Personalized Theratyping Trial

NCT ID: NCT03587961

Last Updated: 2026-02-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

EARLY_PHASE1

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-08-01

Study Completion Date

2027-09-01

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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

Group Type EXPERIMENTAL

Symdeko

Intervention Type 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.

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.

Group Type EXPERIMENTAL

Symdeko

Intervention Type 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.

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

Group Type EXPERIMENTAL

Symdeko

Intervention Type 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.

Interventions

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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.

Intervention Type DRUG

Other Intervention Names

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Orkambi Ivacaftor

Eligibility Criteria

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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
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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George Solomon

OTHER

Sponsor Role lead

Responsible Party

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George Solomon

Principal Investigator

Responsibility Role SPONSOR_INVESTIGATOR

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Heather Hathorne, PhD

Role: CONTACT

205-638-9568

Facility Contacts

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Heather Hathorne, PhD

Role: primary

205-638-9568

Ginger Reeves, BS

Role: backup

205-638-5970

Other Identifiers

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IMPACT-Solomon

Identifier Type: OTHER

Identifier Source: secondary_id

IRB-300001867

Identifier Type: -

Identifier Source: org_study_id

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