Not Yet RecruitingPhase 1

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

24 participantsStarted 2026-06-02

Plain-language 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.

Who can participate

Age range18 Years – 55 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Percentage Change From Baseline in 24-Hour Urine Volume (Uvol)

Timeframe: Up to Day 10

Percentage Change From Baseline in Spot Urine Osmolality (Uosm)

Timeframe: Up to Day 10

Trial details

NCT IDNCT07525960

SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-04-14

Contact for this trial

Otsuka Call Center

844-687-8522 otsukaprofessionalservices@otsuka-us.com

View on ClinicalTrials.gov More X-linked Congenital Nephrogenic Diabetes Insipidus trials