Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases (SIDS)

Ann & Robert H Lurie Children's Hospital of Chicago

Status and phase: Unknown; Phase 2

Conditions: Severe Immunodeficiency Diseases

Treatments: Drug: Fludarabine, Busulfan, and Anti-Thymocyte Globulin

Study type: Interventional

Funder types: Other

Identifiers

NCT00613561

IRB # 2007-13271

SCT 0707

Details and patient eligibility

About

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.

Enrollment: 25 estimated patients

Sex: All

Ages: Under 11 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)