Your donation will make a difference:
Cystic Fibrosis Trust

Please Log in

Log in

Not a registered user? Register with us

This trial will be added to your watchlist.

Do you want to receive email notifications when the trial status changes?

No Yes

Unfollowing a trial will remove it from the watchlist and stop status change email notifications

Are you sure you want to unfollow this trial?

Yes, remove from watchlist

VERTPh1D

Details

Therapeutic category
The type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise

Restore CFTR Function

Trial status

Closed - follow up complete

Phase
The different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use

Phase I

Full title

A Phase 1, Randomized, Double-blind, Placebo-controlled, Dose Escalation, and Bioavailability Study Evaluating the Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Subjects With Cystic Fibrosis

Cystic fibrosis (CF) is an autosomal recessive chronic disease with serious morbidities and frequent premature mortality. At present, there is no cure. CF affects approximately 70,000 individuals worldwide The aims of the study is to evaluate the safety and tolerability of VX-659 in triple combination with TEZ/IVA for 14 days in subjects with CF This is a randomized, double-blind, placebo-controlled, single- and multiple-dose, first-in-human dose escalation study of VX-659 that includes evaluations of relative and absolute bioavailability. Part D (Subjects with CF) Part D has a randomized, double-blind, placebo-controlled, parallel-group design. Approximately 12 subjects will be enrolled and randomized 3:1 to TC:placebo. Subjects will receive 14 days of treatment. Part D may be initiated while Parts A, B, and C are ongoing after review of safety, tolerability, and available PK data. The dose of VX-659 will be at least 1 dose level below the highest Part C dose for which safety and tolerability results are available and supportive. The dosage of TEZ/IVA will be TEZ 100 mg qd/IVA 150 mg q12h, which will be administered as TEZ 100-mg/IVA 150-mg FDC in the morning and IVA 150 mg in the evening. The decision to initiate successive cohorts and dose selection will be based on safety and tolerability data from preceding dose group(s) and available PK data from a minimum of 6 subjects (to ensure 4 subjects receiving active drug) in the preceding cohort.

Trial type

Medication

Length of participation
The length of time a participant will take part in a trial, from the first to the last appointment

6 months

Intervention
The name of the treatment or therapy being researched

VX-659, Tezacaftor, Ivacaftor or Matching Placebos

Recruitment target
The number of participants who need to be recruited for the trial in the UK

12

Last edited date

04 September 2017

Sponsor

Vertex Pharmaceuticals Incorporated

Sponsor type

Commercial

Who can take part?

Mutation

One copy of F508del

Age

18+

Top inclusion criteria
  • Heterozygous for F508del and a minimal function CFTR mutation
  • Subjects must have an forced expiratory volume in 1 second (FEV1) ≥ 40% and ≤ 90% of predicted normal for age, sex, and height
  • Sweat chloride value ≥ 60 mmol/L at screening
Top exclusion criteria
  • History of cirrhosis with portal hypertension
  • Risk factors for Torsade de Pointes
  • History of hemolysis

CF centres running this trial

Closed

Heart of England NHS Foundation Trust

Address

Birmingham Heartlands Hospital Bordesley Green East Birmingham West Midlands B9 5SS

Recruitment starts

March 2017

Recruitment ends

June 2017

Contact

Nash, Edward

Get in touch

Closed

Liverpool Heart and Chest Hospitals NHS Foundation Trust

Address

Thomas Drive Liverpool Merseyside L14 3PE

Recruitment starts

March 2017

Recruitment ends

July 2017

Contact

Ledson, Martin

Get in touch

Closed

Papworth Hospital NHS Foundation Trust

Address

Papworth Hall Papworth Everard Cambridge Cambridgeshire CB23 3RE

Recruitment starts

March 2017

Contact

Haworth, Charles

Get in touch

Closed

Royal Brompton and Harefield NHS Foundation Trust

Address

Royal Brompton Hospital Sydney Street London Greater London SW3 6NP

Recruitment starts

March 2017

Contact

Davies, Jane C

Get in touch
Show all participating centres