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New RNA drug targets fatty liver in people with specific gene

NCT ID NCT05648214

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental drug called ALN-PNP in healthy adults and people with a liver condition called MASLD who have a specific gene variant. The main goal is to check safety and how the body handles the drug. It is a first-in-human trial, so the drug is not yet approved for any use.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ALN-PNP (an experimental RNA-based drug)
What this could lead to
If successful, this could point toward a treatment that reduces liver fat in people with MASLD who have a specific gene variant.
What could go wrong
This is a very early, first-in-human study, so safety and effectiveness are not yet known. The drug may not work or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 172 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2022

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key 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 (BMI) 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. 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 Key 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 (HCV) 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 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 NOTE: Other protocol defined inclusion / exclusion criteria apply

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    5 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • California Clinical Trials Medical Group

    COMPLETED

    Glendale, California, 91206, United States

  • Genoma Research Group, Inc

    RECRUITING

    Miami, Florida, 33173, United States

  • Med Research of Florida, LLC

    RECRUITING

    Miami, Florida, 33186, United States

  • Pioneer Research Solutions

    RECRUITING

    Houston, Texas, 77099, United States

  • Tandem Clinical Research

    RECRUITING

    Marrero, Louisiana, 70072, United States

  • Velocity Clinical research

    RECRUITING

    Los Angeles, California, 90057, United States

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