Study

A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders

Study ID Alternative Stable ID Type
phs001392 Longitudinal

Study Description

Individuals, including infants who were identified by newborn screening, with a recent/new diagnosis of severe combined immune deficiency (SCID) may be eligible to be enrolled in the PIDTC research study: A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders. To determine eligibility for this study, potential participants (or, in the case of children, their parents/guardians) should consult with their doctors. The study follows participants with SCID prospectively. That is, the study enrolls participants who already have a plan to receive a blood and marrow transplant (BMT), enzyme therapy, or gene therapy in the future. After the procedure, participants are followed according to a study schedule. The participants' study visits will coincide with the follow-up visits with their doctors as part of their regular ongoing medical care. Participants with “leaky SCID”, reticular dysgenesis, and Omenn syndrome may also be eligible to participate in this study. As the PIDTC recognizes that there are many complex factors ... (Show More)

Archive Link Archive Accession
dbGaP phs001392

Who archives the data?

There are no publications available