An Extension Study to Evaluate the Long-Term Efficacy, Safety and Tolerability of UCB0599 in Study Participants With Parkinson's Disease

• ClinicalTrials.gov Identifier: NCT05543252
• Recruitment Status: Enrolling by invitation
• First Posted: September 16, 2022
• Last Update Posted: March 17, 2023
• Sponsor: UCB Biopharma SRL
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma SRL )

Study Description

Brief Summary:

The purpose of the study is to estimate the pharmacodynamic effects of UCB0599 on brain pathophysiology in Early-start versus Delayed-start participants originally diagnosed with new onset Parkinson's disease.

Condition or disease	Intervention/treatment	Phase
Parkinson's Disease	Drug: UCB0599	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 270 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Dose-Blinded Extension Study to Evaluate the Long-Term Efficacy, Safety, and Tolerability of UCB0599 in Study Participants With Parkinson's Disease
• Actual Study Start Date: August 29, 2022
• Estimated Primary Completion Date: December 10, 2029
• Estimated Study Completion Date: December 10, 2029

Arms and Interventions

Arm	Intervention/treatment
Experimental: UCB0599 High Dose Arm; Participants will receive a predefined high dosage of UCB0599 during the Treatment Period.	Drug: UCB0599; UCB0599 Pharmaceutical form: Granules in capsules Route of administration: Oral use Participants will receive UCB0599 in a pre-specified sequence during the Treatment Period.
Experimental: UCB0599 Low Dose Arm; Participants will receive a predefined low dosage of UCB0599 during the Treatment Period.	Drug: UCB0599; 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. Baseline adjusted Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) whole striatum SBR at PD0055 Month 18 [ Time Frame: From Baseline up to Month 18 ]

   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.

   Baseline will refer to PD0053 (NCT04658186) Screening Visit date.

Secondary Outcome Measures :

1. Cumulative Levodopa Equivalent Daily Dose; (LEDD) at PD0055 Month 18 [ Time Frame: From Baseline up to Month 18 ]
   The Cumulative Levodopa Equivalent Daily Dose (LEDD) will be calculated for each participant at each visit and at the end of study. This is the sum of all the LEDDs taken up to that visit. Any changes in medication (type, dose, or dosing regimen) should be accounted for when calculating cumulative doses.
2. Incidence of treatment-emergent adverse event (TEAEs) [ Time Frame: From Baseline to the Safety Follow-up Visit (Month 31) ]
   Any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related.
3. Incidence of serious adverse events (SAEs) [ Time Frame: From Baseline to the Safety Follow-up Visit (Month 31) ]
   Any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. Serious criteria of 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.
4. Incidence of TEAEs leading to withdrawal from study [ Time Frame: From Baseline to the Safety Follow-up Visit (Month 31) ]
   Any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related.

Eligibility Criteria

• Ages Eligible for Study: 40 Years to 78 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria

• Participant completed the Treatment Period of PD0053 (NCT04658186). The Baseline Visit for PD0055 (Visit 2) needs to be within 4 weeks following the end of treatment (EOT) Visit in PD0053 (NCT04658186)
• A male study participant must agree to use contraception during the Treatment Period and for at least 90 days after the last dose of the IMP and refrain from donating sperm during this period.

• A female study participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 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 investigational medicinal product (IMP). The study participant must have a negative urine pregnancy test at Screening (Visit 1), which is to be confirmed negative by urine testing prior to the first dose of IMP at PD0055 Baseline Visit. If oral contraception is used, an additional barrier method will be required during the study as an IMP-related gastrointestinal upset or a drug interaction by cytochrome P450 3A4 (CYP3A4) induction could interfere with efficacy

• Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the Informed Consent form (ICF) and in this protocol.

Exclusion Criteria:

• Study participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the study participant's ability to participate in this study
• A female study participant who tests positive for pregnancy, plans to get pregnant during the participation in the study, or who is breastfeeding
• Study participant had previously participated in PD0055
• Study participant meets any withdrawal criteria in PD0053 (NCT04658186)

Contacts and Locations

Locations

United States, California

Pd0055 50385

Fresno, California, United States, 93710

United States, Florida

Pd0055 50396

Boca Raton, Florida, United States, 33486

United States, Illinois

Pd0055 50401

Chicago, Illinois, United States, 60611

Pd0055 50399

Winfield, Illinois, United States, 60190

United States, Michigan

Pd0055 50386

Farmington Hills, Michigan, United States, 48334

United States, Nevada

Pd0055 50397

Las Vegas, Nevada, United States, 89123

United States, Oklahoma

Pd0055 50398

Tulsa, Oklahoma, United States, 74136

United States, Texas

Pd0055 50400

San Antonio, Texas, United States, 78229-3900

United States, West Virginia

Pd0055 50402

Crab Orchard, West Virginia, United States, 25827

France

Pd0055 40525

Paris, France

Germany

Pd0055 40532

Haag in Oberbayern, Germany

Netherlands

Pd0055 40359

Nijmegen, Netherlands

Poland

Pd0055 40539

Katowice, Poland

Pd0055 40538

Krakow, Poland

Pd0055 40535

Oswiecim, Poland

Sponsors and Collaborators

UCB Biopharma SRL

Investigators

• Study Director: UCB Cares; 001 844 599 2273

More Information

• Responsible Party: UCB Biopharma SRL
• ClinicalTrials.gov Identifier: NCT05543252
• Other Study ID Numbers: PD0055; 2022-500424-30-00 ( Registry Identifier: EU CT Number ); U1111-1279-2323 ( Other Identifier: Universal Trial Number (UTN) )
• First Posted: September 16, 2022
• Last Update Posted: March 17, 2023
• Last Verified: March 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• 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.
• Supporting Materials: Study Protocol; Statistical Analysis Plan (SAP); Clinical Study Report (CSR)
• 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.
• 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.
• URL: https://vivli.org/

• 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 SRL ):

Parkinson's disease.; UCB0599; Phase 2

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