Study of Baricitinib, a JAK1/2 Inhibitor, in Chronic Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

Conditions

• Chronic Graft vs Host Disease
• Chronic Graft-Versus-Host Disease

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Baricitinib — DRUG
  Cycle=28 days: Baricitinib: 1mg-4mg by mouth (PO) every day (QD)

Study Details

Background: Chronic graft versus host disease (cGVHD) can affect people who had a hematopoietic stem cell transplant using donor cells. It is often fatal. It is usually treated with high doses of steroids. But that helps only about half the people in the long term. Researchers want to see if a drug called baricitinib can help people with cGVHD that has not responded to therapy. The drug inhibits the proteins involved in communication in the immune system. These proteins may play a role in cGVHD and other inflammatory diseases. Objectives: To test the safety and effectiveness of baricitinib in people with cGVHD that has not responded to therapy. Eligibility: Adults 18 and older with cGVHD that has not responded to therapy. Design: Participants will be screened with a medical history, physical exam, and blood and urine tests. They will have lung and heart tests and chest scans. Baseline visit: Participants will have: Medical history Physical exam Blood tests Tests for infectious diseases Skin, eye, and teeth evaluations Rehabilitation and occupational medicine evaluations Photos of any lesions Gynecology evaluation (females) The study will occur in 28-day cycles. Participants will take the study drug by mouth every day for 3 cycles. Some will take it for 3 or 6 more cycles. Participants will have a few visits during each cycle. They will repeat some previous tests. They may also have scans and questionnaires. Participants will have a visit when they stop taking the drug and another 3 months later. They will repeat a few study tests. They will have follow-up calls for 2 years.

Key Dates

Start date

Nov 1, 2016

Status verified

Sep 2024

Primary completion

Jun 30, 2022

Completion

May 30, 2024

Study Design

Enrollment

24 participants (actual)

Allocation

NA

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Baricitinib
  Single arm, intra-patient dose escalation. Arm 1 (starting dose) -Baricitinib starting at a dose of 2mg daily for 12 weeks. If the response at 12 weeks is a complete response (CR) and there has not been a dose-limiting toxicity (DLT), the dose will remain at 2mg daily for an additional 12 weeks. Arm 2 (dose escalation) - If the response is a partial response (PR) or stable disease (from cohort 1), the dose will be increased to 4mg daily for an additional 12 weeks. If there has been a DLT within the first 4 weeks will have a dose reduction back to 2mg daily. Each participant is exposed to both dose levels (with the exception of participants who developed toxicity or progressive disease that caused them to discontinue study early before they were able to undergo the per-protocol dose escalation at 12 weeks).

Primary Outcome Measure

Phase 1: Number of Participants With Dose-Limiting Toxicities (DLT) [ Time Frame: Starting at each dose level initiation, every 2 weeks up to the first 4 weeks of each dose ]

Locations (1)

Find similar trials in Bethesda, MD