CystinuriaPhase 2NCT03663855

Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria

A Phase 2 study testing Tiopronin 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.

Trial summary

The purpose of this study is to determine the minimum effective dose of the cysteine binding thiol drug (CBTD) Tiopronin on urine cystine capacity, which is a measure of cystine solubility in the urine, in patients with cystinuria to evaluate the effect of escalating doses of cystine binding thiol drugs on the cystine… 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
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 10 participants
Where: 1 siteUnited States
Study dates: 1 Nov 2018to 10 Sep 2019
Phase: Phase 2
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

Patients with a confirmed laboratory diagnosis of cystinuria who meet the following criteria: (1) stone analysis demonstrating cystine as a component, or (2) increased urinary cystine excretion (>250mg/24hrs in adults).
A medical regimen that includes Tiopronin.
Willing to use a medically accepted form of birth control, if female and of child bearing- potential
Ability to reliably urinate in a collection vessel and measure urine volume.
Ability to give informed consent.
Documentation of a stable complete blood count (CBC) and urinalysis (UA) in the six month period prior to the date of enrollment.
Enrollment in Rare Kidney Stone Consortium (RKSC) Protocol 6401 (Cystinuria Registry)

Who cannot join

Women who are pregnant, breastfeeding, or trying to become pregnant
Patients with renal colic
Patients who are scheduled to undergo a surgical procedure
Inability to give informed consent

Trial locations and contacts

1 site in 1 country.

United States1 site

New York University School of Medicine
New York, New York, 10016

Other trials in United States

About this study

Cystinuria is a rare genetic disease that can lead to significant morbidity in affected patients due to the recurrent nature of the disease. This study will follow the levels of urine cystine capacity in order to help guide treatment and to use lower than usually prescribed Tiopronin doses to decrease the potential side effects while maintaining therapeutic benefit. This will increase adherence with the medications by decreasing the burden of the large number of pills that need to be taken daily.

Conditions: Cystinuria
Being tested: Tiopronin
Medical subject headings: Cystinuria

What participants receive

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

TioproninDrug
Phase 1: No medication, Phase 2: 500mg PO daily x 7 days (total 500mg), Phase 3: 500mg PO BID x 7 days (total 1g), Phase 4: 1g PO BID x 7 days (total 2g)

Study groups in NCT03663855
Study group	Role	What they receive
Cystinuria Patients	Experimental	Not described Receives: Drug: Tiopronin

What is being measured

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

Change in Cystine Capacity From Baseline
This measure reflects the ability of urine to take up more cystine
Measured: Baseline; Day 7

Timeline

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

1 Nov 201810 Sep 2019

First posted10 Sep 2018
Study start1 Nov 2018
Primary completion10 Sep 2019
Study completion10 Sep 2019

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 2
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: NCT03663855
Sponsor's protocol ID: 18-00642
Status last verified: Nov 2021
First submitted: 6 Sep 2018
Last updated: 17 Dec 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: All of the individual participant data collected during the trial, after deidentification. Researchers who provide a methodologically sound proposal.

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 17 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria
ClinicalTrials.gov ID: NCT03663855
Acronym / sponsor ID: 18-00642
Conditions: Cystinuria
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 10 participants (actual)
Lead sponsor: NYU Langone HealthUniversity, hospital or other
Study start: 1 Nov 2018 (actual)
Primary completion: 10 Sep 2019 (actual)
Study completion: 10 Sep 2019 (actual)
First posted: 10 Sep 2018 (actual)
Results: Posted 17 Dec 2021

Clinical summary

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

Study design

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

Arm	Type	Receives
Cystinuria Patients	Experimental	Drug: Tiopronin

Who may qualify

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

Key inclusion criteria (7 listed)

Patients with a confirmed laboratory diagnosis of cystinuria who meet the following criteria: (1) stone analysis demonstrating cystine as a component, or (2) increased urinary cystine excretion (>250mg/24hrs in adults).
A medical regimen that includes Tiopronin.
Willing to use a medically accepted form of birth control, if female and of child bearing- potential
Ability to reliably urinate in a collection vessel and measure urine volume.
Ability to give informed consent.

Key exclusion criteria (4 listed)

Women who are pregnant, breastfeeding, or trying to become pregnant
Patients with renal colic
Patients who are scheduled to undergo a surgical procedure
Inability to give informed consent

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
TioproninPhase 1: No medication, Phase 2: 500mg PO daily x 7 days (total 500mg), Phase 3: 500mg PO BID x 7 days (total 1g), Phase 4: 1g PO BID x 7 days (total 2g)	Drug	Cystinuria Patients

Primary outcome

Change in Cystine Capacity From Baseline
Time frame: Baseline; Day 7
This measure reflects the ability of urine to take up more cystine

Results and safety

Results posted to the registry on 17 Dec 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
Cystinuria Patients	10	10

Primary outcome: Change in Cystine Capacity From Baseline

Time frame: Baseline; Day 7. Mean (Standard Deviation). Unit: mg/L

Measure	Cystinuria Patients n = 10
Phase 1	-63.6 (24.8)
Phase 2	76 (18.1)
Phase 3	53.4 (17.7)
Phase 4	103.1 (27.4)

Adverse events

Time frame: 30 days. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cystinuria 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 17 Dec 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Yes. All of the individual participant data collected during the trial, after deidentification. Researchers who provide a methodologically sound proposal.

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 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?

A Phase 2 study testing Tiopronin 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 7 inclusion and 4 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 17 Dec 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 17 Dec 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?

Change in Cystine Capacity From Baseline (time frame: Baseline; Day 7). 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 NCT03663855.

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 17 Dec 2021.