Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease

Ionis Pharmaceuticals

Status and phase

Completed

Phase 2

Phase 1

Conditions

Mild Alzheimer's Disease

Treatments

Other: Placebo

Drug: IONIS MAPTRx

Study type

Interventional

Funder types

Industry

Identifiers

NCT03186989

ISIS 814907-CS1

2016-002713-22 (EudraCT Number)

NL60032.000.16 (Other Identifier)

Details and patient eligibility

About

The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS-MAPTRx in patients with Mild Alzheimer's Disease

Full description

This is a randomized, double-blind, placebo-controlled study, followed by an Open-Label Extension in up to 44 participants. This study will consist in two parts:

Part 1: a randomized, double-blind, placebo-controlled multiple ascending dose period in participants with Mild Alzheimer's Disease, followed by Part 2: the open-label, long-term extension period.

Enrollment

46 patients

Sex

All

Ages

50 to 74 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria for Part 1:

Males or females aged 50-74 years, inclusive, at the time of informed consent
Diagnosed with mild Alzheimers disease, including CSF biomarkers consistent with this diagnosis
Body Mass Index BMI ≥ 18 and ≤ 35 kg/m2 and total body weight > 50 kg (110 lbs)
Able and willing to meet all study requirements, including toleration for MRI scans, blood draws and lumbar punctures, travel to Study Center and participation in all procedures and measurements at study visits
Have a trial partner who is reliable, competent and at least 18 years of age, is willing to accompany the patient to select trial visits and to be available to the Study Center by phone if needed
Reside within 4 hours travel of the Study Center

Exclusion Criteria for Part 1:

Treatment with another Study Drug, biological agent, or device within one-month of Screening or 5 half-lives of investigational agent, whichever is longer
Clinically significant medical condition which would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study
Use of a disallowed CNS-active or antipsychotic medication within 4 weeks prior to Screening punctures

Inclusion Criteria for Part 2:

Must have completed the Treatment Evaluation and Post-Treatment Periods in Part 1

Exclusion Criteria for Part 2 (only applicable to participants in Cohorts A and B, as participants from Cohorts C and D will seamlessly transition to Part 2):

Treatment with another Study Drug, biological agent, or device within one-month of Screening or 5 half-lives of investigational agent, whichever is longer
Clinically significant medical condition which would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study
Use of a disallowed CNS-active or antipsychotic medication within 4 weeks prior to Screening punctures