Autosomal Dominant Polycystic Kidney DiseasePhase 1NCT05521191
A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease
A Phase 1 study testing RGLS8429 in people with Autosomal Dominant Polycystic Kidney Disease and ADPKD, run by Regulus Therapeutics Inc. at 21 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney Disease, ADPKD, Polycystic Kidney, Autosomal Dominant
- What is being tested?
- RGLS8429
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 68 participants
- Where
- 21 sitesUnited States
- Study dates
- 6 Oct 2022to 27 Mar 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
21 sites in 1 country.
21 sites in 1 country
United States21 sites
Southeastern Clinical Research Institute, LLC
Augusta, Georgia, 30904
University of Maryland School of Medicine, Nephrology
Baltimore, Maryland, 21201
Northeast Clinical Research Center
Bethlehem, Pennsylvania, 18017
CARE Institute
Boise, Idaho, 83706
Tufts Medical Center
Boston, Massachusetts, 02111
University of Chicago
Chicago, Illinois, 60617
Research by Design, LLC
Chicago, Illinois, 60643
CARE Institute
Chubbuck, Idaho, 83202
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
The Idaho Kidney Institute
Idaho Falls, Idaho, 83404
University of Iowa
Iowa City, Iowa, 52242
Mayo Clinic - Florida
Jacksonville, Florida, 32224
University of Kansas Medical Center Jared Grantham Kidney Institute
Kansas City, Kansas, 66160
Academic Medical Research Institute
Los Angeles, California, 90022
Centricity Research Phoenix Multispecialty
Mesa, Arizona, 85206
Yale Nephrology Outpatient Clinic
New Haven, Connecticut, 06510
Elixia
Orlando, Florida, 32806
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
St. Clair Nephrology Research
Roseville, Michigan, 48066
Swedish Center for Comprehensive Care
Seattle, Washington, 98104
Witchita Nephrology Group, PA
Wichita, Kansas, 67214
About this study
Official title: A Phase 1b, Double-Blind, Placebo-Controlled, Multiple Ascending Dose and an Open-Label Fixed-Dose Study in Patients With Autosomal Dominant Polycystic Kidney Disease to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of RGLS8429
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
Read the detailed description
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.
- Conditions
- Autosomal Dominant Polycystic Kidney Disease, ADPKD, Polycystic Kidney, Autosomal Dominant
- Being tested
- RGLS8429, Placebo
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RGLS8429Drug
Solution for subcutaneous injection
PlaceboDrug
Solution for subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| RGLS8429 | Experimental | 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 Receives: Drug: RGLS8429 |
| Placebo | Experimental | 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 Receives: Drug: Placebo |
| Open Label Fixed Dose RGLS8429 | Experimental | 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. Receives: Drug: RGLS8429 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of RGLS4829: Incidence of AEs
Incidence of adverse events over time
Measured: Baseline to Day 113
Impact of RGLS8429 on ADPKD biomarkers
Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers
Measured: Baseline to Day 113
Secondary outcomes (4)
Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
Change from baseline in htTKV
Measured: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Cmax
Maximum observed concentration (Cmax) of RGLS8429
Measured: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Tmax
Time to maximum observed concentration (Tmax) of RGLS8429
Measured: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: t½
Half-life of RGLS8429 (t½)
Measured: Baseline to Day 113
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2022
- Study start6 Oct 2022
- Primary completion27 Jan 2025
- Study completion27 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Autosomal Dominant Polycystic Kidney DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- The study will consist of two parts. 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 (approximately 36 subjects total). Part B will be an open-label fixed dose cohort of up to 30 subjects. .
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regulus Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rekha Garg, MD, Study director, Regulus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05521191
- Sponsor's protocol ID
- RGLS8429-02
- Status last verified
- May 2025
- First submitted
- 24 Aug 2022
- Last updated
- 3 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Joy MS, Gbadegesin RA, Conway PT, Chertow GM, Liu KD, Lim MD, Kretzler M, Bonventre JV; Precision Medicine Workshop Speakers and Moderators. National Institutes of Health Workshop to Accelerate Therapeutic Innovation by Optimizing Kidney Precision Medicine Clinical Trials. Clin J Am Soc Nephrol. 2026 Feb 17. doi: 10.2215/CJN.0000001031. Online ahead of print.
PubMed 41701531Yilmaz G, Singha SK, Savaliya B, Abdelfattah A, Elsekaily W, Xu X, Zhang Y, Hanna C, Hogan MC, Chade AR, Eirin A, Irazabal MV. Vascular transcriptional and metabolic changes precede progressive intrarenal microvascular rarefaction in autosomal dominant polycystic kidney disease. Am J Physiol Renal Physiol. 2026 Mar 1;330(3):F326-F345. doi: 10.1152/ajprenal.00266.2025. Epub 2026 Feb 5.
PubMed 41643210Chen CY, Hadla M, Khambati I, Kashyap S, Westerfield V, Fedeles S, Besse W, Hopp K, Harris PC, Patel V, Chini E, Salih M, Barry MA, Chebib FT. Emerging Therapies in Autosomal Dominant Polycystic Kidney Disease. Kidney360. 2026 Jul 1;7(7):1662-1676. doi: 10.34067/KID.0000001109. Epub 2025 Dec 12.
PubMed 41385299St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.
PubMed 39356039Tekendo-Ngongang C, Gleeson JG, Mignon L. Treating the Untreatable: Antisense Oligonucleotides as an Individualized Therapy for Rare Genetic Kidney Diseases. J Am Soc Nephrol. 2024 Dec 1;35(12):1774-1777. doi: 10.1681/ASN.0000000532. Epub 2024 Sep 27. No abstract available.
PubMed 39331470
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Double-Blind, Placebo-Controlled, Multiple Ascending Dose and an Open-Label Fixed-Dose Study in Patients With Autosomal Dominant Polycystic Kidney Disease to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of RGLS8429
- ClinicalTrials.gov ID
- NCT05521191
- Acronym / sponsor ID
- RGLS8429-02
- Conditions
- Autosomal Dominant Polycystic Kidney Disease, ADPKD, Polycystic Kidney, Autosomal Dominant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Regulus Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Oct 2022 (actual)
- Primary completion
- 27 Jan 2025 (actual)
- Study completion
- 27 Mar 2025 (actual)
- First posted
- 30 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
- Model description
- The study will consist of two parts. 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 (approximately 36 subjects total). Part B will be an open-label fixed dose cohort of up to 30 subjects. .
| Arm | Type | Receives |
|---|---|---|
| RGLS8429 | Experimental | Drug: RGLS8429The 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 |
| Placebo | Experimental | Drug: PlaceboThe 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 |
| Open Label Fixed Dose RGLS8429 | Experimental | Drug: RGLS8429The 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 may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RGLS8429Solution for subcutaneous injection | Drug | Open Label Fixed Dose RGLS8429, RGLS8429 |
| PlaceboSolution for subcutaneous injection | Drug | Placebo |
Primary outcome
Safety and tolerability of RGLS4829: Incidence of AEs
Time frame: Baseline to Day 113
Incidence of adverse events over time
Impact of RGLS8429 on ADPKD biomarkers
Time frame: Baseline to Day 113
Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers
4 secondary outcomes
Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
Time frame: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Cmax
Time frame: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Tmax
Time frame: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: t½
Time frame: Baseline to Day 113
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2025.
- Derived from this study: PubMed 41701531Joy MS, Gbadegesin RA, Conway PT, Chertow GM, Liu KD, Lim MD, Kretzler M, Bonventre JV; Precision Medicine Workshop Speakers and Moderators. National Institutes of Health Workshop to Accelerate Therapeutic Innovation by Optimizing Kidney Precision Medicine Clinical Trials. Clin J Am Soc Nephrol. 2026 Feb 17. doi: 10.2215/CJN.0000001031. Online ahead of print.
- Derived from this study: PubMed 41643210Yilmaz G, Singha SK, Savaliya B, Abdelfattah A, Elsekaily W, Xu X, Zhang Y, Hanna C, Hogan MC, Chade AR, Eirin A, Irazabal MV. Vascular transcriptional and metabolic changes precede progressive intrarenal microvascular rarefaction in autosomal dominant polycystic kidney disease. Am J Physiol Renal Physiol. 2026 Mar 1;330(3):F326-F345. doi: 10.1152/ajprenal.00266.2025. Epub 2026 Feb 5.
- Derived from this study: PubMed 41385299Chen CY, Hadla M, Khambati I, Kashyap S, Westerfield V, Fedeles S, Besse W, Hopp K, Harris PC, Patel V, Chini E, Salih M, Barry MA, Chebib FT. Emerging Therapies in Autosomal Dominant Polycystic Kidney Disease. Kidney360. 2026 Jul 1;7(7):1662-1676. doi: 10.34067/KID.0000001109. Epub 2025 Dec 12.
- Derived from this study: PubMed 39356039St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.
- Derived from this study: PubMed 39331470Tekendo-Ngongang C, Gleeson JG, Mignon L. Treating the Untreatable: Antisense Oligonucleotides as an Individualized Therapy for Rare Genetic Kidney Diseases. J Am Soc Nephrol. 2024 Dec 1;35(12):1774-1777. doi: 10.1681/ASN.0000000532. Epub 2024 Sep 27. No abstract available.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rekha Garg, MD study director
Regulus Therapeutics
21 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RGLS8429 in people with Autosomal Dominant Polycystic Kidney Disease and ADPKD, run by Regulus Therapeutics Inc. at 21 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of RGLS4829: Incidence of AEs (time frame: Baseline to Day 113). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05521191, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2025.