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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

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ClinicalTrials.gov Identifier: NCT04025632
Recruitment Status : Terminated (No relevant safety differences between Zilucoplan and placebo were identified.)
First Posted : July 19, 2019
Last Update Posted : September 16, 2021
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)
Actual Enrollment : 27 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
Actual Primary Completion Date : March 4, 2021
Actual Study Completion Date : June 15, 2021

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. Percentage Change from Baseline to Week 8 in Creatine Kinase (CK) Levels [ Time Frame: Baseline (Day 1) to Week 8 ]
  2. Number of Participants Who Experience a Treatment-Emergent Adverse Event (TEAE) [ Time Frame: Day 1 to up to 40 days after last dose (up to an overall duration of approximately 21 months) ]

Secondary Outcome Measures :
  1. Number of Participants Who Achieve at Least Minimal Response Based on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria Scale [ Time Frame: Baseline (Day 1) to Week 8 ]
  2. Change from Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time [ Time Frame: Baseline (Day 1) to Week 8 ]
    The 3TUG test will only be performed in participants who are ambulatory.

  3. Change from Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Grade [ Time Frame: Baseline (Day 1) to Week 8 ]
  4. Change from Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score [ Time Frame: Baseline (Day 1) to Week 8 ]
  5. Change from Baseline to Week 8 in Patient Global Activity Visual Analogue Scale (VAS) Score [ Time Frame: Baseline (Day 1) to Week 8 ]
  6. Change from Baseline to Week 8 in Health Assessment Questionnaire (HAQ) Score [ Time Frame: Baseline (Day 1) to Week 8 ]
  7. Change from Baseline to Week 8 in Myositis Disease Activity Assessment Tool (MDAAT) Extramuscular Disease Activity Visual Analogue Scale (VAS) Score [ Time Frame: Baseline (Day 1) to Week 8 ]
  8. Change from Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score [ Time Frame: Baseline (Day 1) to Week 8 ]


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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-3-hydroxy-3-methyl-glutaryl-coenzyme A reductase (HMGCR) or anti-signal recognition particle (SRP) autoantibodies
  • Clinical evidence of weakness (≤ grade 4 out of 5) on manual muscle testing in at least one proximal limb muscle group
  • Creatine kinase (CK) 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


Locations
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Sponsors and Collaborators
Ra Pharmaceuticals
Investigators
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Study Director: UCB Cares UCB Pharma
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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, 2021
Last Verified: September 2021

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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