Nephrogenic Diabetes Insipidus Clinical Trial
Official title:
Pharmacologic Treatment of Congenital Nephrogenic Diabetes Insipidus
The purpose of this research study is to determine if two investigational medications will be more effective in decreasing urine output than the currently available and routinely used medications in patients with congenital nephrogenic diabetes insipidus (NDI).
The study involves the use of the investigational medications sildenafil and calcitonin.
These medications have shown promise as treatment for NDI in laboratory (non-human) studies
but have not been used for treatment of NDI in humans. At this time, there is no guarantee
that these investigational medications will provide additional benefit to people with NDI.
The study is open to males, between the ages of 5 and 25 years who have been diagnosed with
Nephrogenic diabetes insipidus (NDI) and who have normal kidney and bladder function. A total
of 40 patients with NDI will be enrolled in the study. The study will involve two outpatient
clinic visits, followed by a 9-night hospital stay, followed by a final follow-up outpatient
clinic visit. All visits will take place within a 20-day time period.
At the first clinic visit, blood and urine testing for kidney and liver function and blood
count will be performed. If the genetic alteration which causes your NDI has not been
previously identified, blood for DNA testing will also be obtained. If a kidney and bladder
ultrasound has not been performed in the past 6 months, it will be obtained. The ultrasound
is to make sure that there is no problem with drainage of urine from the kidneys and bladder.
Subjects will be asked to fill out food preference questionnaires to use for planning of
meals for the hospital stay. Subjects will be given containers to collect two consecutive
24-hour urine samples at home. These urine collections will help determine how well the
subjects routine medicines are working to control their NDI.
At the second clinic visit, subjects will bring in the two 24-hour urine samples. Blood will
again be collected for further testing of kidney function. Subjects will be given containers
to collect another 24-hour urine just prior to the hospital visit.
The third visit requires hospital admission and will be scheduled at the study site closest
to the subjects home (The Children's Hospital, Denver, Colorado; University of Aarhus,
Denmark). For the hospital visit, subjects will need to stop their usual NDI medications for
48 hours prior to the visit. Subjects will perform another 24-hour urine collection on the
day prior to your hospital admission. This urine sample will be turned in to the laboratory
when you are admitted to the hospital for the research study. The length of the hospital stay
is 10 days/9 nights. During the stay, subjects can expect to have their weight, heart rate,
and blood pressure checked three times a day. All urine will be collected. Blood testing will
be performed every other day. Subjects will need to eat the meals provided at the hospital;
all meals will be provided according to a low-salt diet restriction. Subjects may drink
fluids as desired but they will need to avoid caffeine-containing beverages and alcohol.
On the first day of the hospital stay, testing will be performed to confirm the diagnosis of
NDI. This test involves administration of the medicine dDAVP (Desmopressin) through an IV
catheter (into a vein) with collection of urine every 30 minutes for 4 hours. subjects will
be randomized (like the toss of a coin) to receive either the investigational medication
treatment for 4 days followed by the routine medication treatment for 4 days or vice versa.
When subjects receive the routine medication treatment, they will receive placebos (inactive
substances like a sugar pill) in place of the investigational medicines. In this way, neither
the subject nor the investigator will know whether the subjects are receiving the
investigational or the routine medication treatment first. Medicines will be given twice a
day during the hospital stay. On the last day of the hospital stay, subjects will be
instructed to resume their normal diet and medications.
At a final outpatient clinic visit, blood testing and urinalysis will be performed.
Potential benefits of participation include a no-cost health examination, laboratory studies,
and an evaluation of current management of NDI. There is no cost for participation in this
research study. No pay will be given to participants in this research study.
This research study has been approved by the ethical review boards of the following
institutions: Colorado Multiple Institutional Review Board (#06-0588), Emory University
Institutional Review Board (#729-2005), and the University of Aarhus (#20050183). Individuals
who decide to take part in this research study will need to sign a specific consent form at a
participating institution as well as a release for use of personal health information (HIPAA
form).
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