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DermatomyositisPhase 1STIRNCT03002649

Study of Tofacitinib in Refractory Dermatomyositis

A Phase 1 study testing Tofacitinib in people with Dermatomyositis, run by Johns Hopkins University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to obtain preliminary data regarding the safety and efficacy of Janus kinase (JAK) inhibitor, tofacitinib, in adults with active, treatment-refractory dermatomyositis. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Dermatomyositis
What is being tested?
Tofacitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
Jan 2017to 30 Sep 2020
Phase
Phase 1
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
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Study subjects must meet the following criteria:
  • Definite or probable dermatomyositis by Bohan and Peter Criteria at least 6 months before screening
  • Active skin disease as defined by a CDASI score of at least 5
  • Skin biopsy proven disease
  • Although not mandatory, patients with muscle weakness are eligible for enrollment. Those with active muscle disease must have a Manual Muscle Testing (MMT-8) score < 142 out of 150
  • Age > 18
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  • Refractory myositis is defined by active disease despite a 12 week trial of steroids and with failure of response to at least prednisone and 1 other first line immunosuppressive agents (e.g. methotrexate, mycophenolate mofetil, or azathioprine) OR have demonstrated significant toxicity or intolerance to such therapies
  • Maximum prednisone dose allowed will be 20mg/daily at time of entry to study provided that the dose has been stable for at least 2 weeks prior to baseline. Patients should not have received a daily therapy of more than 80mg of prednisone equivalent within 8 weeks prior to study entry
  • Negative cancer screening conducted in the 6 months prior to screening visit
  • Washout of immunosuppressive agents will be as follows:
  • Azathioprine, mycophenolate, tacrolimus: 12-16 weeks prior to first dose of study drug;
  • Rituximab: 12 months;
  • Intravenous Immunoglobulin (IVIg): 3 months;
  • Cyclophosphamide: 1 year;
  • Methotrexate: 12-16 weeks.
  • Women of child-bearing potential must have negative pregnancy test and be willing to undergo urine pregnancy testing at every on-site visit for the duration of the study
  • Must provide informed consent
  • Must be willing and able to comply with the requirements of the protocol

Who cannot join

  • The presence of any of the following excludes subject participation in the study:
  • Use of other investigational drugs at the time of enrollment
  • History of hypersensitivity to any of the study drugs or drugs of similar chemical classes
  • DM patients having overlap myositis attributable to other causes such as scleroderma, arthritis, statin myopathy, steroid induced myopathy and/or significant organ damage e.g. lupus nephritis, central nervous system are present
  • Late stage DM whose muscle weakness, according to the Investigator, could be attributable to muscle damage rather than myositis disease activity
  • Patients with other types of myositis or myopathies: polymyositis, paraneoplastic myositis, inclusion body myositis, metabolic or drug induced myopathy, dystrophies
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  • Inclusion body myositis, Juvenile dermatomyositis or polymyositis, or myositis in overlap with other rheumatic diseases such as lupus, scleroderma, Sjogren's, or vasculitis
  • Patients with advanced clinically symptomatic interstitial lung disease
  • Pregnancy or breast-feeding patients
  • History of bowel rupture or inflammatory bowel diseases
  • History of tuberculosis or mycobacterial infections
  • Recent infection in the past 4 weeks before entry of study
  • History of any malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases
  • Active systemic bacterial, viral or fungal infections, or diagnosis of AIDS, Hepatitis B, Hepatitis C infection
  • Diverticulitis or ulcers in stomach or intestines
  • Evidence of any other acute or chronic infectious diseases
  • Have received any live or live attenuated vaccines (including varicella or measles) within 2 months prior to study enrollment
  • Patients with any of the following hepatic conditions prior to study: (a) history of chronic liver or biliary disease, (b) total conjugated bilirubin greater than 1.5 times upper limits of normal (ULN) range, unless in the context of Gilbert's syndrome, (c) alkaline phosphatase greater than 1.5 times the ULN range, (d) aspartate transaminase (AST), alanine transaminase (ALT) greater than 3 times the ULN if the elevation of AST or ALT, according to the investigator, is attributable to liver disease. Patients with elevated AST/ALT due to myositis disease activity are eligible, (e) Gamma-glutamyltransferase (GGT) greater than 3 times the ULN range
  • Current or recent history of uncontrolled renal, hepatic, hematological, gastrointestinal, metabolic, endocrine, pulmonary, cardiac, or neurological disease
  • Blood dyscrasias within 3 months prior to the first dose of study medication, including confirmed:
  • Hemoglobin <9 g/dL or Hematocrit <30%;
  • White blood cell count <3.0 x 109/L;
  • Absolute neutrophil count <1.2 x 109/L;
  • Platelet count <100 x 109/L.
  • Estimated glomerular filtration rate (GFR) <40 ml/min based on Cockcroft-Gault calculation

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Johns Hopkins University

    Baltimore, Maryland, 21224

Other trials in United States

About this study

Official title: Study of Tofacitinib in Refractory Dermatomyositis (STIR): Proof of Concept, Open-Label Study of 10 Patients

The purpose of this study is to obtain preliminary data regarding the safety and efficacy of Janus kinase (JAK) inhibitor, tofacitinib, in adults with active, treatment-refractory dermatomyositis.

Read the detailed description

Dermatomyositis (DM) is a rare, progressively debilitating disorder that affects the muscle (causing weakness) and skin (causing a rash) in most affected patients. DM can also involve multiple body systems including the lungs, joints, gut and heart. Therapy for DM involves administering corticosteroids, typically with an immunosuppressive agent, but treatment options for refractory DM are very limited. This research is being done to determine the safety and effectiveness of a Janus kinase (JAK) inhibitor called Tofacitinib in adults with active, treatment-refractory dermatomyositis. The investigators will also look for specific biomolecular changes in blood, skin, and muscle when it is exposed to Tofacitinib. Tofacitinib is approved by the Food and Drug Administration (FDA) for the treatment of rheumatoid arthritis. The study aim is to conduct a 12-week, open-label, pilot study with up to 10 patients who have refractory DM to assess whether a JAK inhibitor effectively and safely reduces the symptoms of DM in both the skin and muscle. This study consists of up to 9 study visits over 6 months. There is an optional treatment extension period of 4 weeks. All subjects will undergo follow-up assessments 4 weeks after stopping treatment. Results from this study will contribute to the design of future trials that will further define the role of JAK inhibitors in the treatment of patients with dermatomyositis.

Conditions
Dermatomyositis
Being tested
Tofacitinib
Keywords
Dermatomyositis, Tofacitinib, Refractory dermatomyositis
Medical subject headings
Dermatomyositis

What participants receive

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

  • TofacitinibDrug

    Also called Xeljanz XR, Tofacitinib citrate

    Tofacitinib comes as an extended-release (XR) (long-acting) tablet to take by mouth. The extended-release tablet is usually taken with or without food once daily.

Study groups in NCT03002649
Study groupRoleWhat they receive
TofacitinibExperimentalAll subjects will be provided Tofacitinib 11mg tablets for oral administration once daily. 12-week treatment period with optional 4-week treatment extension period.
Receives: Drug: Tofacitinib

What is being measured

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

  1. Number of participants who achieve International Myositis Assessment and Clinical Studies (IMACS) Definition of Improvement (DOI)

    IMACS DOI is 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 manual muscle testing)

    Measured: Up to 12 weeks

Secondary outcomes (3)

  1. Change from baseline in CDASI activity score

    Measured: Up to 16 weeks

  2. Safety and tolerability of tofacitinib as assessed by frequency of adverse events reported and observed

    Measured: Up to 16 weeks

  3. Safety and tolerability of tofacitinib as assessed by incidence of adverse events reported and observed

    Measured: Up to 16 weeks

Timeline

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

  1. First posted26 Dec 2016 (estimated)
  2. Study startJan 2017
  3. Primary completion30 Sep 2020
  4. Study completion30 Sep 2020

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03002649
Sponsor's protocol ID
IRB00084227
Acronym
STIR
Status last verified
Jun 2020
First submitted
20 Dec 2016
Last updated
6 Nov 2020
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Publications

  • Paik JJ, Casciola-Rosen L, Shin JY, Albayda J, Tiniakou E, Leung DG, Gutierrez-Alamillo L, Perin J, Florea L, Antonescu C, Leung SG, Purwin G, Koenig A, Christopher-Stine L. Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients. Arthritis Rheumatol. 2021 May;73(5):858-865. doi: 10.1002/art.41602. Epub 2021 Mar 24.

    PubMed 33258553

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Tofacitinib in people with Dermatomyositis, run by Johns Hopkins University 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 18 inclusion and 25 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 6 Nov 2020. 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.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 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?

Number of participants who achieve International Myositis Assessment and Clinical Studies (IMACS) Definition of Improvement (DOI) (time frame: Up to 12 weeks). 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 NCT03002649, which also lists 1 linked publication.

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 6 Nov 2020.

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