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Trial record 1 of 1 for:    nct05521191
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A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05521191
Recruitment Status : Recruiting
First Posted : August 30, 2022
Last Update Posted : February 2, 2023
Sponsor:
Information provided by (Responsible Party):
Regulus Therapeutics Inc.

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

Condition or disease Intervention/treatment Phase
Autosomal Dominant Polycystic Kidney Disease ADPKD Polycystic Kidney, Autosomal Dominant Drug: RGLS8429 Drug: Placebo Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention 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: Double (Participant, Investigator)
Masking Description: This will be a double-blind, randomized, placebo-controlled study.
Primary Purpose: Treatment
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
Actual Study Start Date : October 6, 2022
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023


Arm Intervention/treatment
Experimental: RGLS8429

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
Drug: RGLS8429
Solution for subcutaneous injection

Experimental: 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
Drug: Placebo
Solution for subcutaneous injection




Primary Outcome Measures :
  1. Safety and tolerability of RGLS4829: Incidence of AEs [ Time Frame: Baseline to Day 113 ]
    Incidence of adverse events over time

  2. Impact of RGLS8429 on ADPKD biomarkers [ Time Frame: Baseline to Day 113 ]
    Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers


Secondary Outcome Measures :
  1. Impact of RGLS8429 on height-adjusted total kidney volume (htTKV) [ Time Frame: Baseline to Day 113 ]
    Change from baseline in htTKV

  2. Pharmacokinetic properties of RGLS8429: Cmax [ Time Frame: Baseline to Day 113 ]
    Maximum observed concentration (Cmax) of RGLS8429

  3. Pharmacokinetic properties of RGLS8429: Tmax [ Time Frame: Baseline to Day 113 ]
    Time to maximum observed concentration (Tmax) of RGLS8429

  4. Pharmacokinetic properties of RGLS8429: t½ [ Time Frame: Baseline to Day 113 ]
    Half-life of RGLS8429 (t½)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05521191


Contacts
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Contact: Kristen Gillotti 858-202-6300 kgillotti@regulusrx.com

Locations
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United States, Arizona
Centricity Research Phoenix Multispecialty Recruiting
Mesa, Arizona, United States, 85206
Contact: Alondra Salomon    614-501-6164 ext 5026    Alondra.salomon@centricityresearch.com   
United States, California
Amicis Research Center Recruiting
Granada Hills, California, United States, 91344
Contact: Harvey Rosales    818-644-2242    harvey rosales <harvey.rosales@amicisresearch.com>   
California Institute of Renal Research Recruiting
La Mesa, California, United States, 91942
Contact: Yen Vo    619-461-3894    yvo@balboaunited.org   
Academic Medical Research Institute Recruiting
Los Angeles, California, United States, 90022
Contact: Kriss Grande    323-725-0051    kriss@amrionline.net   
Valiance Clinical Research Recruiting
S. Gate, California, United States, 90280
Contact: Carolina Cruz    626-592-6536    C.CRUZ@JPJRESEARCH.COM   
United States, Florida
Horizon Research Group, LLC Recruiting
Coral Gables, Florida, United States, 33134
Contact: Laura Lamas    305-239-0240    llamas@horizonmedresearch.com   
United States, Idaho
CARE Institute Recruiting
Chubbuck, Idaho, United States, 83202
Contact: Farrah Ishtiaq    208-984-2273    Farrah@cardiorenalinstitute.com   
Boise Kidney and Hypertension, PLLC Recruiting
Nampa, Idaho, United States, 83687
Contact: Sarah Anderson    208-995-5747    sarah.anderson12@frenova.com   
Contact: Brianna Atkinson    269-382-9176    brianna.atkinson@frenova.com   
United States, Kansas
University of Kansas Medical Center Jared Grantham Kidney Institute Recruiting
Kansas City, Kansas, United States, 66160
Contact: Cathy Creed, RN, CCRP    913-588-0053    ccreed@kumc.edu   
Contact: Elisabeth Laundy    913-588-8983    elaundy@kumc.edu   
Witchita Nephrology Group, PA Recruiting
Wichita, Kansas, United States, 67214
Contact: Michelle Hershberger    316-494-4912    michelle@wngpa.com   
United States, Michigan
St. Clair Nephrology Research Recruiting
Roseville, Michigan, United States, 48066
Contact: Rosemarie Henschel, RN    313-432-6273    rhenschel@scsp.net   
United States, Nevada
Nevada Kidney Disease and Hypertension Centers Recruiting
Las Vegas, Nevada, United States, 89106
Contact: Angela Gomez       angela.gomez1@frenova.com   
United States, Pennsylvania
Northeast Clinical Research Center Recruiting
Bethlehem, Pennsylvania, United States, 18017
Contact: Nelson Kopyt, MD    610-433-4100      
Sponsors and Collaborators
Regulus Therapeutics Inc.
Investigators
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Study Director: Rekha Garg, MD Regulus Therapeutics
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Responsible Party: Regulus Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT05521191    
Other Study ID Numbers: RGLS8429-02
First Posted: August 30, 2022    Key Record Dates
Last Update Posted: February 2, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Arthrogryposis
Kidney Diseases
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Urologic Diseases
Joint Diseases
Musculoskeletal Diseases
Muscular Diseases
Musculoskeletal Abnormalities
Congenital Abnormalities
Kidney Diseases, Cystic
Abnormalities, Multiple
Ciliopathies
Genetic Diseases, Inborn