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Trial record 1 of 1 for:    NCT04025632
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Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04025632
Recruitment Status : Recruiting
First Posted : July 19, 2019
Last Update Posted : September 16, 2020
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

Brief Summary:
The purpose of the study is to evaluate the safety and efficacy of zilucoplan in patients with Immune-Mediated Necrotizing Myopathy (IMNM). Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or matching placebo for 8 weeks.

Condition or disease Intervention/treatment Phase
Immune Mediated Necrotizing Myopathy Drug: zilucoplan Other: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
Actual Study Start Date : December 3, 2019
Estimated Primary Completion Date : November 2020
Estimated Study Completion Date : November 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscle Disorders

Arm Intervention/treatment
Experimental: 0.3 mg/kg zilucoplan
Daily subcutaneous (SC) injection
Drug: zilucoplan
Daily subcutaneous (SC) inection

Placebo Comparator: Placebo
Daily subcutaneous (SC) injection
Other: Placebo
Daily subcutaneous (SC) inection




Primary Outcome Measures :
  1. Change from Baseline in creatine kinase levels. [ Time Frame: 8 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinical diagnosis of IMNM (Immune-Mediated Necrotizing Myopathy)
  • Positive serology for anti-HMGCR or anti-SRP autoantibodies
  • Clinical evidence of weakness (≤ grade 4 out of 5) on manual muscle testing in at least one proximal limb muscle group
  • CK (creatine kinase) of >1000 U/L at Screening
  • No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study
  • No changes in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study

Exclusion Criteria:

  • History of meningococcal disease
  • Current or recent systemic infection within 2 weeks prior to Screening or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Screening
  • Recent initiation of intravenous immunoglobulin (IVIG) (i.e., first cycle administered less than 90 days prior to Baseline)
  • Rituximab use within 90 days prior to Baseline or anticipated to occur during study
  • Statin use within 30 days prior to Baseline or anticipated to occur during study
  • Plasma exchange within 4 weeks prior to Baseline or expected to occur during the 8-week Treatment Period

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04025632


Contacts
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Contact: Ra Pharmaceuticals, Inc +1 617 401 4060 trials@rapharma.com

Locations
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Sponsors and Collaborators
Ra Pharmaceuticals
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Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04025632    
Other Study ID Numbers: RA101495-02.202
First Posted: July 19, 2019    Key Record Dates
Last Update Posted: September 16, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases