NCT02918292

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Medical College of Wisconsin · Academic / Other
• Sex: All
• Age: 75 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Antithymocyte Globulin (ATG)	Administration of ATG will be 0.5 mg/kg IV on Day -9 over 6 hours and 2 mg/kg IV on Days -8 and -7 over 4 hours.
DRUG	Fludarabine	Fludarabine dose will be 30 mg/m\^2 IV daily for 5 days from Day -6 to Day -2.
DRUG	Cyclophosphamide	Cyclophosphamide dose will be 14.5 mg/kg IV daily for 2 days (Day -6 to Day -5) prior to transplantation and 50 mg/kg IV daily for 2 days (Day +3 to Day +4) after transplantation.
RADIATION	Total Body Irradiation (TBI)	TBI is to be delivered in a single dose of 200 cGy on Day -1.
PROCEDURE	Haplo HSCT	Eligible patients without a fully matched related or unrelated donor available will undergo haploidentical bone marrow transplant.
DRUG	Tacrolimus	Tacrolimus should be started on Day +5 and administered to maintain a level of 10-15 ng/mL.
DRUG	Mycophenolate mofetil (MMF)	MMF dose will be 15 mg/kg PO three times a day (TID) up to 1 gm TID (or IV equivalent) starting on Day +5.
DRUG	G-CSF	G-CSF will be given IV or SQ starting on Day +5 at 5 mcg/kg/day until ANC is \> 1500 for 3 days.

Timeline

• Start date: 2017-07-03
• Primary completion: 2021-08-17
• Completion: 2021-08-17
• First posted: 2016-09-28
• Last updated: 2026-03-31
• Results posted: 2022-07-12

Locations

28 sites across 1 country: United States

Regulatory

• FDA-regulated drug study