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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NDI-5001 | Experimental | Participants receive NDI-5001, orally, once daily (QD), for 9 consecutive days starting on Day 1. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NDI-5001 | Drug | Capsule. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Change From Baseline in 24-Hour Urine Volume (Uvol) | Up to Day 10 | |
| Percentage Change From Baseline in Spot Urine Osmolality (Uosm) | Up to Day 10 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Time-Matched Baseline in Uvol | Up to Day 10 | |
| Change From Time-Matched Baseline in Uosm | Up to Day 10 | |
| Change From Baseline in 24-Hour Uosm |
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Inclusion Criteria
3) BMI between 18.0 to 32.0 kilograms per square meter (kg/m^2), inclusive.
Exclusion Criteria
Note: Other protocol-specified inclusion/exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Otsuka Call Center | Contact | 844-687-8522 | otsukaprofessionalservices@otsuka-us.com |
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| Label | URL |
|---|---|
| Otsuka Clinical Trial Website: | View source |
| Otsuka Clinical Trial Transparency: | View source |
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| Up to Day 10 |
| 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 |
| Correlation Coefficient Between Change From Baseline in Uvol and Change From Baseline in Uosm by Interval | Up to Day 10 |
| Number of Participants With Adverse Events (AEs) | Up to Day 39 |
| Number of Participants With Clinically Relevant Changes in Clinical Laboratory Values | Up to Day 31 |
| 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 |