Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing MyopathyPhase 2NCT06599697
The MIGHT Trial - An Exploratory Clinical Trial of IVIG in Anti-HMGCR Immune Mediated Necrotizing Myopathy
A Phase 2 study testing Intravenously administered pooled human immunoglobulin (IVIG) in people with Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy, run by University of Alabama at Birmingham at 5 sites in United States.
Trial summary
This is a randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of intravenously administered pooled human immunoglobulin (IVIG) in anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) immune mediated necrotizing myopathy (IMNM). Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy
- What is being tested?
- Intravenously administered pooled human immunoglobulin (IVIG)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 5 sitesUnited States
- Study dates
- 27 Oct 2025to 30 Jun 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age > 16 years
- Anti-HMGCR antibody positive
- MMT-8 score < 142 (range 0-160)
- Serum CK > 5x upper limit of normal
- Anti-HMGCR IMNM disease duration < 36 months at screening
- No moderate or severe respiratory or swallowing dysfunction due to anti-HMGCR IMNM at screening
- No history of dermatomyositis rash
- Must reside in a state with a participating research site
Who cannot join
- Oral glucocorticoid (GC) daily dose > 15mg at screening
- Change in oral GC dose < 2 weeks prior to screening
- Prior IVIG treatment for anti-HMGCR IMNM
- ->1 oral conventional synthetic DMARD (e.g. methotrexate, mycophenolate mofetil, azathioprine) use at screening
- Change in concomitant DMARD dose < 4 weeks prior to screening
- Rituximab < 6 months prior to screening
Show all 20Show fewer
- Plasma exchange, cyclophosphamide, or biologic immunosuppressive medication < 3 months prior to screening
- Use of statin medication at screening
- History of anaphylactic reaction to IVIG
- History of angina pectoris, myocardial infarction, transient ischemic attack, or stroke < 12 months prior to screening
- Females of child-bearing potential who are pregnant, breastfeeding, or are unwilling to practice a highly effective method of contraception during the study
- Wells Criteria for DVT score of 2 or more at screening
- Wells Criteria for PE score of 4 or more at screening
- Weight >120kg
- History of cancer (excluding non-melanomatous skin cancer) < 5 years prior to screening
- History of pulmonary embolism or deep venous thromboembolism < 3 years prior to screening
- History of hyperviscosity or hypercoagulable state
- 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, ticlopidine).
- Glomerular filtration rate (GFR) <60mL/min at the time of screening
- Any medical condition which, in the investigator's judgment, makes participation in the clinical trial unadvisable or which would interfere with evaluation of the study treatment.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 4 recruiting
- Not yet recruiting
Johns Hopkins University
Baltimore, Maryland, 21218
Lisa Christopher-Stine, MD, MPH, 410-550-6962, lchrist4@jhmi.edu
Grazyna Purwin, gpurwin1@jhmi.edu
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35233
James Andrews, MD, (205) 934-1564, jaandrews@uabmc.edu
Hannah E Howell, MS, 205-996-6552, heburns@uabmc.edu
- Recruiting
University of Texas Health Science Center at Houston
Houston, Texas, 77030
Eleni Tiniakou, MD, 713-500-4435, eleni.tiniakou@uth.tmc.edu
Enrique Rodriguez, Enrique.Rodriguez@uth.tmc.edu
- Recruiting
University of Pittsburgh
Pittsburgh, Pennsylvania, 15260
Rohit Aggarwal, MD, MS, 412-624-4141, aggarwalr@upmc.edu
Diane C Koontz, dik4@pitt.edu
- Active, not recruiting
University of Washington
Seattle, Washington, 98195
About this study
Official title: The MIGHT Trial - An Exploratory Clinical Trial of Intravenous Immunoglobulin (IVIG) in Anti-3-Hydroxy-3-Methylglutaryl-CoA Reductase (HMGCR) Immune Mediated Necrotizing Myopathy (IMNM)
This is a randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of intravenously administered pooled human immunoglobulin (IVIG) in anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) immune mediated necrotizing myopathy (IMNM). Planned enrollment is 12 individuals with active anti-HMGCR IMNM meeting inclusion and exclusion criteria. Assuming 20% drop-out, the investigators anticipate 10 participants will complete all study assessments. Enrolled participants will be randomized 1:1 to either IVIG 2g/kg or placebo (0.9% sodium chloride at equivalent volume) at weeks 0, 4, and 8. The primary efficacy and co-primary safety and tolerability endpoints will be assessed at week 12. After the randomized phase of the trial, all participants will be offered to continue on to an open-label extension phase in which participants will receive IVIG at weeks 12, 16, and 20. Participants will then return at week 24 for a final non-infusion visit to reassess safety, tolerability, and efficacy outcome.
- Conditions
- Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy
- Being tested
- Intravenously administered pooled human immunoglobulin (IVIG)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravenously administered pooled human immunoglobulin (IVIG)Biological
Also called Octagram -Immune Globulin Intravenous (Human)
IVIG 2g/kg every 4 weeks for 12 weeks (3 doses)
| Study group | Role | What they receive |
|---|---|---|
| Intravenously Administered Pooled Human Immunoglobulin (IVIG) | Experimental | Participants will receive intravenously administered pooled human immunoglobulin (IVIG) 2g/kg every 4 weeks for 24 weeks. Receives: Biological: Intravenously administered pooled human immunoglobulin (IVIG) |
| Placebo | No intervention | Participants will receive an infusion of 0.9% sodium chloride solution every 4 weeks for 16 weeks at equivalent volume to corresponding IVIG weight-based dose. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage change in serum creatine kinase (CK)
Primary Efficacy Outcome
Measured: Week 0 to 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start27 Oct 2025
- Primary completion30 Dec 2026 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing MyopathyStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- A randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of intravenously administered pooled human immunoglobulin (IVIG) in anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) immune mediated necrotizing myopathy (IMNM). Planned enrollment is 12 individuals with active anti-HMGCR IMNM meeting inclusion and exclusion criteria. Enrolled participants will be randomized 1:1 to receive either IVIG 2g/kg or placebo (0.9% sodium chloride at equivalent volume) at weeks 0, 4, and 8. The 2g/kg q4 weeks regimen of IVIG was chosen based on the investigators' clinical experience and prior studies. The primary endpoints will be assessed at week 12. After the randomized phase of the trial, all participants will be offered an open-label extension to receive IVIG at weeks 12, 16, and 20.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Octapharma USA, Inc., CSI Pharmacy
- Responsible party
- Principal investigator, James Andrews, University of Alabama at Birmingham
- Investigators
- James Andrews, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06599697
- Sponsor's protocol ID
- 300013467
- Other IDs
- 1R21AR083566-01A1
- Status last verified
- Aug 2026
- First submitted
- 12 Sep 2024
- Last updated
- 5 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The MIGHT Trial - An Exploratory Clinical Trial of Intravenous Immunoglobulin (IVIG) in Anti-3-Hydroxy-3-Methylglutaryl-CoA Reductase (HMGCR) Immune Mediated Necrotizing Myopathy (IMNM)
- ClinicalTrials.gov ID
- NCT06599697
- Acronym / sponsor ID
- 300013467
- Conditions
- Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Octapharma USA, Inc., CSI Pharmacy
- Study start
- 27 Oct 2025 (actual)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of intravenously administered pooled human immunoglobulin (IVIG) in anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) immune mediated necrotizing myopathy (IMNM). Planned enrollment is 12 individuals with active anti-HMGCR IMNM meeting inclusion and exclusion criteria. Assuming 20% drop-out, the investigators anticipate 10 participants will complete all study assessments. Enrolled participants will be randomized 1:1 to either IVIG 2g/kg or placebo (0.9% sodium chloride at equivalent volume) at weeks 0, 4, and 8. The primary efficacy and co-primary safety and tolerability endpoints will be assessed at week 12. After the randomized phase of the trial, all participants will be offered to continue on to an open-label extension phase in which participants will receive IVIG at weeks 12, 16, and 20. Participants will then return at week 24 for a final non-infusion visit to reassess safety, tolerability, and efficacy outcome.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- A randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of intravenously administered pooled human immunoglobulin (IVIG) in anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) immune mediated necrotizing myopathy (IMNM). Planned enrollment is 12 individuals with active anti-HMGCR IMNM meeting inclusion and exclusion criteria. Enrolled participants will be randomized 1:1 to receive either IVIG 2g/kg or placebo (0.9% sodium chloride at equivalent volume) at weeks 0, 4, and 8. The 2g/kg q4 weeks regimen of IVIG was chosen based on the investigators' clinical experience and prior studies. The primary endpoints will be assessed at week 12. After the randomized phase of the trial, all participants will be offered an open-label extension to receive IVIG at weeks 12, 16, and 20.
| Arm | Type | Receives |
|---|---|---|
| Intravenously Administered Pooled Human Immunoglobulin (IVIG) | Experimental | Biological: Intravenously administered pooled human immunoglobulin (IVIG)Participants will receive intravenously administered pooled human immunoglobulin (IVIG) 2g/kg every 4 weeks for 24 weeks. |
| Placebo | No intervention | –Participants will receive an infusion of 0.9% sodium chloride solution every 4 weeks for 16 weeks at equivalent volume to corresponding IVIG weight-based dose. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Age > 16 years
- Anti-HMGCR antibody positive
- MMT-8 score < 142 (range 0-160)
- Serum CK > 5x upper limit of normal
- Anti-HMGCR IMNM disease duration < 36 months at screening
Key exclusion criteria (20 listed)
- Oral glucocorticoid (GC) daily dose > 15mg at screening
- Change in oral GC dose < 2 weeks prior to screening
- Prior IVIG treatment for anti-HMGCR IMNM
- ->1 oral conventional synthetic DMARD (e.g. methotrexate, mycophenolate mofetil, azathioprine) use at screening
- Change in concomitant DMARD dose < 4 weeks prior to screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intravenously administered pooled human immunoglobulin (IVIG)Also: Octagram -Immune Globulin Intravenous (Human)IVIG 2g/kg every 4 weeks for 12 weeks (3 doses) | Biological | Intravenously Administered Pooled Human Immunoglobulin (IVIG) |
Primary outcome
Percentage change in serum creatine kinase (CK)
Time frame: Week 0 to 12
Primary Efficacy Outcome
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2026.
- Individual participant dataSharing planned: Undecided
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
James Andrews, MD principal investigator
University of Alabama at Birmingham
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Intravenously administered pooled human immunoglobulin (IVIG) in people with Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy, run by University of Alabama at Birmingham at 5 sites 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 8 inclusion and 20 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: looking for participants now, as of the sponsor's last update on 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact James Andrews, MD ((205) 934-1564, jaandrews@uabmc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage change in serum creatine kinase (CK) (time frame: Week 0 to 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Aug 2026.