Monotherapy IVIG Gamunex-C for HMG-CoA Reductase Auto-Antibody Positive Necrotizing Myopathy Treatment (The MIGHT Trial)

NCT04450654 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: STUDY00009143
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase 2, pilot, randomized, placebo-controlled trial of Gamunex-C IVIG as mono-therapy for HMGCoA reductase auto-antibody positive (HMGCR) necrotizing myopathy. The trial will test the feasibility and initial efficacy of Gamunex-C IVIG mono-therapy in HMGCR necrotizing myopathy.

Conditions

Immune-Mediated Necrotizing Myopathy

Keywords

HMG-CoA Reductase Auto-Antibody Necrotizing Myopathy

Outcome Measures

Primary Outcomes (1)

• ACR/EULAR 2016 Clinical Response Criteria for Myositis

  The percentage of patients in each arm with at least a minimal response

  week 8

Secondary Outcomes (7)

• Manual Muscle Strength Testing (MMT-8) score

  week 8

• ACR/EULAR Clinical Response Criteria for Myositis Total Improvement Score

  week 8

• HMG-CoA Reductase Auto-antibody titer level

  week 8

• Creatine kinase

  week 8

• Number of patients screened to achieve planned enrollment

  2 years

Study Arms (2)

Gamunex-C IVIG

EXPERIMENTAL

Gamunex-C IVIG dosed at 2g/kg will be given on week 0 and week 4.

Drug: Gamunex-C

Placebo

PLACEBO COMPARATOR

Albumin in a 1% solution at an equivalent volume to the corresponding Gamunex-C IVIG dose will be given at week 0 and week 4.

Drug: Albumin

Interventions

Gamunex-C DRUG

Gamunex-C IVIG will be given at week 0 and week 4

Also known as: IVIG

Gamunex-C IVIG

Albumin DRUG

1% albumin solution dosed at equivalent volume to the corresponding weight-based Gamunex-C IVIG dose will be given at week 0 and week 4

Placebo

Eligibility Criteria

• Age: 18 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Anti-HMGCR positive. Patients will be screened by commercially-available ELISA.
• Age ≥ 18 years
• Demonstrable proximal muscle weakness: score of \<135 on the Proximal Manual Muscle Strength Testing 8-Muscle Group Assessment (MMT-8) (range 0-160).
• Serum creatinine kinase (CK) more than 5 times the upper limit of normal
• Muscle biopsy will not be required for eligibility in order to minimize the time to enrollment and initiation of treatment. Muscle biopsy will be obtained whenever possible as part of the standard of care.
• Subjects must be willing and able to provide written informed consent.

You may not qualify if:

• Disease duration greater than 36 months.
• Participants taking oral or intravenous glucocorticoids where the dose has changed within 4 weeks of screening.
• Exposure to immunoglobulin treatment (IV, IM, or SubQ) in the prior 3 months
• Exposure to plasma exchange (PEX) in the prior 3 months
• Exposure to other immunosuppressive medications (e.g. methotrexate, leflunomide, azathioprine, mycophenolate mofetil) in the prior 6 months
• Exposure to rituximab or any monoclonal antibody in the prior 12 months
• Currently taking a statin medication
• History of dermatomyositis rash (either biopsy-proven, or history of photosensitive rash).
• Presence of respiratory or swallowing dysfunction due to HMGCR myopathy
• Inadequate venous access
• History of anaphylactic reactions or severe reactions to any blood-derived product
• History of intolerance to any component of the IP
• History of thrombotic complication to polyclonal IVIG therapy
• History of pulmonary embolism or deep venous thromboembolism
• History of hyperviscosity or hypercoagulable state

Sponsors & Collaborators

• University of Washington: lead
• Grifols Biologicals, LLC: collaborator

Study Sites (1)

University of Washington

Seattle, Washington, 98195, United States

Location

MeSH Terms

Interventions

Hizentra; Immunoglobulins, Intravenous; Albumins

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• James S Andrews, MD

  University of Washington

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor, School of Medicine: Rheumatology

Model Details: This is a randomized, double-blinded, placebo-controlled phase 2 trial.

Study Record Dates

• First Submitted: June 24, 2020
• First Posted: June 29, 2020
• Study Start: May 1, 2022
• Primary Completion: July 25, 2022
• Study Completion: July 25, 2022
• Last Updated: October 24, 2022

Record last verified: 2022-10

Locations

US (1)