Condition or disease | Intervention/treatment | Phase |
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Spinal Muscular Atrophy Spinal Muscular Atrophy Type 3 Spinal Muscular Atrophy Type 2 SMA Neuromuscular Diseases Muscular Atrophy Atrophy Muscular Atrophy, Spinal Neuromuscular Manifestations | Biological: SRK-015 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 58 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Masking Description: | Active treatment, randomized, double-blind for Cohort 3 |
Primary Purpose: | Treatment |
Official Title: | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ) |
Actual Study Start Date : | April 22, 2019 |
Actual Primary Completion Date : | January 12, 2021 |
Estimated Study Completion Date : | April 2023 |
Arm | Intervention/treatment |
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Experimental: Cohort 1
Ambulatory Type 3 SMA
|
Biological: SRK-015
SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 will be administered every 4 weeks by intravenous infusion.
|
Experimental: Cohort 2
Type 2 SMA / Non-Ambulatory Type 3 SMA
|
Biological: SRK-015
SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 will be administered every 4 weeks by intravenous infusion.
|
Experimental: Cohort 3
Type 2 SMA
|
Biological: SRK-015
SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 will be administered every 4 weeks by intravenous infusion.
|
Ages Eligible for Study: | 2 Years to 21 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Receiving the same background SMA therapy (e.g., on an approved survival motor neuron (SMN) upregulator therapy such as nusinersen, or not on any SMA therapy) for at least 6 months prior to screening and anticipated to remain on that therapy throughout the duration of the study.
Exclusion Criteria:
United States, Arizona | |
Phoenix Children's Hospital | |
Phoenix, Arizona, United States, 85016 | |
United States, California | |
Stanford University | |
Palo Alto, California, United States, 94304 | |
United States, Colorado | |
Children's Hospital Colorado | |
Aurora, Colorado, United States, 80045 | |
United States, Maryland | |
The Johns Hopkins University | |
Baltimore, Maryland, United States, 21287 | |
United States, Massachusetts | |
Boston Children's Hospital | |
Boston, Massachusetts, United States, 02115 | |
United States, Michigan | |
Helen DeVos Children's Hospital | |
Grand Rapids, Michigan, United States, 49503 | |
United States, New York | |
Columbia University | |
New York, New York, United States, 10032 | |
United States, North Carolina | |
Wake Forest School of Medicine | |
Winston-Salem, North Carolina, United States, 27157 | |
United States, Oregon | |
Oregon Health & Science University | |
Portland, Oregon, United States, 97239 | |
United States, Texas | |
Childrens Medical Center Dallas | |
Dallas, Texas, United States, 75235 | |
United States, Virginia | |
Children's Hospital of The King's Daughters | |
Norfolk, Virginia, United States, 23507 | |
Italy | |
ASST Grande Ospedale Metropolitano Niguarda | |
Milano, Italy, 20162 | |
Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore | |
Roma, Italy | |
Netherlands | |
Universitair Medisch Centrum Utrecht | |
Utrecht, Netherlands, 3584 | |
Spain | |
Hospital Sant Joan de Déu | |
Barcelona, Spain | |
Hospital Universitari i Politecnic La Fe | |
Valencia, Spain, 46026 |
Principal Investigator: | Thomas O. Crawford, MD | Johns Hopkins University |
Tracking Information | |||||
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First Submitted Date ICMJE | April 16, 2019 | ||||
First Posted Date ICMJE | April 19, 2019 | ||||
Last Update Posted Date | March 17, 2021 | ||||
Actual Study Start Date ICMJE | April 22, 2019 | ||||
Actual Primary Completion Date | January 12, 2021 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
|
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||
Original Secondary Outcome Measures ICMJE | Not Provided | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy | ||||
Official Title ICMJE | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ) | ||||
Brief Summary | The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients. | ||||
Detailed Description | Not Provided | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 2 | ||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Masking Description: Active treatment, randomized, double-blind for Cohort 3 Primary Purpose: Treatment
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Condition ICMJE |
|
||||
Intervention ICMJE | Biological: SRK-015
SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 will be administered every 4 weeks by intravenous infusion.
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Actual Enrollment ICMJE |
58 | ||||
Original Estimated Enrollment ICMJE |
55 | ||||
Estimated Study Completion Date ICMJE | April 2023 | ||||
Actual Primary Completion Date | January 12, 2021 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE |
Inclusion Criteria:
Exclusion Criteria:
|
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Sex/Gender ICMJE |
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Ages ICMJE | 2 Years to 21 Years (Child, Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Italy, Netherlands, Spain, United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT03921528 | ||||
Other Study ID Numbers ICMJE | SRK-015-002 | ||||
Has Data Monitoring Committee | Not Provided | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Scholar Rock, Inc. | ||||
Study Sponsor ICMJE | Scholar Rock, Inc. | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Scholar Rock, Inc. | ||||
Verification Date | March 2021 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |