Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria

Cystinuria Clinical Trial

Official title:

Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03663855
• Other study ID #: 18-00642
• Status: Completed
• Phase: Phase 2
• Start date: November 1, 2018
• Est. completion date: September 10, 2019

Study information

• Verified date: November 2021
• Source: NYU Langone Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to determine the minimum effective dose of the cysteine binding thiol drug (CBTD) Tiopronin on urine cystine capacity, which is a measure of cystine solubility in the urine, in patients with cystinuria to evaluate the effect of escalating doses of cystine binding thiol drugs on the cystine capacity of the urine. The overall goal will be to help guide therapy and ultimately minimize unnecessary side effects caused by larger dosages.

Cystinuria is a rare genetic disease that can lead to significant morbidity in affected patients due to the recurrent nature of the disease. This study will follow the levels of urine cystine capacity in order to help guide treatment and to use lower than usually prescribed Tiopronin doses to decrease the potential side effects while maintaining therapeutic benefit. This will increase adherence with the medications by decreasing the burden of the large number of pills that need to be taken daily.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: September 10, 2019
• Est. primary completion date: September 10, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

Inclusion Criteria:

• Patients with a confirmed laboratory diagnosis of cystinuria who meet the following criteria: (1) stone analysis demonstrating cystine as a component, or (2) increased urinary cystine excretion (>250mg/24hrs in adults).

• A medical regimen that includes Tiopronin.

• Willing to use a medically accepted form of birth control, if female and of child bearing- potential

• Ability to reliably urinate in a collection vessel and measure urine volume.

• Ability to give informed consent.

• Documentation of a stable complete blood count (CBC) and urinalysis (UA) in the six month period prior to the date of enrollment.

• Enrollment in Rare Kidney Stone Consortium (RKSC) Protocol 6401 (Cystinuria Registry)

Exclusion Criteria:

• Women who are pregnant, breastfeeding, or trying to become pregnant

• Patients with renal colic

• Patients who are scheduled to undergo a surgical procedure

• Inability to give informed consent

Related Conditions & MeSH terms

• Cystinuria

Intervention

Drug:
• Tiopronin
Phase 1: No medication, Phase 2: 500mg PO daily x 7 days (total 500mg), Phase 3: 500mg PO BID x 7 days (total 1g), Phase 4: 1g PO BID x 7 days (total 2g)

Locations

Country	Name	City	State
United States	New York University School of Medicine	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	Change in Cystine Capacity From Baseline	This measure reflects the ability of urine to take up more cystine	Baseline; Day 7