NCT00186797

Brief Summary

This study is for patients with Severe Aplastic Anemia (SAA). A stem cell transplant from a genetically matched sibling donor can help or cure this disease in 85 to 100 percent of patients. Stem cells are immature blood cells that grow to become red blood cells, white blood cells or platelets. A genetic "match" means a brother or sister has same immune type (HLA type) as the patient. Unfortunately, few patients have a matched sibling donor. The chance of negative outcomes is much higher with other types of donors. This study will test the success of a new approach to stem cell transplant for SAA. Patients in this study will receive drugs and radiation treatment to destroy their diseased bone marrow and to prepare them for stem cell transplant. Bone marrow is the tissue inside the bones where stem cells are made.Stem cells will be harvested from the blood or bone marrow of genetically matched unrelated donors or partially matched family donors. The stem cells will be filtered using a new device that is currently under study. The patients will receive large doses of the filtered stem cells (stem cell graft). Researchers want to find out how the study treatment affects patients, the disease, and the chances for survival.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
28

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Dec 2002

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2002

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2005

Completed
8 days until next milestone

First Submitted

Initial submission to the registry

September 9, 2005

Completed
7 days until next milestone

First Posted

Study publicly available on registry

September 16, 2005

Completed
1.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2007

Completed
Last Updated

April 9, 2010

Status Verified

April 1, 2010

Enrollment Period

2.8 years

First QC Date

September 9, 2005

Last Update Submit

April 7, 2010

Conditions

Aplastic Anemia

Keywords

Aplastic Anemia

Outcome Measures

Primary Outcomes (4)

  • To learn the safety of stem cell transplantation in patients with unrelated donors or partially matched family donors

  • To study the effects (good and bad) of this treatment on the patients, the aplastic anemia, and survival

  • To learn how well the donor bone marrow grows in patients who receive the research treatment

  • To learn how many patients need extra T-cells or extra stem cells from the donor to help the donor's blood stem cells grow

Interventions

Allogeneic stem cell transplantPROCEDURE
Fludarabine, CyclophosphamideDRUG

Eligibility Criteria

AgeUp to 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age \< 21 years
  • Diagnosis of severe aplastic anemia. (As defined by at least 2 of the following: ANC \< 500/μl, platelet count \< 20,000/μl, and a reticulocyte count \< 1% after correction for the hematocrit. In addition, the diagnostic bone marrow biopsy must contain less than 25% of the normal cellularity).
  • Patient must have failed one or more courses of immunosuppressive therapy that included ATG. As immunosuppression may take up to 6 months to demonstrate a response, patients must have been observed to have failed immunosuppression for a minimum of six months.
  • Absence of suitable HLA-matched sibling donor.
  • Negative serum pregnancy test for females with child bearing potential.
  • Patient/parent/guardian is able to provide informed consent.

You may not qualify if:

  • Patients with a life expectancy \< 6 weeks.
  • Patients with severe renal disease (creatinine clearance \< 40cc/min/1.73m2)
  • Patients with pre-existing severe restrictive pulmonary disease (FVC \<40% of predicted)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

fludarabineCyclophosphamide

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Study Officials

  • Paul Woodard, MD

    St. Jude Children's Research Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER

Study Record Dates

First Submitted

September 9, 2005

First Posted

September 16, 2005

Study Start

December 1, 2002

Primary Completion

September 1, 2005

Study Completion

May 1, 2007

Last Updated

April 9, 2010

Record last verified: 2010-04

Locations

US(1)