Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases (SIDS)
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ClinicalTrials.gov Identifier: NCT00613561 |
Recruitment Status
: Unknown
Verified May 2009 by Ann & Robert H Lurie Children's Hospital of Chicago.
Recruitment status was: Recruiting
First Posted
: February 13, 2008
Last Update Posted
: May 27, 2009
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Severe Immunodeficiency Diseases | Drug: Fludarabine, Busulfan, and Anti-Thymocyte Globulin | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 25 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases |
Study Start Date : | December 2007 |
Estimated Primary Completion Date : | December 2012 |
Estimated Study Completion Date : | December 2017 |
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Drug: Fludarabine, Busulfan, and Anti-Thymocyte Globulin
- To evaluate the efficacy of an optimized reduced intensity conditioning regimen using Fludarabine, Busulfan, and Anti-Thymocyte Globulin in preparing severe immunodeficiency patients for allogeneic hematopoietic progenitor cell transplantation [ Time Frame: 5 years ]
- To prospectively follow the natural course of severe immunodeficiency diseases after transplantation [ Time Frame: 5 years ]
- To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen. [ Time Frame: 5 years ]

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Ages Eligible for Study: | up to 11 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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Must show one of the following diseases:
- SCIDS
- Hyper-IgM
- Wiskott-Aldrich Syndrome
- Chediak-Higashi and Griscelli Syndromes
- X-Linked Lymphoproliferative Diseases
- IPEX Syndrome
- NEMO Syndrome
- other severe immunodeficiency diseases not stated above at the discretion of the Principal Investigator
- Informed Consent
- Adequate Renal Function
- Adequate Liver Function
- Adequate Cardiac Function
- Adequate Pulmonary Function
- Adequate Performance Statue
- Adequate Venous Access
Exclusion Criteria:
- Patient/Family has not signed informed consent
- Patient does not have a clear diagnosis of a severe immunodeficiency disease
- A suitable donor for the patient cannot be found
- Patient is HIV positive
- Patient has active Hepatitis B
- Patient is pregnant
- Patient is considered unsuitable for transplant at the discretion of the Principal Investigators or the medical director

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00613561
Contact: Morris Kletzel, MD | 773-880-4562 | mkletzel@childrensmemorial.org | |
Contact: Colleen E Schaefer, BS | 773-880-3459 | cschaefer@childrensmemorial.org |
United States, Illinois | |
Children's Memorial Hospital | Recruiting |
Chicago, Illinois, United States, 60614 |
Principal Investigator: | Morris Kletzel, MD | Ann & Robert H Lurie Children's Hospital of Chicago |
Responsible Party: | Morris Kletzel, MD, Children's Memorial Hospital |
ClinicalTrials.gov Identifier: | NCT00613561 History of Changes |
Other Study ID Numbers: |
SCT 0707 IRB # 2007-13271 |
First Posted: | February 13, 2008 Key Record Dates |
Last Update Posted: | May 27, 2009 |
Last Verified: | May 2009 |
Keywords provided by Ann & Robert H Lurie Children's Hospital of Chicago:
Patients with the Severe Immunodeficiency diseases Severe Immunodeficiency diseases |
Additional relevant MeSH terms:
Immunologic Deficiency Syndromes Immune System Diseases Fludarabine Fludarabine phosphate Busulfan Antilymphocyte Serum Antineoplastic Agents Antimetabolites, Antineoplastic |
Antimetabolites Molecular Mechanisms of Pharmacological Action Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Alkylating Agents Antineoplastic Agents, Alkylating Myeloablative Agonists |