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]
Recruiting
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Study Details

Medical Condition
  • X-linked congenital NDI (nephrogenic diabetes insipidus)
Study Drug
  • Drug: NDI-5001
Date
Jul 2026 - Aug 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
  • 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) before consuming any food or drink after awakening.
  • BMI between 18.0 to 35.0 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

Location
Status
Location
NRC Research Institute
Santa Ana, California, United States, 92705
Status
Will Be Recruiting
Location
Flourish Jacksonville
Jacksonville, Florida, United States, 32277
Status
Recruiting
Location
Flourish San Antonio
San Antonio, Texas, United States, 78023
Status
Recruiting
© 2025 Otsuka America Pharmaceutical,
Inc All rights reserved.

July 2025 01US25EUC0279

Requirements information
  • 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) before consuming any food or drink after awakening.
  • BMI between 18.0 to 35.0 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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