Chronic Graft vs Host DiseasePhase 1/2NCT02759731
Study of Baricitinib, a JAK1/2 Inhibitor, in Chronic Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
A Phase 1/2 study testing Baricitinib in people with Chronic Graft vs Host Disease and Chronic Graft-Versus-Host Disease, run by National Cancer Institute (NCI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Graft vs Host Disease, Chronic Graft-Versus-Host Disease
- What is being tested?
- Baricitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Nov 2016to 30 May 2024
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
None listed.
Who cannot join
- Systemic immune suppression or systemic therapy for cGVHD started within preceding 4 weeks.
- Hypersensitivity to JAK inhibitors.
- Any serious medical condition within the previous 4 weeks which places the subject at an unacceptable risk if he or she were to participate in the study or confounds the ability to interpret data from the study, including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmias, acute kidney injury, or psychiatric illness/social situations that would limit compliance with study requirements.
- Uncontrolled infection, including active human immunodeficiency virus (HIV-1), Hepatitis B virus (HBV) and/or Hepatitis C virus (HCV) infection (positive HBV or HCV viral load in the setting of positive HBV core antibody or surface antibody or HCV antibody). History of HBV or HCV is allowed if there is no uncontrolled viral infection. Because the study agent may impact response to infections, patients with any active viral infection are excluded.
- Recurrent or progressive malignancy requiring anticancer treatment.
- Other cancer except that for which the transplant was done <2 years before study entry, except non-melanoma skin cancer or carcinoma in situ of the uterine cervix or breast.
Show all 9Show fewer
- Patients who are receiving any other investigational agents.
- NIH lung score 3.
- Pregnant women are excluded from this study because the teratogenic effects of baricitinib are unknown. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with baricitinib, breastfeeding should be discontinued if the mother is treated with this agent.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Phase 1/2 Study of Baricitinib, a JAK1/2 Inhibitor, in Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (SCT)
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.
Read more
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.
Read the detailed description
- Background:
- Chronic graft-versus-host disease (cGVHD) is the leading cause of non-relapse morbidity and mortality in persons after allogeneic hematopoietic stem cell transplantation (SCT).
- Approximately 50% of patients with cGVHD have disease refractory to systemic corticosteroids; currently, there is no standard second-line therapy.
- The Janus kinase/signal transducers and activators of transcription (JAK/STAT) pathway relays the signaling function of several inflammatory cytokines that have a role in GVHD (interferon (IFN)-gamma, Interleukin-2 (IL-2), Interleukin-6 (IL-6), Interleukin-12 (IL-12)).
- Murine models have demonstrated activity of JAK inhibitors in graft-versus-host disease.
- Baricitinib is a potent and selective inhibitor of Janus kinase 1 (JAK1) and Janus kinase 1 (JAK2) that has demonstrated anti-inflammatory effects and a good safety profile in patients with rheumatoid arthritis but has not been evaluated in GVHD.
- Objectives:
- To determine the safety and tolerability of baricitinib in patients with cGVHD that is refractory to steroids
- To determine the efficacy of baricitinib in patients with cGVHD that is refractory to steroids
- Eligibility:
- Inclusion:
- Age greater than or equal to 18 years
- Moderate or severe cGVHD per NIH consensus criteria
- Karnofsky performance status greater than or equal to 50%
- cGVHD 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)
- Receiving stable or tapering doses of systemic therapy in the preceding 4 weeks if taking systemic therapy for cGVHD
- Exclusion:
- Neutrophils <1.0x10^9/L, platelets <50X10^9/L, creatinine greater than or equal to 1.5 times the upper limit of normal or estimated creatinine clearance <50mL/min/1.73m^2 (Cockroft-Gault formula), serum aspartate aminotransferase or alanine aminotransferase concentration >3x upper limit of normal (ULN) or total bilirubin greater than or equal to 1.5x ULN
- Progressive malignancy, uncontrolled infection or any major organ dysfunction as defined by the protocol
- Design:
- This is a Phase 1/2 trial to determine the safety and efficacy of baricitinib in patients with cGVHD that is refractory to steroids.
- Patients will initially be treated with baricitinib at 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, with the primary response assessment at 24 weeks of total treatment. If the response is a partial response (PR) or stable disease, the dose will be increased to 4mg daily for an additional 12 weeks, with the primary response assessment at 24 weeks of total treatment. If there is progression of disease at any time within the first 12 weeks, the dose can be increased to 4mg daily at that time, and patients will continue for a total of 24 weeks of treatment. Patients will have the option to continue baricitinib for an additional 6 months as tolerated if they have stable or responding disease.
- The co-primary endpoint of safety will be determined by rate, severity, and duration of adverse events based on Common Terminology Criteria for Adverse Events (CTCAE) v4 criteria. Assessment for DLTs will occur every 2 weeks during the first 4 weeks of each dose level. Safety monitoring will occur every 4 weeks thereafter.
- The co-primary endpoint of efficacy will be defined as rate of overall response at 24 weeks per National Institutes of Health (NIH) consensus criteria (CR or PR).
- Peripheral blood samples will be collected prior to treatment, at 2 weeks, at 12 weeks and every 12 weeks thereafter to evaluate cytokine and cellular profiles, STAT phosphorylation, candidate chronic GVHD biomarkers. Pharmacokinetic studies will also be performed at each dose level.
- In an initial futility analysis, if 0 of the first 7 patients enrolled in cohort 1 have responded at 12 weeks, then a 2nd cohort of patients will be accrued to start treatment at the higher dose (4mg daily). Otherwise, if 1 or more of the first 7 patients respond in cohort 1, then 21 evaluable patients will be treated in cohort 1. Similarly, if the second cohort is used, and if 0 of the 7 patients enrolled in this second cohort have responded at 12 weeks, then no further patients will be accrued. Otherwise, if 1 or more of the first 7 patients respond in cohort 2, then 21 evaluable patients will be treated in cohort 2.
- A total of 21 evaluable patients will be enrolled in either cohort 1 or 2 as appropriate, in order to have 80% power to detect a response rate consistent with 30% and ruling out 10%, with a one-sided significance level of 0.10 for the cohort. As an early stopping rule for safety, if 2/3 or greater patients at any given dose level experiences a dose limiting toxicity requiring dose reduction or discontinuation, that dose will not be subsequently used, and no further dose escalation will take place.
- Being tested
- Baricitinib
- Keywords
- Murine Models, Rheumatoid Arthritis, Corticosteroids, Systemic Therapy, Inflammatory Cytokines
- Medical subject headings
- Bronchiolitis Obliterans Syndrome, Arthritis, Rheumatoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BaricitinibDrug
Also called Olumiant
Cycle=28 days: Baricitinib: 1mg-4mg by mouth (PO) every day (QD)
| Study group | Role | What they receive |
|---|---|---|
| Baricitinib | Experimental | 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). Receives: Drug: Baricitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: Number of Participants With Dose-Limiting Toxicities (DLT)
DLT is defined as any grade ≥3 non-hematologic or grade ≥4 hematologic adverse event, or hemoglobin <6.5 g/dL, within 4 weeks of starting any dose level (1mg, 2mg, or 4 mg) except those that are clearly and incontrovertibly due to extraneous causes. Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). Grade 3 is severe. Grade 4 is life-threatening.
Measured: 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
Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). Grade 3 is severe. Grade 4 is life-threatening. And Grade 5 is death related to adverse event.
Measured: every 4 weeks through study completion (up to 48 weeks)
Phase 2: Overall Response at 24 Weeks
Response was assessed by the National Institutes of Health (NIH) chronic graft-versus-host disease (cGVHD) response criteria. Complete Response (CR) is defined as resolution of all manifestations in each organ or site. Partial Response (PR) is defined as improvement at least 1 organ or site without progression in any other organ or site. Lack of Response including unchanged, mixed response, and disease progression. Mixed response is a CR or PR in at least 1 organ accompanied by progression in another organ. Unchanged is outcomes that do not meet the criteria for CR, PR, disease progression, or mixed response. Disease Progression is a change in the manifestations in each organ or site that does not meet the criteria for CR, PR, mixed response, unchanged or lack of response.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2016 (estimated)
- Study start1 Nov 2016
- Primary completion30 Jun 2022
- Study completion30 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Graft vs Host DiseaseStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Principal investigator, Steven Pavletic, M.D., National Cancer Institute (NCI)
- Investigators
- Steven Z Pavletic, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02759731
- Sponsor's protocol ID
- 160094
- Other IDs
- 16-C-0094
- Status last verified
- Sep 2024
- First submitted
- 14 Apr 2016
- Last updated
- 8 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request.
Publications
Holtzman NG, Im A, Curtis L, Schulz E, Sharma R, Pirsl F, Berman A, Ostojic A, Magone de Quadros Costa MT, Cowen EW, Mays J, Comis LE, Joe G, Peer C, Figg W, Rose J, El Jurdi N, Pouzolles M, Hakim F, Taylor N, Pavletic SZ. Baricitinib for refractory chronic graft-versus-host disease: results of a phase 1/2 study. Blood Adv. 2026 Sep 8;10(17):5751-5761. doi: 10.1182/bloodadvances.2025018898.
PubMed 42200447
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study of Baricitinib, a JAK1/2 Inhibitor, in Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (SCT)
- ClinicalTrials.gov ID
- NCT02759731
- Acronym / sponsor ID
- 160094
- Keywords
- Murine Models, Rheumatoid Arthritis, Corticosteroids, Systemic Therapy, Inflammatory Cytokines
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 1 Nov 2016 (actual)
- Primary completion
- 30 Jun 2022 (actual)
- Study completion
- 30 May 2024 (actual)
- First posted
- 3 May 2016 (estimated)
- Results
- Posted 25 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
- Background:
- Chronic graft-versus-host disease (cGVHD) is the leading cause of non-relapse morbidity and mortality in persons after allogeneic hematopoietic stem cell transplantation (SCT).
- Approximately 50% of patients with cGVHD have disease refractory to systemic corticosteroids; currently, there is no standard second-line therapy.
- The Janus kinase/signal transducers and activators of transcription (JAK/STAT) pathway relays the signaling function of several inflammatory cytokines that have a role in GVHD (interferon (IFN)-gamma, Interleukin-2 (IL-2), Interleukin-6 (IL-6), Interleukin-12 (IL-12)).
- Murine models have demonstrated activity of JAK inhibitors in graft-versus-host disease.
- Baricitinib is a potent and selective inhibitor of Janus kinase 1 (JAK1) and Janus kinase 1 (JAK2) that has demonstrated anti-inflammatory effects and a good safety profile in patients with rheumatoid arthritis but has not been evaluated in GVHD.
- Objectives:
- To determine the safety and tolerability of baricitinib in patients with cGVHD that is refractory to steroids
- To determine the efficacy of baricitinib in patients with cGVHD that is refractory to steroids
- Eligibility:
- Inclusion:
- Age greater than or equal to 18 years
- Moderate or severe cGVHD per NIH consensus criteria
- Karnofsky performance status greater than or equal to 50%
- cGVHD 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)
- Receiving stable or tapering doses of systemic therapy in the preceding 4 weeks if taking systemic therapy for cGVHD
- Exclusion:
- Neutrophils <1.0x10^9/L, platelets <50X10^9/L, creatinine greater than or equal to 1.5 times the upper limit of normal or estimated creatinine clearance <50mL/min/1.73m^2 (Cockroft-Gault formula), serum aspartate aminotransferase or alanine aminotransferase concentration >3x upper limit of normal (ULN) or total bilirubin greater than or equal to 1.5x ULN
- Progressive malignancy, uncontrolled infection or any major organ dysfunction as defined by the protocol
- Design:
- This is a Phase 1/2 trial to determine the safety and efficacy of baricitinib in patients with cGVHD that is refractory to steroids.
- Patients will initially be treated with baricitinib at 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, with the primary response assessment at 24 weeks of total treatment. If the response is a partial response (PR) or stable disease, the dose will be increased to 4mg daily for an additional 12 weeks, with the primary response assessment at 24 weeks of total treatment. If there is progression of disease at any time within the first 12 weeks, the dose can be increased to 4mg daily at that time, and patients will continue for a total of 24 weeks of treatment. Patients will have the option to continue baricitinib for an additional 6 months as tolerated if they have stable or responding disease.
- The co-primary endpoint of safety will be determined by rate, severity, and duration of adverse events based on Common Terminology Criteria for Adverse Events (CTCAE) v4 criteria. Assessment for DLTs will occur every 2 weeks during the first 4 weeks of each dose level. Safety monitoring will occur every 4 weeks thereafter.
- The co-primary endpoint of efficacy will be defined as rate of overall response at 24 weeks per National Institutes of Health (NIH) consensus criteria (CR or PR).
- Peripheral blood samples will be collected prior to treatment, at 2 weeks, at 12 weeks and every 12 weeks thereafter to evaluate cytokine and cellular profiles, STAT phosphorylation, candidate chronic GVHD biomarkers. Pharmacokinetic studies will also be performed at each dose level.
- In an initial futility analysis, if 0 of the first 7 patients enrolled in cohort 1 have responded at 12 weeks, then a 2nd cohort of patients will be accrued to start treatment at the higher dose (4mg daily). Otherwise, if 1 or more of the first 7 patients respond in cohort 1, then 21 evaluable patients will be treated in cohort 1. Similarly, if the second cohort is used, and if 0 of the 7 patients enrolled in this second cohort have responded at 12 weeks, then no further patients will be accrued. Otherwise, if 1 or more of the first 7 patients respond in cohort 2, then 21 evaluable patients will be treated in cohort 2.
- A total of 21 evaluable patients will be enrolled in either cohort 1 or 2 as appropriate, in order to have 80% power to detect a response rate consistent with 30% and ruling out 10%, with a one-sided significance level of 0.10 for the cohort. As an early stopping rule for safety, if 2/3 or greater patients at any given dose level experiences a dose limiting toxicity requiring dose reduction or discontinuation, that dose will not be subsequently used, and no further dose escalation will take place.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Baricitinib | Experimental | Drug: BaricitinibSingle 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (9 listed)
- Systemic immune suppression or systemic therapy for cGVHD started within preceding 4 weeks.
- Hypersensitivity to JAK inhibitors.
- Any serious medical condition within the previous 4 weeks which places the subject at an unacceptable risk if he or she were to participate in the study or confounds the ability to interpret data from the study, including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmias, acute kidney injury, or psychiatric illness/social situations that would limit compliance with study requirements.
- Uncontrolled infection, including active human immunodeficiency virus (HIV-1), Hepatitis B virus (HBV) and/or Hepatitis C virus (HCV) infection (positive HBV or HCV viral load in the setting of positive HBV core antibody or surface antibody or HCV antibody). History of HBV or HCV is allowed if there is no uncontrolled viral infection. Because the study agent may impact response to infections, patients with any active viral infection are excluded.
- Recurrent or progressive malignancy requiring anticancer treatment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BaricitinibAlso: OlumiantCycle=28 days: Baricitinib: 1mg-4mg by mouth (PO) every day (QD) | Drug | Baricitinib |
Primary outcome
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
DLT is defined as any grade ≥3 non-hematologic or grade ≥4 hematologic adverse event, or hemoglobin <6.5 g/dL, within 4 weeks of starting any dose level (1mg, 2mg, or 4 mg) except those that are clearly and incontrovertibly due to extraneous causes. Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). Grade 3 is severe. Grade 4 is life-threatening.
Phase 2: Number of Participants With Any Serious and/or Non-Serious Grades 3, 4, and/or 5 Adverse Events Due to Drug
Time frame: every 4 weeks through study completion (up to 48 weeks)
Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). Grade 3 is severe. Grade 4 is life-threatening. And Grade 5 is death related to adverse event.
Phase 2: Overall Response at 24 Weeks
Time frame: 24 weeks
Response was assessed by the National Institutes of Health (NIH) chronic graft-versus-host disease (cGVHD) response criteria. Complete Response (CR) is defined as resolution of all manifestations in each organ or site. Partial Response (PR) is defined as improvement at least 1 organ or site without progression in any other organ or site. Lack of Response including unchanged, mixed response, and disease progression. Mixed response is a CR or PR in at least 1 organ accompanied by progression in another organ. Unchanged is outcomes that do not meet the criteria for CR, PR, disease progression, or mixed response. Disease Progression is a change in the manifestations in each organ or site that does not meet the criteria for CR, PR, mixed response, unchanged or lack of response.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42200447Holtzman NG, Im A, Curtis L, Schulz E, Sharma R, Pirsl F, Berman A, Ostojic A, Magone de Quadros Costa MT, Cowen EW, Mays J, Comis LE, Joe G, Peer C, Figg W, Rose J, El Jurdi N, Pouzolles M, Hakim F, Taylor N, Pavletic SZ. Baricitinib for refractory chronic graft-versus-host disease: results of a phase 1/2 study. Blood Adv. 2026 Sep 8;10(17):5751-5761. doi: 10.1182/bloodadvances.2025018898.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven Z Pavletic, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Baricitinib in people with Chronic Graft vs Host Disease and Chronic Graft-Versus-Host Disease, run by National Cancer Institute (NCI) 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 0 inclusion and 9 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 8 Oct 2024. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
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). The study lists 3 primary outcomes in all. 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 NCT02759731, 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 8 Oct 2024.