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

Study Identifier:
X10-201-00003
ClinicalTrials.gov Identifier:
NCT07525960
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Contact Information:
844-687-8522 or [email protected]
Will Be Recruiting
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Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: NDI-5001
Date
Jun 2026 - Apr 2027
Phase 1/2
Phase 2/3
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 18 - 55 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion 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.

Protocol 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.

Study Locations

No locations found.
© 2025 Otsuka America Pharmaceutical,
Inc All rights reserved.

July 2025 01US25EUC0279

Requirements information
Inclusion 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.

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