Clinical Trial Results:
High Dose Antioxidant Treatment for Patients with Cystic Fibrosis
Summary
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EudraCT number |
2013-005481-19 |
Trial protocol |
DK |
Global end of trial date |
30 Sep 2015
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Results information
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Results version number |
v1(current) |
This version publication date |
22 Jun 2016
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First version publication date |
22 Jun 2016
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
2013-01
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Aarhus University Hospital
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Sponsor organisation address |
Brendstrupsgaardvej 100, Aarhus N, Denmark, 8200
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Public contact |
Pediatric Department A, Aarhus University Hospital, peschi@rm.dk
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Scientific contact |
Pediatric Department A, Aarhus University Hospital, peschi@rm.dk
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
30 Sep 2015
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
30 Sep 2015
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Global end of trial reached? |
Yes
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Global end of trial date |
30 Sep 2015
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To evaluate if high dose antioxdant treatment (per oral and inhalation) can inhibit the oxidativ stress mediated inflammation in cystic fibrosis patients lung. The inflammation with neutrophil leucocytes is monitored by Pet/CT scan
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Protection of trial subjects |
To minimize pain in relation to blood sampling the patients were offered local anestetics. No other measures were taken
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Background therapy |
Pancreatic enzymes - vitamin supplementation- PEP mask theraphy- pulmozyme inhalation | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
01 May 2014
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Denmark: 11
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Worldwide total number of subjects |
11
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EEA total number of subjects |
11
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
3
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Adolescents (12-17 years) |
8
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
Patients were contacted during rutine control visits in the outpatient Clinic and asked if they wanted to participate. Recruitement period August 2014 - April 2015 | ||||||
Pre-assignment
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Screening details |
14 pts. were screened. 2 did not want to participate and one suffered from liver disease and had to be excluded. | ||||||
Period 1
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Period 1 title |
Overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||
Allocation method |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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medical intervention | ||||||
Arm description |
All patients had the same run in period where they should be clinically stable and with out anti inflammatory treatment , then scan and tests were performed and there after they recieved Medical intervention (N acetylcysteine) for 14 dys. All tests were then repeated and the trial was over. | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
Mucomyst or ACC
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Effervescent tablet, Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
100 mg/kg/24hrs in 3 divided doses given per oral. The patients decided themselves whether they wanted mucomyst or ACC
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Investigational medicinal product name |
Glutathione (TAD)
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Inhalation vapour, solution
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Routes of administration |
Intrapulmonary use
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Dosage and administration details |
Inhalations were given 2 times daily 600 mg x2
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Baseline characteristics reporting groups
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Reporting group title |
Overall trial
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
medical intervention
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Reporting group description |
All patients had the same run in period where they should be clinically stable and with out anti inflammatory treatment , then scan and tests were performed and there after they recieved Medical intervention (N acetylcysteine) for 14 dys. All tests were then repeated and the trial was over. | ||
Subject analysis set title |
Post treatment
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Subject analysis set type |
Per protocol | ||
Subject analysis set description |
Post treatment
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End point title |
neutrophil metabolic activity | ||||||||||||
End point description |
PET-CT scan was carried out 60 minutes after iv injection of 18 F-FDG injection and patient was scanned 6 mins over parts of the lung of interest.
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End point type |
Primary
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End point timeframe |
Pet CT scan was performed early on the day when antioxidant theraphy was begun and Again 14 days later.
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Notes [1] - 4 patients did not complete the trial. Pre-post pairs are included. |
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Statistical analysis title |
Wilcoxon signed rank test | ||||||||||||
Comparison groups |
medical intervention v Post treatment
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Number of subjects included in analysis |
14
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
< 0.001 | ||||||||||||
Method |
Wilcoxon (Mann-Whitney) | ||||||||||||
Confidence interval |
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Adverse events information
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Timeframe for reporting adverse events |
2 weeks.
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Adverse event reporting additional description |
patients diaries, Anamnestic review at the final visit with investigator. Blood tests and physical examination.
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Assessment type |
Systematic | ||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||
Dictionary version |
19
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Reporting groups
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Reporting group title |
1medical intervention
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Reporting group description |
All patients had the same run in period where they should be clinically stable and with out anti inflammatory treatment , then scan and tests were performed and there after they recieved Medical intervention (N acetylcysteine) for 14 dys. All tests were then repeated and the trial was over. | ||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |