A Phase 1, Double-blind, Randomized Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Single Dose and Multiple Doses of CG-0416 in Healthy Participants.

NCT07724431 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: CG-AU-0416-01
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 1

Brief Summary

A Phase 1, double-blind, randomized study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of single dose and multiple doses of CG-0416 in healthy participants. HongKong Curegene Innovation Co. Limited is developing the study drug CG-0416 as a potential new treatment for overweight and obesity. The purpose of this research is to investigate the safety, tolerability (if any side effects occur), pharmacokinetics (the amount of study drug or any of its breakdown products in your body) and pharmacodynamics (how the study drug affects your body), of single and multiple oral doses of a study drug called CG-0416.

Conditions

Overweight and Obesity

Keywords

weight loss; CG-0416; Curegene; Obesity; MASH

Outcome Measures

Primary Outcomes (3)

• To determine number of participants with treatment-related adverse events as assessed by CTCAE v6.0 of single dose and multiple doses of CG-0416 in healthy participants.

  Number of Incidence and frequency of adverse events (AEs) / SAEs possibly or probably related to study drug.

  up to 3 weeks

• To determine the safety and tolerability of single dose and multiple doses of CG-0416 in healthy participants

  Concentration changes from baseline in clinical laboratory safety tests: thyroid hormones \[TH\] , thyroid-stimulating hormone (TSH), free triiodothyronine (FT3), free thyroxine (FT4)\], follicle-stimulating hormone (FSH), luteinizing hormone, estradiol, testosterone, and free testosterone.

  up to 3 weeks

• To determine the safety and tolerability of single dose and multiple doses of CG-0416 in healthy participants.

  Baseline and placebo-corrected QTcF (ΔΔQTcF).

  up to 3 weeks

Secondary Outcomes (8)

• To define pharmacokinetics (PK) profile of CG-0416 and its active metabolite CG-0400

  up to 3 weeks

• To define pharmacokinetics (PK) profile of CG-0416 and its active metabolite CG-0400

  up to 3 weeks

• To define pharmacokinetics (PK) profile of CG-0416 and its active metabolite CG-0400

  up to 3 weeks

• To define pharmacokinetics (PK) profile of CG-0416 and its active metabolite CG-0400

  up to 3 weeks

• To define pharmacokinetics (PK) profile of CG-0416 and its active metabolite CG-0400

  Up to 3 weeks

Study Arms (8)

Cohort 1

EXPERIMENTAL

8 healthy participants will be randomly assigned in a ratio of 3:1 to receive a single dose of CG-0416 or placebo

Drug: CG-0416

Cohort 2

EXPERIMENTAL

8 healthy participants will be randomly assigned in a ratio of 3:1 to receive a single dose of CG-0416 or placebo

Drug: CG-0416

Cohort 3

EXPERIMENTAL

8 healthy participants will be randomly assigned in a ratio of 3:1 to receive a single dose of CG-0416 or placebo

Drug: CG-0416

Cohort 4

EXPERIMENTAL

8 healthy participants will be randomly assigned in a ratio of 3:1 to receive a single dose of CG-0416 or placebo

Drug: CG-0416

Cohort 5

EXPERIMENTAL

8 healthy participants will be randomly assigned in a ratio of 3:1 to receive a single dose of CG-0416 or placebo

Drug: CG-0416

Cohort 1a

EXPERIMENTAL

8 healthy participants will be randomly assigned in a ratio of 3:1 to receive a low dose of CG-0416 or placebo once daily for 14 consecutive days.

Drug: CG-0416

Cohort 2a

EXPERIMENTAL

8 healthy participants will be randomly assigned in a ratio of 3:1 to receive a middle dose of CG-0416 or placebo once daily for 14 consecutive days.

Drug: CG-0416

Cohort 3a

EXPERIMENTAL

8 healthy participants will be randomly assigned in a ratio of 3:1 to receive a high dose of CG-0416 or placebo once daily for 14 consecutive days.

Drug: CG-0416

Interventions

CG-0416 DRUG

CG-0416 is a novel selective, liver-directed thyroid hormone receptor beta (THR-β) agonist, given orally once daily.

Cohort 1; Cohort 1a; Cohort 2; Cohort 2a; Cohort 3; Cohort 3a; Cohort 4; Cohort 5

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Male and female participants aged 18 to 60 years, inclusive, at the time of screening.
• BMI of ≥ 18 kg/m2 to ≤ 32 kg/m2 for SAD and ≥ 21 kg/m2 to ≤ 35 kg/m2 for MAD at screening, and body weight \> 50 kg for SAD and MAD.
• Participants will be assessed to be in good health by the investigator, such as no clinically relevant abnormalities that will affect participant safety in the opinion of the investigator based on medical history, physical examination, clinical laboratory assessments (hematology, serum chemistry, coagulation, urinalysis, TH), and 12-lead ECG.
• LDL-C \> 1.8 mmol/L (70 mg/dL) in SAD, LDL-C \> 2.6 mmol/L (100 mg/dL) in MAD.
• Self-reported stable weight (within ± 5% change) for at least 3 months prior to screening.
• Participant and his/her sexual partner are not planning to fall pregnant and male participants can't donate sperm for 95 days after the last dose of study drug and willing to use two or more effective contraception methods (contraception guidance is provided in Appendix 17.1). Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test within 24 hours prior to the start of study drug and must not be breastfeeding, lactating or planning pregnancy during the study period. WOCBP are defined as any female who has experienced menarche and who has not undergone surgical sterilization (hysterectomy, bilateral salpingectomy or bilateral oophorectomy) and is not postmenopausal. Menopause is defined as 12 months of amenorrhea in the absence of other biological causes. In addition, females under the age of 55 years must have a documented serum FSH level \> 40 mIU/mL to confirm menopause. Male participants with potentially postmenopausal partners who are under the age of 55 years must use condoms unless their partner's postmenopausal status has been confirmed by FSH level.
• Participants have a full understanding of the trial content, process and possible adverse reactions, and voluntarily signed the informed consent form (ICF).

You may not qualify if:

• Clinically significant infection and/or cardiovascular, hematologic, renal, hepatic, pulmonary, endocrine, gastrointestinal, immunologic, dermatologic, neurologic, current or history of ADHD, anxiety or depression, or psychiatric disease at screening that, or its treatment, may have interfered with the conduct of the study (e.g., study procedure compliance, safety assessment, study results interpretation) or, in the judgment of the investigator, will have placed the participant at an unacceptable risk if participating in the study.
• Clinically significant diseases (including but not limited to gastrointestinal, renal, hepatic, neurological, hematological, endocrine, neoplastic, pulmonary, immunological, psychiatric or cardiovascular disease) within 6 months before screening.
• Thyroid diseases: a) active hyperthyroidism; b) TSH \>7 IU/L with symptoms of hypothyroidism or \>10 IU/L without symptoms.
• Diagnosis of type 1 or 2 diabetes or other autoimmune diabetes (fully resolved gestational diabetes will be permitted).
• Glycated hemoglobin (HbA1c) \> 6.5% or fasting blood glucose ≥ 7.0 mmol/L.
• Fasting (TG ≥ 500 mg/dL (or 5.65 mmol/L) at screening.
• Participant meets any of the following:
• Transaminases (AST, ALT) \> 1.5 × ULN.
• Alkaline phosphatase (ALP) \> 1.5 × ULN.
• Serum total bilirubin \> 1.5 × ULN (participants diagnosed with Gilbert's syndrome at screening are allowed to meet all other criteria).
• Platelet count \< 100,000/mm³.
• International normalized ratio (INR) \> ULN (as per the local laboratory reference range).
• Albumin \< 35 g/L.
• Estimated glomerular filtration rate \< 60 mL/min/1.73m2 per Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) 2021 formula.
• Severe allergic diseases or suspected allergy to any component of the study drug, allergic constitution (multiple drug and food allergy) judged by the investigator.

Sponsors & Collaborators

• HongKong Curegene Innovation Co., Limited: lead

Study Sites (1)

Nucleus Network Pty Ltd.

Melbourne, Victoria, 3004, Australia

Location

MeSH Terms

Conditions

Overweight; Obesity; Weight Loss

Condition Hierarchy (Ancestors)

Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Body Weight Changes

Study Officials

• Philip Ryan, MD

  Nucleus Network Pty Ltd.

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Philip Ryan, MD

CONTACT

61-0385939801; p.ryan@nucleusnetwork.com.au

Jerneen Williams

CONTACT

61-0883613222; bellberry@bellberry.com.au

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part 1 will be a single ascending dose (SAD) design, progressing from lowest dose group to highest dose group. Part 2 will be a multiple ascending dose (MAD) design, given orally once daily for 14 days.

Study Record Dates

• First Submitted: July 9, 2026
• First Posted: July 24, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): April 1, 2027
• Last Updated: July 24, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)