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

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Severe Immunodeficiency Diseases	Drug: Fludarabine, Busulfan, and Anti-Thymocyte Globulin	Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	25 participants
Allocation:	Non-Randomized
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases
Study Start Date :	December 2007
Estimated Primary Completion Date :	December 2012
Estimated Study Completion Date :	December 2017

Arms and Interventions

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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.

Outcome Measures

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Primary Outcome Measures :

1. 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 :

1. To prospectively follow the natural course of severe immunodeficiency diseases after transplantation [ Time Frame: 5 years ]
2. To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen. [ Time Frame: 5 years ]

Eligibility Criteria

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Ages Eligible for Study:	up to 11 Years (Child)
Sexes Eligible for Study:	All
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

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
First Posted:	February 13, 2008
Last Update Posted:	May 27, 2009
Last Verified:	May 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; Fludarabine phosphate; Busulfan; Antilymphocyte Serum; Antineoplastic Agents; Antimetabolites, Antineoplastic	Antimetabolites; Molecular Mechanisms of Pharmacological Action; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Alkylating Agents; Antineoplastic Agents, Alkylating; Myeloablative Agonists