Clinical Trial Results:
An open-label study to assess the safety, pharmacokinetics and pharmacodynamics of inhaled PC945 in adult Cystic Fibrosis (CF) patients with persistent pulmonary Aspergillus fumigatus infection.
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Summary
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EudraCT number |
2018-000243-87 |
Trial protocol |
GB |
Global end of trial date |
01 Jun 2020
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Results information
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Results version number |
v1(current) |
This version publication date |
04 Aug 2021
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First version publication date |
04 Aug 2021
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Other versions |
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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 |
PC_ASP_003
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT03870841 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Pulmocide Ltd
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Sponsor organisation address |
44 Southampton Buildings, London, United Kingdom, WC2A 1AP
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Public contact |
Dr Lance Berman, Pulmocide Ltd, +34 660 745 200 , Lance@pulmocide.com
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Scientific contact |
Dr Lance Berman, Pulmocide Ltd, +34 660 745 200 , Lance@pulmocide.com
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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 |
15 Apr 2021
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
03 Jan 2020
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Global end of trial reached? |
Yes
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Global end of trial date |
01 Jun 2020
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
• To investigate the safety and tolerability of once daily treatment with inhaled PC945 for 28 days in adult subjects with CF who have persistent pulmonary Aspergillus fumigatus infection
• To obtain estimates of derived systemic pharmacokinetic parameters of PC945 and the potential circulating metabolite(s), if detectable, following single and repeat doses of PC945
The study was terminated early as a result of the COVID-19 outbreak as the study was being conducted in a vulnerable patient group that was at very high risk of severe illness from COVID-19 and recruitment was halted by the hospitals involved in the study. As it was not possible to predict when recruitment could recommence, and it was unlikely that the trials could be completed without significant changes to the protocols, the Sponsor stopped the study on 01 June 2020.
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Protection of trial subjects |
This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with Good Clinical Practice and applicable regulatory requirements. Known instances of non-conformance were documented and are not considered to have had an impact on the overall conclusions of the study.
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Background therapy |
Subjects received PC945 in addition to their standard of care treatments | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
23 May 2019
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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 |
United Kingdom: 4
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Worldwide total number of subjects |
4
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EEA total number of subjects |
0
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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) |
4
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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 |
Eighteen subjects were to be enrolled to ensure that at least 10 subjects received PC945 and completed the study | ||||||||||
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Pre-assignment
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Screening details |
Six subjects were entered into the study, two of whom failed screening. Four subjects were recruited, one of whom was an earlier screening failure who was re-screened and then successfully entered the study. One subject was discontinued when the study was halted temporarily; the study was then terminated early as a result of COVID-19. | ||||||||||
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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 |
Not applicable
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Blinding used |
Not blinded | ||||||||||
Blinding implementation details |
This was an open label study with no blinding implementation.
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Arms
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Arm title
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PC945 | ||||||||||
Arm description |
Once daily doses of PC945 5 mg (emitted dose) once daily for 28 days | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
PC945
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Nebuliser suspension
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Routes of administration |
Inhalation use
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Dosage and administration details |
5mg (emitted dose) once daily for 28 days administered as a suspension for inhalation via a nebuliser
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Baseline characteristics reporting groups
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Reporting group title |
PC945
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Reporting group description |
Once daily doses of PC945 5 mg (emitted dose) once daily for 28 days | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
PC945
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Reporting group description |
Once daily doses of PC945 5 mg (emitted dose) once daily for 28 days | ||
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End point title |
PC945 Cmax Day 1 [1] | ||||||||
End point description |
Maximum PC945 plasma concentration observed from time 0 to 2 hours post dose; summary statistics provided.
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End point type |
Primary
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End point timeframe |
Day 1
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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: Summary statistics provided - no formal analysis conducted. |
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| No statistical analyses for this end point | |||||||||
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End point title |
PC945 Cmax Day 14 [2] | ||||||||
End point description |
Maximum PC945 plasma concentration from time 0 to 2 hour post dose; summary statistics provided.
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End point type |
Primary
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End point timeframe |
Day 14
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| Notes [2] - 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: Summary statistics provided - no formal analysis conducted. |
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| No statistical analyses for this end point | |||||||||
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End point title |
PC945 AUC 0-2h Day 1 [3] | ||||||||
End point description |
Area under the PC945 plasma concentration - time curve from 0 to 2 hour post dose; summary statistics provided.
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End point type |
Primary
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End point timeframe |
Day 1
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| Notes [3] - 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: Summary statistics provided - no formal analysis conducted. |
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| No statistical analyses for this end point | |||||||||
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End point title |
PC945 AUC 0-2h Day 14 [4] | ||||||||
End point description |
Area under PC945 plasma concentration-time curve from 0 to 2 hour post dose; summary statistics provided.
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End point type |
Primary
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End point timeframe |
Day 14
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| Notes [4] - 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: Summary statistics provided - no formal analysis conducted. |
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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 |
All AEs were to be reported from Visit 2 until completion of the subject's last study-related procedure.
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Adverse event reporting additional description |
Prior to Visit 2, from the time of signing consent, only AEs considered to be related to study procedures will be recorded.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
21.1
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Reporting groups
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Reporting group title |
PC945
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Reporting group description |
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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? Yes | ||||||||||
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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. | ||||||||||
| Due to the early termination and the limited number of subjects who completed the study no formal analysis are presented for secondary endpoints. | ||||||||||
