Clinical Trial: Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents
Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Observational
Official Title: Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents
Brief Summary:
Background:
- Severe combined immunodeficiency (SCID) is a rare inherited disorder in which certain white blood cells have impaired function and are unable to properly fight infections. SCID typically appears within the first year of life and is characterized by multiple, recurrent severe infections. More than 10 percent of all cases of SCID involve a deficiency of an enzyme called adenosine deaminase (ADA), and these SCID patients also tend to have impaired brain function or psychiatric disorders. Researchers are attempting to treat ADA-SCID patients with an experimental gene therapy, and a research protocol has been established for those who are participating in this therapy.
- Little is known about quality of life in individuals with ADA-SCID, but researchers believe that the effects of the disease and the treatments may cause a decreased quality of life in both patients and their parents. Another potential cause of decreased quality of life in ADA-SCID is the associated psychiatric and neurological problems caused by the disease. Researchers are interested in studying quality of life in individuals with ADA-SCID and their parents to provide more information about the disease.
Objectives:
- To evaluate whether gene therapy alters the quality of life or neuropsychiatric status of children with ADA-SCID.
- To monitor for intellectual, attention, memory, or specific learning disorders in children with ADA-SCID.
- To evaluate whether undergoing gene therapy has an effect on parenting stress of parents whose children have ADA-SCID.
Eligibility:
