RECRUITING

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

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Conditions

Severe Aplastic AnemiaAcquired Amegakaryocytic ThrombocytopeniaAcquired Pure Red Cell AplasiaParoxysmal Nocturnal HemoglobinuriaHCTRICSAAPTCyaATaPRCA

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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

A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and/or clonal hematopoiesis.

Official Title

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Quick Facts

Study Start:2024-06-05
Study Completion:2036-05-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06412497

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:0 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Idiopathic Severe Aplastic Anemia (SAA), characterized by one of the following:
  2. 1. Refractory cytopenia(s), with 1+ of the following:
  3. 1. Platelets \<20,000/uL or transfusion dependent
  4. 2. Absolute neutrophil count \<500/uL without hematopoietic growth factor support
  5. 3. Absolute reticulocyte count \<60,000/uL AND bone marrow cellularity \<50% (with \< 30% residual hematopoietic cells)
  6. 2. Early myelodysplastic features (bone marrow (BM) blasts \<5%), without history of MDS/AML pre-treatment.
  7. 3. Idiopathic SAA with post-HCT graft failure (blood/marrow donor chimerism \<5%) requiring a 2nd allogeneic HCT
  8. * Paroxysmal Nocturnal Hemoglobinuria (PNH), including AA-PNH overlap syndrome, acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT), characterized by one of the following:
  9. 1. Refractory cytopenia(s), with 1+ of the following:
  10. 1. Platelets \<20,000/uL or transfusion dependent
  11. 2. Absolute neutrophil count \<500/uL without hematopoietic growth factor support
  12. 3. Absolute reticulocyte count \<60,000/uL or red cell transfusion dependent AND Bone marrow evidence of 1 to 3-lineage aplasia OR peripheral blood PNH clone \>/= 10%
  13. 2. Early myelodysplastic features (bone marrow (BM) blasts \<5%) without history of MDS/AML pre-treatment.
  14. 3. Idiopathic PNH, aPRCA, or aAT with post-HCT graft failure (blood/marrow donor chimerism \<5%) requiring a 2nd allogeneic HCT
  15. * Adequate organ function within 30 days of conditioning regimen
  1. * Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days of the start of treatment
  2. * Uncontrolled infection
  3. * Evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
  4. * Known allergy to any of the study components
  5. * Prior radiation therapy deemed excessive by radiation therapist for proposed low dose TBI exposure on this protocol
  6. * Diagnosis of an inherited bone marrow failure disorder such as Fanconi anemia, Telomere biology disorder, or Schwachman-Diamond syndrome, unless reviewed by the principal investigator and deemed appropriate for this approach (e.g. GATA2 deficiency)
  7. * Advanced myelodysplastic syndrome (MDS; BM blasts \>5%) or acute myeloid leukemia
  8. * Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements
  9. * Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study

Contacts and Locations

Study Contact

Christen Ebens, MD, MPH
CONTACT
612-624-1791
ebens012@umn.edu

Study Locations (Sites)

University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
United States

Collaborators and Investigators

Sponsor: Masonic Cancer Center, University of Minnesota

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 Date2024-06-05
Study Completion Date2036-05-01

Study Record Updates

Study Start Date2024-06-05
Study Completion Date2036-05-01

Terms related to this study

Keywords Provided by Researchers

  • HCT
  • RIC
  • SAA
  • PTCy
  • aAT
  • aPRCA

Additional Relevant MeSH Terms

  • Severe Aplastic Anemia
  • Acquired Amegakaryocytic Thrombocytopenia
  • Acquired Pure Red Cell Aplasia
  • Paroxysmal Nocturnal Hemoglobinuria