Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases (SIDS)
This study is currently recruiting participants.
(see Contacts and Locations)
Verified May 2009 by Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor:
Ann & Robert H Lurie Children's Hospital of Chicago
Information provided by:
Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier:
NCT00613561
First received: January 31, 2008
Last updated: May 26, 2009
Last verified: May 2009
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Purpose
The hypothesis of this study is that children with severe primary immunodeficiencies will benefit from early stem cell transplantation utilizing a reduced intensity conditioning regimen. This regimen is associated with a low risk of complications and will lead to correction of the underlying immunological defects.
| Condition | Intervention | Phase |
|---|---|---|
|
Severe Immunodeficiency Diseases |
Drug: Fludarabine, Busulfan, and Anti-Thymocyte Globulin |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases |
Resource links provided by NLM:
Further study details as provided by Ann & Robert H Lurie Children's Hospital of Chicago:
Primary Outcome Measures:
- 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 ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- To prospectively follow the natural course of severe immunodeficiency diseases after transplantation [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
- To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen. [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 25 |
| Study Start Date: | December 2007 |
| Estimated Study Completion Date: | December 2017 |
| Estimated Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
Intervention Details:
-
Drug: Fludarabine, Busulfan, and Anti-Thymocyte Globulin
Patients on this study will receive 5 days of Fludarabine, 1 day of Busulfan Test Dose, 2 Days of Busulfan Regimen Dose, and 4 days of Anti-Thymocyte Globulin
Other Name: Anti-Thymocyte Globulin is also refered to as ATG.
Eligibility| Ages Eligible for Study: | up to 11 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
-
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
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00613561
Please refer to this study by its ClinicalTrials.gov identifier: NCT00613561
Contacts
| Contact: Morris Kletzel, MD | 773-880-4562 | mkletzel@childrensmemorial.org | |
| Contact: Colleen E Schaefer, BS | 773-880-3459 | cschaefer@childrensmemorial.org |
Locations
| United States, Illinois | |
| Children's Memorial Hospital | Recruiting |
| Chicago, Illinois, United States, 60614 | |
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Investigators
| Principal Investigator: | Morris Kletzel, MD | Ann & Robert H Lurie Children's Hospital of Chicago |
More Information
No publications provided
| 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 |
| Study First Received: | January 31, 2008 |
| Last Updated: | May 26, 2009 |
| Health Authority: | United States: Institutional Review Board |
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 Antilymphocyte Serum Immunologic Factors |
Immunosuppressive Agents Pharmacologic Actions Physiological Effects of Drugs |
ClinicalTrials.gov processed this record on March 01, 2015