Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-12-31

Study Completion Date

2017-12-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Severe Immunodeficiency Diseases

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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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

Intervention Type DRUG

Other Intervention Names

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Anti-Thymocyte Globulin is also refered to as ATG.

Eligibility Criteria

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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

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No