MyositisNCT03414086
Predictor of Clinical Response to Acthar in Myositis
An observational study of Myositis and Dermatomyositis, run by University of Pittsburgh at 1 site in United States.
Trial summary
Comparing the clinical effects of Acthar Gel before and after treatment and compare it to patients with inactive disease. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Myositis, Dermatomyositis, Polymyositis
- What is being tested?
- Myositis in Remission
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Nov 2017to 30 Jul 2022
- Phase
- Observational
- 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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Arthritis and Autoimmunity Center
Who may be able to join
- Healthy Controls:
- An individual will be eligible to be a control subject if his/her age is 18 years or greater.
- Myositis Remission Control Group
- Definite or probable PM or 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 (Aggarwal or Oddis) to ensure non-PM patients are not enrolled. This step is necessary since there are well known mimics of PM.
- Age is greater than or equal to 18 years
- Remission of myositis as defined by a myositis disease global activity score <1 on the MDAAT and no new immunosuppressive or glucocorticoid therapy or dose change within one year.
Who cannot join
- Healthy Controls:
- An existing diagnosis of a CTD
- A potential immune compromised state, for example, treatment with immunosuppressant or anti-rejection medication or a diagnosis of an immune deficiency disease
- Myositis Remission Control Group:
- 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.
- Severe muscle damage defined as a baseline global muscle damage score on the MDI (Myositis Damage Index) of greater than or equal to five centimeters on a ten centimeter VAS.
- Patients with malignancy within three years of screening (except basal cell cancer or squamous cell cancer of skin.
- Uncontrolled diabetes, hepatic or renal disease.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Pittsburgh
Pittsburgh, Pennsylvania, 15261
About this study
Official title: Predictor of Clinical Response to Acthar in Myositis: Phase II of Acthar Clinical Trial
Comparing the clinical effects of Acthar Gel before and after treatment and compare it to patients with inactive disease.
Read the detailed description
To compare the clinical impact of Acthar Gel at the cellular and molecular level before and after treatment and compare it to patients with inactive disease. The cohort of active myositis subjects are not actually enrolled in this study, but rather the data for those with active myositis will be obtained from a previously completed trial entitled "Efficacy and safety of Adenocorticotropic Hormone Gel in Refractory dermatomyositis and polymyositis".
- Conditions
- Myositis, Dermatomyositis, Polymyositis
- Being tested
- Healthy Control, Myositis in Remission
- Keywords
- PM, DM
- Medical subject headings
- Myositis, Dermatomyositis, Polymyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Healthy ControlOther
Ten healthy adult patients evaluating serum, PBMC's, and RNA.
Myositis in RemissionOther
Ten patients (four who have polymyositis, six dermatomyositis) from our database who are followed in the Myositis Center. We will collect serum, PBMC's, and RNA samples will be obtained at baseline and at six months. Remission is defined as a global myositis disease activity score less than or equal to 1 on the MDAAT assessments with no new immunosuppressive drug or glucocorticoid use and no increase in dose of either in the preceding year.
| Study group | Role | What they receive |
|---|---|---|
| Myositis in Remission | Subjects who are in remission with their myositis diagnosis. Receives: Other: Myositis in Remission | |
| Healthy Controls | Subjects who do not have a myositis diagnosis. Receives: Other: Healthy Control |
What is being measured
The primary outcome is the main question the study is designed to answer.
IMACS Core Set Measures Improvement
The International Myositis Assessment \& Clinical Studies (IMACS) definition of improvement: Three of any of the six core set measures improved by greater than or equal to twenty percent, with no more than two core set measuring by greater than or equal to twenty five percent.
Measured: 6 Months
Secondary outcomes (1)
Myositis Response Criteria
This criteria yields a continuous improvement score which can be more readily extrapolated to individual response in subjects allowing correlation with baseline and longitudinal immunologic markers in these subjects.
Measured: 6 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2018
- Study start6 Nov 2017
- Primary completion30 Jul 2022
- Study completion30 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (46)Papers the sponsor has linked to this study.
- Other trials for MyositisStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rohit Aggarwal, MD, University of Pittsburgh
- Investigators
- Rohit Aggarwal, MD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03414086
- Sponsor's protocol ID
- PRO16100125
- Status last verified
- Aug 2022
- First submitted
- 22 Jan 2018
- Last updated
- 9 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Outside researchers could obtain de-identified raw research data once approved from an ancillary committee.
Publications
Bernstein RM, Morgan SH, Chapman J, Bunn CC, Mathews MB, Turner-Warwick M, Hughes GR. Anti-Jo-1 antibody: a marker for myositis with interstitial lung disease. Br Med J (Clin Res Ed). 1984 Jul 21;289(6438):151-2. doi: 10.1136/bmj.289.6438.151.
PubMed 6430389Catania 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 22787386Page G, Chevrel G, Miossec P. Anatomic localization of immature and mature dendritic cell subsets in dermatomyositis and polymyositis: Interaction with chemokines and Th1 cytokine-producing cells. Arthritis Rheum. 2004 Jan;50(1):199-208. doi: 10.1002/art.11428.
PubMed 14730617Tournadre A, Lenief V, Eljaafari A, Miossec P. Immature muscle precursors are a source of interferon-beta in myositis: role of Toll-like receptor 3 activation and contribution to HLA class I up-regulation. Arthritis Rheum. 2012 Feb;64(2):533-41. doi: 10.1002/art.33350.
PubMed 22094963Tournadre A, Porcherot M, Cherin P, Marie I, Hachulla E, Miossec P. Th1 and Th17 balance in inflammatory myopathies: interaction with dendritic cells and possible link with response to high-dose immunoglobulins. Cytokine. 2009 Jun;46(3):297-301. doi: 10.1016/j.cyto.2009.02.013. Epub 2009 Mar 19.
PubMed 19303320Acosta-Rodriguez EV, Napolitani G, Lanzavecchia A, Sallusto F. Interleukins 1beta and 6 but not transforming growth factor-beta are essential for the differentiation of interleukin 17-producing human T helper cells. Nat Immunol. 2007 Sep;8(9):942-9. doi: 10.1038/ni1496. Epub 2007 Aug 5.
PubMed 17676045McGeachy MJ, Cua DJ. The link between IL-23 and Th17 cell-mediated immune pathologies. Semin Immunol. 2007 Dec;19(6):372-6. doi: 10.1016/j.smim.2007.10.012. Epub 2007 Dec 3.
PubMed 18319054Chevrel G, Page G, Granet C, Streichenberger N, Varennes A, Miossec P. Interleukin-17 increases the effects of IL-1 beta on muscle cells: arguments for the role of T cells in the pathogenesis of myositis. J Neuroimmunol. 2003 Apr;137(1-2):125-33. doi: 10.1016/s0165-5728(03)00032-8.
PubMed 12667656Tournadre A, Lenief V, Miossec P. Expression of Toll-like receptor 3 and Toll-like receptor 7 in muscle is characteristic of inflammatory myopathy and is differentially regulated by Th1 and Th17 cytokines. Arthritis Rheum. 2010 Jul;62(7):2144-51. doi: 10.1002/art.27465.
PubMed 20309865Bilgic H, Ytterberg SR, Amin S, McNallan KT, Wilson JC, Koeuth T, Ellingson S, Newman B, Bauer JW, Peterson EJ, Baechler EC, Reed AM. Interleukin-6 and type I interferon-regulated genes and chemokines mark disease activity in dermatomyositis. Arthritis Rheum. 2009 Nov;60(11):3436-46. doi: 10.1002/art.24936.
PubMed 19877033
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predictor of Clinical Response to Acthar in Myositis: Phase II of Acthar Clinical Trial
- ClinicalTrials.gov ID
- NCT03414086
- Acronym / sponsor ID
- PRO16100125
- Conditions
- Myositis, Dermatomyositis, Polymyositis
- Keywords
- PM, DM
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- Study start
- 6 Nov 2017 (actual)
- Primary completion
- 30 Jul 2022 (actual)
- Study completion
- 30 Jul 2022 (actual)
- First posted
- 29 Jan 2018 (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.
Comparing the clinical effects of Acthar Gel before and after treatment and compare it to patients with inactive disease.
Detailed description
To compare the clinical impact of Acthar Gel at the cellular and molecular level before and after treatment and compare it to patients with inactive disease. The cohort of active myositis subjects are not actually enrolled in this study, but rather the data for those with active myositis will be obtained from a previously completed trial entitled "Efficacy and safety of Adenocorticotropic Hormone Gel in Refractory dermatomyositis and polymyositis".
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Myositis in Remission | – | Other: Myositis in RemissionSubjects who are in remission with their myositis diagnosis. |
| Healthy Controls | – | Other: Healthy ControlSubjects who do not have a myositis diagnosis. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Arthritis and Autoimmunity Center
Key inclusion criteria (7 listed)
- Healthy Controls:
- Myositis Remission Control Group
Key exclusion criteria (8 listed)
- Healthy Controls:
- Myositis Remission Control Group:
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 |
|---|---|---|
| Healthy ControlTen healthy adult patients evaluating serum, PBMC's, and RNA. | Other | Healthy Controls |
| Myositis in RemissionTen patients (four who have polymyositis, six dermatomyositis) from our database who are followed in the Myositis Center. We will collect serum, PBMC's, and RNA samples will be obtained at baseline and at six months. Remission is defined as a global myositis disease activity score less than or equal to 1 on the MDAAT assessments with no new immunosuppressive drug or glucocorticoid use and no increase in dose of either in the preceding year. | Other | Myositis in Remission |
Primary outcome
IMACS Core Set Measures Improvement
Time frame: 6 Months
The International Myositis Assessment \& Clinical Studies (IMACS) definition of improvement: Three of any of the six core set measures improved by greater than or equal to twenty percent, with no more than two core set measuring by greater than or equal to twenty five percent.
1 secondary outcome
Myositis Response Criteria
Time frame: 6 Months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2022.
- Background: PubMed 6430389Bernstein RM, Morgan SH, Chapman J, Bunn CC, Mathews MB, Turner-Warwick M, Hughes GR. Anti-Jo-1 antibody: a marker for myositis with interstitial lung disease. Br Med J (Clin Res Ed). 1984 Jul 21;289(6438):151-2. doi: 10.1136/bmj.289.6438.151.
- 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 14730617Page G, Chevrel G, Miossec P. Anatomic localization of immature and mature dendritic cell subsets in dermatomyositis and polymyositis: Interaction with chemokines and Th1 cytokine-producing cells. Arthritis Rheum. 2004 Jan;50(1):199-208. doi: 10.1002/art.11428.
- Background: PubMed 22094963Tournadre A, Lenief V, Eljaafari A, Miossec P. Immature muscle precursors are a source of interferon-beta in myositis: role of Toll-like receptor 3 activation and contribution to HLA class I up-regulation. Arthritis Rheum. 2012 Feb;64(2):533-41. doi: 10.1002/art.33350.
- Background: PubMed 19303320Tournadre A, Porcherot M, Cherin P, Marie I, Hachulla E, Miossec P. Th1 and Th17 balance in inflammatory myopathies: interaction with dendritic cells and possible link with response to high-dose immunoglobulins. Cytokine. 2009 Jun;46(3):297-301. doi: 10.1016/j.cyto.2009.02.013. Epub 2009 Mar 19.
- Background: PubMed 17676045Acosta-Rodriguez EV, Napolitani G, Lanzavecchia A, Sallusto F. Interleukins 1beta and 6 but not transforming growth factor-beta are essential for the differentiation of interleukin 17-producing human T helper cells. Nat Immunol. 2007 Sep;8(9):942-9. doi: 10.1038/ni1496. Epub 2007 Aug 5.
- Background: PubMed 18319054McGeachy MJ, Cua DJ. The link between IL-23 and Th17 cell-mediated immune pathologies. Semin Immunol. 2007 Dec;19(6):372-6. doi: 10.1016/j.smim.2007.10.012. Epub 2007 Dec 3.
- Individual participant dataSharing planned: Yes. Outside researchers could obtain de-identified raw research data once approved from an ancillary committee.
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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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myositis and Dermatomyositis, run by University of Pittsburgh 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 8 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 9 Aug 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
IMACS Core Set Measures Improvement (time frame: 6 Months). 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 NCT03414086, which also lists 46 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 9 Aug 2022.