Test of NDI-5001 in adult men with X-linked nephrogenic diabetes insipidus

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 1 interventional study evaluating safety, tolerability, and pharmacokinetics of daily oral NDI-5001 in adult males with AVPR2-linked nephrogenic diabetes insipidus. Participants will receive NDI-5001 capsules over multiple days and undergo PK/PD assessments to understand absorption and effects on water balance. Eligibility requires confirmed AVPR2 mutations, low morning urine osmolality, and a BMI within 18.0–35.0 kg/m^2, among other criteria. Key exclusions include high sodium intake, positive drug screens, kidney function concerns, prior NDI-5001 exposure, and recent investigational drug use. The study is sponsored by Otsuka and conducted at sites in California, Florida, and Texas.

Why it matters

Who can join: the full rules

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.

Where it's running

• Flourish Jacksonville
  Jacksonville, Florida, United States Recruiting
• Flourish San Antonio
  San Antonio, Texas, United States Recruiting
• NRC Research Institute
  Santa Ana, California, United States Not yet recruiting

Study contacts

• Study coordinator: Otsuka Call Center
• Email: Otsuka-ProfessionalServices@otsuka-us.com
• Phone: 844-687-8522

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• How oral estrogens affect blood oxytocin in healthy people and those with AVP deficiency

  This study will test whether two oral estrogens can trigger oxytocin release in healthy adults and in people with AVP-deficiency (central diabetes insipidus).

  Recruiting University Hospital, Basel, Switzerland · Basel · NCT07361263

• Effects of intranasal oxytocin on emotion recognition and stress in diabetes insipidus patients

  This study is testing if a nasal spray of oxytocin can help people with diabetes insipidus better recognize emotions and lower their stress levels compared to a placebo.

  Recruiting Phase 2 University Hospital, Basel, Switzerland · Basel · NCT06676774

• Lower dose radiotherapy for treating children with CNS germinoma

  This study is testing if giving a lower dose of radiotherapy after chemotherapy can effectively treat children with CNS germinomas while reducing side effects.

  Recruiting at 113 sites Phase 2 Children's Oncology Group · Mobile, Alabama and 114 other locations · NCT06368817

• Registry and clinical study for Wolfram syndrome and related disorders

  This study is trying to gather information from people with Wolfram syndrome to better understand the condition and its effects, while also looking for specific gene mutations.

  Recruiting Washington University School of Medicine · St Louis, Missouri · NCT02841553

• Intranasal oxytocin for sexual well‑being in people with arginine vasopressin deficiency and matched healthy adults

  This trial will test whether a nasal oxytocin spray can improve sexual well‑being, arousal, intimacy and related emotions in adults with arginine vasopressin deficiency and in matched healthy adults.

  Recruiting Phase 2 University Hospital, Basel, Switzerland · Basel · NCT06808516

• Effects of low-dose MDMA on oxytocin levels in patients with vasopressin deficiency and healthy controls

  This study is testing whether low-dose MDMA can increase oxytocin levels in people with a vasopressin deficiency and healthy individuals to see how it affects them differently.

  Recruiting University Hospital, Basel, Switzerland · Basel · NCT06789705

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.