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

NCT03663855 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 18-00642
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Cystinuria

Outcome Measures

Primary Outcomes (1)

• Change in Cystine Capacity From Baseline

  This measure reflects the ability of urine to take up more cystine

  Baseline; Day 7

Study Arms (1)

Cystinuria Patients

EXPERIMENTAL

Drug: Tiopronin

Interventions

Tiopronin DRUG

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)

Cystinuria Patients

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

New York University School of Medicine

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Cystinuria

Interventions

Tiopronin

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

Intervention Hierarchy (Ancestors)

Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins; N-substituted Glycines; Glycine

Results Point of Contact

• Title: David Goldfarb, MD
• Organization: NYU Langone Health

David.Goldfarb@nyulangone.org

(212) 263-7300

Study Officials

• David Goldfarb, MD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 6, 2018
• First Posted: September 10, 2018
• Study Start: November 1, 2018
• Primary Completion: September 10, 2019
• Study Completion: September 10, 2019
• Last Updated: December 17, 2021
• Results First Posted: December 17, 2021

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: CSR, ANALYTIC CODE
• Time Frame: Beginning 9 months and ending 36 months following article publication.
• Access Criteria: Proposals may be submitted up to 36 months following article publication. After 36 months the data will be available in our University's data warehouse but without investigator support other than deposited metadata. Information regarding submitting requests and accessing data may be found at (Link to be provided

Locations

US (1)