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| Sponsor: | Baylor College of Medicine |
|---|---|
| Information provided by: | Baylor College of Medicine |
| ClinicalTrials.gov Identifier: | NCT00578643 |
Purpose
Chronic Granulomatous Disease (CGD) is a life threatening primary immunodeficiency caused by the abnormal function of any of four components of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase system present in the phagocytic cells. The estimated incidence of CGD in the United States is between 1/200,000 - 1 /255,000 live births.
Patients will be treated with Campath (Alemtuzumab) an antibody directed against the CD52 antigen, which is present on most lymphocytes. This agent has shown efficacy as an immunosuppressive promoting donor engraftment and prevention of GVHD.
| Condition | Intervention | Phase |
|---|---|---|
|
Chronic Granulomatous Disease |
Drug: Busulfan Drug: alemtuzumab Drug: Cyclophosphamide Drug: Fludarabine Drug: cyclosporine Drug: MESNA Procedure: stem cell infusion |
Phase I |
| Study Type: | Interventional |
| Study Design: | Treatment, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study |
| Official Title: | HLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease |
| Estimated Enrollment: | 15 |
| Study Start Date: | February 2004 |
| Estimated Study Completion Date: | February 2012 |
| Estimated Primary Completion Date: | February 2012 (Final data collection date for primary outcome measure) |
days -9 through -6
1 mg/kg initially (based on weight)
Show Detailed Description
Eligibility| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Patients with acute or chronic leukemia or advanced Hodgkin or non Hodgkin lymphoma or myelodsyplastic/myeloproliferative disease who are unlikely to be cured by standard chemotherapy treatments. This includes patients who have relapsed after standard chemotherapy treatments and patients in first remission with unfavorable prognostic features.
Patient must have a genotype HLA identical stem cell donor.
Exclusion Criteria:
Patients with a life expectancy (less than or equal to 6 weeks) limited by disease other than leukemia.
Patients with symptomatic cardiac failure unrelieved by medical therapy or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction <20%).
Patients with severe renal disease (i.e., creatinine greater than 3 times normal for age).
Patients with pre-existing severe restrictive pulmonary disease (FVC less than 40% of predicted).
Patients with severe hepatic disease (direct bilirubin greater than 3 mg/dl or AST greater than 500 IU/L).
Patients with severe personality disorder or mental illness.
Patients with severe infection that in the estimation of the principal investigator prohibits the use of ablative chemotherapy.
Patients who are documented HIV positive.
Patients with a Karnofsky performance score <60% or Lansky performance score <50%.
NOTE: Patients who would be excluded from treatment on this protocol strictly for laboratory or performance abnormalities can be included at the principal investigator's discretion after consultation with the members of the SCT Policy and Procedures Committee.
Contacts and Locations| Contact: Robert Krance, MD | 832-824-4661 | rkrance@bcm.tmc.edu |
| United States, Texas | |
| Texas Children's Hospital | Recruiting |
| Houston, Texas, United States, 77030 | |
| Sub-Investigator: Bambi J Grilley | |
| Sub-Investigator: Malcolm K Brenner, MD | |
| Sub-Investigator: Helen E Heslop, MD | |
| Sub-Investigator: Stephen M Gottschalk, MD | |
| Sub-Investigator: Catherine M Bollard, MD | |
| Sub-Investigator: Caridad A Martinez, MD | |
| Principal Investigator: | Robert Krance, MD | Baylor College of Medicine |
More Information
| Responsible Party: | Baylor College of Medicine ( Robert A. Krance ) |
| Study ID Numbers: | H-14771 |
| Study First Received: | December 19, 2007 |
| Last Updated: | December 3, 2009 |
| ClinicalTrials.gov Identifier: | NCT00578643 History of Changes |
| Health Authority: | United States: Institutional Review Board |
|
Transplant Granulomatous Disease |
|
Anti-Infective Agents Cyclosporine Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs Leukocyte Disorders Cyclophosphamide Cyclosporins Pathologic Processes Antifungal Agents Alemtuzumab Therapeutic Uses Genetic Diseases, X-Linked Dermatologic Agents |
Alkylating Agents Phagocyte Bactericidal Dysfunction Immune System Diseases Hematologic Diseases Enzyme Inhibitors Granuloma Immunosuppressive Agents Immunologic Deficiency Syndromes Pharmacologic Actions Lymphatic Diseases Genetic Diseases, Inborn Granulomatous Disease, Chronic Myeloablative Agonists Fludarabine Antineoplastic Agents, Alkylating |