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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

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

Who can join

Ages 18 to 55 · Men

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.

About the study

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.

What is being tested

• NDI-5001 (drug)

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. · Participants: 24 · Started: Jul 7, 2026

Contact the study team

• Otsuka Call Center · Phone: 844-687-8522

Official record on ClinicalTrials.gov — NCT07525960

Locations in the U.S.

Conditions

• Diabetes Insipidus

From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.