Clinical trial NCT05429073: A Study of RGLS8429 in Healthy Volunteers
Official title: A Phase 1, Double-Blind, Placebo-Controlled, Single Ascending Dose Study in Healthy Volunteers to Evaluate the Safety, Tolerability, and Pharmacokinetics of RGLS8429
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy Volunteers
Keywords: RGLS8429
Autosomal Dominant Polycystic Kidney Disease
ADPKD
Lead sponsor: Regulus Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2022-06-23
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: Primary Objective

• To assess the safety and tolerability of single ascending doses of RGLS8429

Secondary Objectives

* To identify dose-limiting toxicity (DLT) and to determine the maximum tolerated dose (MTD) of a single SC dose of RGLS8429
* To characterize the pharmacokinetic (PK) properties of RGLS8429

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

1. Male or female, 18 to 55 years of age
2. Body mass index (BMI) 18 to 35 kg/m2
3. Medically healthy, with no clinically significant medical history in the opinion of the Investigator
4. Estimated glomerular filtration rate (eGFR) ≥ 90 mL/min/1.73 m2
5. Must understand and consent to the study procedures explained in the informed consent form (ICF) and be willing and able to comply with the protocol

Exclusion Criteria:

1. Subject is mentally incapacitated or has significant emotional problems
2. 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
3. History or presence of alcoholism or drug abuse within the past 2 years prior to Screening
4. 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 and 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.

Primary outcomes:
measure: Incidence of adverse events (AEs)
timeFrame: Baseline to Day 29
Secondary outcomes:
measure: Incidence of DLT and determination of MTD
timeFrame: Baseline to Day 29
measure: Plasma half-life (T1/2) of RGLS8429
timeFrame: Baseline to Day 15
measure: Plasma clearance of RGLS8429
timeFrame: Baseline to Day 15
measure: Area under the plasma concentration vs time curve (AUC) of RGLS8429
timeFrame: Baseline to Day 15
measure: Amount of RGLS8429 excreted in urine
timeFrame: Baseline to 24-hour
Other outcomes:
Locations:
city: San Antonio
country: United States
facility: ICON Early Phase Services
state: Texas
Study officials:
affiliation: Regulus Therapeutics
name: Rekha Garg, MD
role: STUDY_DIRECTOR
References: