Personalized Theratyping Trial

George Solomon

Status and phase: Enrolling; Early Phase 1

Conditions: Cystic Fibrosis

Treatments: Drug: Symdeko

Study type: Interventional

Funder types: Other

Identifiers

NCT03587961

IRB-300001867

IMPACT-Solomon (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 20 estimated patients

Sex: All

Ages: 6+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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20 participants in 3 patient groups

Symdeko

Experimental group

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

Treatment:

Drug: Symdeko

Ivacaftor

Experimental group

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.

Treatment:

Drug: Symdeko

Orkambi

Experimental group

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

Treatment:

Drug: Symdeko