AZD7503 for Non-alcoholic Fatty Liver Disease

No longer recruiting at 1 trial location
AC
Overseen ByAstraZeneca Clinical Study Information Center
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: AstraZeneca
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to explore the effects of a new treatment, AZD7503, for individuals with non-alcoholic fatty liver disease (NAFLD) or non-alcoholic steatohepatitis (NASH). In the first part of the study, participants will undergo a liver biopsy to confirm their condition but will not receive the drug. In the second part, those with confirmed NAFLD or NASH will receive the treatment to assess its safety and effectiveness. The trial seeks adults with signs of NAFLD or NASH, such as unusual liver test results or imaging showing liver fat, who are not pregnant or likely to become pregnant. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications, but it mentions that any changes to medications within one month before screening are not allowed. This might mean you should maintain your current medication regimen without changes.

Is there any evidence suggesting that AZD7503 is likely to be safe for humans?

Research shows that AZD7503 is being tested for safety and tolerance in people with non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). Earlier studies injected AZD7503 under the skin to observe how the body processes the drug over time. These studies focus on identifying side effects and assessing how well participants tolerate the drug.

Since this trial is in an early stage, the main goal is to learn about the safety of AZD7503. Early-stage trials usually involve a small number of participants to closely monitor any side effects. Although specific results are not yet available, this careful monitoring helps quickly identify potential risks.

It's important to understand that reaching this stage of testing means the treatment has passed initial lab tests and is promising enough for human trials. However, a complete understanding of its safety will only emerge as more data is collected over time.12345

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for non-alcoholic fatty liver disease (NAFLD), which often focus on lifestyle changes and medications that indirectly address liver health, AZD7503 directly targets hepatic HSD17B13 mRNA expression. This novel mechanism aims to reduce liver inflammation and damage from the root by knocking down a specific enzyme linked to liver disease progression. Researchers are excited about AZD7503 because it represents a more targeted therapeutic approach, potentially offering improved efficacy and safety for patients with NAFLD or NASH.

What evidence suggests that AZD7503 might be an effective treatment for non-alcoholic fatty liver disease?

Research shows that AZD7503, the investigational treatment in this trial, targets and reduces a specific liver enzyme called HSD17B13. This enzyme is linked to the development of liver conditions like non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). Early studies suggest that lowering HSD17B13 levels may help reduce liver inflammation and damage, major issues in these conditions. While researchers continue to gather detailed human data, treatments like AZD7503 that focus on genes show promise for improving long-term liver health in people with liver diseases. Initial findings indicate that this approach could effectively stop or slow the progression of the disease.36789

Are You a Good Fit for This Trial?

This trial is for adults aged 18 to 70 with suspected or confirmed non-alcoholic fatty liver disease (NAFLD) or non-alcoholic steatohepatitis (NASH). They must have certain lab values indicating liver issues, a BMI of at least 20 kg/m2, and histologic evidence of NAFLD/NASH. Women must be of non-childbearing potential.

Inclusion Criteria

Inclusion Criteria # Part A
Participants with suspected or confirmed NAFLD or NASH including laboratory values with any of the following deviations at screening
ALT > ULN
See 5 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Part A - Baseline Assessment

Participants undergo a diagnostic liver biopsy to determine NAFLD Activity Score and fibrosis stage

1 week
1 visit (in-person)

Part B - Treatment

Participants receive multiple doses of AZD7503 and undergo repeat liver biopsy at the end-of-treatment

99 days
Multiple visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks
2 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

  • AZD7503
Trial Overview The study tests AZD7503's effects on knocking down HSD17B13 mRNA in patients with NAFLD/NASH. Part A involves diagnosis without intervention; Part B treats confirmed cases with the drug to assess its pharmacokinetics, safety, and tolerability.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Intervention/ DrugExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

AstraZeneca

Lead Sponsor

Trials
4,491
Recruited
290,540,000+

Sir Pascal Soriot

AstraZeneca

Chief Executive Officer since 2012

Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris

Dr. Cristian Massacesi

AstraZeneca

Chief Medical Officer since 2021

MD from Marche Polytechnic University, Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology

Pascal Soriot

AstraZeneca

Chief Executive Officer since 2012

Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris

Cristian Massacesi

AstraZeneca

Chief Medical Officer since 2021

MD from Marche Polytechnic University, Medical Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology

Published Research Related to This Trial

Non-alcoholic fatty liver disease (NAFLD) is a growing global health issue linked to metabolic disorders, with significant risks for disease progression and mortality, yet there are currently no approved medications for its treatment.
This paper reviews emerging therapeutic strategies and potential drug targets for NAFLD, emphasizing the importance of ongoing clinical trials in developing effective pharmacotherapies.
Pharmacotherapy for Non-Alcoholic Fatty Liver Disease: Emerging Targets and Drug Candidates.Prikhodko, VA., Bezborodkina, NN., Okovityi, SV.[2023]
In a study of 50 patients with biopsy-proven non-alcoholic fatty liver disease (NAFLD), common clinical features included fatigue (100%), hypertriglyceridemia (80%), and hepatomegaly (72%), indicating a clear pattern of symptoms associated with the disease.
Histopathological analysis revealed that 62% of patients had simple fatty liver, while 38% had nonalcoholic steatohepatitis (NASH), with a significant relationship found between hypertriglyceridemia and the presence of NASH.
A clinical and biochemical profile of biopsy-proven non-alcoholic Fatty liver disease subjects.Khurram, M., Ashraf, MM.[2022]

Citations

Antifibrotic therapies for metabolic dysfunction-associated ...Summary. Metabolic dysfunction-associated steatotic liver disease (MASLD) affects more than a quarter of the adult population worldwide.
Metabolic Dysfunction-associated Steatohepatitis (MASH) ...Not Available, completed. NCT05560607. Knockdown of HSD17B13 mRNA, Pharmacokinetics, Safety, and Tolerability, of AZD7503 in Non-Alcoholic Fatty Liver Disease.
Non-Alcoholic Fatty Liver Disease Pipeline 2024The MAESTRO-NASH OUTCOMES study is a double-blind, randomized, placebo-controlled Phase III trial that includes 845 patients with compensated NASH cirrhosis and ...
Review Gene-based therapies for steatotic liver diseaseGene-based therapies show significant potential for preventing the progression of SLD and enhancing long-term liver health.
AZD7503 for Non-alcoholic Fatty Liver DiseaseThis trial tests a new drug called AZD7503 to treat adults with specific liver diseases (NAFLD or NASH) who cannot have children.
A study to investigate Safety, Tolerability, and ...The purpose of this study is to measure the safety, tolerability, and PK (measurement of drug activity in the body over time) of AZD7503 injected subcutaneously ...
7.plpl.www.astrazenecaclinicaltrials.complpl.www.astrazenecaclinicaltrials.com/study/D9230C00002
A study to investigate Safety, Tolerability, and Pharmacokinetics ...The purpose of this study is to measure the safety, tolerability, and PK (measurement of drug activity in the body over time) of AZD7503 injected subcutaneously ...
Oligonucleotide therapies for nonalcoholic steatohepatitis - PMCThis article systematically reviews the pathogenesis of NASH/NAFLD, the promising druggable targets proven by current studies in chemical compounds or ...
Trial | NCT05560607This is a two-part study. In Part A, eligible participants will undergo a baseline diagnostic liver biopsy to determine non-alcoholic fatty liver disease ...
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