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URMC / Research / Clinical Trials / Study Details

PHEM15022 / PIDTC-6901 / Jeffrey Andolina

Basic Study Information

Purpose:
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important in determining outcome, study investigators will uniformly collect pre-, post- and peri-transplant (or other treatment) information on all children enrolled into this study. Children will be divided into three strata: Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell function Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function), and Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy. Each Group/Cohort Stratum will be analyzed separately.

Location: University of Rochester Medical Center/ Golisano Children's Hospital

Lead Researcher (Principal Investigator)

Lead Researcher:  Jeffrey Andolina

Study Contact Information

Study Coordinator: Jessica Mackowiak
Email: jessica_ellis@urmc.rochester.edu

Additional Study Details

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