RECRUITING

Reduced Intensity Haploidentical Transplantation for the Treatment of Primary or Secondary Myelofibrosis

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Conditions

Primary MyelofibrosisSecondary Myelofibrosis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This initial cohort of this phase II trial studied the outcomes of using a JAK inhibitor prior to reduced intensity haploidentical (Haplo) transplantation for the treatment of primary or secondary myelofibrosis (MF). The primary risk of using Haplo HCT in patients with MF is graft failure. In the first cohort, all patients engrafted. There were no instances of graft failure. However, a large number of patients did have graft versus host disease as a complication of their transplant. JAK inhibitors have since been approved for the indication of graft versus host disease treatment. And we are also using them for graft versus host disease prevention in a study of MF patients with sibling and unrelated donors. Therefore, we are opening a new cohort of the current study using the JAK inhibitor prior to, during and after Haplo transplant. Our goal is to decrease graft versus host disease in patients receiving a Haplo MF transplant without increasing the risk of graft failure.

Official Title

Pilot Study of JAK Inhibitor Therapy Followed by Reduced Intensity Haploidentical Transplantation for Patients With Myelofibrosis

Quick Facts

Study Start:2021-02-09
Study Completion:2029-08-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04370301

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * PART 1: JAK INHIBITOR ADMINISTRATION INCLUSION CRITERIA
  2. * Age \> 18 years
  3. * Diagnosis of primary myelofibrosis (PMF) as defined by the 2016 World Health Organization classification system or diagnosis of secondary MF as defined by the International Working Group (IWG) for Myeloproliferative Neoplasms Research and Treatment criteria
  4. * Patients meeting the criteria for intermediate-1, intermediate-2 or high-risk disease by the Dynamic International Prognostic Scoring System (DIPSS)-plus scoring system (DIPSS may be used if all data from DIPSS are not available)
  5. * Ability to understand and the willingness to sign a written informed consent document (or legally authorized representative)
  6. * Patient must be a potential hematopoietic stem cell transplant candidate
  7. * PART 2: ALLOGENEIC STEM CELL TRANSPLANT INCLUSION CRITERIA
  8. * Meeting criteria for 1st phase as above, at time of initiation of JAK inhibitor, including ability to understand and willingness to sign a written informed consent (or legally authorized representative). Patients arriving to our institution for transplant and not enrolled in Part 1 may still be enrolled in Part 2 if Part 1 criteria met. These patients will have Part 1 endpoints transcribed from medical records
  9. * Received JAK inhibitor for at least 8 weeks immediately prior to conditioning and be willing to continue until 9-12 months post-transplant as tolerated
  10. * Karnofsky performance status score \>= 70
  11. * Calculated creatinine clearance using the Cockcroft-Gault formula or 24 hour (hr) urine creatinine clearance must be \> 60 ml/min
  12. * Total serum bilirubin must be \< 3 mg/dL unless the elevation is thought to be due to Gilbert's disease or hemolysis
  13. * Transaminases must be \< 3 x the upper limit of normal
  14. * Patients with clinical or laboratory evidence of liver disease will be evaluated for the cause of liver disease, its clinical severity in terms of liver function, and the degree of portal hypertension. Patients with fulminant liver failure, cirrhosis with evidence of portal hypertension or bridging fibrosis, alcoholic hepatitis, hepatic encephalopathy, or correctable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin \> 3 mg/dL, and symptomatic biliary disease will be excluded
  15. * Diffusion capacity of the lung for carbon monoxide (DLCO) corrected \> 60% normal; may not be on supplemental oxygen
  16. * Left ventricular ejection fraction \> 40% OR shortening fraction \> 26%
  17. * Comorbidity Index \< 5 at the time of pre-transplant evaluation
  18. * DONOR: Patients must be screened prior to transplant for donor-specific anti-HLA antibodies (DSA). Patients with DSA will be reviewed by the principal investigator and considered for desensitization treatment
  19. * DONOR: Children are preferred over siblings and parents
  20. * DONOR: Younger donors are preferred over older donors
  21. * DONOR: ABO matched donors are preferred over minor ABO mismatched and over major ABO mismatch donors
  1. * PART 1: JAK INHIBITOR ADMINISTRATION EXCLUSION CRITERIA
  2. * Contraindication to receiving a JAK inhibitor including:
  3. * Patients who have known hypersensitivity to JAK inhibitors
  4. * Clinical or laboratory evidence of significant renal or hepatic impairment including cirrhosis
  5. * Active uncontrolled infection
  6. * Known human immunodeficiency virus (HIV) positivity
  7. * Women who are pregnant or trying to conceive
  8. * Caution should be used in patients with platelets \< 100 though adjustments in dose can be made to accommodate anyone with platelets \> 50
  9. * History of prior allogeneic transplant
  10. * Leukemic transformation (\> 20% blasts)
  11. * PART 2: ALLOGENEIC STEM CELL TRANSPLANT EXCLUSION CRITERIA
  12. * Uncontrolled viral or bacterial infection at the time of study enrollment
  13. * Active or recent (prior 6 month) invasive fungal infection without infectious disease (ID) consult and approval
  14. * Known HIV positivity
  15. * Pregnant or breastfeeding
  16. * Availability of an human leukocyte antigen (HLA)-identical or 1-allele-mismatched related donor or an HLA 10 of 10 matched unrelated donor

Contacts and Locations

Study Contact

Rachel B. Salit
CONTACT
206-667-1317
rsalit@fredhutch.org

Principal Investigator

Rachel B. Salit
PRINCIPAL_INVESTIGATOR
Fred Hutch/University of Washington Cancer Consortium

Study Locations (Sites)

Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109
United States

Collaborators and Investigators

Sponsor: Fred Hutchinson Cancer Center

  • Rachel B. Salit, PRINCIPAL_INVESTIGATOR, Fred Hutch/University of Washington Cancer Consortium

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-02-09
Study Completion Date2029-08-31

Study Record Updates

Study Start Date2021-02-09
Study Completion Date2029-08-31

Terms related to this study

Additional Relevant MeSH Terms

  • Primary Myelofibrosis
  • Secondary Myelofibrosis