Clinical trial NCT06132360: the Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects in Adult Subjects With Elevated LDL-C
Official title: A Phase 1, Randomized, Single-blinded, Placebo-controlled, Single-ascending-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects in Adult Subjects With Elevated LDL-C
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: To Reduce the LDL-C Level in Hypercholesteremia Patients
Lead sponsor: Shanghai Rona Therapeutics Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2023-11-15
Enrollment: 32
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: This is a randomized, placebo-controlled, single ascending-dose (SAD) study of administered SC to adult subjects with elevated LDL-C. Subjects who have signed an Ethics Committee (EC)-approved informed consent form (ICF) and have met all the protocol eligibility criteria during screening may be enrolled into the study.


Eligibility criteria: Inclusion Criteria:

1. Male and female subjects, aged 18 to 60 years, inclusive
2. Body mass index between 18 and 32 kg/m2, inclusive, with body weight \> 45 kg for females and \>50 kg for males
3. Serum LDL-C ≥100 mg/dL (2.6 mmol/L) at screening and Day -1
4. Fasting triglycerides \< 400 mg/dL (\<4.52 mmol/L) at screening and Day -1

Exclusion Criteria:

1. Known underlying disease or medical condition that may have a potential impact on general safety assessment, lipid metabolism, or glucose metabolism, or surgical condition (including, but not limited to, bariatric surgery) that, in the opinion of the investigator, may interfere with the interpretation of the results of the clinical study
2. Received any medication including, but not limited to, statins, ezetimibe, or lipid-altering nutrients within 30 days prior to screening, or PCSK9 antibody within 90 days prior to screening, or any LDL-C-lowering siRNA therapy within 12 months prior to screening
3. History of multiple drug allergies or allergic reactions to oligonucleotides or N-acetylglucosamine (GalNAc)
4. Subjects with alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) \> 1.5 times the upper limit of normal (ULN) at the time of screening (one repeat of the screening assay is permitted) with clinically significant ALT and/or AST \> ULN and ≤ 1.5 times the ULN as determined by the investigator
5. Any medical condition that, in the opinion of the Investigator, makes the subject unsuitable for enrollment or may interfere with the subject's participation in or completion of the study. This includes, but is not limited to: history or presence of cardiovascular disease (including peripheral arterial and cerebrovascular disease); diabetes mellitus (except for diabetes mellitus of pregnancy in remission); cerebrovascular accidents; and abnormal thyroid function.

Primary outcomes:
measure: To determine the safety and tolerability of RN0191 administered as escalating single subcutaneous (SC) doses in adult subjects with elevated low-density lipoprotein-cholesterol (LDL-C)
timeFrame: For each subject in the study, the duration of the study clinic visits is approximately 18 weeks from screening to Day 85 EOS examination.
Secondary outcomes:
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Third Hospital
Study officials:
References: