NCT06024408CompletedPhase 1
A Trial to Learn if Receiving ALN-PNP siRNA is Safe and Well Tolerated, and How it Works in Adult Participants With Nonalcoholic Fatty Liver Disease (NAFLD) and a Genetic Risk Factor
A Three-Part, Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Dose Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALN-PNP siRNA in Participants With Nonalcoholic Fatty Liver Disease (NAFLD) and a PNPLA3 Genetic Risk Factor
Source: ClinicalTrials.gov · Last updated 31 Jul 2025
Brief summary (registry, in English)
This study is researching an experimental drug called ALN-PNP. This study is focused on participants who are known to have nonalcoholic fatty liver disease (NAFLD), and a specific variant of the patatin-like phospholipase domain containing 3 (PNPLA3) gene. The aim of this study is to see how safe, tolerable, and effective the study drug is. This study is looking at several other research questions, including: * What side effects may happen from taking the study drug * How the study drug works to change liver fat content in NAFLD * How much study drug and study drug metabolites (byproduct of the body breaking down the study drug) are in your blood at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) * Better understanding of the study drug and NAFLD
- Status (registry)
- Completed
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 9 (actual)
- First submitted
- 16 Aug 2023
- Start date
- 21 May 2024
- Primary completion date
- 7 Jul 2025
- Lead sponsor
- Regeneron Pharmaceuticals
- Diseases
- Fatty liver disease (MASLD/MASH)
- NCT number
- NCT06024408
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 9
- From 21 May 2024 to the primary completion date, 7 Jul 2025
- 5 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Bariatric surgery within approximately 5 years prior or planned during the study period
- Has lost or gained more than 4.0% body weight over the 3 months prior to or during the screening period
Other illness
- Evidence of other forms of known chronic liver disease, as defined in the protocol
- Has a contraindication to MRI examinations, such as persons with cardiac pacemaker and implants made out of metal (for example, cochlear implant, nerve stimulators, magnetic vascular clips, and metallic heart valve), severe claustrophobia, or other contraindications for MRI
- Is taking a medication to treat a co-morbid condition that is not permitted during the study
- History of Type 1 diabetes
- 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
Lab values
- Has any laboratory parameter assessments at screening, as defined in the protocol
Other
- Was hospitalized (ie, \>24 hours) for any reason within 30 days of the screening visit
- Note: Other protocol defined Inclusion/Exclusion Criteria apply
Who the study is looking for
- Participants from 18 (or country's legal age of adulthood) to 65 years of age, inclusive, at screening visit 1
- Body mass index (BMI) from 23.0 kg/m\^2 to 40.0 kg/m\^2, inclusive, for East Asians (including but not limited to South Koreans, Chinese, Taiwanese, and Japanese) and BMI from 27.0 kg/m\^2 to 40.0 kg/m\^2, inclusive, for any other ethnicity at screening visit 1
- Meets genotype criteria for the rs738409:G PNPLA3 risk allele: homozygotes (for Part A and Part B) or heterozygotes (optional Part C); p.I148M variant (PNPLA3 rs738409:G \[p.I148M\]) at screening visit 1
- Liver fat content ≥8.5% as measured by MRI-PDFF at screening visit 3
- Generally stable diet (based on participant's recall) for at least 3 months prior to the screening visit
Age in the registry: 18 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Chungbuk National University Hospital Coordinator of this site? Confirm its status | Cheongju-si | South Korea | Unknown status |
| CHA Bundang Medical Center Coordinator of this site? Confirm its status | Seongnam-si | South Korea | Unknown status |
| Asan Medical Center Coordinator of this site? Confirm its status | Seoul | South Korea | Unknown status |
| Severance hospital Coordinator of this site? Confirm its status | Seoul | South Korea | Unknown status |
| Seoul National University Hospital Coordinator of this site? Confirm its status | Seoul | South Korea | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.