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 ID
X10-201-00003
Clinicaltrials.gov ID
NCT07525960
EudraCT ID
N/A
EUCTIS ID
N/A
Sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Contact Information
844-687-8522 or [email protected]
Recruiting
Sponsor ID
X10-201-00003
Trial Details
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Age
18 - 55 Years
Therapeutic Area
  • X-linked congenital NDI (nephrogenic diabetes insipidus)
    • Sex
    Male
    Trial Dates
    Jul 2026 - Aug 2027
    Healthy Volunteers
    No
    • 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.
    • 3\) 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.

    Study 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.

    Additional Study Details

    Phase
    Phase 1
    Study Drug
    Drug: NDI-5001
    Type
    Interventional
    Masking
    None (Open Label)
    Estimated Enrollment
    24
    Additional Study Details

    Inclusion and Exclusion Criteria

    Inclusion 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).

    Exclusion Criteria

    Inclusion and Exclusion Criteria
    • 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.
    • 3\) 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.

    Locations

    Location
    Status
    Location
    Flourish Jacksonville
    Jacksonville, Florida, United States, 32277
    Status
    Recruiting
    Location
    Flourish San Antonio
    San Antonio, Texas, United States, 78023
    Status
    Recruiting

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    January 2026 01US25EUC0489

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