Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

• Status: Unknown status
• Trial end date: 2017-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
• Treatments: Antilymphocyte Serum; Busulfan; Fludarabine; Fludarabine phosphate

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