Progressive Supranuclear Palsy
Conditions
Keywords
PSP, C2N-8E12, tauopathy, Progressive Supranuclear Palsy, Steele Richardson Olszewski Syndrome, Humanized anti-tau antibody
Brief summary
This study will evaluate the safety and tolerability (maximum tolerated dose (MTD) within the specified dosing range) of single intravenous (IV) infusion of C2N-8E12 in patients with progressive supranuclear palsy (PSP).
Detailed description
This study evaluates the safety, tolerability, pharmacokinetics, and maximum tolerated dose (within dosing range) of intravenous (IV) infusion of C2N-8E12 in 32 patients with progressive supranuclear palsy (PSP). Four sequential cohorts will receive increasing single doses of either C2N-8E12 or placebo. Out of every 4 patients enrolled 3 patients will receive drug and 1 will receive placebo. Study participants will be followed for a minimum of 2 months post-treatment to monitor for the safety, tolerability, pharmacokinetics, and immunogenicity of C2N-8E12.
Interventions
DRUGSingle dose C2N-8E12
C2N-8E12 is a humanized recombinant anti-human tau antibody.
Subjects will be block randomized to receive a single dose of C2N-8E12 or placebo in two blocks of 4 subjects (3:1, C2N-8E12:placebo) per cohort.
Sponsors
C2N Diagnostics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
50 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Meets NINDS-SPSP possible or probable criteria as modified for NNIPPS and AL-108-231 clinical trials * Brain MRI at Screening is consistent with PSP; * Stable medications for Parkinsonism for at least 2 months prior to Screening; * Agree to use protocol specified methods of contraception. Key
Exclusion criteria
* Signs of a progressive neurological disorder that better meets the criteria for types of neurological disorders other than PSP; * Currently on any other biologic or immunomodulatory therapy; * Subjects that reside at a skilled nursing or dementia care facility; * Diagnosis of any other significant unrelated neurological or psychiatric disorders that could account for cognitive deficits; * Untreated major depression at baseline evaluation, based on clinical judgment and results in geriatric depression scale; * Unable to tolerate MRI scan at Screening or any other contraindication to MRI; * Any contraindication to or unable to tolerate lumbar puncture at Screening, including use of anti-coagulant medications.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability, as measured by number of participants experiencing adverse events (AEs), serious AEs, and abnormalities in clinical laboratory tests, vital signs, ECGs, MRI, and physical and neurological exams. | up to 4 months |
Secondary
| Measure | Time frame |
|---|---|
| Immunogenicity as measured by the number of participants developing anti drug antibodies. | up to 4 months |
| Area under the concentration vs time curve (AUC) of C2N-8E12 | up to 4 months |
| Elimination half-life of C2N-8E12 | up to 4 months |
Countries
United States
Outcome results
None listed