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Trial · Interventional

CompletedPhase 2Results postedRegistry data

Acthar in Treatment of Refractory Dermatomyositis and Polymyositis

Overview

Status
Completed
Phase
Phase 2
Enrollment
12 participants (actual)
Sponsor
Rohit Aggarwal, MD · Other
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

United States

Start
Sept 2013
Primary completion
Sept 2015
Completion
May 2016
First posted
24 Jul 2013 (estimated)
Last update posted
1 Sept 2017
Conditions
Dermatomyositis · Polymyositis

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20132014201520162017
  1. Ph 2

    Acthar in Treatment of Refractory Dermatomyositis and Polymyositis

    NCT01906372

    Sept 2013 to May 2016

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The purpose of this research study is to evaluate the effectiveness of the study drug, ACTH Gel in people diagnosed with dermatomyositis a disease that causes muscle weakness and is associated with a rash (DM) or polymyositis (PM) a disease that causes muscle weakness without a rash. The study doctors want to evaluate whether ACTH Gel will improve the symptoms of this disease. This drug is approved by the Food and Drug Administration (FDA) for dermatomyositis (DM) and polymyositis (PM). ACTH gel has been an FDA-approved treatment for myositis since 1952, and in 2010 the FDA retained PM and DM as diseases approved for ACTH gel use.

Interventions

  • Adrenocorticotropic Hormone GelDrug

Arms1

  • Experimental

    Acthar Gel

    Acthar Gel (Adrenocorticotropic Hormone Gel)in refractory PM and DM patients using an open label design for 6 months. We will enroll 10 active and refractory PM/DM patients over a 15 month period, followed by 6 months of additional follow-up for each subject. Study subjects will self-administer subcutaneously H.P. Acthar Gel 80 units (1 ml) twice a week for a period of six months. Outcome measures were not evaluated on subjects who did not reach the 8 week time point in the trial.

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Specific Aim 1: Number of Subjects Meeting IMACS Preliminary Definition of Improvement (DOI).

    Time frame · Primary end point: IMACS preliminary definition of improvement (DOI)

    Registry description

    3 of any of the 6 core set measures (CSM) improved by ≥ 20%, with no more than 2 CSM worsening by ≥25% (worsening measure cannot include the MMT). The DOI should be met at least once on any of the 6 follow up visits and maintained until week 24. Subjects not meeting DOI during the trial are treatment failures.

Secondary outcomes(1)
  1. Steroid-sparing Effect of H.P. Acthar Gel in Refractory Adult PM and DM Patients.

    Time frame · Steroid sparing effect and safety and tolerability at 24 weeks compared to baseline

    Registry description

    Mean change in glucocorticoid dose (equivalent prednisone dose) at 24 weeks compared to baseline.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· Primary end point: IMACS preliminary definition of improvement (DOI)

Specific Aim 1: Number of Subjects Meeting IMACS Preliminary Definition of Improvement (DOI).

GroupValue (Participants)
Acthar Gel7
Secondary and other outcome results(1)
Secondary outcomeMean· Steroid sparing effect and safety and tolerability at 24 weeks compared to baseline

Steroid-sparing Effect of H.P. Acthar Gel in Refractory Adult PM and DM Patients.

GroupValue (mg)Spread
Acthar Gel2.33.2

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Acthar GelActhar Gel (Adrenocorticotropic Hormone Gel)in refractory PM and DM patients using an open label design for 6 months. 10 active and refractory PM/DM patients were evaluated over a 15 month period, followed by 6 months of additional follow-up for each subject. Study subjects will self-administer subcutaneously H.P. Acthar Gel 80 units (1 ml) twice a week for a period of six months.–3 / 107 / 10

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies6

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Randomized controlled trial · 2011

3 results

Five-day regimen of intramuscular or subcutaneous self-administered adrenocorticotropic hormone gel for acute exacerbations of multiple sclerosis: a prospective, randomized, open-label pilot trial.

Simsarian et al. 2011Drug design, development and therapycited by 27

PMID 21792296PMC3140290DOI 10.2147/dddt.s19331PUB-21792296

Free to read · publisher termsCC BY-NC3 results

Indexing hint: Randomized controlled trial · 1996

High-dose corticotropin (ACTH) versus prednisone for infantile spasms: a prospective, randomized, blinded study.

Baram et al. 1996Pediatricscited by 229

PMID 8604274PMC3100715DOI 10.1542/peds.97.3.375PUB-08604274

Rights unknown
Source link

More studies

4 studies on this page

Indexing hint: Clinical trial · 2024

Corrected

Efficacy and safety of repository corticotropin injection for refractory cutaneous dermatomyositis: a prospective, open-label study.

Fernandez et al. 2024Rheumatology (Oxford, England)cited by 2

PMID 37941470DOI 10.1093/rheumatology/kead595PUB-37941470

Full text at the publisher

Indexing hint: Clinical trial · 2018

Efficacy and safety of adrenocorticotropic hormone gel in refractory dermatomyositis and polymyositis.

Aggarwal et al. 2018Annals of the rheumatic diseasescited by 35

PMID 29237618DOI 10.1136/annrheumdis-2017-212047PUB-29237618

Full text at the publisher
Publisher page

Indexing hint: Case report · 2012

Corrected

Treating refractory dermatomyositis or polymyositis with adrenocorticotropic hormone gel: a retrospective case series.

Levine et al. 2012Drug design, development and therapycited by 48

PMID 22787386PMC3392138DOI 10.2147/dddt.s33110PUB-22787386

Free to read · publisher termsCC BY-NC

Indexing hint: Case report · 2011

Treatment of nephrotic syndrome with adrenocorticotropic hormone (ACTH) gel.

Bomback et al. 2011Drug design, development and therapycited by 62

PMID 21448451PMC3063118DOI 10.2147/dddt.s17521PUB-21448451

Free to read · publisher termsCC BY-NC
Read at source

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * Definite or probable polymyositis (PM) or dermatomyositis (DM) by Bohan and Peter criteria. * PM patients must either possess a myositis-associated autoantibody or undergo adjudication for confirmation of the PM diagnosis by consensus of two experts to ensure non-PM patients are not enrolled. This step is necessary since there are well-known mimics of PM. * Age ≥ 18 years. * Active myositis as defined by baseline Manual Muscle Testing (MMT-8) no greater than 125/150 and at least 2 additional CSM meeting the criteria stipulated below: 1. Patient global with a minimum value of 2.0 cm on a 10 cm visual analog scale(VAS) 2. Physician global with a minimum value of 2.0 cm on a 10 cm VAS scale 3. Health Assessment Questionnaire (HAQ) disability index with a minimum value of 0.25 4. Elevation of at least one of the muscle enzymes \[which includes creatine kinase (CK), aldolase, lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and aspartate aminotransferase (AST)\] at a minimum level of 1.3 x the upper limit of normal. 5. Global extramuscular disease activity score with a minimum value of 1.0 cm on a 10 cm VAS scale \[this measure is the physician's composite evaluation and is based on assessments of activity scores on the constitutional, cutaneous, skeletal, gastrointestinal, pulmonary and cardiac scales of the Myositis Disease Activity Assessment Tool (MDAAT)\]. * To ensure that we can enroll active DM patients with a severe rash who may not meet the MMT-8 criterion noted above, we propose additional enrollment criteria such that the International Myositis Assessment and Clinical Studies (IMACS) definition of improvement (DOI) can potentially be met: 1. Cutaneous VAS score on MDAAT \> 3 cm on a 10 cm VAS scale, and 2. At least 3 of the above 5 (a through e under 4.) criteria. * Refractory myositis is defined by active disease despite an adequate glucocorticoid trial (\> 2 months of usual glucocorticoid therapy or intolerance to such therapy) and/or ≥ 1 conventional immunosuppressive agent (e.g. methotrexate, azathioprine, tacrolimus, cyclosporine, mycophenolate mofetil, IVIG, anti-TNF or rituximab) for a reasonable dose and duration (\> 3 months or intolerance to therapy). It is recommended to enroll refractory patients failing (or intolerant to) both glucocorticoids and at least 1 conventional immunosuppressive agent. * If the enrolling physician is planning to continue current immunosuppressive agents or glucocorticoids as concomitant therapy with Acthar gel during the trial, then patient must be on a stable glucocorticoid and/or immunosuppressive dose 2 weeks prior to visit 1. The patient should have been on that immunosuppressive medication for at least 8 weeks (and at least 4 weeks for glucocorticoids) prior to visit 1. * If the enrolling physician is planning to discontinue current immunosuppressive agent or glucocorticoids, then following wash out period is required prior to visit 1. * If previous concomitant medications were discontinued, the following wash out periods are required prior to Visit 1 * Methotrexate -4 weeks * Other IS agent (e.g. azathioprine, cyclosporine, tacrolimus, leflunomide, mycophenolate mofetil) - 4 weeks * IVIg or cyclophosphamide - 2 months * rituximab -6 months * infliximab or adalimumab -8 weeks * glucocorticoids - 2 weeks * etanercept -2 weeks * anakinra -1 week Exclusion Criteria: * Juvenile DM or PM, myositis in overlap with another connective tissue disease, cancer associated myositis, inclusion body myositis, or any other non immune-mediated myopathy. * Hypersensitivity to Acthar * Severe cardiac or pulmonary involvement * Severe muscle damage defined as a baseline global muscle damage score on the MDI (Myositis Damage Index) of ≥ 5 cm on a 10 cm VAS. * Patients with malignancy within 3 years of screening (except basal cell cancer or squamous cell cancer of skin). * Uncontrolled diabetes, hepatic or renal disease. * Ongoing active or chronic infections. * Pregnant or lactating females. * For any medical or physical or socio-psychological reasons that PI feels would not allow the subject to complete the study.

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