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

Study Purpose

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

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years - 70 Years
Gender	All

More Inclusion & Exclusion Criteria

Key

Inclusion Criteria:

1. Male or female ADPKD patients, 18 to 70 years old. 2. 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) 3. eGFR between 30 to 90 mL/min/1.73 m2. 4. Body mass index (BMI) 18 to 35 kg/m2. 5. 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:

1. Administration of tolvaptan in the 28 days before randomization. 2. Subject is mentally incapacitated or has significant emotional problems. 3. 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. 4. History or presence of alcoholism or drug abuse within the past 2 years prior to screening. 5. Only one kidney or kidney transplant recipient. 6. 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 Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT05521191
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 1
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Regulus Therapeutics Inc.
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Rekha Garg, MD
Principal Investigator Affiliation	Regulus Therapeutics
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Autosomal Dominant Polycystic Kidney Disease, ADPKD, Polycystic Kidney, Autosomal Dominant

Additional Details

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.

Arms & Interventions

Arms

Experimental: RGLS8429

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

Experimental: Placebo

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

Experimental: Open Label Fixed Dose RGLS8429

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.

Interventions

Drug: - RGLS8429

Solution for subcutaneous injection

Drug: - Placebo

Solution for subcutaneous injection

Contact a Trial Team

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Mesa, Arizona

Status

Terminated

Address

Centricity Research Phoenix Multispecialty

Mesa, Arizona, 85206

Site Contact

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