A Study to Test the Safety, Tolerability, and Pharmacokinetics of UCB0599 in Healthy Study Participants and Patients With Parkinson's Disease (PD)

• ClinicalTrials.gov Identifier: NCT04875962
• Recruitment Status: Completed
• First Posted: May 6, 2021
• Last Update Posted: May 6, 2021
• Sponsor: UCB Biopharma S.P.R.L.
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma S.P.R.L. )

Study Description

Brief Summary:

The purpose of the study is to evaluate the safety and tolerability after administration of multiple doses and the pharmacokinetics (PK) of single and multiple doses of UCB0599 in healthy study participants and participants with Parkinson's Disease (PD).

Condition or disease	Intervention/treatment	Phase
Parkinson's Disease	Drug: UCB0599; Drug: Placebo	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 31 participants
• Allocation: Randomized
• Intervention Model: Sequential Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Basic Science
• Official Title: 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)
• Actual Study Start Date: May 6, 2019
• Actual Primary Completion Date: February 19, 2020
• Actual Study Completion Date: February 19, 2020

Arms and Interventions

Arm	Intervention/treatment
Experimental: Cohort 1 - UCB0599; Participants will be randomized to receive a predefined dosage of UCB0599.	Drug: UCB0599; Participants will receive an assigned dosage regimen of UCB0599 during the Treatment Period.
Experimental: Cohort 2 - UCB0599; Participants will be randomized to receive a predefined dosage of UCB0599.	Drug: UCB0599; Participants will receive an assigned dosage regimen of UCB0599 during the Treatment Period.
Placebo Comparator: Cohort 1 - Placebo; Participants will be randomized to receive a predefined dosage of Placebo.	Drug: Placebo; Participants will receive an assigned dosage regimen of Placebo during the Treatment Period to maintain the blinding.
Placebo Comparator: Cohort 2 - Placebo; Participants will be randomized to receive a predefined dosage of Placebo.	Drug: Placebo; Participants will receive an assigned dosage regimen of Placebo during the Treatment Period to maintain the blinding.

Outcome Measures

Primary Outcome Measures :

1. Treatment-Emergent Adverse Avents (TEAEs) from Baseline to End of Study visit [ Time Frame: From Baseline to End of study visit (up to Week 7) ]
   An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

Secondary Outcome Measures :

1. Maximum observed plasma concentration (Cmax) in healthy participants on Day 1 [ Time Frame: Day 1: Predose up to 12 hours post dose ]
   Cmax: Maximum observed plasma concentration
2. Maximum observed plasma concentration (Cmax) in healthy participants on Day 28 [ Time Frame: Day 28: Predose up to 24 hours post dose ]
   Cmax: Maximum observed plasma concentration
3. Time to maximum observed plasma concentration (tmax) in healthy participants on Day 1 [ Time Frame: Day 1: Predose up to 12 hours post dose ]
   Tmax: Time of observed Cmax
4. Time to maximum observed plasma concentration (tmax) in healthy participants on Day 28 [ Time Frame: Day 28: Predose up to 24 hours post dose ]
   Tmax: Time of observed Cmax
5. Area under the concentration - time curve (AUC(0-12h)) from time 0 to 12 hours in healthy participants on Day 1 [ Time Frame: Day 1: Predose up to 12 hours post dose ]
   AUC(0-12h): Area under the curve from time 0 to 12 hours
6. Area under the concentration-time curve for the dosing interval (AUCtau) in healthy participants on Day 28 [ Time Frame: Day 28: Predose up to 24 hours post dose ]
   AUCtau: Area under the concentration-time curve for the dosing interval at steady state
7. Maximum observed plasma concentration (Cmax) in patients on Day 1 [ Time Frame: Day 1: Predose up to 12 hours post dose ]
   Cmax: Maximum observed plasma concentration
8. Maximum observed plasma concentration (Cmax) in patients on Day 28 [ Time Frame: Day 28: Predose up to 24 hours post dose ]
   Cmax: Maximum observed plasma concentration
9. Time to maximum observed plasma concentration (tmax) in patients on Day 1 [ Time Frame: Day 1: Predose up to 12 hours post dose ]
   Tmax: Time of observed Cmax
10. Time to maximum observed plasma concentration (tmax) in patients on Day 28 [ Time Frame: Day 28: Predose up to 24 hours post dose ]
    Tmax: Time of observed Cmax
11. Area under the concentration - time curve (AUC(0-12h)) from time 0 to 12 hours in patients on Day 1 [ Time Frame: Day 1: Predose up to 12 hours post dose ]
    AUC(0-12h): Area under the curve from time 0 to 12 hours
12. Area under the concentration-time curve for the dosing interval (AUCtau) in patients on Day 28 [ Time Frame: Day 28: Predose up to 24 hours post dose ]
    AUCtau: Area under the concentration-time curve for the dosing interval at steady state

Eligibility Criteria

• Ages Eligible for Study: 40 Years to 80 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Criteria

Inclusion Criteria:

• Participant must be 40 to 80 years of age inclusive, at the time of signing the informed consent
• Study participant with Parkinson's disease (PD) must have a clinical diagnosis of PD. The following diagnostic criteria must be met: Bradykinesia AND at least ONE of the following: muscular rigidity or resting tremor
• Study participant with PD must have a historic brain image (magnetic resonance imaging (MRI) or computerized tomography (CT) obtained at any point from the time of clinical diagnosis to the time of Screening that does not show any brain abnormalities that could cause symptomatic Parkinsonism
• Study participant must have a Hoehn and Yahr Stage: 1 to 3
• Study participant must be either untreated, or treated with a stable regimen (at least 4 weeks prior to Baseline Visit) of antiparkinsonian drugs and is expected to remain on this regimen for the duration of the study
• Body weight >50 kg (110 lbs) and body mass index (BMI) within the range 18 to 32 kg/m^2 (inclusive)

Exclusion Criteria:

• Study participant has a known hypersensitivity to any components of the study medication or comparative drugs as stated in this protocol
• Study participant has a known relevant allergy, a pre-existing history of a relevant allergic condition, or a predisposition for an allergic reaction (ie, total immunoglobulin E [IgE] value above normal range at Screening); this study participant's inclusion should be discussed with the Medical Monitor
• Study participant has a history of levodopa-induced motor fluctuations or dyskinesia expected to interfere with his/her ability to participate in the study
• Study participant has ongoing significant inflammatory gastrointestinal disorders and/or clinical signs of significant gastrointestinal problems at Screening
• Study participant has a historic brain scan (MRI scan or CT scan) or an MRI scan performed at Screening indicative of a clinically significant abnormality
• Study participant has a diagnosis of a significant Central nervous system (CNS) disease other than PD or history of epilepsy or seizure disorder other than febrile seizures as a child
• Abnormalities in lumbar spine previously known or determined by a screening lumbar x-ray (if conducted)
• History of clinically significant back pain, back pathology, and/or back injury (for example, degenerative disease, spinal deformity, or spinal surgery) that may predispose participant to complications or technical difficulty with lumbar puncture
• Evidence or history of significant active bleeding or coagulation disorder or use of drugs that affect coagulation or platelet function within 14 days prior to lumbar catheter insertion
• Current or chronic history of liver disease, or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones)
• Study participant has a history or present condition of respiratory or cardiovascular disorders at Screening (eg, cardiac insufficiency, coronary heart disease, uncontrolled hypertension, arrhythmia, tachyarrhythmia, or myocardial infarction) which is considered clinically significant by the Investigator
• Study participant has medical history or current diagnosis of diabetes
• Study participant has clinical significant electrocardiogram (ECG) abnormality at Screening, in the opinion of the Investigator
• Study participant has had prior treatment with an investigational vaccine for PD (including active immunization or passive immunotherapy with monoclonal antibodies)
• Study participant has had prior surgical treatment of PD involving intracranial surgery or implantation of a device (including deep brain stimulation) or duodopa

Contacts and Locations

Locations

United States, California

Up0077 102

Long Beach, California, United States, 90806

United States, Florida

Up0077 103

Bay Harbor Islands, Florida, United States, 33154

Up0077 105

DeLand, Florida, United States, 32720

United States, Georgia

Up0077 107

Atlanta, Georgia, United States, 30331

United States, Michigan

Up0077 101

Farmington Hills, Michigan, United States, 48334

United States, North Carolina

Up0077 104

Raleigh, North Carolina, United States, 27612

Sponsors and Collaborators

UCB Biopharma S.P.R.L.

Investigators

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

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Smit JW, Basile P, Prato MK, Detalle L, Mathy FX, Schmidt A, Lalla M, Germani M, Domange C, Biere AL, Bani M, Carson S, Genius J. Phase 1/1b Studies of UCB0599, an Oral Inhibitor of alpha-Synuclein Misfolding, Including a Randomized Study in Parkinson's Disease. Mov Disord. 2022 Oct;37(10):2045-2056. doi: 10.1002/mds.29170. Epub 2022 Aug 12.

• Responsible Party: UCB Biopharma S.P.R.L.
• ClinicalTrials.gov Identifier: NCT04875962
• Other Study ID Numbers: UP0077
• First Posted: May 6, 2021
• Last Update Posted: May 6, 2021
• Last Verified: May 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No
• Plan Description: Due to the small sample size in this trial, IPD cannot be adequately anonymized i.e., there is a reasonable likelihood that individual participants could be re-identified. For this reason, data from this trial cannot be shared.

• 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 Pharma ( UCB Biopharma S.P.R.L. ):

UCB0599; Parkinson's Disease; Phase 1 study; Healthy participants; Patients

Additional relevant MeSH terms:

Parkinson Disease; Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases