Personalized Theratyping Trial

Cystic Fibrosis Clinical Trial

Official title:

Personalized Theratyping Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03587961
• Other study ID #: IRB-300001867
• Status: Recruiting
• Phase: Early Phase 1
• Start date: August 1, 2019
• Est. completion date: January 1, 2025

Study information

• Verified date: September 2023
• Source: University of Alabama at Birmingham
• Contact: Heather Hathorne, PhD
• Phone: 205-638-9568
• Email: hhathorne[@]peds.uab.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: January 1, 2025
• Est. primary completion date: September 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis

Intervention

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.

Locations

Country	Name	City	State
United States	University of Alabama at Birmingham	Birmingham	Alabama

Sponsors (1)

Lead Sponsor	Collaborator
University of Alabama at Birmingham

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	spirometry	change in lung function as measured via spirometry	32 weeks