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Recruiting NCT07525960

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

Phase I Interventional Diabetes Insipidus

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: NDI-5001.
Who it may be relevant to
Registry conditions: Diabetes Insipidus. Basic parameters: 18 years — 55 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

Interventions

  • Drug NDI-5001
    Capsule.

Primary outcome measures

  • Percentage Change From Baseline in 24-Hour Urine Volume (Uvol) [Time frame: Up to Day 10]
  • Percentage Change From Baseline in Spot Urine Osmolality (Uosm) [Time frame: Up to Day 10]
Secondary outcome measures (12)
  • Change From Time-Matched Baseline in Uvol [Time frame: Up to Day 10]
  • Change From Time-Matched Baseline in Uosm [Time frame: Up to Day 10]
  • Change From Baseline in 24-Hour Uosm [Time frame: Up to Day 10]
  • Correlation Coefficient Between 24-Hour Uvol and Uvol by interval [Time frame: Up to Day 10]
  • Correlation Coefficient Between 24-Hour Uvol and Uosm by Interval [Time frame: Up to Day 10]
  • Correlation Coefficient Between 24-Hour Uvol and Spot Uosm [Time frame: Up to Day 10]
  • Correlation Coefficient Between Change From Baseline in Uvol and Change From Baseline in Uosm by Interval [Time frame: Up to Day 10]
  • Number of Participants With Adverse Events (AEs) [Time frame: Up to Day 39]
  • Number of Participants With Clinically Relevant Changes in Clinical Laboratory Values [Time frame: Up to Day 31]
  • Number of Participants With Potentially Clinically Relevant Changes in Vital Signs [Time frame: Up to Day 31]
  • Number of Participants With Clinically Significant Abnormalities in Physical Examination Findings [Time frame: Up to Day 10]
  • Number of Participants With Potentially Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Parameters [Time frame: Up to Day 10]

Eligibility 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 2 centers
  • Flourish Jacksonville — Jacksonville
  • Flourish San Antonio — San Antonio

Identifiers

NCT: NCT07525960 · X10-201-00003

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗