A Study to Test the Safety and Tolerability of UCB0107 in Study Participants With Progressive Supranuclear Palsy (PSP)

A Participant-Blind, Investigator-Blind, Placebo-Controlled, Phase 1b Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of UCB0107 in Study Participants With Progressive Supranuclear Palsy (PSP)

The purpose of the study is to assess the safety and tolerability of UCB0107 in study participants with Progressive Supranuclear Palsy (PSP).

Study Overview

• Status: Completed
• Conditions: Progressive Supranuclear Palsy
• Intervention / Treatment: Drug: bepranemab; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 1

Contacts and Locations

Study Locations

• Belgium
  • Edegem, Belgium
    • Psp003 40122
  • Leuven, Belgium
    • Psp003 40002
• Germany
  • Bochum, Germany
    • Psp003 40277
  • Düsseldorf, Germany
    • Psp003 40276
  • Essen, Germany
    • Psp003 40278
  • Hannover, Germany
    • Psp003 40024
• Spain
  • Barcelona, Spain
    • Psp003 40159
  • Barcelona, Spain
    • Psp003 40267
  • Madrid, Spain
    • Psp003 40100
  • Madrid, Spain
    • Psp003 40268
• United Kingdom
  • London, United Kingdom
    • Psp003 40166
  • London, United Kingdom
    • Psp003 40175
  • Southampton, United Kingdom
    • Psp003 40165

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 38 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participant must be ≥40 years of age at the time of signing the informed consent
• Participants meet the criteria for possible or probable Progressive Supranuclear Palsy (PSP) Richardson's Syndrome according the Movement Disorder Society (MDS)-PSP criteria
• Participant is able to walk at least 5 steps with minimal or no assistance (stabilization of one arm or use of cane/walker)
• Participant has reliable caregiver support during the whole study period or the participant is able to independently follow the study protocol
• Participant is stable on all treatments for at least 2 weeks prior to the Baseline Visit
• Participant has a body mass index (BMI) within the range 16.0 to 32.0 kg/m^2 (inclusive)
• Participants can be male or female
• Participant (and caregiver or legal representative, if applicable) is 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. Informed consent must be obtained before initiating any study procedures

Exclusion Criteria:

• Ongoing, recurrent, severe headaches, including migraines
• Evidence of any clinically significant neurological disorder (including any clinically significant abnormalities on the screening magnetic resonance imaging) other than Progressive Supranuclear Palsy (PSP)
• Participant has a lifetime history of suicide attempt, or has suicidal ideation with at least some intent to act in the past 12 months as indicated by a positive response ("Yes") to either Question 4 or Question 5 of the "Screening/Baseline" version of the Columbia-suicide severity rating scale (C-SSRS) at Screening
• Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator or medical monitor, contraindicates participation in the study

• The following liver enzyme test results:

  • Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP) are >2.0x upper limit of normal (ULN)
  • Bilirubin >1.5x ULN (isolated bilirubin >1.5x ULN is acceptable if bilirubin is fractionated and direct bilirubin is <35 %)
• The mean QT interval value (corrected by Fredericia's formula for the heart rate, QTcF) of the 3 Screening ECGs is >450 msec for male participants or >470 msec for female participants or QTcF is >480 msec in participants with bundle branch block
• Abnormalities in lumbar spine previously known or determined by a Screening lumbar x-ray (if conducted) that may jeopardize the execution of the lumbar puncture
• Participant was previously treated with tau-protein targeting drugs and/or tau-protein targeting antibodies or vaccines.
• Treatment with biologic agents (such as monoclonal antibodies including marketed drugs) within 3 months or 5 half-lives (whichever is longer) prior to the first dose

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: bepranemab; Subjects will be randomized to receive bepranemab.	Drug: bepranemab; bepranemab will be administered in a predefined dosage.; Pharmaceutical Form: Solution for infusion; Route of Administration: Intravenous; Other Names: UCB0107
Placebo Comparator: Placebo; Subjects will be randomized to receive Placebo.	Drug: Placebo; Pharmaceutical Form: Solution for infusion; Concentration: 0.9% w/v sodium chloride aqueous solution; Route of Administration: Intravenous

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment emergent adverse events (TEAEs) from Baseline to the last 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 up to Week 68

Collaborators and Investigators

• Sponsor: UCB Biopharma SRL
• Investigators: Study Director: UCB Cares, 001 844 599 2273 (UCB)

Study record dates

Study Major Dates

• Study Start (Actual): December 3, 2019
• Primary Completion (Actual): November 17, 2021
• Study Completion (Actual): November 17, 2021

Study Registration Dates

• First Submitted: December 2, 2019
• First Submitted That Met QC Criteria: December 2, 2019
• First Posted (Actual): December 4, 2019

Study Record Updates

• Last Update Posted (Estimated): December 1, 2023
• Last Update Submitted That Met QC Criteria: November 27, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: Progressive Supranuclear Palsy; PSP; UCB0107; Phase 1 study
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Eye Diseases; Neurologic Manifestations; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Tauopathies; Cranial Nerve Diseases; Ocular Motility Disorders; Ophthalmoplegia; Paralysis; Supranuclear Palsy, Progressive
• Other Study ID Numbers: PSP003; 2019-002377-61 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Due to the small sample size in this trial, Individual Patient Data cannot be adequately anonymized and there is a reasonable likelihood that individual participants could be re-identified. For this reason, data from this trial cannot be shared.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No