AZD1613 for Polycystic Kidney Disease

(PIONEER-PKD Trial)

Not yet recruiting at 10 trial locations
AC
Overseen ByAstraZeneca Clinical Study Information Center
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: AstraZeneca
Must be taking: Antihypertensives
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 tests a new treatment called AZD1613 for individuals with autosomal dominant polycystic kidney disease (ADPKD), a condition that causes cysts to grow in the kidneys. The study aims to determine if AZD1613 is safe, tolerable, and how the body processes it once administered. Participants will receive either AZD1613 or a placebo over four sessions. The trial seeks individuals diagnosed with ADPKD who have specific kidney function levels and meet other health criteria. As a Phase 1 trial, this research focuses on understanding how AZD1613 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop taking your current medications. However, if you are on anti-hypertensive medication, you must be on a stable treatment plan for at least 30 days before the screening.

Is there any evidence suggesting that AZD1613 is likely to be safe for humans?

Research shows that AZD1613 is being tested for safety and tolerability. In studies with healthy adults, AZD1613 has been administered both subcutaneously and intravenously. These studies aim to understand how the body processes the drug and identify any side effects.

Currently, limited information exists about the safety of AZD1613, as it remains in the early testing stages. At this point, scientists are primarily assessing whether the drug is safe enough for further testing. Since AZD1613 is in this phase, its safety for individuals with autosomal dominant polycystic kidney disease (ADPKD) is not yet fully known.

The ongoing trials aim to gather more detailed information on AZD1613's tolerability and potential negative effects. Safety studies like these are crucial first steps in drug development to ensure safety before wider use.12345

Why do researchers think this study treatment might be promising for polycystic kidney disease?

Unlike the standard treatments for Polycystic Kidney Disease, which often focus on managing symptoms or slowing cyst growth, AZD1613 targets the underlying mechanisms of the disease. Researchers are excited about AZD1613 because it potentially offers a new approach by directly interfering with the pathway responsible for cyst growth. This could lead to more effective management of the condition, potentially improving kidney function and patient outcomes.

What evidence suggests that AZD1613 might be an effective treatment for polycystic kidney disease?

Research is investigating AZD1613 to determine its potential benefits for autosomal dominant polycystic kidney disease (ADPKD). This condition often enlarges the kidneys and can lead to kidney failure. Although limited information exists on AZD1613's effectiveness for ADPKD in humans, ongoing studies are assessing its safety and pharmacokinetics. The primary goal is to evaluate its safety and absorption in the body. While specific results on effectiveness are not yet available, researchers are examining whether it can improve kidney function and slow disease progression. Participants in this trial will receive either AZD1613 or a placebo to help determine these outcomes.13678

Are You a Good Fit for This Trial?

This trial is for adults with Autosomal Dominant Polycystic Kidney Disease (ADPKD). Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may be excluded based on factors like other medical conditions or treatments that could interfere with the study.

Inclusion Criteria

My ADPKD is classified as Mayo Class IB-IE.
My kidney function, measured by eGFR, is between 45 to 90 mL/min.
My weight is at least 45 kg and my BMI is between 18 and 35.
See 1 more

Exclusion Criteria

Haemoglobin below the lower limit of the normal range or any other clinically significant haematological abnormality as judged by the investigator
Any clinically important abnormalities in clinical chemistry, haematology, coagulation, or urinalysis results other than those specifically described as exclusion criteria herein, as judged by the investigator
Positive hepatitis C antibody, hepatitis B virus surface antigen, or human immunodeficiency virus test, at screening
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 4 doses of AZD1613 or placebo on days 1, 29, 57, and 85

12 weeks
4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

14 weeks
Scheduled visits through Day 189

What Are the Treatments Tested in This Trial?

Interventions

  • AZD1613
Trial Overview The trial is testing AZD1613, a potential treatment for ADPKD. Participants will receive either AZD1613 or a placebo through subcutaneous (under the skin) or intravenous (into a vein) administration to evaluate its safety, tolerability, and how the body processes it.
How Is the Trial Designed?
3Treatment groups
Experimental Treatment
Group I: Part B - Chinese CohortExperimental Treatment3 Interventions
Group II: Part A - Cohort A2Experimental Treatment2 Interventions
Group III: Part A - Cohort A1Experimental Treatment2 Interventions

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

Citations

Safety, Tolerability and Pharmacokinetics of AZD1613 in ...This Phase I, randomised, single-blind, placebo-controlled study will assess the safety and tolerability of AZD1613 and characterise the ...
A Systematic Review of Reported Outcomes in ADPKD StudiesMost ADPKD studies report outcomes concerning kidney function, change in total kidney volume (TKV), change in creatinine clearance, and the development of ESKD.
PKD Outcomes Consortium (PKDOC)PKD is marked by a long period of stable kidney function (as measured by the currently accepted endpoints in drug development) during which the kidneys expand ...
NCT06995820 | A Study to Investigate Safety, Tolerability ...The purpose of the study is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of AZD1613 in healthy ...
Polycystic Kidney Disease Outcomes ConsortiumC-Path's Polycystic Kidney Disease (PKD) Outcomes Consortium is a collaborative partnership whose research leads to discovery of treatments for PKD.
Safety, tolerability and pharmacokinetics of AZD1613 in ...Safety, tolerability and pharmacokinetics of AZD1613 in adults with autosomal dominant polycystic kidney disease.
A study to investigate safety, tolerability, pharmacokinetics ...The purpose of the study is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of AZD1613 in healthy ...
Polycystic Kidney Disease Drug Development - PubMed CentralThis Special Report reviews the key points discussed during the summit and provides future direction related to PKD drug development tools.
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