Recruiting
Phase 1

NDI-5001

Sponsor:

Otsuka Pharmaceutical Development & Commercialization, Inc.

Code:

NCT07525960

Conditions

Diabetes Insipidus

Eligibility Criteria

Sex: Male

Age: 18 - 55

Healthy Volunteers: Not accepted

Interventions

NDI-5001

Study Details

Brief 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.

Conditions

Diabetes Insipidus

Study ID

NCT07525960

Start date

Jul 7, 2026

Status verified date

Jul, 2026

Completion date

Aug 25, 2027

Anticipated

Primary completion date

Aug 25, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 18 - 55

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

24 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: NDI-5001

Participants receive NDI-5001, orally, once daily (QD), for 9 consecutive days starting on Day 1.

Interventions

NDI-5001

Capsule.

Primary outcome measure

  • Percentage Change From Baseline in 24-Hour Urine Volume (Uvol) [ Time Frame: Up to Day 10 ]
  • Percentage Change From Baseline in Spot Urine Osmolality (Uosm) [ Time Frame: Up to Day 10 ]

Central Contacts and Locations

Locations

Flourish Jacksonville

Recruiting

Jacksonville, Florida, United States, 32277

Contacts

Flourish San Antonio

Recruiting

San Antonio, Texas, United States, 78023

Contacts

More Information

Sponsor

Otsuka Pharmaceutical Development & Commercialization, Inc.

Last update posted

Jul 31, 2026

Last verified

Jul, 2026

Keywords

  • X-linked Congenital Nephrogenic Diabetes Insipidus

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Otsuka Pharmaceutical Development & Commercialization, Inc. on 2026-07-31.