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

Otsuka

Status and phase: Active, not recruiting; Phase 1

Conditions: Diabetes Insipidus

Treatments: Drug: NDI-5001

Study type: Interventional

Funder types: Industry

Identifiers

NCT07525960

X10-201-00003

Details and patient eligibility

About

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.

Enrollment: 24 estimated patients

Sex: Male

Ages: 18 to 55 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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, 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

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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24 participants in 1 patient group

NDI-5001

Experimental group

Description:

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

Treatment:

Drug: NDI-5001