Patients Treated for SCID (1968-Present)

ADA-SCID Clinical Trial

Official title: A Retrospective and Cross-Sectional Analysis of Patients Treated for SCID Since January 1,1968 (RDCRN PIDTC-6902)

Status Enrolling by invitation

Clinical Trial Summary

Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant, gene therapy, or enzyme replacement in the past may be eligible for this study. The purpose of study is to look backwards at what has already been done in the. Over 800 patients with SCID are expected to be enrolled, making this one of the largest studies ever to describe outcomes for patients with SCID treated at many different hospitals around North America.

Clinical Trial Description

One of the most important components of this study is the "cross sectional" aspect. Patients who have received their treatments (BMT, gene therapy, enzyme replacement) many years ago are asked to come back to the hospital where they were treated. During this visit, additional research bloodwork is drawn, and information is gathered regarding long-term transplant outcomes such as infections, graft-versus-host disease, autoimmune diseases, and quality of life. This will allow PIDTC researchers to better understand long-term outcomes from procedures that occurred many years ago (sometimes over 30 years ago). This will help researchers to best design new treatments and clinical trials in the future for children with SCID. ;

Related Conditions & MeSH terms

• ADA-SCID
• Leaky SCID
• Omenn Syndrome
• Reticular Dysgenesis
• SCID
• Severe Combined Immunodeficiency
• XSCID

• NCT number: NCT01346150
• Study type: Observational
• Source: National Institute of Allergy and Infectious Diseases (NIAID)
• Status: Enrolling by invitation
• Start date: May 15, 2011
• Completion date: August 2023