Phase I Study of Retrovirus-Mediated Transfer of cDNA for Human CD18 Into Peripheral Blood Repopulating Cells of Patients With Leukocyte Adherence Deficiency - Tabular View

Tracking Information

First Received Date ^ICMJE

October 18, 1999

Last Updated Date

June 23, 2005

Start Date ^ICMJE

October 1999

Primary Completion Date

Current Primary Outcome Measures ^ICMJE

Original Primary Outcome Measures ^ICMJE

Change History

Complete list of historical versions of study NCT00004470 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Original Secondary Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

Phase I Study of Retrovirus-Mediated Transfer of cDNA for Human CD18 Into Peripheral Blood Repopulating Cells of Patients With Leukocyte Adherence Deficiency

Official Title ^ICMJE

Brief Summary

OBJECTIVES:

I. Investigate the efficiency and safety of transducing the human CD18 DNA into filgrastim (G-CSF) mobilized, CD34 enriched peripheral blood repopulating cells from patients with a severe or moderate deficiency form of leukocyte adherence deficiency (LAD) by retrovirus-mediated gene transfer.

II. Investigate whether the human CD18 cDNA is expressed sufficiently to benefit patients with this disease.

III. Determine whether repeated infusions of transduced peripheral blood repopulating cells increase the number of surface CD11/CD18 positive cells in these patients.

IV. Determine the extent of long-term persistence of transduced peripheral blood repopulating cells in LAD patients not receiving prior myeloablation.

Detailed Description

PROTOCOL OUTLINE:

Patients receive filgrastim (G-CSF) subcutaneously daily on days 1-5 with peripheral blood cell collections on days 4 and 5. Patients receive infusions of transduced CD34+ peripheral blood cells on day 8. Patients may be offered a second course of transduced peripheral blood cells for a total of 4 infusions.

Patients are followed monthly for one year and then annually for 4 years.

Study Phase

Phase I

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment

Condition ^ICMJE

Leukocyte Adhesion Deficiency Syndrome

Intervention ^ICMJE

Drug: filgrastim
Drug: Retrovirus vector LgCD18

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Active, not recruiting

Enrollment ^ICMJE

Completion Date

Primary Completion Date

Eligibility Criteria ^ICMJE

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Leukocyte adherence deficiency (LAD): Severe or moderate deficiency LAD disease confirmed by surface immunofluorescence of peripheral blood leukocytes of less than 10% normal

Measurable clinical disease in the form of at least one episode of life-threatening disease

Allogeneic bone marrow transplantation offered if HLA-matched sibling donor available

--Patient Characteristics--

Performance status: Karnofsky 60-100%

Hematopoietic: WBC greater than 2,000/mm3; Absolute neutrophil count greater than 1,000/mm3; Platelet count greater than 50,000/mm3; Prothrombin time and partial thromboplastin time less than 1.5 times upper limit of normal (ULN)

Hepatic: Bilirubin no greater than 2.5 mg/dL; SGOT and SGPT no greater than 5 times ULN; Alkaline phosphatase no greater than 2 times ULN

Renal: Creatinine clearance greater than 50 mL/min

Cardiovascular: Normal cardiac function

Pulmonary: No cardiorespiratory instability

Other: Triglycerides less than 400 mg/dL; Amylase no greater than 1.5 times ULN; HIV negative; No acute infection; Not pregnant or nursing; Fertile patients must use effective contraception

Gender

Both

Ages

4 Years to 65 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

Administrative Information

NCT ID ^ICMJE

NCT00004470

Responsible Party

Study ID Numbers ^ICMJE

199/13939, CHMC-S-IRB-198-99-07, CHMC-S-DK47754, CHMC-S-HL54881, CHMC-S-IRB-207-9708, CHMC-S-IRB-216-00-07, FHCRC-981, NIH/ORDA-9707-204, UWASH-198-99-07, UWASH-207-9708

Study Sponsor ^ICMJE

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Collaborators ^ICMJE

Seattle Children's Hospital

Investigators ^ICMJE

Study Chair:

Dennis D. Hickstein

Seattle Children's Hospital

Information Provided By

Office of Rare Diseases (ORD)

Verification Date

May 2001

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