- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00613561
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases (SIDS)
May 26, 2009 updated by: Ann & Robert H Lurie Children's Hospital of Chicago
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.
Study Overview
Status
Unknown
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Anticipated)
25
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Morris Kletzel, MD
- Phone Number: 773-880-4562
- Email: MKletzel@childrensmemorial.org
Study Contact Backup
- Name: Colleen E Schaefer, BS
- Phone Number: 773-880-3459
- Email: cschaefer@childrensmemorial.org
Study Locations
-
-
Illinois
-
Chicago, Illinois, United States, 60614
- Recruiting
- Children's Memorial Hospital
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 11 years (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
|
5 years
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
To prospectively follow the natural course of severe immunodeficiency diseases after transplantation
Time Frame: 5 years
|
5 years
|
To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.
Time Frame: 5 years
|
5 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Morris Kletzel, MD, Ann & Robert H Lurie Children's Hospital of Chicago
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
December 1, 2007
Primary Completion (Anticipated)
December 1, 2012
Study Completion (Anticipated)
December 1, 2017
Study Registration Dates
First Submitted
January 31, 2008
First Submitted That Met QC Criteria
February 12, 2008
First Posted (Estimate)
February 13, 2008
Study Record Updates
Last Update Posted (Estimate)
May 27, 2009
Last Update Submitted That Met QC Criteria
May 26, 2009
Last Verified
May 1, 2009
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Immune System Diseases
- Immunologic Deficiency Syndromes
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Antineoplastic Agents, Alkylating
- Alkylating Agents
- Myeloablative Agonists
- Fludarabine
- Busulfan
- Antilymphocyte Serum
Other Study ID Numbers
- SCT 0707
- IRB # 2007-13271
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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