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| Sponsors and Collaborators: |
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Children's Hospital and Regional Medical Center, Seattle |
| 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 |
| Study Type: | Interventional |
| Study Design: | Treatment |
| Estimated Enrollment: | 4 |
| Study Start Date: | October 1999 |
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 |
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| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
| Children's Hospital and Regional Medical Center, Seattle |
| Study Chair: | Dennis D. Hickstein | Children's Hospital and Regional Medical Center, Seattle |
More Information
| 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 |
| First Received: | October 18, 1999 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00004470 |
| Health Authority: | United States: Federal Government |
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