Open Label Prospective Observational Registry of Thiola EC Therapy
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- NYU Langone Health
- Enrollment
- 25
- Locations
- 4
- Primary Endpoint
- Short Form-36 Health Survey (SF-36v2) Score
Study Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •18 years of age or older
- •Patients with cystinuria and a history of kidney stones
- •Taking Thiola EC
- •Willing and able to provide consent
Exclusion Criteria
- •Unable to sign informed consent.
Outcomes
Primary Outcomes
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.
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.
Secondary Outcomes
No secondary outcomes reported