Registry of Thiola EC Therapy

Cystinuria Clinical Trial

Official title:

Open Label Prospective Observational Registry of Thiola EC Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05048563
• Other study ID #: 20-00331
• Status: Completed
• Start date: March 2, 2021
• Est. completion date: October 31, 2023

Study information

• Verified date: February 2024
• Source: NYU Langone Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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. 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: October 31, 2023
• Est. primary completion date: October 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystinuria

Locations

Country	Name	City	State
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	University of Texas Southwestern	Dallas	Texas
United States	Duke University	Durham	North Carolina
United States	NYU Langone Health	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
NYU Langone Health

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline
Primary	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.	Month 6
Primary	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.	Month 12
Primary	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.	Month 18
Primary	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.	Month 24
Primary	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.	Baseline
Primary	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.	Month 6
Primary	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.	Month 12
Primary	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.	Month 18
Primary	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.	Month 24