Evidence map›Paper›PMID 35959805›Full record

Trial reportMovement disorders : official journal of the Movement Disorder Society2022

Phase 1/1b Studies of UCB0599, an Oral Inhibitor of α-Synuclein Misfolding, Including a Randomized Study in Parkinson's Disease.

Johan Willem Smit, Peter Basile, Maria Key Prato, Laurent Detalle, François-Xavier Mathy, Astrid Schmidt, Marianna Lalla, Massimiliano Germani, Coralie Domange, Anja-Leona Biere and 3 more

2 registry-linked trialsOpen access · hybridAbstract readClinical Trial, Phase IRandomized Controlled Trial
In one paragraph

Trial report in Movement disorders : official journal of the Movement Disorder Society, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 35 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
35citing papers in PubMed, 2 pooled it
5.7field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04658186 phase2completednot on this map

A Double-Blind, Placebo-Controlled, Randomized, 18-Month Phase 2a Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Oral UCB0599 in Study Participants With Early Parkinson's Disease

TypeinterventionalSponsorUCB Biopharma SRLRan2020 to 2024Enrolled496ConditionsEarly-stage Parkinson's DiseaseArmsUCB0599, Placebo
NCT04875962 phase1completednot on this map

A Double-Blind, Placebo-Controlled, Multiple Dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of UCB0599 in Healthy Study Participants and Patients With Parkinson's Disease (PD)

TypeinterventionalSponsorUCB Biopharma S.P.R.L.Ran2019 to 2020Enrolled31ConditionsParkinson's DiseaseArmsUCB0599, Placebo
3 · Its place in the literature

Who cites it

35 citing papers in PubMed, 2 syntheses or guidelines pooled it, 53 citations in OpenAlex.

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  3. Cross-disease protein aggregate interactions in neurodegeneration: from molecular mechanisms to therapeutic strategies.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors at 4 institutions in 4 countries.

Johan Willem SmitUCB Pharma, Braine-l'Alleud, Belgium.
Peter BasileUCB Pharma, Slough, United Kingdom.
Maria Key PratoUCB Pharma, Braine-l'Alleud, Belgium.
Laurent DetalleUCB Pharma, Braine-l'Alleud, Belgium.
François-Xavier MathyUCB Pharma, Braine-l'Alleud, Belgium.
Astrid SchmidtUCB Pharma, Monheim am Rhein, Germany.
Marianna LallaUCB Pharma, Slough, United Kingdom.
Massimiliano GermaniUCB Pharma, Braine-l'Alleud, Belgium.
Coralie DomangeUCB Pharma, Brussels, Belgium.
Anja-Leona BiereUCB Pharma, Brussels, Belgium.
Massimo BaniUCB Pharma, Braine-l'Alleud, Belgium.
Stan CarsonUCB Pharma, Morrisville, North Carolina, USA.ORCID 0000-0001-5578-6520
Just GeniusUCB Pharma, Braine-l'Alleud, Belgium.
UCB Pharma (Belgium) · BEUCB Pharma (United Kingdom) · GBUCB Pharma (Germany) · DEUCB Pharma (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundParkinson's disease (PD) and its progression are thought to be caused and driven by misfolding of α-synuclein (ASYN). UCB0599 is an oral, small-molecule inhibitor of ASYN misfolding, aimed at slowing disease progression.

objectiveThe aim was to investigate safety/tolerability and pharmacokinetics (PK) of single and multiple doses of UCB0599.

methodsSafety/tolerability and PK of single and multiple doses of UCB0599 and its metabolites were investigated in two phase 1 studies in healthy participants (HPs), where food effect and possible interaction with itraconazole (ITZ) were assessed (UP0030 [randomized, placebo-controlled, dose-escalation, crossover study, N = 65] and UP0078 [open-label study, N = 22]). Safety/tolerability and multi-dose PK of UCB0599 were subsequently investigated in a phase 1b randomized, double-blind, placebo-controlled study of participants with PD (UP0077 [NCT04875962], N = 31).

resultsAcross all studies, UCB0599 displayed rapid absorption with linear, time-independent PK properties; PK of multiple doses of UCB0599 were predictable from single-dose exposures. No notable food-effect was observed; co-administration with ITZ affected UCB0599 disposition (maximum plasma concentration and area under the curve increased ~1.3- and ~2 to 3-fold, respectively) however, this did not impact the safety profile. Hypersensitivity reactions were reported in UP0030 (n = 2) and UP0077 (n = 2). Treatment-related adverse events occurred in 43% (UCB0599), and 30% (placebo) of participants with PD were predominantly mild-to-moderate in intensity and were not dose related.

conclusionsSeventy-three HPs and 21 participants with PD received UCB0599 doses; an acceptable safety/tolerability profile and predictable PK support continued development of UCB0599 for the slowing of PD progression. A phase 2 study in early-stage PD is underway (NCT04658186). © 2022 UCB Pharma. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Indexed as

alpha-SynucleinParkinson DiseaseCross-Over StudiesDose-Response Relationship, DrugDouble-Blind MethodHumansItraconazolealpha-SynucleinItraconazoledisease modificationmisfolding inhibitorParkinson's diseasephase 1 clinical trialα-synuclein

Identifiers

PMID35959805
PMCPMC9804489
OpenAlexW4296748423

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.