A Study in Adult Males With X-linked Congenital Nephrogenic Diabetes Insipidus to Test the Effects of NDI-5001 Given for Multiple Days and to Test How NDI-5001 is Tolerated and Taken up in the Body
Otsuka Pharmaceutical Development & Commercialization, Inc.
Phase
Phase 1
Started
2026-07-07
Last updated
2026-07-31
Condition(s) studied
Diabetes Insipidus
Investigational drug(s) / intervention(s)
NDI-5001
NDI-5001: Capsule.
Study 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.
Eligibility
Sex
MALE
Min age
18 Years
Max age
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/exclusion criteria may apply.
Primary outcome measure(s)
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
Trial sites (2)
Facility
City
Region
Status
Flourish Jacksonville
Jacksonville
Florida
Recruiting
Flourish San Antonio
San Antonio
Texas
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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