Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria
2 other identifiers
interventional
10
1 country
1
Brief Summary
The purpose of this study is to evaluate the effect of escalating doses of cystine biding thiol drugs, including tiopronin and d-penicillamine, on the urinary cystine capacity, which is a measure of the amount of cystine in the urine, in patients with cystinuria. The overall goal will be to help guide therapy and ultimately minimize unnecessary side effects caused by larger doses.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_4
Started Jun 2012
Longer than P75 for phase_4
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2012
CompletedFirst Submitted
Initial submission to the registry
April 24, 2014
CompletedFirst Posted
Study publicly available on registry
April 29, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2017
CompletedResults Posted
Study results publicly available
August 23, 2021
CompletedAugust 23, 2021
July 1, 2021
5.5 years
April 24, 2014
July 17, 2019
July 28, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Cystine Capacity
We will test for a negative correlation between progressive increases in doses of thiol drugs and cystine capacity
4 weeks on assigned therapy
Study Arms (1)
CBTD Patients
EXPERIMENTALPart 1: patients will stop taking CBTDs for seven days and perform a 24-hour urine collection on day 7 Part 2: patients will take their usual CBTD, either tiopronin or d-penicillamine, 1g per day for 7 days, taken as 500 mg twice a day Part 3: patients will take a total of 2g of tiopronin or D-penicillamine daily for 7 days Part 4: patients will take a total of 3g/d of tiopronin or D-penicillamine, also for a 7 day period
Interventions
500mg PO BID x 7 days
1g PO BID x 7 days
1.5g PO BID x 7 days
Eligibility Criteria
You may qualify if:
- Be a patient with a confirmed diagnosis of cystinuria.
- Be already taking a CBTD (either D-penicillamine (Cuprimine®) or tiopronin (Thiola®) as part of your medication regimen.
- Be between 18 and 80 years of age
- Be enrolled in the Cystinuria Registry.
You may not qualify if:
- You are not a patient with cystinuria
- You are not already taking a cystine binding thiol drug
- You have renal colic (if you are passing a stone)
- You are scheduled to undergo a urologic procedure
- You are unwilling or unable to provide informed consent in order to be able to participate.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
NYU Langone Medical Center
New York, New York, 10016, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- David Goldfarb
- Organization
- NYU Langone Health
Study Officials
- PRINCIPAL INVESTIGATOR
David S Goldfarb, MD
NYU Langone Medical Center
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 24, 2014
First Posted
April 29, 2014
Study Start
June 1, 2012
Primary Completion
December 1, 2017
Study Completion
December 1, 2017
Last Updated
August 23, 2021
Results First Posted
August 23, 2021
Record last verified: 2021-07