Diabetes Insipidus
Conditions
Keywords
X-linked Congenital Nephrogenic Diabetes Insipidus
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.
Interventions
DRUGNDI-5001
Capsule.
Sponsors
Otsuka Pharmaceutical Development & Commercialization, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
1. X-linked congenital NDI with a genotype-confirmed arginine vasopressin receptor 2 (gene) (AVPR2) mutation and no mutation in aquaporin-2 (AQP2). 2. 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
1. Participant who is not willing to follow a low-sodium (\<=1 gram/day) diet during the in-clinic portion of the trial. 2. A positive drug screen for substances of abuse (including THC, cannabidiol) at screening or check-in. 3. Estimated glomerular filtration rate \<60 milliliters per minute per 1.73-kilogram square meters by serum creatinine and cystatin C at screening. 4. Participants who have taken an investigational drug within 30 days prior to screening. 5. Previous exposure to NDI-5001. 6. Any history of significant bleeding or hemorrhagic tendencies. Note: Other protocol-specified inclusion/
Design outcomes
Primary
| Measure | 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 |
Secondary
| Measure | 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 | 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 |
Countries
United States
Contacts
CONTACTOtsuka Call Center
Outcome results
None listed