NCT07228364 · AstraZeneca
Safety, Tolerability and Pharmacokinetics of AZD1613 in Adults With Autosomal Dominant Polycystic Kidney Disease
(PIONEER-PKD)
What this study is about
A study to investigate safety, tolerability, and how the drug moves through the body of AZD1613 following injected under the skin or given through a vein (IV) administration in participants with autosomal dominant polycystic kidney disease (ADPKD).
View original scientific description
A study to investigate safety, tolerability, and pharmacokinetics of AZD1613 following subcutaneous or intravenous administration in participants with autosomal dominant polycystic kidney disease (ADPKD).
Interventions
DRUG
AZD1613 - Part A
Part A - Participants will be administered doses of AZD1613 on days 1, 29, 57, and 85 according to randomization in IRT.
DRUG
Placebo - Part A
Part A - Participants will be administered doses of placebo on days 1, 29, 57, and 85 according to randomization in IRT.
DRUG
AZD1613 - Part B
Part B - Participants will be administered doses of AZD1613 on days 1, 29, 57, and 85 according to randomization in IRT.
DRUG
Placebo - Part B
Part B - Participants will be administered doses of placebo on days 1, 29, 57, and 85 according to randomization in IRT.
Primary outcome measures
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: From randomization (Day 1) through end of follow-up (up to Day 189 ±3 days)
Number of participants with at least one TEAE and SAE, including events leading to discontinuation or death; coded by system organ class and preferred term. Unit: participants.
Change From Baseline in Safety 12-Lead ECG QTcF
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in QT interval corrected using Fridericia's formula (QTcF) measured on single 12-lead safety ECGs. Unit: milliseconds (ms).
Change From Baseline in Safety 12-Lead ECG PR Interval
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in PR interval measured on single 12-lead safety ECGs. Unit: milliseconds (ms).
Change From Baseline in Safety 12-Lead ECG QRS Duration
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in QRS duration measured on single 12-lead safety ECGs. Unit: milliseconds (ms).
Change From Baseline in Heart Rate (12-Lead Safety ECG)
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in heart rate measured on single 12-lead safety ECGs. Unit: beats per minute (bpm).
Change From Baseline in ALT
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in alanine aminotransferase. Unit: U/L.
Change From Baseline in AST
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in aspartate aminotransferase. Unit: U/L.
Change From Baseline in Total Bilirubin
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in total bilirubin. Unit: mg/dL.
Change From Baseline in Serum Creatinine
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in serum creatinine. Unit: mg/dL.
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR; CKD-EPI 2021)
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in eGFR calculated using CKD-EPI 2021. Unit: mL/min/1.73 m².
Change From Baseline in INR
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in international normalized ratio- (INR). Unit: unitless.
Change From Baseline in Prothrombin Time (PT)
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in prothrombin time. Unit: seconds (s).
Change From Baseline in Activated Partial Thromboplastin Time (aPTT)
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in aPTT. Unit: seconds (s).
Change From Baseline in Urinary Albumin-to-Creatinine Ratio (UACR)
Time frame: Baseline (Day -1) and scheduled visits through Day 189 ±3 days; UACR as triplicate first-morning voids per visit
Change from baseline in UACR (geometric mean of triplicates at each visit). Unit: mg/g.
Change From Baseline in Systolic Blood Pressure
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in supine systolic blood pressure. Unit: mmHg.
Change From Baseline in Diastolic Blood Pressure
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in supine diastolic blood pressure. Unit: mmHg.
Change From Baseline in Heart Rate (Vital Signs)
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in supine heart rate. Unit: bpm.
Change From Baseline in Body Temperature
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in oral body temperature. Unit: °C.
Change From Baseline in Respiratory Rate
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in respiratory rate. Unit: breaths per minute.
Change From Baseline in Oxygen Saturation (SpO2)
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change from baseline in pulse oximetry oxygen saturation. Unit: percent (%).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Patients with ADPKD Mayo Class (IB-IE), as per clinical diagnosis (MIC) assessed centrally. Genetic testing results will not be used for eligibility purposes
- eGFR = 45 to 90 mL/min /1.73m2
- Body weight ≥ 45 kg and body mass index within the range 18 to 35 kg/m2 (inclusive).
- Females are to be of non-childbearing potential
Exclusion criteria
- As judged by the investigator, any evidence of cardiac, vascular, and other renal conditions which in the investigator's opinion makes it undesirable for the participant to participate in the study.
- Positive hepatitis C antibody, hepatitis B virus surface antigen, or human immunodeficiency virus test, at screening.
- History of QT prolongation associated with other medications that required discontinuation of that medication.
- Congenital long QT syndrome.
- History of ventricular arrhythmia requiring treatment. Patients with atrial fibrillation/flutter and controlled ventricular rate HR \< 100 bpm can be eligible as judged by the investigator.
- 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.
- Systolic BP \> 160 mmHg or diastolic BP \> 100mmHg or HR \< 50 bpm or \> 100 bpm at screening. Patients taking anti-hypertensive medication should be on a stable treatment regimen of antihypertensive therapy for at least 30 days prior to the screening visit.
- Any clinically important abnormalities in rhythm, conduction, or morphology of the resting ECG and any abnormalities in the 12-lead ECG that, as considered by the investigator, may interfere with the interpretation of QTc interval changes, including abnormal ST-T-wave morphology or left ventricular hypertrophy.
- Kidney cyst interventions such as cyst aspiration or cyst fenestration within 12 weeks prior to screening and during the screening period, or such interventions planned or anticipated within the follow-up period.
Where
- Birmingham, Alabama
- Loma Linda, California
- Jacksonville, Florida
- Orlando, Florida
- Lenexa, Kansas
- Baltimore, Maryland
- Rochester, Minnesota
- San Antonio, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 28, 2026 · Source of record for eligibility and locations