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.

Phase 1

• Conditions: Pemphigus vulgaris; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]; MedDRA version: 20.0Level: LLTClassification code 10052802Term: Pemphigus vulgarisSystem Organ Class: 100000004858

• Registration Number: EUCTR2019-001727-12-DE
• Lead Sponsor: Topas Therapeutics GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-06-12
• Last Update Posted: 28 March 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 24

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.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 12

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the safety and tolerability of the intravenous infusion of TPM203 in pemphigus vulgaris (PV) patients.;Secondary Objective: Secondary: Pharmacokinetics of TPM203 in PV patients<br>;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.;Timepoint(s) of evaluation of this end point: Evaluation during and within 28 days after first administration of TPM203<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Pharmacokinetics<br>;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<br><br>