A Double-Blind, Placebo Controlled, Single Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of C2N-8E12 in Subjects With Progressive Supranuclear Palsy
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Sponsor
- Enrollment
- 32
- Locations
- 10
- Primary Endpoint
- 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.
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 50 Years to 85 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
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.
Arms & Interventions
Single dose C2N-8E12 level 2
Single IV infusion of C2N-8E12
Intervention: Single dose C2N-8E12 (Drug)
Single dose C2N-8E12 level 3
Single IV infusion of C2N-8E12
Intervention: Single dose C2N-8E12 (Drug)
Single dose C2N-8E12 level 4
Single IV infusion of C2N-8E12
Intervention: Single dose C2N-8E12 (Drug)
Single dose C2N-8E12 level 1
Single IV infusion of C2N-8E12
Intervention: Single dose C2N-8E12 (Drug)
Single dose placebo
Single IV infusion of placebo
Intervention: Single dose placebo (Drug)
Outcomes
Primary Outcomes
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.
Time Frame: up to 4 months
Secondary Outcomes
- 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)