Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03186989 | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease | PHASE1 | COMPLETED | 46 | — | — | Oct 12, 2017 | May 12, 2022 | Apr 8, 2025 | 13 | Canada, Finland +4 |
An adverse event (AE) was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of medicinal (investigational) product, whether or not the AE was considered related to the medicinal (investigational) product. A TEAE was defined as any AE that starts or worsens on or after the date of first dose of study treatment. TEAEs were categorised as mild, moderate, and severe to aid in severity assessment.
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of medicinal (investigational) product, whether or not the AE was considered related to the medicinal (investigational) product. A TEAE was defined as any AE that starts or worsens on or after the date of first dose of study treatment. TEAEs were categorised as mild, moderate, and severe to aid in severity assessment.
| Arm | Type | Description |
|---|---|---|
| Part 1: Cohort A: ISIS 814907 10 mg | EXPERIMENTAL | Participants received 10 milligrams (mg) ISIS 814907 diluted in 20 milliliters (mL) artificial cerebrospinal fluid (CSF), intrathecally, every four weeks (Q4W) on Days 1, 29, 57, and 85 in Part 1 of the study. |
| Part 1: Cohort B: ISIS 814907 30 mg | EXPERIMENTAL | Participants received 30 mg ISIS 814907 diluted in 20 mL in artificial CSF, intrathecally, Q4W on Days 1, 29, 57, and 85 in Part 1 of the study. |
| Part 1: Cohort C: ISIS 814907 60 mg | EXPERIMENTAL | Participants received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q4W on Days 1, 29, 57, and 85 in Part 1 of the study. |
| Part 1: Cohort D: ISIS 814907 115 mg | EXPERIMENTAL | Participants received 115 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, every 12 weeks (Q12W) on Days 1 and 85 in Part 1 of the study. |
| Part 1: Pooled Placebo | PLACEBO_COMPARATOR | Participants received 20 mL artificial CSF, intrathecally, as placebo on Days 1, 29, 57, and 85 for the 4-dose regimens, or on Days 1 and 85 for the 2-dose regimens in Part 1 of the study. |
| Part 2: Late Start Cohort A + Cohort B + Cohort C + ISIS 814907 60 mg | EXPERIMENTAL | Participants from MAD Cohorts A, B, C that were placebo-treated, received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Late Start Cohort D + ISIS 814907 115 mg | EXPERIMENTAL | Participants from MAD Cohort D that were placebo-treated, received 115 mg ISIS 814907 diluted up in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Early Start Cohort A + ISIS 814907 60 mg | EXPERIMENTAL | Participants from MAD Cohort A that were ISIS 814907 10 mg -treated, received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Early Start Cohort B + ISIS 814907 60 mg | EXPERIMENTAL | Participants from MAD Cohort B that were ISIS 814907 30 mg-treated, received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Early Start Cohort C + ISIS 814907 60 mg | EXPERIMENTAL | Participants from MAD Cohort C that were ISIS 814907 60 mg-treated, received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Early Start Cohort D + ISIS 814907 115 mg | EXPERIMENTAL | Participants from MAD Cohort D that were ISIS 814907 115 mg-treated, received 115 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Name | Type | Description |
|---|---|---|
| IONIS MAPTRx | DRUG | IONIS MAPTRx injections. |
| Placebo | OTHER | Artificial CSF injections. |
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 ...