ACLF: GENFIT’s clinical trials
What is ACLF ?
Acute-on-Chronic Liver Failure (ACLF) is a severe and potentially life-threatening condition that affects patients with advanced liver disease, including those with cirrhosis, a condition in which the liver becomes progressively scarred, impairing its ability to function properly.
This condition is marked by the sudden deterioration of liver function, often accompanied by the failure of other vital organs such as the kidneys, brain, lungs, circulatory system, or blood clotting system.
ACLF can be classified into different stages based on the number of organ failures and the severity of the condition. These stages (ACLF 1, 2, 3…) help medical professionals determine the prognosis and appropriate treatment strategies.
ACLF can be triggered by various precipitating events, such as infections, alcohol consumption, or other stressors on the liver. Once triggered, ACLF commonly leads to a rapid and progressive decline in organ function.
In milder cases, about 7% of patients may not survive after 28 days, reaching around
70% for the most severe cases. Because ACLF can progress rapidly, early medical care
is essential to improve outcomes.
1. Arroyo V et al., Nat. Rev. Dis. Primers 2 (2016)
Patients with ACLF may experience a variety of symptoms, including jaundice (yellowing of the skin and eyes), ascites (fluid accumulation in the abdomen), hepatic encephalopathy (confusion and altered mental state), and variceal bleeding (bleeding
from enlarged veins in the esophagus or stomach).
Other signs and symptoms, such as
kidney dysfunction, difficulty breathing, and increased risk of infections, may also occur. Recognizing these signs and symptoms early can be crucial for timely medical care and better outcomes.
Currently, there are no approved therapies specifically for ACLF. Treatment focuses on
managing the triggering event (e.g., treating the infection) and providing supportive care.
For some patients, liver transplantation is the only potential cure, but unfortunately, it is not available to all who need it.
ACLF occurs when a patient with chronic liver disease or
cirrhosis experiences acute liver deterioration accompanied by the failure of one or
more organs. It is diagnosed through a combination of clinical evaluation,
laboratory tests, and imaging studies.
Specialized scoring systems, such as the CLIF-C ACLF score help assess disease
severity and predict patient outcomes.
About Clinical Study
What is a Clinical Study?
A clinical study, also known as a clinical trial, is a carefully designed research study that evaluates the safety and effectiveness of new investigational drugs or treatments for specific medical conditions.
Clinical studies are typically conducted in phases, each with a specific purpose. Phase I trials focus on safety and dosage in healthy volunteers, Phase II trials assess proof of concept, safety and dosage in patients, Phase III trials II assesses
safety and efficacy in patients, and Phase IV trials gather additional information on long-term risks and benefits. Before any new drug or treatment can be approved and made widely available, it must undergo rigorous testing in clinical studies.
Regulatory authorities, such as the FDA, use the results of these studies to determine if a new treatment is safe and effective. Every clinical study is meticulously reviewed by an Institutional Review Board (IRB) or Ethics Committee to
ensure it meets strict safety guidelines and that the rights and well-being of participants are protected. These studies are conducted by experienced medical professionals who closely monitor the safety of participants throughout their participation in the trial.
Every clinical study is meticulously reviewed by an Independent Review Board or Ethics Committee to ensure it meets strict safety guidelines and that the rights and well-being of participants are protected. These studies are conducted by experienced medical professionals who closely monitor the health and progress of participants throughout the trial.
Before enrolling in a clinical study, participants must give informed consent. This process involves a detailed explanation of the study’s purpose, procedures, potential risks, and benefits. Participants are encouraged to ask questions and are given time to consider their decision. Informed consent ensures that participants are fully aware of what the study entails.
Clinical studies adhere to strict confidentiality and data protection protocols. Participants’ personal information and medical data are kept secure and are only accessible to authorized study personnel. This ensures that participants’ privacy is respected and their data is used ethically and responsibly.
Why Participate in a Clinical Study?
Specialized
Learning Opportunities
Advancing Research
Access to New Investigational Treatments:
It’s important to note that not everyone enrolled in a study will directly benefit from the treatment being tested. Some participants may experience improvements in their health, while others may not. There is also a possibility that some participants may feel worse. Some participants won’t receive the investigational drug but will receive the placebo instead.
Participation in a clinical study is entirely voluntary, and you can choose to withdraw at any time without penalty.