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

NCT04875962 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: UP0077
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 6

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).

Conditions

Parkinson's Disease

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Treatment-Emergent Adverse Avents (TEAEs) from Baseline to End of Study visit

  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.

  From Baseline to End of study visit (up to Week 7)

Secondary Outcomes (12)

• Maximum observed plasma concentration (Cmax) in healthy participants on Day 1

  Day 1: Predose up to 12 hours post dose

• Maximum observed plasma concentration (Cmax) in healthy participants on Day 28

  Day 28: Predose up to 24 hours post dose

• Time to maximum observed plasma concentration (tmax) in healthy participants on Day 1

  Day 1: Predose up to 12 hours post dose

• Time to maximum observed plasma concentration (tmax) in healthy participants on Day 28

  Day 28: Predose up to 24 hours post dose

• Area under the concentration - time curve (AUC(0-12h)) from time 0 to 12 hours in healthy participants on Day 1

  Day 1: Predose up to 12 hours post dose

Study Arms (4)

Cohort 1 - UCB0599

EXPERIMENTAL

Participants will be randomized to receive a predefined dosage of UCB0599.

Drug: UCB0599

Cohort 2 - UCB0599

EXPERIMENTAL

Participants will be randomized to receive a predefined dosage of UCB0599.

Drug: UCB0599

Cohort 1 - Placebo

PLACEBO COMPARATOR

Participants will be randomized to receive a predefined dosage of Placebo.

Drug: Placebo

Cohort 2 - Placebo

PLACEBO COMPARATOR

Participants will be randomized to receive a predefined dosage of Placebo.

Drug: Placebo

Interventions

UCB0599 DRUG

Participants will receive an assigned dosage regimen of UCB0599 during the Treatment Period.

Cohort 1 - UCB0599; Cohort 2 - UCB0599

Placebo DRUG

Participants will receive an assigned dosage regimen of Placebo during the Treatment Period to maintain the blinding.

Cohort 1 - Placebo; Cohort 2 - Placebo

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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 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

Sponsors & Collaborators

• UCB Biopharma S.P.R.L.: lead

Study Sites (6)

Up0077 102

Long Beach, California, 90806, United States

Location

Up0077 103

Bay Harbor Islands, Florida, 33154, United States

Location

Up0077 105

DeLand, Florida, 32720, United States

Location

Up0077 107

Atlanta, Georgia, 30331, United States

Location

Up0077 101

Farmington Hills, Michigan, 48334, United States

Location

Up0077 104

Raleigh, North Carolina, 27612, United States

Location

Related Publications (2)

• Mercier J, Bani M, Colson AO, Germani M, Lalla M, Plisson C, Huiban M, Searle G, Mathy FX, Nicholl R, Otoul C, Smit JW, van Asch V, Wagneur M, Maguire RP. Evaluation and Application of a PET Tracer in Preclinical and Phase 1 Studies to Determine the Brain Biodistribution of Minzasolmin (UCB0599). Mol Imaging Biol. 2024 Apr;26(2):310-321. doi: 10.1007/s11307-023-01878-7. Epub 2023 Dec 18.

  PMID: 38110790 DERIVED

• 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.

  PMID: 35959805 DERIVED

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

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

Study Officials

• UCB Cares

  001 844 599 2273 (UCB)

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2021
• First Posted: May 6, 2021
• Study Start: May 6, 2019
• Primary Completion: February 19, 2020
• Study Completion: February 19, 2020
• Last Updated: May 6, 2021

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)