Loading clinical trials...

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502) | Clinical Trials | Clareo Health

COMPLETEDPHASE2

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

Optimizing Haploidentical Aplastic Anemia Transplantation (CHAMP) (BMT CTN 1502)

NCT02918292•Medical College of Wisconsin

Summary

This study is a prospective, multicenter phase II study with patients receiving haploidentical transplantation for Severe Aplastic Anemia (SAA). The primary objective is to assess overall survival (OS) at 1 year post-hematopoietic stem cell transplantation (HSCT).

Detailed Description

Acquired SAA is a rare bone marrow failure disorder with an estimated annual incidence of 2 cases per million and with over 600 new cases in the United States each year. A major challenge in treating acquired SAA is the management of patients who are refractory to immunosuppressant therapy (IST) or have relapsed after IST. HSCT is the only curative option for these patients but many are ineligible because they lack a suitable donor. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) seeks to increase options for these patients by using novel therapeutic strategies of GVHD prophylaxis with PTCy to expand the donor pool to include haploidentical donors. The goal of this protocol is to test whether optimized approaches using haploidentical donors will achieve acceptable outcomes in SAA patients.

Eligibility

Age

0 - 75 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Patient is \< 75 years of age at time of enrollment.
•2. Confirmed diagnosis of SAA, either from initial diagnosis or follow-up assessments, defined as:
•1. Bone marrow cellularity \< 25% or marrow cellularity \< 50% but with \< 30% residual hematopoietic cells.
•2. Two out of three of the following (in peripheral blood): Neutrophils \< 0.5 x10\^9/L, Platelets \< 20 x10\^9/L, or Reticulocyte count \< 20 x10\^9/L (\<60 x 10\^9/L using an automated analysis)
•3. No suitable fully matched related sibling donor (6/6 match for human leukocyte antigen (HLA)-A and B at intermediate or high resolution and DRB1 at high resolution using DNA-based typing) available.
•4. Failed at least one trial of immunosuppressive therapy (IST) by being refractory or having relapsed. IST could have included ATG based regimens, calcineurin inhibitors and/or other higher dose therapy directed at the treatment of primary SAA.
•5. Available relative of the patient who is a haploidentical match, including biological parents, siblings or half siblings, children, uncles/aunts, first cousins, etc. Eligible haploidentical donors will have 2-4 mismatches if HLA-A, -B, -C, and -DRB1 typing is used; 2-5 mismatches if HLA-A, -B, -C, -DRB1, and -DQB1 typing is used; and 2-6 mismatches if HLA-A, -B, -C, -DRB1, -DQB1, and -DPB1 typing is used. A unidirectional mismatch in either the graft versus host or host versus graft direction is considered a mismatch. The donor and recipient must demonstrate that they are a full haplotype match by being identical at a minimum of one allele (at high resolution DNA-based typing) at the following genetic loci: HLA-A, -B, -C, and DRB1 if 8 allele typing is used; HLA-A, -B, -C, -DRB1, and -DQB1 if 10 allele typing is used; and HLA-A, -B, -C, -DRB1-, DQB1, and -DPB1 is 12 allele typing is used by the local center. See Section 2.4 for additional information.
•6. Patient and/or legal guardian must sign informed consent for HSCT.
•7. The haplo donor and/or legal guardian must be able to sign informed consent documents.
•8. The potential haplo donor must be willing and able to donate bone marrow.
+8 more criteria

Exclusion Criteria

•1. Inherited bone marrow failure syndromes such as Fanconi anemia must be ruled out according to center standard.
•2. Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (e.g. Monosomy 7).
•3. Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement-dependent cytotoxicity or flow cytometric testing or the presence of anti-donor HLA antibody to the high expression loci HLA-A, B, C, DRB1, or DPB1 with mean fluorescence intensity (MFI) \> 1000 by solid phase immunoassay).
•4. Prior allogeneic stem cell transplant.
•5. Prior solid organ transplant.
•6. Known life-threatening reaction (i.e., anaphylaxis) to Thymoglobulin® that would prohibit use for the patient as this study requires use of the Thymoglobulin® preparation of ATG.
•7. Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment.
•8. Seropositive for the human immunodeficiency virus (HIV).
•9. Active Hepatitis B or C determined by a detectable viral load of HBV or HCV.
•10. Female patients who are pregnant (per institutional practice) or breast-feeding.
+2 more criteria

Locations (28)

City of Hope National Medical Center

Duarte, California, United States

Children's Hospital of Los Angeles

Los Angeles, California, United States

Children's Hospital and Research Center Oakland

Oakland, California, United States

Stanford Hospital and Clinics

Stanford, California, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

University of Florida College of Medicine

Gainesville, Florida, United States

Miami Children's Hospital

Miami, Florida, United States

All Children's Hospital

St. Petersburg, Florida, United States

H. Lee Moffitt Cancer Center

Tampa, Florida, United States

Northside Hospital

Atlanta, Georgia, United States

Key Dates

Start Date

July 3, 2017

Primary Completion Date

August 17, 2021

Completion Date

August 17, 2021

Last Updated

November 30, 2022

Enrollment

ACTUAL participants

Conditions

Severe Aplastic Anemia

Interventions

Antithymocyte Globulin (ATG)

DRUG

Fludarabine

DRUG

Cyclophosphamide

DRUG

Total Body Irradiation (TBI)

RADIATION

Haplo HSCT

PROCEDURE

Tacrolimus

DRUG

Mycophenolate mofetil (MMF)

DRUG

G-CSF

DRUG

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

NCT05012111

Severe Aplastic AnemiaTelomere Biology Disorders+1 more

View study

RECRUITINGPHASE2

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

NCT03520647

Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)+1 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: November 30, 2022Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

Inclusion Criteria

Exclusion Criteria

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

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

Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia