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

Complete title: Allogeneic Hematopoietic Cell Transplantation for Patients with Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen

Research Study Number       2256.00
Principal Investigator       Lauri Burroughs, MD
Phase       II

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Research Study Description

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.

Eligibility Criteria (must meet the following to participate in this study)

Ages Eligible for Study: up to 54 Years

Genders Eligible for Study: Both

- Patients with a nonmalignant disease treatable by allogeneic HCT

- Patients with a known nonmalignant disease that is not clearly defined will need to be discussed with the protocol principal investigator (PI) (Dr. Lauri Burroughs) and potentially the nonmalignant board to determine if they are eligible for HCT on this study

- DONOR: Human leukocyte antigens (HLA)-identical related donors or unrelated donors matched for HLA-A, B, C, DRB1, and DQB1 or mismatched for a single allele at HLA-A, B, C, DRB1 or a single DQB1 antigen or allele mismatch by high resolution deoxyribonucleic acid (DNA) typing

- DONOR: Bone marrow is the preferred cell source; PBSC are allowed if donor refuses or is unable to give marrow or as clinically indicated or following discussion with the PI

- DONOR: Umbilical Cord Blood: Unit selection is based on the cryopreserved total nucleated cell (TNC) dose and matching at HLA-A, B antigen level and DRB1 allele level typing; while HLA-C antigen/allele level typing is not considered in the matching criteria, if available, may be used to optimize unit selection

- DONOR: Umbilical Cord Blood: The patient and the cord blood unit(s) must be matched for at least 4 of 6 loci as defined above

- DONOR: Umbilical Cord Blood: Selection of two umbilical cord blood (UCB) units is allowed to provide sufficient cell dose

Other eligibility criteria may apply.

Exclusions (conditions that would prevent participation in this study)

- Patients with idiopathic aplastic anemia and Fanconi anemia; (patients with aplastic anemia associated with paroxysmal nocturnal hemoglobinuria [PNH] or inherited marrow failure syndromes, except Fanconi anemia, will be allowed)

- Patients with impaired cardiac function as evidenced by ejection fraction < 35% (or, if unable to obtain ejection fraction, shortening fraction of < 26%) or cardiac insufficiency requiring treatment or symptomatic coronary artery disease; patients with a shortening fraction < 26% may be enrolled if approved by a cardiologist

- Patients with impaired pulmonary function as evidenced by diffusion capacity of the lung for carbon monoxide (DLCO) < 50% of predicted (or, if unable to perform pulmonary function tests, then oxygen [O2] saturation < 92% on room air)

- Patients with impaired renal function as evidenced by creatinine-clearance < 50% for age, weight, height or serum creatinine > 2 x upper normal limit or dialysis-dependent

- Patients with evidence of synthetic dysfunction or severe cirrhosis requiring deferral of conditioning as recommended by a gastroenterology specialist

- Patients with an active infectious disease requiring deferral of conditioning; as recommended by an infectious disease specialist

- Patients who are positive for human immunodeficiency virus (HIV)

- Females who are pregnant or breast-feeding

- Patients with a known hypersensitivity to treosulfan and/or fludarabine

- Receiving another experimental drug within 4 weeks of initiation of conditioning (day -6) unless approved by the PI

- DONOR: Deemed unable to undergo marrow harvesting or PBSC mobilization and leukapheresis

- DONOR: HIV-positive

- DONOR: With active infectious hepatitis

- DONOR: Females with a positive pregnancy test

- DONOR: Umbilical Cord Blood: Any cord blood units with < 1.5 x 10^7 total nucleated cells per kilogram recipient weight

- DONOR: Umbilical Cord Blood: Any cord blood units without full maternal testing and negative results for hepatitis A, B, C, HIV, and human T-cell lymphotropic virus 1 (HTLV-1) viruses; any additional available virology results on the unit itself will be reviewed but are not mandated, complete or always available; cord blood units are presumed to be cytomegalovirus (CMV) negative regardless of serologic testing due to passive transmission of maternal CMV antibodies

Other exclusion criteria may apply.

Research Study Number       2256.00
Contact       Seattle Cancer Care Alliance Intake Office
Telephone       800-804-8824 / 206-288-1024

Bone Marrow Transplant / Hematopoietic Stem Cell Transplant (BMT/HSCT); Diamond Blackfan Anemia; Immunodeficiency Syndromes; Sickle Cell Anemia; Umbilical Cord Blood Transplant (UCBT); Non-malignant Condition

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Please remember:

  • Talk to your health care providers first before making decisions about your health care.
  • Whether you are eligible for a research study depends on many things. There are specific requirements to be in research studies. These requirements are different for each study.

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