Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

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).

Conditions

• Severe Aplastic Anemia

Interventions

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.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• National Cancer Institute (NCI)

  collaborator NIH

• Blood and Marrow Transplant Clinical Trials Network

  collaborator NETWORK

• National Marrow Donor Program

  collaborator OTHER

• Medical College of Wisconsin

  lead OTHER

Principal Investigators

• Mary Horowitz, MD, MS · Center for International Blood and Marrow Transplant Research

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Max Age

75 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2017-07-03

Primary Completion

2021-08-17

Completion

2021-08-17

FDA Drug

Yes

Countries

• United States

Study Locations