Trial Outcomes & Findings for Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria (NCT NCT02125721)

NCT ID: NCT02125721

Last Updated: 2021-08-23

Results Overview

We will test for a negative correlation between progressive increases in doses of thiol drugs and cystine capacity

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

10 participants

Primary outcome timeframe

4 weeks on assigned therapy

Results posted on

2021-08-23

Participant Flow

Participant flow data was not provided "per sequence". Multiple efforts were made to obtain information, but have not been successful.

Participant milestones

Participant milestones
Measure	CBTD Patients Part 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
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CBTD Patients n=10 Participants Part 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
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks on assigned therapy

Population: Multiple efforts to contact the PI/study team for the relevant data have failed. Therefore, per intervention data are not available to be reported for this assessment.

We will test for a negative correlation between progressive increases in doses of thiol drugs and cystine capacity

Outcome measures

Outcome measures
Measure	Increasing Doses of CBTD n=7 Participants Intervention: CBTD 0-3 gm CBTD 0-3 gm: Oral CBTD 0-3 gm dose/day for 7 days, dose escalation
Cystine Capacity	43.1 mg/L Standard Deviation 131.2

Adverse Events

CBTD Patients

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

David Goldfarb

NYU Langone Health

Phone: 212 263 0744

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place