E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Progressive supranuclear palsy |
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E.1.1.1 | Medical condition in easily understood language |
A rare and progressive condition that can cause problems with balance, movement, vision, speech and swallowing |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10036813 |
E.1.2 | Term | Progressive supranuclear palsy |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the efficacy of BIIB092, compared to placebo, as measured by a change from baseline in the Progressive Supranuclear Palsy Rating Scale (PSPRS) at Week 52.
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. |
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E.2.2 | Secondary objectives of the trial |
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 Parkinsons's Disease Rating Scale (MDS-UPDRS) Part II at Week 52. - the Clinical Global Impression of Change (CGI-C) at Week 52. - a change in baseline in the Repeatable Battery for the Assessment of Neuropsychological Disease Severity (RBANS) at Week 52.
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 (PSP-QoL) scale at Week 52. |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
This study includes an optional substudy to measure quantitative movement assessments (QMAs ) using wearable sensors. The QMAs will measure gait, postural instability, motor function, and falls in a subset of study participants participating in the long-term extension period. |
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E.3 | Principal inclusion criteria |
• Participants with probable or possible Progressive Supranuclear Palsy (PSP) • Able to ambulate independently or with assistance • Able to tolerate Magnetic Resonance Imaging (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 |
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E.4 | Principal exclusion criteria |
• Presence of other significant neurological or psychiatric disorders • Diagnosis of amyotrophic lateral sclerosis (ALS) or other motor neauron disease • History of early, prominent rapid eye movement (REM) sleep behaviour 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Change From Baseline in PSP (Progressive Supranuclear Palsy) Rating Scale (PSPRS) at week 52 2. Percentage of Participants with Death, Serious Adverse Events (SAEs), Adverse Events Leading to Discontinuation, and Grade 3 and 4 Laboratory Abnormalities
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1) Baseline, week 52 2) Up to 52 weeks |
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E.5.2 | Secondary end point(s) |
1. Change From Baseline in Movement Disorder Society (MDS)-sponsored revision of the Unified Parkinson’s Disease Rating Scale (MDSUPDRS) Part II at Week 52. 2. Clinical Global Impression of Change (CGI-C) scale score . 3. Change from Baseline Impact in Repeatable Battery for the Assessment of Neuropsychological Disease Severity (RBANS) scale. 4. Change from Baseline in Progressive Supranuclear Palsy Quality of Life scale (PSP-QoL) scale. 5. Change From Baseline in Schwab and England Activities of Daily Living (SEADL) Scale Score at Week 48 6. Change From Baseline in Clinical Global Impression of Severity (CGIS) Score at Week 52 7. Change From Baseline in Phonemic Fluency Test Score at Week 48 8. Change From Baseline in Letter Number Sequencing Test at Week 48 9. Change From Baseline in Color Trails Test at Week 48 10. Change From Baseline in Montreal Cognitive Assessment (MoCA) Score at Week 48 11. Number of Participants with Drug Antibodies (anti-BIIB092) in Serum 12. Percent Change from Baseline of Brain Volumes as Determined by MRI |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) Baseline, Week 52 2) Week 52 3) Baseline, Week 52 4) Baseline, Week 52 5) Baseline, Week 48 6) Baseline, Week 52 7) Baseline, Week 48 8) Baseline, Week 48 9) Baseline, Week 48 10) Baseline, Week 48 11) Up to Week 52 12) Up to Week 52 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Immunogenicity, biomarker analysis |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Double-blind treatment period followed by an open-label extension period |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 40 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Austria |
Canada |
France |
Germany |
Greece |
Italy |
Japan |
Korea, Republic of |
Russian Federation |
Spain |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The date that the last participant completes the last study visit or scheduled procedure, will be defined as the end of the study. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |