Evaluating ALN-APP for Early-Onset Alzheimer's Disease
A Randomized, Double-blind, Placebo-controlled Single Ascending Dose and Open-label Multi-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-APP in Adult Patients With Early-onset Alzheimer's Disease (EOAD)
This study is testing a new treatment called ALN-APP to see if it is safe and can help people with early-onset Alzheimer's disease.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Alnylam Pharmaceuticals Industry-sponsored |
| Locations | 8 sites (La Jolla, California and 7 other locations) |
| Trial ID | NCT05231785 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety and tolerability of ALN-APP, an investigational treatment, in patients with early-onset Alzheimer's Disease (EOAD). Participants will receive either a single dose or multiple doses of ALN-APP via intrathecal injection, with the first part of the study focusing on a single dose and the second part lasting up to 12 months. The study will also evaluate the pharmacodynamics and pharmacokinetics of the treatment. Patients will be monitored for cognitive impairment and overall health throughout the trial.
Who should consider this trial
Good fit: Ideal candidates include adults with mild cognitive impairment or mild dementia due to early-onset Alzheimer's Disease.
Not a fit: Patients with non-Alzheimer's disease dementia or those with significant liver or kidney dysfunction may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from early-onset Alzheimer's Disease.
How similar studies have performed: While this approach is novel, similar studies targeting Alzheimer's Disease have shown promise, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * Has Non-Alzheimer's disease dementia * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>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 Note: other protocol defined inclusion / exclusion criteria apply
Where this trial is running
La Jolla, California and 7 other locations
- Clinical Trial Site — La Jolla, California, United States (Completed)
- Clinical Trial Site — Indianapolis, Indiana, United States (Recruiting)
- Clinical Trial Site — Montreal, Canada (Recruiting)
- Clinical Trial Site — Toronto, Canada (Recruiting)
- Clinical Trial Site — Amsterdam, Netherlands (Recruiting)
- Clinical Trial Site — Groningen, Netherlands (Completed)
- Clinical Trial Site — Huntley Street, United Kingdom (Recruiting)
- Clinical Trial Site — Sheffield, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Alnylam Clinical Trial Information Line
- Email: clinicaltrials@alnylam.com
- Phone: 1-877-ALNYLAM
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.