RECRUITING

Fludarabine Based RIC for Bone Marrow Failure Syndromes

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Conditions

Bone Marrow Failure SyndromesAcquired aplastic anemiaInherited bone marrow failure

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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 is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).

Official Title

Fludarabine-Based Conditioning for Matched Related Donor Bone Marrow Transplantation in Patients With Bone Marrow Failure Syndromes

Quick Facts

Study Start:2015-04
Study Completion:2026-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT02928991

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:Not specified to 22 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Ages 0-22 years at time of enrollment
  2. 2. Diseases:
  3. * Patients with severe or very severe acquired AA, defined by:
  4. * Bone marrow biopsy demonstrating cellularity of \<25% (at least 2 weeks from last dose of G-CSF), in addition to 2 of the following: absolute neutrophil count (ANC) \<500/µL, platelets \< 20,000/µL and absolute reticulocytes \<40,000/µL
  5. * Negative evaluation for inherited bone marrow failure conditions and negative evaluation for dysplasia or cytogenetic abnormalities associated with myelodysplastic syndromes
  6. * Patients with concurrent paroxysmal nocturnal hemoglobinuria (PNH) clones are eligible, as long as they meet criteria for severe or very severe aplastic anemia as defined above
  7. * Patients with clinically diagnosed and/or genetically proven iBMF syndromes, resulting in chronic red blood cell or platelet-transfusion dependence and/or an absolute neutrophil count \<500/µL. These disorders include, but are not limited to:
  8. * Fanconi Anemia
  9. * Dyskeratosis Congenita
  10. * Severe Congenital Neutropenia
  11. * Diamond-Blackfan Anemia
  12. * Congenital Dyserythropoietic/Sideroblastic Anemias
  13. * Congenital Amegakaryocytic Thrombocytopenia
  14. * Shwachman-Diamond Syndrome
  15. 3. Lansky or Karnofsky performance \>60
  16. 4. HLA matched related donor available.
  17. 5. No active untreated infection
  18. 6. Females of childbearing potential must have negative pregnancy test.
  19. * Serum creatinine \<1.5xupper limit of normal for age Hepatic: Transaminases \<5x normal
  20. * Cardiac shortening fraction \>27%
  21. * Bilirubin \<2.5x normal (unless elevation due to Gilberts disease).
  22. * Donor selection will comply with U.S. Food and Drug Administration's Code of Federal Regulations
  23. * Fully HLA-matched related donor.
  24. * Donor must be at least 6 months of age
  25. * Donor suitable for bone marrow collection and meets eligibility for donation, including fulfilling infectious disease criteria as per SOP, including HIV, Hepatitis B, Hepatitis C Polymerase chain reaction (PCR) negative.
  26. * If subject has confirmed iBMF syndrome, donor must be evaluated for this disorder and testing must be negative
  27. * Children's Hospital of Philadelphia (CHOP) bone marrow transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases.
  28. * Donor evaluation and collection procedure as per CHOP Standard Operating Procedures (SOP)
  1. * Uncontrolled bacterial, viral or fungal infections
  2. * HLA matched related donor unable to donate bone marrow.
  3. * No eligible fully HLA-matched related donor
  4. * Pregnant females
  5. * Patients with a clinical diagnosis of Myelodysplastic syndrome (MDS) defined by combination of bone marrow dysplasia and classic cytogenetic lesion (Monosomy 7, Trisomy 8 eg.), with or without excess blasts.
  6. * Patients with PNH without underlying bone marrow aplasia

Contacts and Locations

Study Contact

Megan Atkinson
CONTACT
215-590-2820
cttsbmtintake@chop.edu
Patricia Hankins, BSN, RN, CCRC
CONTACT
215-590-5168
hankinsp@chop.edu

Principal Investigator

Timothy Olson, MD, PhD
PRINCIPAL_INVESTIGATOR
Children's Hospital of Philadelphia

Study Locations (Sites)

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States

Collaborators and Investigators

Sponsor: Children's Hospital of Philadelphia

  • Timothy Olson, MD, PhD, PRINCIPAL_INVESTIGATOR, Children's Hospital of Philadelphia

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 Date2015-04
Study Completion Date2026-12

Study Record Updates

Study Start Date2015-04
Study Completion Date2026-12

Terms related to this study

Keywords Provided by Researchers

  • Acquired aplastic anemia
  • Inherited bone marrow failure

Additional Relevant MeSH Terms

  • Bone Marrow Failure Syndromes