Cystic FibrosisNCT01381289
VX-770 Expanded Access Program
A study testing VX-770 in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated.
Trial summary
The purpose of this expanded access program is to provide VX-770 prior to its commercial availability to people with cystic fibrosis (CF) who have at least one copy of the G551D-CFTR mutation and who are in critical medical need and who are not eligible for participation in other Vertex-sponsored studies. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- VX-770
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 6 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Male or female with confirmed diagnosis of CF, with a sweat chloride >60 mmol/L OR 2 CF-causing mutations AND chronic sinopulmonary disease OR gastrointestinal/nutritional abnormalities.
- Have the G551D-CFTR mutation in at least 1 allele
- Will be aged 6 years or older on the date of signed informed consent form
- Highest FEV1 in the 6 months prior to screening is ≤ 40% predicted value or patient is documented to be active on the lung transplant wait list
Who cannot join
- If female, currently pregnant
- Abnormal liver function, at screening on recent clinical laboratory testing, defined as >3 × upper limit of normal (ULN), of any 3 or more of the following: AST, ALT, GGT, serum alkaline phosphatase, total bilirubin
- Is currently requiring invasive mechanical ventilation
- Is currently participating, or has participated in the past 30 days in another therapeutic or clinical study
About this study
Official title: VX-770 Expanded Access Program (EAP)
The purpose of this expanded access program is to provide VX-770 prior to its commercial availability to people with cystic fibrosis (CF) who have at least one copy of the G551D-CFTR mutation and who are in critical medical need and who are not eligible for participation in other Vertex-sponsored studies.
Read the detailed description
VX-770, a compound being developed by Vertex Pharmaceuticals Incorporated (Vertex) for the treatment of CF, is an orally bioavailable small molecule that targets the underlying defect in CF, the dysfunctional CFTR protein. In Phase 3 studies of VX-770 in patients with CF and a G551D CFTR mutation, improvements in CFTR function (measured by reduction in sweat chloride concentration) and improvements in lung function were observed.
Patients who are interested in the VX-770 Expanded Access should contact their CF physician about participation.
Physicians interested in participating as a site should contact 800-745-4484.
- Conditions
- Cystic Fibrosis
- Being tested
- VX-770
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-770Drug
150 mg tablet, oral use, twice daily every 12 hours (q12h)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2011 (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.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01381289
- Sponsor's protocol ID
- VX11-770-901
- Status last verified
- Feb 2012
- First submitted
- 22 Jun 2011
- Last updated
- 9 Feb 2012
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- VX-770 Expanded Access Program (EAP)
- ClinicalTrials.gov ID
- NCT01381289
- Acronym / sponsor ID
- VX11-770-901
- Conditions
- Cystic Fibrosis
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- First posted
- 27 Jun 2011 (estimated)
- 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.
The purpose of this expanded access program is to provide VX-770 prior to its commercial availability to people with cystic fibrosis (CF) who have at least one copy of the G551D-CFTR mutation and who are in critical medical need and who are not eligible for participation in other Vertex-sponsored studies.
Detailed description
VX-770, a compound being developed by Vertex Pharmaceuticals Incorporated (Vertex) for the treatment of CF, is an orally bioavailable small molecule that targets the underlying defect in CF, the dysfunctional CFTR protein. In Phase 3 studies of VX-770 in patients with CF and a G551D CFTR mutation, improvements in CFTR function (measured by reduction in sweat chloride concentration) and improvements in lung function were observed.
Patients who are interested in the VX-770 Expanded Access should contact their CF physician about participation.
Physicians interested in participating as a site should contact 800-745-4484.
Study design
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male or female with confirmed diagnosis of CF, with a sweat chloride >60 mmol/L OR 2 CF-causing mutations AND chronic sinopulmonary disease OR gastrointestinal/nutritional abnormalities.
- Have the G551D-CFTR mutation in at least 1 allele
- Will be aged 6 years or older on the date of signed informed consent form
- Highest FEV1 in the 6 months prior to screening is ≤ 40% predicted value or patient is documented to be active on the lung transplant wait list
Key exclusion criteria (4 listed)
- If female, currently pregnant
- Abnormal liver function, at screening on recent clinical laboratory testing, defined as >3 × upper limit of normal (ULN), of any 3 or more of the following: AST, ALT, GGT, serum alkaline phosphatase, total bilirubin
- Is currently requiring invasive mechanical ventilation
- Is currently participating, or has participated in the past 30 days in another therapeutic or clinical study
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 |
|---|---|---|
| VX-770150 mg tablet, oral use, twice daily every 12 hours (q12h) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2012.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing VX-770 in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated.
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 4 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 9 Feb 2012. Status can change between updates, so confirm with the study contact before making plans.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
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 9 Feb 2012.