ANZCTR - Registration

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Trial registered on ANZCTR

Registration number

ACTRN12619000708156

Ethics application status

Approved

Date submitted

19/04/2019

Date registered

13/05/2019

Date last updated

13/05/2019

Date data sharing statement initially provided

13/05/2019

Date results provided

13/05/2019

Type of registration

Retrospectively registered

Titles & IDs

Public title

Observation effect of Ivacaftor/lumacaftor on lung function and exercise tolerance in subjects with Cystic Fibrosis and severe lung disease

Scientific title

Observation effect of Ivacaftor/lumacaftor on lung function and exercise tolerance in subjects with Cystic Fibrosis and severe lung disease, homozygous for phe508del

Secondary ID [1]

nil

Universal Trial Number (UTN)

Trial acronym

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Cystic Fibrosis

Condition category

Condition code

Human Genetics and Inherited Disorders

Cystic fibrosis

Intervention/exposure

Study type

Observational

Patient registry

True

Target follow-up duration

Target follow-up type

Months

Description of intervention(s) / exposure

Participants received Lumacaftor 400mg and Ivacaftor 250mg twice daily for 12 months. They received these as part of treatment, not for the study.Participation did not require any additional investigations or review. The data from the medical record was reviewed. All investigations were done as part of usual care.

Intervention code [1]

Not applicable

Comparator / control treatment

10 adult historical controls, who attended the same centre from 2012 to 2015, aged at least 18years, homozygous for the Phe508del mutation, who also would have been eligible for access to LUM/IVA as above, who either had ppFEV1 of 40% or less when clinically stable at their annual review and or were judged to require referral for transplant at this time.

Control group

Historical

Outcomes

Primary outcome [1]

Six minute walk test distance, measured in metres.

Timepoint [1]

52 weeks post treatment.

Secondary outcome [1]

FEV1 percent predicted, percentage change from baseline. Measured by spirometry. Standards used Global Lung Initiative.(GLI)

Timepoint [1]

52 weeks post treatment.

Eligibility

Key inclusion criteria

Diagnosis of Cystic fibrosis, aged 18years, and homozygous for Phe508del mutation, eligible for access to LUM/IVA through a compassionate access programme provided by Vertex. To be eligible they had to have an FEV1? 40% when clinically stable, or experienced a 20% or more fall in FEV1, or had been referred for lung transplantation assessment.

Minimum age

18 Years

Maximum age

No limit

Sex

Both males and females

Can healthy volunteers participate?

Key exclusion criteria

Unable to participate in the measures required.

Study design

Purpose

Natural history

Duration

Longitudinal

Selection

Convenience sample

Timing

Retrospective

Statistical methods / analysis

Analysis of continuous variables was performed using parametric techniques to compare differences as these were normally distributed. This was done using a one-way ANOVA with Dunnett’s multiple comparisons test. Categorical comparisons were assessed using Fisher’s exact test. Analysis was carried out using Graphpad Prism version 7.

Recruitment

Recruitment status

Completed

Date of first participant enrolment

Anticipated

Actual

10/03/2015

Date of last participant enrolment

Anticipated

Actual

24/06/2015

Date of last data collection

Anticipated

Actual

19/07/2016

Sample size

Target

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

NSW

Recruitment hospital [1]

John Hunter Hospital - New Lambton

Recruitment postcode(s) [1]

2305 - New Lambton

Funding & Sponsors

Funding source category [1]

Hospital

Name [1]

John Hunter Hospital

Address [1]

John Hunter Hospital Lookout Rd NSW 2305.

Country [1]

Australia

Primary sponsor type

Hospital

Name

John Hunter Hospital

Address

John Hunter Hospital Lookout Rd NSW 2305.

Country

Australia

Secondary sponsor category [1]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Hunter New England LHD

Ethics committee address [1]

AREA HQ LOOKOUT RD New Lambton NSW 2305

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

13/01/2015

Approval date [1]

27/02/2015

Ethics approval number [1]

Low risk application

Summary

Brief summary

Our aim was to determine a sensitive clinical measure of change, in patients with CF and severe airways disease as measured by an FEV1<40% when stable, following treatment with Lumacaftor/Ivacaftor. We assessed response with spirometry and compared this to changes seen in exercise capacity as measured by the six minute walk test (6MWT), the nitrogen multi-breath washout test, the carbon monoxide gas transfer factor and lung volumes measured by plethysmography.

Trial website

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Prof Peter Alexander Blanch Wark

Address

Centre for Healthy Lungs HMRI, Lookout Rd New Lambton NSW 2305.

Country

Australia

Phone

+61249213470

Fax

[email protected]

Contact person for public queries

Name

Peter Alexander Blanch Wark

Address

Centre for Healthy Lungs HMRI, Lookout Rd New Lambton NSW 2305.

Country

Australia

Phone

+61249213470

Fax

[email protected]

Contact person for scientific queries

Name

Peter Alexander Blanch Wark

Address

Centre for Healthy Lungs HMRI, Lookout Rd New Lambton NSW 2305.

Country

Australia

Phone

+61249213470

Fax

[email protected]

Data sharing statement

Will individual participant data (IPD) for this trial be available (including data dictionaries)?

No/undecided IPD sharing reason/comment

Clinical data recorded as for patient care, anonymised forms not possible.

What supporting documents are/will be available?

No Supporting Document Provided

Results publications and other study-related documents

Documents added manually

Type	Is Peer Reviewed?	DOI	Citations or Other Details	Attachment
Basic results	No			377438-(Uploaded-25-11-2020-12-29-49)-Basic results summary.docx
Study results article	Yes		Wark PAB, Cookson K, Thiruchelvam T, Brannan J, Do... [More Details]

Documents added automatically

Source	Title	Year of Publication	DOI
Embase	Lumacaftor/ Ivacaftor improves exercise tolerance in patients with Cystic Fibrosis and severe airflow obstruction.	2019	https://dx.doi.org/10.1186/s12890-019-0866-y

N.B. These documents automatically identified may not have been verified by the study sponsor.

Trial Review

Documents added manually

Documents added automatically