NCT02918292: CHAMP

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

Completed Phase 2 Results posted Last updated 31 March 2026

What this trial tests

Phase 2 trial testing Antithymocyte Globulin (ATG) in Severe Aplastic Anemia in 32 participants. Completed in 17 August 2021.

Timeline

3 July 2017

Primary endpoint
17 August 2021

17 August 2021

Quick facts

Lead sponsor	Medical College of Wisconsin
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	32
Start date	3 July 2017
Primary completion	17 August 2021
Estimated completion	17 August 2021
Sites	28 locations across United States

Drugs / interventions tested

• Antithymocyte Globulin (ATG)
• Fludarabine (FLUDARABINE) — full drug profile →
• Cyclophosphamide (cyclophosphamide) — full drug profile →
• Total Body Irradiation (TBI)
• Haplo HSCT
• Tacrolimus (TACROLIMUS) — full drug profile →
• Mycophenolate mofetil (MMF) — full drug profile →
• G-CSF

Conditions studied

• Severe Aplastic Anemia — all drugs for Severe Aplastic Anemia →

Sponsor

Medical College of Wisconsin

Who can join

Under 75, any sex, with Severe Aplastic Anemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage of Participants With Overall Survival (OS) Primary · 1 year

Overall survival (OS) is the primary endpoint of this study. The time to this event is the time from transplant to death from any cause or last follow-up or 1 year from transplant, whichever occurs first. The one-year OS probability and its 95% confidence interval were estimated using the Kaplan-Meier estimator and Greenwood's formula.

Group	Value	95% CI
Haplo Bone Marrow HSCT	80.6	61.9 – 90.8

Percentage of Participants With Neutrophil Recovery Secondary · Day 28 and 56

Neutrophil recovery is achieving an absolute neutrophil count (ANC) \> 0.5 x10\^9/L for three consecutive measurements on different days, with the first of the three days being defined as the day of neutrophil engraftment. The cumulative percentage of neutrophil engraftment was estimated with a 95% confidence interval using the Aalen-Johansen estimator with death prior to neutrophil engraftment treated as a competing risk.

Day 28

Group	Value	95% CI
Haplo Bone Marrow HSCT	93.5	72.4 – 98.6

Day 56

Group	Value	95% CI
Haplo Bone Marrow HSCT	93.5	72.4 – 98.6

Percentage of Participants With Platelet Recovery Secondary · Day 100

Platelet recovery is defined by achieving a platelet count \> 20 x 10\^9/L with no platelet transfusions in the preceding seven days. The first day of the sustained platelet count will be defined as the day of platelet engraftment. The cumulative percentage of platelet engraftment was estimated with a 95% confidence interval using the Aalen-Johansen estimator with death prior to platelet engraftment treated as a competing risk.

Group	Value	95% CI
Haplo Bone Marrow HSCT	77.4	57.3 – 88.9

Participants Alive With Sustained Engraftment Secondary · 1 year

Being alive and engrafted is defined as not having experienced death, primary graft failure, or secondary graft failure. Donor cell engraftment is defined as donor chimerism greater than or equal to 5% on or after Day 56 after transplantation. Chimerism may be evaluated in whole blood or blood cell fractions, including CD3 and CD33 or CD15 fractions. For this protocol, lineage-specific, myeloid, and T cell chimerisms are required. This endpoint was adjudicated by the Endpoint Review Committee (ERC) and ERC data was used for the analysis.

Group	Value	95% CI
Haplo Bone Marrow HSCT	24
Haplo Bone Marrow HSCT	4
Haplo Bone Marrow HSCT	1
Haplo Bone Marrow HSCT	2

Percentage of Participants With Graft-Failure-Free Survival Secondary · 1 year

Events for Graft-Failure-Free Survival (GFFS) including death, primary graft failure, secondary graft failure. The time to this event is the time from transplant to death from any cause, or graft failure, or last follow-up, or 1 year from transplant, whichever occurs first. For patients experiencing primary graft failure, Day 0.1 was used for primary graft failure event date to count the event in. The one-year GFFS probability and its 95% confidence interval were estimated using the Kaplan-Meier estimator and Greenwood's formula.

Group	Value	95% CI
Haplo Bone Marrow HSCT	77.4	58.4 – 88.5

Percentage of Participants With Primary Graft Failure Secondary · Day 56

Primary graft failure is defined by the lack of neutrophil engraftment by Day 56 post-HSCT or failure to achieve at least 5% donor chimerism (whole blood or marrow) on any measurements up to and including Day +56. For this protocol, lineage-specific, myeloid, and T cell chimerisms are required. Myeloid engraftment might not proceed at the same rate as T cell engraftment. If myeloid has greater than or equal to 5% donor, even if T cell compartment does not, this is not considered primary graft failure. Secondary graft failure is defined by initial neutrophil engraftment (ANC greater than or equ

Group	Value	95% CI
Haplo Bone Marrow HSCT	12.9	3.6 – 29.8

Percentage of Participants With Secondary Graft Failure Secondary · 1 year

Secondary graft failure is defined as any one of the following: 1. Initial neutrophil engraftment (ANC greater than or equal to 0.5 x10\^9/L measured for three consecutive measurements on different days) followed by sustained subsequent decline in ANC to less than 0.5 x 10\^9/L for three consecutive measurements on different days; 2. Initial whole blood or marrow donor chimerism greater than or equal to 5%, but then declining to less than 5% on subsequent measurements; 3. Second infusion/transplant given after Day 56 for graft failure.

Group	Value	95% CI
Haplo Bone Marrow HSCT	3.2	0.1 – 16.7

Participants Alive With Autologous Recovery Secondary · 1 year

Autologous recovery is defined as ANC \> 0.5 x 10\^9/L and transfusion independence but with \< 5% donor chimerism (whole blood or m. arrow). This endpoint was reviewed and adjudicated by ERC. The analysis is based on ERC data.

Group	Value	95% CI
Haplo Bone Marrow HSCT	0
Haplo Bone Marrow HSCT	6
Haplo Bone Marrow HSCT	25

Percentage of Participants With Acute Graft-vs-host-disease (GVHD) Secondary · Day 100

Acute GVHD is graded by consensus grading (Przepiorka 1995) per BMTCTN manual of procedures (MOP). Acute GVHD is graded by consensus grading (Przepiorka 1995) per BMTCTN manual of procedures (MOP). The time of onset of grades II-IV and grades III-IV acute GVHD were recorded. This endpoint is evaluated through 100 days post-transplant. Cumulative percentage of acute GVHD post-transplant are estimated using the cumulative incidence function, treating death prior to acute GVHD as the competing risk.

Group	Value	95% CI
Haplo Bone Marrow HSCT	16.1	5.7 – 31.1

Participants With Maximum Acute GVHD Secondary · Day 100

Acute GVHD is graded by consensus grading (Przepiorka 1995) per BMTCTN manual of procedures (MOP). Acute GVHD grading is performed by the consensus conference criteria (Przepiorka et al. 1995) with higher grade indicating worse outcomes. Grade I acute GVHD is defined as Skin stage of 1-2 and stage 0 for both GI and liver organs. Grade II is stage 3 of skin, or stage 1 of GI, or stage 1 of liver. Grade III is stage 2-4 for GI, or stage 2-3 of liver. Grade IV is stage 4 of skin, or stage 4 of liver. Maximum grade of acute GVHD through 100 days post transplant is reported.

Group	Value	95% CI
Haplo Bone Marrow HSCT	23
Haplo Bone Marrow HSCT	3
Haplo Bone Marrow HSCT	5
Haplo Bone Marrow HSCT	0

Percentage of Participants With Chronic GVHD Secondary · 1 year

The event for this secondary endpoint is any chronic GVHD based on 2014 NIH Consensus Criteria. This includes mild, moderate and severe chronic GVHD. The analyses of Chronic GVHD use the site-reported data. The cumulative percentage of chronic GVHD is computed using the cumulative incidence function, treating death prior to chronic GVHD as a competing risk. Eight organs will be scored on a 0-3 scale to reflect degree of chronic GVHD involvement. Liver and pulmonary function test results and use of systemic therapy for treatment of chronic GVHD will also be recorded. This secondary endpoint of

Group	Value	95% CI
Haplo Bone Marrow HSCT	25.8	11.9 – 42.2

Number of Participants Experiencing Chronic GVHD With Maximum Severity Secondary · 1 year

The event for this secondary endpoint is any chronic GVHD based on 2014 NIH Consensus Criteria. Eight organs will be scored on a 0-3 scale to reflect degree of chronic GVHD involvement. Liver and pulmonary function test results and use of systemic therapy for treatment of chronic GVHD will also be recorded. The overall chronic GVHD severity is based on the eight organs score. The maximin severity level of chronic GVHD include mild, moderate and severe.

Group	Value	95% CI
Haplo Bone Marrow HSCT	23
Haplo Bone Marrow HSCT	7
Haplo Bone Marrow HSCT	1
Haplo Bone Marrow HSCT	0

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse event reporting and monitoring were conducted throughout the study, up to 1 year.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Enrolled Participants

Serious: 1/32 (3%)

Deaths: 7/32

Serious adverse events (1 terms)

Reaction	System	Enrolled Participants
INCARCERATED INGUINAL HERNIA	Gastrointestinal disorders	—

Most-reported serious reactions: INCARCERATED INGUINAL HERNIA.

Data from ClinicalTrials.gov NCT02918292 adverse events section.

Sponsor's own description

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).

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide.
   DeZern AE, Zahurak ML, Symons HJ, Cooke KR, et al · · 2020 · cited 116× · PMID 32343796 · DOI 10.1182/bloodadvances.2020001729
2. Haploidentical bone marrow transplantation in patients with relapsed or refractory severe aplastic anaemia in the USA (BMT CTN 1502): a multicentre, single-arm, phase 2 trial.
   DeZern AE, Eapen M, Wu J, Talano JA, et al · · 2022 · cited 58× · PMID 35907408 · DOI 10.1016/s2352-3026(22)00206-x
3. Haploidentical Donor Bone Marrow Transplantation for Severe Aplastic Anemia.
   DeZern AE, Brodsky RA. · · 2018 · cited 20× · PMID 30047416 · DOI 10.1016/j.hoc.2018.04.001
4. Post-transplantation cyclophosphamide to facilitate HLA-haploidentical hematopoietic cell transplantation: Mechanisms and results.
   Elmariah H, Fuchs EJ. · · 2019 · cited 8× · PMID 31202428 · DOI 10.1053/j.seminhematol.2018.09.002
5. Priorities for Improving Outcomes for Nonmalignant Blood Diseases: A Report from the Blood and Marrow Transplant Clinical Trials Network.
   Levine JE, Antin JH, Allen CE, Burroughs LM, et al · · 2020 · cited 4× · PMID 32035274 · DOI 10.1016/j.bbmt.2020.01.024
6. Future directions in transplantation for aplastic anemia.
   Sharma A. · · 2025 · PMID 41348014 · DOI 10.1182/hematology.2025000767