RECRUITING Phase 1 ClinicalTrials.gov

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

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. • ClinicalTrials.gov ID: NCT07525960

Registry updated: 2026-10-20

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Description

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

NDI-5001 (Drug)
Capsule.

Eligibility criteria

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) before consuming any food or drink after awakening. 3. BMI between 18.0 to 35.0 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.

Conditions

Diabetes Insipidus

Locations

NRC Research Institute — Santa Ana, California, United States
Flourish Jacksonville — Jacksonville, Florida, United States
Flourish San Antonio — San Antonio, Texas, United States

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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