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

This study is ongoing, but not recruiting participants.

First Received: October 18, 1999 Last Updated: June 23, 2005 History of Changes

Sponsors and Collaborators:	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Seattle Children's Hospital
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004470

Purpose

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.

Condition	Intervention	Phase
Leukocyte Adhesion Deficiency Syndrome	Drug: filgrastim Drug: Retrovirus vector LgCD18	Phase I

MedlinePlus related topics: Adhesions

Drug Information available for: Filgrastim

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:	4
Study Start Date:	October 1999

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.

Eligibility

Ages Eligible for Study:	4 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004470

Sponsors and Collaborators

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

Seattle Children's Hospital

Investigators

Study Chair:

Dennis D. Hickstein

Seattle Children's Hospital

More Information

No publications provided

Study ID Numbers:	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 First Received:	October 18, 1999
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004470 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

immunologic disorders and infectious disorders
leukocyte adhesion deficiency syndrome
primary immunodeficiency disease
rare disease

Study placed in the following topic categories:

Rare Diseases
Adhesions
Leukocyte-Adhesion Deficiency Syndrome
Immunologic Deficiency Syndromes

Additional relevant MeSH terms:

Disease
Pathologic Processes
Immune System Diseases
Syndrome

Adhesions
Leukocyte-Adhesion Deficiency Syndrome
Immunologic Deficiency Syndromes

ClinicalTrials.gov processed this record on May 07, 2009