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An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy (TOPAZ)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03921528
Recruitment Status : Active, not recruiting
First Posted : April 19, 2019
Last Update Posted : January 30, 2020
Sponsor:
Information provided by (Responsible Party):
Scholar Rock, Inc.

Tracking Information
First Submitted Date  ICMJE April 16, 2019
First Posted Date  ICMJE April 19, 2019
Last Update Posted Date January 30, 2020
Actual Study Start Date  ICMJE April 22, 2019
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 16, 2019)
  • Cohort 1: Change from Baseline in the Revised Hammersmith Scale (RHS) [ Time Frame: Baseline up to 12 months ]
  • Cohort 2 and Cohort 3: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) [ Time Frame: Baseline up to 12 months ]
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
Condition  ICMJE
  • Spinal Muscular Atrophy
  • Spinal Muscular Atrophy Type 3
  • Spinal Muscular Atrophy Type 2
  • SMA
  • Neuromuscular Diseases
  • Muscular Atrophy
  • Atrophy
  • Muscular Atrophy, Spinal
  • Neuromuscular Manifestations
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.
Study Arms  ICMJE
  • Experimental: Cohort 1
    Ambulatory Type 3 SMA
    Intervention: Biological: SRK-015
  • Experimental: Cohort 2
    Type 2 SMA / Non-Ambulatory Type 3 SMA
    Intervention: Biological: SRK-015
  • Experimental: Cohort 3
    Type 2 SMA
    Intervention: Biological: SRK-015
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: January 29, 2020)
58
Original Estimated Enrollment  ICMJE
 (submitted: April 16, 2019)
55
Estimated Study Completion Date  ICMJE April 2021
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Age 5 through 21 years old at the time of screening for Cohorts 1 and 2; Age ≥2 years old at the time of screening for Cohort 3.
  • Documented diagnosis of 5q SMA.
  • Diagnosed as later-onset (e.g., Type 2 or Type 3) SMA prior to receiving any treatment with therapy approved for SMA.
  • Non-ambulatory 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.
  • Ambulatory patients must have the ability to independently ambulate without aids or orthotics over 10 meters in 30 seconds or less at screening.
  • 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.

    • If receiving the SMN upregulator therapy nusinersen, must have completed the loading regimen and initiated maintenance dosing (i.e., completed at least one maintenance dose) with at least 4 weeks after the first maintenance dose having elapsed prior to screening.
  • Nutritional status stable over the past 6 months and anticipated to be stable throughout the duration of the study.
  • 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.

Exclusion Criteria:

  • Use of tracheostomy with positive pressure.
  • 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 14 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.
  • 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).
  • Use of systemic corticosteroids within 60 days prior to screening. Inhaled or topical steroids are allowed.
  • 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 betaagonist, botulinum toxin, or muscle relaxants) or muscle-enhancing supplements within 60 days prior to screening.
  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
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
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Scholar Rock, Inc.
Study Sponsor  ICMJE Scholar Rock, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Thomas O. Crawford, MD Johns Hopkins University
PRS Account Scholar Rock, Inc.
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP