What side effects are associated with this type of intervention?

Common treatments for Polycystic Kidney Disease (PKD) include tolvaptan, metformin, and dietary interventions. Tolvaptan, a vasopressin V2-receptor antagonist, is known to cause side effects such as increased thirst, frequent urination, and potential liver toxicity. Metformin, an AMPK activator, is generally well-tolerated but can cause gastrointestinal issues like nausea and diarrhea. Dietary interventions, such as caloric restriction and ketogenic diets, may have fewer direct side effects but require careful management to avoid nutritional deficiencies. These treatments aim to slow disease progression by targeting pathways involved in cyst growth and kidney function.

What prior FDA approvals exist?

As of now, the FDA has approved tolvaptan for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). Tolvaptan is a vasopressin V2 receptor antagonist that slows the progression of kidney function decline in ADPKD by reducing cyst growth and kidney volume. This treatment aligns with the goals of investigational drugs like RGLS8429, which also aim to impact disease biomarkers and improve renal function.

What other types of research exist?

Promising alternatives to RGLS8429 for Polycystic Kidney Disease patients may include tolvaptan, which is approved for slowing kidney function decline in ADPKD, and other investigational drugs targeting similar pathways or biomarkers. Consulting with a healthcare provider for the latest clinical trial options and emerging therapies is recommended.

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Antisense Oligonucleotide

RGLS8429 for Polycystic Kidney Disease

Phase 1

Recruiting

Research Sponsored by Regulus Therapeutics Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to day 113

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed, participants will be monitored for any adverse reactions due to RGLS8429 which could include typical drug-related issues such as nausea, allergic reactions, or changes in blood work indicative of organ stress.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to day 113

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to day 113

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to day 113 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Polycystic Kidney, Autosomal Dominant

Safety and tolerability of RGLS4829: Incidence of AEs

Secondary study objectives

Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)

Pharmacokinetic properties of RGLS8429: Cmax

Pharmacokinetic properties of RGLS8429: Tmax

+1 more

Trial Design

3Treatment groups

Experimental Treatment

Group I: RGLS8429Experimental Treatment1 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: PlaceboExperimental Treatment1 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 RGLS8429Experimental Treatment1 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

RGLS8429

2022

Completed Phase 1

~40

Placebo

1995

Completed Phase 3

~2670

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Polycystic Kidney Disease (PKD) often target pathways involved in cyst growth and kidney function. RGLS8429, an investigational drug, aims to impact disease biomarkers and improve kidney function. Tolvaptan, another treatment, works by inhibiting vasopressin to reduce cyst growth and slow disease progression. Metformin, an AMPK activator, downregulates mTOR and CFTR pathways, which are crucial for cyst expansion. Dietary interventions like caloric restriction and ketogenic diets also activate AMPK signaling, reducing cyst growth. These treatments are significant for PKD patients as they offer potential to slow disease progression, preserve kidney function, and improve quality of life.

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