NCT07525960RecruitingPhase 1
A Phase 1b, Open-label Trial to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of NDI-5001 in a Capsule Following Daily Oral Doses to Adult Males With X-linked Congenital Nephrogenic Diabetes Insipidus
Otsuka Pharmaceutical Development & Commercialization, Inc.4 sites in 1 country24 target enrollmentStarted: July 7, 2026Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 1
- Status
- Recruiting
- Enrollment
- 24
- Locations
- 4
- Primary Endpoint
- Percentage Change From Baseline in 24-Hour Urine Volume (Uvol)
Study Overview
Brief Summary
The primary purpose of this trial is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NDI-5001 administered as a capsule following daily oral dosing in adult males with X-linked congenital nephrogenic diabetes insipidus (NDI) due to vasopressin receptor Type 2 (V2R) mutations.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 55 Years (Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •X-linked congenital NDI with a genotype-confirmed arginine vasopressin receptor 2 (gene) (AVPR2) mutation and no mutation in aquaporin-2 (AQP2).
- •Urine osmolality of ≤ 200 milliosmoles per kilogram of water (mOsm/kg) in a spot urine sample obtained from a first morning void upon awakening (at screening, check-in [Day -4], and Day -1) before consuming any food or drink after awakening.
- •3) BMI between 18.0 to 32.0 kilograms per square meter (kg/m^2), inclusive.
Exclusion Criteria
- •Participant who is not willing to follow a low-sodium (<=1 gram/day) diet during the in-clinic portion of the trial.
- •A positive drug screen for substances of abuse (including THC, cannabidiol) at screening or check-in.
- •Estimated glomerular filtration rate <60 milliliters per minute per 1.73-kilogram square meters by serum creatinine and cystatin C at screening.
- •Participants who have taken an investigational drug within 30 days prior to screening.
- •Previous exposure to NDI-
- •Any history of significant bleeding or hemorrhagic tendencies.
- •Note: Other protocol-specified inclusion/exclusion criteria may apply.
Arms & Interventions
NDI-5001
Experimental
Participants receive NDI-5001, orally, once daily (QD), for 9 consecutive days starting on Day 1.
Intervention: NDI-5001 (Drug)
Outcomes
Primary Outcomes
Percentage Change From Baseline in 24-Hour Urine Volume (Uvol)
Time Frame: Up to Day 10
Percentage Change From Baseline in Spot Urine Osmolality (Uosm)
Time Frame: Up to Day 10
Secondary Outcomes
- Change From Time-Matched Baseline in Uosm(Up to Day 10)
- Change From Baseline in 24-Hour Uosm(Up to Day 10)
- Number of Participants With Adverse Events (AEs)(Up to Day 39)
- Correlation Coefficient Between 24-Hour Uvol and Uvol by interval(Up to Day 10)
- Correlation Coefficient Between 24-Hour Uvol and Uosm by Interval(Up to Day 10)
- Correlation Coefficient Between 24-Hour Uvol and Spot Uosm(Up to Day 10)
- Number of Participants With Clinically Relevant Changes in Clinical Laboratory Values(Up to Day 31)
- Correlation Coefficient Between Change From Baseline in Uvol and Change From Baseline in Uosm by Interval(Up to Day 10)
- Change From Time-Matched Baseline in Uvol(Up to Day 10)
- Number of Participants With Potentially Clinically Relevant Changes in Vital Signs(Up to Day 31)
- Number of Participants With Clinically Significant Abnormalities in Physical Examination Findings(Up to Day 10)
- Number of Participants With Potentially Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Parameters(Up to Day 10)
- Maximum (Peak) Plasma Concentration (Cmax) of NDI-5001(Day 1 and Day 9)
- Time to Reach Maximum (Peak) Observed Plasma Concentration (Tmax) of NDI-5001(Day 1 and Day 9)
- Area Under the Concentration-time Curve From Time Zero to 24 hours (AUC0-24h) of NDI-5001(Up to 24 hours, post dose on Day 1 and Day 9)
- Dose-Normalized Maximum Observed Plasma Concentration (Cmax/Dose) of NDI-5001(Day 1 and Day 9)
- Dose-Normalized Area Under the Plasma Concentration-Time Curve From 0 to 24 Hours (AUC0-24h/Dose) of NDI-5001(Up to 24 hours, post dose on Day 1 and Day 9)
Investigators
Study Sites (4)
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