免费获得国外相关药品，最快 1 个工作日回馈药物信息

出境医 / 临床实验 / Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy

Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy

Study Description
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

Study Design
Layout table for study information
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
Actual Primary Completion Date : March 4, 2021
Estimated Study Completion Date : November 2022
Arms and Interventions
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
Outcome Measures
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 ]

Eligibility Criteria
Layout table for eligibility information
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
Contacts and Locations

Locations
Show Show 18 study locations
Sponsors and Collaborators
Ra Pharmaceuticals
Tracking Information
First Submitted Date  ICMJE July 17, 2019
First Posted Date  ICMJE July 19, 2019
Last Update Posted Date April 26, 2021
Actual Study Start Date  ICMJE December 3, 2019
Actual Primary Completion Date March 4, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 22, 2021)
  • Percentage Change from Baseline to Week 8 in Creatine Kinase (CK) Levels [ Time Frame: Baseline (Day 1) to Week 8 ]
  • 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) ]
Original Primary Outcome Measures  ICMJE
 (submitted: July 17, 2019) 		Change from Baseline in creatine kinase levels. [ Time Frame: 8 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 22, 2021)
  • 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 ]
  • 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.
  • Change from Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Grade [ Time Frame: Baseline (Day 1) to Week 8 ]
  • Change from Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score [ Time Frame: Baseline (Day 1) to Week 8 ]
  • Change from Baseline to Week 8 in Patient Global Activity Visual Analogue Scale (VAS) Score [ Time Frame: Baseline (Day 1) to Week 8 ]
  • Change from Baseline to Week 8 in Health Assessment Questionnaire (HAQ) Score [ Time Frame: Baseline (Day 1) to Week 8 ]
  • 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 ]
  • 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 ]
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 Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Immune Mediated Necrotizing Myopathy
Intervention  ICMJE
  • Drug: zilucoplan
    Daily subcutaneous (SC) inection
  • Other: Placebo
    Daily subcutaneous (SC) inection
Study Arms  ICMJE
  • Experimental: 0.3 mg/kg zilucoplan
    Daily subcutaneous (SC) injection
    Intervention: Drug: zilucoplan
  • Placebo Comparator: Placebo
    Daily subcutaneous (SC) injection
    Intervention: Other: Placebo
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
Estimated Enrollment  ICMJE
 (submitted: July 17, 2019) 		24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2022
Actual Primary Completion Date March 4, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04025632
Other Study ID Numbers  ICMJE RA101495-02.202
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 Not Provided
Responsible Party Ra Pharmaceuticals
Study Sponsor  ICMJE Ra Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Ra Pharmaceuticals
Verification Date April 2021

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

治疗医院

新药特效药

前沿医讯