Evaluation of the ReX-C System, in Patients Receiving CFTR Modulators for the Treatment of Cystic Fibrosis (CF).

Dosentrx

Status: Unknown

Conditions: Adherence, Medication; Cystic Fibrosis

Treatments: Device: ReX-C system

Study type: Interventional

Funder types: Industry

Identifiers

NCT04215796

RXC-054-2019-CLE

Details and patient eligibility

About

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.

Full description

ReX is a hand-held, mobile device intended to provide solid oral medication on patient's demand according to a pre-programmed treatment protocol. ReX addresses poor patient adherence, tracks patient's response to treatment and enhances patient engagement to therapy. The system comprises a reusable drug dispensing unit (Dispenser), a disposable Cassette containing the prescribed medication, a cellphone app, and the ReX cloud. The Dispenser manages pill administration. It includes a touch screen which guides the user and presents patient-specific clinical surveys and therapy information. The Dispenser contains a chargeable battery and indicators demonstrating device and battery status; a pill window enabling pills to be viewed; operational sensors; and communication to an app on cellphones. The patient receives the Cassette from the pharmacy, pre-filled with his/her specific medication. The Cassette is inserted into the Dispenser where it is locked in place. All therapy and patient survey data are transferred to a patient-specific domain on DosentRx' proprietary web-based cloud, named ReX cloud.

Enrollment: 10 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

1. 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.

2. Subject failed to extract 2 "demo" pills during ReX-C training.

3. Subject is participating in another clinical study that does not permit participation in two studies simultaneously.

4. Subject is at end stage or terminal illness with anticipated life expectancy of 6 months or less.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

ReX-C intervention

Experimental group

Description:

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.

Treatment:

Device: ReX-C system