A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04565717 |
Recruitment Status :
Active, not recruiting
First Posted : September 25, 2020
Last Update Posted : January 17, 2023
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Nonalcoholic Steatohepatitis NASH | Drug: ALN-HSD Drug: Placebo | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 103 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1, Randomized, Double-Blind, Placebo-Controlled, 2-Part Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Dose ALN-HSD in Healthy Adult Subjects and Multiple Dose ALN-HSD in Adult Patients With Nonalcoholic Steatohepatitis (NASH) |
Actual Study Start Date : | October 9, 2020 |
Estimated Primary Completion Date : | January 4, 2023 |
Estimated Study Completion Date : | January 4, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: Part A: ALN-HSD
Participants will be administered a single dose of ALN-HSD.
|
Drug: ALN-HSD
ALN-HSD will be administered by subcutaneous (SC) injection. |
Placebo Comparator: Part A: Placebo
Participants will be administered a single dose of ALN-HSD-matching placebo.
|
Drug: Placebo
Normal saline (0.9% NaCl) matching volume of ALN-HSD doses will be administered SC. |
Experimental: Part B: ALN-HSD
Participants will be administered multiple doses of ALN-HSD.
|
Drug: ALN-HSD
ALN-HSD will be administered by subcutaneous (SC) injection. |
Placebo Comparator: Part B: Placebo
Participants will be administered multiple doses of ALN-HSD-matching placebo.
|
Drug: Placebo
Normal saline (0.9% NaCl) matching volume of ALN-HSD doses will be administered SC. |
- Frequency of Adverse Events [ Time Frame: Part A: Up to 3.5 months; Part B: up to 12.5 months ]
- Area Under the Plasma Concentration-time Curve (AUC) for ALN-HSD and Potential Metabolites [ Time Frame: Part A: Day 1 predose and up to 48 hours postdose; Part B: Day 1 and Month 3 predose and up to 4 hours postdose ]
- Maximum Plasma Concentration (Cmax) for ALN-HSD and Potential Metabolites [ Time Frame: Part A: Day 1 predose and up to 48 hours postdose; Part B: Day 1 and Month 3 predose and up to 4 hours postdose ]
- Fraction Excreted in Urine (fe) of ALN-HSD and Potential Metabolites [ Time Frame: Part A and B: Day 1 up to 24 hours postdose ]
- Part B: Change from Baseline of Liver Hydroxysteroid 17β Dehydrogenase 13 (HSD17B13) Messenger Ribonucleic Acid (mRNA) [ Time Frame: Predose and up to 9 months postdose ]Hepatic HSD17B13 mRNA will be measured by quantitative reverse-transcription polymerase chain reaction using ribonucleic acid (RNA) isolated from liver biopsy.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
Inclusion Criteria:
-
Part A Only
- Has body mass index (BMI) ≥18 kg/m^2 and ≤28 kg/m^2
- Has normal 12-lead electrocardiogram (ECG)
-
Part B Only:
- Has BMI ≥18 kg/m^2 and ≤40 kg/m^2
- Has a diagnosis of NASH documented in the patient's medical history or a clinical suspicion of NASH based on defined study criteria
- Has screening liver biopsy with NASH activity score (NAS) score of ≥3 per NASH Clinical Research Network (CRN) criteria
Exclusion Criteria:
-
Parts A and B:
- Has any clinical safety laboratory result considered clinically significant and unacceptable by the Investigator
- Has known active human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV) infection
- Has known history or evidence of drug abuse, within 12 months prior to screening
- Has evidence of other forms of known chronic liver disease
- Has recently received an investigational agent
- Has any uncontrolled or serious disease, medical or surgical condition that my interfere with participation or data interpretation
- Has excessive alcohol intake for ≥ 3 months during past year
- Has history of intolerance to SC injection(s)
- Has international normalized ratio (INR) >1.2
- Has platelet count <140x10^9/L
-
Part A Only
- Has systolic blood pressure (BP) >140 mmHg and diastolic >90 mmHg;
- Has used certain prescription drugs within last 14 days prior to screening
- Has used certain over the counter (OTC) medication within 7 days prior to screening
- Has estimated glomerular filtration rate (GFR) ≤60 mL/min/1.73m^2 at screening
-
Part B Only
- Has abnormal ECG
- Has changes in certain prescription medications defined in the protocol within the specified timeframe prior to screening
- Has GFR<45ml/min/1.73m^2

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04565717
United States, Florida | |
Clinical Trial Site | |
Fleming Island, Florida, United States, 32003 | |
United States, Louisiana | |
Clinical Trial Site | |
Marrero, Louisiana, United States, 70072 | |
United States, Maryland | |
Clinical Trial Site | |
Baltimore, Maryland, United States, 21202 | |
United States, Tennessee | |
Clinical Trial Site | |
Hermitage, Tennessee, United States, 37076 | |
United States, Texas | |
Clinical Trial Site | |
San Antonio, Texas, United States, 78215 | |
Clinical Trial Site | |
San Antonio, Texas, United States, 78229 | |
Clinical Trial Site | |
San Antonio, Texas, United States, 78230 | |
Belgium | |
Clinical Trial Site | |
Brussels, Belgium | |
Bulgaria | |
Clinical Trial Site | |
Sofia, Bulgaria | |
Turkey | |
Clinical Trial Site | |
Balçova, Turkey | |
United Kingdom | |
Clinical Trial Site | |
Edinburgh, United Kingdom | |
Clinical Trial Site | |
London, United Kingdom |
Study Director: | Medical Director | Alnylam Pharmaceuticals |
Responsible Party: | Alnylam Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT04565717 |
Other Study ID Numbers: |
ALN-HSD-001 2020-000847-29 ( EudraCT Number ) |
First Posted: | September 25, 2020 Key Record Dates |
Last Update Posted: | January 17, 2023 |
Last Verified: | January 2023 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Hepatobiliary disorders Non-alcoholic fatty liver disease NAFLD Fatty liver RNAi therapeutic |
Fatty Liver Non-alcoholic Fatty Liver Disease Liver Diseases Digestive System Diseases |