Datum der Studienregistrierung
13.03.2020
Einschluss der ersten teilnehmenden Person
10.04.2020
Rekrutierungsstatus
Authorised-recruitment may be ongoing or finished
Wissenschaftlicher Titel
(Datenquelle: WHO)
First in Human Study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of PPSGG in anti-MAG neuropathy patients
Studientyp
(Datenquelle: WHO)
Interventional clinical trial of medicinal product
Design der Studie
(Datenquelle: WHO)
Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: yes
Other trial design description: During SAD open non controlled and during MAD double-blind placebo controlled, randomised
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: no
Number of treatment arms in the trial: 2
Phase
(Datenquelle: WHO)
Human pharmacology (Phase I): yesTherapeutic exploratory (Phase II): yesTherapeutic confirmatory - (Phase III): noTherapeutic use (Phase IV): no
Primäre Endpunkte
(Datenquelle: WHO)
Timepoint(s) of evaluation of this end point: ECG: 1-lead during infusions, 12-lead predose, 1h, 2h, 8h post dose. On Day 2 and EOS during SAD and during MAD on infusion days (Day 1-5, 8, 14, 21, 28, 35, 42, 56), 70, 98, EOS. The same days for vital signs
Safety lab: SAD: SCR, baseline, Day 8, EOS
Safety lab: MAD: SCR, baseline, Day 8, 28, 42, 98, EOS.;Primary end point(s): Assessment of safety based on vital signs, physical examination, ECGs, laboratory assessments, Signs of infusion-related reactions, including clinical signs and symptoms, changes in diastolic or systolic blood pressure, heart rate, oxygen saturation, and skin reactions or local reactions at the infusion site and collection of AEs assessed from baseline until the end of the study visit. Presence of Anti-drug antibodies will also be investigated;Secondary Objective: pharmacokinetics of PPSGG after single and multiple intravenous administrations, pharmacodynamics, preliminary efficacy;Main Objective: safety and tolerability
Sekundäre Endpunkte
(Datenquelle: WHO)
Secondary end point(s): PK: Non-compartmental parameters related to PPSGG, including but not limited to Tmax, Cmax, as well as trough (pre-dose) levels after multiple dose, PD: Reduction of anti-MAG antibodies and time to reduction, Time to anti-MAG IgM rebound (to pre-treatment BTU levels), Paraprotein levels (g/L), Total IgM levels (g/L), Anti-human natural killer-1 (HNK-1) IgM titers, Reduction of anti-MAG antibodies by =50% and time to reduction, preliminary efficacy: Change in ONLS score, Time to walk 10 meters, RODS, Ataxia score, Modified ISS, MUNIX (motor unit number index), Grip Strength;Timepoint(s) of evaluation of this end point: PK during infusion: 5, 30, 60min, 2, 6, 8 h,
PK and PD on SCR, baseline, Day 2, 4, 8 and 14 during SAD and on Day 1-5,8,14,21,28,35,42,56,70, EOS and FU during MAD
Scores: SCR, Day 14, 42, 98, EOS and 180 during MAD
Kontakt für Auskünfte
(Datenquelle: WHO)
Polyneuron Pharmaceuticals AG