PN-1007-001
Research type
Research Study
Full title
First in Human Study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of PPSGG in anti-MAG neuropathy patients
IRAS ID
282277
Contact name
Michael Lunn
Contact email
Sponsor organisation
Polyneuron Pharmaceuticals AG
Eudract number
2020-000067-23
Duration of Study in the UK
1 years, 3 months, 2 days
Research summary
The aim of this study is to test a new drug, PPSGG,
that might help patients with anti-MAG neuropathy.
At the moment, there is no treatment that is
licenced for use in the UK. The medicines used
currently to treat anti-MAG neuropathy are licenced
for other conditions, and are not that effective and
have various side effects. Anti-MAG antibodies in the
blood cause the neuropathy in anti-MAG
paraproteinaemic peripheral neuropathy. The
purpose of this drug is to reduce or even eliminate
the level of the anti-MAG antibodies in the blood
that cause the neuropathy in anti-MAG neuropathy.
The drug is not a chemotherapy. The drug acts by
specifically absorbing the anti-MAG antibodies only
from the blood and carrying them away. If the levels
of anti-MAG autoantibodies are low, the patient
could have better outcome. An increase in these
autoantibodies normally indicates that the disease is
worsening.
The main aim of this study is to determine how safe
it is, to see how well PPSGG works, to see how it is
broken down in the body after one dose and several
doses, as well as to see how it affects patient
outcome.
This study will consist of 2 parts or phases. The first
phase of the study is called the Single Ascending
Dose (SAD) phase. Each patient in each cohort will
be given a single dose of drug, with the dose
increasing in each cohort. All patients will receive
the study drug. The second phase of the study is
called the Multiple Ascending Dose (MAD) phase. In
the MAD phase the patients will receive either the
study drug or a placebo (dummy) drug and neither
participants nor the treating doctor will know which
they have received (a double blind study). Several
doses of drug will be given to each patient and the
dose of drug will increase through the study.REC name
London - Central Research Ethics Committee
REC reference
20/LO/0951
Date of REC Opinion
21 Sep 2020
REC opinion
Further Information Favourable Opinion