First-In-Human Study to Evaluate Single and Multiple Ascending Doses of JUV-161 in Healthy Adult Volunteers

NCT06918925 | Active Not Recruiting Phase 1

• 1 other identifier: JUV-161-101
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

Brief Summary

The present First-In-Human (FIH) study (JUV-161-101) aims to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple subcutaneous (SC) doses of JUV-161 in healthy volunteers. The study design is well-established for FIH studies and appropriate to assess the preliminary safety and tolerability of new drug candidates. Data from this study will support conduct studies in patients with DM1 as well as supporting studies in other degenerative myopathies and other disorders for which preclinical efficacy data have been obtained.JUV-161 has not been previously been administered to human subjects.

Conditions

Healthy Volunteer

Outcome Measures

Primary Outcomes (5)

• Incidence of treatment-emergent adverse events

  From enrollment through to safety follow-up Visit on Day 60

• Number of participants with treatment-emergent potentially clinically-significant safety abnormalities in safety laboratory parameters

  From enrollment through to safety follow-up Visit on Day 60

• Number of participants who have changes from baseline in electrocardiogram values in QTcF intervals

  From enrollment through the safety follow-up visit on Day 60

• Number of participants who have changes from baseline in heart rate ( beats per minute)

  From enrollment through the safety follow-up visit on Day 60

• Number of participants who have a change from baseline in systolic and diastolic blood pressure (mmHg)

  From enrollment through the safety follow-up visit on Day 60

Secondary Outcomes (4)

• Maximum observed plasma concentration (Cmax)

  Day 1 pre-dose and at 1, 2, 6, 12, 24, 36, 48, 60 and 72 hours Day 6,8,11,15,18,21 and 60

• Time to the maximum measured plasma concentration (Tmax):

  Up to 60 days

• Area under the concentration-time curve from time zero through to infinity (AUC0-∞)

  Up to 60 days.

• Terminal elimination half-life in plasma (t1/2)

  Up to 60 days

Study Arms (2)

JUV-161

ACTIVE COMPARATOR

Single-ascending dose administration of JUV-161/ 5 cohorts of 6 subjects SAD 1 JUV-161 0.10 mg/kg 6 subjects SAD 2 JUV-161 0.30 mg/kg 6 subjects SAD 3 JUV-161 1.00 mg/kg 6 subjects SAD 4 JUV-161 3.00 mg/kg 6 subjects SAD 5 JUV-161 5.0 mg/kg 6 subjects SAD 6 JUV-161 10.0mg/kg 6 subjects MAD 1 JUV-161 0.10 mg/kg 6 subjects X 3 doses MAD2 JUV-161 3.00 mg/kg 6 subjects X 3 doses MAD3 JUV-161 5.0 mg/kg 6 subjects x 3 doses

Drug: JUV-161, Placebo

Placebo-Controlled

PLACEBO COMPARATOR

SAD1 placebo 2 subjects X 1 dose SAD 2 placebo 2 subjects X 1 dose SAD 3 placebo 2 subjects X 1 dose SAD 4 placebo 2 subjects X 1 dose SAD 5 placebo 2 subjects X 1 dose SAD 6 placebo 2 subjects X 1 dose MAD 1 placebo 2 subjects x 3 doses MAD 2 placebo 2 subjects x 3 doses MAD 3 placebo 2 subjects x 3 doses

Drug: JUV-161, Placebo

Interventions

JUV-161, Placebo DRUG

Single-Ascending, Placebo-Controlled

JUV-161; Placebo-Controlled

Eligibility Criteria

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

You may qualify if:

• Are males or nonpregnant females, ages 18 to 60 (inclusive) at time of signing Informed Consent with body mass index (BMI) 18 to 35 kg/m2
• Are willing and able to give informed consent and follow all study procedures and requirements
• Are able to understand the requirements of the study protocol
• Agree to complete all required study visits
• Are healthy, based on physical examination, medical history laboratory tests, vital signs and resting electrocardiograms
• Are willing to abstain from caffeine and nicotine while in the Study Unit
• Have negative screens for alcohol and drugs of abuse at screening and admission
• Are willing to abstain from all alcoholic beverages and cannabinoids for 48 h prior to dosing through Post-dosing visit on Study Day 6.
• Females must be either:
• of non-childbearing potential (defined as having undergone surgical sterilization (hysterectomy, bilateral salpingectomy, bilateral oophorectomy or being postmenopausal (i.e., greater than 45 years old with amenorrhea for ≥ 12 months).) \[Women under the age of 55 years must have a follicle stimulating hormone (FSH) level \> 40mIU/mL to confirm menopause\] OR
• of child-bearing potential and using at least one of the following acceptable methods of contraception from at least 30 days prior to the time of informed consent through the time of study drug administration and for 8 weeks after last administration of study drug:
• Hormonal methods of contraception, including oral contraceptives containing combined estrogen and progesterone, a vaginal ring, injectable and implantable hormonal contraceptives, intrauterine hormone-releasing system (e.g., Mirena) and progestogen-only hormonal contraception associated with inhibition of ovulation
• Nonhormonal intrauterine device (IUD)
• Bilateral tubal occlusion
• Vasectomized subject/partner with documented azoospermia 90 days after procedure, if that partner is the sole sexual partner NOTE: WOCBP who are not exclusively in same-sex relationships must agree to use adequate contraception. WOCBP who are continuously not heterosexually active are exempt from contraceptive requirements. However, WOCBP must still undergo pregnancy testing as per protocol.

You may not qualify if:

• Are unwilling or unable to comply with study procedures, including follow-up, as specified by the protocol, or unwilling to cooperate fully with the Investigator
• Have a history of drug or alcohol abuse within 3 months of Screening
• Have an active malignancy or have a history of malignancy within the 5 years prior to Screening. (Subjects with prior basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that were successfully treated may be enrolled.)
• Have any of the following known active infections:
• Infection requiring systemic antiviral or antimicrobial therapy that would not have been completed within 30 days prior to screening
• Known history or positive test result for HIV, HBV or HCV
• Have any clinical history or other active medical condition, psychiatric disorder or clinically significant laboratory abnormality, vital sign, ECG abnormality or other finding that, in the investigator's opinion, is likely to increase the risk of study participation, confound study results, or interfere with study conduct or adherence
• Have any of the following:
• History of diabetes
• History of bleeding disorder or excessive bleeding
• Impaired renal function (estimated glomerular filtration rate \[eGFR\] \<60ml/min/1.73m2, using the CKD-EPI (2021 equation) \[See Appendix 1.\]
• Platelet count \< 125 X 109/L
• INR \> ULN
• Electrocardiogram (ECG) showing QTcF \> 470 msec female or \> 450 msec male
• Have received

Sponsors & Collaborators

• Juvena Therapeutics: lead

Study Sites (1)

Nucleus Network Pty Ltd

Melbourne, Victoria, 3004, Australia

Location

Study Officials

• Philip Ryan, MD

  Nucleus Network

  PRINCIPAL INVESTIGATOR

Study Design

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

Model Details: double-blind, placebo controlled

Study Record Dates

• First Submitted: March 24, 2025
• First Posted: April 9, 2025
• Study Start: April 14, 2025
• Primary Completion: April 30, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: April 20, 2026

Record last verified: 2026-04

Locations

AU (1)