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NCT Number: NCT07525960

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

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.

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Key information

Conditions

Diabetes Insipidus Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Kidney Diseases Male Urogenital Diseases Pituitary Diseases Urogenital Diseases Urologic Diseases

Age range

18 year–55 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1

Primary location

Flourish Jacksonville, Jacksonville, Florida, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

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.

Note: Other protocol-specified inclusion/exclusion criteria may apply.

Treatment and study plan

NDI-5001

Drug

Capsule.

Primary outcomes

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

    Time frame: Up to Day 10

  2. Percentage Change From Baseline in Spot Urine Osmolality (Uosm)

    Time frame: Up to Day 10

Secondary outcomes

  1. Change From Time-Matched Baseline in Uvol

    Time frame: Up to Day 10

  2. Change From Time-Matched Baseline in Uosm

    Time frame: Up to Day 10

  3. Change From Baseline in 24-Hour Uosm

    Time frame: Up to Day 10

  4. Correlation Coefficient Between 24-Hour Uvol and Uvol by interval

    Time frame: Up to Day 10

  5. Correlation Coefficient Between 24-Hour Uvol and Uosm by Interval

    Time frame: Up to Day 10

  6. Correlation Coefficient Between 24-Hour Uvol and Spot Uosm

    Time frame: Up to Day 10

  7. Correlation Coefficient Between Change From Baseline in Uvol and Change From Baseline in Uosm by Interval

    Time frame: Up to Day 10

  8. Number of Participants With Adverse Events (AEs)

    Time frame: Up to Day 39

  9. Number of Participants With Clinically Relevant Changes in Clinical Laboratory Values

    Time frame: Up to Day 31

  10. Number of Participants With Potentially Clinically Relevant Changes in Vital Signs

    Time frame: Up to Day 31

  11. Number of Participants With Clinically Significant Abnormalities in Physical Examination Findings

    Time frame: Up to Day 10

  12. Number of Participants With Potentially Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Parameters

    Time frame: Up to Day 10

  13. Maximum (Peak) Plasma Concentration (Cmax) of NDI-5001

    Time frame: Day 1 and Day 9

  14. Time to Reach Maximum (Peak) Observed Plasma Concentration (Tmax) of NDI-5001

    Time frame: Day 1 and Day 9

  15. Area Under the Concentration-time Curve From Time Zero to 24 hours (AUC0-24h) of NDI-5001

    Time frame: Up to 24 hours, post dose on Day 1 and Day 9

  16. Dose-Normalized Maximum Observed Plasma Concentration (Cmax/Dose) of NDI-5001

    Time frame: Day 1 and Day 9

  17. Dose-Normalized Area Under the Plasma Concentration-Time Curve From 0 to 24 Hours (AUC0-24h/Dose) of NDI-5001

    Time frame: Up to 24 hours, post dose on Day 1 and Day 9

Study contacts

Contact information is provided by the study sponsor or research team.

Otsuka Call Center

CONTACT

[email protected]

844-687-8522

Sponsors and collaborators

Lead sponsor

Otsuka Pharmaceutical Development & Commercialization, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Apr 13, 2026
Registry last updated
Jul 31, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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