An interventional study of Tolvaptan in Cystinuria, sponsored by Caleb Nelson. Completed at 1 site in United States. Open to participants aged 12 Years to 29 Years. Per ClinicalTrials.gov, last updated 2020-07-10.
Sponsored by Caleb Nelson · Not applicable, Interventional, and Treatment
The goal of this research project is to establish that in short-term use, tolvaptan is a safe and potentially effective new therapy for cystinuria, by conducting a short-term pilot study of the safety and tolerability of this drug, and assess impact on urinary stone risk parameters, among adolescent and young adult patients with clinical cystinuria.
21 studies on the registry are indexed under Cystinuria; 5 are open to participants now.
Browse Cystinuria studies →This is the only study on the registry with Caleb Nelson as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Tolvaptan
Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8)
The primary outcome was urinary cystine supersaturation as measured by "cystine capacity". This proprietary test (Litholink Corp., Chicago IL) is reported as a value in mg/L above or below zero, with positive values indicating urine undersaturated with cystine, while negative values indicate that the urine is supersaturated with cystine. Four 24-hour urine samples were obtained during the study: one at baseline 3-6 days prior to the measurement, one on day 3-4 of the dosing period, one on day 7-8 of the dosing period, and one 3-6 days after the washout period. Each 24 hour urine sample was sent individually for analysis by Litholink Corp, which performs the cystine assays.
Time frame: 23 days
Urine Osmolality at High Dose (Day 8)
Secondary outcomes included serum sodium and other electrolyte levels. Serum electrolytes for each subject before, during, and after tolvaptan treatment (Sodium/Potassium/Chloride in mmol/L).
Time frame: 11 days
Recruitment and Enrollment period was between August 2016 to December 2018. Subjects were recruited from our existing database of cystinuria patients, from new patients presenting during the research period, and via advertising through the International Cystinuria Foundation, Rare Kidney Stone Consortium, and social media.
| Milestone | Tolvaptan |
|---|---|
| Started | 4 |
| Completed | 4 |
| Not completed | 0 |
The primary outcome was urinary cystine supersaturation as measured by "cystine capacity". This proprietary test (Litholink Corp., Chicago IL) is reported as a value in mg/L above or below zero, with positive values indicating urine undersaturated with cystine, while negative values indicate that the urine is supersaturated with cystine. Four 24-hour urine samples were obtained during the study: one at baseline 3-6 days prior to the measurement, one on day 3-4 of the dosing period, one on day 7-8 of the dosing period, and one 3-6 days after the washout period. Each 24 hour urine sample was sent individually for analysis by Litholink Corp, which performs the cystine assays.
| mg/L | Tolvaptan |
|---|---|
| Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8) | 70 (-3 to 111) |
Secondary outcomes included serum sodium and other electrolyte levels. Serum electrolytes for each subject before, during, and after tolvaptan treatment (Sodium/Potassium/Chloride in mmol/L).
| mmol/L | Tolvaptan |
|---|---|
| Sodium | 141 (139 to 142) |
| Potassium | 4.05 (3.7 to 4.4) |
| Chloride | 102 (101 to 103) |
Collected over 2 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Tolvaptan | 0/4 (0%) | 0/4 (0%) | 4/4 (100%) |
| Event | Tolvaptan |
|---|---|
| Severe ThirstInvestigations | 4/4 |
| Age, Categorical(Participants) | Tolvaptan |
|---|---|
| <=18 years | 2 |
| Between 18 and 65 years | 2 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Tolvaptan |
|---|---|
| Female | 2 |
| Male | 2 |
| Race and Ethnicity Not Collected(Participants) | Tolvaptan |
|---|
PDFs hosted by the registry, ClinicalTrials.gov.
This study is completed, as verified in May 2020. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Tolvaptan