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Immune-Mediated Necrotizing MyopathyPhase 2NCT04450654

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

A Phase 2 study testing Gamunex-C and Albumin in people with Immune-Mediated Necrotizing Myopathy, run by University of Washington at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 120 years
Conditions the study is for
Immune-Mediated Necrotizing Myopathy
What is being tested?
Gamunex-C and Albumin
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
0 participants
Where
1 siteUnited States
Study dates
May 2022to 25 Jul 2022
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 120 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • A subject must meet all of the following inclusion criteria at screening to be eligible for participation in this study:
  • 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.

Who cannot join

  • A subject meeting any of the following exclusion criteria at screening is NOT eligible for participation in this study:
  • 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
Show all 36Show fewer
  • 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
  • History of myocardial infarction or stroke in the last 12 months
  • Currently receiving anti-coagulation therapy (vitamin K antagonists, non-vitamin K oral anticoagulants [e.g. dabigatran, rivaroxaban, apixaban], parenteral anticoagulants [e.g fondaparinux]. Note that oral anti-platelet agents are allowed (e.g. aspirin, clopidogrel, ticodipine).
  • Females of child-bearing potential who are pregnant, have a positive serum pregnancy test (human chorionic gonadotropin [HCG]-based assay), breastfeeding, or are unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device or intrauterine system, condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence*) throughout the study.
  • True abstinence: When this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, post-ovulation methods], declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.)
  • Renal impairment (i.e., estimated glomerular filtration rate (eGFR) below 60ml/min)
  • History of chronic liver disease
  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) levels exceeding more than 2.5 times the ULN for the expected normal range for the testing laboratory, not due to HMGCR myopathy.
  • Hemoglobin level <9 g/dL
  • Known Immunoglobulin A (IgA) deficiency and anti-IgA serum antibodies
  • History of chronic alcoholism or illicit drug abuse (addiction) in the prior 12 months
  • Active psychiatric illness that interferes with compliance or communication with healthcare personnel
  • Currently receiving, or having received, within 1 month prior any investigational medicinal product or device. In the case of an investigational medicinal product trial, at least five half- lives (if known) must have elapsed prior to Screening.
  • Any medical condition which makes the clinical trial participation unadvisable or which is likely to interfere with the evaluation of the study treatment and/or the satisfactory conduct of the clinical trial according to the investigator's judgment. Any factor that in the opinion of the investigator would compromise the ability of the subject to complete the trial
  • Weight > 120kg. Individuals weighing >100kg and ≤120kg will be eligible at the discretion of the investigators.
  • History of angina pectoris or transient ischemic attack (TIA) in the last 12 months
  • Wells Criteria Score for DVT of 2 or more at the time of screening.
  • Wells Criteria Score for PE of 4 or more at the time of screening.
  • Presence of a central, in-dwelling catheter (such as a PICC line) at the time of informed consent.
  • Currently taking a nephrotoxic drug (eg gentamicin or vancomycin) at the time of informed consent.
  • Severe cardiac failure at the time of informed consent.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Washington

    Seattle, Washington, 98195

Other trials in United States

About this study

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.

Read the detailed description

This is a phase 2, double-blinded, randomized, placebo-controlled, multi-center trial of Gamunex-C IVIG as mono-therapy for HMGCR necrotizing myopathy. Up to 10 treatment-naïve patients will be enrolled and randomized to receive either Gamunex-C IVIG dosed at 2g/kg or placebo at week 0 and week 4. The primary efficacy outcome is the percentage of patients at week 8 with at least minimal improvement per the 2016 ACR/EULAR myositis clinical response criteria.

Being tested
Gamunex-C, Albumin
Keywords
HMG-CoA Reductase Auto-Antibody Necrotizing Myopathy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Gamunex-CDrug

    Also called IVIG

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

  • AlbuminDrug

    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

Study groups in NCT04450654
Study groupRoleWhat they receive
Gamunex-C IVIGExperimentalGamunex-C IVIG dosed at 2g/kg will be given on week 0 and week 4.
Receives: Drug: Gamunex-C
PlaceboPlacebo comparatorAlbumin in a 1% solution at an equivalent volume to the corresponding Gamunex-C IVIG dose will be given at week 0 and week 4.
Receives: Drug: Albumin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. ACR/EULAR 2016 Clinical Response Criteria for Myositis

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

    Measured: week 8

Secondary outcomes (7)

  1. Manual Muscle Strength Testing (MMT-8) score

    Manual muscle strength testing in 8 muscle groups will be scored. Range 0-160, with higher scores reflecting increased muscle strength.

    Measured: week 8

  2. ACR/EULAR Clinical Response Criteria for Myositis Total Improvement Score

    The total improvement score based on ACR/EULAR 2016 criteria will be calculated. Range 0-100, with higher scores reflecting greater improvement.

    Measured: week 8

  3. HMG-CoA Reductase Auto-antibody titer level

    Autoantibody titer level will be assessed

    Measured: week 8

  4. Creatine kinase

    Serum creatine kinase level will be measured

    Measured: week 8

  5. Number of patients screened to achieve planned enrollment

    Measured: 2 years

  6. Number of days needed to enroll planned sample

    Measured: 2 years

  7. Percentage of enrolled patients completing all primary and secondary measures

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Jun 2020
  2. Study startMay 2022 (estimated)
  3. Primary completion25 Jul 2022
  4. Study completion25 Jul 2022

Why it stopped: No Participants Enrolled

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
This is a randomized, double-blinded, placebo-controlled phase 2 trial.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04450654
Sponsor's protocol ID
STUDY00009143
Status last verified
Oct 2022
First submitted
24 Jun 2020
Last updated
24 Oct 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Gamunex-C and Albumin in people with Immune-Mediated Necrotizing Myopathy, run by University of Washington at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 36 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 24 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

ACR/EULAR 2016 Clinical Response Criteria for Myositis (time frame: week 8). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04450654.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Oct 2022.

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