Clinical trial NCT04025632: Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Immune Mediated Necrotizing Myopathy
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2019-07-19
Enrollment: 27
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Percentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
measure: Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
timeFrame: Baseline (Day 1) to end of Main Portion (Week 8)
Secondary outcomes:
measure: 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
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
measure: Change From Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
measure: Change From Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Score
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
measure: Change From Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
measure: Change From Baseline to Week 8 in Patient Global Activity VAS Score
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
measure: Change From Baseline to Week 8 in HAQ Score
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
measure: Change From Baseline to Week 8 in MDAAT Extramuscular Disease Activity VAS Score
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
measure: Change From Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score
timeFrame: Baseline (Day 1) and end of Main Portion (Week 8)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: Orange
country: United States
facility: University of California Irvine
state: California
city: Jacksonville
country: United States
facility: University of Florida Health Jacksonville
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Bethesda
country: United States
facility: National Institute of Health
state: Maryland
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine in Saint Louis
state: Missouri
city: Great Neck
country: United States
facility: Northwell Health Neuroscience Institute
state: New York
city: Columbus
country: United States
facility: The Ohio State University
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Memphis
country: United States
facility: Wesley Neurology Clinic
state: Tennessee
city: Austin
country: United States
facility: Austin Neuromuscular Center
state: Texas
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
city: Paris
country: France
facility: Hôpital Universitaire Pitié Salpêtrière
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC
city: London
country: United Kingdom
facility: University College London Hospitals NHS Foundation Trust
city: Manchester
country: United Kingdom
facility: Salford Royal NHS Barnes Clinical Research Facility
Study officials:
affiliation: UCB Pharma
name: UCB Cares
role: STUDY_DIRECTOR
References: