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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03068468




Registration number
NCT03068468
Ethics application status
Date submitted
27/02/2017
Date registered
1/03/2017
Date last updated
21/12/2020

Titles & IDs
Public title
Study of BIIB092 in Participants With Progressive Supranuclear Palsy
Scientific title
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
Secondary ID [1] 0 0
2016-002554-21
Secondary ID [2] 0 0
251PP301
Universal Trial Number (UTN)
Trial acronym
PASSPORT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Supranuclear Palsy, Progressive 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BIIB092
Treatment: Drugs - Placebo

Experimental: 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.

Placebo comparator: 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.


Treatment: Drugs: BIIB092
BIIB092 intravenous infusion on specified days

Treatment: Drugs: Placebo
Placebo intravenous infusion on specified days
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Progressive Supranuclear Palsy Rating Scale (PSPRS) at Week 52
Assessment method [1] 0 0
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.
Timepoint [1] 0 0
Baseline, Week 52
Primary outcome [2] 0 0
Percentage of Participants With Death, Serious Adverse Events (SAEs), Adverse Events (AEs) and Adverse Events (AEs) Leading to Discontinuation of Drug
Assessment method [2] 0 0
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.
Timepoint [2] 0 0
up to 52 weeks
Secondary outcome [1] 0 0
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
Assessment method [1] 0 0
The MDS-UPRDRS Part 2 includes 13 items assessing motor aspects of experiences of daily living (M-EDL) these include speech, saliva and drooling, chewing and swallowing, handwriting, doing hobbies and other activities, eating tasks, tremor, dressing, hygiene, turning in bed, getting out of bed, walking and balance, and freezing. All items have 5 responses with uniform anchors of 0= normal, 1= slight, 2= mild, 3= moderate, and 4= severe. Total score ranges from 0 to 52, higher score indicating severe conditions. A positive change from baseline indicates worsening.
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [2] 0 0
Clinical Global Impression of Change (CGI-C) Scale Score
Assessment method [2] 0 0
The CGI-C scale measures the change in the patient's clinical status from a specific point in time. Using a 7-point scale, ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change.
Timepoint [2] 0 0
Week 52
Secondary outcome [3] 0 0
Change From Baseline in Progressive Supranuclear Palsy (PSP)-Cognitive Composite Battery Z-Score at Week 52
Assessment method [3] 0 0
The PSP cognitive composite battery is used to identify and characterize abnormal cognitive decline in PSP participants. The PSP cognitive composite battery includes 13 sub-tests in total: 11 tests from the RBANS (only the picture naming is excluded), letter number sequencing test, and phonemic fluency test. Three domains are identified: Memory and learning, Visual-Motor function, and Working memory and Executive. A z-score transformation is applied for each component test at each visit, and the final total composite z-score is the average of the three-domain z-scores. A z-score of 0 is equal to the estimated mean adjusted by age and is considered average for this study population. Lower values are indicative of cognitive decline. A negative change from baseline indicates worsening.
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Disease Severity (RBANS) Scale at Week 52
Assessment method [4] 0 0
The RBANS provides both a total scale score and scores for 5 different cognitive domains. Specifically, the test measures immediate memory, visuospatial/constructional ability, language, attention, and delayed memory. Scores from all subtests are aggregated into a total composite score. RBANS data were age-normed and analyzed as index scores (also referred to as standard scores), which have a mean of 100 and a standard deviation of 15. Higher scores on each sub measure and index indicate better performance. A negative change from baseline indicates worsening.
Timepoint [4] 0 0
Baseline, Week 52
Secondary outcome [5] 0 0
Change From Baseline in Progressive Supranuclear Palsy Quality of Life Scale (PSP-QoL) Score
Assessment method [5] 0 0
The PSP-QoL is a patient-reported outcome measure developed specifically for assessing the health-related quality of life in people living with PSP. It is validated 45-item questionnaire and visual analog scale that is comprised of 2 subscales: physical health state (22 items), which covers mobility, dysarthria, dysphagia, visual disturbances, self-care, and activities of daily living, and mental health state (23 items), which covers emotional, cognitive and social functioning. Items are given a 6-reponse option format (No Problem, Slight Problem, Moderate Problem, Marked Problem, Extreme Problem and Not Applicable). The subscale results are derived by summing the respective items for that subscale and transforming the scores into a range of 0 to 100, the higher the scores indicating a greater impact of the disease on the aspect measured. The PSP-QoL also comprises of a Life Satisfaction rating gauge, which is a visual analog scale with a range of 0 (worst) to 100 (best).
Timepoint [5] 0 0
Baseline, Week 52
Secondary outcome [6] 0 0
Change From Baseline in Schwab and England Activities of Daily Living (SEADL) Scale Score at Week 48
Assessment method [6] 0 0
The SEADL scale is a means of assessing a person's ability to perform daily activities in terms of speed and independence, with 100% indicating total independence, falling to 0%, which indicates a state of complete dependence. The individual is asked to rate his or her function using an 11-point scale (10% increments), from 100% (completely independent; able to do all chores without slowness, difficulty, or impairment; essentially normal; unaware of any difficulty) to 0% (vegetative functions such as swallowing, bladder and bowels are not functioning; bedridden). A negative change from baseline indicates worsening.
Timepoint [6] 0 0
Baseline, Week 48
Secondary outcome [7] 0 0
Change From Baseline in Clinical Global Impression of Severity (CGI-S) Score at Week 52
Assessment method [7] 0 0
The Clinical Global Impression of Severity (CGI-S) Rating evaluates the severity of individual symptoms and treatment response in participants with mental disorders. The CGI-S is a 7-point scale that that requires the clinician to rate the severity of the patient's illness at the time of assessment. A rating of 1 is considered normal, or with the least severe symptoms, a rating of 7 is extremely ill, or the worst symptoms.
Timepoint [7] 0 0
Baseline, Week 52
Secondary outcome [8] 0 0
Change From Baseline in Phonemic Fluency Test Score at Week 48
Assessment method [8] 0 0
Phonemic fluency is a sensitive test for assessing frontal lobe dysfunction. Participants are given a letter of the alphabet and asked to name as many words as they can that start with that letter in 1 minute. The score for each trial is auto-calculated as follows: Trial 1: Total number of correct responses for the first letter (range 0 to 40); Trial 2: Total number of correct responses for the second letter (range 0 to 40). The total score from the two trials will be used for analysis (range 0 to 80). More number of words correlates to better phonemic fluency. A negative change from baseline indicates worsening.
Timepoint [8] 0 0
Baseline, Week 48
Secondary outcome [9] 0 0
Change From Baseline in Letter-Number Sequencing Test at Week 48
Assessment method [9] 0 0
Letter number is a test of working memory which involves ordering a series of up to 8 letters and numbers in which the numbers are repeated back first in order starting with the lowest number, then followed by the letters in alphabetical order. LNS consists of 10 items and each item has 3 trials rated as Incorrect (0) or Correct (1). The LNS total raw score (range 0 to 30) is auto-calculated by summing the 10 individual item scores (range 0 to 3 for each item). Higher number of correct items correlated to better performance and a negative change from baseline indicates worsening.
Timepoint [9] 0 0
Baseline, Week 48
Secondary outcome [10] 0 0
Change From Baseline in Color Trails at Week 48
Assessment method [10] 0 0
The Color Trails test is a language free version of the Trail Making Test and was developed to allow for broader cross cultural assessment. For Part 1 (color trails test 1), the respondent uses a pencil to rapidly connect circles numbered 1-25 in sequence. For Part 2 (color trails test 2), the respondent rapidly connects number circles in sequence, but alternates between pink and yellow background. The length of time to complete each trial is recorded, along with qualitative features of performance indicative of brain dysfunction, such as near-misses, prompts, number sequence errors, and color sequence errors. Less time indicates better performance. A positive change from baseline indicates worsening.
Timepoint [10] 0 0
Baseline, Week 48
Secondary outcome [11] 0 0
Change From Baseline in Montreal Cognitive Assessment (MoCA) Score at Week 48
Assessment method [11] 0 0
The MOCA was designed as a rapid screening instrument for mild cognitive dysfunction. It assesses different cognitive domains: attention and concentration, executive function, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. Scores on the MOCA range from 0-30, with higher score being better performance. A negative change from baseline indicates worsening.
Timepoint [11] 0 0
Baseline, Week 48
Secondary outcome [12] 0 0
Number of Participants With Treatment Emergent Antibodies (Anti-BIIB092) Positive Results in Serum
Assessment method [12] 0 0
Timepoint [12] 0 0
Up to Week 48
Secondary outcome [13] 0 0
Change From Baseline of Brain Volumes as Determined by MRI at Week 52
Assessment method [13] 0 0
A 3 dimension (3D) T1-weighted MRI was performed to estimate brain volumes (e.g., ventricles, whole brain, midbrain, pons, superior cerebellar peduncle, third ventricle, and frontal lobes).
Timepoint [13] 0 0
Baseline, Week 52

Eligibility
Key inclusion criteria
Key

* 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

Key
Minimum age
41 Years
Maximum age
86 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
7/02/2020
Sample size
Target
Accrual to date
Final
490
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Research Site - North Melbourne
Recruitment postcode(s) [1] 0 0
- North Melbourne
Recruitment outside Australia
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United States of America
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Arizona
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United States of America
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California
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United States of America
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Florida
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United States of America
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Illinois
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United States of America
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Indiana
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United States of America
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Kansas
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United States of America
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Louisiana
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United States of America
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Maryland
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Massachusetts
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Michigan
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Minnesota
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New Jersey
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New York
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Pennsylvania
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South Carolina
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Texas
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Virginia
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Washington
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Austria
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Tirol
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Austria
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Vienna
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Canada
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Ontario
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France
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Bordeaux
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France
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Lille
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Hessen
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Mecklenburg-Western-Pommerania
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Ulm
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Greece
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Campania
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Italy
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Pisa
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Tokyo
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Tottori
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Japan
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Shizuoka
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Korea, Republic of
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Seoul
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Russian Federation
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Krasnoyarsk
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Russian Federation
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Moscow
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Spain
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Navarra
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Vizcaya
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Barcelona
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Madrid
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Sevilla
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Valencia
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Cambridgeshire
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East Sussex
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Hampshire
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Merseyside
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Tyne And Wear
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Wales
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United Kingdom
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London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Biogen
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Biogen
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol
Statistical analysis plan



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT03068468