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Trial record 3 of 277864 for:    ALL

Personalized Theratyping Trial

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ClinicalTrials.gov Identifier: NCT03587961
Recruitment Status : Not yet recruiting
First Posted : July 16, 2018
Last Update Posted : July 16, 2018
Sponsor:
Information provided by (Responsible Party):
George Solomon, University of Alabama at Birmingham

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Symdeko Early Phase 1

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Personalized Theratyping Trial
Estimated Study Start Date : August 1, 2018
Estimated Primary Completion Date : August 1, 2020
Estimated Study Completion Date : August 1, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Ivacaftor

Arm Intervention/treatment
Experimental: mutation that responds to a CFTR corrector
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
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.
Other Names:
  • Orkambi
  • Ivacaftor

Experimental: response to a potentiator of CFTR function
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
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.
Other Names:
  • Orkambi
  • Ivacaftor

Experimental: the patient is 6-12 years old
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.
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.
Other Names:
  • Orkambi
  • Ivacaftor




Primary Outcome Measures :
  1. Lung function [ Time Frame: 32 weeks ]
    change in lung function



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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03587961


Contacts
Contact: Heather Hathorne, PhD 205-638-9568 hhathorne@peds.uab.edu

Locations
United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Sponsors and Collaborators
University of Alabama at Birmingham

Responsible Party: George Solomon, MD, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT03587961     History of Changes
Other Study ID Numbers: IRB-300001867
First Posted: July 16, 2018    Key Record Dates
Last Update Posted: July 16, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Additional relevant MeSH terms:
Cystic Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action