A 18-month Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of Oral UCB0599 in Study Participants With Early-stage Parkinson's Disease

NCT ID: NCT04658186

Last Updated: 2025-10-31

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

496 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-12-30

Study Completion Date

2024-09-06

Brief Summary

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The purpose of the study is to assess the safety and tolerability of UCB0599 and to demonstrate the superiority of UCB0599 over placebo with regard to clinical symptoms of disease progression over 12 and 18 months in participants diagnosed with early-stage Parkinson's Disease.

Detailed Description

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Conditions

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Early-stage Parkinson's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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UCB0599 High Dose Arm

Participants will be randomized to receive a predefined high dosage of UCB0599 during the Treatment Period.

Group Type EXPERIMENTAL

UCB0599

Intervention Type DRUG

Drug: UCB0599 Pharmaceutical form: Granules in capsules Route of administration: Oral use Participants will receive UCB0599 in a pre-specified sequence during the Treatment Period.

Placebo Arm

Participants will be randomized to receive a predefined dosage of Placebo during the Treatment Period.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Drug: Placebo Pharmaceutical form: Capsules Route of administration: Oral use Participants will receive Placebo in a pre-specified sequence during the Treatment Period.

UCB0599 Low Dose Arm

Participants will be randomized to receive a predefined low dosage of UCB0599 during the Treatment Period.

Group Type EXPERIMENTAL

UCB0599

Intervention Type DRUG

Drug: UCB0599 Pharmaceutical form: Granules in capsules Route of administration: Oral use Participants will receive UCB0599 in a pre-specified sequence during the Treatment Period.

Interventions

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UCB0599

Drug: UCB0599 Pharmaceutical form: Granules in capsules Route of administration: Oral use Participants will receive UCB0599 in a pre-specified sequence during the Treatment Period.

Intervention Type DRUG

Placebo

Drug: Placebo Pharmaceutical form: Capsules Route of administration: Oral use Participants will receive Placebo in a pre-specified sequence during the Treatment Period.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Study participant must be 40 to 75 years of age inclusive, at the time of signing the informed consent
* Study participant has Parkinson's Disease (PD), with a diagnosis made by a neurologist according to the 2015 Movement Disorder Society criteria within 2 years of Baseline Visit (including diagnosis during Screening)
* The following diagnostic criteria must be met: bradykinesia AND at least ONE of the following: muscular rigidity, or resting tremor
* A Screening Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT), or a historical DaT-SPECT within 3 months of the Screening Visit that has been qualified by the central reader, shows evidence of dopamine transporter deficit per study requirements and as determined by a central reader
* Study participant is in the ≤2.5 modified Hoehn and Yahr stage at Screening
* Study participant has never taken medications for the treatment of motor symptoms of PD and is not expected to require starting symptomatic treatment (ST) with a high likelihood in the next 6 months as far as clinical judgement allows
* Study participant has never taken part in disease-modifying treatment studies directed at neurodegenerative disease (NDD)
* Study participant does not take N-acetyl cysteine or other cysteine donors or glutathione precursors on a regular basis as a food supplement
* Study participant is willing, competent, and able to comply with all aspects of the protocol, including follow-up schedule and biospecimen collection
* Study participant has a body mass index (BMI) of 16 to 34kg/m² (inclusive)
* Contraception i) A male participant must agree to use contraception during the Treatment Period and for at least 90 days after the last dose ofstudy medication and refrain from donating sperm during this period ii) A female participant is eligible to participate if she is not pregnant, not breastfeeding, andat least one of the following conditions applies: Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 1 month after the last dose of study medication. The study participant must have a negative serum pregnancy test at Screening, which is to be confirmed negative by urine testing prior to the first dose of study medication at Baseline (Visit 3). If oral contraception is used, an additional barrier method will be required during the study as a study medication related-gastrointestinal upset or a drug interaction by CYP3A4 induction could interfere with efficacy

Exclusion Criteria

* Study participant has a known hypersensitivity to any components (and/or its excipients) of the study medication or comparative drugs as stated in the protocol
* Study participant has a brain magnetic resonance imaging (MRI) scan performed during Screening indicative of a clinically significant abnormality or a historical MRI scan during the 6 months before Screening Visit 1 of sufficient quality to show such abnormalities. In case of doubt, the significance is determined on a case-by-case basis in close collaboration with the Medical Monitor and should not include abnormalities like age-appropriate brain atrophy, minor white matter signals, or mild vasculopathy
* Study participant has any contraindication for the brain MRI or Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) imaging
* Study participant has a Montreal Cognitive Assessment (MoCA) score less than 23, indicating mild cognitive impairment or other significant cognitive impairment or clinical dementia at Screening that, in the opinion of the Investigator, would interfere with study evaluation
* Study participant has abnormalities in lumbar spine previously known or determined by a Screening lumbar x-ray (if conducted) that could preclude lumbar puncture, in the opinion of the Investigator. The participant must be excluded from lumbar puncture but not from study participation
* Study participant has clinically significant electrocardiogram (ECG) abnormality at Screening, in the opinion of the Investigator
* Study participant has past history of use of medications for the treatment of motor symptoms of PD. Short (up to 4 weeks) past use of medications for the treatment of motor symptoms is permitted following a sufficient washout period. Medications included are: levodopa (maximum 400mg per day), dopamine agonists, monoamine oxidase B (MAO-B) inhibitors, anticholinergics, or amantadine. A sufficient washout period is at least 3 months prior to the Baseline Visit
Minimum Eligible Age

40 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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The Parkinson Study Group

NETWORK

Sponsor Role collaborator

UCB Biopharma SRL

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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UCB Cares

Role: STUDY_DIRECTOR

001 844 599 2273

Locations

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Pd0053 50140

Birmingham, Alabama, United States

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Pd0053 50506

Phoenix, Arizona, United States

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Pd0053 50081

Phoenix, Arizona, United States

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Pd0053 50391

Tucson, Arizona, United States

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Pd0053 50539

Little Rock, Arkansas, United States

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Pd0053 50519

Fountain Valley, California, United States

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Pd0053 50385

Fresno, California, United States

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Pd0053 50416

La Jolla, California, United States

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Pd0053 50118

Los Angeles, California, United States

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Pd0053 50531

Englewood, Colorado, United States

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Pd0053 50392

Danbury, Connecticut, United States

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Pd0053 50538

Farmington, Connecticut, United States

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Pd0053 50396

Boca Raton, Florida, United States

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Pd0053 50524

Bradenton, Florida, United States

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Pd0053 50199

Miami, Florida, United States

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Pd0053 50394

Tampa, Florida, United States

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Pd0053 50544

Augusta, Georgia, United States

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Pd0053 50401

Chicago, Illinois, United States

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Pd0053 50310

Chicago, Illinois, United States

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Pd0053 50399

Winfield, Illinois, United States

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Pd0053 50549

Iowa City, Iowa, United States

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Pd0053 50074

Kansas City, Kansas, United States

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Pd0053 50121

Lexington, Kentucky, United States

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Pd0053 50395

New Orleans, Louisiana, United States

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Pd0053 50529

Baltimore, Maryland, United States

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Pd0053 50547

Baltimore, Maryland, United States

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Pd0053 50243

Boston, Massachusetts, United States

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Pd0053 50546

Worcester, Massachusetts, United States

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Pd0053 50386

Farmington Hills, Michigan, United States

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Pd0053 50536

Saint Paul, Minnesota, United States

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Pd0053 50397

Las Vegas, Nevada, United States

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Pd0053 50299

New Brunswick, New Jersey, United States

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Pd0053 50077

New York, New York, United States

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Pd0053 50521

New York, New York, United States

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Pd0053 50119

New York, New York, United States

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Pd0053 50530

Stony Brook, New York, United States

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Pd0053 50535

Williamsville, New York, United States

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Pd0053 50372

Cleveland, Ohio, United States

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Pd0053 50311

Cleveland, Ohio, United States

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Pd0053 50255

Columbus, Ohio, United States

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Pd0053 50527

Toledo, Ohio, United States

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Pd0053 50398

Tulsa, Oklahoma, United States

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Pd0053 50510

Portland, Oregon, United States

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Pd0053 50526

Philadelphia, Pennsylvania, United States

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Pd0053 50084

Charleston, South Carolina, United States

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Pd0053 50532

Knoxville, Tennessee, United States

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Pd0053 50543

Memphis, Tennessee, United States

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Pd0053 50525

Houston, Texas, United States

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Pd0053 50113

Houston, Texas, United States

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Pd0053 50400

San Antonio, Texas, United States

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Pd0053 50107

Burlington, Vermont, United States

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Pd0053 50542

Charlottesville, Virginia, United States

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Pd0053 50410

Fairfax, Virginia, United States

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Pd0053 50534

Virginia Beach, Virginia, United States

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Pd0053 50292

Kirkland, Washington, United States

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Pd0053 50419

Spokane, Washington, United States

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Pd0053 50402

Crab Orchard, West Virginia, United States

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Pd0053 50374

Calgary, , Canada

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Pd0053 50390

Kelowna, , Canada

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Pd0053 50387

Ottawa, , Canada

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Pd0053 50389

Toronto, , Canada

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Pd0053 40197

Amiens, , France

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Pd0053 40527

Bordeaux, , France

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Pd0053 40424

Créteil, , France

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Pd0053 40526

Lille, , France

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Pd0053 40130

Marseille, , France

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Pd0053 40635

Nantes, , France

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Pd0053 40524

Nîmes, , France

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Pd0053 40525

Paris, , France

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Pd0053 40131

Strasbourg, , France

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Pd0053 40528

Toulouse, , France

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Pd0053 40515

Berlin, , Germany

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Pd0053 40138

Bonn, , Germany

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Pd0053 40530

Dresden, , Germany

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Pd0053 40711

Erbach im Odenwald, , Germany

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Pd0053 40023

Erlangen, , Germany

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Pd0053 40710

Essen, , Germany

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Pd0053 40532

Haag in Oberbayern, , Germany

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Pd0053 40024

Hanover, , Germany

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Pd0053 40249

Kiel, , Germany

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Pd0053 40174

Mainz, , Germany

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Pd0053 40529

Marburg, , Germany

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Pd0053 40531

Regensburg, , Germany

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Pd0053 40555

Brescia, , Italy

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Pd0053 40533

Padua, , Italy

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Pd0053 40257

Roma, , Italy

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Pd0053 40534

Roma, , Italy

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Pd0053 40697

Roma, , Italy

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Pd0053 40359

Nijmegen, , Netherlands

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Pd0053 40694

Bydgoszcz, , Poland

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Pd0053 40719

Jelenia Góra, , Poland

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Pd0053 40539

Katowice, , Poland

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Pd0053 40538

Krakow, , Poland

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Pd0053 40696

Krakow, , Poland

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Pd0053 40700

Lodz, , Poland

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Pd0053 40702

Lublin, , Poland

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Pd0053 40535

Oświęcim, , Poland

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Pd0053 40536

Warsaw, , Poland

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Pd0053 40699

Warsaw, , Poland

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Pd0053 40705

Warsaw, , Poland

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Pd0053 40045

A Coruña, , Spain

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Pd0053 40159

Barcelona, , Spain

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Pd0053 40267

Barcelona, , Spain

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Pd0053 40046

Córdoba, , Spain

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Pd0053 40540

Madrid, , Spain

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Pd0053 40542

Móstoles, , Spain

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Pd0053 40352

Pamplona, , Spain

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Pd0053 40541

San Sebastián, , Spain

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Pd0053 40049

Seville, , Spain

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Pd0053 40175

London, , United Kingdom

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Pd0053 40543

London, , United Kingdom

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Pd0053 40698

London, , United Kingdom

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Pd0053 40544

Motherwell, , United Kingdom

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Pd0053 40306

Newcastle upon Tyne, , United Kingdom

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Pd0053 40457

Plymouth, , United Kingdom

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Countries

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United States Canada France Germany Italy Netherlands Poland Spain United Kingdom

References

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Price DL, Khan A, Angers R, Cardenas A, Prato MK, Bani M, Bonhaus DW, Citron M, Biere AL. In vivo effects of the alpha-synuclein misfolding inhibitor minzasolmin supports clinical development in Parkinson's disease. NPJ Parkinsons Dis. 2023 Jul 17;9(1):114. doi: 10.1038/s41531-023-00552-7.

Reference Type DERIVED
PMID: 37460603 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2020-003265-19

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PD0053

Identifier Type: -

Identifier Source: org_study_id

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