Registry of Thiola EC Therapy

NCT05048563 | Completed

• 1 other identifier: 20-00331
• Study Type: observational
• Target: 25
• Locations: 1 country
• Sites: 4

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.

Conditions

Cystinuria

Outcome Measures

Primary Outcomes (10)

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with cystinuria on Thiola EC.

You may qualify if:

• years of age or older
• Patients with cystinuria and a history of kidney stones
• Taking Thiola EC
• Willing and able to provide consent

You may not qualify if:

• \. Unable to sign informed consent.

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (4)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

NYU Langone Health

New York, New York, 10016, United States

Location

Duke University

Durham, North Carolina, 27708, United States

Location

University of Texas Southwestern

Dallas, Texas, 75390, United States

Location

MeSH Terms

Conditions

Cystinuria

Condition Hierarchy (Ancestors)

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

Study Officials

• David Goldfarb, MD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2021
• First Posted: September 17, 2021
• Study Start: March 2, 2021
• Primary Completion: October 31, 2023
• Study Completion: October 31, 2023
• Last Updated: February 29, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: Beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research.
• Access Criteria: The investigator who proposed to use the data, researchers who provide a methodologically sound proposal, and investigators whose proposed use of the data has been approved by an independent review committee ("learned intermediary") identified for this purpose will have access to the data upon reasonable request. Requests should be directed to david.goldfarb@nyulangone.org. To gain access, data requestors will need to sign a data access agreement.

Locations

US (4)