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Personalized Theratyping Trial

Sponsor
University of Alabama at Birmingham (Other)
Overall Status
Recruiting
CT.gov ID
NCT03587961
Collaborator
(none)
20
Enrollment
1
Location
3
Arms
41
Anticipated Duration (Months)
0.5
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
Early Phase 1

Detailed Description

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. Symdeko (Tezacaftor/Ivacaftor), Orkambi (Ivacaftor and Lumacaftor), correctors of CFTR misfolding and Kalydeco (Ivacaftor), a potentiator of abnormal CFTR gating, will be explored as a treatment for patients with other CF mutations than those currently approved. Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, or Orkambi, depending on the in vitro response pattern. 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. If the patient is 6-12 years old, we will only study Orkambi or ivacaftor as symdeko is not yet FDA approved in this patient population.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
20 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Personalized Theratyping Trial
Actual Study Start Date :
Aug 1, 2019
Anticipated Primary Completion Date :
Sep 1, 2022
Anticipated Study Completion Date :
Jan 1, 2023

Arms and Interventions

Arm Intervention/Treatment
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

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.
Other Names:
  • Orkambi
  • Ivacaftor

    		•
    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.

    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.
    Other Names:
  • Orkambi
  • 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

    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.
    Other Names:
  • Orkambi
  • Ivacaftor

    		•

    Outcome Measures

    Primary Outcome Measures

    1. spirometry [32 weeks]

      change in lung function as measured via spirometry

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Diagnosis of CF

    • Age ≥6 y.o.

    • CFTR mutation that may respond to approved correctors/potentiators in the opinion of the study investigators

    • Informed Consent/Assent

    • Stable CF pulmonary regimen

    Exclusion Criteria:

    • Exacerbation requiring antibiotic or steroids for >28 days before trial entry

    • Ongoing participation in a CFTR modulator study

    • Active smoking in the past 6 months

    • History of solid organ transplant

    • Any condition which precludes the use of CFTR modulators: e.g. advanced cirrhosis, End-stage Renal Disease (ESRD)

    • Any condition that precludes the patient from participation in the opinion of the investigator

    • Any meds that have significant drug-drug interactions or any other off label use of CFTR modulators

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Alabama at Birmingham Birmingham Alabama United States 35233

    Sponsors and Collaborators

    • University of Alabama at Birmingham

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    George Solomon, Principal Investigator, University of Alabama at Birmingham
    ClinicalTrials.gov Identifier:
    NCT03587961
    Other Study ID Numbers:
    • IRB-300001867
    First Posted:
    Jul 16, 2018
    Last Update Posted:
    Sep 24, 2021
    Last Verified:
    Sep 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Product Manufactured in and Exported from the U.S.:
    No
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Ivacaftor

    Study Results

    No Results Posted as of Sep 24, 2021