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Personalized Theratyping Trial

Primary Purpose

Cystic Fibrosis

Status

Recruiting

Phase

Early Phase 1

Locations

United States

Study Type

Interventional

Intervention

Symdeko

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

University of Alabama at BirminghamRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Symdeko

Ivacaftor

Orkambi

Arm Description

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

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.

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

Outcomes

Primary Outcome Measures

spirometry

change in lung function as measured via spirometry

Secondary Outcome Measures

Full Information

NCT ID

NCT03587961

First Posted

July 3, 2018

Last Updated

September 8, 2023

Sponsor

University of Alabama at Birmingham

1. Study Identification

Unique Protocol Identification Number

NCT03587961

Brief Title

Personalized Theratyping Trial

Official Title

Personalized Theratyping Trial

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Recruiting

Study Start Date

August 1, 2019 (Actual)

Primary Completion Date

September 1, 2024 (Anticipated)

Study Completion Date

January 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

University of Alabama at Birmingham

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Early Phase 1

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Symdeko

Arm Type

Experimental

Arm Description

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

Arm Title

Ivacaftor

Arm Type

Experimental

Arm Description

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.

Arm Title

Orkambi

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Symdeko

Other Intervention Name(s)

Orkambi, Ivacaftor

Intervention Description

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.

Primary Outcome Measure Information:

Title

spirometry

Description

change in lung function as measured via spirometry

Time Frame

32 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Heather Hathorne, PhD

Phone

205-638-9568

hhathorne@peds.uab.edu

Facility Information:

Facility Name

University of Alabama at Birmingham

City

Birmingham

State/Province

Alabama

ZIP/Postal Code

35233

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Heather Hathorne, PhD

Phone

205-638-9568

hhathorne@peds.uab.edu

First Name & Middle Initial & Last Name & Degree

Ginger Reeves, BS

Phone

205-638-5970

greeves@peds.uab.edu

First Name & Middle Initial & Last Name & Degree

George Solomon, MD

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Personalized Theratyping Trial

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