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

• Sponsor: UCB Biopharma SRL (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04658186
• Collaborator: (none)
• Enrollment: 450
• Locations: 98
• Arms: 3
• Anticipated Duration (Months): 42
• Patients Per Site: 4.6
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Early-stage Parkinson's Disease	Drug: UCB0599; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 450 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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
• Actual Study Start Date: Dec 30, 2020
• Anticipated Primary Completion Date: Jul 1, 2024
• Anticipated Study Completion Date: Jul 1, 2024

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures

1. Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III sum score [From Baseline up to 18 Months]

   Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) parts I-III includes several items assessing motor and non-motor signs and symptoms including cognitive impairment, sleep problems, speech, facial expression etc. Each of the items in the UPDRS parts is measured on a scale of 0 to 4, where 0 is normal and 4 (higher score) represents severe abnormalities/worse outcome. The scores from all 3 parts will be added together in which higher scores indicate worse disease.

Secondary Outcome Measures

1. MDS-UPDRS Part III subscale [From Baseline up to 18 Months]

   Movement Disorder Society Unified Parkinson's Disease Rating Scale (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. Each of the items in the UPDRS is measured on a scale of 0 to 4, where 0 is normal and 4 (higher score) represents severe abnormalities/worse outcome.

2. MDS-UPDRS Part II subscale [From Baseline up to 18 Months]

   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. Each of the items in the UPDRS is measured on a scale of 0 to 4, where 0 is normal and 4 (higher score) represents severe abnormalities/worse outcome.

3. MDS-UPDRS Part I subscale [From Baseline up to 18 Months]

   Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part I includes 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. Each of the items in the UPDRS is measured on a scale of 0 to 4, where 0 is normal and 4 (higher score) represents severe abnormalities/worse outcome.

4. Time to worsening of the disease on MDS-UPDRS Part III scale [From Baseline up to 18 Months]

   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.

5. Montreal Cognitive Assessment (MoCA) [From Screening up to 18 Months]

   The Montreal Cognitive Assessment (MoCA) assesses different cognitive domains (visuospatial/executive, naming, memory, attention, language, abstraction, delayed recall, and orientation). Participants are assessed on a 30-point scale. A score of 26 or above is considered normal, a lower score indicate cognitive impairment.

6. Change in Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) mean striatum specific binding ratios (SBR) [From Screening up to 18 Months]

   The change from baseline (screening) in mean striatum specific binding ratios (SBR) will be assessed by Dopamine Transporter Imaging with Single Photon Emission Computed Tomography using 123I-Ioflupane as radiopharmaceutical.

7. Time to start of symptomatic treatment (ST) [From Baseline up to 18 Months]

   Time to start of symptomatic treatment (ST) within the 18-month period.

8. Symptomatic treatment intake [From Baseline up to 18 Months]

   Number of participants on symptomatic treatment (ST) at 18 months

9. Incidence of treatment-emergent adverse events (TEAEs) [From Baseline up to 19 Months]

   Adverse event: Any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related.

10. Incidence of serious adverse events (SAEs) [From Screening up to 19 Months]

    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.

11. Incidence of TEAEs leading to participant withdrawal [From Baseline up to 19 Months]

    Adverse event: Any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 40 Years to 75 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Pd0053 50140	Birmingham	Alabama	United States	35233
2	Pd0053 50506	Phoenix	Arizona	United States	85004-1150
3	Pd0053 50391	Tucson	Arizona	United States	85710
4	Pd0053 50519	Fountain Valley	California	United States	92708
5	Pd0053 50385	Fresno	California	United States	93710
6	Pd0053 50416	La Jolla	California	United States	92037
7	Pd0053 50118	Los Angeles	California	United States	90033-5315
8	Pd0053 50392	Danbury	Connecticut	United States	06810
9	Pd0053 50538	Farmington	Connecticut	United States	06030-5357
10	Pd0053 50396	Boca Raton	Florida	United States	33486
11	Pd0053 50524	Bradenton	Florida	United States	34205
12	Pd0053 50394	Tampa	Florida	United States	33613
13	Pd0053 50401	Chicago	Illinois	United States	60611
14	Pd0053 50310	Chicago	Illinois	United States	60612-3863
15	Pd0053 50399	Winfield	Illinois	United States	60190
16	Pd0053 50074	Kansas City	Kansas	United States	66160
17	Pd0053 50121	Lexington	Kentucky	United States	40536-0284
18	Pd0053 50395	New Orleans	Louisiana	United States	70121
19	Pd0053 50547	Baltimore	Maryland	United States	21287
20	Pd0053 50546	Worcester	Massachusetts	United States	01655
21	Pd0053 50545	East Lansing	Michigan	United States	48824
22	Pd0053 50386	Farmington Hills	Michigan	United States	48334
23	Pd0053 50536	Saint Paul	Minnesota	United States	55130
24	Pd0053 50397	Las Vegas	Nevada	United States	89104
25	Pd0053 50299	New Brunswick	New Jersey	United States	08901
26	Pd0053 50077	New York	New York	United States	10021-4823
27	Pd0053 50530	Stony Brook	New York	United States	11794
28	Pd0053 50535	Williamsville	New York	United States	14221
29	Pd0053 50311	Cleveland	Ohio	United States	44195
30	Pd0053 50255	Columbus	Ohio	United States	43210
31	Pd0053 50398	Tulsa	Oklahoma	United States	74136
32	Pd0053 50526	Philadelphia	Pennsylvania	United States	19107
33	Pd0053 50084	Charleston	South Carolina	United States	29425
34	Pd0053 50532	Knoxville	Tennessee	United States	37920
35	Pd0053 50543	Memphis	Tennessee	United States	38157
36	Pd0053 50113	Houston	Texas	United States	77030
37	Pd0053 50400	San Antonio	Texas	United States	78229-3900
38	Pd0053 50107	Burlington	Vermont	United States	05401
39	Pd0053 50410	Fairfax	Virginia	United States	22031
40	Pd0053 50534	Virginia Beach	Virginia	United States	23456
41	Pd0053 50292	Kirkland	Washington	United States	98034-3030
42	Pd0053 50419	Spokane	Washington	United States	99202
43	Pd0053 50402	Crab Orchard	West Virginia	United States	25827
44	Pd0053 50374	Calgary		Canada
45	Pd0053 50390	Kelowna		Canada
46	Pd0053 50387	Ottawa		Canada
47	Pd0053 50388	Toronto		Canada
48	Pd0053 50389	Toronto		Canada
49	Pd0053 40197	Amiens		France
50	Pd0053 40527	Bordeaux		France
51	Pd0053 40424	Créteil		France
52	Pd0053 40526	Lille		France
53	Pd0053 40130	Marseille		France
54	Pd0053 40635	Nantes		France
55	Pd0053 40524	Nimes		France
56	Pd0053 40525	Paris		France
57	Pd0053 40131	Strasbourg		France
58	Pd0053 40528	Toulouse Cedex 09		France
59	Pd0053 40138	Bonn		Germany
60	Pd0053 40530	Dresden		Germany
61	Pd0053 40711	Erbach		Germany
62	Pd0053 40023	Erlangen		Germany
63	Pd0053 40140	Göttingen		Germany
64	Pd0053 40532	Haag I.ob		Germany
65	Pd0053 40249	Kiel		Germany
66	Pd0053 40174	Mainz		Germany
67	Pd0053 40529	Marburg		Germany
68	Pd0053 40531	Regensburg		Germany
69	Pd0053 40555	Brescia		Italy
70	Pd0053 40533	Padova		Italy
71	Pd0053 40257	Roma		Italy
72	Pd0053 40534	Roma		Italy
73	Pd0053 40359	Nijmegen		Netherlands
74	Pd0053 40694	Bydgoszcz		Poland
75	Pd0053 40539	Katowice		Poland
76	Pd0053 40538	Krakow		Poland
77	Pd0053 40696	Krakow		Poland
78	Pd0053 40700	Lodz		Poland
79	Pd0053 40702	Lublin		Poland
80	Pd0053 40535	Oswiecim		Poland
81	Pd0053 40536	Warszawa		Poland
82	Pd0053 40699	Warszawa		Poland
83	Pd0053 40705	Warszawa		Poland
84	Pd0053 40045	A Coruna		Spain
85	Pd0053 40159	Barcelona		Spain
86	Pd0053 40267	Barcelona		Spain
87	Pd0053 40046	Cordoba		Spain
88	Pd0053 40540	Madrid		Spain
89	Pd0053 40542	Móstoles		Spain
90	Pd0053 40352	Pamplona		Spain
91	Pd0053 40541	San Sebastián		Spain
92	Pd0053 40049	Sevilla		Spain
93	Pd0053 40175	London		United Kingdom
94	Pd0053 40543	London		United Kingdom
95	Pd0053 40698	London		United Kingdom
96	Pd0053 40544	Motherwell		United Kingdom
97	Pd0053 40306	Newcastle Upon Tyne		United Kingdom
98	Pd0053 40457	Plymouth		United Kingdom

Sponsors and Collaborators

• UCB Biopharma SRL

Investigators

• Study Director: UCB Cares, 001 844 599 2273 (UCB)

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: UCB Biopharma SRL
• ClinicalTrials.gov Identifier: NCT04658186
• Other Study ID Numbers: PD0053; 2020-003265-19
• First Posted: Dec 8, 2020
• Last Update Posted: Aug 19, 2022
• Last Verified: Aug 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• 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
• Keywords provided by UCB Biopharma SRL: Early-stage parkinson's disease; UCB0599; Phase 2; Early-stage PD
• Additional relevant MeSH terms: Parkinson Disease

Study Results

No Results Posted as of Aug 19, 2022