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Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases (SIDS)

Primary Purpose

Severe Immunodeficiency Diseases

Status
Unknown status
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Fludarabine, Busulfan, and Anti-Thymocyte Globulin
Sponsored by
Ann & Robert H Lurie Children's Hospital of Chicago
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Severe Immunodeficiency Diseases focused on measuring Patients with the Severe Immunodeficiency diseases, Severe Immunodeficiency diseases

Eligibility Criteria

undefined - 11 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Memorial HospitalRecruiting

Outcomes

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

Secondary Outcome Measures

To prospectively follow the natural course of severe immunodeficiency diseases after transplantation
To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.

Full Information

First Posted
January 31, 2008
Last Updated
May 26, 2009
Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
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1. Study Identification

Unique Protocol Identification Number
NCT00613561
Brief Title
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases
Acronym
SIDS
Official Title
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases
Study Type
Interventional

2. Study Status

Record Verification Date
May 2009
Overall Recruitment Status
Unknown status
Study Start Date
December 2007 (undefined)
Primary Completion Date
December 2012 (Anticipated)
Study Completion Date
December 2017 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Immunodeficiency Diseases
Keywords
Patients with the Severe Immunodeficiency diseases, Severe Immunodeficiency diseases

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
25 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Fludarabine, Busulfan, and Anti-Thymocyte Globulin
Other Intervention Name(s)
Anti-Thymocyte Globulin is also refered to as ATG.
Intervention Description
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
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
To prospectively follow the natural course of severe immunodeficiency diseases after transplantation
Time Frame
5 years
Title
To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.
Time Frame
5 years

10. Eligibility

Sex
All
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Morris Kletzel, MD
Phone
773-880-4562
Email
mkletzel@childrensmemorial.org
First Name & Middle Initial & Last Name or Official Title & Degree
Colleen E Schaefer, BS
Phone
773-880-3459
Email
cschaefer@childrensmemorial.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Morris Kletzel, MD
Organizational Affiliation
Ann & Robert H Lurie Children's Hospital of Chicago
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Individual Site Status
Recruiting

12. IPD Sharing Statement

Learn more about this trial

Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

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