DermatomyositisPhase 2NCT01906372
Acthar in Treatment of Refractory Dermatomyositis and Polymyositis
A Phase 2 study testing Adrenocorticotropic Hormone Gel in people with Dermatomyositis and Polymyositis, run by Rohit Aggarwal, MD at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Dermatomyositis, Polymyositis
- What is being tested?
- Adrenocorticotropic Hormone Gel
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- Sep 2013to May 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- Patient global with a minimum value of 2.0 cm on a 10 cm visual analog scale(VAS)
- Physician global with a minimum value of 2.0 cm on a 10 cm VAS scale
Show all 24Show fewer
- Health Assessment Questionnaire (HAQ) disability index with a minimum value of 0.25
- 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.
- 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:
- Cutaneous VAS score on MDAAT > 3 cm on a 10 cm VAS scale, and
- 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
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
North Shore LIJ Medical Center
Great Neck, New York, 11021
University of Pittsburgh
Pittsburgh, Pennsylvania, 15261
About this study
Official title: Open Label Proof of Concept Study to Evaluate Efficacy and Safety of Adrenocorticotropic Hormone Gel in Refractory Dermatomyositis or Polymyositis
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.
Read the detailed description
Despite its FDA approval there is very limited data on its clinical effectiveness in PM and DM. There was a recent study published in the peer-review journal Drug Design, Development and Therapy on a retrospective case series evaluating Acthar in the treatment of PM and DM. Acthar was administered to five patients who had previously failed multiple steroid and immunosuppressant treatment regimens. The patients received injections of Acthar over the course of 12 weeks or more. Improvement in PM and DM symptoms related to disease exacerbations was seen in all five patients. Symptom improvements included increased muscle strength, resolution of disease-related skin manifestations and improvements in the ability to perform tasks associated with daily living. All of these patients tolerated the treatment well with no significant side effects reported. The paper, "Treating refractory dermatomyositis or polymyositis with adrenocorticotropic hormone gel: a retrospective case series," was authored by Dr. Todd Levine, M.D., Co-Director of the Neurophysiology Department at Banner Good Samaritan Medical Center, Assistant Professor at the University of Arizona in Neurology, and Member of Phoenix Neurological Associates.
H.P. Acthar® Gel, or Acthar, is a prescription medication containing the hormone adrenocorticotropin (hormone produced and secreted by the anterior pituitary gland), also known as ACTH. H.P. Acthar Gel is a highly purified preparation of adrenocorticotropic hormone (ACTH) in a gel that is designed to provide extended release of the ACTH following injection. Acthar was originally approved by the FDA in 1952. It is approved for use in 19 different conditions including dermatomyositis and polymyositis.
Acthar is designed to provide a prolonged release of the medication after it is injected. Acthar is not a steroid; it works by helping your body produce its own natural steroid hormones, such as cortisol, corticosterone, and aldosterone. Acthar is an injection that is given intramuscularly (into the muscle). Subjects enrolled in the study will be asked to self administer Acthar two times per week. Subjects will be provided training by the principal investigator on how to perform the self injections.
- Conditions
- Dermatomyositis, Polymyositis
- Being tested
- Adrenocorticotropic Hormone Gel
- Keywords
- dermatomyositis, polymyositis
- Medical subject headings
- Dermatomyositis, Polymyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adrenocorticotropic Hormone GelDrug
Also called H.P. Acthar Gel
H.P. Acthar Gel 80 units will be self-administered subcutaneously twice weekly by the subject for a period of 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Acthar Gel | Experimental | 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. Receives: Drug: Adrenocorticotropic Hormone Gel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Specific Aim 1: Number of Subjects Meeting IMACS Preliminary Definition of Improvement (DOI).
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.
Measured: Primary end point: IMACS preliminary definition of improvement (DOI)
Secondary outcomes (1)
Steroid-sparing Effect of H.P. Acthar Gel in Refractory Adult PM and DM Patients.
Mean change in glucocorticoid dose (equivalent prednisone dose) at 24 weeks compared to baseline.
Measured: Steroid sparing effect and safety and tolerability at 24 weeks compared to baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2013 (estimated)
- Study startSep 2013
- Primary completionSep 2015
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for DermatomyositisStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rohit Aggarwal, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt
- Responsible party
- Sponsor-investigator, Rohit Aggarwal, MD, University of Pittsburgh
- Investigators
- Rohit Aggarwal, MD, Principal investigator, University of Pittsburgh, Division of Rheumatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01906372
- Sponsor's protocol ID
- PRO13050507
- Status last verified
- Aug 2017
- First submitted
- 5 Jul 2013
- Last updated
- 1 Sep 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Catania A, Gatti S, Colombo G, Lipton JM. Targeting melanocortin receptors as a novel strategy to control inflammation. Pharmacol Rev. 2004 Mar;56(1):1-29. doi: 10.1124/pr.56.1.1.
PubMed 15001661Catania A, Lonati C, Sordi A, Carlin A, Leonardi P, Gatti S. The melanocortin system in control of inflammation. ScientificWorldJournal. 2010 Sep 14;10:1840-53. doi: 10.1100/tsw.2010.173.
PubMed 20852827Baram TZ, Mitchell WG, Tournay A, Snead OC, Hanson RA, Horton EJ. High-dose corticotropin (ACTH) versus prednisone for infantile spasms: a prospective, randomized, blinded study. Pediatrics. 1996 Mar;97(3):375-9.
PubMed 8604274Bomback AS, Tumlin JA, Baranski J, Bourdeau JE, Besarab A, Appel AS, Radhakrishnan J, Appel GB. Treatment of nephrotic syndrome with adrenocorticotropic hormone (ACTH) gel. Drug Des Devel Ther. 2011 Mar 14;5:147-53. doi: 10.2147/DDDT.S17521.
PubMed 21448451Simsarian JP, Saunders C, Smith DM. 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. Drug Des Devel Ther. 2011;5:381-9. doi: 10.2147/DDDT.S19331. Epub 2011 Jul 11.
PubMed 21792296Levine T. Treating refractory dermatomyositis or polymyositis with adrenocorticotropic hormone gel: a retrospective case series. Drug Des Devel Ther. 2012;6:133-9. doi: 10.2147/DDDT.S33110. Epub 2012 Jun 11.
PubMed 22787386Rider LG, Giannini EH, Brunner HI, Ruperto N, James-Newton L, Reed AM, Lachenbruch PA, Miller FW; International Myositis Assessment and Clinical Studies Group. International consensus on preliminary definitions of improvement in adult and juvenile myositis. Arthritis Rheum. 2004 Jul;50(7):2281-90. doi: 10.1002/art.20349.
PubMed 15248228Oddis CV, Reed AM, Aggarwal R, Rider LG, Ascherman DP, Levesque MC, Barohn RJ, Feldman BM, Harris-Love MO, Koontz DC, Fertig N, Kelley SS, Pryber SL, Miller FW, Rockette HE; RIM Study Group. Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: a randomized, placebo-phase trial. Arthritis Rheum. 2013 Feb;65(2):314-24. doi: 10.1002/art.37754.
PubMed 23124935Fernandez AP, Gallop J, Polly S, Khanna U. Efficacy and safety of repository corticotropin injection for refractory cutaneous dermatomyositis: a prospective, open-label study. Rheumatology (Oxford). 2024 Dec 1;63(12):3370-3379. doi: 10.1093/rheumatology/kead595.
PubMed 37941470Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Efficacy and safety of adrenocorticotropic hormone gel in refractory dermatomyositis and polymyositis. Ann Rheum Dis. 2018 May;77(5):720-727. doi: 10.1136/annrheumdis-2017-212047. Epub 2017 Dec 13.
PubMed 29237618
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Proof of Concept Study to Evaluate Efficacy and Safety of Adrenocorticotropic Hormone Gel in Refractory Dermatomyositis or Polymyositis
- ClinicalTrials.gov ID
- NCT01906372
- Acronym / sponsor ID
- PRO13050507
- Conditions
- Dermatomyositis, Polymyositis
- Keywords
- dermatomyositis, polymyositis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Rohit Aggarwal, MDUniversity, hospital or other
- Collaborators
- Mallinckrodt
- Study start
- Sep 2013
- Primary completion
- Sep 2015 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 24 Jul 2013 (estimated)
- Results
- Posted 2 Aug 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Despite its FDA approval there is very limited data on its clinical effectiveness in PM and DM. There was a recent study published in the peer-review journal Drug Design, Development and Therapy on a retrospective case series evaluating Acthar in the treatment of PM and DM. Acthar was administered to five patients who had previously failed multiple steroid and immunosuppressant treatment regimens. The patients received injections of Acthar over the course of 12 weeks or more. Improvement in PM and DM symptoms related to disease exacerbations was seen in all five patients. Symptom improvements included increased muscle strength, resolution of disease-related skin manifestations and improvements in the ability to perform tasks associated with daily living. All of these patients tolerated the treatment well with no significant side effects reported. The paper, "Treating refractory dermatomyositis or polymyositis with adrenocorticotropic hormone gel: a retrospective case series," was authored by Dr. Todd Levine, M.D., Co-Director of the Neurophysiology Department at Banner Good Samaritan Medical Center, Assistant Professor at the University of Arizona in Neurology, and Member of Phoenix Neurological Associates.
H.P. Acthar® Gel, or Acthar, is a prescription medication containing the hormone adrenocorticotropin (hormone produced and secreted by the anterior pituitary gland), also known as ACTH. H.P. Acthar Gel is a highly purified preparation of adrenocorticotropic hormone (ACTH) in a gel that is designed to provide extended release of the ACTH following injection. Acthar was originally approved by the FDA in 1952. It is approved for use in 19 different conditions including dermatomyositis and polymyositis.
Acthar is designed to provide a prolonged release of the medication after it is injected. Acthar is not a steroid; it works by helping your body produce its own natural steroid hormones, such as cortisol, corticosterone, and aldosterone. Acthar is an injection that is given intramuscularly (into the muscle). Subjects enrolled in the study will be asked to self administer Acthar two times per week. Subjects will be provided training by the principal investigator on how to perform the self injections.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Acthar Gel | Experimental | Drug: Adrenocorticotropic Hormone GelActhar 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (24 listed)
- 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:
- 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:
Key exclusion criteria (9 listed)
- 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).
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 |
|---|---|---|
| Adrenocorticotropic Hormone GelAlso: H.P. Acthar GelH.P. Acthar Gel 80 units will be self-administered subcutaneously twice weekly by the subject for a period of 6 months. | Drug | Acthar Gel |
Primary outcome
Specific Aim 1: Number of Subjects Meeting IMACS Preliminary Definition of Improvement (DOI).
Time frame: Primary end point: IMACS preliminary definition of improvement (DOI)
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.
1 secondary outcome
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 15001661Catania A, Gatti S, Colombo G, Lipton JM. Targeting melanocortin receptors as a novel strategy to control inflammation. Pharmacol Rev. 2004 Mar;56(1):1-29. doi: 10.1124/pr.56.1.1.
- Background: PubMed 20852827Catania A, Lonati C, Sordi A, Carlin A, Leonardi P, Gatti S. The melanocortin system in control of inflammation. ScientificWorldJournal. 2010 Sep 14;10:1840-53. doi: 10.1100/tsw.2010.173.
- Background: PubMed 8604274Baram TZ, Mitchell WG, Tournay A, Snead OC, Hanson RA, Horton EJ. High-dose corticotropin (ACTH) versus prednisone for infantile spasms: a prospective, randomized, blinded study. Pediatrics. 1996 Mar;97(3):375-9.
- Background: PubMed 21448451Bomback AS, Tumlin JA, Baranski J, Bourdeau JE, Besarab A, Appel AS, Radhakrishnan J, Appel GB. Treatment of nephrotic syndrome with adrenocorticotropic hormone (ACTH) gel. Drug Des Devel Ther. 2011 Mar 14;5:147-53. doi: 10.2147/DDDT.S17521.
- Background: PubMed 21792296Simsarian JP, Saunders C, Smith DM. 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. Drug Des Devel Ther. 2011;5:381-9. doi: 10.2147/DDDT.S19331. Epub 2011 Jul 11.
- Background: PubMed 22787386Levine T. Treating refractory dermatomyositis or polymyositis with adrenocorticotropic hormone gel: a retrospective case series. Drug Des Devel Ther. 2012;6:133-9. doi: 10.2147/DDDT.S33110. Epub 2012 Jun 11.
- Background: PubMed 15248228Rider LG, Giannini EH, Brunner HI, Ruperto N, James-Newton L, Reed AM, Lachenbruch PA, Miller FW; International Myositis Assessment and Clinical Studies Group. International consensus on preliminary definitions of improvement in adult and juvenile myositis. Arthritis Rheum. 2004 Jul;50(7):2281-90. doi: 10.1002/art.20349.
- Background: PubMed 23124935Oddis CV, Reed AM, Aggarwal R, Rider LG, Ascherman DP, Levesque MC, Barohn RJ, Feldman BM, Harris-Love MO, Koontz DC, Fertig N, Kelley SS, Pryber SL, Miller FW, Rockette HE; RIM Study Group. Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: a randomized, placebo-phase trial. Arthritis Rheum. 2013 Feb;65(2):314-24. doi: 10.1002/art.37754.
- Derived from this study: PubMed 37941470Fernandez AP, Gallop J, Polly S, Khanna U. Efficacy and safety of repository corticotropin injection for refractory cutaneous dermatomyositis: a prospective, open-label study. Rheumatology (Oxford). 2024 Dec 1;63(12):3370-3379. doi: 10.1093/rheumatology/kead595.
- Derived from this study: PubMed 29237618Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Efficacy and safety of adrenocorticotropic hormone gel in refractory dermatomyositis and polymyositis. Ann Rheum Dis. 2018 May;77(5):720-727. doi: 10.1136/annrheumdis-2017-212047. Epub 2017 Dec 13.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Rohit Aggarwal, MD principal investigator
University of Pittsburgh, Division of Rheumatology
2 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 Adrenocorticotropic Hormone Gel in people with Dermatomyositis and Polymyositis, run by Rohit Aggarwal, MD at 2 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 24 inclusion and 9 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: finished, as of the sponsor's last update on 1 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
Yes. Results are posted in the registry (first posted 2 Aug 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Specific Aim 1: Number of Subjects Meeting IMACS Preliminary Definition of Improvement (DOI). (time frame: Primary end point: IMACS preliminary definition of improvement (DOI)). 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 NCT01906372, which also lists 10 linked publications.
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 1 Sep 2017.