Central Diabetes Insipidus (CDI) Clinical Trials

Central diabetes insipidus (CDI) results from deficiency of arginine vasopressin (antidiuretic hormone, ADH) due to dysfunction of the hypothalamic-pituitary system. Causes include pituitary or hypothalamic tumors (craniopharyngiomas, germinomas, adenomas), pituitary surgery or radiation therapy, traumatic brain injury, infections (meningitis, encephalitis, tuberculous meningitis), autoimmune hypophysitis, or idiopathic forms accounting for 30-50% of cases. Congenital forms result from genetic mutations affecting vasopressin synthesis (AVP gene mutations, DIDMOAD syndrome).

The most common symptoms of Central Diabetes Insipidus include Severe polydipsia (excessive thirst), Polyuria (excessive urination) with dilute urine, Dehydration and hypernatremia if intake inadequate, Nocturia and enuresis in children, Difficulty concentrating dilute urine, among others. Diabetes insipidus of all types affects about 1 in 25,000 people; central DI can be congenital or acquired. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Central Diabetes Insipidus from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Desmopressin (1-deamino-8-D-arginine vasopressin), synthetic vasopressin replacement, is the standard therapeutic agent and is highly effective. Several forms are available: nasal spray, oral tablets, an oral liquid (Desmoda, FDA-approved in February 2026 for adults and children), and injection under the skin or into a vein.

Yes. As of October 14, 2026, 1 FDA-approved medicine has a current label that names Central Diabetes Insipidus: Desmoda (desmopressin acetate oral solution). Corticosteroids and immune globulins count once each.

Central Diabetes Insipidus is caused by variants in the AVP gene. Only the rare familial form is genetic (AVP variants, almost always autosomal dominant); most cases are acquired from surgery, tumors, head injury, or infiltrative disease, and 30 to 50% are idiopathic.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it. Diabetes insipidus of all types affects about 1 in 25,000 people; central DI can be congenital or acquired. Genetic testing confirms the diagnosis.

Yes. Genetic testing for Central Diabetes Insipidus looks at the AVP gene. Only the rare familial form is genetic (AVP variants, almost always autosomal dominant); most cases are acquired from surgery, tumors, head injury, or infiltrative disease, and 30 to 50% are idiopathic.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it.

A confirmed variant establishes the diagnosis and is often required to enroll in gene-specific trials. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Central Diabetes Insipidus clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Central Diabetes Insipidus clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.