Trial document




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  DRKS00020286

Trial Description

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Title

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.

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Trial Acronym

TPV11

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URL of the Trial

[---]*

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Brief Summary in Lay Language

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

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Brief Summary in Scientific Language

A multi-centre, open-label, phase 1 study in patients with pemphigus vulgaris. The study consists of two parts: Part A with single ascending doses (4 dose levels) and Part B with multiple dose applications (2 dose levels) of the investigational medicinal product (IMP) TPM203. Primary objective is the evaluation of the safety and tolerability of the IMP, secondary objective the characterization of the pharmacokinetics of TPM203. In addition immunological effects are evaluated in an exploratory manner, with respect to early signs of effectiveness of induction of antigen-specific immune tolerance with TPM203 in pemphigus vulgaris patients.

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Do you plan to share individual participant data with other researchers?

No

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Description IPD sharing plan:

No (Phase 1 study)

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Organizational Data

  •   DRKS00020286
  •   2019/12/12
  •   [---]*
  •   no
  •   Approved
  •   99/19 A-ff, Ethik-Kommission des Fachbereichs Medizin der Philipps-Universität Marburg
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Secondary IDs

  •   2019-001727-12 
  •   TPV11 
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Health Condition or Problem studied

  •   L10.0 -  Pemphigus vulgaris
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Interventions/Observational Groups

  •   Part A: single doses, ascending dose levels (4 levels with 3 patients each); Part B: multiple doses, ascending dose levels (2 dose levels with 6 patients each).
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Characteristics

  •   Interventional
  •   [---]*
  •   Single arm study
  •   Open (masking not used)
  •   [---]*
  •   Uncontrolled/Single arm
  •   Treatment
  •   Single (group)
  •   I
  •   No
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Primary Outcome

Safety and tolerability of TPM203 measured by frequency and severity of treatment-emergent adverse events (TEAEs) and worsening of the disease pemphigus vulgaris (PV) during IMP administration and within 28 days after study treatment.

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Secondary Outcome

Pharmacokinetics of TPM203

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Countries of Recruitment

  •   Germany
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Locations of Recruitment

  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
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Recruitment

  •   Actual
  •   2019/12/12
  •   24
  •   Multicenter trial
  •   National
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Inclusion Criteria

  •   Both, male and female
  •   18   Years
  •   70   Years
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Additional 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 ≤30 kg/m2;
4. Diagnosis with PV (documented), based on clinical symptoms, immune fluorescence; (skin/mucosa) and serum IgG (immunoglobulin G) against Dsg3 by ELISA;
5. At screening, complete clinical remission or low to moderate clinical disease activity (i.e. ABSIS ≤17 and PDAI ≤15);
6. Presence of anti-Dsg3 IgG antibodies in blood at screening;
7. Presence of peripheral blood CD4+ T cells specific for at least one of the Dsg3 peptides employed in TPM203 in blood samples at screening;
8. Agreement to contraception (applicable patients);
9. Negative result in a pregnancy test (applicable female patients).
10. Apart from PV, good general health.

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Exclusion Criteria

1. Immunosuppressive or immunomodulatory treatment during the study other than prednisolone ≤10 mg/d;
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.

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Addresses

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    • Topas Therapeutics GmbH
    • Mr.  Dr.  Rupert  Sandbrink 
    • Falkenried 88
    • 20251  Hamburg
    • Germany
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    • Topas Therapeutics GmbH
    • Mr.  Dr.  Rupert  Sandbrink 
    • Falkenried 88
    • 20251  Hamburg
    • Germany
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    • Universitätsklinikum Marburg (UKGM) Klinik für Dermatologie und Allergologie
    • Mr.  Prof. Dr.  Michael  Hertl 
    • Baldingerstraße
    • 35043  Marburg
    • Germany
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    • Universitätsklinikum Marburg (UKGM) Klinik für Dermatologie und Allergologie
    • Mr.  Prof. Dr.  Michael  Hertl 
    • Baldingerstraße
    • 35043  Marburg
    • Germany
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  • start of 1:1-Block address investigator
    • Universitätsklinikum Marburg (UKGM) Klinik für Dermatologie und Allergologie
    • Mr.  Prof. Dr.  Michael  Hertl 
    • Baldingerstraße
    • 35043  Marburg
    • Germany
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Sources of Monetary or Material Support

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    • Topas Therapeutics GmbH
    • Falkenried 88
    • 20251  Hamburg
    • Germany
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Status

  •   Recruiting ongoing
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Trial Publications, Results and other Documents

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* This entry means the parameter is not applicable or has not been set.