A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)

May 16, 2024 updated by: Alnylam Pharmaceuticals

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, 3-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)

The purpose of this study is to evaluate the safety and tolerability of single ascending doses of ALN-HSD in healthy participants (Part A) and multiple doses of ALN-HSD in patients with NASH (Parts B and C).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
6

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Belgium
      • Brussels, Belgium
        • Clinical Trial Site
  • Bulgaria
      • Sofia, Bulgaria
        • Clinical Trial Site
  • Turkey
      • Balçova, Turkey
        • Clinical Trial Site
      • İzmir, Turkey
        • Clinical Trial Site
  • United Kingdom
      • Edinburgh, United Kingdom
        • Clinical Trial Site
      • London, United Kingdom
        • Clinical Trial Site
  • United States
    • Florida
      • Fleming Island, Florida, United States, 32003
        • Clinical Trial Site
    • Louisiana
      • Marrero, Louisiana, United States, 70072
        • Clinical Trial Site
    • Maryland
      • Baltimore, Maryland, United States, 21202
        • Clinical Trial Site
    • Tennessee
      • Hermitage, Tennessee, United States, 37076
        • Clinical Trial Site
    • Texas
      • San Antonio, Texas, United States, 78229
        • Clinical Trial Site
      • San Antonio, Texas, United States, 78215
        • Clinical Trial Site
      • San Antonio, Texas, United States, 78230
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Description

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)

  • Parts B and C 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, B and C:

    • 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) <90 mL/min/1.73m^2 at screening

  • Parts B and C 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.
Experimental: Part C: ALN-HSD
Participants will be administered multiple doses of ALN-HSD.
Drug: ALN-HSD
ALN-HSD will be administered by subcutaneous (SC) injection.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Parts A and B: Frequency of Adverse Events
Time Frame: Part A: Up to 3.5 months; Part B: up to 12.5 months
Part A: Up to 3.5 months; Part B: up to 12.5 months
Part C: Change from Baseline of Liver Hydroxysteroid 17β Dehydrogenase 13 (HSD17B13) Messenger Ribonucleic Acid (mRNA)
Time Frame: Baseline and Month 6
Baseline and Month 6

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Part A: Area Under the Plasma Concentration-time Curve (AUC) for ALN-HSD and Potential Metabolites
Time Frame: Day 1 predose and up to 48 hours postdose
Day 1 predose and up to 48 hours postdose
Part B: Change from Baseline of Liver HSD17B13 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.
Predose and up to 9 months postdose
Part C: Frequency of Adverse Events
Time Frame: Up to 6 months
Up to 6 months
Pat A: Maximum Plasma Concentration (Cmax) for ALN-HSD and Potential Metabolites
Time Frame: Day 1 predose and up to 48 hours postdose
Day 1 predose and up to 48 hours postdose
Part A: Fraction Excreted in Urine (fe) of ALN-HSD and Potential Metabolites
Time Frame: Day 1 up to 24 hours postdose
Day 1 up to 24 hours postdose
Part B: Plasma Concentrations of ALN-HSD and Potential Major Metabolite(s)
Time Frame: Day 1 and Month 3 predose and up to 4 hours postdose
Day 1 and Month 3 predose and up to 4 hours postdose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alnylam Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 9, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 6, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 21, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 21, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 22, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 25, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 17, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 16, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ALN-HSD-001
  • 2020-000847-29 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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