CystinuriaTCUPSNCT02538016
TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study
A study testing Tolvaptan in people with Cystinuria, run by Caleb Nelson at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 12 to 29 years
- Conditions the study is for
- Cystinuria
- What is being tested?
- Tolvaptan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2016to Dec 2018
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 29 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Males and females age 12 - 29 years
- Weight ≥ 25kg (55 lbs)
- Confirmed cystinuria diagnosis
- Specific blood test levels (done within the past 6 months)
Who cannot join
- Concurrent non-renal disease that might increase risk of complications due to aquaresis
- Liver or biliary disease (chronic or acute)
- Malabsorption syndrome or other gastrointestinal condition that may interfere with response to therapy
- Non-cutaneous malignancy within last 5 years
- History of adverse reaction or allergy to Tolvaptan or other arginine vasopressin V2-receptor antagonists
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
Official title: Use of Tolvaptan, a Vasopressin Antagonist, to Increase Urine Dilution and Reduce Cystine Urolithiasis Among Patients With Homozygous Cystinuria: a Pilot Investigation
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.
- Conditions
- Cystinuria
- Being tested
- Tolvaptan
- Keywords
- Cystinuria, Kidney Stones
- Medical subject headings
- Cystinuria, Kidney Calculi
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TolvaptanDrug
| Study group | Role | What they receive |
|---|---|---|
| Tolvaptan | Other | Not described Receives: Drug: Tolvaptan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 23 days
Secondary outcomes (1)
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).
Measured: 11 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2015 (estimated)
- Study startOct 2016
- Primary completionDec 2018
- Study completionDec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for CystinuriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Caleb Nelson
- Sponsor type
- University, hospital or other
- Collaborators
- Otsuka America Pharmaceutical
- Responsible party
- Sponsor-investigator, Caleb Nelson, Boston Children's Hospital
- Investigators
- Caleb Nelson, MD, MPH, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02538016
- Sponsor's protocol ID
- 16726
- Acronym
- TCUPS
- Status last verified
- May 2020
- First submitted
- 21 Jul 2015
- Last updated
- 10 Jul 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Tolvaptan, a Vasopressin Antagonist, to Increase Urine Dilution and Reduce Cystine Urolithiasis Among Patients With Homozygous Cystinuria: a Pilot Investigation
- ClinicalTrials.gov ID
- NCT02538016
- Acronym / sponsor ID
- TCUPS / 16726
- Conditions
- Cystinuria
- Keywords
- Cystinuria, Kidney Stones
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Caleb NelsonUniversity, hospital or other
- Collaborators
- Otsuka America Pharmaceutical
- Study start
- Oct 2016
- Primary completion
- Dec 2018 (actual)
- Study completion
- Dec 2018 (actual)
- First posted
- 2 Sep 2015 (estimated)
- Results
- Posted 4 Jun 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tolvaptan | Other | Drug: Tolvaptan |
Who may qualify
- Ages
- 12 to 29 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Males and females age 12 - 29 years
- Weight ≥ 25kg (55 lbs)
- Confirmed cystinuria diagnosis
- Specific blood test levels (done within the past 6 months)
Key exclusion criteria (5 listed)
- Concurrent non-renal disease that might increase risk of complications due to aquaresis
- Liver or biliary disease (chronic or acute)
- Malabsorption syndrome or other gastrointestinal condition that may interfere with response to therapy
- Non-cutaneous malignancy within last 5 years
- History of adverse reaction or allergy to Tolvaptan or other arginine vasopressin V2-receptor antagonists
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tolvaptan | Drug | Tolvaptan |
Primary outcome
Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8)
Time frame: 23 days
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.
1 secondary outcome
Urine Osmolality at High Dose (Day 8)
Time frame: 11 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Caleb Nelson, MD, MPH principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Tolvaptan in people with Cystinuria, run by Caleb Nelson at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Jun 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8) (time frame: 23 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2020.