Safety, Tolerability and Pharmacokinetics of AZD1613 in Adults With Autosomal Dominant Polycystic Kidney Disease

NCT07228364 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: D9050C00002Identifier Number
• Study Type: interventional
• Target: 40
• Locations: 3 countries
• Sites: 15

Brief Summary

A study to investigate safety, tolerability, and pharmacokinetics of AZD1613 following subcutaneous or intravenous administration in participants with autosomal dominant polycystic kidney disease (ADPKD).

Conditions

Autosomal Dominant Polycystic Kidney Disease

Outcome Measures

Primary Outcomes (20)

• 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.

  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).

  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).

  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).

  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).

  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.

  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.

  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.

  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.

  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.

  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.

  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).

  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).

  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.

  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.

  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.

  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.

  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.

  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.

  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 (%).

  Baseline (Day -1) and post-dose at scheduled visits through Day 189 ±3 days

Secondary Outcomes (9)

• 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

• Area Under the Concentration-Time Curve Over the Dosing Interval at Steady State (AUCtau) of AZD1613

  Over the dosing interval (τ = 28 days) at steady state; sampling through Day 189

• Time to Maximum Observed Serum Concentration (Tmax) of AZD1613

  Intensive PK sampling from Day 1 through Day 189 per protocol schedule

Study Arms (3)

Part A - Cohort A1

EXPERIMENTAL

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

Drug: AZD1613 - Part A; Drug: Placebo - Part A

Part A - Cohort A2

EXPERIMENTAL

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

Drug: AZD1613 - Part A; Drug: Placebo - Part A

Part B - Chinese Cohort

EXPERIMENTAL

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

Interventions

AZD1613 - Part A DRUG

Part A - Participants will be administered doses of AZD1613 on days 1, 29, 57, and 85 according to randomization in IRT.

Part A - Cohort A1; Part A - Cohort A2; Part B - Chinese Cohort

Placebo - Part A DRUG

Part A - Participants will be administered doses of placebo on days 1, 29, 57, and 85 according to randomization in IRT.

Part A - Cohort A1; Part A - Cohort A2

AZD1613 - Part B DRUG

Part B - Participants will be administered doses of AZD1613 on days 1, 29, 57, and 85 according to randomization in IRT.

Part B - Chinese Cohort

Placebo - Part B DRUG

Part B - Participants will be administered doses of placebo on days 1, 29, 57, and 85 according to randomization in IRT.

Part B - Chinese Cohort

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• AstraZeneca: lead

Study Sites (15)

Research Site

Birmingham, Alabama, 35233, United States

NOT YET RECRUITING

Research Site

Loma Linda, California, 92354, United States

NOT YET RECRUITING

Research Site

Jacksonville, Florida, 32216, United States

NOT YET RECRUITING

Research Site

Orlando, Florida, 32808, United States

RECRUITING

Research Site

Lenexa, Kansas, 66219, United States

RECRUITING

Research Site

Baltimore, Maryland, 21201, United States

NOT YET RECRUITING

Research Site

Rochester, Minnesota, 55905, United States

NOT YET RECRUITING

Research Site

San Antonio, Texas, 78212, United States

NOT YET RECRUITING

Research Site

Chengdu, 610072, China

NOT YET RECRUITING

Research Site

Hangzhou, 310003, China

NOT YET RECRUITING

Research Site

Nanjing, 210009, China

NOT YET RECRUITING

Research Site

Shanghai, 200025, China

NOT YET RECRUITING

Research Site

Wuhan, 430022, China

NOT YET RECRUITING

Research Site

Xiamen, 361101, China

NOT YET RECRUITING

Research Site

London, NW3 2QG, United Kingdom

NOT YET RECRUITING

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

1-877-240-9479; information.center@astrazeneca.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This study will be single-blinded in order to minimize bias in the collection and evaluation of data during its conduct.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a Phase I, first-in-patient, randomized, single-blind, placebo-controlled study with a sequential MAD design in participants with autosomal dominant polycystic kidney disease (ADPKD). AZD1613 will be administered either subcutaneously or intravenously in up to 5 cohorts.

Study Record Dates

• First Submitted: October 23, 2025
• First Posted: November 14, 2025
• Study Start: November 10, 2025
• Primary Completion (Estimated): January 4, 2027
• Study Completion (Estimated): January 4, 2027
• Last Updated: March 12, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• Access Criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Locations

US (8); CN (6); GB (1)