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

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

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.

Study Overview

• Status: Completed
• Conditions: Early-stage Parkinson's Disease
• Intervention / Treatment: Drug: UCB0599; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 496
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Calgary, Canada
    • Pd0053 50374
  • Kelowna, Canada
    • Pd0053 50390
  • Ottawa, Canada
    • Pd0053 50387
  • Toronto, Canada
    • Pd0053 50389
• France
  • Amiens, France
    • Pd0053 40197
  • Bordeaux, France
    • Pd0053 40527
  • Créteil, France
    • Pd0053 40424
  • Lille, France
    • Pd0053 40526
  • Marseille, France
    • Pd0053 40130
  • Nantes, France
    • Pd0053 40635
  • Nîmes, France
    • Pd0053 40524
  • Paris, France
    • Pd0053 40525
  • Strasbourg, France
    • Pd0053 40131
  • Toulouse, France
    • Pd0053 40528
• Germany
  • Berlin, Germany
    • Pd0053 40515
  • Bonn, Germany
    • Pd0053 40138
  • Dresden, Germany
    • Pd0053 40530
  • Erbach im Odenwald, Germany
    • Pd0053 40711
  • Erlangen, Germany
    • Pd0053 40023
  • Essen, Germany
    • Pd0053 40710
  • Haag in Oberbayern, Germany
    • Pd0053 40532
  • Hanover, Germany
    • Pd0053 40024
  • Kiel, Germany
    • Pd0053 40249
  • Mainz, Germany
    • Pd0053 40174
  • Marburg, Germany
    • Pd0053 40529
  • Regensburg, Germany
    • Pd0053 40531
• Italy
  • Brescia, Italy
    • Pd0053 40555
  • Padua, Italy
    • Pd0053 40533
  • Roma, Italy
    • Pd0053 40257
  • Roma, Italy
    • Pd0053 40534
  • Roma, Italy
    • Pd0053 40697
• Netherlands
  • Nijmegen, Netherlands
    • Pd0053 40359
• Poland
  • Bydgoszcz, Poland
    • Pd0053 40694
  • Jelenia Góra, Poland
    • Pd0053 40719
  • Katowice, Poland
    • Pd0053 40539
  • Krakow, Poland
    • Pd0053 40538
  • Krakow, Poland
    • Pd0053 40696
  • Lodz, Poland
    • Pd0053 40700
  • Lublin, Poland
    • Pd0053 40702
  • Oświęcim, Poland
    • Pd0053 40535
  • Warsaw, Poland
    • Pd0053 40536
  • Warsaw, Poland
    • Pd0053 40699
  • Warsaw, Poland
    • Pd0053 40705
• Spain
  • A Coruña, Spain
    • Pd0053 40045
  • Barcelona, Spain
    • Pd0053 40159
  • Barcelona, Spain
    • Pd0053 40267
  • Córdoba, Spain
    • Pd0053 40046
  • Madrid, Spain
    • Pd0053 40540
  • Móstoles, Spain
    • Pd0053 40542
  • Pamplona, Spain
    • Pd0053 40352
  • San Sebastián, Spain
    • Pd0053 40541
  • Seville, Spain
    • Pd0053 40049
• United Kingdom
  • London, United Kingdom
    • Pd0053 40175
  • London, United Kingdom
    • Pd0053 40543
  • London, United Kingdom
    • Pd0053 40698
  • Motherwell, United Kingdom
    • Pd0053 40544
  • Newcastle upon Tyne, United Kingdom
    • Pd0053 40306
  • Plymouth, United Kingdom
    • Pd0053 40457
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Pd0053 50140
  • Arizona
    • Phoenix, Arizona, United States, 85004-1150
      • Pd0053 50506
    • Phoenix, Arizona, United States, 85013
      • Pd0053 50081
    • Tucson, Arizona, United States, 85710
      • Pd0053 50391
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • Pd0053 50539
  • California
    • Fountain Valley, California, United States, 92708
      • Pd0053 50519
    • Fresno, California, United States, 93710
      • Pd0053 50385
    • La Jolla, California, United States, 92037
      • Pd0053 50416
    • Los Angeles, California, United States, 90033-5315
      • Pd0053 50118
  • Colorado
    • Englewood, Colorado, United States, 80113
      • Pd0053 50531
  • Connecticut
    • Danbury, Connecticut, United States, 06810
      • Pd0053 50392
    • Farmington, Connecticut, United States, 06030
      • Pd0053 50538
  • Florida
    • Boca Raton, Florida, United States, 33486
      • Pd0053 50396
    • Bradenton, Florida, United States, 34205
      • Pd0053 50524
    • Miami, Florida, United States, 33136
      • Pd0053 50199
    • Tampa, Florida, United States, 33613
      • Pd0053 50394
  • Georgia
    • Augusta, Georgia, United States, 30912-0004
      • Pd0053 50544
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Pd0053 50401
    • Chicago, Illinois, United States, 60612-3863
      • Pd0053 50310
    • Winfield, Illinois, United States, 60190
      • Pd0053 50399
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Pd0053 50549
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • Pd0053 50074
  • Kentucky
    • Lexington, Kentucky, United States, 40536-0284
      • Pd0053 50121
  • Louisiana
    • New Orleans, Louisiana, United States, 70121
      • Pd0053 50395
  • Maryland
    • Baltimore, Maryland, United States, 21201-1606
      • Pd0053 50529
    • Baltimore, Maryland, United States, 21287
      • Pd0053 50547
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Pd0053 50243
    • Worcester, Massachusetts, United States, 01655
      • Pd0053 50546
  • Michigan
    • Farmington Hills, Michigan, United States, 48334
      • Pd0053 50386
  • Minnesota
    • Saint Paul, Minnesota, United States, 55130
      • Pd0053 50536
  • Nevada
    • Las Vegas, Nevada, United States, 89104
      • Pd0053 50397
  • New Jersey
    • New Brunswick, New Jersey, United States, 08903
      • Pd0053 50299
  • New York
    • New York, New York, United States, 10029
      • Pd0053 50521
    • New York, New York, United States, 10032
      • Pd0053 50119
    • New York, New York, United States, 10021
      • Pd0053 50077
    • Stony Brook, New York, United States, 11794
      • Pd0053 50530
    • Williamsville, New York, United States, 14221
      • Pd0053 50535
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • Pd0053 50372
    • Cleveland, Ohio, United States, 44195
      • Pd0053 50311
    • Columbus, Ohio, United States, 43210
      • Pd0053 50255
    • Toledo, Ohio, United States, 43606
      • Pd0053 50527
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74136
      • Pd0053 50398
  • Oregon
    • Portland, Oregon, United States, 97239
      • Pd0053 50510
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Pd0053 50526
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Pd0053 50084
  • Tennessee
    • Knoxville, Tennessee, United States, 37920
      • Pd0053 50532
    • Memphis, Tennessee, United States, 38157
      • Pd0053 50543
  • Texas
    • Houston, Texas, United States, 77030-5301
      • Pd0053 50525
    • Houston, Texas, United States, 77030
      • Pd0053 50113
    • San Antonio, Texas, United States, 78229
      • Pd0053 50400
  • Vermont
    • Burlington, Vermont, United States, 05401-1473
      • Pd0053 50107
  • Virginia
    • Charlottesville, Virginia, United States, 22908
      • Pd0053 50542
    • Fairfax, Virginia, United States, 22031
      • Pd0053 50410
    • Virginia Beach, Virginia, United States, 23456
      • Pd0053 50534
  • Washington
    • Kirkland, Washington, United States, 98034-3030
      • Pd0053 50292
    • Spokane, Washington, United States, 99202
      • Pd0053 50419
  • West Virginia
    • Crab Orchard, West Virginia, United States, 25827
      • Pd0053 50402

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: UCB0599 High Dose Arm; Participants will be randomized to receive a predefined high dosage of UCB0599 during the Treatment Period.	Drug: 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.
Placebo Comparator: Placebo Arm; Participants will be randomized to receive a predefined dosage of Placebo during the Treatment Period.	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.
Experimental: UCB0599 Low Dose Arm; Participants will be randomized to receive a predefined low dosage of UCB0599 during the Treatment Period.	Drug: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Day 0	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Day 0
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 2	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 2
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 4	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 4
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 6	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 6
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 8	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 8
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 10	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 10
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 12	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 12
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 14	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 14
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 16	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 16
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 18	MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.	Month 18

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
MDS-UPDRS Part III Subscale	MDS-UPDRS part III includes motor items assessing speech, facial expression, rigidity, finger tapping, hand movements, pronation supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement (body bradykinesia), postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, and constancy of rest tremor. It included 33 scores based on 18-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range was from 0 to 132, with a lower score indicating better motor function and a higher score indicating more severe motor symptoms.	Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items	The early-stage Parkinson's disease (ePD) subscore is derived from a 15-item subset of the MDS-UPDRS Part III (Motor Examination). It includes all rigidity assessments (neck, upper limbs [right/left], and lower limbs [right/left]) and bradykinesia-related tasks: finger tapping (right/left), hand movements (right/left), pronation-supination of hands (right/left), toe tapping (right/left), and leg agility (right/left). Each item is scored on a 5-point likert scale (0 = no problem to 4 = severe), resulting in a total ePD subscore range of 0 to 60. Higher scores indicate greater motor impairment, while lower scores reflect better motor function.	Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
MDS-UPDRS Part II Subscale	MDS-UPDRS part II includes motor items assessing speech, saliva and drooling, chewing and swallowing, eating tasks (cutting food and handling utensils), dressing, hygiene, handwriting, doing hobbies and other activities, turning in bed, tremor, getting out of bed, a car or a deep chair, walking and balance, and freezing. Each of the items in the UPDRS is measured on a scale. It included 13-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range for Part II was 0-52, with higher scores reflecting greater severity.	Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
MDS-UPDRS Part I Subscale	MDS-UPDRS part I include several non-motor aspects of experiences of daily living including cognitive impairment, hallucinations and psychosis, depressed mood, anxious mood, apathy, features of dopamine dysregulation syndrome during part 1A and sleep problems, daytime sleepiness, pain and other sensation, urinary problems, constipation problems, lightheadedness on standing, and fatigue during Part 1B. Part I assessed non-motor experiences of daily living and has 2 components (Range 0-52). Part IA contained 6 questions and were assessed by examiner (Range 0-24). Part IB contained 7 questions on non-motor experiences of daily living which were completed by participant (Range 0-28). Each of items in UPDRS is measured on a scale of 0 to 4, where 0 is normal and 4 (higher score) represents severe abnormalities/worse outcome. Total Score Range 0 to 52. Total score is sum of Part IA (0-24) and Part IB (0-28) subscale scores. Higher scores indicated greater severity of non-motor symptoms.	Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II	The participant was considered to have an emerging symptom for the item, if the change from Baseline for the item is greater than 0 for 2 consecutive visits. The magnitude of change from Baseline was not considered to determine the emerging symptom. Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part II includes motor items assessing speech, saliva and drooling, chewing and swallowing, eating tasks (cutting food and handling utensils), dressing, hygiene, handwriting, doing hobbies and other activities, turning in bed, tremor, getting out of bed, a car or a deep chair, walking and balance, and freezing. This included 13-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range for Part II was 0-52, with higher scores reflecting greater severity.	Baseline to Month 18
Time to Worsening of the Disease as Measured by MDS-UPDRS Part III	Time to worsening of the disease on the MDS-UPDRS III scale as defined by a 5-point increase in MDS-UPDRS III, within the 18-month period. MDS-UPDRS part III includes motor items assessing speech, facial expression, rigidity, finger tapping, hand movements, pronation supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement (body bradykinesia), postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, and constancy of rest tremor. It included 33 scores based on 18-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range was from 0 to 132, with a lower score indicating better motor function and a higher score indicating more severe motor symptoms.	Baseline to Month 18
Montreal Cognitive Assessment (MoCA)	The Montreal Cognitive Assessment (MoCA) is a standardized screening tool used to evaluate mild cognitive impairment across multiple cognitive functions i.e. visuospatial/executive function, naming, memory, attention, language, abstraction, delayed recall, and orientation. The total possible score is calculated by summing the scores across all functions ranges from: 0 to 30. A score of 26 or above is considered normal, a lower score indicates cognitive impairment.	Screening, Month 18
Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR)	The change from screening in mean striatum specific binding ratios (SBR) was assessed by DaT-SPECT using 123I-Ioflupane as radiopharmaceutical. The whole striatum was calculated as the average of the SBR data values for the four following "small" regions: left caudate small, left putamen small, right caudate small and right putamen small. The SBR was calculated for each region with the occipital cortex as a reference region, where a lower SBR indicates worse disease. The following formula was used to calculate this: (Average [Small region] - Average [Occipital region])/ (Average [Occipital region]).	Screening, Months 12 and 18
Time to Start of Symptomatic Treatment (ST)	Time to start of symptomatic treatment (ST) within the 18-month period.	Baseline to Month 18
Symptomatic Treatment (ST) Intake	Cumulative number of participants on symptomatic treatment (ST) at 18 months are reported.	Month 18
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)	An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE is defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment.	From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
Percentage of Participants With Serious TEAEs	Serious adverse event: Death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. Important medical events that may not result in death, be life-threatening, or require hospitalization may be considered serious when, based upon appropriate medical judgment, they may jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the outcomes listed in this definition.	From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
Percentage of Participants With TEAEs Leading to Participant Withdrawal	An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE is defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment.	From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)

Collaborators and Investigators

• Sponsor: UCB Biopharma SRL
• Collaborators: The Parkinson Study Group
• Investigators: Study Director: UCB Cares, 001 844 599 2273

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): December 30, 2020
• Primary Completion (Actual): September 6, 2024
• Study Completion (Actual): September 6, 2024

Study Registration Dates

• First Submitted: December 1, 2020
• First Submitted That Met QC Criteria: December 1, 2020
• First Posted (Actual): December 8, 2020

Study Record Updates

• Last Update Posted (Estimated): October 31, 2025
• Last Update Submitted That Met QC Criteria: October 17, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: Phase 2; Early-stage parkinson's disease; UCB0599; Early-stage PD
• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Parkinson Disease
• Other Study ID Numbers: PD0053; 2020-003265-19 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
• IPD Sharing Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• IPD Sharing Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No