Safety Study of AZD0233 in Healthy Adults

AC
Overseen ByAstraZeneca Clinical Study Information Center

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AZD0233 (also known as ION449) to assess its safety and behavior in the body at various doses. Participants will either receive the actual treatment or a placebo (a non-active substance). The trial targets healthy individuals with good veins for blood draws who meet specific weight and BMI criteria. It is suitable for those without major medical conditions or a history of drug or alcohol abuse. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the unique opportunity to be among the first to receive it.

Will I have to stop taking my current medications?

Yes, participants must stop taking any prescribed or nonprescribed medications, including antacids, analgesics (except paracetamol/acetaminophen), herbal remedies, and mega dose vitamins and minerals, during the 2 weeks before the study starts. Hormone replacement therapy is allowed for female participants.

Is there any evidence suggesting that AZD0233 is likely to be safe for humans?

Research has shown that AZD0233 has been studied for its safety in healthy adults. The studies found that AZD0233 is generally well-tolerated in both single and multiple doses. Participants reported few side effects, and those that did occur were mild. This suggests AZD0233 could be safe for humans at the doses tested so far.

However, these studies remain in the early stages. While the initial results are promising, more research is needed to fully understand the safety of AZD0233. People interested in participating should consider these findings and any potential risks when deciding whether to join clinical trials.12345

Why do researchers think this study treatment might be promising?

Researchers are excited about AZD0233 because it offers a potentially novel approach to treatment with its unique mechanism of action. Unlike many traditional treatments that rely on well-established pathways, AZD0233 may target different pathways or involve a new active ingredient, setting it apart from the current standard of care. This could lead to improved outcomes or reduced side effects, making it a promising candidate for future therapeutic options.

What evidence suggests that AZD0233 could be effective?

Research shows that AZD0233, also called ION449, may help lower cholesterol levels. In an earlier study, a similar drug reduced "bad" cholesterol (LDL-C) by 62.3%, a very positive result. Most participants in the studies did not experience serious side effects, indicating the drug was generally well tolerated. This trial will assess the safety of AZD0233 at different doses, with some participants receiving varying doses of AZD0233 and others receiving a placebo. While these results are promising, more research is needed to determine its effectiveness and safety at different doses.12678

Are You a Good Fit for This Trial?

Healthy adults, particularly those of Japanese descent with both parents and all grandparents being Japanese. Participants must have a BMI between 18-30 kg/m², weigh at least 50 kg, and suitable veins for blood draws. Women must not be pregnant and men with fertile partners must use contraception. Excludes vegans, those with dietary restrictions or recent minor medical issues that could affect the study.

Inclusion Criteria

For the healthy Japanese sub-Cohorts: healthy Japanese participants (e.g., natives of Japan or Japanese Americans) are defined as having both parents and 4 grandparents who are Japanese. This includes healthy second and third generation participants of Japanese descent whose parents or grandparents are living in a country other than Japan
I have veins suitable for repeated needle insertions.
I have taken a pregnancy test and it was negative.
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Exclusion Criteria

Participants who are vegans or have medical dietary restrictions
Judgment by the investigator that the participant should not participate in the study if they have any ongoing or recent (ie, during the Screening period) minor medical complaints that may interfere with the interpretation of study data or are considered unlikely to comply with study procedures, restrictions, and requirements
Vulnerable participants, e.g., kept in detention, protected adults under guardianship, trusteeship, or committed to an institution by governmental or juridical order
See 21 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

26 days

Treatment Part A (SAD)

Participants receive single ascending doses of AZD0233 or placebo. Includes a food effect study for Cohort 3A.

7 days
Inpatient stay from Day -1 to Day 6

Follow-up Part A

Participants are monitored for safety and effectiveness after treatment in Part A.

7 days
1 visit on Day 8 or Day 10

Treatment Part B (MAD)

Participants receive multiple ascending doses of AZD0233 or placebo.

14 days
Inpatient stay from Day -1 to Day 13

Follow-up Part B

Participants are monitored for safety and effectiveness after treatment in Part B.

7 days
1 visit on Day 17

What Are the Treatments Tested in This Trial?

Interventions

  • AZD0233
Trial Overview The trial is testing AZD0233's safety and how the body processes it after one or more doses in healthy people compared to a placebo (a substance with no therapeutic effect). It involves taking either the drug or placebo to see what happens in their bodies over time.
How Is the Trial Designed?
11Treatment groups
Experimental Treatment
Placebo Group
Group I: Part B (MAD): Cohort 3B - AZD0233 (dose 8)Experimental Treatment1 Intervention
Group II: Part B (MAD): Cohort 2B - AZD0233 (dose 7)Experimental Treatment1 Intervention
Group III: Part B (MAD): Cohort 1B - AZD0233 (dose 6)Experimental Treatment1 Intervention
Group IV: Part A (SAD): Food Effect (FE) extended Cohort 3AExperimental Treatment1 Intervention
Group V: Part A (SAD): Cohort 5A - AZD0233 (dose 5)Experimental Treatment1 Intervention
Group VI: Part A (SAD): Cohort 4A - AZD0233 (dose 4)Experimental Treatment1 Intervention
Group VII: Part A (SAD): Cohort 3A - AZD0233 (dose 3)Experimental Treatment1 Intervention
Group VIII: Part A (SAD): Cohort 2A - AZD0233 (dose 2)Experimental Treatment1 Intervention
Group IX: Part A (SAD): Cohort 1A - AZD0233 (dose 1)Experimental Treatment1 Intervention
Group X: Part B (MAD): Placebo cohortPlacebo Group1 Intervention
Group XI: Part A (SAD): Placebo cohortPlacebo Group1 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

Parexel

Industry Sponsor

Trials
322
Recruited
137,000+
Peyton Howell profile image

Peyton Howell

Parexel

Chief Executive Officer

Master of Healthcare Administration from The Ohio State University, Bachelor of Arts in Health Communications from the University of Illinois

Dr. Austin Smith profile image

Dr. Austin Smith

Parexel

Chief Medical Officer since 2023

MD from the Royal College of Surgeons in Ireland

Citations

Ionis provides update on development program evaluating ...60mg of ION449 (AZD8233) administered monthly achieved a statistically significant 62.3% (p<0.001) reduction in low-density lipoprotein cholesterol (LDL-C) ...
A study to investigate safety, tolerability, and ...This Phase 1 study assesses the safety, tolerability, and pharmacokinetics of AZD0233 in healthy adults, using single and multiple ascending doses, compared to ...
Ionis announces positive data for ETESIAN Phase 2b study ...ETESIAN Phase 2b study of ION449 (AZD8233) met its primary and secondary endpoints; ION449 was generally well tolerated.
Ionis announces positive data for ETESIAN Phase 2b study ...ETESIAN Phase 2b study of ION449 (AZD8233) met its primary and secondary endpoints; ION449 was generally well tolerated.
A Study to Investigate Safety, Tolerability, and ...The purpose of this study is to assess the safety, tolerability, and pharmacokinetics of AZD0233 following single and multiple ascending dose ( ...
Promising new data for Ionis' antisense medicine targeting ...ION449 was observed to be safe and well tolerated at all dose levels. ... An oral formulation of ION449 is currently being evaluated in a Phase 1 study in healthy ...
A Study to Investigate Safety, Tolerability, and Pharmaco...To assess the safety and tolerability of AZD0233 following oral administration of single ascending doses (Part A) and multiple ascending ...
Safety Study of AZD0233 in Healthy AdultsThe purpose of this study is to assess the safety, tolerability, and pharmacokinetics of AZD0233 following single and multiple ascending dose (SAD and MAD) ...
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