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OpenTrials
Completed

NCT Number: NCT05048563

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.

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Key information

Conditions

Cystinuria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Kidney Diseases Male Urogenital Diseases Renal Aminoacidurias Renal Tubular Transport, Inborn Errors Urogenital Diseases Urologic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Alabama at Birmingham, Birmingham, Alabama, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. 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.

Sponsors and collaborators

Lead sponsor

NYU Langone Health

Other

Registry information

Official study title

Open Label Prospective Observational Registry of Thiola EC Therapy

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Sep 17, 2021
Registry last updated
Feb 29, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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