A Trial to Learn if ALN-PNP is Safe and Well Tolerated in Healthy Adults and Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

156 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-27

Study Completion Date

2027-08-10

Brief Summary

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This study is researching an experimental drug called ALN-PNP (called "study drug"). This is a first in human study. The study drug is not approved by any public health agency such as the United States Food and Drug Administration (FDA) for any kind of treatment.

This study consists of 3 parts. Part A is focused on healthy participants. Parts B and C of the study are focused on participants who are known to have MASLD and a specific variant of the PNPLA3 gene.

The aim of the study is to see how safe, tolerable and effective the study drug is.

The study is looking at several other research questions, including:

* What side effects may happen from taking the study drug (Parts A, B and C)
* How much study drug (Parts A, B and C) and study drug metabolites (byproduct of the body breaking down the study drug) (Parts B and C) are in the blood at different times
* Whether the body makes antibodies against the study drug (which could make the study drug less effective or could lead to side effects) (Part A, B and C)
* Explore impact of Japanese ethnicity on safety and PK (Pharmacokinetics, or study of what the body does to the drug) of single doses of ALN-PNP over time (Part A)
* How the study drug works to change liver fat content in MASLD (Part B and C)
* Better understanding of the study drug and MASLD (Part B and C)

Detailed Description

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Conditions

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Healthy Volunteers Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)

Keywords

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Liver steatosis Fibrosis Hepatocytes Non-Alcoholic Steatohepatitis (NASH) Non-Alcoholic Fatty Liver Disease (NAFLD) Metabolic dysfunction-Associated Steatohepatitis (MASH)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Part A: Sequential ascending dose Part B and Part C: Parallel

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Administered per the protocol

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Part A (Healthy Adults):

1. From 18 to 55 years of age
2. For Japanese cohorts ONLY; the Japanese participant must:

1. Be Japanese, born in Japan, and have both biologic parents and 4 biologic grandparents who are ethnically Japanese and born in Japan
2. Have maintained a Japanese lifestyle, with no significant change since leaving Japan, including having access to Japanese food and adhering to a Japanese diet
3. Be living \<10 years outside of Japan
3. Has a body mass index between 18 and 32 kg/m\^2, inclusive, at the screening visit
4. Is judged by the investigator to be in good health, as described in the protocol
5. Is in good health based on laboratory safety testing obtained at the screening visit and approximately within 24 hours prior to administration of study drug

Part B and Part C (Participants with MASLD):

1. Part B: From 18 to 65 years of age
2. Part C: From 18 to 75 years of age
3. Body mass index (BMI) from 23.0 kg/m2 to 40.0 kg/m2, inclusive, for East Asians (including but not limited to South Koreans, Chinese, Taiwanese, and Japanese) and BMI from 27.0 kg/m2 to 40.0 kg/m2, inclusive, for any other ethnicity at screening visit 1
4. Liver fat content ≥8.5% as measured by MRI-PDFF at screening visit 3

Exclusion Criteria

Part A:

1. History of clinically significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, psychiatric, neurological, or dermatologic disease, as assessed by the investigator, that may confound the results of the study or poses an additional risk to the participant by study participation
2. Presents any concern to the study investigator that might confound the results of the study or poses an additional risk to the participant by their participation in the study
3. Hospitalized for any reason within 30 days of the screening visit
4. Using the Modification of Diet in Renal Disease equation, has a glomerular filtration rate as described in the protocol at the screening visit
5. Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) or total bilirubin above the upper limit of normal (ULN) range
6. Is a current smoker or former smoker, including e-cigarettes, who stopped smoking within 3 months prior to the screening visit
7. Has a history of alcohol or drug abuse per investigator opinion
8. Is positive for hepatitis C antibody and if so, positive for qualitative (ie, detected or not detected) hepatitis C virus ribonucleic acid (RNA) test at the screening visit

Part B and Part C:

1. Evidence of other forms of known chronic liver disease, as defined in the protocol
2. Has a contraindication to MRI examinations, as defined in the protocol
3. History of Type 1 diabetes
4. Has lost or gained more than 4.0% body weight over the 3 months prior to or during the screening period
5. Has known human immunodeficiency virus (HIV) infection, evidence of current or chronic hepatitis B virus (HBV) infection, or current or chronic hepatitis C virus (HCV) infection, as defined in the protocol
6. Bariatric surgery within approximately 5 years (Part B) or 3 years (Part C) prior or planned during the study period

Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trial Management

Role: STUDY_DIRECTOR

Regeneron Pharmaceuticals

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Clinical Trials Administrator

Role: CONTACT

Phone: 844-734-6643

Email: [email protected]

Other Identifiers

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ALN-PNP-HV-2227

Identifier Type: -

Identifier Source: org_study_id

A Trial to Learn if ALN-PNP is Safe and Well Tolerated in Healthy Adults and Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

Part A: Cohorts

ALN-PNP

Placebo (PB)

Part A: Optional Cohort

ALN-PNP

Placebo (PB)

Part A: JPN Cohorts

ALN-PNP

Placebo (PB)

Part B: Cohorts

ALN-PNP

Placebo (PB)

Part C: Cohorts

ALN-PNP

Placebo (PB)

Interventions

ALN-PNP

Placebo (PB)

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Regeneron Pharmaceuticals

Responsible Party

Principal Investigators

Clinical Trial Management

Locations

California Clinical Trials Medical Group

Velocity Clinical research

Genoma Research Group, Inc

Med Research of Florida, LLC

Tandem Clinical Research

Pioneer Research Solutions

Countries

Central Contacts

Clinical Trials Administrator

Other Identifiers

ALN-PNP-HV-2227