Personalized Theratyping Trial
NCT03587961 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 20
Last updated 2026-02-02
Summary
The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.
Conditions
Interventions
- DRUG
-
Symdeko
explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.
Sponsors & Collaborators
-
George Solomon
lead OTHER
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- PARALLEL
Eligibility
- Min Age
- 6 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2019-08-01
- Primary Completion
- 2027-01-01
- Completion
- 2027-09-01
- FDA Drug
- Yes
Countries
- United States
Study Locations
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