Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06504589
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-06-27
Brief Title:
A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The REACH study is for people with CF who do not take cystic fibrosis transmembrane conductance regulator CFTR modulators The goal of the REACH study is to collect research data including health data and specimens from people with CF who do not take CFTR modulators This data may be used to inform CF research help design CF clinical trials and support the development of new treatments for people with CF who do not take CFTR modulators
Another goal of this study is to learn about research involvement for people with CF who do not take CFTR modulators engage them in research and give them an opportunity to learn about what is involved in participating in a CF research study
Another goal of this study is to learn about research involvement for people with CF who do not take CFTR modulators engage them in research and give them an opportunity to learn about what is involved in participating in a CF research study
Detailed Description:
People with Cystic Fibrosis pwCF who are genetically ineligible andor not taking cystic fibrosis transmembrane conductance regulator CFTR modulators currently face future health that is considerably different from the approximately 90 of pwCF in the United States who benefit from CFTR modulators New treatments are being advanced for pwCF who are genetically ineligible or not taking CFTR modulators and these therapies will include both nucleic acid-based therapies NABTs to address the underlying defect in CFTR and symptomatic therapies aimed at targeting the symptoms of CF A key concern for this limited and underserved patient population is being able to advance multiple therapeutic programs in parallel To complete these studies CF researchers will need to be able to reach this community effectively while also promoting the use of innovative trial designs
The REACH Study is a prospective longitudinal observational research study to obtain research quality ie monitored research CF outcome data Primary outcome endpoints of the Core study collected across all study participants are aligned with anticipated clinical trial outcome endpoints needed in overall development of therapies for the CF population unable to benefit from CFTR modulators This study will also include sub-studies to obtain specialized measures which may help inform efficacy and safety evaluations of new therapies by providing CF control data Finally this study also seeks to assess research solicitation and research participation for the CF population that is modulator ineligible or not taking modulators The observational data collected within this study may be used in characterizing this CF population developing innovative trial designs for comparison when evaluating new or experimental CF therapies andor in CF research
The REACH Study is a prospective longitudinal observational research study to obtain research quality ie monitored research CF outcome data Primary outcome endpoints of the Core study collected across all study participants are aligned with anticipated clinical trial outcome endpoints needed in overall development of therapies for the CF population unable to benefit from CFTR modulators This study will also include sub-studies to obtain specialized measures which may help inform efficacy and safety evaluations of new therapies by providing CF control data Finally this study also seeks to assess research solicitation and research participation for the CF population that is modulator ineligible or not taking modulators The observational data collected within this study may be used in characterizing this CF population developing innovative trial designs for comparison when evaluating new or experimental CF therapies andor in CF research
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