Clinical Trials Register

Summary
EudraCT Number:	2019-001727-12
Sponsor's Protocol Code Number:	TPV11
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2019-06-12
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

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A. Protocol Information

A.1

Member State Concerned

Germany - BfArM

A.2

EudraCT number

2019-001727-12

A.3

Full title of the trial

A multi-centre, open-label, phase 1 study, Part A single ascending dose and Part B multiple dose, to evaluate the safety, tolerability and pharmacokinetics, and to explore early signs of effectiveness of induction of antigen-specific immune tolerance with TPM203 in pemphigus vulgaris patients

Multizentrische, offene Phase-1-Studie, Teil A mit Einzeldosen in aufsteigenden Konzentrationen, Teil B mit Mehrfachdosen, zur Bewertung der Sicherheit, Verträglichkeit und Pharmakokinetik und zur Untersuchung früher Anzeichen der Wirksamkeit bei der Induktion der antigenspezifischen Immuntoleranz durch TPM203 bei PV-Patienten

A.3.1

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

First use of TPM203 in various doses in patients with pemphigus vulgaris to to evaluate the safety, tolerability, and metabolism and to explore early signs of effectiveness.

Erste Anwendung der Prüfsubstanz TPM203 in verschiedenen Dosen bei Patienten mit Pemphigus vulgaris, um die Sicherheit, Verträglichkeit und das Verhalten der Substanz im Körper zu testen und ggf. erste Wirksamkeitsanzeichen zu sehen.

A.4.1

Sponsor's protocol code number

TPV11

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Topas Therapeutics GmbH
B.1.3.4	Country	Germany
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Topas Therapeutics GmbH
B.4.2	Country	Germany
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Koordinierungszentrum für Klinische Studien Marburg (KKS)
B.5.2	Functional name of contact point	Susanne Harnisch
B.5.3	Address:
B.5.3.1	Street Address	Karl-von-Frisch-Straße 4
B.5.3.2	Town/ city	Marburg
B.5.3.3	Post code	35043
B.5.3.4	Country	Germany
B.5.4	Telephone number	+49064212866553
B.5.5	Fax number	+49064212866517
B.5.6	E-mail	susanne.harnisch@kks.uni-marburg.de

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	No
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	TPM203
D.3.4	Pharmaceutical form	Dispersion for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
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	Yes
D.3.11.13.1	Other medicinal product type	Non-biological complex drug (NBCD) TPM203 is a mixture of four Topas Particle Conjugates (TPC0002, TPC0003,TPC0005 and TPC0012)

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

Pemphigus vulgaris

E.1.1.1

Medical condition in easily understood language

Pemphigus vulgaris is a blistering skin disorder in which antibodies target the body's own tissues.

Pemphigus vulgaris ist eine blasenbildende Hauterkrankung, bei der sich Antikörper gegen körpereigenes Gewebe richten.

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	10052802
E.1.2	Term	Pemphigus vulgaris
E.1.2	System Organ Class	100000004858

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the safety and tolerability of the intravenous infusion of TPM203 in pemphigus vulgaris (PV) patients.

Untersuchung der Sicherheit und Verträglichkeit der intravenösen Infusion von TPM203 bei Patienten mit Pemphigus vulgaris (PV).

E.2.2

Secondary objectives of the trial

Secondary: Pharmacokinetics of TPM203 in PV patients

Sekundär: Pharmakokinetik von TPM203 bei PV-Patienten

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent to take part in the study
2.Patient (male or female) age ≥18 years and ≤70 years
3.Body weight of ≥50 kg and a body mass index ≥18.5 and ≤32 kg/m2
4.Diagnosis with PV (documented) and at screening complete clinical remission or low to moderate clinical disease activity (i.e. ABSIS ≤17 and PDAI ≤15)
5.Presence of anti-Dsg3 IgG (immunoglobulin G) antibodies and peripheral blood CD4+ T cells specific for at least one of the Dsg3 peptides employed in TPM203 in blood samples at screening or presence of human leucocyte antigen (HLA)-DRB1*04:02 and/or HLA-DQB1*05:03.

1. Schriftliche Einverständniserklärung zur Teilnahme an der Studie
2. Alter des Patienten (männlich oder weiblich) zwischen ≥ 18 Jahre und ≤ 70 Jahre
3. Körpergewicht ≥ 50 kg und Body-Mass-Index ≥ 18,5 und ≤ 32 kg / m2
4. Dokumentierte Diagnose von PV und beim Screening vollständige klinische Remission oder geringe bis mäßige klinische Krankheitsaktivität (d. H. ABSIS ≤17 und PDAI ≤15)
5. Beim Screening Anwesenheit von Anti-Dsg3-IgG (Immunglobulin G) -Antikörpern und CD4 + -T-Zellen in peripherem Blut, die für mindestens eines der in TPM203 verwendeten Dsg3-Peptide spezifisch sind oder Vorhandensein des humanen Leukozyten-Antigens (HLA)-DRB1*04:02 und/oder HLA-DQB1*05:03

E.4

Principal exclusion criteria

1.Immunosuppressive or immunomodulatory treatment during the study other than prednisolone ≤10 mg/d for patients with body weight <70 kg or ≤12.5 mg/d for patients with body weight ≥ 70 kg (or equipotent doses of other steroids)
2.Conditions including previous or concomitant medication that might present a risk to the patient and/or impede the attainment of the study's objectives.

1.Andere immunsuppressive oder immunmodulatorische Behandlung während der Studie als Prednisolon ≤ 10 mg / d für Patienten mit einem Körpergewicht <70 kg oder ≤12,5 mg/d für Patienten mit einem Körpergewicht ≥ 70 kg (oder äquipotente Dosen anderer Steroide)
2.Erkrankungen einschließlich früherer oder begleitender Medikamente, die ein Risiko für den Patienten darstellen und / oder die Erreichung der Ziele der Studie behindern könnten.

E.5 End points

E.5.1

Primary end point(s)

Primary endpoints: safety and tolerability of TPM203 measured by frequency and severity of treatment-emergent adverse events (TEAEs) and worsening of PV during IMP administration and within 28 days after study treatment.

Primäre Endpunkte: Sicherheit und Verträglichkeit von TPM203, bestimmt anhand der Häufigkeit und Schwere von Nebenwirkungen (TEAEs) und einer Verschlechterung der PV während und innerhalb von 28 Tagen nach der Applikation des Prüfpräparates.

E.5.1.1

Timepoint(s) of evaluation of this end point

Evaluation during and within 28 days after first administration of TPM203

Erfassung während und innerhalb von 28 Tagen nach der Applikation von TPM203

E.5.2

Secondary end point(s)

Pharmacokinetics

Pharmakokinetik

E.5.2.1

Timepoint(s) of evaluation of this end point

Part A only from pre-dose (day 1) up to 23 hours after start of IMP administration

Nur in Part A im Zeitraum von pre-dose (Tag 1) bis zu 23 Stunden nach dem Start der IMP-Gabe

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

E.6.4

Safety

Yes

E.6.5

Efficacy

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

Yes

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)

Yes

E.7.1.1

First administration to humans

Yes

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)

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.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Israel

Germany

Italy

United Kingdom

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

Data base closure

Datenbankschluss

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

E.8.9.2

In all countries concerned by the trial months

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.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

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)

Not different from the expected normal treatment of that condition

Nicht anders als die normale Behandlung von PV

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

2019-10-24

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-10-24

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2023-07-25

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