific guidelines
- Genetic diagnosis of 5q-autosomal recessive SMA, including confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the SMN1 gene
- Absence of clinical signs or symptoms at screening (Day -42 to Day -2) or at baseline (Day -1) that are, in the opinion of the investigator, strongly suggestive of SMA
Key exclusion criteria (15 listed)
- Concomitant or previous participation in any investigational drug or device study at any time
- Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide, SMN2-splicing modifier, or gene therapy either in a clinical study or as part of medical care
- Presence of significant concurrent syndromes or diseases
- In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures
- Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RisdiplamAlso: EvrysdiRisdiplam will be administered orally. | Drug | Open-label Risdiplam |
Primary outcome
Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support
Time frame: At Month 12
The Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. An exact binomial test was performed. If the lower limit of the two-sided 90% CI was above the 5% threshold, the primary objective of the study was considered achieved.
27 secondary outcomes
Percentage of Participants Developing Clinically Manifested SMA
Time frame: At Month 12 and 24
Time to Permanent Ventilation and/or Death
Time frame: Up to 7 years
Percentage of Participants Who Are Alive Without Permanent Ventilation
Time frame: At Month 12 and 24
Percentage of Participants Alive
Time frame: At Month 12 and 24
Percentage of Participants Who Achieve the Attainment Level of the Motor Milestones as Assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2)
Time frame: At Month 12 and 24
Percentage of Participants With Two Copies of the SMN2 Gene Sitting Without Support for 5 Seconds (Independent of the CMAP Value at Baseline).
Time frame: At Month 12
Percentage of Participants Sitting Without Support for 5 Seconds
Time frame: At Month 24
Percentage of Participants Sitting Without Support for 30 Seconds
Time frame: At Month 12 and 24
Percentage of Participants Standing for at Least 3 Seconds
Time frame: At Month 24
Percentage of Participants Walking (Takes at Least 3 Steps)
Time frame: At Month 24
Percentage of Participants Demonstrating the Ability to Achieve a Scaled Score on BSID-III Gross Motor Subtests Within 1.5 Standard Deviations of Chronological Reference Standard
Time frame: At Month 24 and 42
Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12
Time frame: Baseline, Month 12
Percentage of Participants Who Achieve a Score of 40 or Higher, 50 or Higher, and 60 or Higher in the CHOP INTEND Motor Function Scale at Month 12
Time frame: At Month 12
Percentage of Participants Who Meet CHOP INTEND Stopping Criteria at Any Point
Time frame: Up to Month 24
Change From Baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) Score
Time frame: At Month 60
Number and Percentage of Participants Within 3rd Percentile of Normal Range for Weight-for-Age, Length/Height-for-Age and Weight-for-Length/Height
Time frame: At Month 12, 24, 36, 48 and 60
Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age
Time frame: At Month 12 and 24
Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height
Time frame: At Month 12, 24, 36, 48 and 60
Change From Baseline Percentiles for Head Circumference- For-age
Time frame: At Month 12 and 24
Change From Baseline in Chest Circumference
Time frame: At Month 12 and 24
Ratio Between Chest and Head Circumferences
Time frame: At Month 12 and 24
Percentage of Participants With the Ability to Swallow and to Feed Orally
Time frame: At Month 12, 24, 36, 48 and 60
Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitude
Time frame: At Month 12 and 24
Measurement of Pharmacodynamic Marker Levels in Blood
Time frame: Day 1, 56, 196, 364, 728 and at early withdrawal
Percentage of Participants With Adverse Events
Time frame: Up to 7 years
Ophthalmological Examination as Appropriate for Age
Time frame: Up to 7 years
Plasma Concentration of Risdiplam and Its Metabolites to Characterize the PK Profile
Time frame: Up to 7 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40802943Finkel RS, Servais L, Vlodavets D, Zanoteli E, Mazurkiewicz-Beldzinska M, Jong YJ, Navas-Nazario A, Al-Muhaizea M, Araujo APQC, Nelson L, Wang Y, Jaber B, Gorni K, Kletzl H, Palfreeman L, Rabbia M, Summers D, Gaki E, Wagner KR, Fontoura P, Farrar MA, Bertini E; RAINBOWFISH Study Group. Risdiplam in Presymptomatic Spinal Muscular Atrophy. N Engl J Med. 2025 Aug 14;393(7):671-682. doi: 10.1056/NEJMoa2410120.
- Individual participant dataSharing planned: Yes. For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
7 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Risdiplam in people with Muscular Atrophy, Spinal, run by Hoffmann-La Roche at 7 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Australia, Belgium, Brazil, Poland and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support (time frame: At Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03779334, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2026.