Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Primary Objective
• To assess the safety and tolerability of single ascending doses of RGLS8429
Secondary Objectives
In this randomized, double-blind, placebo-controlled Phase 1 study, a single ascending dose of RGLS8429 or placebo will be administered via subcutaneous (SC) injection to healthy volunteers to evaluate the safety, tolerability, and PK of RGLS8429.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RGLS8429, first dose level | Experimental | Eligible participants will receive subcutaneous injection of the first dose level RGLS8429 or placebo |
|
| RGLS8429, second dose level | Experimental | Eligible participants will receive subcutaneous injection of the second dose level RGLS8429 or placebo |
|
| RGLS8429, third dose level | Experimental | Eligible participants will receive subcutaneous injection of the third dose level RGLS8429 or placebo |
|
| RGLS8429, fourth dose level | Experimental | Eligible participants will receive subcutaneous injection of the fourth dose level RGLS8429 or placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RGLS8429 | Drug | solution for subcutaneous injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events (AEs) | Incidence and severity of adverse events after administration of RGLS8429 | Baseline to Day 29 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of DLT and Determination of MTD | Identify dose limiting toxicity and the maximum tolerated dose after RGLS8429 administration | Baseline to Day 29 |
| Plasma Half-life (T1/2) of RGLS8429 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Rekha Garg, MD | Regulus Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| ICON Early Phase Services | San Antonio | Texas | 78209 | United States |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | RGLS8429, First Dose Level | Eligible participants will receive subcutaneous injection of the first dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| FG001 | RGLS8429, Second Dose Level | Eligible participants will receive subcutaneous injection of the second dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| FG002 | RGLS8429, Third Dose Level | Eligible participants will receive subcutaneous injection of the third dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| FG003 | RGLS8429, Fourth Dose Level | Eligible participants will receive subcutaneous injection of the fourth dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| FG004 | Matching Placebo | Eligible participants will receive subcutaneous injection of the placebo volume-matching RGLS8429 dose. Placebo volume-matching RGLS8429 dose: solution for subcutaneous injection |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | RGLS8429, First Dose Level | Eligible participants will receive subcutaneous injection of the first dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| BG001 | RGLS8429, Second Dose Level |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Incidence of Adverse Events (AEs) | Incidence and severity of adverse events after administration of RGLS8429 | Safety Population: all subjects who received a dose of study drug; subjects were analyzed based on treatment received | Posted | Number | participants | Baseline to Day 29 |
|
All Adverse Events were collecting beginning with the start of study drug administration until the Day 29 End of Study Visit.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | RGLS8429, First Dose Level | Eligible participants will receive subcutaneous injection of the first dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eustachian tube dysfunction | Ear and labyrinth disorders | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Kristen Gillotti, Senior Director Clinical Operations | Regulus Therapeutics | 203-313-8185 | kgillotti@regulusrx.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 6, 2022 | Dec 19, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 19, 2022 | Dec 19, 2023 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D016891 | Polycystic Kidney, Autosomal Dominant |
| ID | Term |
|---|---|
| D007690 | Polycystic Kidney Diseases |
| D052177 | Kidney Diseases, Cystic |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
This will be a double-blinded, randomized, placebo-controlled study.
| Placebo volume-matching RGLS8429 dose | Drug | solution for subcutaneous injection |
|
Measure plasma elimination half-life after RGLS8429 administration
| Baseline to Day 15 |
| Plasma Clearance of RGLS8429 | Measure plasma clearance after RGLS8429 administration | Baseline to Day 15 |
| Area Under the Plasma Concentration vs Time Curve (AUC) of RGLS8429 | Measure area under the plasma concentration vs time curve after RGLS8429 administration | Baseline to Day 15 |
| Amount of RGLS8429 Excreted in Urine | Measure the amount of RGLS8429 excreted in urine after RGLS8429 administration | Baseline to 24-hour |
Eligible participants will receive subcutaneous injection of the second dose level RGLS8429.
RGLS8429: solution for subcutaneous injection
| BG002 | RGLS8429, Third Dose Level | Eligible participants will receive subcutaneous injection of the third dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| BG003 | RGLS8429, Fourth Dose Level | Eligible participants will receive subcutaneous injection of the fourth dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| BG004 | Matching Placebo | Eligible participants will receive subcutaneous injection of the placebo volume-matching RGLS8429 dose. Placebo volume-matching RGLS8429 dose: solution for subcutaneous injection |
| BG005 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG002 | RGLS8429, Third Dose Level | Eligible participants will receive subcutaneous injection of the third dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| OG003 | RGLS8429, Fourth Dose Level | Eligible participants will receive subcutaneous injection of the fourth dose level RGLS8429. RGLS8429: solution for subcutaneous injection |
| OG004 | Matching Placebo | Eligible participants will receive subcutaneous injection of the placebo volume-matching RGLS8429 dose. Placebo volume-matching RGLS8429 dose: solution for subcutaneous injection |
|
|
| Secondary | Incidence of DLT and Determination of MTD | Identify dose limiting toxicity and the maximum tolerated dose after RGLS8429 administration | Not Posted | Baseline to Day 29 | Participants |
| Secondary | Plasma Half-life (T1/2) of RGLS8429 | Measure plasma elimination half-life after RGLS8429 administration | Not Posted | Baseline to Day 15 | Participants |
| Secondary | Plasma Clearance of RGLS8429 | Measure plasma clearance after RGLS8429 administration | Not Posted | Baseline to Day 15 | Participants |
| Secondary | Area Under the Plasma Concentration vs Time Curve (AUC) of RGLS8429 | Measure area under the plasma concentration vs time curve after RGLS8429 administration | Not Posted | Baseline to Day 15 | Participants |
| Secondary | Amount of RGLS8429 Excreted in Urine | Measure the amount of RGLS8429 excreted in urine after RGLS8429 administration | Not Posted | Baseline to 24-hour | Participants |
| 0 |
| 6 |
| 0 |
| 6 |
| 1 |
| 6 |
| EG001 | RGLS8429, Second Dose Level | Eligible participants will receive subcutaneous injection of the second dose level RGLS8429. RGLS8429: solution for subcutaneous injection | 0 | 6 | 0 | 6 | 1 | 6 |
| EG002 | RGLS8429, Third Dose Level | Eligible participants will receive subcutaneous injection of the third dose level RGLS8429. RGLS8429: solution for subcutaneous injection | 0 | 6 | 0 | 6 | 3 | 6 |
| EG003 | RGLS8429, Fourth Dose Level | Eligible participants will receive subcutaneous injection of the fourth dose level RGLS8429. RGLS8429: solution for subcutaneous injection | 0 | 6 | 0 | 6 | 1 | 6 |
| EG004 | Matching Placebo | Eligible participants will receive subcutaneous injection of the placebo volume-matching RGLS8429 dose. Placebo volume-matching RGLS8429 dose: solution for subcutaneous injection | 0 | 8 | 0 | 8 | 3 | 8 |
| Dry mouth | Gastrointestinal disorders | Systematic Assessment |
|
| Gastroesophageal reflux disease | Gastrointestinal disorders | Systematic Assessment |
|
| Medical device site reaction | General disorders | Systematic Assessment |
|
| Sinusitis | Infections and infestations | Systematic Assessment |
|
| Skin abrasion | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Ecchymosis | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
Not provided
Not provided
| D052776 |
| Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |