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
- Conditions
- L10.0Pemphigus vulgaris
- Registration Number
- DRKS00020286
- Lead Sponsor
- Topas Therapeutics GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 17
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), 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.
1. Immunosuppressive or immunomodulatory treatment during the study other than prednisolone =10 mg/d (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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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.
- Secondary Outcome Measures
Name Time Method Pharmacokinetics of TPM203