Clinical trial NCT04185415: A Study to Test the Safety and Tolerability of UCB0107 in Study Participants With Progressive Supranuclear Palsy (PSP)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Progressive Supranuclear Palsy
Keywords: Progressive Supranuclear Palsy
UCB0107
Phase 1 study
PSP
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2019-12-04
Enrollment: 25
Sex: ALL
Minimum age (years): 40
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

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


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of treatment emergent adverse events (TEAEs) from Baseline to the last Visit
timeFrame: From Baseline up to Week 68
Secondary outcomes:
Other outcomes:
Locations:
city: Edegem
country: Belgium
facility: Psp003 40122
city: Leuven
country: Belgium
facility: Psp003 40002
city: Bochum
country: Germany
facility: Psp003 40277
city: Düsseldorf
country: Germany
facility: Psp003 40276
city: Essen
country: Germany
facility: Psp003 40278
city: Hannover
country: Germany
facility: Psp003 40024
city: Barcelona
country: Spain
facility: Psp003 40159
city: Barcelona
country: Spain
facility: Psp003 40267
city: Madrid
country: Spain
facility: Psp003 40100
city: Madrid
country: Spain
facility: Psp003 40268
city: London
country: United Kingdom
facility: Psp003 40166
city: London
country: United Kingdom
facility: Psp003 40175
city: Southampton
country: United Kingdom
facility: Psp003 40165
Study officials:
affiliation: 001 844 599 2273 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References: