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
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

January 31, 2008
May 26, 2009
December 2007
December 2012   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT00613561 on ClinicalTrials.gov Archive Site
  • 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 ]
  • 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 transplatation using the reduced intensity transplant regimen. [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

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.

Not Provided
Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Severe Immunodeficiency Diseases
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.
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
25
December 2017
December 2012   (final data collection date for primary outcome measure)

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
Both
up to 11 Years
No
Contact: Morris Kletzel, MD 773-880-4562 mkletzel@childrensmemorial.org
Contact: Colleen E Schaefer, BS 773-880-3459 cschaefer@childrensmemorial.org
United States
 
NCT00613561
SCT 0707, IRB # 2007-13271
No
Morris Kletzel, MD, Children's Memorial Hospital
Ann & Robert H Lurie Children's Hospital of Chicago
Not Provided
Principal Investigator: Morris Kletzel, MD Ann & Robert H Lurie Children's Hospital of Chicago
Ann & Robert H Lurie Children's Hospital of Chicago
May 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP