Clinical Trial Results:
Open-label, multi-centre, proof of concept phase IIa clinical trial on the efficacy and tolerability of an 8 week oral treatment with once daily 9 mg budesonide in patients with active ulcerative colitis
Summary
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EudraCT number |
2014-005635-14 |
Trial protocol |
DE LV HU LT |
Global end of trial date |
20 Feb 2017
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Results information
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Results version number |
v1(current) |
This version publication date |
11 Oct 2018
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First version publication date |
11 Oct 2018
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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 |
BUX-3/UCA
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02550418 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Dr. Falk Pharma GmbH
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Sponsor organisation address |
Leinenweberstrasse 5, Freiburg, Germany, 79288
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Public contact |
Clinical Research and Development, Dr. Falk Pharma GmbH, 0049 7611514-0, zentrale@drfalkpharma.de
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Scientific contact |
Clinical Research and Development, Dr. Falk Pharma GmbH, 0049 7611514-0, zentrale@drfalkpharma.de
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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 |
22 Feb 2018
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
27 Jan 2017
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Global end of trial reached? |
Yes
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Global end of trial date |
20 Feb 2017
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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 |
The primary objective is to evaluate the efficacy of a 8-week treatment with once-daily 9 mg budesonide in patients with active ulcerative colitis.
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Protection of trial subjects |
Close supervision of subjects by implementing interim visits every 14 days to guarantee their safety and wellbeing. Prior to recruitment of patients, all relevant documents of the clinical study were submitted to and proved by the Independent Ethics Committees (IECs) responsible for the participating investigators. Written consent documents embodied the elements of informed consent as described in the Declaration of Helsinki, the ICH Guidelines for Good Clinical Practice (GCP) and were in accordance with all applicable laws and regulations. The informed consent form and patient information sheet described the planned and permitted uses, transfers and disclosures of the patient's personal data and personal health information for purposes of conducting the study. The informed consent form and the patient information sheet further explained the nature of the study, its objectives and potential risks and benefits as well as the date informed consent was given. Before being enrolled in the clinical trial, every patient was informed that participation in this trial was voluntary and that he/she could withdraw from the study at any time without giving a reason and without having to fear any loss in his/her medical care. The patient’s consent was obtained in writing before the start of the study. By signing the informed consent, the patient declared that he/she was participating voluntarily and intended to follow the study protocol instructions and the instructions of the investigator and to answer the questions asked during the course of the trial.
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Background therapy |
None | ||
Evidence for comparator |
Not applicable | ||
Actual start date of recruitment |
13 Oct 2015
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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 |
Germany: 4
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Country: Number of subjects enrolled |
Hungary: 7
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Country: Number of subjects enrolled |
Latvia: 37
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Country: Number of subjects enrolled |
Lithuania: 13
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Worldwide total number of subjects |
61
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EEA total number of subjects |
61
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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) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
56
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From 65 to 84 years |
5
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85 years and over |
0
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Recruitment
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Recruitment details |
In total 61 patients from Germany, Hungary, Lithuania and Latvia were enrolled and treated. | ||||||||||||||
Pre-assignment
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Screening details |
Patients signing the informed consent form were screened for 7 to 10 days to evaluate eligibility for the study. A total of 66 patients was screened for enrolment into the study. Five patients could not be included into the treatment phase. The most frequent reason for screening failure was violation of eligibility criteria. | ||||||||||||||
Period 1
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Period 1 title |
8-week open-label treatment phase (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 | ||||||||||||||
Blinding implementation details |
Not applicable
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Arms
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Arm title
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Open-label treatment with budesonide | ||||||||||||||
Arm description |
8-week treatment with one budesonide 9 mg capsule, hard (BUX-PV) once-daily. Analysis set: Full Analysis Set, which was defined according to the intention-to-treat principle and included all patients who received at least one dose of the investigational medicinal product. | ||||||||||||||
Arm type |
Experimental | ||||||||||||||
Investigational medicinal product name |
Budesonide 9 mg capsule, hard (BUX-PV)
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Capsule, hard
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Routes of administration |
Oral use
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Dosage and administration details |
One budesonide 9 mg capsule, hard (BUX-PV) once-daily in the morning.
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Baseline characteristics reporting groups
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Reporting group title |
Open-label treatment with budesonide
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Reporting group description |
8-week treatment with one budesonide 9 mg capsule, hard (BUX-PV) once-daily. Analysis set: Full Analysis Set, which was defined according to the intention-to-treat principle and included all patients who received at least one dose of the investigational medicinal product. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Open-label treatment with budesonide
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Reporting group description |
8-week treatment with one budesonide 9 mg capsule, hard (BUX-PV) once-daily. Analysis set: Full Analysis Set, which was defined according to the intention-to-treat principle and included all patients who received at least one dose of the investigational medicinal product. |
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End point title |
Clinical remission at week 8 / EOT [1] | ||||||
End point description |
Percentage of patients being in clinical remission at week 8 / EOT. Clinical remission was defined as Clinical Activity Index (CAI) total score ≤ 4, with subscores 1 and 2 of 0 at week 8 / EOT.
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End point type |
Primary
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End point timeframe |
After 8-week treatment: week 8 / EOT
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No formal statistical hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only. |
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No statistical analyses for this end point |
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End point title |
Clinical and endoscopic remission at week 8 / EOT | ||||||
End point description |
The proportion of patients being in combined clinical and endoscopic remission at week 8 / EOT. Combined clinical and endoscopic remission was defined as modified Disease Activity Index (mDAI) stool frequency subscore = 0, rectal bleeding subscore = 0, mucosal appearance subscore = 0 or 1 and physician's rating of disease activity subscore = 0 or 1 at week 8 / EOT.
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End point type |
Secondary
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End point timeframe |
After 8-week treatment: week 8 / EOT
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
From baseline to week 8 / EOT
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Adverse event reporting additional description |
All adverse events which occurred from the first drug administration to week 8 / EOT.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
19.1
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Reporting groups
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Reporting group title |
Open-label treatment with budesonide
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Reporting group description |
8-week treatment with one budesonide 9 mg capsule, hard (BUX-PV) once-daily. Analysis set: The Safety Analysis Set included all patients who received at least one dose of the investigational medicinal product and had at least one follow-up value for the safety variables to be analysed. If the administration of any investigational medicinal product was not certain, the patient was included in the Safety Analysis Set. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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 |