Progressive Supranuclear Palsy Trial with C2N-8E12

  • Study Director: Adam Boxer, MD, PhD
  • Sponsor: C2N Diagnostics
  • Recruiting? No
  • Official Study Title: A Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of C2N-8E12 in Subjects with Progressive Supranuclear Palsy
  • ClinicalTrials.gov Identifier: NCT02494024
  • Conditions Studied: Progressive supranuclear palsy (PSP)
  • Intervention: C2N-8E12 or placebo control will be administered once as an intravenous infusion; subjects will be monitored for up to two months post-infusion to monitor for safety, tolerability, pharmacokinetics, and immunogenicity of C2N-8E12
  • Phase: Phase 1
  • Duration of Participation: Approximately eight visits over a 2- to 4-month period

Purpose of the Study

The primary purpose of this study is to determine the safety, tolerability, and maximally tolerated dose of C2N-8E12, and anti-tau monoclonal antibody. C2N-8E12 is an immunotherapy (antibody) drug designed to bind to and to remove tau protein. The tau protein is believed to build up to abnormally high levels in the brains of individuals with PSP. This abnormal accumulation is associated with clinical progression of the disease.

Eligibility

Inclusion Criteria: Subjects must be between 50–85 years of age and be able to walk 5 steps with minimal assistance (stabilization of one arm or use of cane/walker). Subjects must meet NINDS-SPSP possible or probable PSP criteria and be willing to have two brain MRI scans, as well as two lumbar punctures, performed; must have a reliable caregiver who has at least 5 hours of contact per week and is willing to accompany the subject to study visits; and must have stable medications for Parkinsonism for at least 2 months prior to screening.

Exclusion Criteria: Subjects must not have signs of a progressive neurological disorder that better meets criteria for types of neurological disorders other than PSP; must not be currently on any other biologic or immunomodulatory therapy; must not reside at a skilled nursing or dementia care facility; or have diagnosis of any other significant unrelated neurological or psychiatric disorders that could account for cognitive deficits; or have disease duration greater than 5 years since onset of symptoms.

What to Expect

Testing: Two brain MRIs, two lumbar punctures, neurological and physical examinations, cognitive testing and neuropsychiatric assessments, ECGs, blood and urine specimen collection, vital signs

The Frequency of Visits:

  • Screening phase: 2–3 visits within 56 days of C2N-8E12 infusion
  • Dose administration: One intravenous infusion followed by overnight stay at UCSF
  • Follow-up: Safety visits on days 2, 7, 14, 28, and 56 after infusion day

Materials Needed Before Evaluation: Participants meet criteria for probable or possible PSP (based on the National Institute of Neurological Disorders and Stroke & Society for Progressive Supranuclear Palsy).

Costs: No costs will be charged for any of the study procedures. Parking will be validated at UCSF public garages for all study visits, and subjects will be reimbursed $75 for each scheduled visit and $75 for additional MRI scanning and/or lumbar puncture visits to help defray any incidental expenses incurred in the course of participating in this study.

Contact Information

Clinical Trials Nurse: Mary Koestler, RN, PhD, CCRC – Mary.Koestler@ucsf.edu, 415.476.0661