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

This study is currently recruiting participants.
Verified May 2009 by Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor:
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
History of Changes
  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:

U.S. FDA Resources

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
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
Busulfan
Fludarabine monophosphate
Fludarabine
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
 Pharmacologic Actions
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Therapeutic Uses
Myeloablative Agonists
Antimetabolites, Antineoplastic
Antimetabolites

ClinicalTrials.gov processed this record on May 16, 2013