Spinal Muscular AtrophyPhase 3SAPPHIRENCT05156320
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
A Phase 3 study testing Apitegromab in people with Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, run by Scholar Rock, Inc. at 50 sites in 9 countries.
Trial summary
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam)… Read more about this study
- Who is it for?
- Ages 2 to 21 years
- Conditions the study is for
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2
- What is being tested?
- Apitegromab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 188 participants
- Where
- 50 sitesin 9 countries
- Study dates
- 14 Apr 2022to 18 Dec 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 2 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Males and females 2 through 21 years old at Screening.
- Documented diagnosis of 5q SMA.
- Diagnosed with later-onset SMA (i.e., Type 2 and Type 3 SMA) before receiving an approved SMN upregulator therapy (i.e., either nusinersen or risdiplam).
- Must be Nonambulatory at Screening. Nonambulatory patients must be able to sit independently (sits up straight with head erect for at least 10 seconds; does not use arms or hands to balance body or support position) per World Health Organization (WHO) motor milestones definition at Screening.
- Receiving one background therapy for SMA (i.e., either nusinersen or risdiplam) for the time period specified below and anticipated to remain on that same treatment throughout the trial:
- If receiving the SMN upregulator therapy nusinersen, must have completed at least 10 months of dosing (i.e., completed the loading regimen and at least 2 maintenance doses) before Screening;
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- If receiving the SMN upregulator therapy risdiplam, must have completed at least 6 months of dosing before Screening.
- Motor Function Score (HFMSE) ≥10 and ≤45 at Screening.
- Have no physical limitations that would prevent the patient from undergoing motor function outcome measures throughout the duration of the study.
- Able to receive study drug infusions and provide blood samples through the use of a peripheral intravenous (IV) or a long-term IV access device that the patient has placed for reasons independent from the study throughout the duration of the study.
- Able to adhere to the requirements of the protocol, including travel to the study center and completing all study procedures and study visits.
- For patients who are expected to have reached reproductive maturity by the end of the study, adhere to study specific contraception requirements.
Who cannot join
- Received ZOLGENSMA® (onasemnogene abeparvovec-xioi) at any time and previous treatment with apitegromab.
- Use of invasive ventilation and tracheostomy.
- Use of chronic daytime non-invasive ventilatory support for >16 hours daily in the 2 weeks prior to dosing, or anticipated to regularly receive such daytime ventilator support chronically over the duration of the study.
- Any acute or co-morbid condition interfering with the well-being of the patient within 7 days of screening, including active systemic infection, the need for acute treatment or inpatient observation due to any reason.
- Severe scoliosis and/or contractures at screening. Based on clinical judgement, any scoliosis or contractures present must be stable over the past 6 months, anticipated to be stable for the duration of the study and not prevent the patient from being evaluated on any functional outcome measures throughout the duration of the study.
- Pregnant or breastfeeding.
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- Major orthopedic or other interventional procedure, including spine or hip surgery, considered to have the potential to substantially limit the ability of the patient to be evaluated on any functional outcome measures, within 6 months prior to Screening, or anticipated for the duration of the study.
- Prior history of a hypersensitivity reaction to a monoclonal antibody (mAb) or recombinant protein bearing an Fc domain (such as a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab.
- Treatment with investigational drugs within 3 months prior to Screening.
- Use of therapies with potentially significant muscle effects (such as androgens, insulin-like growth factor, growth hormone, systemic beta-agonist, botulinum toxin, or muscle relaxants or muscle-enhancing supplements) or potentially significant neuromuscular effects (such as acetylcholinesterase inhibitors) within 60 days prior to screening.
- Nutritional status not stable over the past 6 months and not anticipated to be stable throughout the duration of the study.
- Patient has any other condition, which in the opinion of the Investigator may compromise safety or compliance, would preclude the patient from successful completion of the study, or interfere with the interpretation of the results.
Trial locations and contacts
50 sites in 9 countries.
50 sites in 9 countries
United States26 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
The Johns Hopkins University
Baltimore, Maryland, 21287
Children's of Alabama
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Medical Center Dallas
Dallas, Texas, 75235
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Children's Hospital of Los Angeles
Los Angeles, California, 90027
University of Wisconsin School of Medicine and Public Health
Madison, Wisconsin, 53705
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Columbia University, SMA Clinical Research Center
New York, New York, 10032
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
Nemours Children's Hospital
Orlando, Florida, 32827
Stanford University Medical Center
Palo Alto, California, 94304
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Oregon Health & Sciences University
Portland, Oregon, 97239
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
University of Utah
Salt Lake City, Utah, 84112
Rady's Children's Hospital/UCSD
San Diego, California, 92123
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
Italy5 sites
IRCCS Istituto Giannina Gaslini
Genoa
A.O.U Policlinico G. Martino
Messina, 98125
Foundation I.R.C.C.S. Carlo Besta Neurological Institute
Milan, 20133
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162
Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore
Roma, 00168
Germany4 sites
Universitätsklinikum Bonn
Bonn, 53127
Universitätsklinikum Essen
Essen, 45147
Universitaetsklinikum Freiburg
Freiburg im Breisgau, 79106
Dr. von Haunersches Kinderspital
Munich, 80337
Belgium3 sites
UZ Gent
Ghent, 9000
UZ Leuven
Leuven, 3000
Chr de La Citadelle
Liège, 4000
France3 sites
CHU Lille - Hôpital Jeanne de Flandre
Lille, 59037
Hôpital Armand Trousseau, I-Motion
Paris, 75012
CHU Toulouse - Hopital des Enfants
Toulouse, 31059
Poland3 sites
Uniwersyteckie Centrum Kliniczne w Gdańsku
Gdansk, 80-001
Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu
Poznan, 61-701
Instytut Pomnik - Centrum Zdrowia Dziecka: CZD Warszawa
Warsaw, 04-736
United Kingdom3 sites
Leeds Teaching Hospitals NHS Trust
Leeds, LS1 3EX
Great Ormond Street Hospital for Children NHS Foundation Trust
London
University of Oxford
Oxford, OX3 0ER
Spain2 sites
Hospital Sant Joan de Déu
Barcelona
Hospital Universitario y Politécnico La Fe
Valencia
About this study
Official title: Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy
- Being tested
- Apitegromab, Placebo
- Medical subject headings
- Muscular Atrophy, Spinal, Spinal Muscular Atrophies of Childhood, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Neuromuscular Manifestations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ApitegromabDrug
Also called SRK-015
Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 was administered every 4 weeks by intravenous (IV) infusion.
PlaceboDrug
Placebo was administered every 4 weeks by intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| Main Efficacy Population (Apitegromab 10 mg/kg) | Experimental | Aged 2-12 years at Screening. Participants were randomized to receive apitegromab 10 mg/kg for up to 52 weeks. Receives: Drug: Apitegromab |
| Main Efficacy Population (Apitegromab 20 mg/kg) | Experimental | Aged 2-12 years at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks. Receives: Drug: Apitegromab |
| Main Efficacy Population (Placebo) | Placebo comparator | Aged 2-12 years at Screening. Participants were randomized to receive placebo for up to 52 weeks. Receives: Drug: Placebo |
| Exploratory Subpopulation (Apitegromab) | Experimental | Aged 13-21 years at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks. Receives: Drug: Apitegromab |
| Exploratory Subpopulation (Placebo) | Placebo comparator | Aged 13-21 years at Screening. Participants were randomized to receive placebo for up to 52 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.
The HFMSE assesses the physical abilities of patients with Type 2 and Type 3 SMA. It comprises of 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function.
Measured: Baseline up to 12 months.
Secondary outcomes (7)
Main Efficacy Population: Change from Baseline in Revised Upper Limb Module (RULM) total score.
The RULM is a 20 item assessment of upper limb function in nonambulatory patients with SMA that was performed for patients who were 30 months of age or older at baseline. The 19 scored items assess functions that relate to everyday life, such as pressing a button and picking up a token; these items are scored 0, 1, or 2, where 0 denotes unable, 1 denotes able with modification, and 2 denotes able with no modification. The maximum score achievable is 37. Higher scores increased great upper limb function.
Measured: Baseline up to 12 months.
Main Efficacy Population: Proportion of patients with ≥3-point change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.
The HFMSE assesses the physical abilities of patients with Type 2 and Type 3 SMA. It comprises of 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function.
Measured: Baseline up to 12 months.
Main Efficacy Population: Change from Baseline in number of WHO motor development milestones attained at 12 months.
Measured: Baseline up to 12 months.
Main Efficacy Population and Exploratory Subpopulation combined: Incidence of Treatment Emergent Adverse Events (TEAEs) and Severe Adverse Events (SAEs) by severity.
Measured: Baseline up to 12 months.
Main Efficacy Population and Exploratory Subpopulation combined: Apitegromab concentrations in serum from blood samples.
Measured: Baseline up to 12 months.
Main Efficacy Population and Exploratory Subpopulation combined: Circulating latent myostatin concentrations in blood samples.
Measured: Baseline up to 12 months.
Main Efficacy Population and Exploratory Subpopulation combined: Presence or absence of Antidrug Antibody (ADA) against apitegromab in serum from blood samples.
Measured: Baseline up to 12 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2021
- Study start14 Apr 2022
- Primary completion18 Dec 2024
- Study completion18 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 188 participants (actual)
Sponsor and investigators
- Lead sponsor
- Scholar Rock, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05156320
- Sponsor's protocol ID
- SRK-015-003
- Acronym
- SAPPHIRE
- Status last verified
- Jan 2026
- First submitted
- 1 Dec 2021
- Last updated
- 22 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Crawford TO, Servais L, Mercuri E, Kolbel H, Kuntz N, Finkel RS, Krueger J, Batley K, Young SD, Marantz JL, Song G, Yao B, Zhao G, Rossello J, Tirucherai GS, Mazzone ES, Butterfield RJ, de la Banda MGG, Seferian AM, Sansone VA, De Waele L, van der Pol WL, Cances C, Pechmann A, Darras BT; SAPPHIRE Study Group. Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2025 Sep;24(9):727-739. doi: 10.1016/S1474-4422(25)00225-X.
PubMed 40818473
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy
- ClinicalTrials.gov ID
- NCT05156320
- Acronym / sponsor ID
- SAPPHIRE / SRK-015-003
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 188 participants (actual)
- Lead sponsor
- Scholar Rock, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Apr 2022 (actual)
- Primary completion
- 18 Dec 2024 (actual)
- Study completion
- 18 Dec 2024 (actual)
- First posted
- 14 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Main Efficacy Population (Apitegromab 10 mg/kg) | Experimental | Drug: ApitegromabAged 2-12 years at Screening. Participants were randomized to receive apitegromab 10 mg/kg for up to 52 weeks. |
| Main Efficacy Population (Apitegromab 20 mg/kg) | Experimental | Drug: ApitegromabAged 2-12 years at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks. |
| Main Efficacy Population (Placebo) | Placebo comparator | Drug: PlaceboAged 2-12 years at Screening. Participants were randomized to receive placebo for up to 52 weeks. |
| Exploratory Subpopulation (Apitegromab) | Experimental | Drug: ApitegromabAged 13-21 years at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks. |
| Exploratory Subpopulation (Placebo) | Placebo comparator | Drug: PlaceboAged 13-21 years at Screening. Participants were randomized to receive placebo for up to 52 weeks. |
Who may qualify
- Ages
- 2 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- Males and females 2 through 21 years old at Screening.
- Documented diagnosis of 5q SMA.
- Diagnosed with later-onset SMA (i.e., Type 2 and Type 3 SMA) before receiving an approved SMN upregulator therapy (i.e., either nusinersen or risdiplam).
- Must be Nonambulatory at Screening. Nonambulatory patients must be able to sit independently (sits up straight with head erect for at least 10 seconds; does not use arms or hands to balance body or support position) per World Health Organization (WHO) motor milestones definition at Screening.
- Receiving one background therapy for SMA (i.e., either nusinersen or risdiplam) for the time period specified below and anticipated to remain on that same treatment throughout the trial:
Key exclusion criteria (12 listed)
- Received ZOLGENSMA® (onasemnogene abeparvovec-xioi) at any time and previous treatment with apitegromab.
- Use of invasive ventilation and tracheostomy.
- Use of chronic daytime non-invasive ventilatory support for >16 hours daily in the 2 weeks prior to dosing, or anticipated to regularly receive such daytime ventilator support chronically over the duration of the study.
- Any acute or co-morbid condition interfering with the well-being of the patient within 7 days of screening, including active systemic infection, the need for acute treatment or inpatient observation due to any reason.
- Severe scoliosis and/or contractures at screening. Based on clinical judgement, any scoliosis or contractures present must be stable over the past 6 months, anticipated to be stable for the duration of the study and not prevent the patient from being evaluated on any functional outcome measures throughout the duration of the study.
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 |
|---|---|---|
| ApitegromabAlso: SRK-015Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 was administered every 4 weeks by intravenous (IV) infusion. | Drug | Exploratory Subpopulation (Apitegromab), Main Efficacy Population (Apitegromab 10 mg/kg), Main Efficacy Population (Apitegromab 20 mg/kg) |
| PlaceboPlacebo was administered every 4 weeks by intravenous (IV) infusion. | Drug | Exploratory Subpopulation (Placebo), Main Efficacy Population (Placebo) |
Primary outcome
Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.
Time frame: Baseline up to 12 months.
The HFMSE assesses the physical abilities of patients with Type 2 and Type 3 SMA. It comprises of 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function.
7 secondary outcomes
Main Efficacy Population: Change from Baseline in Revised Upper Limb Module (RULM) total score.
Time frame: Baseline up to 12 months.
Main Efficacy Population: Proportion of patients with ≥3-point change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.
Time frame: Baseline up to 12 months.
Main Efficacy Population: Change from Baseline in number of WHO motor development milestones attained at 12 months.
Time frame: Baseline up to 12 months.
Main Efficacy Population and Exploratory Subpopulation combined: Incidence of Treatment Emergent Adverse Events (TEAEs) and Severe Adverse Events (SAEs) by severity.
Time frame: Baseline up to 12 months.
Main Efficacy Population and Exploratory Subpopulation combined: Apitegromab concentrations in serum from blood samples.
Time frame: Baseline up to 12 months.
Main Efficacy Population and Exploratory Subpopulation combined: Circulating latent myostatin concentrations in blood samples.
Time frame: Baseline up to 12 months.
Main Efficacy Population and Exploratory Subpopulation combined: Presence or absence of Antidrug Antibody (ADA) against apitegromab in serum from blood samples.
Time frame: Baseline up to 12 months.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2026.
- Derived from this study: PubMed 40818473Crawford TO, Servais L, Mercuri E, Kolbel H, Kuntz N, Finkel RS, Krueger J, Batley K, Young SD, Marantz JL, Song G, Yao B, Zhao G, Rossello J, Tirucherai GS, Mazzone ES, Butterfield RJ, de la Banda MGG, Seferian AM, Sansone VA, De Waele L, van der Pol WL, Cances C, Pechmann A, Darras BT; SAPPHIRE Study Group. Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2025 Sep;24(9):727-739. doi: 10.1016/S1474-4422(25)00225-X.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
50 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Apitegromab in people with Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, run by Scholar Rock, Inc. at 50 sites in 9 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 12 inclusion and 12 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: finished, as of the sponsor's last update on 22 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, Italy, Germany, Belgium, France and 4 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score. (time frame: Baseline up to 12 months.). 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 NCT05156320, 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 22 Jan 2026.