NCT04215796

Brief Summary

Cystic fibrosis (CF) is a progressive multisystem disorder characterized by abnormalities in the transport of chloride ions in human airway epithelial cells, leading to frequent lung infections, decreased pulmonary function, inability to properly digest food and absorb essential nutrients, and complications with many organs. Patients with CF spend hours daily, in treatments required to manage their disease, including hours of physiotherapy and inhalation and treatment with many daily pills. CF treatment load heavy burden on patients and families and the inevitable consequence of these treatment demands is widespread non-adherence to therapy. CFTR modulators (trade name Kalydeco, Orkambi, Simdeco) is a highly efficient drug approved to treat CF in patients with certain mutations. It is the first drug that treats the underlying cause rather than the symptoms of the disease. It is also one of the most expensive drugs, costing over $300,000 per patient per year. Despite of its proven efficacy and approved reimbursement for certain patients, non-adherence is common among CF patients, resulting from the heavy burden of daily treatment required to manage CF disease.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started May 2020

Typical duration for not_applicable

Geographic Reach
1 country

2 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 29, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

January 2, 2020

Completed
4 months until next milestone

Study Start

First participant enrolled

May 12, 2020

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2022

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

July 31, 2022

Completed
Last Updated

September 23, 2021

Status Verified

September 1, 2021

Enrollment Period

2.1 years

First QC Date

December 29, 2019

Last Update Submit

September 22, 2021

Conditions

Outcome Measures

Primary Outcomes (3)

  • Number of safety events during administration of oral oncolytic medications by ReX-C

    Safety events include: pill overdose, pill inhalation during pill ingestion, pill malformation upon dispensing and any severe adverse events related to pill intake by the ReX-C device.

    12 months

  • Rate of subjects feel comfortable to use ReX-C for pill intake and medication managment and define the system as "Easy to use"

    At least 70% of subjects: A. Complete at least 6 months with Rex-C. B. Define the system as easy to use and are willing to continue using it for their medication treatment.

    12 months

  • Rate of adherence (number of pill intakes) during treatment with ReX-C

    A. Adherence rate when using the ReX-C system is \> 80%. B. Adherence rate is enhanced by the following measures: * At least 80% of missed/ delayed doses recorded by ReX-C are subsequently taken by subjects following real-time reminder communication. * Subject's self-report by adherence questionnaire

    12 months

Secondary Outcomes (1)

  • Rate of improvement in patients health condition when using ReX-C compared with historical data of the same subject, taking identical CFTR modulator in the conventional way

    24 months (12 months historical data, before study initiation), 12 months ReX intervention

Study Arms (1)

ReX-C intervention

EXPERIMENTAL

Subjects use ReX-C to receive CFTR modulators medications. Adherence data, side effects and response to treatment are monitored online in real time via ReX-C cloud.

Device: ReX-C system

Interventions

CFTR modulators are dispensed to patients via ReX-C system

ReX-C intervention

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Male or Female, at least 12 years of age
  • Subject is able to swallow pills and during training successfully achieves 2 administrations of "demo" pills (Tic-Tac sweets) by ReX-C.
  • Subject is able to read and understand the Informed Consent Form.
  • Subject was diagnosed with CF and receives CFTR modulators medication.
  • Subject is intended to receive Kalydeco OR Orkambi OR Symdeko during the 12 months study period.
  • Subject takes medication therapy at home.
  • Subject is fluent in one of the following languages: Hebrew, Arabic.

You may not qualify if:

  • Subject has significant physical disability including; poor fine motor skills, impaired visual or auditory faculties, mental disorders or other impairment affecting ability to provide Informed Consent or use the ReX-C dispensing unit effectively.
  • Subject failed to extract 2 "demo" pills during ReX-C training.
  • Subject is participating in another clinical study that does not permit participation in two studies simultaneously.
  • Subject is at end stage or terminal illness with anticipated life expectancy of 6 months or less.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Hadassah Medical Organization

Jerusalem, Select One, Israel

RECRUITING

Carmel Medical Center

Haifa, Israel

RECRUITING

MeSH Terms

Conditions

Cystic FibrosisMedication Adherence

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesPatient CompliancePatient Acceptance of Health CareTreatment Adherence and ComplianceHealth BehaviorBehavior

Study Officials

  • David Shoseyyov, MD

    Hadassah Medical Center, Pediatric department, Jerusalem, Israel

    PRINCIPAL INVESTIGATOR
  • Galit Livnat-Levanon, MD

    Pediatric unit, Lady Davis Carmel Medical Center, Haifa, Israel

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Ronit Shtrichman, Ph.D

CONTACT

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Open study for subjects receiving home-based medication: CFTR modulators (Kalydeco, Orkambi, Simdeko) treatment. Subjects receive these medication by ReX-C device. Medication adherence, treatment instructions and side effects are recorded and monitored online in real time during the Study, by the ReX-C system.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 29, 2019

First Posted

January 2, 2020

Study Start

May 12, 2020

Primary Completion

June 30, 2022

Study Completion

July 31, 2022

Last Updated

September 23, 2021

Record last verified: 2021-09

Locations