At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- ALN-APP (drug), Placebo (drug)
- How long the study runs
- Study runs about 86 months (dates as stated)
- About the drug or intervention
- ALN-APP — drug: ALN-APP will be administered intrathecally (IT) · Placebo — drug: Placebo will be administered IT
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 18 Years and over
- Who
- All
- Number of participants
- 60
- Started
- 2022-02-04
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for early-onset alzheimer disease
- • Phase1 - 60 participants
- • 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)
Who can take part?
- • Ages 18 Years and over
- • Diagnosed with early-onset alzheimer disease
Where?
- • Huntley Street - Clinical Trial Site
- • Sheffield - Clinical Trial Site
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years and over
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Has mild cognitive impairment or mild dementia due to EOAD
- ✓Has Clinical Dementia Rating (CDR) global score 0.5 or 1.0 and Mini Mental State Examination (MMSE) \>20
Who cannot take part
- ✗Has Non-Alzheimer's disease dementia
- ✗Has alanine aminotransferase (liver enzymes) or aspartate aminotransferase (liver enzymes) \>2×upper limit of normal (ULN)
- ✗Has estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73m\^2 at Screening
- ✗Has recently received an investigational agent
- ✗Has recent treatment with amyloid-targeting antibody
See the full criteria
Where Is This Study? (2 UK sites)
Clinical Trial Site
Huntley Street WC1E 6AG, United Kingdom
Clinical Trial Site
Sheffield S10 2JF, United Kingdom
How to Get in Touch
Alnylam Clinical Trial Information Line
Sponsor contactCONTACT
Alnylam Clinical Trial Information Line
Sponsor contactCONTACT
