A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease

R

Regulus Therapeutics

Status and phase

Enrolling
Phase 1

Conditions

ADPKD
Polycystic Kidney, Autosomal Dominant
Autosomal Dominant Polycystic Kidney Disease

Treatments

Drug: RGLS8429
Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT05521191
RGLS8429-02

Details and patient eligibility

About

Primary Objectives To assess the safety and tolerability of RGLS8429 To assess the impact of RGLS8429 on ADPKD biomarkers Secondary Objectives To assess the impact of RGLS8429 on height-adjusted total kidney volume (htTKV) To characterize the pharmacokinetic (PK) properties of RGLS8429 To assess the impact of RGLS8429 on renal function

Full description

This is a randomized, double-blind, placebo-controlled multiple ascending dose and an open-label fixed-dose Phase 1b study consisting of two parts, Part A and Part B. In Part A, multiple ascending doses of RGLS8429 or placebo will be administered via subcutaneous injection to subjects with ADPKD to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of RGLS8429. In Part B, a fixed-dose of RGLS8429 will be administered via subcutaneous injection to subjects with ADPKD to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of RGLS8429.

Enrollment

70 estimated patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

  • Male or female ADPKD patients, 18 to 70 years old
  • Class 1C, 1D, or 1E Mayo Imaging Classification of ADPKD (based upon either the MRI obtained during screening, or a prior MRI obtained within 5 years of screening with documented Mayo classification)
  • eGFR between 30 to 90 mL/min/1.73 m2
  • Body mass index (BMI) 18 to 35 kg/m2
  • Must understand and consent to the study procedures explained in the ICF and be willing and able to comply with the protocol

Key Exclusion Criteria:

  • Administration of tolvaptan in the 28 days before randomization
  • Subject is mentally incapacitated or has significant emotional problems
  • Any medical condition or social circumstance that, in the opinion of the Investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements; or may pose a risk to the subject's safety
  • History or presence of alcoholism or drug abuse within the past 2 years prior to screening
  • Only one kidney or kidney transplant recipient
  • Participation in another clinical trial and/or exposure to any investigational drug or approved therapy for investigational use within 28 days or 5 half-lives of the investigational drug's dosing, whichever is longer, prior to dosing. The 28-day or 5-half-life windows will be calculated from the date of the last dosing in the previous study to Day 1 of the current study.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

Double Blind

70 participants in 3 patient groups

RGLS8429
Experimental group
Description:
The randomized,double blind part of the study (Part A) will consist of three sequential cohorts of approximately 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total). Cohort 1: first dose level of RGLS8429 or placebo Cohort 2: second dose level of RGLS8429 or placebo Cohort 3: third dose level of RGLS8429 or placebo
Treatment:
Drug: RGLS8429
Placebo
Experimental group
Description:
The randomized double blind part of the study (Part A) will consist of three sequential cohorts of approximately 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total). Cohort 1: first dose level of RGLS8429 or placebo Cohort 2: second dose level of RGLS8429 or placebo Cohort 3: third dose level of RGLS8429 or placebo
Treatment:
Drug: Placebo
Open Label Fixed Dose RGLS8429
Experimental group
Description:
The open-label fixed dose part of the study (Part B and Cohort 4) will consist of a single cohort of up to 30 subjects each receiving 300 mg RGLS8429.
Treatment:
Drug: RGLS8429

Trial contacts and locations

28

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Central trial contact

Kristen Gillotti

Data sourced from clinicaltrials.gov

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