Personalized Theratyping Trial

NCT03587961 | Recruiting Early Phase 1 FDA Drug

• 2 other identifiers: IRB-300001867IMPACT-Solomon
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• spirometry

  change in lung function as measured via spirometry

  32 weeks

Study Arms (3)

Symdeko

EXPERIMENTAL

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

Ivacaftor

EXPERIMENTAL

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

Orkambi

EXPERIMENTAL

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

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.

Also known as: Orkambi, Ivacaftor

Ivacaftor; Orkambi; Symdeko

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• George Solomon: lead

Study Sites (1)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

tezacaftor, ivacaftor drug combination; lumacaftor, ivacaftor drug combination; ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Central Study Contacts

Heather Hathorne, PhD

CONTACT

205-638-9568; hhathorne@peds.uab.edu

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 3, 2018
• First Posted: July 16, 2018
• Study Start: August 1, 2019
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: February 2, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)