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Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Phase 2

Locations

Netherlands

Study Type

Interventional

Intervention

LUM/IVA

Placebo

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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

Sites / Locations

HagaZiekenhuis
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Treatment Sequence 1

Treatment Sequence 2

Arm Description

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

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT03061331

First Posted

February 9, 2017

Last Updated

September 6, 2018

Sponsor

Vertex Pharmaceuticals Incorporated

1. Study Identification

Unique Protocol Identification Number

NCT03061331

Brief Title

Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation

Official Title

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-CFTR Mutation

Study Type

Interventional

2. Study Status

Record Verification Date

September 2018

Overall Recruitment Status

Completed

Study Start Date

January 31, 2017 (Actual)

Primary Completion Date

September 6, 2017 (Actual)

Study Completion Date

October 4, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Yes

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

20 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Treatment Sequence 1

Arm Type

Experimental

Arm Description

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

Arm Title

Treatment Sequence 2

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

LUM/IVA

Other Intervention Name(s)

lumacaftor/ivacaftor

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

No Active Drug

Primary Outcome Measure Information:

Title

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

Description

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

Time Frame

Study Baseline, Through Week 8

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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

Facility Information:

Facility Name

HagaZiekenhuis

City

Den Haag

Country

Netherlands

Facility Name

University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

City

Heidelberglaan

Country

Netherlands

12. IPD Sharing Statement

Citations:

PubMed Identifier

33249003

Citation

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.

Results Reference

derived

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Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation

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