DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Information source: Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Severe Immunodeficiency Diseases

Intervention: Fludarabine, Busulfan, and Anti-Thymocyte Globulin (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Ann & Robert H Lurie Children's Hospital of Chicago

Official(s) and/or principal investigator(s):
Morris Kletzel, MD, Principal Investigator, Affiliation: Ann & Robert H Lurie Children's Hospital of Chicago

Overall contact:
Morris Kletzel, MD, Phone: 773-880-4562, Email: mkletzel@childrensmemorial.org

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.

Clinical Details

Official title: Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

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

Primary outcome: 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

Secondary outcome:

To prospectively follow the natural course of severe immunodeficiency diseases after transplantation

To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.

Eligibility

Minimum age: N/A. Maximum age: 11 Years. Gender(s): Both.

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

Locations and Contacts

Morris Kletzel, MD, Phone: 773-880-4562, Email: mkletzel@childrensmemorial.org

Children's Memorial Hospital, Chicago, Illinois 60614, United States; Recruiting
Additional Information

Starting date: December 2007
Last updated: May 26, 2009

Page last updated: August 20, 2015

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017