Ignite Creation Date:
2024-06-16 @ 11:48 AM
Last Modification Date:
2024-10-26 @ 3:30 PM
Study NCT ID:
NCT06435000
Status:
RECRUITING
Last Update Posted:
2024-07-03
First Post:
2024-05-24
Brief Title:
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 STGD1 Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
Sponsor:
Splice Bio
Organization:
Splice Bio
Study Overview
Official Title:
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 STGD1 Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily a Member 4 ABCA4 Gene
Status:
RECRUITING
Status Verified Date:
2024-09
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:
POLARIS
Brief Summary:
This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 STGD1 Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
This is a multicenter study which will enroll approximately 75 subjects
This is a multicenter study which will enroll approximately 75 subjects
Detailed Description:
Comprehensive knowledge of a disease is essential to the design and conduct of well-controlled interventional clinical trials Understanding of the disease state is important for identifying the patient population for a clinical trial study duration and selection of clinically meaningful endpoints
Observational studies play an important role in the understanding of rare diseases and facilitating effective development of potential therapies To support clinical research observational studies can help define the clinical features of a rare disease rate of progression pathophysiology and other important factors Further following the course of a disease over time allows investigators to identify demographic variables genotypic and phenotypic features and other characteristics that may correlate with disease and outcomes in the absence of treatment Thus observational studies are useful in guiding the design of therapeutic studies including selection of the patient population trial duration and the types of outcome measures to evaluate efficacy and safety
Results of a natural history study evaluating the progression of atrophy secondary to Stargardt Disease have been published using retrospective and prospective cohorts of patients ProgStar Strauss et al 2016
In summary the current study is a prospective observational study of patients with STGD1 the aim of which is to further enhance understanding of disease progression and structural and functional markers that can be used to evaluate the efficacy and safety of therapeutic interventions especially in light of advancements in imaging technology
Observational studies play an important role in the understanding of rare diseases and facilitating effective development of potential therapies To support clinical research observational studies can help define the clinical features of a rare disease rate of progression pathophysiology and other important factors Further following the course of a disease over time allows investigators to identify demographic variables genotypic and phenotypic features and other characteristics that may correlate with disease and outcomes in the absence of treatment Thus observational studies are useful in guiding the design of therapeutic studies including selection of the patient population trial duration and the types of outcome measures to evaluate efficacy and safety
Results of a natural history study evaluating the progression of atrophy secondary to Stargardt Disease have been published using retrospective and prospective cohorts of patients ProgStar Strauss et al 2016
In summary the current study is a prospective observational study of patients with STGD1 the aim of which is to further enhance understanding of disease progression and structural and functional markers that can be used to evaluate the efficacy and safety of therapeutic interventions especially in light of advancements in imaging technology
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None