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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-05-15

Study Completion Date

2021-06-24

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Sarcoidosis, Pulmonary Sarcoidosis Lung Sarcoidosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open-label, interventional, proof of concept, hypothesis-generating study

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Tofacitinib 5mg Oral Tablet [Xeljanz] 16 week trial

Intervention Type DRUG

Tofacitinib 5mg oral table twice daily for 16 weeks

Spirometry

Intervention Type DIAGNOSTIC_TEST

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

RNA Sequencing

Intervention Type GENETIC

RNA sequencing test at baseline and week 16

Laboratory testing

Intervention Type DIAGNOSTIC_TEST

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

Corticosteroid

Intervention Type DRUG

Taper corticosteroids starting at week 4

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

Intervention Type DRUG

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

Interventions

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Tofacitinib 5mg Oral Tablet [Xeljanz] 16 week trial

Tofacitinib 5mg oral table twice daily for 16 weeks

Intervention Type DRUG

Spirometry

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

Intervention Type DIAGNOSTIC_TEST

RNA Sequencing

RNA sequencing test at baseline and week 16

Intervention Type GENETIC

Laboratory testing

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

Intervention Type DIAGNOSTIC_TEST

Corticosteroid

Taper corticosteroids starting at week 4

Intervention Type 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.

Intervention Type DRUG

Other Intervention Names

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Xeljanz tofacitinib Pulmonary function test Blood work Corticosteroid taper Prednisone taper Steroid taper tofacitinib Xeljanz

Eligibility Criteria

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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
* Forced vital capacity 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).

Minimum Eligible Age

18 Years

Maximum Eligible Age

89 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No