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JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure

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Conditions

Severe Aplastic AnemiaSingle Lineage Cytopenias, T-LGLHypoplastic MDSHematologic toxicityRuxolitinib

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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

Background: Immune bone marrow failure is a condition that occurs when a person s immune system attacks the cells of the bone marrow. This can lead to diseases including different types of anemias and blood cancers. Some of these diseases can be deadly. Better treatments are needed. Objective: To test a drug (ruxolitinib) in people with different types of immune bone marrow failure. Eligibility: Adults aged 18 and older with an immune bone marrow failure. Design: Participants will be screened. They will have a physical exam. They will give samples of blood and saliva. They will have a bone marrow biopsy: A large needle will be inserted into a small cut to remove a sample of the soft tissue inside the bone. Some participants may have a skin biopsy: A small piece of skin will be removed. Some may have a computed tomography (CT) scan: They will lie on a table that slides into a donut-shaped machine that uses X-rays to make pictures of the inside of the body. Ruxolitinib is a tablet taken by mouth. Participants will take the drug twice a day for up to 6 months. Participants will have blood tests every week while they are taking the drug. These tests can be done by the participant s own physician and the results sent to the researchers. Participants will have clinic visits after taking the drug for 3 months and 6 months and then after 1, 2, and 3 years. The blood tests and bone marrow biopsy will be repeated. Participants who improve while taking the drugs may go on to an extension phase of the study.

Official Title

A Phase I/II Study of the JAK1/2 Inhibitor Ruxolitinib for Relapsed / Refractory Immune Bone Marrow Failure

Quick Facts

Study Start:2024-02-20
Study Completion:2032-06-03
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05998408

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 to 99 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Ability of the participant or legally authorized representative (LAR) to understand and be willing to sign a written informed consent document
  2. * Age 18 or older
  3. * For females of childbearing potential, stated willingness to use an accepted method of contraception for the duration of the study. Accepted methods of contraception are:
  4. * Total abstinence
  5. * Use of an implanted or intrauterine hormonal device for at least 30 consecutive days before study drug administration
  6. * Use of oral, patch or injectable contraceptives or a vaginal hormonal device for at least 30 consecutive days before study drug infusion
  7. * Use of a non-hormonal intrauterine device for at least 30 consecutive days before study drug administration
  8. * Two barrier methods such as a diaphragm with spermicide or a condom with spermicide
  9. * For sexually active males with a female partner of childbearing potential, stated willingness to agree to use a condom with spermicide for the duration of the study.
  10. * Diagnosis of immune bone marrow failure (see specific cohort)
  11. * Absolute neutrophil count \< 0.5 x 10\^9/L
  12. * Platelet count \< 20 x 10\^9/L
  13. * Absolute Reticulocyte count \< 60 x 10\^9/L
  14. * Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy.
  15. * Not suitable for transplant due to age, co-morbidities, lack of suitable donor, or participant choice.
  16. * Patients who have a documented historic diagnosis of SAA and have received an ATG-based therapy in the past and are now relapsed / refractory may be included in this cohort even if documentation of original CBC and bone marrow are unavailable.
  17. * Aplastic anemia (hypocellular bone marrow for age) with no evidence for other disease processes causing marrow failure, and depression of at least two out of three blood counts below the normal values but not fulfilling the criteria for SAA:
  18. * Absolute neutrophil count \<= l.2 x 10\^9/L
  19. * Platelet count \<= 70 x 10\^9/L
  20. * Anemia with hemoglobin \<= 9 g/dL and absolute reticulocyte count \< 60 x 10\^9/L or transfusion dependence
  21. * Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy.
  22. * No evidence of viral or drug suppression of the marrow, T-LGL, dysplasia, or underproduction anemias secondary to B12, folate, iron or other reversible causes.
  23. * Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy.
  24. * Clinical history supportive of the diagnosis of T-LGL leukemia (i.e., a history of cytopenias with peripheral blood morphologic evidence of LGLs).
  25. * Immunophenotypic studies of peripheral blood showing an increased population of TLGLs (suggested by staining with CD3+, CD8+ and CD16+ or CD57+) or gamma-delta T cells.
  26. * Restricted or clonal rearrangement of the T-cell receptor by PCR
  27. * Neutropenia: Absolute neutrophil count \< 0.5 x 10\^9/L
  28. * Thrombocytopenia: Platelet count \< 30 x 10\^9/L or platelet transfusion dependence
  29. * Anemia: Hemoglobin \< 9g/dL or red cell transfusion-dependence or absolute reticulocyte count \<60 x 10\^9/L
  30. * Relapsed or refractory disease as evidenced by a course of at least 1 prior therapy
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Ivana Darden, R.N.
CONTACT
(301) 827-2988
ivana.darden@nih.gov
Emma M Groarke, M.D.
CONTACT
(301) 496-5093
emma.groarke@nih.gov

Principal Investigator

Emma M Groarke, M.D.
PRINCIPAL_INVESTIGATOR
National Heart, Lung, and Blood Institute (NHLBI)

Study Locations (Sites)

National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
United States

Collaborators and Investigators

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

  • Emma M Groarke, M.D., PRINCIPAL_INVESTIGATOR, National Heart, Lung, and Blood Institute (NHLBI)

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 Date2024-02-20
Study Completion Date2032-06-03

Study Record Updates

Study Start Date2024-02-20
Study Completion Date2032-06-03

Terms related to this study

Keywords Provided by Researchers

  • Hematologic toxicity
  • Ruxolitinib

Additional Relevant MeSH Terms

  • Severe Aplastic Anemia
  • Single Lineage Cytopenias, T-LGL
  • Hypoplastic MDS