A Phase 1 interventional study of AZD1613 - Part A and Placebo - Part A in Autosomal Dominant Polycystic Kidney Disease, sponsored by AstraZeneca. Recruiting at 15 sites in 3 countries. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-09-11.
Sponsored by AstraZeneca · Phase 1, Interventional, and Treatment
A study to investigate safety, tolerability, and pharmacokinetics of AZD1613 following subcutaneous or intravenous administration in participants with autosomal dominant polycystic kidney disease (ADPKD).
This Phase I, randomised, single-blind, placebo-controlled study will assess the safety and tolerability of AZD1613 and characterise the pharmacokinetics (PK) of AZD1613 in participants with autosomal dominant polycystic kidney disease (ADPKD), following subcutaneous (SC) or intravenous (IV) administration. Inclusion of participants receiving placebo is appropriate for benchmarking the safety and tolerability of AZD1613. Furthermore, the safety and PK profile will be evaluated in Chinese participants with ADPKD to assess any potential race effect in this population.
Exclusion Criteria:
Participants will receive 4 doses of AZD1613 or placebo on days 1, 29, 57 and 85.
Drug: AZD1613 - Part A · Drug: Placebo - Part A
Participants will receive 4 doses of AZD1613 or placebo on days 1, 29, 57 and 85.
Drug: AZD1613 - Part A · Drug: Placebo - Part A
Participants will receive 4 doses of AZD1613 or placebo on days 1, 29, 57 and 85.
Drug: AZD1613 - Part A · Drug: AZD1613 - Part B · Drug: Placebo - Part B
Part A - Participants will be administered doses of AZD1613 on days 1, 29, 57, and 85 according to randomization in IRT.
Part A - Participants will be administered doses of placebo on days 1, 29, 57, and 85 according to randomization in IRT.
Part B - Participants will be administered doses of AZD1613 on days 1, 29, 57, and 85 according to randomization in IRT.
Part B - Participants will be administered doses of placebo on days 1, 29, 57, and 85 according to randomization in IRT.
Incidence of Treatment-Emergent Adverse Events (TEAEs)
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.
Time frame: From randomization (Day 1) through end of follow-up (up to Day 189 ±3 days)
Change From Baseline in Safety 12-Lead ECG QTcF
Change from baseline in QT interval corrected using Fridericia's formula (QTcF) measured on single 12-lead safety ECGs. Unit: milliseconds (ms).
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Safety 12-Lead ECG PR Interval
Change from baseline in PR interval measured on single 12-lead safety ECGs. Unit: milliseconds (ms).
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Safety 12-Lead ECG QRS Duration
Change from baseline in QRS duration measured on single 12-lead safety ECGs. Unit: milliseconds (ms).
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Heart Rate (12-Lead Safety ECG)
Change from baseline in heart rate measured on single 12-lead safety ECGs. Unit: beats per minute (bpm).
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in ALT
Change from baseline in alanine aminotransferase. Unit: U/L.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in AST
Change from baseline in aspartate aminotransferase. Unit: U/L.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Total Bilirubin
Change from baseline in total bilirubin. Unit: mg/dL.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Serum Creatinine
Change from baseline in serum creatinine. Unit: mg/dL.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR; CKD-EPI 2021)
Change from baseline in eGFR calculated using CKD-EPI 2021. Unit: mL/min/1.73 m².
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in INR
Change from baseline in international normalized ratio- (INR). Unit: unitless.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Prothrombin Time (PT)
Change from baseline in prothrombin time. Unit: seconds (s).
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Activated Partial Thromboplastin Time (aPTT)
Change from baseline in aPTT. Unit: seconds (s).
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Urinary Albumin-to-Creatinine Ratio (UACR)
Change from baseline in UACR (geometric mean of triplicates at each visit). Unit: mg/g.
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 Systolic Blood Pressure
Change from baseline in supine systolic blood pressure. Unit: mmHg.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Diastolic Blood Pressure
Change from baseline in supine diastolic blood pressure. Unit: mmHg.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Heart Rate (Vital Signs)
Change from baseline in supine heart rate. Unit: bpm.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Body Temperature
Change from baseline in oral body temperature. Unit: °C.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Respiratory Rate
Change from baseline in respiratory rate. Unit: breaths per minute.
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Change From Baseline in Oxygen Saturation (SpO2)
Change from baseline in pulse oximetry oxygen saturation. Unit: percent (%).
Time frame: Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days
Maximum Observed Serum Concentration (Cmax) of AZD1613
Maximum observed serum concentration following subcutaneous or intravenous administration. Unit: µg/mL.
Time frame: Intensive PK sampling from Day 1 through Day 189 per protocol schedule
Area Under the Concentration-Time Curve to Last Quantifiable Concentration (AUClast) of AZD1613
AUC from time zero to last quantifiable concentration. Unit: h·µg/mL (or day·µg/mL; align with bioanalytical report).
Time frame: Intensive PK sampling from Day 1 through Day 189 per protocol schedule
Area Under the Concentration-Time Curve From Time Zero to Infinity (AUCinf) of AZD1613
AUC from time zero extrapolated to infinity. Unit: h·µg/mL (or day·µg/mL).
Time frame: Intensive PK sampling from Day 1 through Day 189 per protocol schedule
Area Under the Concentration-Time Curve Over the Dosing Interval at Steady State (AUCtau) of AZD1613
AUC over the dosing interval at steady state. Unit: h·µg/mL (or day·µg/mL).
Time frame: Over the dosing interval (τ = 28 days) at steady state; sampling through Day 189
Time to Maximum Observed Serum Concentration (Tmax) of AZD1613
Time to reach Cmax after dosing. Unit: hours (h).
Time frame: Intensive PK sampling from Day 1 through Day 189 per protocol schedule
Terminal Elimination Half-Life (t½) of AZD1613
Half-life associated with terminal slope (λz) of the concentration-time curve. Unit: hours (h) or days (d).
Time frame: Intensive PK sampling from Day 1 through Day 189 per protocol schedule
Incidence of Anti-Drug Antibodies (ADA) to AZD1613
Number of participants with ADA-positive response based on validated tiered assay (screen and confirm). Unit: participants.
Time frame: Predose on dosing days and during follow-up through Day 189 ±3 days
ADA Titer to AZD1613
Titer among confirmed ADA-positive samples. Unit: reciprocal dilution (titer).
Time frame: Predose on dosing days and during follow-up through Day 189 ±3 days
Change From Baseline in PD Markers of PAPPA-1 Inhibition
Change from baseline in pharmacodynamic marker of PAPPA-1 inhibition. Unit: ng/mL (or assay-specific unit).
Time frame: Baseline to scheduled post-dose time points through Day 189 ±3 days
Plan to share: Yes — Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
No publications or documents are linked to this record.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Polycystic Kidney, Autosomal Dominant→
AstraZeneca