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

Primary Purpose

Autosomal Dominant Polycystic Kidney Disease, ADPKD, Polycystic Kidney, Autosomal Dominant

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
RGLS8429
Placebo
Sponsored by
Regulus Therapeutics Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autosomal Dominant Polycystic Kidney Disease

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Centricity Research Phoenix MultispecialtyRecruiting
  • Amicis Research Center
  • National Institute of Clinical Research, Inc.Recruiting
  • Amicis Research CenterRecruiting
  • California Institute of Renal ResearchRecruiting
  • Academic Medical Research InstituteRecruiting
  • Valiance Clinical Research
  • Yale Nephrology Outpatient ClinicRecruiting
  • Horizon Research Group, LLC
  • Mayo Clinic - FloridaRecruiting
  • ElixiaRecruiting
  • Southeastern Clinical Research Institute, LLCRecruiting
  • CARE InstituteRecruiting
  • The Idaho Kidney InstituteRecruiting
  • Boise Kidney and Hypertension, PLLCRecruiting
  • University of Chicago
  • Research by Design, LLCRecruiting
  • University of IowaRecruiting
  • University of Kansas Medical Center Jared Grantham Kidney InstituteRecruiting
  • Witchita Nephrology Group, PARecruiting
  • University of Maryland School of Medicine, NephrologyRecruiting
  • Tufts Medical Center
  • St. Clair Nephrology ResearchRecruiting
  • Mayo Clinic - RochesterRecruiting
  • Nevada Kidney Disease and Hypertension CentersRecruiting
  • Northeast Clinical Research CenterRecruiting
  • University of Texas Southwestern Medical CenterRecruiting
  • Swedish Center for Comprehensive CareRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

RGLS8429

Placebo

Arm Description

The study will consist of three sequential cohorts of 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

The study will consist of three sequential cohorts of 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

Outcomes

Primary Outcome Measures

Safety and tolerability of RGLS4829: Incidence of AEs
Incidence of adverse events over time
Impact of RGLS8429 on ADPKD biomarkers
Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers

Secondary Outcome Measures

Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
Change from baseline in htTKV
Pharmacokinetic properties of RGLS8429: Cmax
Maximum observed concentration (Cmax) of RGLS8429
Pharmacokinetic properties of RGLS8429: Tmax
Time to maximum observed concentration (Tmax) of RGLS8429
Pharmacokinetic properties of RGLS8429: t½
Half-life of RGLS8429 (t½)

Full Information

First Posted
August 24, 2022
Last Updated
September 5, 2023
Sponsor
Regulus Therapeutics Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05521191
Brief Title
A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease
Official Title
A Phase 1b, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study in Patients With Autosomal Dominant Polycystic Kidney Disease to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of RGLS8429
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 6, 2022 (Actual)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Regulus Therapeutics Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
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
Detailed Description
In this randomized, double-blind, placebo-controlled Phase 1b study, 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autosomal Dominant Polycystic Kidney Disease, ADPKD, Polycystic Kidney, Autosomal Dominant

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Model Description
The study will consist of three sequential cohorts of 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total).
Masking
ParticipantInvestigator
Masking Description
This will be a double-blind, randomized, placebo-controlled study.
Allocation
Randomized
Enrollment
36 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
RGLS8429
Arm Type
Experimental
Arm Description
The study will consist of three sequential cohorts of 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
Arm Title
Placebo
Arm Type
Experimental
Arm Description
The study will consist of three sequential cohorts of 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
Intervention Type
Drug
Intervention Name(s)
RGLS8429
Intervention Description
Solution for subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Solution for subcutaneous injection
Primary Outcome Measure Information:
Title
Safety and tolerability of RGLS4829: Incidence of AEs
Description
Incidence of adverse events over time
Time Frame
Baseline to Day 113
Title
Impact of RGLS8429 on ADPKD biomarkers
Description
Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers
Time Frame
Baseline to Day 113
Secondary Outcome Measure Information:
Title
Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
Description
Change from baseline in htTKV
Time Frame
Baseline to Day 113
Title
Pharmacokinetic properties of RGLS8429: Cmax
Description
Maximum observed concentration (Cmax) of RGLS8429
Time Frame
Baseline to Day 113
Title
Pharmacokinetic properties of RGLS8429: Tmax
Description
Time to maximum observed concentration (Tmax) of RGLS8429
Time Frame
Baseline to Day 113
Title
Pharmacokinetic properties of RGLS8429: t½
Description
Half-life of RGLS8429 (t½)
Time Frame
Baseline to Day 113

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Kristen Gillotti
Phone
858-202-6300
Email
kgillotti@regulusrx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rekha Garg, MD
Organizational Affiliation
Regulus Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
Centricity Research Phoenix Multispecialty
City
Mesa
State/Province
Arizona
ZIP/Postal Code
85206
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alondra Salomon
Phone
614-501-6164
Ext
5026
Email
Alondra.salomon@centricityresearch.com
Facility Name
Amicis Research Center
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Larissa Arevalo
Phone
818-924-4708
Email
larissa.arevalo@amicisresearch.com
Facility Name
National Institute of Clinical Research, Inc.
City
Garden Grove
State/Province
California
ZIP/Postal Code
92844
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Peter Nguyen
Email
pnguyen@nicresearch.com
First Name & Middle Initial & Last Name & Degree
Alan Cantillep, MD
Phone
7144622000
Email
acantillep@nicresearch.com
Facility Name
Amicis Research Center
City
Granada Hills
State/Province
California
ZIP/Postal Code
91344
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tyrone Rosales
Email
tyrone.rosales@amicisresearch.com
Facility Name
California Institute of Renal Research
City
La Mesa
State/Province
California
ZIP/Postal Code
91942
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yen Vo
Phone
619-461-3894
Email
yvo@balboaunited.org
Facility Name
Academic Medical Research Institute
City
Los Angeles
State/Province
California
ZIP/Postal Code
90022
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kriss Grande
Phone
323-725-0051
Email
kriss@amrionline.net
Facility Name
Valiance Clinical Research
City
S. Gate
State/Province
California
ZIP/Postal Code
90280
Country
United States
Individual Site Status
Withdrawn
Facility Name
Yale Nephrology Outpatient Clinic
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lauren Liberti
Phone
203-737-8010
Email
lauren.liberti@yale.edu
First Name & Middle Initial & Last Name & Degree
Lawrence Ullman
Phone
203-213-6293
Email
lawrence.ullmanjr@yale.edu
Facility Name
Horizon Research Group, LLC
City
Coral Gables
State/Province
Florida
ZIP/Postal Code
33134
Country
United States
Individual Site Status
Withdrawn
Facility Name
Mayo Clinic - Florida
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Fouad Chebib, MD
Phone
904-956-8989
Email
Chebib.Fouad@mayo.edu
First Name & Middle Initial & Last Name & Degree
Cameron King
Phone
904-953-4254
Email
King.Cameron@mayo.edu
Facility Name
Elixia
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sahil Mehta
Phone
561-797-0336
Email
SMehta@elixiacrc.com
Facility Name
Southeastern Clinical Research Institute, LLC
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30904
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Matthew Diamond, MD
Phone
706-922-3005
Email
mdiamond@nacp.care
First Name & Middle Initial & Last Name & Degree
Katherine Peacock
Phone
706-922-3005
Email
kpeacock@nacp.care
Facility Name
CARE Institute
City
Chubbuck
State/Province
Idaho
ZIP/Postal Code
83202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jill Heinz, MHS, CCRC, CRCP
Phone
208-984-2273
Ext
102
Email
Jill@cardiorenalinstitute.com
Facility Name
The Idaho Kidney Institute
City
Idaho Falls
State/Province
Idaho
ZIP/Postal Code
83404
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jill Heinz, MHS, CCRC, CRCP
Phone
208-984-2273
Email
Jill@CardioRenalInstitute.com
Facility Name
Boise Kidney and Hypertension, PLLC
City
Nampa
State/Province
Idaho
ZIP/Postal Code
83687
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Samantha Stinemetz
Email
Samantha.hickey@frenova.com
First Name & Middle Initial & Last Name & Degree
Brianna Atkinson
Phone
269-382-9176
Email
brianna.atkinson@frenova.com
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60617
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elizabeth Salvo Brugarolas
Email
Elizabeth.SalvoBrugarolas@bsd.uchicago.ed
Facility Name
Research by Design, LLC
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60643
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Baldemar Garcia
Phone
708-952-4644
Email
bgarcia@researchbd.com
First Name & Middle Initial & Last Name & Degree
Briget Ross
Phone
708-952-4644
Email
bross@researchbd.com
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jissele Verdinez
Email
jissele-verdinez@uiowa.edu
Facility Name
University of Kansas Medical Center Jared Grantham Kidney Institute
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Cathy Creed, RN, CCRP
Phone
913-588-0053
Email
ccreed@kumc.edu
First Name & Middle Initial & Last Name & Degree
Elisabeth Laundy
Phone
913-588-8983
Email
elaundy@kumc.edu
Facility Name
Witchita Nephrology Group, PA
City
Wichita
State/Province
Kansas
ZIP/Postal Code
67214
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Michelle Hershberger
Phone
316-494-4912
Email
michelle@wngpa.com
Facility Name
University of Maryland School of Medicine, Nephrology
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Charalett Diggs, RN, MSN
Phone
410-706-2122
Email
charalett.diggs@som.umaryland.edu
Facility Name
Tufts Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nicole Merullo
Phone
617-636-8117
Email
nicole.merullo@tuftsmedicine.org
Facility Name
St. Clair Nephrology Research
City
Roseville
State/Province
Michigan
ZIP/Postal Code
48066
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rosemarie Henschel, RN
Phone
313-432-6273
Email
rhenschel@scsp.net
Facility Name
Mayo Clinic - Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Cassie Howe
Phone
507-266-1230
Email
Howe.cassie@mayo.edu
Facility Name
Nevada Kidney Disease and Hypertension Centers
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89106
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Angela Gomez
Email
angela.gomez1@frenova.com
Facility Name
Northeast Clinical Research Center
City
Bethlehem
State/Province
Pennsylvania
ZIP/Postal Code
18017
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nelson Kopyt, MD
Phone
610-433-4100
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ronak Lakhia, MD
Phone
214-648-4576
Email
ronak.lakhia@utsouthwestern.edu
First Name & Middle Initial & Last Name & Degree
Luis Madrigal
Phone
214-648-7494
Email
luis.madrigal@utsouthwestern.edu
Facility Name
Swedish Center for Comprehensive Care
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Adel Islam
Phone
206-215-6532
Email
adel.islam@swedish.org

12. IPD Sharing Statement

Plan to Share IPD
No

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

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