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Trial registered on ANZCTR


Registration number
ACTRN12611001041943
Ethics application status
Approved
Date submitted
26/09/2011
Date registered
4/10/2011
Date last updated
10/10/2011
Type of registration
Retrospectively registered

Titles & IDs
Public title
Is the effectiveness of Pulmozyme related to timing? Pre or Post Physiotherapy?
Scientific title
In adults with cystic fibrosis (CF), does lung function and quality of life change when Pulmozyme is given after physiotherapy rather than the standard timing given before physiotherapy?
Secondary ID [1] 273163 0
Adult CF Lung Function and QOL study when Pulmozyme is given after rather than before physiotherapy.
Universal Trial Number (UTN)
U1111-1124-8603
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic fibrosis 270866 0
Condition category
Condition code
Respiratory 279042 279042 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
1. Pulmozyme was given once daily via nebuliser for 2 weeks @ 2.5mg/dose

2. During days 1-14 the subjects were given one order of therapy (eg placebo saline, followed by physio then Pulmozyme) and then days 15-28 they were given the reverse order, in this case (Pulmozyme, followed by physio then placebo)

3. the federal government allows doctors to trial Pulmozyme for one month (28-30 days) as part of the PBS S100 listing. We designed the trial to keep within this requirement.

4. Physiotherapy was optimised by an experienced physiotherapist before the trial. The patients performed the same physio techniques every day during the trial – by themselves, individually, at home, as per standard CF Physiotherapy used clinically–

Generally it takes about 30-40 minutes, but the patient does the same thing every day before during and after the trial, with the endpoint that they have no more sputum that requires clearance techniques.


This trial will directly compare Pulmozyme given before versus after physiotherapy. To maintain blinding, a matched saline nebulized therapy will be given at the other time point.
The study comprises three components – baseline, first period (two weeks) followed immediately (no wash out) where two weeks of the reverse order. This duration was chosen to maintain within the Pulmozyme one month trial.
Intervention code [1] 269455 0
Treatment: Drugs
Comparator / control treatment
Intervention = Pulmozyme given after physiotherapy
Pulmozyme given after physiotherapy is the test, compared with the control of Pulmozyme given before physiotherapy.
Given the requirement for a placebo, the two arms are
1) pre/post:- Pulmozyme/physiotherapy/saline followed by saline/physiotherapy/Pulmozyme., which is tested in half the subjects.
2) Post/pre:- The other half of the subjects undertake the trial in the reverse order.

Group 1 = Post / Pre = . During days 1-14 the subjects were given each day the following treatments : (saline, followed by physio then Pulmozyme) and then days 15-28 they were given (Pulmozyme, followed by physio then placebo).

Group 2 = Pre / Post =. During days 1-14 the subjects were given each day the following treatments : (Pulmozyme, followed by physio then placebo) and then days 15-28 they were given (saline, followed by physio then Pulmozyme).
Control group
Active

Outcomes
Primary outcome [1] 279697 0
Change in lung function (FEV1/FVC) both acutely and over a 14 day trial period.
Timepoint [1] 279697 0
14 day
Secondary outcome [1] 294237 0
Sputum weight during the physiotherapy clearance technique, plus one hour afterwards and during a 24 hour period. Exercise tests, maximum VO2 uptake.
Timepoint [1] 294237 0
During technique, plus one hour and during a 24 hour period

Eligibility
Key inclusion criteria
1 diagnosis of cystic fibrosis.
2 Clinically stable for one month before randomization.
3 FVC greater than 40% predicted.
4 Reproducible lung function tests.
5 Informed consent.
Minimum age
18 Years
Maximum age
70 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1 Inability to comply with protocol
2 Lack of informed consent.
3 Clinical instability
4 Pregnancy or breast feeding.
5 Allergy or intolerance to Pulmozyme..

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)
Allocation to intervention was concealed using sealed envelopes as the allocation group performed in blocks of four, with allocation performed by a pharmacist who was not involved in trial.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Sequence generation created by randomized table from computer.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Crossover
Other design features
Phase
Phase 4
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment postcode(s) [1] 4525 0
2120
Recruitment postcode(s) [2] 4526 0
2121
Recruitment postcode(s) [3] 4527 0
2123
Recruitment postcode(s) [4] 4528 0
2124
Recruitment postcode(s) [5] 4529 0
2125
Recruitment postcode(s) [6] 4530 0
2126
Recruitment postcode(s) [7] 4531 0
2127
Recruitment postcode(s) [8] 4532 0
2128
Recruitment postcode(s) [9] 4533 0
2129
Recruitment postcode(s) [10] 4534 0
2130
Recruitment postcode(s) [11] 4535 0
2131
Recruitment postcode(s) [12] 4536 0
2132
Recruitment postcode(s) [13] 4537 0
2133
Recruitment postcode(s) [14] 4538 0
2134
Recruitment postcode(s) [15] 4539 0
2135
Recruitment postcode(s) [16] 4540 0
2136
Recruitment postcode(s) [17] 4541 0
2137
Recruitment postcode(s) [18] 4542 0
2138
Recruitment postcode(s) [19] 4543 0
2140
Recruitment postcode(s) [20] 4544 0
2141
Recruitment postcode(s) [21] 4545 0
2142
Recruitment postcode(s) [22] 4546 0
2143
Recruitment postcode(s) [23] 4547 0
2144
Recruitment postcode(s) [24] 4548 0
2145
Recruitment postcode(s) [25] 4549 0
2146
Recruitment postcode(s) [26] 4550 0
2147
Recruitment postcode(s) [27] 4551 0
2148
Recruitment postcode(s) [28] 4552 0
2150
Recruitment postcode(s) [29] 4553 0
2151
Recruitment postcode(s) [30] 4554 0
2153
Recruitment postcode(s) [31] 4555 0
2154
Recruitment postcode(s) [32] 4556 0
2156
Recruitment postcode(s) [33] 4557 0
2157
Recruitment postcode(s) [34] 4558 0
2158
Recruitment postcode(s) [35] 4559 0
2159
Recruitment postcode(s) [36] 4560 0
2160
Recruitment postcode(s) [37] 4561 0
2161
Recruitment postcode(s) [38] 4562 0
2162
Recruitment postcode(s) [39] 4563 0
2163
Recruitment postcode(s) [40] 4564 0
2164
Recruitment postcode(s) [41] 4565 0
2165
Recruitment postcode(s) [42] 4566 0
2166
Recruitment postcode(s) [43] 4567 0
2167

Funding & Sponsors
Funding source category [1] 269938 0
Commercial sector/Industry
Name [1] 269938 0
Roche Pharmaceuticals
Address [1] 269938 0
Roche Products Pty. Ltd
4-10 Inman Rd
DEE WHY NSW 2099
Country [1] 269938 0
Australia
Primary sponsor type
Individual
Name
Jenny Bishop
Address
Jenny Bishop
Physiotherapist
Department of Respiratory Medicine
Westmead Hospital
Hawkesbury Rd
WESTMEAD NSW 2145
Country
Australia
Secondary sponsor category [1] 268936 0
None
Name [1] 268936 0
Address [1] 268936 0
Country [1] 268936 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 271903 0
Sydney Western Area Health Service Human Research Ethics Committee
Ethics committee address [1] 271903 0
Research Office
Room 2020 Clinical Sciences Corridor
Westmead Hospital
Hawkesbury Road
Westmead NSW 2145
Ethics committee country [1] 271903 0
Australia
Date submitted for ethics approval [1] 271903 0
01/09/1998
Approval date [1] 271903 0
11/09/1998
Ethics approval number [1] 271903 0
HREC98/9/4.8 (695)

Summary
Brief summary
In Australia Pulmozyme is licensed for use in CF with mild/moderate lung function impairment.
All CF patients are eligible for an initial one-month trial, but continued funding after one month dependent on a 10% improvement in FEV1.
The purpose of this one month trial of Pulmozyme is to provide information for each individual patient as to whether they benefit more with Pulmozyme before or Pulmozyme after their physiotherapy technique.
Following a period of optimization of their therapy, the patients undergo 2 two-week periods: Pulmozyme given before physiotherapy, and saline nebulized after physiotherapy with the second two weeks in the reverse direction.
The other half of the subjects are randomly allocated to the reverse order of interventions. Outcomes include lung function, quality of life, sputum, weight and exercise testing.
Trial website
Nil
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 33203 0
Address 33203 0
Country 33203 0
Phone 33203 0
Fax 33203 0
Email 33203 0
Contact person for public queries
Name 16450 0
Associate Professor Peter Middleton
Address 16450 0
Associate Professor Respiratory Medicine
Westmead Hospital
Level 2
Department of Respiratory Medicine
WESTMEAD NSW 2145
Country 16450 0
Australia
Phone 16450 0
+61 (02)98456797
Fax 16450 0
+61 (02)98457286
Email 16450 0
peter.middleton@sydney.edu.au
Contact person for scientific queries
Name 7378 0
Associate Professor Peter Middleton
Address 7378 0
Associate Professor Respiratory Medicine
Westmead Hospital
Level 2
Department of Respiratory Medicine
WESTMEAD NSW 2145
Country 7378 0
Australia
Phone 7378 0
+61 (02)98456797
Fax 7378 0
+61 (02)98457286
Email 7378 0
peter.middleton@sydney.edu.au

No information has been provided regarding IPD availability
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary