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

Recruiting now · Phase 1

Conditions studied: Diabetes Insipidus

In brief

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.

Key facts

Study ID

NCT07525960

Run by

Otsuka Pharmaceutical Development & Commercialization, Inc.

People needed

24

Starts

2026-07-07

Expected to finish

2027-08-25

Last updated by the study team

2026-07-31

Who can join

Age: 18 and older, up to 55. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• BMI between 18.0 to 32.0 kilograms per square meter (kg/m\^2), inclusive.

You may not qualify if…

• 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-5001.
• Any history of significant bleeding or hemorrhagic tendencies.
• Note: Other protocol-specified inclusion/exclusion criteria may apply.

Where it is running

• Flourish Jacksonville — Jacksonville, Florida, United States (enrolling)
• Flourish San Antonio — San Antonio, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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