Personalized Theratyping Trial

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
George Solomon
Study ID
NCT03587961
Phase
EARLY_PHASE1
Status
Recruiting
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Conditions

  • Cystic Fibrosis

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Symdeko — DRUG
    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.

Study Details

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.

Key Dates

Start date
Aug 1, 2019
Status verified
Jan 2026
Primary completion
Jan 1, 2027
Completion
Sep 1, 2027

Study Design

Enrollment
20 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Symdeko
    Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, depending on the in vitro response pattern
  • Experimental: Ivacaftor
    Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy.. Patients with a mutation equivalent to wild type will be given Ivacaftor.
  • Experimental: Orkambi
    Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Orkambi, depending on the in vitro response pattern

Primary Outcome Measure

spirometry [ Time Frame: 32 weeks ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of Alabama at BirminghamBirminghamAlabama35233
Heather Hathorne, PhD
[email protected]
205-638-9568
Ginger Reeves, BS
[email protected]
205-638-5970
George Solomon, MD (PRINCIPAL_INVESTIGATOR)

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By research site
University of Alabama at Birmingham· Birmingham, AL

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