Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00004294
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
Sponsor:
Collaborators:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
University of Pittsburgh
Information provided by:
National Center for Research Resources (NCRR)

Tracking Information
First Submitted Date  ICMJE October 18, 1999
First Posted Date  ICMJE October 19, 1999
Last Update Posted Date June 24, 2005
Study Start Date  ICMJE November 1999
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE Not Provided
Original Primary Outcome Measures  ICMJE Not Provided
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease
Official Title  ICMJE Not Provided
Brief Summary

OBJECTIVES: I. Transfer the human glucocerebrosidase (GC) gene into peripheral blood stem cells (PBSC) obtained from patients with type I Gaucher disease using a retroviral vector.

II. Transplant the autologous transduced PBSC in these patients. III. Measure the carriage and expression of the transferred gene and its duration in peripheral blood leukocytes.

IV. Assess the clinical effects of transplanting genetically corrected PBSC.

Detailed Description

PROTOCOL OUTLINE: Patients undergo autologous transplantation using peripheral blood stems cells (PBSC) stimulated with filgrastim (G-CSF) and transduced with a retroviral vector containing the human glucocerebrosidase gene (R-GC). Patients may receive up to 4 transplants if a deficient glucocerebrosidase level is found in peripheral leukocytes 1 month following transplantation.

The G-CSF-stimulated PBSC are enriched for CD34-positive stem cells and transduced with the R-GC vector. Stem cells with normal gene activity are selected for transplantation.

Patients are followed every month for 6 months, every 6 months for 18 months, and then annually thereafter.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Primary Purpose: Treatment
Condition  ICMJE Gaucher's Disease
Intervention  ICMJE Genetic: human glucocerebrosidase gene into autologous peripheral blood stem cells
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE
 (submitted: June¬†23,¬†2005)
15
Original Enrollment  ICMJE Same as current
Study Completion Date  ICMJE Not Provided
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Enzyme proven type I Gaucher disease Glucocerebrosidase (GC) activity less than 30% of normal GC mutation identified Significant signs and symptoms of disease prior to therapy initiation At least 1 of the following after 12 months of therapy: Liver at least 2 times normal size Spleen at least 5 times normal size Platelet count greater than 150,000/mm3 Clinical bone disease with pain, fractures, or infarctions Multiple sites of marrow involvement Angiotensin-converting enzyme at least 1.5 times normal No GC antibody At least 3 of the following responses to therapy: Hemoglobin increase of 2 g/dL Platelet count increase of 50% Spleen or liver size decrease at least 25% Improvement in MRI or x-ray of the bones Nontartrate inhibitable acid phosphatase decrease of 50% Angiotensin-converting enzyme decrease of 50% OR Previously untreated and immediate enzyme therapy would not be life saving Meets at least 2 of the following criteria: Spleen at least 5 times normal size or liver at least 2 times normal size by physical exam and MRI Hemoglobin less than 11 g/dL Platelet count less than 90,000/mm3 Disabling bone pain with degenerative changes on x-ray Multiple sites of bone marrow infiltration and evidence of bony changes Pulmonary compromise with clubbing and PaO2 less than 70 mm Hg Biopsy proven cirrhosis and elevated hepatic parenchymal enzymes Bleeding esophageal varices --Prior/Concurrent Therapy-- At least 3 months since any prior investigational therapy Concurrent enzyme replacement therapy may be tapered on study --Patient Characteristics-- HIV negative No malignant disease No known sensitivity to egg or murine products Not pregnant or nursing Fertile patients must use effective contraception

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00004294
Other Study ID Numbers  ICMJE 199/11727
UPITTS-GAUCHER
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE National Center for Research Resources (NCRR)
Collaborators  ICMJE
  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • University of Pittsburgh
Investigators  ICMJE
Study Chair: John Barranger University of Pittsburgh
PRS Account National Center for Research Resources (NCRR)
Verification Date December 2003

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP