MedPath

Registry of Thiola EC Therapy

Completed
Conditions
Cystinuria
Registration Number
NCT05048563
Lead Sponsor
NYU Langone Health
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
25
Inclusion Criteria
  1. 18 years of age or older
  2. Patients with cystinuria and a history of kidney stones
  3. Taking Thiola EC
  4. Willing and able to provide consent
Exclusion Criteria
  1. Unable to sign informed consent.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Short Form-36 Health Survey (SF-36v2) ScoreMonth 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) ScoreMonth 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 Outcome Measures
NameTimeMethod

Trial Locations

Locations (4)

Duke University

🇺🇸

Durham, North Carolina, United States

NYU Langone Health

🇺🇸

New York, New York, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

University of Texas Southwestern

🇺🇸

Dallas, Texas, United States

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