Itacitinib for the Treatment of Bronchiolitis Obliterans Syndrome After Donor Hematopoietic Cell Transplant

NCT ID: NCT04239989

Last Updated: 2025-11-10

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-08
• Study Completion Date: 2025-10-31

Brief Summary

This phase I trial studies how well itacitinib works for the treatment of bronchiolitis obliterans syndrome after donor hematopoietic cell transplant. Itacitinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed Description

PRIMARY OBJECTIVE:

I. To assess the safety of itacitinib in patients with bronchiolitis obliterans syndrome (BOS) after allogeneic hematopoietic cell transplantation (HCT).

SECONDARY OBJECTIVES:

I. To assess treatment failure at 3 months and 6 months.

II. To assess change in symptom-based lung score at 3 months and 6 months.

III. To assess change in the St. George Respiratory Questionnaire and Study Short Form 36 at 3 months and 6 months.

IV. To assess change in the Lee chronic graft versus host disease (GVHD) symptom scale at 3 months and 6 months post-treatment.

V. To assess change in 6-minute walk test at 3 months and 6 months.

VI. To assess failure-free survival at 6 months.

VII. To assess non-relapse mortality at 6 months.

VIII. To assess overall survival at 6 months.

OUTLINE:

Patents receive itacitinib orally (PO) once daily (QD) for up to 1 year in the absence of disease progression or unacceptable toxicity.

Conditions

Bronchiolitis Obliterans

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment (itacitinib)

Patents receive itacitinib PO QD for up to 1 year in the absence of disease progression or unacceptable toxicity.

Group Type: EXPERIMENTAL

Itacitinib

Intervention Type: DRUG

Given PO

Itacitinib Adipate

Intervention Type: DRUG

Given PO

Interventions

Itacitinib

Given PO

Intervention Type: DRUG

Itacitinib Adipate

Given PO

Intervention Type: DRUG

Other Intervention Names

INCB 039110; INCB-039110; INCB039110; INCB-039110 Adipate; INCB039110 Adipate

Eligibility Criteria

Inclusion Criteria

1. BOS diagnosed within the past 6 months of enrollment, defined by 2015 National Institutes of Health (NIH) Consensus Criteria 126

2. Age 18-75 years

3. Undergone allogeneic SCT

4. ANC >1,000/µL, hemoglobin > 8 gm/dL (untransfused) and platelet count >25,000/ µL (untransfused)

5. Karnofsky performance score >60

6. The ability to understand and sign a written informed consent form

7. Contraception for women and men of child bearing potential. Permitted methods should be at least 99% effective in preventing pregnancy.

8. Male patients must be willing to refrain from donating sperm during their participation in the study and for at least 3 months after completing the study.

Exclusion Criteria

1. Prior treatment with any other JAK inhibitor (including Ruxolitinib) for BOS or any other indication within the past 6 months of enrolment.

2. Patients on mechanical ventilation or resting by pulse oximetry O2 saturation <88%

3. FEV1 <40% predicted

4. Relapsed primary malignancy for which SCT was performed

5. History of progressive multifocal leuko-encephalopathy (PML)

6. Active uncontrolled bacterial, fungal, parasitic, or viral infection

7. Known human immunodeficiency virus (HIV) infection or active hepatitis B or C infections.

8. History of tuberculosis anytime after SCT

9. Severe renal dysfunction defined by serum creatinine > 2 mg/dL, creatinine clearance <60 mL/minute or dialysis dependence

10. Serum transaminases > 5 × upper limit of normal

11. inability to perform PFT reliably

12. Positive Beta HCG test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.

13. Lactating/nursing women

14. Life expectancy < 6 months

15. Other severe organ dysfunction unrelated to underlying GVHD. For example, uncontrolled or significant cardiac disease, including any of the following: recent myocardial infarction (within last 6 months from randomization); New York Heart Association Class III or IV congestive heart failure; unstable angina (within last 6 months prior to randomization); clinically significant (symptomatic) cardiac arrhythmias (e.g., sustained ventricular; tachycardia, and clinically significant second or third degree AV block without a pacemaker); uncontrolled hypertension. Or any other concurrent severe and/or uncontrolled medical conditions which, in the opinion of the investigator, could compromise participation in the study, pose a significant risk to the subject, or interfere with study results.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

M.D. Anderson Cancer Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Amin Alousi, MD

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

M D Anderson Cancer Center

Houston, Texas, United States

Countries

United States

Provided Documents

Document Type: Informed Consent Form

View Document

Related Links

http://www.mdanderson.org

MD Anderson Cancer Center

Other Identifiers

NCI-2019-08252

Identifier Type: REGISTRY

Identifier Source: secondary_id

2018-0489

Identifier Type: OTHER

Identifier Source: secondary_id

2018-0489

Identifier Type: -

Identifier Source: org_study_id