RGLS8429 for Polycystic Kidney Disease

Recruiting at 21 trial locations

Overseen By Kristen Gillotti

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Regulus Therapeutics Inc.

Must not be taking: Tolvaptan

Disqualifiers: Alcoholism, Drug abuse, Kidney transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 7

68 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have taken tolvaptan in the 28 days before joining. It's best to discuss your medications with the trial team.

What is the purpose of this trial?

This trial tests a new drug called RGLS8429 to see if it is safe and effective for people with a specific kidney disease called ADPKD. The drug is given through an injection under the skin. Researchers will check if it helps improve kidney health and function.

Research Team

Rekha Garg, MD

Principal Investigator

Regulus Therapeutics

Eligibility Criteria

This trial is for adults aged 18-70 with Autosomal Dominant Polycystic Kidney Disease (ADPKD), specifically those classified as 1C, 1D, or 1E by Mayo Imaging. Participants must have a certain level of kidney function (eGFR between 30 to 90 mL/min/1.73 m2) and a BMI of 18 to 35 kg/m2. They cannot have taken the drug tolvaptan in the last month or be part of another study recently.

Inclusion Criteria

Must understand and consent to the study procedures explained in the ICF and be willing and able to comply with the protocol

You have a certain classification of ADPKD based on an MRI scan taken during screening or within the past 5 years.

Your kidney function, measured by eGFR, needs to be between 30 and 90 mL/min/1.73 m2.

Exclusion Criteria

You have taken tolvaptan within the 28 days before the study starts.

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Multiple ascending doses of RGLS8429 or placebo administered via subcutaneous injection every other week for 7 doses

14 weeks

7 visits (in-person)

Treatment Part B

Fixed-dose of RGLS8429 administered via subcutaneous injection

Duration not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Placebo
• RGLS8429

Trial Overview The trial tests RGLS8429's safety and its effect on ADPKD biomarkers compared to a placebo. It also looks at how it affects kidney size related to height (htTKV), its behavior in the body (pharmacokinetics), and overall kidney function.

Participant Groups

Group I: RGLS8429 Experimental Treatment 1 Intervention

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

Group II: Placebo Experimental Treatment 1 Intervention

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

Group III: Open Label Fixed Dose RGLS8429 Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Regulus Therapeutics Inc.

Lead Sponsor

Trials

3

Recruited

130+