A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function

NCT07713758 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: 359-201-00009
• Study Type: interventional
• Target: 32
• Locations: 0 countries
• Sites: N/A

Brief Summary

This trial is intended to understand how the body processes repinatrabit in people with moderate liver impairment and severe kidney impairment and also to compare these results with people of similar age, weight, and sex who have normal liver function.

Conditions

Phenylketonuria

Outcome Measures

Primary Outcomes (3)

• Groups 1 and 2: Unbound and Total Area Under the Concentration-Time Curve Calculated to the Last Observable Concentration at Time t (AUC0-t) of Repinatrabit

  From Day 1 to Day 8

• Groups 1 and 2: Unbound and Total Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC0-inf) of Repinatrabit

  From Day 1 to Day 8

• Groups 1 and 2: Unbound and Total Maximum (Peak) Plasma Concentration (Cmax) of Repinatrabit

  From Day 1 to Day 8

Secondary Outcomes (5)

• All Groups: Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Severe Adverse Events (SAEs)

  Up to Day 14

• All Groups: Number of Participants With Potentially Clinically Relevant Clinical Laboratory Test Results

  Up to Day 8

• All Groups: Number of Participants With Potentially Clinically Relevant Changes in Vital Signs

  Up to Day 8

• All Groups: Number of Participants With Potentially Clinically Relevant Changes in Physical Examination Findings

  Up to Day 8

• All Groups: Number of Participants With Potentially Clinically Relevant Changes in 12-lead Electrocardiogram (ECG) Results

  Up to Day 8

Study Arms (3)

Group 1: Moderate Hepatic Impairment

EXPERIMENTAL

Participants with moderate hepatic impairment will receive a single dose of repinatrabit tablet, under fasted conditions on Day 1.

Drug: Repinatrabit

Group 2: Severe Renal Impairment

EXPERIMENTAL

Participants with severe renal impairment will receive a single dose of repinatrabit tablet, under fasted conditions on Day 1.

Drug: Repinatrabit

Group 3: Matched Normal Hepatic and Renal Function

EXPERIMENTAL

Participants matched by age, weight, and sex with normal hepatic and renal function will receive a single dose of repinatrabit tablet, under fasted conditions on Day 1.

Drug: Repinatrabit

Interventions

Repinatrabit DRUG

Oral tablet

Also known as: JNT-517

Group 1: Moderate Hepatic Impairment; Group 2: Severe Renal Impairment; Group 3: Matched Normal Hepatic and Renal Function

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Body mass index (BMI) from 18.0 to 38.0 kilograms per square meter (kg/m\^2) (inclusive).
• Able to provide written, informed consent.
• Male participants and female participants of childbearing potential (POCBP) must agree to remain fully abstinent from intercourse where pregnancy can occur OR practice 2 different contraceptive methods, where at least one must be highly effective.
• Otherwise be in generally good health.
• Additional criteria for Group 1:
• i. Have moderate hepatic impairment determined using Child-Pugh classification (Class B).
• ii. Have relatively stable hepatic function for the duration of the study as determined by the investigator and liver function test at the time of screening or stable hepatic function 1 year prior to screening.
• iii. Have normal renal function. iv. Have a clinical diagnosis of hepatic cirrhosis based on biopsy and/or clinical criteria.
• Additional criteria for Group 2:
• i. Have estimated glomerular filtration rate based on the Chronic Kidney Disease-Epidemiology Collaboration (eGFRCKD-EPI) lesser than (\<) 30 milliliters per minute per 1.73 square meters (mL/min/1.73 m\^2) indicating severe renal impairment.
• ii. Not currently be on dialysis. iii. Have relatively stable renal function as determined by eGFRCKD-EPI. 7) Additional criteria for Group 3: i. eGFRCKD-EPI greater than or equal to (≥) 90 mL/min/1.73 m\^2 indicating normal renal function.
• ii. Have liver enzymes within normal limits.

You may not qualify if:

• Participants who are breastfeeding.
• Participants who have a positive pregnancy test result prior to receiving the investigational medical product (IMP).
• Participants with international normalized ratio (INR) greater than (\>) 2.3. Participants with hepatic impairment with elevated INR will be excluded based on the investigator's discretion.
• Participants with hepatic carcinoma or porto-hepatic shunts that have been surgically created or planted.
• A history of difficulty in obtaining intravenous access.
• The donation of blood or plasma \> 250 milliliters (mL) within 30 days prior to screening or received \> 50 mL of blood or plasma within 30 days prior to screening.
• Participants who have taken an investigational drug within 30 days prior to screening.
• Exposure to any substances known to stimulate hepatic microsomal enzymes within 30 days prior to screening (eg, occupational exposure to pesticides, organic solvents, etc).
• Any participant who, in the opinion of the investigator, should not participate in the trial.
• Previous exposure to repinatrabit.
• Additional criteria for Group 1: History of serious mental disorder including participants who have greater than Grade 1 encephalopathy as assessed by the Child-Pugh score and/or the investigator's judgment.
• Additional criteria for Group 2:
• i) Participants that have undergone a renal transplant. ii) Participants expected to require dialysis within 45 days. iii) Clinically significant abnormal findings in the serum chemistry, hematology, or urinalysis results obtained at screening or on Day -1, other than those associated with underlying renal conditions and other stable medical conditions consistent with the disease processes.
• Additional criteria for Group 3: History of or current hepatitis, or carriers of hepatitis B surface antigen (HBsAg) and/or hepatitis C antibodies (anti-HC).

Sponsors & Collaborators

• Otsuka Pharmaceutical Development & Commercialization, Inc.: lead

Related Links

• Otsuka Clinical Trial Website:
• Otsuka Clinical Trial Transparency:

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Otsuka Call Center

CONTACT

844-687-8522; Otsuka-ProfessionalServices@otsuka-us.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 15, 2026
• First Posted: July 20, 2026
• Study Start: July 17, 2026
• Primary Completion (Estimated): February 9, 2027
• Study Completion (Estimated): February 15, 2027
• Last Updated: July 20, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be available after marketing approval in global markets, or beginning 1-3 years following article publication. There is no end date to the availability of the data.
• Access Criteria: Otsuka will share data on the Vivli data sharing platform: https://vivli.org/ourmember/Otsuka/