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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-508363-79-00 | Other Identifier | EU CT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: ALN-APP | Experimental | Participants will be administered a single dose of ALN-APP. |
|
| Part A: Placebo | Placebo Comparator | Participants will be administered a single dose of placebo. |
|
| Part B: | Experimental | Participants will be administered multiple doses of ALN-APP. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALN-APP | Drug | ALN-APP will be administered intrathecally (IT) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Frequency of Adverse Events | Up to 12 months | |
| Part B: Frequency of Adverse Events | Up to 42 months |
| Measure | Description | Time Frame |
|---|---|---|
| Part A and Part B: Change from Baseline in Levels of Cerebrospinal Fluid (CSF) Soluble Amyloid Precursor Protein Alpha (sAPPα) and Soluble Amyloid Precursor Protein Beta (sAPPβ) | Part A up to 12 months; Part B up to 24 months | |
| Part A and Part B: Area Under the Plasma Concentration-time Curve (AUC) of ALN-APP and of Potential Metabolites |
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Inclusion Criteria:
Exclusion Criteria:
Note: other protocol defined inclusion / exclusion criteria apply
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Alnylam Clinical Trial Information Line | Contact | 1-877-ALNYLAM | clinicaltrials@alnylam.com | |
| Alnylam Clinical Trial Information Line | Contact | 1-877-256-9526 | clinicaltrials@alnylam.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Completed | La Jolla | California | 92037 | United States | |
| Clinical Trial Site |
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| ID | Term |
|---|---|
| D000544 | Alzheimer Disease |
| ID | Term |
|---|---|
| D003704 | Dementia |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| Placebo | Drug | Placebo will be administered IT |
|
| Part A up to 12 months; Part B up to 24 months |
| Part A and Part B: Maximum Observed Plasma Concentration (Cmax) of ALN-APP and of Potential Metabolites | Part A up to 12 months; Part B up to 24 months |
| Part A: Fraction of ALN-APP and Potential Metabolites Excreted in the Urine (fe) | Up to 1 day |
| Part A and Part B: Concentration of ALN-APP and Potential Metabolites at Time 't' (Ct) in Plasma and CSF | Part A up to 12 months; Part B up to 24 months |
| Recruiting |
| Indianapolis |
| Indiana |
| 46202 |
| United States |
| Clinical Trial Site | Recruiting | Montreal | H3A 2B4 | Canada |
| Clinical Trial Site | Recruiting | Toronto | M3B 2S7 | Canada |
| Clinical Trial Site | Recruiting | Amsterdam | 1081 BT | Netherlands |
| Clinical Trial Site | Completed | Groningen | 9713 GG | Netherlands |
| Clinical Trial Site | Recruiting | Huntley Street | WC1E 6AG | United Kingdom |
| Clinical Trial Site | Recruiting | Sheffield | S10 2JF | United Kingdom |
| D024801 |
| Tauopathies |
| D019636 | Neurodegenerative Diseases |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |