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

NCT07525960 | Recruiting Phase 1 FDA Drug

• 1 other identifier: X10-201-00003
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 2

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

Keywords

X-linked Congenital Nephrogenic Diabetes Insipidus

Outcome Measures

Primary Outcomes (2)

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

  Up to Day 10

• Percentage Change From Baseline in Spot Urine Osmolality (Uosm)

  Up to Day 10

Secondary Outcomes (17)

• Change From Time-Matched Baseline in Uvol

  Up to Day 10

• Change From Time-Matched Baseline in Uosm

  Up to Day 10

• Change From Baseline in 24-Hour Uosm

  Up to Day 10

• Correlation Coefficient Between 24-Hour Uvol and Uvol by interval

  Up to Day 10

• Correlation Coefficient Between 24-Hour Uvol and Uosm by Interval

  Up to Day 10

Study Arms (1)

NDI-5001

EXPERIMENTAL

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

Drug: NDI-5001

Interventions

NDI-5001 DRUG

Capsule.

NDI-5001

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Otsuka Pharmaceutical Development & Commercialization, Inc.: lead

Study Sites (2)

Flourish Jacksonville

Jacksonville, Florida, 32277, United States

RECRUITING

Flourish San Antonio

San Antonio, Texas, 78023, United States

RECRUITING

Related Links

• Otsuka Clinical Trial Website:
• Otsuka Clinical Trial Transparency:

MeSH Terms

Conditions

Diabetes Insipidus

Condition Hierarchy (Ancestors)

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

Central Study Contacts

Otsuka Call Center

CONTACT

844-687-8522; otsukaprofessionalservices@otsuka-us.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 6, 2026
• First Posted: April 13, 2026
• Study Start: July 7, 2026
• Primary Completion (Estimated): August 25, 2027
• Study Completion (Estimated): August 25, 2027
• Last Updated: July 31, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)