Evaluating ALN-CIDEB for liver disease in adults
A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Two-Part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of a Single Dose of ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) and Two Doses of ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatohepatitis (MASH)
This study is testing a new drug called ALN-CIDEB to see if it is safe and helpful for adults with certain liver diseases.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 132 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Regeneron Pharmaceuticals Industry-sponsored |
| Locations | 3 sites (Chandler, Arizona and 2 other locations) |
| Trial ID | NCT06836609 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and tolerability of an experimental drug called ALN-CIDEB in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH). Participants will be monitored for side effects, changes in liver fat content, and the drug's metabolism in the body. The study includes two parts, with specific age and health criteria for each group. It aims to provide insights into the potential treatment of these liver conditions.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 to 65 with a diagnosis of MASLD or MASH and a BMI between 30 and 40.
Not a fit: Patients with liver disease not associated with metabolic dysfunction or those outside the specified age and BMI range may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to a new treatment option for patients suffering from MASLD and MASH.
How similar studies have performed: Other studies have explored treatments for liver diseases, but the specific approach of using ALN-CIDEB is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: 1. Part A: 18 to 55 years at Screening Visit 1 with MASLD, at Screening Visit 1 Part B: 18 to 65 years at Screening Visit 1 with a diagnosis of MASH, at Screening Visit 1 2. Body Mass Index (BMI) ≥30 kg/m2 and ≤40 kg/m2 at Screening Visit 1 3. Controlled-Attenuation Parameter (CAP) ≥285 dB/m by FibroScan during screening as described in the protocol 4. Liver fat content ≥8.5% by MRI-PDFF during screening 5. If on anti-hypertensive and/or lipid lowering medications and/or glucose lowering medications, must be on generally stable dose(s) for at least 12 weeks prior to screening and no changes to the dose(s) are anticipated during the study 6. Part B: A diagnosis of MASH documented in the participant's medical history, or a clinical suspicion of MASH based on non-invasive biomarkers (eg, evidence of fatty liver on imaging and elevated liver enzymes) and clinical risk factors, including having a history of 2 or more elements of metabolic syndrome, as defined in the protocol 7. Part B: Screening percutaneous liver biopsy NAFLD Activity Score (NAS) ≥3 and fibrosis stage, as defined in the protocol Key Exclusion Criteria: 1. Known historical or current diagnosis of portal hypertension or cirrhosis based on clinical assessment, imaging, and/or liver biopsy 2. Known historical or current diagnosis of other forms of chronic liver disease, as defined in the protocol 3. Prior or current suspected or known drug-induced liver injury within 1 year prior to screening 4. History of liver transplant, current placement on a liver transplant list, or Model for End-stage Liver Disease (MELD) score \>12 5. Contraindication to MRI examinations, such as persons with cardiac pacemaker and implants made of metal, severe claustrophobia, size restrictions, or other contraindications for MRI 6. Liver stiffness measurement, laboratory parameter assessment, estimated Glomerular Filtration Rate (GFR), and evidence of uncontrolled hypertension, as defined in the protocol 7. Evidence of Human Immunodeficiency Virus (HIV) infection, Hepatitis B Virus (HBV) infection, or Hepatitis C Virus (HCV) infection during screening, as described in the protocol 8. History of Type 1 Diabetes 9. Bariatric surgery, including any procedures to revise, reverse, or remove any previous bariatric surgery interventions, within approximately 5 years prior to randomization or planned during the study period NOTE: Other protocol-defined inclusion/exclusion criteria apply.
Where this trial is running
Chandler, Arizona and 2 other locations
- Arizona Liver Health — Chandler, Arizona, United States (Recruiting)
- Richmond Pharmacology Limited — London, Greater London, United Kingdom (Recruiting)
- Parexel International Early Phase Clinical Unit — Harrow, London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Clinical Trials Administrator
- Email: clinicaltrials@regeneron.com
- Phone: 844-734-6643
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.