Clinical trial NCT02658916: Extension Study of BIIB092 in Participants With Progressive Supranuclear Palsy (PSP) Who Participated in CN002003
Official title: A Multicenter, Open-Label, Long-Term Treatment Study of Intravenously Administered BIIB092 in Patients With Progressive Supranuclear Palsy Who Participated in Study CN002003
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Supranuclear Palsy, Progressive
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2016-01-20
Enrollment: 47
Sex: ALL
Minimum age (years): 41
Maximum age (years): 86
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the long-term safety and tolerability of multiple intravenous (IV) infusions of BIIB092 in participants with Progressive Supranuclear Palsy (PSP). The study will also assess the pharmacodynamic (PD) effects of BIIB092 on cerebrospinal fluid (CSF) N-terminal tau, pharmacokinetics (PK), and immunogenicity of BIIB092 in participants with PSP.

Detailed description: This study, previously posted by Bristol-Myers Squibb, has transitioned to Biogen under a licensing agreement.

Eligibility criteria: Key Inclusion Criteria:

1. Completed treatment in Study CN002003.
2. A diagnosis of probable or possible PSP consistent with Study CN002003 criteria with no new medical information or diagnoses since enrollment into Study CN002003 that might confer doubt on the PSP diagnosis.
3. Able to tolerate Magnetic Resonance Imaging (MRI).
4. Able to perform all protocol-specified assessments and comply with the study visit schedule.
5. Have reliable caregiver to accompany patient to all study visits. Caregiver must be able to read, understand, and speak local language fluently to ensure comprehension of informed consent and informant-based assessments of patient. Caregiver must also have frequent contact with patient (at least 3 hours per week at one time or at different times) and be willing to monitor the patient's health and concomitant medications throughout the study.
6. Patient must reside outside a skilled nursing facility or dementia care facility at the time of enrollment
7. Women of childbearing potential (WOCBP) and sexually active fertile men with partners who are WOCBP must use highly effective birth control.

Key Exclusion Criteria:

1. Presence of an unstable, clinically significant medical condition other than PSP including, but not limited to: hematological, endocrine, cardiovascular, renal, hepatic, gastrointestinal, immunological, psychological or neurological disease or malignancy.
2. Contraindication to undergoing a lumbar puncture (LP).
3. Recent drug or alcohol abuse as defined in Diagnostic and Statistical Manual of Mental Disorders (4th Edition).
4. Treatment with any investigational drugs (including placebo) other than BIIB092 or placebo given in Study CN002003 or devices within 90 days prior to enrollment
5. Any vaccination within 30 days prior to study drug administration.
6. Contraindication to the MRI examination for any reason
7. Any other sound medical, psychiatric, and/or social reason as determined by the investigator
8. Known history of human immunodeficiency virus.
9. Evidence of organ dysfunction or significant deterioration from prior values in CN002003 beyond what is consistent with the target population or that would place the patient at increased risk or risk of early study discontinuation.
10. Inability to be venipunctured and/or tolerate venous access.
11. History of allergy, hypersensitivity, or serious adverse reaction to monoclonal antibodies or related compounds or allergy to any of the components of the study drug

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Primary outcomes:
measure: Percentage of Participants with Adverse Events (AEs), Serious Adverse Events (SAEs), AEs Leading to Discontinuation, Deaths
timeFrame: Day 1 through study completion (approximately 1.5 years or later)
measure: Percentage of Participants with Marked Abnormalities in Clinical Laboratory Tests, Vital sign Measurements, ECGs, and Physical and Neurological Examinations
timeFrame: Day 1 through study completion (approximately 1.5 years or later)
Secondary outcomes:
measure: Serum Trough Concentration (C-trough) of BIIB092
timeFrame: Up to study completion (approximately 1.5 years or later)
measure: End-of-Infusion Serum Concentration of BIIB092
timeFrame: Day 1 through study completion (approximately 1.5 years or later)
measure: Number of Participants with drug antibodies (anti-BIIB092) in Serum
timeFrame: Up to study completion (approximately 1.5 years or later)
measure: Percent Change from Baseline in Cerebrospinal Fluid (CSF) Concentrations of unbound N-terminal Tau at Week 48
timeFrame: Baseline, Week 48
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The University of Alabama at Birmingham
state: Alabama
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: Los Angeles
country: United States
facility: David Geffen School of Medicine at UCLA
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco, Medical Center at Parnassus
state: California
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton, Inc.
state: Florida
city: Gainesville
country: United States
facility: University of Florida College of Medicine
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Chicago
country: United States
facility: The University of Chicago Medical Center
state: Illinois
city: Minneapolis
country: United States
facility: University of Minnesota Medical School
state: Minnesota
city: New Brunswick
country: United States
facility: Robert Wood Johnson Medical School
state: New Jersey
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Dallas
country: United States
facility: The University of Texas Southwestern Medical Center
state: Texas
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: