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The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALN-HTT02 | Experimental | Participants will be administered a single dose of ALN-HTT02 during the Double-blind Part of the study. Participants who received ALN-HTT02 in the Double-blind part of the study will not receive an additional dose during the Open-label Part of the study. Participants have the option to receive repeat doses of ALN-HTT02 in the Open-label Extension (OLE) Part of the study. |
|
| Placebo + ALN-HTT02 | Placebo Comparator | Participants will be administered a single dose of placebo during the Double-blind Part of the study. Participants will have the option to receive a single dose of ALN-HTT02 during the Open-label Part of the study. Participants have the option to receive repeat doses of ALN-HTT02 in the OLE Part of the study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALN-HTT02 | Drug | ALN-HTT02 will be administered intrathecally |
|
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of Adverse Events (AEs) in the Double-blind Part of the Study | Up to 12 months | |
| Frequency of AEs in the Open-label Part of the Study | Up to 12 months | |
| Frequency of AEs in the OLE Part of the Study | Up to 36 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Levels of Mutant Huntingtin (mHTT) in Cerebrospinal Fluid (CSF) | Baseline up to Month 12 in the Double-blind Part of the study; Baseline up to Month 12 in the Open-label Part of the study ; Baseline up to Month 36 in the OLE Part of the study | |
| Concentrations of ALN-HTT02 in Plasma |
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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 | Recruiting | Edmonton | T6G 2B7 | Canada | ||
| Clinical Trial Site |
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| Placebo | Drug | Placebo will be administered intrathecally |
|
Area Under the Plasma Concentration-time Curve (AUC) Maximum Observed Plasma Concentration (Cmax) Time to Maximum Plasma Concentration (Tmax)
| Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study; Up to Month 36 in the OLE Part of the study |
| Concentrations of ALN-HTT02 in Cerebrospinal Fluid (CSF) | Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study ; Up to Month 36 in the OLE Part of the study |
| Concentrations of ALN-HTT02 in Urine | Fractional Excretion of ALN-HTT02 | Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study; Up to Month 36 in the OLE Part of the study |
| Recruiting |
| Montreal |
| H2W 1T8 |
| Canada |
| Clinical Trial Site | Recruiting | Ottawa | K1Y4E9 | Canada |
| Clinical Trial Site | Recruiting | Vancouver | V6T 2B5 | Canada |
| Clinical Trial Site | Recruiting | Berlin | 10117 | Germany |
| Clinical Trial Site | Recruiting | Bochum | 44791 | Germany |
| Clinical Trial Site | Recruiting | Bonn | 53127 | Germany |
| Clinical Trial Site | Recruiting | Dresden | 01307 | Germany |
| Clinical Trial Site | Recruiting | Münster | 48149 | Germany |
| Clinical Trial Site | Recruiting | Taufkirchen | 84416 | Germany |
| Clinical Trial Site | Recruiting | Ulm | 89081 | Germany |
| Clinical Trial Site | Recruiting | Birmingham | B15 2TT | United Kingdom |
| Clinical Trial Site | Recruiting | Cambridge | CB2 0PY | United Kingdom |
| Clinical Trial Site | Recruiting | Cardiff | CF103AX | United Kingdom |
| Clinical Trial Site | Recruiting | Glasgow | G51 4TF | United Kingdom |
| Clinical Trial Site | Recruiting | Greater Manchester | M13 9WL | United Kingdom |
| Clinical Trial Site | Recruiting | London | WC1N 3BG | United Kingdom |
| Clinical Trial Site | Recruiting | Oxford | OX3 9DU | United Kingdom |
| Clinical Trial Site | Recruiting | Plymouth | PL6 8DH | United Kingdom |
| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| D019636 | Neurodegenerative Diseases |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D003704 | Dementia |
| D002819 | Chorea |
| D020820 | Dyskinesias |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
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