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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
Information provided by:
Ann & Robert H Lurie Children's Hospital of Chicago Identifier:
First received: January 31, 2008
Last updated: May 26, 2009
Last verified: May 2009
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
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 ]

Secondary Outcome Measures:
  • 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 ]

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.

Ages Eligible for Study:   up to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 identifier: NCT00613561

Contact: Morris Kletzel, MD 773-880-4562
Contact: Colleen E Schaefer, BS 773-880-3459

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
Principal Investigator: Morris Kletzel, MD Ann & Robert H Lurie Children's Hospital of Chicago
  More Information

Responsible Party: Morris Kletzel, MD, Children's Memorial Hospital 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

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 phosphate
Antilymphocyte Serum
Antineoplastic Agents
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Alkylating Agents
Antineoplastic Agents, Alkylating
Myeloablative Agonists processed this record on May 24, 2017