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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

RecruitingPhase 1Ages 18 Years - 55 YearsMaleOtsuka Pharmaceutical Development & Commercializa…
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
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. BMI between 18.0 to 35.0 kilograms per square meter (kg/m\^2), inclusive. 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 glomerular filtration rate \<60 milliliters per minute per 1.73-kilogram square meters by serum creatinine and cystatin C at screening. 4. Participants who have taken an investigational 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.
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What it tests

  • NDI-5001
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Where

3 sites
NRC Research Institute
Santa Ana, United States
Flourish Jacksonville
Jacksonville, United States
Flourish San Antonio
San Antonio, United States
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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