CompletedPhase 1/2

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

United States24 participantsStarted 2016-11-01

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Moderate or severe Chronic Graft-Versus-Host Disease (cGVHD) (after allogeneic hematopoietic stem cell transplantation) diagnosed and staged per National Institutes of Health (NIH) criteria. Responses to Janus kinase (JAK) inhibitors have not been restricted to specific organs, so any organ involvement is eligible.
✓. Age greater than or equal to 18 years of age. Because inadequate dosing or adverse event data are currently available on the use of baricitinib in patients \<18 years of age, children are excluded from this study.
✓. Karnofsky performance score \>50%
✓. Chronic GVHD that did not respond to high-dose corticosteroids (prednisone at 1.0 mg/kg/day for at least 1 week or prednisone at 0.5 mg/kg/day or 1 mg/kg every other day for at least 4 weeks), or second-line therapy (any).
✓. If patient is taking systemic therapy for cGVHD at the time of enrollment, they must be on a stable or tapering doses in the preceding 4 weeks.
✓. Patients must have normal organ and marrow function as defined below:
✓. Primary malignancy for which the patient received transplant has been stable for 3 months prior to enrollment on study.
✓. The effects of baricitinib on human fetal development are unknown. Women of child-bearing potential and men must agree to use 2 effective forms of contraception (hormonal or barrier method of birth control; abstinence) for the duration of study participation and for at least 7 days after study drug exposure. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, or if a man's partner becomes pregnant or suspects she is pregnant while he is participating in this study, she or he should inform their treating physician immediately.

What they're measuring

Phase 1: Number of Participants With Dose-Limiting Toxicities (DLT)

Timeframe: Starting at each dose level initiation, every 2 weeks up to the first 4 weeks of each dose

Phase 2: Number of Participants With Any Serious and/or Non-Serious Grades 3, 4, and/or 5 Adverse Events Due to Drug

Timeframe: every 4 weeks through study completion (up to 48 weeks)

Phase 2: Overall Response at 24 Weeks

Timeframe: 24 weeks

Trial details

NCT IDNCT02759731

SponsorNational Cancer Institute (NCI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2022-06-30

Results submitted2023-06-28

View on ClinicalTrials.gov More Chronic Graft vs Host Disease trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Moderate or severe Chronic Graft-Versus-Host Disease (cGVHD) (after allogeneic hematopoietic stem cell transplantation) diagnosed and staged per National Institutes of Health (NIH) criteria. Responses to Janus kinase (JAK) inhibitors have not been restricted to specific organs, so any organ involvement is eligible.
✓. Age greater than or equal to 18 years of age. Because inadequate dosing or adverse event data are currently available on the use of baricitinib in patients \<18 years of age, children are excluded from this study.
✓. Karnofsky performance score \>50%
✓. Chronic GVHD that did not respond to high-dose corticosteroids (prednisone at 1.0 mg/kg/day for at least 1 week or prednisone at 0.5 mg/kg/day or 1 mg/kg every other day for at least 4 weeks), or second-line therapy (any).
✓. If patient is taking systemic therapy for cGVHD at the time of enrollment, they must be on a stable or tapering doses in the preceding 4 weeks.
✓. Patients must have normal organ and marrow function as defined below:
✓. Primary malignancy for which the patient received transplant has been stable for 3 months prior to enrollment on study.
✓. The effects of baricitinib on human fetal development are unknown. Women of child-bearing potential and men must agree to use 2 effective forms of contraception (hormonal or barrier method of birth control; abstinence) for the duration of study participation and for at least 7 days after study drug exposure. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, or if a man's partner becomes pregnant or suspects she is pregnant while he is participating in this study, she or he should inform their treating physician immediately.

What they're measuring

Phase 1: Number of Participants With Dose-Limiting Toxicities (DLT)

Timeframe: Starting at each dose level initiation, every 2 weeks up to the first 4 weeks of each dose

Phase 2: Number of Participants With Any Serious and/or Non-Serious Grades 3, 4, and/or 5 Adverse Events Due to Drug

Timeframe: every 4 weeks through study completion (up to 48 weeks)

Phase 2: Overall Response at 24 Weeks

Timeframe: 24 weeks

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

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

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

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Exclusion criteria