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AZD1613 - Part A

Phase 1

Autosomal Dominant Polycystic Kidney Disease | Small molecule | Nephrology |AstraZeneca PLC|Last Updated: May 11, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment40
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07228364Safety, Tolerability and Pharmacokinetics of AZD1613 in Adults With Autosomal Dominant Polycystic Kidney DiseasePHASE1 RECRUITING 40 — — Nov 10, 2025Jan 26, 2027May 11, 202615 United States, China +1
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Study Endpoints
Primary Endpoints
Incidence of Treatment-Emergent Adverse Events (TEAEs)
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
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
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
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)
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
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
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
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
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)
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
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)
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)
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)
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
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
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)
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
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
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)
Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days

Change from baseline in pulse oximetry oxygen saturation. Unit: percent (%).

Secondary Endpoints
Maximum Observed Serum Concentration (Cmax) of AZD1613
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
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
Intensive PK sampling from Day 1 through Day 189 per protocol schedule
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A - Cohort A1EXPERIMENTALParticipants will receive 4 doses of AZD1613 or placebo on days 1, 29, 57 and 85.
Part A - Cohort A2EXPERIMENTALParticipants will receive 4 doses of AZD1613 or placebo on days 1, 29, 57 and 85.
Part B - Chinese CohortEXPERIMENTALParticipants will receive 4 doses of AZD1613 or placebo on days 1, 29, 57 and 85.
Interventions
NameTypeDescription
AZD1613 - Part ADRUGPart A - Participants will be administered doses of AZD1613 on days 1, 29, 57, and 85 according to randomization in IRT.
Placebo - Part ADRUGPart A - Participants will be administered doses of placebo on days 1, 29, 57, and 85 according to randomization in IRT.
AZD1613 - Part BDRUGPart B - Participants will be administered doses of AZD1613 on days 1, 29, 57, and 85 according to randomization in IRT.
Placebo - Part BDRUGPart B - Participants will be administered doses of placebo on days 1, 29, 57, and 85 according to randomization in IRT.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites15

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)....

Countries:United StatesChinaUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07228364primaryCompletionDate: changed
LOWMay 24, 2026NCT07228364studyFirstPostDate: changed