Rdcrn Pidtc

MK

Miriam Kim, DO

Principal Investigator

Status: Enrolling By Invitation Ages: 17 Years Gender: All Genders Phase: N/A 1 Locations

Brief Description

In this prospective and longitudinal natural history study, the aim is to identify variables that affect long-term outcomes following potentially life-saving Definitive Therapy (DT), which includes either 1) allogeneic hematopoietic cell transplant (HCT), or 2) autologous gene therapy (GT).

Detailed Description

This protocol is a prospective natural history study in which investigators at over 40 centers caring for patients with SCID Disorders in North America will participate. Investigators will: 1) evaluate and treat patients with new suspected or confirmed SCID Disorders according to their institutional practice and protocols, and also 2) enroll the same patients as subjects in this prospective natural history protocol. Thus, subjects will receive comparable baseline and follow up evaluations across all transplant centers, irrespective of the transplant (or other treatment) strategy and methods used at an individual site. 

Eligibility of study

Inclusion Criteria:
  • There will be four (4) cohorts: Cohort 1 will consist of suspected SCID; Cohort 2 will consist of confirmed SCID, defined as one of: a) Typical SCID, b) Leaky SCID, or c) Omenn syndrome; Cohort 3 will consist of confirmed SCID previously enrolled on PIDTC Protocols #6901 or #6902, and having undergone HCT or GT; Cohort 4 will consist of confirmed SCID not previously enrolled on PIDTC Protocols #6901 or #6902, and having undergone HCT or GT. 
Exclusion Criteria:
Patients with other primary immunodeficiency diseases who do not meet the strict entry criteria or patients with secondary immunodeficiency or HIV infection will be excluded from this study. 

Interested in participating?

Enrollment in IU School of Medicine clinical trials is managed through our All IN for Health program. Many studies only accept people who are part of an invited group. Visit allinforhealth.info to connect with the study team, or contact your doctor to see if you are eligible.