Purpose

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

Eligibility

Eligible Ages
Between 18 Years and 75 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
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

Recruiting Locations

University of Kansas Medical Center
Kansas City, Kansas 66160

More Details

Status
Recruiting
Sponsor
Ra Pharmaceuticals

Study Contact

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.