Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis

Study Purpose

This is a pilot study to determine whether further research is warranted to assess whether tofacitinib is an effective steroid sparing treatment for pulmonary sarcoidosis. The primary endpoint for this study is a 50% or greater reduction in corticosteroid requirement.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years - 89 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - Meet World Association of Sarcoidosis and other Granulomatous Disorders (WASOG) definition of pulmonary sarcoid - Histologically proven sarcoid - Evidence of pulmonary sarcoid on chest radiograph - FVC of > 50% - Require 15-30mg/day of prednisone or equivalent corticosteroid to control sarcoidosis.
  • - Stable dose of prednisone or equivalent corticosteroid for 4 weeks prior to enrollment.

Exclusion Criteria:

  • - May be taking methotrexate but not other immunosuppressive or immunomodulatory treatments in the two months prior to study period.
This includes but is not limited to azathioprine, cyclophosphamide, leflunomide, mycophenolate mofetil, cyclosporine, tacrolimus, and biologic medications.
  • - Patients requiring >30mg/day prednisone or equivalent.
  • - Pregnant or lactating women.
  • - Hemoglobin < 9g/dL or hematocrit < 30% - White blood cell count <3.0 K/cu mm - Absolute neutrophil count <1.2 K/cu mm - Platelet count <100 K/cu mm - Subjects with an estimated glomerular filtration rate (GFR) ≤40 ml/min - Subjects with a total bilirubin, aspartate aminotransferase (AST), or alanine aminotransferase (ALT) more than 1.5 times the upper limit of normal at screening.
  • - Severe, progressive, or uncontrolled chronic liver disease including fibrosis, cirrhosis, or recent or active hepatitis.
  • - History of any lymphoproliferative disorder such as Epstein Barr virus (EBV) related lymphoproliferative disorder, history of lymphoma, leukemia, or signs and symptoms suggest of current lymphatic disease.
  • - Current malignancy or history of malignancy, with the exception of adequately treated or excised non-metastatic basal cell or squamous cell cancer of the skin, or cervical carcinoma in situ.
  • - Have or have had an opportunistic infection (e.g., herpes zoster [shingles], cytomegalovirus, Pneumocystis carinii, aspergillosis and aspergilloma, histoplasmosis, or mycobacteria other than TB) within 6 months prior to screening.
  • - Have a known infection with human immunodeficiency virus (HIV) - Have current signs and symptoms of systemic lupus erythematosus, or severe, progressive, or uncontrolled renal, hepatic, hematologic, endocrine, pulmonary, cardiac (New York Heart Association class III or IV), neurologic, or cerebral diseases (with the exception of sarcoidosis).

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03793439
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Oregon Health and Science University
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Jim Rosenbaum, MD
Principal Investigator Affiliation Oregon Health and Science University
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

OtherIndustry
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Sarcoidosis, Pulmonary, Sarcoidosis Lung, Sarcoidosis
Additional Details

Primary Objectives: Objective 1: Test the hypothesis that the addition of tofacitinib will allow patients with sarcoidosis to have 50% or greater reduction in their corticosteroid requirement without a significant decrease in pulmonary function testing, and with a similar quality of life as measured by a validated questionnaire (1). Objective 2: Test the hypothesis that the addition of tofacitinib will result in significantly decreased expression of signal transducer and activator of transcription (STAT)-1 dependent gene expression. Outline: This is a 16-week open-label, interventional, proof of concept, hypothesis-generating study. All subjects will receive Tofacitinib 5mg twice daily for 16 weeks. After four weeks on Tofacitinib, the corticosteroid will be tapered per a pre-defined protocol; once a reduction of 50% has been achieved, any further taper will be per physician discretion. After 16 weeks, subjects who meet the primary end-point will be permitted an optional one year open-label extension.

Arms & Interventions

Arms

Experimental: Open-label treatment

All subjects will receive tofacitinib 5mg twice daily from week 0 to week 16, and a corticosteroid taper starting at week 16. Participants also undergo spirometry, RNA sequence testing, and laboratory evaluations.

Experimental: Open-label extension

After 16 weeks, subjects who meet the primary end-point will be permitted an optional one year open-label extension.

Interventions

Drug: - Tofacitinib 5mg Oral Tablet [Xeljanz] 16 week trial

Tofacitinib 5mg oral table twice daily for 16 weeks

Diagnostic Test: - Spirometry

Spirometry testing at baseline, week 4, week 8, week 12, and week 16

Genetic: - RNA Sequencing

RNA sequencing test at baseline and week 16

Diagnostic Test: - Laboratory testing

Laboratory testing at baseline and weeks 2, 4, 8, 12 and 16

Drug: - Corticosteroid

Taper corticosteroids starting at week 4

Drug: - Tofacitinib 5mg [Xeljanz] 1 year open-label extension

After 16 weeks, subjects who meet the primary end-point will be permitted an optional one year open-label extension.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Oregon Health & Science University, Portland, Oregon

Status

Recruiting

Address

Oregon Health & Science University

Portland, Oregon, 97239

Site Contact

Marcia A Friedman, MD

friedmam@ohsu.edu

503-494-8637

Terms of Service

The PFF does not promote or represent that any investigational new drugs mentioned are safe or effective. The content provided on clinical trials is for informational purposes only and is not a substitute for medical consultation with your healthcare provider. We do not recommend or endorse any specific study and you are advised to discuss the information shown with your healthcare provider. While we believe the information presented on this website to be accurate at the time of writing, we do not guarantee that its contents are correct, complete, or applicable to any particular individual situation. We strongly encourage individuals to seek out appropriate medical advice and treatment from their physicians. We cannot guarantee the availability of any clinical trial listed and will not be responsible if you are considered ineligible to participate in a given clinical trial. We are also not liable for any injury arising as a result of participation. The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. For a full description of terms please refer to our Terms, Conditions & Privacy.

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