Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12610000328077
Ethics application status
Approved
Date submitted
13/04/2010
Date registered
23/04/2010
Date last updated
7/09/2010
Type of registration
Retrospectively registered

Titles & IDs
Public title
Orally inhaled heparin in patients with cystic fibrosis (CF)
Scientific title
A phase I/II randomised, placebo-controlled, double blind trial to assess the safety, tolerability, pharmacodynamics and exploratory efficacy of heparin inhalation in patients with cystic fibrosis (CF)
Secondary ID [1] 251636 0
ISRCTN78613729 issued by International Standard Randomized Controlled Trial Number Register.
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary symptoms of cystic fibrosis 257082 0
Condition category
Condition code
Human Genetics and Inherited Disorders 257231 257231 0 0
Cystic fibrosis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Patients will be randomised to receive one of three daily dose levels of heparin treatment or matching placebo; to be self-administered by inhalation by the patient twice daily for four consecutive weeks. Nominal Daily Doses to be studied are: 11400 International Units (IU), 22800 IU and 45600 IU.

For each patient there will be a screening period of 4 weeks, a treatment period of 4 weeks with a follow-up period of 2 weeks.
Intervention code [1] 256242 0
Treatment: Drugs
Comparator / control treatment
Excipient based placebo self-administered by inhalation
Control group
Placebo

Outcomes
Primary outcome [1] 258116 0
Safety and tolerability assessed by treatment-emergent adverse events, laboratory data (including haematology, clinical chemistry and urinalysis); physical examination; vital signs including blood pressure, heart rate, respiratory rate, temperature and weight; concomitant medication.
Timepoint [1] 258116 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [1] 263760 0
Assessment of Sputum properties (i.e., rheological viscoelasticity/physicochemical measurement parameters)
Timepoint [1] 263760 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [2] 263761 0
Assessment of Sputum inflammatory markers
Timepoint [2] 263761 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [3] 263762 0
Measurement of pH of exhaled breath condensate
Timepoint [3] 263762 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [4] 263763 0
Assessment of Blood plasma inflammatory markers
Timepoint [4] 263763 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [5] 263764 0
Assessment of blood coagulation through Activated Partial Thromboplastin Time and platelet count
Timepoint [5] 263764 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [6] 263765 0
Assessment of Visual Analogue Scale (VAS) parameters including cough; breathlessness; general wellbeing.
Timepoint [6] 263765 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [7] 263766 0
Assessment of Sputum microbiology
Timepoint [7] 263766 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [8] 263767 0
Assessment of Pulmonary function parameters by spirometry including forced expiratory volume in one second (FEV1) and forced vital capacity (FVC)
Timepoint [8] 263767 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
Secondary outcome [9] 263768 0
Assessment of Response to the Cystic Fibrosis Questionnaire
Timepoint [9] 263768 0
Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.

Eligibility
Key inclusion criteria
1. Male or female, aged 16 years or older
2. Non-smoker
3. Written informed consent obtained prior to any trial specific procedures
4. Confirmed diagnosis of CF lung disease (i.e., respiratory clinical symptoms and positive sweat test or disease inducing mutations) by CF expert/investigator
5. Forced expiratory volume in one second (FEV1) at 40 - 90% of predicted value for age, sex and height at screening and baseline
6. FEV1 value at Baseline is within +/-15% of value at screening
7. Regular mucus production due to CF
8. Ease of sputum expectoration as defined by VAS score equal to or less than 80 mm
9. Inflammatory markers above upper limit of normal range.
10. Adequate contraceptive measures.
11. Able to comply with all protocol requirements 12. Able to use inhalation device.
Minimum age
16 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
To be eligible for inclusion into this trial, each patient must not violate any one of the following exclusion criteria at the time of screening, at the time of assessment or as specifically described below:
1. Any contraindication to Monoparin( Registered Trademark) considered clinically relevant
2. Increased bleeding risk
3. History of heparin-induced thrombocytopaenia
4. Patients with bleeding diathesis
5. Evidence of portal hypertension (e.g., hypersplenism or known grade III/IV oesophageal varices)
6. Clinically significant liver disease
7. Pregnancy at screening, or lactation
8. Previous thoracic or scheduled major surgery during trial
9. Any regular anticoagulant therapy (e.g., warfarin, aspirin) in the two weeks prior to screening
10. Modification of medication to treat respiratory disease between screening and baseline (Day 1)
11. Diagnosis or history of aspergilloma
12. Clinically significant serious disease or organ system disease not currently controlled / stable on present therapy
13. Planned hospitalisations which could interfere with trial compliance
14. Unable for any other reason to satisfactorily comply with the protocol (e.g., attendance for trial visits, treatment or assessments)

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1 / Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
26/01/2009
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
64
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment postcode(s) [1] 2834 0
2305
Recruitment postcode(s) [2] 2835 0
4215
Recruitment postcode(s) [3] 2836 0
3052
Recruitment postcode(s) [4] 2837 0
6009
Recruitment outside Australia
Country [1] 2561 0
United Kingdom
State/province [1] 2561 0
Country [2] 2562 0
Ireland
State/province [2] 2562 0
Country [3] 2563 0
Poland
State/province [3] 2563 0
Country [4] 2564 0
Italy
State/province [4] 2564 0

Funding & Sponsors
Funding source category [1] 256751 0
Commercial sector/Industry
Name [1] 256751 0
Vectura Limited (UK)
Country [1] 256751 0
United Kingdom
Primary sponsor type
Commercial sector/Industry
Name
Vectura Limited (UK)
Address
1 Prospect West
Chippenham
Wiltshire
SN14 6FH
Country
United Kingdom
Secondary sponsor category [1] 256024 0
Commercial sector/Industry
Name [1] 256024 0
ORION Clinical Services (Australia) Pty Ltd
Address [1] 256024 0
141 Osborne Street
South Yarra, VIC 3141
Country [1] 256024 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 258751 0
Multi-Centre Research Ethics Committee for Wales
Ethics committee address [1] 258751 0
Ethics committee country [1] 258751 0
United Kingdom
Date submitted for ethics approval [1] 258751 0
21/11/2007
Approval date [1] 258751 0
18/03/2008
Ethics approval number [1] 258751 0
07/MRE09/74
Ethics committee name [2] 258752 0
St Vincents Healthcare Ethics and Medical Research Committee
Ethics committee address [2] 258752 0
Ethics committee country [2] 258752 0
Ireland
Date submitted for ethics approval [2] 258752 0
13/02/2008
Approval date [2] 258752 0
11/11/2008
Ethics approval number [2] 258752 0
none allocated
Ethics committee name [3] 258753 0
Bioethics Committee University of Lodz
Ethics committee address [3] 258753 0
Ethics committee country [3] 258753 0
Poland
Date submitted for ethics approval [3] 258753 0
21/04/2009
Approval date [3] 258753 0
14/07/2009
Ethics approval number [3] 258753 0
RNN/99/09/KE
Ethics committee name [4] 259154 0
Comitato Etico per la Sperimentazione, Azienda Ospedaliera Universitaria Integrata Verona
Ethics committee address [4] 259154 0
Ethics committee country [4] 259154 0
Italy
Date submitted for ethics approval [4] 259154 0
22/02/2010
Approval date [4] 259154 0
07/04/2010
Ethics approval number [4] 259154 0
17940/CE
Ethics committee name [5] 259155 0
Bellberry Human Research Ethics Committee
Ethics committee address [5] 259155 0
Ethics committee country [5] 259155 0
Australia
Date submitted for ethics approval [5] 259155 0
17/03/2010
Approval date [5] 259155 0
20/04/2010
Ethics approval number [5] 259155 0
C22/10
Ethics committee name [6] 259629 0
Hunter New England NSW HREC
Ethics committee address [6] 259629 0
Ethics committee country [6] 259629 0
Australia
Date submitted for ethics approval [6] 259629 0
02/03/2010
Approval date [6] 259629 0
28/05/2010
Ethics approval number [6] 259629 0
10/04/21/3.04
Ethics committee name [7] 259630 0
Gold Coast Health Service District HREC
Ethics committee address [7] 259630 0
Ethics committee country [7] 259630 0
Australia
Date submitted for ethics approval [7] 259630 0
08/04/2010
Approval date [7] 259630 0
15/07/2010
Ethics approval number [7] 259630 0
HREC/10/QGC/55

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 31002 0
Address 31002 0
Country 31002 0
Phone 31002 0
Fax 31002 0
Email 31002 0
Contact person for public queries
Name 14249 0
Dr Albert Shen
Address 14249 0
ORION Clinical Services (Australia) Pty Ltd
141 Osborne Street
South Yarra, VIC 3141
Country 14249 0
Australia
Phone 14249 0
+61 (0) 3 9867 1064
Fax 14249 0
+61 (0) 3 9867 1086
Email 14249 0
[email protected]
Contact person for scientific queries
Name 5177 0
Dr Mark Main
Address 5177 0
Vectura Limited(UK)
1 Prospect West
Chippenham
Wiltshire
SN14 6FH
Country 5177 0
United Kingdom
Phone 5177 0
+ 44 (0) 1249 667 700
Fax 5177 0
Email 5177 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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