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RecruitingNCT07525960Updated Oct 20, 2026

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 interventional study of NDI-5001 in Diabetes Insipidus, sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc.. Recruiting at 3 sites in United States. Open to male participants aged 18 Years to 55 Years. Per ClinicalTrials.gov, last updated 2026-10-20.

Sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc. (part of Otsuka Holdings Co., Ltd.) · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Started Jul 2026; still recruiting 4 months later.
Updated Oct 20, 2026Eligibility revisedStudy contact changed+1 moreGo to Updates ↓
Phase
Phase 1
Study type
Interventional
Enrollment
24
Allocation
Not applicable
Ages
18 Years to 55 Years
Sex
Male
01

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.

02

Conditions studied

  • Diabetes Insipidus

Browse trials for

Keywords

  • X-linked Congenital Nephrogenic Diabetes Insipidus
03

In context

Diabetes Insipidus

191 studies on the registry are indexed under Diabetes Insipidus; 35 are open to participants now.

This study's planned enrollment of 24 is below the median of 75 across 118 interventional studies indexed under Diabetes Insipidus.

Browse Diabetes Insipidus studies →

Lead sponsor

Otsuka Pharmaceutical Development & Commercialization, Inc. is the lead sponsor of 295 studies on the registry; 18 are open to participants now.

Of its 106 completed or terminated interventional studies of FDA-regulated products, 71 (67%) have results posted.

Browse Otsuka Pharmaceutical Development & Commercialization, Inc. studies →All Otsuka Holdings Co., Ltd. studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

The criteria were revised on the registry on Oct 20, 2026. See what changed

Ages eligible
18 Years to 55 Years
Sexes eligible
Male
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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).
  2. 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.
  3. between 18.0 to 35.0 kilograms per square meter (kg/m\^2), inclusive.

Exclusion criteria

Exclusion Criteria

  1. Participant who is not willing to follow a low-sodium (\<=1 gram/day) diet during the in-clinic portion of the trial.
  2. A positive drug screen for substances of abuse (including THC, cannabidiol) at screening or check-in.
  3. Estimated \<60 milliliters per minute per 1.73-kilogram square meters by serum creatinine and cystatin C at screening.
  4. Participants who have taken an drug within 30 days prior to screening.
  5. Previous exposure to NDI-5001.
  6. Any history of significant bleeding or hemorrhagic tendencies.

Note: Other protocol-specified inclusion/exclusion criteria may apply.

Terms in these criteria, in plain language(3)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

BMI (body mass index)

Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.

What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
24 participants (estimated)

Study arms

  • Experimental
    NDI-5001

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

    Drug: NDI-5001

Interventions

  • DrugNDI-5001

    Capsule.

06

What researchers measure

Primary outcomes

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

    Time frame: Up to Day 10

  2. Percentage Change From Baseline in Spot Urine Osmolality (Uosm)

    Time frame: Up to Day 10

Secondary outcomes

  1. Change From Time-Matched Baseline in Uvol

    Time frame: Up to Day 10

  2. Change From Time-Matched Baseline in Uosm

    Time frame: Up to Day 10

  3. Change From Baseline in 24-Hour Uosm

    Time frame: Up to Day 10

  4. Correlation Coefficient Between 24-Hour Uvol and Uvol by interval

    Time frame: Up to Day 10

  5. Correlation Coefficient Between 24-Hour Uvol and Uosm by Interval

    Time frame: Up to Day 10

  6. Correlation Coefficient Between 24-Hour Uvol and Spot Uosm

    Time frame: Up to Day 10

  7. Correlation Coefficient Between Change From Baseline in Uvol and Change From Baseline in Uosm by Interval

    Time frame: Up to Day 10

  8. Number of Participants With Adverse Events (AEs)

    Time frame: Up to Day 39

  9. Number of Participants With Clinically Relevant Changes in Clinical Laboratory Values

    Time frame: Up to Day 31

  10. Number of Participants With Potentially Clinically Relevant Changes in Vital Signs

    Time frame: Up to Day 31

  11. Number of Participants With Clinically Significant Abnormalities in Physical Examination Findings

    Time frame: Up to Day 10

  12. Number of Participants With Potentially Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Parameters

    Time frame: Up to Day 10

  13. Maximum (Peak) Plasma Concentration (Cmax) of NDI-5001

    Time frame: Day 1 and Day 9

  14. Time to Reach Maximum (Peak) Observed Plasma Concentration (Tmax) of NDI-5001

    Time frame: Day 1 and Day 9

  15. Area Under the Concentration-time Curve From Time Zero to 24 hours (AUC0-24h) of NDI-5001

    Time frame: Up to 24 hours, post dose on Day 1 and Day 9

  16. Dose-Normalized Maximum Observed Plasma Concentration (Cmax/Dose) of NDI-5001

    Time frame: Day 1 and Day 9

  17. Dose-Normalized Area Under the Plasma Concentration-Time Curve From 0 to 24 Hours (AUC0-24h/Dose) of NDI-5001

    Time frame: Up to 24 hours, post dose on Day 1 and Day 9

07

Study locations

2 of 3 sites recruiting
08

References and documents

Individual participant data

Plan to share: No

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Jul 2026 (actual). Primary completion is estimated for Aug 2027, 13 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Apr 6, 2026
First posted
Apr 13, 2026
StartActual
Jul 7, 2026
Primary completionEst.
Aug 25, 2027
CompletionEst.
Aug 25, 2027
Last update
Oct 20, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Sites
1 site added
Show site
  • NRC Research Institute · Santa Ana, United States
Oct 20, 2026
Eligibility
2 inclusion criteria edited
What changed in the criteria
Inclusion criteria
  • EditedUrine 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.
  • Edited3) BMI between 18.0 to 3235.0 kilograms per square meter (kg/m\^2), inclusive.
Oct 20, 2026
Contact
Study contact's phone or email changed: o•••@otsuka-us.com→O•••@otsuka-us.com. See the current contact
Oct 20, 2026
Show all 1 update
  1. Oct 20, 2026
    1 site added
    Show site
    • NRC Research Institute · Santa Ana, United States
    Eligibility 2 inclusion criteria edited
    What changed in the criteria
    Inclusion criteria
    • EditedUrine 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.
    • Edited3) BMI between 18.0 to 3235.0 kilograms per square meter (kg/m\^2), inclusive.
    Contact Study contact's phone or email changed: o•••@otsuka-us.com→O•••@otsuka-us.com. See the current contact
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07525960
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Responsible party
Sponsor
First posted
Apr 13, 2026
Start date
Jul 7, 2026
Primary completion
Aug 25, 2027 (estimated)
Completion
Aug 25, 2027 (estimated)
Last update
Oct 20, 2026

Study contacts

Otsuka Call Center
Contact
Otsuka-ProfessionalServices@otsuka-us.com
844-687-8522

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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