Cystic FibrosisNCT04038710
Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.
An observational study of Cystic Fibrosis, run by National Jewish Health at 1 site in United States.
Trial summary
This is an observational study of people with severe cystic fibrosis that are eligible for Vertex's triple combination therapy through its expanded access. Pulmonary health, intestinal health, and the overall health of individuals will be tracked for a year to see how effective triple combination therapy is in these… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Triple combination therapy
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Sep 2019to 26 Feb 2021
- Phase
- Observational
- 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
Study population: Patients at National Jewish Health with Cystic Fibrosis who are eligible to enroll in Vertex's triple combination therapy expanded access program.
Who may be able to join
- Confirmed diagnosis of Cystic Fibrosis
- Ability to reproducibly perform spirometry (according to ATS criteria)
- Physician decision to treat with TCT through the EAP program
- Ability to understand and sign a written informed consent or assent and comply with the requirements of the study
Who cannot join
- History of hypersensitivity to VX 445, VX 659, tezacaftor and/or ivacaftor
- Any acute lower respiratory symptoms treated with oral, inhaled or IV antibiotics or systemic corticosteroids within the 2 weeks prior to Visit 1.
- Major or traumatic surgery within 12 weeks prior to Visit 1.
- Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit 1.
- Use of an investigational agent within 28 days prior to Visit 1.
- History of lung or liver transplantation or listing for organ transplantation.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Observational Study of the Effects on Clinical Outcomes of Expanded Access Program of Vertex Triple Combination Therapy.
This is an observational study of people with severe cystic fibrosis that are eligible for Vertex's triple combination therapy through its expanded access. Pulmonary health, intestinal health, and the overall health of individuals will be tracked for a year to see how effective triple combination therapy is in these people with severe cystic fibrosis disease.
- Conditions
- Cystic Fibrosis
- Being tested
- Triple combination therapy
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Triple combination therapyDrug
Triple combination is compromised of the drugs ivacaftor, tezacaftor and Vertex's next generation modulator.
| Study group | Role | What they receive |
|---|---|---|
| Patients with severe disease | Patients that are eligible to enroll in Vertex's triple combination therapy through the expanded access program. Receives: Drug: Triple combination therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
pulmonary function
FEV1 values.
Measured: For a year post initiation of therapy.
Secondary outcomes (1)
CFQ-R score
cystic fibrosis questionnaire - revised has sections to measure pulmonary health and gastrointestinal health.
Measured: For a year following the initiation of triple combination therapy.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2019
- Study start5 Sep 2019
- Primary completion26 Feb 2021
- Study completion26 Feb 2021
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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Jewish Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Derek Low, MD, Principal investigator, National Jewish Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04038710
- Sponsor's protocol ID
- HS-3235
- Status last verified
- Mar 2021
- First submitted
- 26 Jul 2019
- Last updated
- 11 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Study of the Effects on Clinical Outcomes of Expanded Access Program of Vertex Triple Combination Therapy.
- ClinicalTrials.gov ID
- NCT04038710
- Acronym / sponsor ID
- HS-3235
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- National Jewish HealthUniversity, hospital or other
- Study start
- 5 Sep 2019 (actual)
- Primary completion
- 26 Feb 2021 (actual)
- Study completion
- 26 Feb 2021 (actual)
- First posted
- 31 Jul 2019 (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.
This is an observational study of people with severe cystic fibrosis that are eligible for Vertex's triple combination therapy through its expanded access. Pulmonary health, intestinal health, and the overall health of individuals will be tracked for a year to see how effective triple combination therapy is in these people with severe cystic fibrosis disease.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with severe disease | – | Drug: Triple combination therapyPatients that are eligible to enroll in Vertex's triple combination therapy through the expanded access program. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients at National Jewish Health with Cystic Fibrosis who are eligible to enroll in Vertex's triple combination therapy expanded access program.
Key inclusion criteria (4 listed)
- Confirmed diagnosis of Cystic Fibrosis
- Ability to reproducibly perform spirometry (according to ATS criteria)
- Physician decision to treat with TCT through the EAP program
- Ability to understand and sign a written informed consent or assent and comply with the requirements of the study
Key exclusion criteria (6 listed)
- History of hypersensitivity to VX 445, VX 659, tezacaftor and/or ivacaftor
- Any acute lower respiratory symptoms treated with oral, inhaled or IV antibiotics or systemic corticosteroids within the 2 weeks prior to Visit 1.
- Major or traumatic surgery within 12 weeks prior to Visit 1.
- Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit 1.
- Use of an investigational agent within 28 days prior to Visit 1.
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 |
|---|---|---|
| Triple combination therapyTriple combination is compromised of the drugs ivacaftor, tezacaftor and Vertex's next generation modulator. | Drug | Patients with severe disease |
Primary outcome
pulmonary function
Time frame: For a year post initiation of therapy.
FEV1 values.
1 secondary outcome
CFQ-R score
Time frame: For a year following the initiation of triple combination therapy.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2021.
- Individual participant dataSharing planned: No
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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.
Derek Low, MD principal investigator
National Jewish Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by National Jewish Health at 1 site in United States.
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 4 inclusion and 6 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: finished, as of the sponsor's last update on 11 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
pulmonary function (time frame: For a year post initiation of therapy.). 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 11 Mar 2021.