Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

Summary

This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases. Hematopoietic cell transplantation has been shown to be curative for many patients with nonmalignant (noncancerous) diseases such as primary immunodeficiency disorders, bone marrow failure syndromes, hemoglobinopathies, and inborn errors of metabolism (metabolic disorders). Powerful chemotherapy drugs and/or radiation are often used to condition the patient before infusion of the new healthy donor cells. The purpose of the conditioning therapy is to destroy the patient's abnormal bone marrow which doesn't work properly in order to make way for the new healthy donor cells which functions normally. Although effective in curing the patient's disease, many hematopoietic cell transplantation regimens use intensive chemotherapy and/or radiation which can be quite toxic, have significant side effects, and can potentially be life-threatening. Investigators are investigating whether a new conditioning regimen that uses less intensive drugs (treosulfan and fludarabine phosphate) with or without low dose radiation results in new blood-forming cells (engraftment) of the new donor cells without increased toxicities in patients with nonmalignant (noncancerous) diseases.

Conditions

• Non-Neoplastic Hematologic and Lymphocytic Disorder

Interventions

PROCEDURE

Allogeneic Bone Marrow Transplantation

Infused IV

BIOLOGICAL

Anti-Thymocyte Globulin

Given IV

DRUG

Cyclosporine

Given IV or PO

DRUG

Fludarabine Phosphate

Given IV

OTHER

Laboratory Biomarker Analysis

Correlative studies

DRUG

Methotrexate

Given IV

DRUG

Mycophenolate Mofetil

Given IV or PO

PROCEDURE

Peripheral Blood Stem Cell Transplantation

Infused IV

DRUG

Tacrolimus

Given IV or PO

RADIATION

Total-Body Irradiation

Undergo total body irradiation

DRUG

Treosulfan

Given IV

PROCEDURE

Umbilical Cord Blood Transplantation

Single or double unit umbilical cord blood transplant, infused IV

Sponsors & Collaborators

• medac GmbH

  collaborator INDUSTRY

• National Cancer Institute (NCI)

  collaborator NIH

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

  collaborator NIH

• Fred Hutchinson Cancer Center

  lead OTHER

Principal Investigators

• Lauri M. Burroughs · Fred Hutch/University of Washington Cancer Consortium

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Max Age

49 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2009-07-31

Primary Completion

2020-06-10

Completion

2020-06-10

FDA Drug

Yes

Countries

• United States

Study Locations