RECRUITING

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

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

Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%. With immunosuppression, only 1/3 of patients are cured, 1/3 are dependent on long term immunosuppression, and the other 1/3 relapse or develop a clonal disorder. Recent studies have shown that using a haploidentical donor for transplantation has good response rates and significantly lower rates of acute and chronic GVHD.

Official Title

A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor

Quick Facts

Study Start:2016-09-09
Study Completion:2026-08-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT02828592

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:1 Year to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
  2. * Age \<= 65 years for previously treated and \<= 75 years for previously treated patients
  3. * KPS \>= 70%
  4. * Aplastic Anemia that meets the following criteria:
  5. * \<500 PMN/mm3
  6. * \<20,000 platelets
  7. * absolute reticulocyte count \<40,000/microL
  8. * markedly hypocellular (\<25% of normal cellularity)
  9. * moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above
  1. * poor cardiac function (LVEF \<40%)
  2. * poor pulmonary function (FEV1 \& FVC \<50% predicted)
  3. * poor liver function (bili \>= 2mg/dL)
  4. * poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40mL/min)
  5. * prior allogeneic transplant

Contacts and Locations

Study Contact

Melhem Solh, MD
CONTACT
404-255-1930
msolh@bmtga.com
Stacey Brown
CONTACT
404-851-8238
stacey.brown@northside.com

Principal Investigator

Melhem Solh, MD
PRINCIPAL_INVESTIGATOR
Blood and Marrow Transplant Group of Georgia

Study Locations (Sites)

Blood and Marrow Transplant Group of Georgia
Atlanta, Georgia, 30342
United States
Northside Hospital
Atlanta, Georgia, 30342
United States

Collaborators and Investigators

Sponsor: Northside Hospital, Inc.

  • Melhem Solh, MD, PRINCIPAL_INVESTIGATOR, Blood and Marrow Transplant Group of Georgia

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 Date2016-09-09
Study Completion Date2026-08-31

Study Record Updates

Study Start Date2016-09-09
Study Completion Date2026-08-31

Terms related to this study

Keywords Provided by Researchers

  • SAA

Additional Relevant MeSH Terms

  • Severe Aplastic Anemia