A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

Conditions

• Early-Onset Alzheimer Disease

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• ALN-APP — DRUG
  ALN-APP will be administered intrathecally (IT)
• Placebo — DRUG
  Placebo will be administered IT

Study Details

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment duration for Part B: 12 months.

Key Dates

Start date

Feb 4, 2022

Status verified

May 2026

Primary completion

Apr 20, 2029

Completion

Apr 20, 2029

Study Design

Enrollment

60 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Part A: ALN-APP
  Participants will be administered a single dose of ALN-APP.
• Placebo Comparator: Part A: Placebo
  Participants will be administered a single dose of placebo.
• Experimental: Part B:
  Participants will be administered multiple doses of ALN-APP.

Primary Outcome Measure

Part A: Frequency of Adverse Events [ Time Frame: Up to 12 months ]

Central Contacts

• Alnylam Clinical Trial Information Line
  1-877-ALNYLAM
  [email protected]
• Alnylam Clinical Trial Information Line
  1-877-256-9526
  [email protected]

Locations (2)

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