Trial Outcomes & Findings for Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria (NCT NCT03663855)
NCT ID: NCT03663855
Last Updated: 2021-12-17
Results Overview
This measure reflects the ability of urine to take up more cystine
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Baseline; Day 7
Results posted on
2021-12-17
Participant Flow
Multiple efforts to contact the PI/study team for the relevant data have failed. Therefore, per sequence data are not available to be reported for this assessment.
Participant milestones
| Measure |
Cystinuria Patients
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)
|
|---|---|
|
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 Tiopronin on Cystine Capacity in Patients With Cystinuria
Baseline characteristics by cohort
| Measure |
Cystinuria Patients
n=10 Participants
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)
|
|---|---|
|
Age, Continuous
|
45.3 years
STANDARD_DEVIATION 15.42 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline; Day 7This measure reflects the ability of urine to take up more cystine
Outcome measures
| Measure |
Cystinuria Patients
n=10 Participants
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)
|
|---|---|
|
Change in Cystine Capacity From Baseline
Phase 1
|
-63.6 mg/L
Standard Deviation 24.8
|
|
Change in Cystine Capacity From Baseline
Phase 2
|
76 mg/L
Standard Deviation 18.1
|
|
Change in Cystine Capacity From Baseline
Phase 3
|
53.4 mg/L
Standard Deviation 17.7
|
|
Change in Cystine Capacity From Baseline
Phase 4
|
103.1 mg/L
Standard Deviation 27.4
|
Adverse Events
Cystinuria 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
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place