Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Status

UNKNOWN

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00613561

Acronym

SIDS

Enrollment

Registered

2008-02-13

Start date

2007-12-31

Completion date

2017-12-31

Last updated

2009-05-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Severe Immunodeficiency Diseases

Keywords

Patients with the Severe Immunodeficiency diseases, 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.

Interventions

DRUGFludarabine, 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

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 11 Years

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

Design outcomes

Primary

Measure	Time frame
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	5 years

Secondary

Measure	Time frame
To prospectively follow the natural course of severe immunodeficiency diseases after transplantation	5 years
To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.	5 years

Countries

United States

Contacts

Primary ContactMorris Kletzel, MD

mkletzel@childrensmemorial.org773-880-4562

Backup ContactColleen E Schaefer, BS

cschaefer@childrensmemorial.org773-880-3459

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026