Study of Tamibarotene in Patients With ADPKD

Phase IIa Clinical Trial of Tamibarotene in Patients With Autosomal Dominant Polycystic Kidney Disease

Clinical trial of tamibarotene in patients with Autosomal Dominant Polycystic Kidney Disease

Study Overview

• Status: Recruiting
• Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)
• Intervention / Treatment: Drug: Tamibarotene; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 70
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Ayuto Hayashi; Phone Number: +81 75-744-6858; Email: info@regenephro.co.jp

Study Locations

• Japan
  • Kyoto, Japan, 606-8507
    • Recruiting
    • Kyoto University Hospital
  • Fukuoka
    • Kurume, Fukuoka, Japan, 830-0011
      • Recruiting
      • Kurume University Hospital
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 060-8648
      • Recruiting
      • Hokkaido University Hospital
  • Kanagawa
    • Kamakura, Kanagawa, Japan, 247-8533
      • Recruiting
      • Shonan Kamakura General Hospital
    • Kawasaki, Kanagawa, Japan, 213-8587
      • Recruiting
      • Toranomon Hospital Kajigaya
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8431
      • Recruiting
      • Juntendo University School of Medicine Juntendo Hospital
    • Minato-ku, Tokyo, Japan, 105-8470
      • Recruiting
      • Toranomon Hospital
    • Shinjukuku, Tokyo, Japan, 162-8666
      • Recruiting
      • Tokyo Women's Medical University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients diagnosed as ADPKD by modified Pei-Ravine
• eGFR(CKD-EPI) is equal to or greater than 60 mL/min/1.73m^2
• Patients who are judged to be hard to treat with tolvaptan or who do not wish to be treated with tolvaptan, at the time of obtaining consent
• Patients with systolic blood pressure below 140 mmHg and diastolic blood pressure below 90 mmHg. For patients receiving angiotensin-converting enzyme inhibitors or angiotensin II receptor antagonists, the dose must be constant for at least 6 weeks before obtaining informed consent

Exclusion Criteria:

• Women who are pregnant or may be pregnant
• Nursing mother

• Females with childbearing potential or male subjects with a fertile partner who is unable to use contraception for the following periods:

  1. Female: From informed consent to 2 years after the last administration of the study drug
  2. Male: From informed consent to 6 months after the last administration of the study drug
• Patients within 12 weeks from the last dose of a drug that affects renal cysts, such as tolvaptan, to the first dose of the study drug
• Patients with complications of intracranial aneurysm, malignant tumor, uncontrolled diabetes, osteoporosis, uncontrolled dyslipidemia, or abnormal liver function

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo Group	Drug: Placebo; Subjects are administrated to placebo in daily for 52 weeks.
Active Comparator: Tamibarotene Group	Drug: Tamibarotene; Subjects are administrated to tamibarotene 4 mg in daily for 52 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in TKV from baseline	TKV	52 Week after administration of Investigational Product

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in TLV from baseline	TLV	52 Week after administration of Investigational Product
Changes in eGFR from baseline	eGFR	52 Week after administration of Investigational Product
Incidence of adverse events	Safety	1 year

Collaborators and Investigators

• Sponsor: Rege Nephro Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 22, 2023
• Primary Completion (Estimated): December 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: February 18, 2024
• First Submitted That Met QC Criteria: February 25, 2024
• First Posted (Actual): March 4, 2024

Study Record Updates

• Last Update Posted (Actual): March 4, 2024
• Last Update Submitted That Met QC Criteria: February 25, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Autosomal dominant polycystic kidney disease
• Additional Relevant MeSH Terms: Urologic Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Joint Diseases; Musculoskeletal Diseases; Muscular Diseases; Musculoskeletal Abnormalities; Abnormalities, Multiple; Kidney Diseases, Cystic; Ciliopathies; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant; Arthrogryposis
• Other Study ID Numbers: RP014-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No