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| Sponsor: | Baylor College of Medicine |
|---|---|
| Information provided by: | Baylor College of Medicine |
| ClinicalTrials.gov Identifier: | NCT00579137 |
Purpose
This study is to discover whether children with severe combined immunodeficiency disease (SCID) or other primary immunodeficiency disorder (PID) for which no satisfactory treatment other than stem cell transplantation (SCT) exists can be safely and effectively transplanted from HLA mismatched (up to one haplotype) related donors or unrelated matched or mismatched (up to one antigen) donors, when leukocytolytic monoclonal antibodies and Fludarabine are the sole conditioning agents. Three monoclonal antibodies will be used in combination. Two of them, YTH 24 and YTH 54 are rat IgG1 antibodies directed against two contiguous epitopes on the CD45 (common leucocyte) antigen. They have been safely administered as part of the conditioning regimen for 12 patients receiving allografts (HLA matched and mismatched) at this center. They produce a transient depletion of >90% circulating leucocytes. The third MAb is Campath 1H, a humanized rat anti-CD52 MAb. Campath 1H, Alemtuzumab, has been licensed to treat B-CLL and more recently has been safely given at this and other centers as part of a sub-ablative conditioning regimen to patients with malignant disease. Because these MAb produce both profound immunosuppression and significant, though transient, myelodestruction we believe they may be useful as the sole conditioning regimen in patients with SCID, in whom the use of conventional chemotherapeutic agents for conditioning may produce or aggravate unacceptable and even lethal short term toxicity. We anticipate MAb mediated subablative conditioning will permit engraftment in a high percentage of these patients with little or no immediate or long term toxicity. Campath IH persists in vivo for several days after administration and so will be present over the transplant period to deplete donor T cells as partial GvHD prophylaxis. Additional GvHD prophylaxis may be provided by administration of FK506.
| Condition | Intervention | Phase |
|---|---|---|
|
Immunodeficiency |
Drug: Campath -1H Drug: Fludarabine Biological: Anti-CD45 Procedure: Stem cell infusion |
Phase II |
| Study Type: | Interventional |
| Study Design: | Prevention, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| Official Title: | CD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease And Other Primary Immunodeficiency Disorders |
| Estimated Enrollment: | 21 |
| Study Start Date: | October 2007 |
| Estimated Primary Completion Date: | October 2015 (Final data collection date for primary outcome measure) |
Days -8,-7, and -6:
Intravenous:
Campath dose is weight based: for patients less than 15 kg administer Campath 3 mg; for patients >15 kg to 30 kg administer Campath 5 mg; for patients > 30 kg administer Campath 10 mg
Days -8,-7,-6,-5, and -4:
Intravenous 30 mg/m2
Days -5,-4,-3, and -2:
Intravenous, 400ug/kg over 6 hours
Show Detailed Description
Eligibility| Ages Eligible for Study: | up to 17 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | Yes |
Inclusion Criteria:
- Patients with a diagnosis of: Severe combined immunodeficiency disease
This includes patients whose SCID is characterized by gene specific mutations as well as patients with clinically severe combined immunodeficiency without a defined genetic cause in which the diagnosis will be determined by a combination of clinical course with lymphocyte quantification and function assays.
OR
Severe primary immunodeficiency disorder, including undefined T cell deficiency disorder, Wiskott-Aldrich syndrome, and other severe immunodeficiencies for which satisfactory conventional therapy does not exist.
Exclusion Criteria:
Contacts and Locations| Contact: Robert Krance, MD | 832-824-4661 | rkrance@bcm.tmc.edu |
| Contact: Malcolm Brenner, MD | mkbrenne@txccc.org |
| United States, Texas | |
| Texas Children's Hospital | Recruiting |
| Houston, Texas, United States, 77030 | |
| Principal Investigator: | Robert Krance, MD | Baylor College of Medicine |
| Study Chair: | Malcolm Brenner, MD | Baylor College of Medicine |
More Information
| Responsible Party: | Baylor College of Medicine ( Robert Krance ) |
| Study ID Numbers: | H-21123 |
| Study First Received: | December 19, 2007 |
| Last Updated: | August 6, 2009 |
| ClinicalTrials.gov Identifier: | NCT00579137 History of Changes |
| Health Authority: | United States: Food and Drug Administration |
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immunodeficiency disorders |
|
Metabolic Diseases Immune System Diseases Antineoplastic Agents Severe Combined Immunodeficiency Therapeutic Uses Alemtuzumab |
DNA Repair-Deficiency Disorders Infant, Newborn, Diseases Fludarabine Pharmacologic Actions Immunologic Deficiency Syndromes |