CystinuriaNCT02120105
Cystine Capacity Clinical Study (CysCap)
An observational study of Cystinuria, run by NYU Langone Health at 1 site in United States.
Trial summary
The purpose of this study is to determine whether urinary cystine capacity, an assay used to measure the amount of cystine in the urine, can be used to predict stone recurrence in patients with cystinuria. Read more about this study
- Who is it for?
- Ages 8 to 80 years
- Conditions the study is for
- Cystinuria
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2012to 24 Feb 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
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 8 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: For patients to be enrolled in this study they must have a diagnosis of cystinuria.
Who may be able to join
- Patients with a confirmed laboratory diagnosis of cystinuria will be included in this study. There will be no age barrier to inclusion, but included patients must be able to reliably collect urine for 24 hours. Men and women will be recruited equally and there are no restrictions for racial or ethnic origin in this study.
- Patient enrollment will require signing of an informed consent document approved by the Lenox Hill IRB.
- For children 8-18 years of age, signing an assent to participate will also be required.
Who cannot join
- Patients will be excluded if they cannot sign consent or assent.
- Furthermore if the patient cannot reliably collect urine for 24 hours or adhere to study follow up visit requirements.
Trial locations and contacts
1 site in 1 country.
United States1 site
NYU Langone Medical Center
New York, New York, 10016
About this study
The purpose of this study is to determine whether urinary cystine capacity, an assay used to measure the amount of cystine in the urine, can be used to predict stone recurrence in patients with cystinuria.
Read the detailed description
Cystinuria is a rare genetic cause of kidney stones that leads to significant morbidity due to the recurrent nature of the disease. Despite recent advancements in knowledge about cystinuria, such as the discovery of the genetic defects that cause the disease, there have been very few studies of clinical determinants of recurrent stone formation. An optimal method of measuring cystine solubility in the urine has been lacking, and therefore response to pharmacologic and dietary therapy is often not known. Recently, a new assay to measure the amount of cystine in the urine was developed called Cystine Capacity, or CysCap. It is an assay that adds a pre-formed amount of cystine crystals to urine and measures the amount of cystine the urine can take up from the solid phase (in undersaturated urine) or gives up to solid phase (in supersaturated urine). This study seeks to examine how well this measure of urinary cystine predicts stone occurrence, with the ultimate goal of helping to guide therapy and preventing kidney stones in cystinurics.
This is an observational study over 3 years. Subjects who are enrolled in the study will perform semi-annual 24-hour urine collections. They will undergo imaging of the kidneys every 6 months to monitor kidney stone formation or growth, in order to correlate urinary parameters with clinical events. The study will examine how well the urinary cystine capacity predicts stone formation.
- Conditions
- Cystinuria
- Keywords
- Renal aminoaciduria, cystinuria, D-penicillamine, renal tubular transport, inborn errors, tiopronin, urolithiasis
- Medical subject headings
- Cystinuria, Renal Aminoacidurias, Urolithiasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cystinuria | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cystine capacity will be correlated with recurrence or non-recurrence of stones
The primary end point will be cystine capacity in participants who do and do not have recurrence of stones seen on radiologic imaging in the ipsilateral kidney which was rendered stone-free prior to enrollment.
Subjects who are enrolled in the study will perform semi-annual 24-hour urine collections for measurement of cystine capacity. They will undergo imaging of the kidneys every 6 months to monitor kidney stone formation or growth, in order to correlate urinary parameters with clinical events.
Measured: every 6 months over 3 years
Secondary outcomes (3)
24-hour urine cystine excretion
We will compare 24 hour urine cystine excretion in patients who remain stone free and those who experience stone recurrence.
Measured: every 6 months over 3 years
episodes of renal colic and/or stone passage from a kidney that was previously stone-free
Assessed by renal imaging, either ultrasound or CT scan, every 6 months.
Measured: every 6 months over 3 years
Evidence of new asymptomatic stones noted on the contralateral kidney
Cystine capacity will be compared in participants with or without recurrence of stones in the contralateral kidney by renal ultrasound or CT scan
Measured: every 6 months over 3 year observational study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2014 (estimated)
- Study startSep 2012
- Primary completion24 Feb 2017
- Study completion24 Feb 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for CystinuriaStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- David S Goldfarb, MD, Principal investigator, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02120105
- Sponsor's protocol ID
- 6402
- Status last verified
- Nov 2018
- First submitted
- 4 Jun 2013
- Last updated
- 3 Dec 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Goldfarb DS, Coe FL, Asplin JR. Urinary cystine excretion and capacity in patients with cystinuria. Kidney Int. 2006 Mar;69(6):1041-7. doi: 10.1038/sj.ki.5000104.
PubMed 16501494
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cystine Capacity Clinical Study (CysCap)
- ClinicalTrials.gov ID
- NCT02120105
- Acronym / sponsor ID
- 6402
- Conditions
- Cystinuria
- Keywords
- Renal aminoaciduria, cystinuria, D-penicillamine, renal tubular transport, inborn errors, tiopronin, urolithiasis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- Sep 2012 (actual)
- Primary completion
- 24 Feb 2017 (actual)
- Study completion
- 24 Feb 2017 (actual)
- First posted
- 22 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether urinary cystine capacity, an assay used to measure the amount of cystine in the urine, can be used to predict stone recurrence in patients with cystinuria.
Detailed description
Cystinuria is a rare genetic cause of kidney stones that leads to significant morbidity due to the recurrent nature of the disease. Despite recent advancements in knowledge about cystinuria, such as the discovery of the genetic defects that cause the disease, there have been very few studies of clinical determinants of recurrent stone formation. An optimal method of measuring cystine solubility in the urine has been lacking, and therefore response to pharmacologic and dietary therapy is often not known. Recently, a new assay to measure the amount of cystine in the urine was developed called Cystine Capacity, or CysCap. It is an assay that adds a pre-formed amount of cystine crystals to urine and measures the amount of cystine the urine can take up from the solid phase (in undersaturated urine) or gives up to solid phase (in supersaturated urine). This study seeks to examine how well this measure of urinary cystine predicts stone occurrence, with the ultimate goal of helping to guide therapy and preventing kidney stones in cystinurics.
This is an observational study over 3 years. Subjects who are enrolled in the study will perform semi-annual 24-hour urine collections. They will undergo imaging of the kidneys every 6 months to monitor kidney stone formation or growth, in order to correlate urinary parameters with clinical events. The study will examine how well the urinary cystine capacity predicts stone formation.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cystinuria | – | – |
Who may qualify
- Ages
- 8 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- For patients to be enrolled in this study they must have a diagnosis of cystinuria.
Key inclusion criteria (3 listed)
- Patients with a confirmed laboratory diagnosis of cystinuria will be included in this study. There will be no age barrier to inclusion, but included patients must be able to reliably collect urine for 24 hours. Men and women will be recruited equally and there are no restrictions for racial or ethnic origin in this study.
- Patient enrollment will require signing of an informed consent document approved by the Lenox Hill IRB.
- For children 8-18 years of age, signing an assent to participate will also be required.
Key exclusion criteria (2 listed)
- Patients will be excluded if they cannot sign consent or assent.
- Furthermore if the patient cannot reliably collect urine for 24 hours or adhere to study follow up visit requirements.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Cystine capacity will be correlated with recurrence or non-recurrence of stones
Time frame: every 6 months over 3 years
The primary end point will be cystine capacity in participants who do and do not have recurrence of stones seen on radiologic imaging in the ipsilateral kidney which was rendered stone-free prior to enrollment. Subjects who are enrolled in the study will perform semi-annual 24-hour urine collections for measurement of cystine capacity. They will undergo imaging of the kidneys every 6 months to monitor kidney stone formation or growth, in order to correlate urinary parameters with clinical events.
3 secondary outcomes
24-hour urine cystine excretion
Time frame: every 6 months over 3 years
episodes of renal colic and/or stone passage from a kidney that was previously stone-free
Time frame: every 6 months over 3 years
Evidence of new asymptomatic stones noted on the contralateral kidney
Time frame: every 6 months over 3 year observational study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2018.
- Background: PubMed 16501494Goldfarb DS, Coe FL, Asplin JR. Urinary cystine excretion and capacity in patients with cystinuria. Kidney Int. 2006 Mar;69(6):1041-7. doi: 10.1038/sj.ki.5000104.
- Rare Kidney Stone ConsortiumLink provided by the sponsor.
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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.
David S Goldfarb, MD principal investigator
NYU Langone Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystinuria, run by NYU Langone Health 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 3 inclusion and 2 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 3 Dec 2018. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Cystine capacity will be correlated with recurrence or non-recurrence of stones (time frame: every 6 months over 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02120105, which also lists 1 linked publication.
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 3 Dec 2018.