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Business reasons (not safety related)
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To evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-KHK and to evaluate the safety, tolerability, efficacy, PK and PD of multiple doses of KHK.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: ALN-KHK | Experimental | Participants will be administered a single dose of ALN-KHK. |
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| Part A: Placebo | Placebo Comparator | Participants will be administered a single dose of placebo. |
|
| Part B: ALN-KHK | Experimental | Participants will be administered a multiple doses of ALN-KHK. |
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| Part B: Placebo | Placebo Comparator | Participants will be administered a multiple doses of placebo. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALN-KHK | Drug | ALN-KHK will be administered by subcutaneous (SC) injection. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Frequency of Adverse Events | Up to 9 Months | |
| Part B: Frequency of Adverse Events | Up to 12 Months |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Area Under the Concentration-time Curve (AUC) of ALN-KHK and Potential Major Metabolite(s) | Up to 2 Days following dosing on Day 1 | |
| Part A: Maximum Observed Plasma Concentration (Cmax) of ALN-KHK and Potential Major Metabolite(s) | Up to 2 Days following dosing on Day 1 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Phoenix | Arizona | 85020 | United States | ||
| Clinical Trial Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41388232 | Derived | Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12. |
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The single ascending dose part of this study (Part A) will have a sequential interventional study model. The multi-dose part of this study (Part B) will have a parallel interventional study model and will begin after the completion of Part A.
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| Placebo | Drug | Placebo will be administered by subcutaneous (SC) injection. |
|
| Part A: Time to Maximum Observed Plasma Concentration (Tmax) of ALN-KHK and Potential Major Metabolite(s) | Up to 2 Days following dosing on Day 1 |
| Part A: Fraction of ALN-KHK Excreted in the Urine (fe) and Potential Major Metabolite(s) | Postdose on Day 1 |
| Part A: Percent Change from Baseline in Circulating Fructose in Response to a Fructose Tolerance Test | Baseline up to Month 6 |
| Part A: Percent Change from Baseline in Urinary Fructose in Response to a Fructose Tolerance Test | Baseline up to Month 6 |
| Part A: Percent Change from Baseline in Circulating Fibroblast Growth Factor 21 (FGF21) in Response to a Fructose Tolerance Test | Baseline up to Month 6 |
| Part B: Absolute Change from Baseline in Hemoglobin A1C (HbA1c) at 6 Months | Baseline and Month 6 |
| Parts A and B: Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) in Response to a Glucose Tolerance Test | Part A: Screening up to Month 3; Part B: Month 4 |
| Part B: Fasting Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) | Month 4 |
| Parts A and B: Glucose and Insulin AUC in response to Tolerance Test | Part A: Screening up to Month 3; Part B: Month 4 |
| Part B: Plasma Concentrations of ALN-KHK and Potential Major Metabolite(s) | Day 1 and Month 3 |
| Vista |
| California |
| 92083 |
| United States |
| Clinical Trial Site | Lake Worth | Florida | 33461 | United States |
| Clinical Trial Site | Orlando | Florida | 32806 | United States |
| Clinical Trial Site | Atlanta | Georgia | 30342 | United States |
| Clinical Trial Site | Berlin | New Jersey | 08009 | United States |
| Clinical Trial Site | Monroe | North Carolina | 28110 | United States |
| Clinical Trial Site | Oklahoma City | Oklahoma | 73112 | United States |
| Clinical Trial Site | Dallas | Texas | 75234 | United States |
| Clinical Trial Site | Houston | Texas | 77036 | United States |
| Clinical Trial Site | San Antonio | Texas | 78229 | United States |
| Clinical Trial Site | Sarnia | Ontario | Canada |
| Clinical Trial Site | Montreal | Quebec | Canada |
| Clinical Trial Site | Victoriaville | Quebec | Canada |
| Clinical Trial Site | Toronto | Canada |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Apr 3, 2026 | Apr 22, 2026 | 21 |
| ID | Term |
|---|---|
| D003924 | Diabetes Mellitus, Type 2 |
| D050177 | Overweight |
| D009765 | Obesity |
| D007333 | Insulin Resistance |
| ID | Term |
|---|---|
| D003920 | Diabetes Mellitus |
| D044882 | Glucose Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D004700 | Endocrine System Diseases |
| D044343 | Overnutrition |
| D009748 | Nutrition Disorders |
| D001835 | Body Weight |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D006946 | Hyperinsulinism |
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