Recruiting
Phase 2

Stem Cell Transplantation

Sponsor:

Ann & Robert H Lurie Children's Hospital of Chicago

Code:

NCT00613561

Conditions

Severe Immunodeficiency Diseases

Eligibility Criteria

Sex: All

Age: 0 - 11

Healthy Volunteers: Not accepted

Interventions

Fludarabine, Busulfan, and Anti-Thymocyte Globulin

Study Details

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.

Conditions

Severe Immunodeficiency Diseases

Study ID

NCT00613561

Start date

Dec, 2007

Status verified date

May, 2009

Completion date

Dec, 2017

Anticipated

Primary completion date

Dec, 2012

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 11

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

25 participants

Anticipated

Allocation

Non randomized

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Interventions

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

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 ]

Central Contacts and Locations

Central contacts

Morris Kletzel, MD

773-880-4562mkletzel@childrensmemorial.org

Colleen E Schaefer, BS

773-880-3459cschaefer@childrensmemorial.org

Locations

Children's Memorial Hospital

Recruiting

Chicago, Illinois, United States, 60614

More Information

Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Last update posted

May 27, 2009

Last verified

May, 2009

Keywords

  • Patients with the Severe Immunodeficiency diseases
  • Severe Immunodeficiency diseases

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Ann & Robert H Lurie Children's Hospital of Chicago on 2009-05-27.