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

• Register: ClinicalTrials.gov
• Last refreshed on: 22 April 2024
• Main ID: NCT02828592
• Date of registration: 06/07/2016
• Prospective Registration: Yes
• Primary sponsor: Northside Hospital, Inc.
• Public title: Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
• Scientific 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
• Date of first enrolment: September 9, 2016
• Target sample size: 20
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02828592
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Melhem Solh, MD
• Affiliation: Blood and Marrow Transplant Group of Georgia

• Name: Melhem Solh, MD

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

- Age <= 65 years for previously treated and <= 75 years for previously treated patients

- KPS >= 70%

- Aplastic Anemia that meets the following criteria:

Peripheral Blood (must fulfill 2 of 3):

- <500 PMN/mm3

- <20,000 platelets

- absolute reticulocyte count <40,000/microL

Bone Marrow (must be either):

- markedly hypocellular (<25% of normal cellularity)

- moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral
blood criteria above

Exclusion Criteria:

- poor cardiac function (LVEF <40%)

- poor pulmonary function (FEV1 & FVC <50% predicted)

- poor liver function (bili >= 2mg/dL)

- poor renal function (creatinine >= 2.0mg/dL or creatinine clearance <40mL/min)

- prior allogeneic transplant



Age minimum: 1 Year
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Severe Aplastic Anemia

Intervention(s)

Drug: Cyclophosphamide

Radiation: Total Body Irradiation

Drug: Fludarabine

Drug: Rabbit ATG

Primary Outcome(s)

Demonstrate sustained engraftment after T-cell replete HLA-mismatched haploidentical bone marrow transplantation by collecting chimerism tests monthly following transplant [Time Frame: 2 years]

Secondary Outcome(s)

Determine incidence of chronic GVHD at 6 months and 1 year post transplantation by assessing signs and symptoms of GVHD throughout post-transplant course [Time Frame: 2 years]

Determine the incidence of grade 2-4 and 3-4 acute graft versus host disease at 100 days post transplantation by assessing signs and symptoms of GVHD throughout post-transplant course [Time Frame: 2 years]

Determine the incidence of regimen-related mortality at 100 days post transplantation by recording treatment-related adverse events [Time Frame: 2 years]

Estimate overall survival at 100 days and 1 year post transplantation by collecting survival information at those time points [Time Frame: 2 years]

Secondary ID(s)

NSH 1158

Source(s) of Monetary Support

Please refer to primary and secondary sponsors