Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation

Study at a glance

From the registry record, last updated by the sponsor on 5 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

ClinicalTrials.gov ID

NCT01225211

Acronym / sponsor ID

VX09-809-102

Conditions

Cystic Fibrosis

Phase

Phase 2

Study type

Interventional

Recruitment status

Completed

Enrolment

312 participants (actual)

Lead sponsor

Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)

Study start

Oct 2010

Primary completion

Apr 2014 (actual)

Study completion

Apr 2014 (actual)

First posted

20 Oct 2010 (estimated)

Results

Posted 5 Oct 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose

Treatment

Allocation

Randomised

Intervention model

Factorial

Masking

Triple blind (participant, care provider, investigator)

Number of arms

10

Who may qualify

Ages

18 years and older

Sex

All

Healthy volunteers

Not accepted

Age groups

Adult, Older adult

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. Cohort 1: Safety and Tolerability Based on Adverse Events (AEs)

   Time frame: Cohort 1: Day 1 up to 28 days after last dose (Last dose = Day 21)

   AE: any untoward medical occurrence in a participant during study; irrespective of relationship with treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent. AE includes serious AEs (SAEs) as well as Non-SAEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. AE that started at/after initial dosing of study drug, or increased in severity after initial dosing of study drug is considered treatment-emergent. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 14) and combination therapy period (Period 2: Day 15 to Day 21).

2. Cohort 2 and 3: Safety and Tolerability Based on Adverse Events (AEs)

   Time frame: Cohort 2 and 3: Day 1 up to 28 days after last dose (Last dose = Day 56)

   Detailed description is provided in Outcome Measure 1. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 28) and combination therapy period (Period 2: Day 29 to Day 56).

3. Cohort 4: Safety and Tolerability Assessed by Number of Participants With AEs and SAEs

   Time frame: Cohort 4: Day 1 up to 28 days after last dose (Last dose = Day 56)

   AEs and SAEs are defined in Outcome Measure 1.

4. Cohort 1: Absolute Change From Day 14 in Sweat Chloride at Day 21

   Time frame: Cohort 1: Day 14, Day 21

5. Cohort 2 And 3: Absolute Change From Day 28 in Sweat Chloride at Day 56

   Time frame: Cohort 2 and 3: Day 28, Day 56

6. Cohort 4: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 56

   Time frame: Cohort 4: Baseline, Day 56

   FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. ppFEV1 (predicted for age, gender, and height) was calculated using the Hankinson method.

Results and safety

Loading the posted results from ClinicalTrials.gov…

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2015.
• Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
• Derived from this study: PubMed 37983082Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
• Derived from this study: PubMed 33331662Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
• Derived from this study: PubMed 27898234Rowe SM, McColley SA, Rietschel E, Li X, Bell SC, Konstan MW, Marigowda G, Waltz D, Boyle MP; VX09-809-102 Study Group. Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR. Ann Am Thorac Soc. 2017 Feb;14(2):213-219. doi: 10.1513/AnnalsATS.201609-689OC.
• Derived from this study: PubMed 24973281Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.

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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.

55 sites in 7 countries: see every site and its status.