Purified CD34+ Hematopoietic Stem Cell Transplantation Fr... | NCT00186797

Studies
Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia

NCT00186797

Sponsor

St. Jude Children's Research Hospital

Status

Completed

Last Update Posted

Apr 9, 2010Estimated

Enrollment

Phase

Not Applicable

Conditions

Aplastic Anemia

Interventions

Allogeneic stem cell transplant
Fludarabine, Cyclophosphamide

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2010

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Not provided

Start Date

Dec 2002

Primary Completion Date

Sep 2005Actual

Completion Date

May 2007Actual

First Submitted Date

Sep 9, 2005

First Submission Date that Met QC Criteria

Sep 9, 2005

First Posted Date

Sep 16, 2005Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 7, 2010

Last Update Posted Date

Apr 9, 2010Estimated

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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.

Exclusion Criteria:

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)

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

21 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Paul Woodard, MD	St. Jude Children's Research Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
St. Jude Children's Research Hospital		Memphis	Tennessee	38105	United States

Label	URL
St. Jude Children's Research Hospital	View source

Condition Browse Module

MeSH Terms

ID	Term
D000741	Anemia, Aplastic

Ancestor Terms

ID	Term
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D000080983	Bone Marrow Failure Disorders

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C024352	fludarabine
D003520	Cyclophosphamide

Ancestor Terms

ID	Term
D010752	Phosphoramide Mustards
D009588	Nitrogen Mustard Compounds
D009150	Mustard Compounds
D006846	Hydrocarbons, Halogenated

Browse Leaves

Not provided

Browse Branches

Not provided

Name	Type	Description	Arm Group Labels	Other Names
Allogeneic stem cell transplant	Procedure
Fludarabine, Cyclophosphamide	Drug