Natural History Study of SCID Disorders

This study is looking for children diagnosed with Severe Combined Immunodeficiency (SCID) or similar conditions like Leaky SCID, Omenn Syndrome, or Reticular Dysgenesis, who are planning to have treatments such as a bone marrow transplant, enzyme therapy, or gene therapy.

To join, a child must have specific blood test results that show very low numbers of certain immune cells (T cells) or very poor T cell function, which are hallmarks of SCID. For some types of SCID, researchers will also look for particular genetic changes, but the main focus is on the immune cell counts and their ability to fight off disease.

Essentially, if your child has been diagnosed with a type of SCID where their immune system is severely compromised and they are about to receive, or have recently received, one of the standard treatments for SCID, they might be able to participate. The study is open to children of all ages and genders.

If your child joins this study, they will be observed closely over time. This means doctors will collect information from their regular medical appointments and treatments related to their SCID. They will keep track of how your child's immune system responds after treatment, how they are growing and developing, and their general quality of life.

The study doesn't involve any extra tests or medications that your child wouldn't already be getting as part of their standard medical care. Instead, it uses information from those routine check-ups and procedures. There are no experimental treatments being given within this study. It's about collecting existing data on your child's health journey according to a set schedule, both before and after their treatment for SCID, for a period determined by the study protocol. The overall duration of your child's participation will depend on the follow-up schedule required to gather all the necessary information.