Clinical Trial Results:
Preliminary efficacy and safety of Apremilast in the treatment of acne conglobata: A phase II, single centre, open label, proof of concept study for the treatment of acne conglobata with the PDE-4 inhibitor Apremilast (APACCO-Study)

Trial information Top of page
Trial identification
Sponsor protocol code	TMP-0517
Additional study identifiers
ISRCTN number	-
US NCT number	NCT04161456
WHO universal trial number (UTN)	-
Sponsors
Sponsor organisation name	Fraunhofer Gesellschaft for its Institute Fraunhofer Institute for Molecular Biology and Applied Ecology (IME) - now ITMP
Sponsor organisation address	Theodor-Stern-Kai 7, Frankfurt, Germany, 60596
Public contact	Project Group TMP, Fraunhofer IME, Clinical Research, +49 630180208, clinical.research@ime.fraunhofer.de
Scientific contact	Project Group TMP, Fraunhofer IME, Clinical Research, +49 630180208, clinical.research@ime.fraunhofer.de
Paediatric regulatory details
Is trial part of an agreed paediatric investigation plan (PIP)	No
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?	No
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?	No
Results analysis stage
Analysis stage	Final
Date of interim/final analysis	21 Jun 2021
Is this the analysis of the primary completion data?	No
Global end of trial reached?	Yes
Global end of trial date	17 Feb 2021
Was the trial ended prematurely?	No
General information about the trial
Main objective of the trial	The primary objective of the study is to evaluate the proportion of subjects who achieve at least a 50% reduction in total number of inflammatory lesions at week 24
Protection of trial subjects	Subjects were recruited based on inclusion and exclusion criteria, study visits were performed to ensure safety and well being and compliance of subjects
Background therapy	-
Evidence for comparator	-
Actual start date of recruitment	01 Aug 2019
Long term follow-up planned	No
Independent data monitoring committee (IDMC) involvement?	Yes
Population of trial subjects
Number of subjects enrolled per country
Country: Number of subjects enrolled	Germany: 1
Worldwide total number of subjects	1
EEA total number of subjects	1
Number of subjects enrolled per age group
In utero	0
Preterm newborn - gestational age < 37 wk	0
Newborns (0-27 days)	0
Infants and toddlers (28 days-23 months)	0
Children (2-11 years)	0
Adolescents (12-17 years)	0
Adults (18-64 years)	1
From 65 to 84 years	0
85 years and over	0

Trial information Top of page

Subject disposition Top of page
Recruitment
Recruitment details	16 patients planned, study start 22.08.2019 recruitment period planned for 6 months
Pre-assignment
Screening details	Patient with acne conglobata were searched for inclusion. Intensive recruitment in known patient population, flyer and other advertisement still did not lead to enrolement.
Period 1
Period 1 title	treatment (overall period)
Is this the baseline period?	Yes
Allocation method	Not applicable
Blinding used	Not blinded
Blinding implementation details	design was a open-lable proof of concept study - no blinding was required
Arms
Arm title	treatment
Arm description	apremilast was given with a starting titration and a maintenance dose of 30mg per day over a total period of 24 weeks
Arm type	Experimental
Investigational medicinal product name	Apremilast
Investigational medicinal product code
Other name
Pharmaceutical forms	Tablet
Routes of administration	Oral use
Dosage and administration details	30 mg twice daily, orally, with an initial titration schedule
Number of subjects in period 1 treatment Started 1 Completed 0 Not completed 1      Lost to follow-up 1

Subject disposition Top of page

Baseline characteristics Top of page
Baseline characteristics reporting groups
Reporting group title	treatment
Reporting group description	-
Reporting group values treatment Total Number of subjects 1 1 Age categorical Units: Subjects     In utero 0 0     Preterm newborn infants (gestational age < 37 wks) 0 0     Newborns (0-27 days) 0 0     Infants and toddlers (28 days-23 months) 0 0     Children (2-11 years) 0 0     Adolescents (12-17 years) 0 0     Adults (18-64 years) 1 1     From 65-84 years 0 0     85 years and over 0 0 Gender categorical Units: Subjects     Female 0 0     Male 1 1 Ethnicity Ethnicity of the one enroled subject is described Units: Subjects     caucasian 1 1     other 0 0

Baseline characteristics Top of page

End points Top of page
End points reporting groups
Reporting group title	treatment
Reporting group description	apremilast was given with a starting titration and a maintenance dose of 30mg per day over a total period of 24 weeks

End points Top of page

Primary: To evaluate the proportion of subjects who achieve at least a 50% reduction in total number of inflammatory lesions at week 24 Top of page
End point title	To evaluate the proportion of subjects who achieve at least a 50% reduction in total number of inflammatory lesions at week 24 [1]
End point description	number of lesions at baseline were compared to number of lesions at week 24 after administration of IMP
End point type	Primary
End point timeframe	baseline to week 24 after IMP administration
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: only on subject was enrolled and dropped out, so no statistical analysis was done and no value for primary endpoint can be provided.
End point values treatment Number of subjects analysed 1 [2] Units: percentage     percentage 0
Notes [2] - only one subject was enrolled and has dropped out
No statistical analyses for this end point

Primary: To evaluate the proportion of subjects who achieve at least a 50% reduction in total number of inflammatory lesions at week 24 Top of page

Adverse events Top of page
Adverse events information
Timeframe for reporting adverse events	baseline until end of study (28 weeks after start of treatment)
Assessment type	Non-systematic
Dictionary used for adverse event reporting
Dictionary name	no coding
Dictionary version	na
Reporting groups
Reporting group title	treatment
Reporting group description	adverse events are reported for the one included patient
Serious adverse events treatment Total subjects affected by serious adverse events      subjects affected / exposed 0 / 1 (0.00%)      number of deaths (all causes) 0      number of deaths resulting from adverse events 0
Frequency threshold for reporting non-serious adverse events: 5%
Non-serious adverse events treatment Total subjects affected by non serious adverse events      subjects affected / exposed 1 / 1 (100.00%) Nervous system disorders headache      subjects affected / exposed 1 / 1 (100.00%)      occurrences all number 1 General disorders and administration site conditions belly ache      subjects affected / exposed 1 / 1 (100.00%)      occurrences all number 1 vermehrtes Durstgefühl      subjects affected / exposed 1 / 1 (100.00%)      occurrences all number 1 Skin and subcutaneous tissue disorders schmerzende Haut      subjects affected / exposed 1 / 1 (100.00%)      occurrences all number 1

Adverse events Top of page

More information Top of page
Substantial protocol amendments (globally)
Were there any global substantial amendments to the protocol? No
Interruptions (globally)
Were there any global interruptions to the trial? No
Limitations and caveats
Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
since only one of planned 16 patient could be enrolled no analysis of data was possible and non was done.

More information Top of page