A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BIIB092 in Participants With Progressive Supranuclear Palsy
Overview
- Phase
- Phase 2
- Intervention
- BIIB092
- Conditions
- Supranuclear Palsy, Progressive
- Sponsor
- Biogen
- Enrollment
- 490
- Locations
- 2
- Primary Endpoint
- Change From Baseline in Progressive Supranuclear Palsy Rating Scale (PSPRS) at Week 52
- Status
- Terminated
- Last Updated
- 5 years ago
Overview
Brief Summary
The Primary objective of the study is to evaluate the efficacy of BIIB092, compared to placebo, as measured by a change from baseline in the PSP Rating Scale (PSPRS) at Week 52 and to assess the safety and tolerability of BIIB092, relative to placebo, by measuring the frequency of deaths, SAEs, AEs leading to discontinuation, and Grade 3 & 4 laboratory abnormalities.
The Secondary objective of the study is to evaluate the efficacy of BIIB092, compared to placebo, as measured by a change in baseline in the Movement Disorder Society (MDS)-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II at Week 52, to evaluate the efficacy of BIIB092, compared to placebo, as measured by the Clinical Global Impression of Change (CGI-C) at Week 52, to evaluate the efficacy of BIIB092, compared to placebo, as measured by a change in baseline in the Repeatable Battery for the Assessment of Neuropsychological Disease Severity (RBANS) at Week 52 and to assess the impact of BIIB092 on quality of life, relative to placebo, as measured by change from baseline on the Progressive Supranuclear Palsy Quality of Life scale (PSP-QoL) at Week 52.
Detailed Description
This study, previously posted by Bristol-Myers Squibb, has transitioned to Biogen under a licensing agreement.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Participants with probable or possible PSP
- •Able to ambulate independently or with assistance
- •Able to tolerate MRI
- •Have reliable caregiver to accompany participant to all study visits
- •Score greater or equal to 20 on the Mini Mental State Exam (MMSE) at screening
- •Participant must reside outside a skilled nursing facility or dementia care facility at the time of screening and admission to such a facility must not be planned
Exclusion Criteria
- •Presence of other significant neurological or psychiatric disorders
- •Diagnosis of amyotrophic lateral sclerosis (ALS) or other motor neuron disease
- •History of early, prominent rapid eye movement (REM) sleep behavior disorder
- •History of or screening brain MRI scan indicative of significant abnormality
- •Known history of serum or plasma progranulin level less than one standard deviation below the normal patient mean for the laboratory performing the assay
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Arms & Interventions
BIIB092
Participants will receive BIIB092 50 mg/ml intravenous (IV) infusion once every 4 weeks for 48 weeks in double blind treatment period followed by BIIB092 50 mg/ml IV infusion once every 4 weeks starting at Week 52 up to Week 208.
Intervention: BIIB092
Placebo
Participants will receive BIIB092 matching placebo IV infusion once every 4 weeks for 48 weeks in double blind treatment period followed by BIIB092 50 mg/ml IV infusion once every 4 weeks starting at Week 52 up to Week 208.
Intervention: Placebo
Outcomes
Primary Outcomes
Change From Baseline in Progressive Supranuclear Palsy Rating Scale (PSPRS) at Week 52
Time Frame: Baseline, Week 52
The PSPRS is a quantitative measure of disability in participants with PSP. The PSPRS comprises 28 items in 6 areas. Six items are rated on a 3-point scale (0-2) and 22 are rated on a 5-point scale (0-4). The 6 areas are the History/Daily Activities, Mentation, Bulbar, Ocular Motor, Limb Motor, and Gait. The 28-item PSPRS total score ranges from 0 (normal) to 100. Fifteen items are selected to form a 15-item PSPRS and three domains are identified: Gait/Limb function, Ocular Motor, and Bulbar. The total 15-item PSPRS score ranges from 0 (normal) to 52. A positive change from baseline indicates worsening.
Percentage of Participants With Death, Serious Adverse Events (SAEs), Adverse Events (AEs) and Adverse Events (AEs) Leading to Discontinuation of Drug
Time Frame: up to 52 weeks
AEs: any sign, symptom, or diagnosis/disease that is unfavorable or unintended, that is new, or if pre-existing, worsens in participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. SAEs: an event that results in death; an event that, in the view of the investigator, places the participant at immediate risk of death (a life-threatening event); an outcome that results in a congenital anomaly/birth defect diagnosed in a child of a participant; an event that requires or prolongs inpatient hospitalization; an event that results in persistent or significant disability/incapacity.
Secondary Outcomes
- Number of Participants With Treatment Emergent Antibodies (Anti-BIIB092) Positive Results in Serum(Up to Week 48)
- Change From Baseline in Movement Disorder Society (MDS)-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II at Week 52(Baseline, Week 52)
- Clinical Global Impression of Change (CGI-C) Scale Score(Week 52)
- Change From Baseline in Progressive Supranuclear Palsy (PSP)-Cognitive Composite Battery Z-Score at Week 52(Baseline, Week 52)
- Change From Baseline in Color Trails at Week 48(Baseline, Week 48)
- Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Disease Severity (RBANS) Scale at Week 52(Baseline, Week 52)
- Change From Baseline in Schwab and England Activities of Daily Living (SEADL) Scale Score at Week 48(Baseline, Week 48)
- Change From Baseline in Clinical Global Impression of Severity (CGI-S) Score at Week 52(Baseline, Week 52)
- Change From Baseline in Phonemic Fluency Test Score at Week 48(Baseline, Week 48)
- Change From Baseline in Letter-Number Sequencing Test at Week 48(Baseline, Week 48)
- Change From Baseline in Montreal Cognitive Assessment (MoCA) Score at Week 48(Baseline, Week 48)
- Change From Baseline in Progressive Supranuclear Palsy Quality of Life Scale (PSP-QoL) Score(Baseline, Week 52)
- Change From Baseline of Brain Volumes as Determined by MRI at Week 52(Baseline, Week 52)