Cystic FibrosisNCT04215796
Evaluation of the ReX-C System, in Patients Receiving CFTR Modulators for the Treatment of Cystic Fibrosis (CF).
A study testing ReX-C system in people with Cystic Fibrosis and Adherence, Medication, run by Dosentrx Ltd. at 2 sites in Israel.
Trial 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… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis, Adherence, Medication
- What is being tested?
- ReX-C system
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesIsrael
- Study dates
- 12 May 2020to 31 Jul 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
- -
Trial locations and contacts
2 sites in 1 country.
Israel2 sites, 2 recruiting
- Recruiting
Carmel Medical Center
Haifa
Einat Ben Shoshan, +972 54-447-8092, EinatBe2@clalit.org.il
- Recruiting
Hadassah Medical Organization
Jerusalem, Select One
Batya Nahmani, +972 54-788-8165, hbatya@hadassah.org.il
David Shoseyov, MD
About this study
Official title: Evaluation of the Safety, Usability and Efficacy of the ReX-C® System in Medication Management, in Patients Receiving CFTR Modulators (Kalydeco, Orkambi and/or Symdeko) for the Treatment of Cystic Fibrosis (CF).
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.
Read the detailed 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.
- Conditions
- Cystic Fibrosis, Adherence, Medication
- Being tested
- ReX-C system
- Medical subject headings
- Cystic Fibrosis, Medication Adherence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ReX-C systemDevice
CFTR modulators are dispensed to patients via ReX-C system
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Device: ReX-C system |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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:
- 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.
Measured: 12 months
Rate of adherence (number of pill intakes) during treatment with ReX-C
- Adherence rate when using the ReX-C system is > 80%.
- 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
Measured: 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
Improvement of 10-20% in the following parameters, compared between period of ReX intervention versus historical period when same medication was taken in the conventional way.
- Reduced # of acute exacerbation B. Improved PFT C. Reduced hospitalization events D. Decreased antibiotic treatment events
Measured: 24 months (12 months historical data, before study initiation), 12 months ReX intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jan 2020
- Study start12 May 2020
- Primary completion30 Jun 2022 (estimated)
- Study completion31 Jul 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dosentrx Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Shoseyyov, MD, Principal investigator, Hadassah Medical Center, Pediatric department, Jerusalem, IsraelGalit Livnat-Levanon, MD, Principal investigator, Pediatric unit, Lady Davis Carmel Medical Center, Haifa, Israel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04215796
- Sponsor's protocol ID
- RXC-054-2019-CLE
- Status last verified
- Sep 2021
- First submitted
- 29 Dec 2019
- Last updated
- 23 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Safety, Usability and Efficacy of the ReX-C® System in Medication Management, in Patients Receiving CFTR Modulators (Kalydeco, Orkambi and/or Symdeko) for the Treatment of Cystic Fibrosis (CF).
- ClinicalTrials.gov ID
- NCT04215796
- Acronym / sponsor ID
- RXC-054-2019-CLE
- Conditions
- Cystic Fibrosis, Adherence, Medication
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Dosentrx Ltd.Industry (pharma, biotech, devices)
- Study start
- 12 May 2020 (actual)
- Primary completion
- 30 Jun 2022 (estimated)
- Study completion
- 31 Jul 2022 (estimated)
- First posted
- 2 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| ReX-C intervention | Experimental | Device: ReX-C systemSubjects 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ReX-C systemCFTR modulators are dispensed to patients via ReX-C system | Device | ReX-C intervention |
Primary outcome
Number of safety events during administration of oral oncolytic medications by ReX-C
Time frame: 12 months
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.
Rate of subjects feel comfortable to use ReX-C for pill intake and medication managment and define the system as "Easy to use"
Time frame: 12 months
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.
Rate of adherence (number of pill intakes) during treatment with ReX-C
Time frame: 12 months
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
1 secondary outcome
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
Time frame: 24 months (12 months historical data, before study initiation), 12 months ReX intervention
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Shoseyyov, MD principal investigator
Hadassah Medical Center, Pediatric department, Jerusalem, Israel
Galit Livnat-Levanon, MD principal investigator
Pediatric unit, Lady Davis Carmel Medical Center, Haifa, Israel
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ReX-C system in people with Cystic Fibrosis and Adherence, Medication, run by Dosentrx Ltd. at 2 sites in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ronit Shtrichman, Ph.D (+972526500938, ronit.s@dosentrx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of safety events during administration of oral oncolytic medications by ReX-C (time frame: 12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2021.