NCT03061331

Brief Summary

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, crossover study that will evaluate the efficacy of LUM/IVA in subjects with CF 12 years of age and older who have at least one A455E mutation.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2017

Shorter than P25 for phase_2

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 31, 2017

Completed
9 days until next milestone

First Submitted

Initial submission to the registry

February 9, 2017

Completed
14 days until next milestone

First Posted

Study publicly available on registry

February 23, 2017

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 6, 2017

Completed
28 days until next milestone

Study Completion

Last participant's last visit for all outcomes

October 4, 2017

Completed
12 months until next milestone

Results Posted

Study results publicly available

October 2, 2018

Completed
Last Updated

October 2, 2018

Status Verified

September 1, 2018

Enrollment Period

7 months

First QC Date

February 9, 2017

Results QC Date

September 6, 2018

Last Update Submit

September 6, 2018

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 8

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

    Study Baseline, Through Week 8

Study Arms (2)

Treatment Sequence 1

EXPERIMENTAL

LUM/IVA in Treatment Period 1; washout; placebo in Treatment Period 2

Drug: LUM/IVADrug: Placebo

Treatment Sequence 2

EXPERIMENTAL

Placebo in Treatment Period 1; washout; LUM/IVA in Treatment Period 2

Drug: LUM/IVADrug: Placebo

Interventions

LUM/IVADRUG

LUM 400 mg/IVA 250 mg every 12 hours (q12h)

Also known as: lumacaftor/ivacaftor
Treatment Sequence 1Treatment Sequence 2
PlaceboDRUG

No Active Drug

Treatment Sequence 1Treatment Sequence 2

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Male or female with confirmed diagnosis of CF
  • All subjects must have an A455E mutation on at least 1 CFTR allele; no more than 10 subjects may have an F508del mutation on 1 CFTR allele.
  • Forced expiratory volume in one second (FEV1) ≥30% of predicted and ≤90% of predicted at the Screening Visit, based on the Global Lung Function Initiative (GLI)-2012 multi ethnic all-age reference equations.
  • Stable CF disease as judged by the investigator.
  • Willing to remain on a stable medication regimen for CF from 4 weeks before Day 1 through the Follow up Visit.

You may not qualify if:

  • History of any comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
  • A G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutation on at least one CFTR allele.
  • Pregnant or breastfeeding.
  • Any abnormal laboratory values at the Screening Visit.
  • History of cataract/lens opacity, or evidence of cataract/lens opacity determined to be clinically significant by the ophthalmologist or optometrist during the ophthalmologic examination at the Screening Visit.
  • Use of strong inhibitors or strong inducers of CYP3A, including consumption of certain herbal medications and certain fruit and fruit juices, within 14 days before Day 1
  • Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

Heidelberglaan, Netherlands

Location

HagaZiekenhuis

The Hague, Netherlands

Location

Related Publications (1)

  • Berkers G, van der Meer R, Heijerman H, Beekman JM, Boj SF, Vries RGJ, van Mourik P, Doyle JR, Audhya P, Yuan ZJ, Kinnman N, van der Ent CK. Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation. J Cyst Fibros. 2021 Sep;20(5):761-767. doi: 10.1016/j.jcf.2020.11.007. Epub 2020 Nov 26.

    PMID: 33249003DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

lumacaftor, ivacaftor drug combination

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Results Point of Contact

Title
Medical Monitor
Organization
Vertex Pharmaceuticals Incorporated
medicalinfo@vrtx.com
617-341-6777

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 9, 2017

First Posted

February 23, 2017

Study Start

January 31, 2017

Primary Completion

September 6, 2017

Study Completion

October 4, 2017

Last Updated

October 2, 2018

Results First Posted

October 2, 2018

Record last verified: 2018-09

Locations

NL(2)