Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia

NCT01129323 | Withdrawn DMC

• 1 other identifier: 07081
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to evaluate the effectiveness of allogeneic transplant after a reduced-intensity preparative regimen for patient, to evaluate survival, and to evaluate the side effects of this treatment. The patient will be in the study for two years for treatment and active monitoring. After treatment and active monitoring are over, the patient's medical condition will be followed indefinitely.

Conditions

Severe Aplastic Anemia

Keywords

Anemia, Severe, Aplastic, Reduced-Intensity, Allogeneic, Stem, Cell, Transplantation

Outcome Measures

Primary Outcomes (1)

• Engraftment rate

  Day 42 after allogeneic transplant

Secondary Outcomes (6)

• Relapse

  Days 180, 365 and yearly post transplant

• Development of Graft vs. Host Disease

  Days 180, 365 and yearly post transplant

• Evaluation of the occurrence of secondary malignancies

  Days 180, 365 and yearly post transplant

• Overall survival

  Days 180, 365 and yearly post transplant

• Evaluation of treatment feasibility

  Days 180, 365 and yearly post transplant

Study Arms (1)

Reduced-Intensity Preparative Regimen for Allogeneic SCT

EXPERIMENTAL

Patient in this arm will receive maximally tolerated (reduced) doses of cytotoxic therapy with the goals of suppressing the immune system, and ablate host hematopoiesis to ensure engraftment of the donor's hematopoietic system.

Drug: Cyclophosphamide, Fludarabine, Rabbit ATG

Interventions

Cyclophosphamide, Fludarabine, Rabbit ATG DRUG

Fludarabine 30mg/m2/dose IV and cyclophosphamide 10mg/kg/dose IV are given once per day from day -5 to -2. rATG 1.5 mg/kg/dose IV is given from day -4 to -1.

Reduced-Intensity Preparative Regimen for Allogeneic SCT

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• years old or younger
• Male or female recipients must have histopathologically confirmed diagnosis of severe aplastic anemia. Diagnostic Criteria for Server Aplastic Anemia will be based on the definitions set forth by the international Aplastic Anemia Study Group
• At least two of the following:
• Absolute neutrophil count \<0.5 x 109/L Platelet count \<20 x 109 /L Anemia with corrected reticulocyte count \<1%
• AND
• Bone marrow cellularity \<25%, or bone marrow cellularity \<50% with fewer than 30% hematopoietic cell
• Availability of an HLA identical sibling

You may not qualify if:

• Active and uncontrolled infection
• HIV-1 infection
• Pregnancy or breastfeeding.
• DLCO \<40% predicted
• Left Ventricular Ejection Fraction \< 40%
• Performance scale Karnofsky \<=40% or Lansky\<=40% for patients \<16 years old

Sponsors & Collaborators

• City of Hope Medical Center: lead

MeSH Terms

Conditions

Anemia, Aplastic; Anemia

Interventions

Cyclophosphamide; fludarabine; thymoglobulin

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• Anna Pawlowska, MD

  City of Hope Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: April 21, 2010
• First Posted: May 24, 2010
• Study Start: November 1, 2009
• Primary Completion: September 1, 2011
• Last Updated: March 27, 2012

Record last verified: 2012-03