Registry of Thiola EC Therapy
Study Details
Study Description
Brief 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. 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Study Design
Outcome Measures
Primary Outcome Measures
- Wisconsin Stone Quality Of Life (WiSQoL) Score [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 [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 [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 [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 [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 [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 [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 [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 [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 [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.
Eligibility Criteria
Criteria
Inclusion Criteria:
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18 years of age or older
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Patients with cystinuria and a history of kidney stones
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Taking Thiola EC
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Willing and able to provide consent
Exclusion Criteria:
- Unable to sign informed consent.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Alabama at Birmingham | Birmingham | Alabama | United States | 35294 |
| 2 | NYU Langone Health | New York | New York | United States | 10016 |
| 3 | Duke University | Durham | North Carolina | United States | 27708 |
| 4 | University of Texas Southwestern | Dallas | Texas | United States | 75390 |
Sponsors and Collaborators
- NYU Langone Health
Investigators
- Principal Investigator: David Goldfarb, MD, NYU Langone Health
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 20-00331