Clinical Trials Register

Summary
EudraCT Number:	2017-003386-10
Sponsor's Protocol Code Number:	DMF_PPP
National Competent Authority:	Austria - BASG
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2017-11-03
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Austria - BASG

A.2

EudraCT number

2017-003386-10

A.3

Full title of the trial

Effectiveness and safety of Dimethylfumarate in patients with Palmoplantar Pustulosis – a 24-week, open label, phase II trial

Wirksamkeit und Sicherheit von Dimethylfumarat bei Patienten mit Palmoplantarer Pustulose - eine offene, Phase II Studie über 24 Wochen

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Dimethylfumarate for the treatment of Palmoplantar Pustulosis

Dimethylfumarat in der Behandlung der Palmoplantaren Pustulose

A.4.1

Sponsor's protocol code number

DMF_PPP

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Medical University of Vienna
B.1.3.4	Country	Austria
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Almirall
B.4.2	Country	Spain
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Medical University of Vienna
B.5.2	Functional name of contact point	Department of Dermatology
B.5.3	Address:
B.5.3.1	Street Address	Währinger Gürtel 18-20
B.5.3.2	Town/ city	Wien
B.5.3.3	Post code	1090
B.5.3.4	Country	Austria
B.5.4	Telephone number	+4314040077020
B.5.5	Fax number	+4314040076990
B.5.6	E-mail	adrian.tanew@meduniwien.ac.at

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Skilarence 30 mg
D.2.1.1.2	Name of the Marketing Authorisation holder	Almirall S.A.
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Skilarence
D.3.2	Product code	EMEA/H/C/002157
D.3.4	Pharmaceutical form	Gastro-resistant tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Dimethyl Fumarate
D.3.9.1	CAS number	624-49-7
D.3.9.3	Other descriptive name	DIMETHYL FUMARATE
D.3.9.4	EV Substance Code	SUB13608MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	30
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Skilarence 120 mg
D.2.1.1.2	Name of the Marketing Authorisation holder	Almirall, S.A.
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Skilarence
D.3.2	Product code	EMEA/H/C/002157
D.3.4	Pharmaceutical form	Gastro-resistant tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	dimethyl fumarate
D.3.9.1	CAS number	624-49-7
D.3.9.3	Other descriptive name	DIMETHYL FUMARATE
D.3.9.4	EV Substance Code	SUB13608MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	120
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Palmoplantar Pustulosis

Palmoplantare Pustulose

E.1.1.1

Medical condition in easily understood language

Palmoplantar Pustulosis

Palmoplantare Pustulose

E.1.1.2

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10050185
E.1.2	Term	Palmoplantar pustulosis
E.1.2	System Organ Class	100000004858

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Effectiveness of Dimethylfumarate in terms of 75% reduction of the Palmoplantar Pustulosis Psoriasis Area and Severity Index (ppPASI-75 response)

Wirksamkeit von Dimethylfumarat in Bezug auf eine 75% Reduktion des Palmoplantar Pustulosis Psoriasis Area and Severity Index (ppPASI-75 Antwort)

E.2.2

Secondary objectives of the trial

- Effectiveness of DMF in patients with nail involvement
- Health-related quality of life
- clinical safety and tolerability

- Wirksamkeit von DMF bei Patienten mit Nagelbeteiligung
- Lebensqualität
- Sicherheit und Tolerabilität

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Men or women ≥ 18 years and ≤ 80 years of age at time of screening
- Palmoplantar pustulosis (PPP) for at least 6 months
- moderate - severe disease as defined by a ppPASI score of ≥ 10 for affection of palms and soles, ≥4 for palms only and ≥6 for feet only.
- candidates for systemic treatment after failure of topical treatments
- negative serum pregnancy before enrollment and during the study

- Männer oder Frauen ≥ 18 Jahre und ≤ 80 Jahre
- Erkrankungsdauer von mindestens 6 Monaten
- mittelschwere - schwere Erkrankung, definiert durch einen ppPASI von ≥10 bei Befall von Handflächen und Fußsohlen, ≥4 bei Befall von ausschließlich Handflächen und ≥6 bei Befall von ausschließlich Fußsohlen
- Indikation für Systemtherapie nach frustranen Lokaltherapien
- negativer Schwangerschaftstest vor und während der Studie

E.4

Principal exclusion criteria

- use of prohibited psoriasis treatments
- pregant or nursing women
- WBC count < 3,000 cells/ mm3, lymphocyte count < 1.000/µl
- significant gastrointestinal problems (e.g., ulcer)
- severe renal impairment (eGFR ≤ 30ml/min)
- abnormal liver enzymes
- active infectious diseases
- history of alcohol or intravenous drug abuse
- Immunosuppressive conditions (HIV, organ transplant recipients)
- Known to be hypersensitive to ingredients of the investigational products
- Participation in any other drug investigational trial
- Not willing to give informed consent
- Unable to comply with the requirements of the study or who in the opinion of the investigator should not participate in the study

- Einnahme oder Verwendung verbotener Medikamente
- Schwangere oder stillende Frauen
- Leukozyten < 3,000 Zellen/ mm3, Lymphozyten < 1.000/µl
- relevante gastrointestinale Probleme (z.B. Ulcus)
- schwere Niereninsuffizienz (eGFR ≤ 30ml/min)
- abnorme Leberenzyme
- aktive Infektionserkrankungen
- Vorgeschichte von Alkohol- oder Substanzmissbrauch
- Immunsuppression (HIV oder Organtransplantation)
- Allergie gegen einen der Bestandteile des Arzneimittels
- gleichzeitige Teilnahme an einer anderen Arzneimittelstudie
- kein schriftliches Einverständnis
- Unfähigkeit die Vorgaben der Studie einzuhalten oder aus Sicht des Untersuchers ungeeignet für die Studienteilnahme

E.5 End points

E.5.1

Primary end point(s)

Proportion of patients with a ppPASI-75 response

Anteil der Patienten mit einer ppPASI-75 Antwort

E.5.1.1

Timepoint(s) of evaluation of this end point

week 24

Woche 24

E.5.2

Secondary end point(s)

Objective measures of disease severity:
- Mean reduction in ppPASI and logarithmic ppPASI score
- Mean reduction in target finger- and toenail Nail Area and Severity Index (NAPSI)
- Change in Investigator’s Global Assessment (IGA) for palms, soles, toe- and fingernails
- Proportion of patients achieving an IGA of 0 or 1

Patient reported outcomes:
- Mean reduction in pain- and itch-scale
- Mean reduction in Dermatology Life Quality Index (DLQI)
- Proportion of patients achieving DLQI 0 or 1
- Global Patient Satisfaction
- Change in Subject’s Global Assessment (SGA) for palms/soles and nails

To evaluate the clinical safety and tolerability of DMF as assessed by clinical laboratory variables and adverse events monitoring

Objektive Parameter der Erkrankungsschwere:
- mittlere Reduktion von ppPASI und logarithmischen ppPASI
- mittlere Reduktion des target Nail Area and Severity Index (NAPSI) von Finger- und Zehennägel
- Investigator’s Global Assessment (IGA) für Hand-, Fußflächen, Finger- und Zehennägel
- Anteil jener Patienten, die einen IGA von 0 oder 1 erreichen

Patient reported Outcomes:
- durchschnittliche Verbesserung von Juckreiz und Schmerz
- durchschnittliche Verbesserung des Dermatology Life Quality Index (DLQI)
- Anteil jener Patienten, die einen DLQI von 0 oder 1 erreichen
- Globale Patientenzufriedenheit
- Subject’s Global Assessment (SGA) für Hand-/Fußflächen und Nägel

Sicherheit und Tolerabilität von DMF anhand von Laborergebnissen und berichteten unerwünschten Arzneimittelwirkungen

E.5.2.1

Timepoint(s) of evaluation of this end point

week 24

Woche 24

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

E.6.5

Efficacy

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Treatment after completion of the study will be according to current standard of care for this condition.

Die Patienten werden im Routinebetrieb der Psoriasis Ambulanz der Universitätsklinik für Dermatologie weiter betreut.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2017-12-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-11-24

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2022-11-23

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials