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

Официальное название

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

Обзор

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.

Вмешательства

• Препарат NDI-5001
  Capsule.

Первичные конечные точки

• Percentage Change From Baseline in 24-Hour Urine Volume (Uvol) [Срок оценки: Up to Day 10]
• Percentage Change From Baseline in Spot Urine Osmolality (Uosm) [Срок оценки: Up to Day 10]

Вторичные конечные точки (12)

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

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 2 центра

• Flourish Jacksonville — Jacksonville
• Flourish San Antonio — San Antonio

Идентификаторы

NCT: NCT07525960 · X10-201-00003

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗