CystinuriaPhase 4NCT02125721

Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria

A Phase 4 study testing Tiopronin 1g per day, Tiopronin 2g per day and Tiopronin 3g per day in people with Cystinuria, run by NYU Langone Health at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study is to evaluate the effect of escalating doses of cystine biding thiol drugs, including tiopronin and d-penicillamine, on the urinary cystine capacity, which is a measure of the amount of cystine in the urine, in patients with cystinuria. Read more about this study

Who is it for?: Ages 18 to 80 years
Conditions the study is for: Cystinuria
What is being tested?: Tiopronin 1g per day, Tiopronin 2g per day and Tiopronin 3g per day
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 10 participants
Where: 1 siteUnited States
Study dates: Jun 2012to Dec 2017
Phase: Phase 4
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 18 to 80 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Be a patient with a confirmed diagnosis of cystinuria.
Be already taking a CBTD (either D-penicillamine (Cuprimine®) or tiopronin (Thiola®) as part of your medication regimen.
Be between 18 and 80 years of age
Be enrolled in the Cystinuria Registry.

Who cannot join

You are not a patient with cystinuria
You are not already taking a cystine binding thiol drug
You have renal colic (if you are passing a stone)
You are scheduled to undergo a urologic procedure
You are unwilling or unable to provide informed consent in order to be able to participate.

Trial locations and contacts

1 site in 1 country.

United States1 site

NYU Langone Medical Center
New York, New York, 10016

Other trials in United States

About this study

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. As a result, a significant part of treatment is focused on prevention of stone formation. Current methods of prevention include increasing fluid intake, dietary modifications, alkali therapy, and cystine binding thiol drugs (CBTDs), which help increase the solubility of cystine in the urine. At present, the dosing of CBTDs is empiric, and not titrated to a specific measured effect. Our primary objective will be to measure the effect of increasing doses of CBTDs on cystine capacity. The investigators predict that higher dosages of these medications will lead to a more positive urinary cystine capacity, or the ability of urine to take up more cystine (and therefore decreased risk of stone formation). However, the dose at which increasing dosages of the medications cease to provide additional benefit is unknown. The investigators will directly measure the cystine capacity in the urine in response to increasing doses of medications to determine if there is a dose at which the maximum benefit of the drug exists.

This is a cross-over trial of escalating doses. There will be four parts to the study. In the first part, subjects will stop taking CBTDs for 7 days, and perform a 24-hour urine collection on day 7. In part 2, they will take their usual CBTD (either tiopronin or d-penicillamine) 1 gram per day for 7 days. They will perform another 24-hour urine collection on day 7 of this study period. In part 3, they will take their usual CBTD at 2 grams per day for 7 days and in part 4 they will take their CBTD at 3 grams per day for 7 days. Again, they will perform 24-hour urine collections on day 7 of each period. The order in which the parts of the study are performed will be randomized for each subject. The mean cystine capacities in each part of the study will be compared to determine the effect of drug dosage on urinary cystine capacity.

Conditions: Cystinuria
Being tested: Tiopronin 1g per day, Tiopronin 2g per day, Tiopronin 3g per day
Keywords: cystinuria, D-penicillamine (Cuprimine®), tiopronin (Thiola®)
Medical subject headings: Cystinuria

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Tiopronin 1g per dayDrug
Also called Thiola or tiopronin; d-penicillamine
500mg PO BID x 7 days
Tiopronin 2g per dayDrug
Also called Thiola or tiopronin; d-penicillamine
1g PO BID x 7 days
Tiopronin 3g per dayDrug
Also called Thiola or tiopronin; d-penicillamine
1.5g PO BID x 7 days

Study groups in NCT02125721
Study group	Role	What they receive
CBTD Patients	Experimental	Part 1: patients will stop taking CBTDs for seven days and perform a 24-hour urine collection on day 7 Part 2: patients will take their usual CBTD, either tiopronin or d-penicillamine, 1g per day for 7 days, taken as 500 mg twice a day Part 3: patients will take a total of 2g of tiopronin or D-penicillamine daily for 7 days Part 4: patients will take a total of 3g/d of tiopronin or D-penicillamine, also for a 7 day period Receives: Drug: Tiopronin 1g per day; Drug: Tiopronin 2g per day; Drug: Tiopronin 3g per day

What is being measured

The primary outcome is the main question the study is designed to answer.

Cystine Capacity
We will test for a negative correlation between progressive increases in doses of thiol drugs and cystine capacity
Measured: 4 weeks on assigned therapy

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 2012Dec 2017

First posted29 Apr 2014 (estimated)
Study startJun 2012
Primary completionDec 2017
Study completionDec 2017

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.
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: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 10 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02125721
Sponsor's protocol ID: 16-00108
Other IDs: 1U54DK083908
Status last verified: Jul 2021
First submitted: 24 Apr 2014
Last updated: 23 Aug 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Prospective Research Rare Kidney Stones (ProRKS)
Hyperoxaluria, Cystinuria
RecruitingObservational study
Rare Kidney Stone Consortium Biobank
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Rare Kidney Stone Consortium Patient Registry
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Monogenic Kidney Stone - Genetic Testing
Rare Kidney Stone Diseases
Active, not recruiting
Randall's Plaque Study: Pathogenesis and Relationship to Nephrolithiasis
Nephrocalcinosis, Renal Calculi

All Cystinuria trials

Study at a glance

From the registry record, last updated by the sponsor on 23 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria
ClinicalTrials.gov ID: NCT02125721
Acronym / sponsor ID: 16-00108
Conditions: Cystinuria
Keywords: cystinuria, D-penicillamine (Cuprimine®), tiopronin (Thiola®)
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 10 participants (actual)
Lead sponsor: NYU Langone HealthUniversity, hospital or other
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Study start: Jun 2012
Primary completion: Dec 2017 (actual)
Study completion: Dec 2017 (actual)
First posted: 29 Apr 2014 (estimated)
Results: Posted 23 Aug 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
CBTD Patients	Experimental	Drug: Tiopronin 1g per day, Drug: Tiopronin 2g per day, Drug: Tiopronin 3g per dayPart 1: patients will stop taking CBTDs for seven days and perform a 24-hour urine collection on day 7 Part 2: patients will take their usual CBTD, either tiopronin or d-penicillamine, 1g per day for 7 days, taken as 500 mg twice a day Part 3: patients will take a total of 2g of tiopronin or D-penicillamine daily for 7 days Part 4: patients will take a total of 3g/d of tiopronin or D-penicillamine, also for a 7 day period

Who may qualify

Ages: 18 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Be a patient with a confirmed diagnosis of cystinuria.
Be already taking a CBTD (either D-penicillamine (Cuprimine®) or tiopronin (Thiola®) as part of your medication regimen.
Be between 18 and 80 years of age
Be enrolled in the Cystinuria Registry.

Key exclusion criteria (5 listed)

You are not a patient with cystinuria
You are not already taking a cystine binding thiol drug
You have renal colic (if you are passing a stone)
You are scheduled to undergo a urologic procedure
You are unwilling or unable to provide informed consent in order to be able to participate.

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
Tiopronin 1g per dayAlso: Thiola or tiopronin; d-penicillamine500mg PO BID x 7 days	Drug	CBTD Patients
Tiopronin 2g per dayAlso: Thiola or tiopronin; d-penicillamine1g PO BID x 7 days	Drug	CBTD Patients
Tiopronin 3g per dayAlso: Thiola or tiopronin; d-penicillamine1.5g PO BID x 7 days	Drug	CBTD Patients

Primary outcome

Cystine Capacity
Time frame: 4 weeks on assigned therapy
We will test for a negative correlation between progressive increases in doses of thiol drugs and cystine capacity

Results and safety

Results posted to the registry on 23 Aug 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
CBTD Patients	10	10

Primary outcome: Cystine Capacity

Time frame: 4 weeks on assigned therapy. Mean (Standard Deviation). Unit: mg/L

Measure	Increasing Doses of CBTD n = 7
Value	43.1 (131.2)

Population analysed: Multiple efforts to contact the PI/study team for the relevant data have failed. Therefore, per intervention data are not available to be reported for this assessment.

Adverse events

Time frame: 10 Days. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
CBTD Patients	0 of 10 (0%)	0 of 10 (0%)	0 of 10

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Cystinuria

RecruitingObservational study
Prospective Research Rare Kidney Stones (ProRKS)
Hyperoxaluria, Cystinuria
RecruitingObservational study
Rare Kidney Stone Consortium Biobank
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Rare Kidney Stone Consortium Patient Registry
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Monogenic Kidney Stone - Genetic Testing
Rare Kidney Stone Diseases
Active, not recruiting
Randall's Plaque Study: Pathogenesis and Relationship to Nephrolithiasis
Nephrocalcinosis, Renal Calculi

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 Medical Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Tiopronin 1g per day, Tiopronin 2g per day and Tiopronin 3g per day in people with 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 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 23 Aug 2021. 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.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 Aug 2021). 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?

Cystine Capacity (time frame: 4 weeks on assigned therapy). 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 NCT02125721.

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 23 Aug 2021.