Single- and multiple-dose phase 1a/1b study with TPM203 in PV
Research type
Research Study
Full 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.
IRAS ID
289924
Contact name
Jorg Taubel
Contact email
Sponsor organisation
Topas Therapeutics GmbH
Eudract number
2019-001727-12
Clinicaltrials.gov Identifier
DRKS-ID, DRKS00020286
Duration of Study in the UK
1 years, 4 months, 2 days
Research summary
This study will assess the safety, tolerability, and the pharmacokinetic profile of TPM203 in patients suffering from pemphigus vulgaris (PV), an autoimmune condition where there is painful blistering on the skin and mucous membranes.
TPM203 is a mixture of four different dispersions of nano-sized Topas Particle-Conjugates (TPC) that we have named as TPC0002, TPC0003, TPC0005 and TPC0012. These particle-conjugates mimic blood borne antigens. These are thought to harnesses the natural mechanisms of the liver to promote immune tolerance to blood borne antigens.
Preclinical animal studies in mice and minipigs have shown that there were no adverse effects in the tested dosage range. Only a dark discolouration around the puncture site was observed in some animals, but this was found to be temporary.
This is a multi-centre, open-label, phase 1 study. It will take place in approximately 20 different sites, one of which is Richmond Pharmacology. It has two parts; Part A, which is a single ascending dose and Part B, a multiple dose. This study involves 24 PV patients; 12 in Part A and 12 in Part B. In Part A of the study, patients will be dosed at four different dose levels (three patients per dose level). Part B involves two dose levels, with three doses of TPM203 given at 2-week intervals (6 patients per dose level). Available data suggests an acceptable risk-benefit profile of TPM203.REC name
London - London Bridge Research Ethics Committee
REC reference
20/LO/1153
Date of REC Opinion
30 Nov 2020
REC opinion
Further Information Favourable Opinion