Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia

NCT00427336 | Completed Results

• 1 other identifier: IDP00-266
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

Primary Objectives:

1. 1.To determine the feasibility and toxicity of employing purine-analog based conditioning for allogeneic donor stem cell transplantation in patients with severe aplastic anemia (AA).
2. 2.To determine the engraftment kinetics and degree of chimerism that can be achieved with this strategy.

Conditions

Aplastic Anemia

Keywords

Severe Aplastic Anemia; Bone Marrow Failure; Stem Cell Transplantation; Fludarabine; Cyclophosphamide; Antithymocyte Globulin; SAA; ATG; Cytoxan; Neosar; Fludarabine Phosphate; Thymoglobulin

Outcome Measures

Primary Outcomes (1)

• Number of Patients With Engraftment Response

  Engraftment defined as (1) the first of three consecutive days of an Absolute neutrophil count (ANC) \>500/mL (b) the first of seven consecutive days of an unsupported platelet count 20,000. Patient needs to survive at least 28 days to be evaluable for engraftment. Chimerism studies need to demonstrate donor-derived hematopoiesis (\>90%)

  First 100 days post transplant.

Study Arms (1)

Fludarabine + Cyclophosphamide + ATG

EXPERIMENTAL

Fludarabine 30 mg/m\^2/day by vein (IV), Cyclophosphamide IV 300 mg/m\^2/day, ATG (Antithymocyte Globulin) IV 3.75 mg/kg/day

Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Antithymocyte Globulin

Interventions

Fludarabine DRUG

30 mg/m\^2 by vein daily over 30 minutes

Also known as: Fludarabine Phosphate, Fludara

Fludarabine + Cyclophosphamide + ATG

Cyclophosphamide DRUG

300 mg/m\^2 by vein daily over 2 hours

Also known as: Cytoxan, Neosar

Fludarabine + Cyclophosphamide + ATG

Antithymocyte Globulin DRUG

3.75 mg/kg by vein daily over 4 hours

Also known as: ATG, Thymoglobulin

Fludarabine + Cyclophosphamide + ATG

Eligibility Criteria

• Age: Up to 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients up to 70 years of age with a diagnosis of severe AA (Camitta et al., 1979) and a matched unrelated donor who are unresponsive to IS or who have relapsed after an initial response to IS. Patients with a diagnosis of SAA and an human leukocyte antigen (HLA) - compatible sibling donor are eligible only if they are 40 years of age or older (up to age 70) and regardless whether they have received IS or not. Patients with primary or secondary graft failure following autologous or allogeneic stem cell transplant are eligible.
• Patients must have a serum bilirubin of 2 mg/dl or less, serum creatinine \< 2.0 mg/dl, no symptomatic cardiac or pulmonary disease and a PS of no more than 2. Life expectancy not severely limited by concomitant illness (\> 12 weeks). Left ventricular ejection fraction \> 40%, no uncontrolled arrhythmia or symptomatic cardiac disease. Forced Expiratory Volume in 1 Second (FEV1), Forced Vital Capacity (FVC) and Carbon Monoxide Diffusing Capacity (DLCO) \> 40%. No symptomatic pulmonary disease. Negative pregnancy test.
• Patients must have an HLA-compatible related or unrelated donor willing to donate marrow or rhG-CSF-mobilized peripheral blood stem cells. In the event of transplants from matched unrelated donors, a high-resolution allele match for HLA-A, -B, -C, -DRB1 and DQB1 ("10 of 10 match") is preferred. However, a one-antigen mismatch ("micromismatch") is also considered acceptable matching ("9 of 10 match").
• Patients must sign informed consent. In the event of a pediatric patient (i.e., a minor), consent will be provided by their guardian/parent.
• Lack of clonal cytogenetic abnormalities associated with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or other hematologic malignancies.

You may not qualify if:

• Life expectancy of less than 8 weeks. Inability to provide informed consent.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

U.T.M.D. Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• The University of Texas M.D.Anderson Cancer Center

MeSH Terms

Conditions

Anemia, Aplastic; Bone Marrow Failure Disorders

Interventions

fludarabine; fludarabine phosphate; Cyclophosphamide; Antilymphocyte Serum; thymoglobulin

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures

Results Point of Contact

• Title: Paolo Anderlini, MD / Professor
• Organization: UT MD Anderson Cancer Center

mvpacheco@mdanderson.org

Study Officials

• Paolo Anderlini, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 25, 2007
• First Posted: January 29, 2007
• Study Start: December 1, 2000
• Primary Completion: August 1, 2009
• Study Completion: August 1, 2009
• Last Updated: October 27, 2011
• Results First Posted: October 27, 2011

Record last verified: 2011-09

Locations

US (1)