RDCRN PIDTC
M
Miriam Kim, DO
Primary Investigator
Enrolling By Invitation
17 years and younger
All
Phase
N/A
12 participants needed
1 Location
Brief description of study
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 of study
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
You may be eligible for this study if you meet the following criteria:
- Conditions: SCID; infection disease, cancer, Riley
-
Age: - 17 Years
-
Gender: All
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.
Updated on
13 Sep 2024.
Study ID: 16964; PHO-PIDTC-RDCRN-SCID
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