Ignite Creation Date:
2024-05-05 @ 10:46 PM
Last Modification Date:
2024-10-26 @ 10:24 AM
Study NCT ID:
NCT01186913
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2020-11-10
First Post:
2010-08-20
Brief Title:
Natural History Study of SCID Disorders
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institute of Allergy and Infectious Diseases NIAID
Study Overview
Official Title:
A Prospective Natural History Study of Diagnosis Treatment and Outcomes of Children With SCID Disorders RDCRN PIDTC-6901
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2020-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is a prospective evaluation of children with Severe Combined Immune Deficiency SCID who are treated under a variety of protocols used by participating institutions In order to determine the patient recipient and transplant-related variables that are most important in determining outcome study investigators will uniformly collect pre- post- and peri-transplant or other treatment information on all children enrolled into this study
Children will be divided into three strata
Stratum A Typical SCID with virtual absence of autologous T cells and poor T cell function
Stratum B Atypical SCID leaky SCID Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function and
Stratum C ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy
Each GroupCohort Stratum will be analyzed separately
Children will be divided into three strata
Stratum A Typical SCID with virtual absence of autologous T cells and poor T cell function
Stratum B Atypical SCID leaky SCID Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function and
Stratum C ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy
Each GroupCohort Stratum will be analyzed separately
Detailed Description:
This study follows participants with SCID prospectively meaning the study enrolls participants where there is a plan to receive a blood and marrow transplant enzyme therapy or gene therapy in the future Participants are then followed according to a schedule set out by the study protocol after the procedure There are no experimental therapies on this study
The goal of this study is to learn more about 1 outcomes from the treatment of SCID in the modern era of medicine 2 what factors lead to the best long-term outcomes such as best donor conditioning regimen timing of transplant etc and 3 what impact newborn screening and the early diagnosis of SCID has had on the long-term outcomes following BMT or gene therapy Information is also being gathered on how and when the immune system recovers after bone marrow transplant BMT quality of life for long-term survivors and about whether children develop normally after treatment
This natural history study is the largest coordinated prospective study of participants with SCID ever performed Information that investigators will learn both now and in the future will help doctors and other health professionals to better treat children with SCID
The goal of this study is to learn more about 1 outcomes from the treatment of SCID in the modern era of medicine 2 what factors lead to the best long-term outcomes such as best donor conditioning regimen timing of transplant etc and 3 what impact newborn screening and the early diagnosis of SCID has had on the long-term outcomes following BMT or gene therapy Information is also being gathered on how and when the immune system recovers after bone marrow transplant BMT quality of life for long-term survivors and about whether children develop normally after treatment
This natural history study is the largest coordinated prospective study of participants with SCID ever performed Information that investigators will learn both now and in the future will help doctors and other health professionals to better treat children with SCID
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None