ALN-CIDEB for Fatty Liver Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests an experimental drug called ALN-CIDEB to determine its safety and impact on liver fat in individuals with fatty liver disease. It targets two conditions: MASLD, characterized by excessive liver fat, and MASH, which also involves liver inflammation. Researchers aim to understand the side effects and how the drug alters liver fat. Suitable participants may include those with MASLD or MASH, who struggle with higher body weight, and have maintained stable medication for blood pressure or cholesterol. As a Phase 1, Phase 2 trial, this research seeks to understand how the treatment works in people and measure its effectiveness in an initial, smaller group.
Is there any evidence suggesting that ALN-CIDEB is likely to be safe for humans?
Research has shown that changes in the CIDEB gene, targeted by the ALN-CIDEB drug, link to a lower risk of fatty liver disease. This finding is promising, suggesting the drug might protect the liver. However, since ALN-CIDEB remains under study, limited safety information is currently available.
The study is in its early stages, focusing primarily on the drug's safety and tolerability in humans. At this stage, researchers are just beginning to gather information on side effects. Early studies often emphasize safety, so a complete picture is not yet available. The study's continuation suggests researchers consider it potentially safe enough for further testing.
Although specific data on ALN-CIDEB's side effects is not yet available, the genetic background supports its potential safety and benefits. More information will emerge as the study progresses.12345Why do researchers think this study treatment might be promising?
Most treatments for fatty liver disease focus on lifestyle changes, such as diet and exercise, or medications that target underlying conditions like diabetes or high cholesterol. But ALN-CIDEB works differently by specifically targeting a protein involved in fat metabolism in the liver. Researchers are excited about ALN-CIDEB because it offers a new mechanism of action that directly addresses the buildup of fat in liver cells. This targeted approach could potentially result in more effective treatment outcomes with fewer side effects compared to current options.
What evidence suggests that ALN-CIDEB might be an effective treatment for fatty liver disease?
Research has shown that ALN-CIDEB might help treat fatty liver disease by targeting the CIDEB gene, which plays a role in storing fat in the liver. Studies have found that rare changes in the CIDEB gene link to a 33% lower risk of liver disease. In animal studies, reducing CIDEB activity resulted in less fat and damage in the liver. This trial will evaluate ALN-CIDEB in two parts, Part A and Part B, to determine its potential to lower liver fat and improve liver health in people.12567
Who Is on the Research Team?
Clinical Trial Management
Principal Investigator
Regeneron Pharmaceuticals
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single dose of ALN-CIDEB in Part A and two doses in Part B to evaluate safety, tolerability, and pharmacokinetics
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ALN-CIDEB
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Randomized per the protocol
Randomized per the protocol
Find a Clinic Near You
Who Is Running the Clinical Trial?
Regeneron Pharmaceuticals
Lead Sponsor
Leonard Schleifer
Regeneron Pharmaceuticals
Chief Executive Officer since 1988
MD and PhD in Medicine
George Yancopoulos
Regeneron Pharmaceuticals
Chief Medical Officer since 1997
MD from Harvard Medical School
Citations
NCT06836609 | A Study to Evaluate ALN-CIDEB in Adult ...
The study is focused on participants with metabolic dysfunction-associated steatotic liver disease (MASLD) (Part A) and metabolic dysfunction-associated ...
Advances in the Genetics of Nonalcoholic Fatty Liver Disease
Rare coding variants in CIDEB, which encodes a structural protein found in hepatic lipid droplets, were protective with 33% lower odds of any cause of liver ...
3.
clinicaltrials.regeneron.com
clinicaltrials.regeneron.com/clinical-trials/a0MPr000000bxsLMAQ/nct06836609A Study to Evaluate ALN-CIDEB in Adult Participants With ...
This study is researching an experimental drug called ALN-CIDEB, also referred to as "study drug". The study is focused on participants with metabolic ...
Liver-specific inactivation of Cideb improves metabolic ...
In the CDAA-HFD model, Cideb siRNA treatment significantly reduced liver TC and TG levels. Furthermore, remarkable reductions of hepatic steatosis and the ...
Regeneron and Alnylam Report Promising Data from ...
In the first two Part B cohorts, ALN-HSD was associated with robust target knockdown and numerically lower liver enzymes and biopsy-derived ...
6.
investor.regeneron.com
investor.regeneron.com/news-releases/news-release-details/regeneron-genetics-center-discovers-rare-mutations-cideb-gene/Regeneron Genetics Center Discovers Rare Mutations In ...
The study also found that CIDEB mutations had greater protective associations in individuals with obesity or type 2 diabetes, who are ...
Mechanistic basis for protection against fatty liver disease by ...
Collectively, these studies suggest that CIDEB mutations are protective against lipotoxic liver disease. Consistently, whole-body CIDEB deletion in mice reduces ...
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