CystinuriaNCT05048563
Registry of Thiola EC Therapy
An observational study of Cystinuria, run by NYU Langone Health at 4 sites in United States.
Trial summary
Thiola EC represents several modifications of Thiola that promise better, more efficacious therapy of cystinuria. First, pill size has changed from 100 mg to 300 mg, meaning that typical pill burden will be reduced from, on average, 10 pills per day to 3-5 pills per day. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystinuria
- How many people
- 25 participants
- Where
- 4 sitesUnited States
- Study dates
- 2 Mar 2021to 31 Oct 2023
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with cystinuria on Thiola EC.
Who may be able to join
- 18 years of age or older
- Patients with cystinuria and a history of kidney stones
- Taking Thiola EC
- Willing and able to provide consent
Who cannot join
- 1. Unable to sign informed consent.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
University of Alabama at Birmingham
Birmingham, Alabama, 35294
University of Texas Southwestern
Dallas, Texas, 75390
Duke University
Durham, North Carolina, 27708
NYU Langone Health
New York, New York, 10016
About this study
Official title: Open Label Prospective Observational Registry of Thiola EC Therapy
Thiola EC represents several modifications of Thiola that promise better, more efficacious therapy of cystinuria. First, pill size has changed from 100 mg to 300 mg, meaning that typical pill burden will be reduced from, on average, 10 pills per day to 3-5 pills per day. This change will be welcomed by patients whose fluid intake and administration of potassium citrate are daily impositions. Second, the preparation is now enteric-coated, formulated to offer delayed release of active tiopronin. Lastly, Thiola EC can be taken with food which is an improvement to the inconvenient dosing regimen of Thiola, which can only be taken one hour before, or two hours after meals. These changes may affect compliance and side effect profiles compared to those of Thiola. In combination with potassium citrate or other alkali preparations, adverse GI effects are relatively common in actively-treated patients with cystinuria. It is possible that GI side effects may be reduced by Thiola EC.
- Conditions
- Cystinuria
- Medical subject headings
- Cystinuria
What is being measured
The primary outcome is the main question the study is designed to answer.
Wisconsin Stone Quality Of Life (WiSQoL) Score
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Measured: Baseline
Wisconsin Stone Quality Of Life (WiSQoL) Score
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Measured: Month 6
Wisconsin Stone Quality Of Life (WiSQoL) Score
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Measured: Month 12
Wisconsin Stone Quality Of Life (WiSQoL) Score
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Measured: Month 18
Wisconsin Stone Quality Of Life (WiSQoL) Score
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Measured: Month 24
Short Form-36 Health Survey (SF-36v2) Score
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Measured: Baseline
Short Form-36 Health Survey (SF-36v2) Score
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Measured: Month 6
Short Form-36 Health Survey (SF-36v2) Score
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Measured: Month 12
Short Form-36 Health Survey (SF-36v2) Score
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Measured: Month 18
Short Form-36 Health Survey (SF-36v2) Score
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Measured: Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2021
- Study start2 Mar 2021
- Primary completion31 Oct 2023
- Study completion31 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- David Goldfarb, MD, Principal investigator, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05048563
- Sponsor's protocol ID
- 20-00331
- Status last verified
- Feb 2024
- First submitted
- 13 Sep 2021
- Last updated
- 29 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices) will be shared upon reasonable request.
Study at a glance
From the registry record, last updated by the sponsor on 29 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Prospective Observational Registry of Thiola EC Therapy
- ClinicalTrials.gov ID
- NCT05048563
- Acronym / sponsor ID
- 20-00331
- Conditions
- Cystinuria
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- 2 Mar 2021 (actual)
- Primary completion
- 31 Oct 2023 (actual)
- Study completion
- 31 Oct 2023 (actual)
- First posted
- 17 Sep 2021 (actual)
- 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.
Thiola EC represents several modifications of Thiola that promise better, more efficacious therapy of cystinuria. First, pill size has changed from 100 mg to 300 mg, meaning that typical pill burden will be reduced from, on average, 10 pills per day to 3-5 pills per day. This change will be welcomed by patients whose fluid intake and administration of potassium citrate are daily impositions. Second, the preparation is now enteric-coated, formulated to offer delayed release of active tiopronin. Lastly, Thiola EC can be taken with food which is an improvement to the inconvenient dosing regimen of Thiola, which can only be taken one hour before, or two hours after meals. These changes may affect compliance and side effect profiles compared to those of Thiola. In combination with potassium citrate or other alkali preparations, adverse GI effects are relatively common in actively-treated patients with cystinuria. It is possible that GI side effects may be reduced by Thiola EC.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with cystinuria on Thiola EC.
Key inclusion criteria (4 listed)
- 18 years of age or older
- Patients with cystinuria and a history of kidney stones
- Taking Thiola EC
- Willing and able to provide consent
Key exclusion criteria (1 listed)
- 1. Unable to sign 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.
Primary outcome
Wisconsin Stone Quality Of Life (WiSQoL) Score
Time frame: Baseline
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Wisconsin Stone Quality Of Life (WiSQoL) Score
Time frame: Month 6
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Wisconsin Stone Quality Of Life (WiSQoL) Score
Time frame: Month 12
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Wisconsin Stone Quality Of Life (WiSQoL) Score
Time frame: Month 18
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Wisconsin Stone Quality Of Life (WiSQoL) Score
Time frame: Month 24
The WiSQoL questionnaire is designed to understand the quality of life of patients with a history of kidney stones. There are 7 main questions that each have sub-questions. The questions are answered on a scale from 1 (very true / always or almost always) to 5 (not at all true / not at all, never). The total range of questions is 28-140; the lower the score, the worse the quality of life.
Short Form-36 Health Survey (SF-36v2) Score
Time frame: Baseline
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Short Form-36 Health Survey (SF-36v2) Score
Time frame: Month 6
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Short Form-36 Health Survey (SF-36v2) Score
Time frame: Month 12
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Short Form-36 Health Survey (SF-36v2) Score
Time frame: Month 18
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
Short Form-36 Health Survey (SF-36v2) Score
Time frame: Month 24
Scales are standardized with a scoring algorithm or by the SF-36v2 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status.
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 29 Feb 2024.
- Individual participant dataSharing planned: Yes. Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices) will be shared upon reasonable request.
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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 Goldfarb, MD principal investigator
NYU Langone Health
4 sites 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 4 sites 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 1 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 29 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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?
Wisconsin Stone Quality Of Life (WiSQoL) Score (time frame: Baseline). The study lists 10 primary outcomes in all. 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 29 Feb 2024.