Diabetes InsipidusPhase 1NCT07525960
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
A Phase 1 study testing NDI-5001 in people with Diabetes Insipidus, run by Otsuka Pharmaceutical Development & Commercialization, Inc. at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 55 years, men only
- Conditions the study is for
- Diabetes Insipidus
- What is being tested?
- NDI-5001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 3 sitesUnited States
- Study dates
- 7 Jul 2026to 25 Aug 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Flourish Jacksonville
Jacksonville, Florida, 32277
Chebib, 844-687-8522, Otsuka-ProfessionalServices@otsuka-us.com
- Recruiting
Flourish San Antonio
San Antonio, Texas, 78023
Denham, 844-687-8522, Otsuka-ProfessionalServices@otsuka-us.com
- Not yet recruiting
NRC Research Institute
Santa Ana, California, 92705
Chueh, 844-687-8522, Otsuka-ProfessionalServices@otsuka-us.com
About this study
Official 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
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
- Being tested
- NDI-5001
- Keywords
- X-linked Congenital Nephrogenic Diabetes Insipidus
- Medical subject headings
- Diabetes Insipidus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NDI-5001Drug
Capsule.
| Study group | Role | What they receive |
|---|---|---|
| NDI-5001 | Experimental | Participants receive NDI-5001, orally, once daily (QD), for 9 consecutive days starting on Day 1. Receives: Drug: NDI-5001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Change From Baseline in 24-Hour Urine Volume (Uvol)
Measured: Up to Day 10
Percentage Change From Baseline in Spot Urine Osmolality (Uosm)
Measured: Up to Day 10
Secondary outcomes (17)
Change From Time-Matched Baseline in Uvol
Measured: Up to Day 10
Change From Time-Matched Baseline in Uosm
Measured: Up to Day 10
Change From Baseline in 24-Hour Uosm
Measured: Up to Day 10
Correlation Coefficient Between 24-Hour Uvol and Uvol by interval
Measured: Up to Day 10
Correlation Coefficient Between 24-Hour Uvol and Uosm by Interval
Measured: Up to Day 10
Correlation Coefficient Between 24-Hour Uvol and Spot Uosm
Measured: Up to Day 10
Correlation Coefficient Between Change From Baseline in Uvol and Change From Baseline in Uosm by Interval
Measured: Up to Day 10
Number of Participants With Adverse Events (AEs)
Measured: Up to Day 39
Number of Participants With Clinically Relevant Changes in Clinical Laboratory Values
Measured: Up to Day 31
Number of Participants With Potentially Clinically Relevant Changes in Vital Signs
Measured: Up to Day 31
Number of Participants With Clinically Significant Abnormalities in Physical Examination Findings
Measured: Up to Day 10
Number of Participants With Potentially Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Parameters
Measured: Up to Day 10
Maximum (Peak) Plasma Concentration (Cmax) of NDI-5001
Measured: Day 1 and Day 9
Time to Reach Maximum (Peak) Observed Plasma Concentration (Tmax) of NDI-5001
Measured: Day 1 and Day 9
Area Under the Concentration-time Curve From Time Zero to 24 hours (AUC0-24h) of NDI-5001
Measured: Up to 24 hours, post dose on Day 1 and Day 9
Dose-Normalized Maximum Observed Plasma Concentration (Cmax/Dose) of NDI-5001
Measured: Day 1 and Day 9
Dose-Normalized Area Under the Plasma Concentration-Time Curve From 0 to 24 Hours (AUC0-24h/Dose) of NDI-5001
Measured: Up to 24 hours, post dose on Day 1 and Day 9
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2026
- Study start7 Jul 2026
- Primary completion25 Aug 2027 (estimated)
- Study completion25 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetes InsipidusStudies of the same condition, open and under way.
- More trials run by Otsuka Pharmaceutical Development & Commercialization, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07525960
- Sponsor's protocol ID
- X10-201-00003
- Status last verified
- Oct 2026
- First submitted
- 6 Apr 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No