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

Tracking Information

First Received Date ^ICMJE

January 31, 2008

Last Updated Date

May 26, 2009

Start Date ^ICMJE

December 2007

Estimated Primary Completion Date

December 2012 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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 ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00613561 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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 ]

Original Secondary Outcome Measures ^ICMJE

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 ]

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Official Title ^ICMJE

Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Severe Immunodeficiency Diseases

Intervention ^ICMJE

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.

Study Arm (s)

Not Provided

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

December 2017

Estimated Primary Completion Date

December 2012 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

up to 11 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Morris Kletzel, MD	773-880-4562	mkletzel@childrensmemorial.org
Contact: Colleen E Schaefer, BS	773-880-3459	cschaefer@childrensmemorial.org

Location Countries ^ICMJE

United States

Administrative Information

NCT Number ^ICMJE

NCT00613561

Other Study ID Numbers ^ICMJE

SCT 0707, IRB # 2007-13271

Has Data Monitoring Committee

Responsible Party

Morris Kletzel, MD, Children's Memorial Hospital

Study Sponsor ^ICMJE

Ann & Robert H Lurie Children's Hospital of Chicago

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

Morris Kletzel, MD

Ann & Robert H Lurie Children's Hospital of Chicago

Information Provided By

Ann & Robert H Lurie Children's Hospital of Chicago

Verification Date

May 2009

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

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