PHEM15022 / PIDTC-6901 / Jeffrey Andolina
Basic Study Information
Purpose:Location: University of Rochester Medical Center/ Golisano Children's Hospital
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
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.
Lead Researcher (Principal Investigator)
Study Contact InformationStudy Coordinator: Jessica Mackowiak
Additional Study Details
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