Trial Outcomes & Findings for Lipoic Acid Supplement for Cystine Stone (NCT NCT02910531)
NCT ID: NCT02910531
Last Updated: 2026-04-15
Results Overview
The primary efficacy endpoint will be assessed in two ways: 1. symptomatic stone recurrences, defined as renal colic, stone passage, or surgical removal of a stone; 2. silent stone recurrences, classified as stone growth or new stones, diagnosed on the basis of renal ultrasound, plain KUB x-ray, or if clinically indicated, computed tomography.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
50 participants
Primary outcome timeframe
3 years
Results posted on
2026-04-15
Participant Flow
Potential participants were screened by their Primary Urologist outside of UCSF or from the UCSF urology clinic under the care of Drs. Stoller and Chi. Eligibility was determined by the following inclusion criteria: age 18 or older; documented cystinuria on prior 24-hour urine collection or stone analysis; and being able and willing to provide consent. This information was present at the time of the clinic visit for new participants or at follow-up visits for current patients of study PIs.
Participants were computer sequence-randomized to receive either oral daily dosing of 1200 mg ALA or placebo for three years.
Participant milestones
| Measure |
ALA Supplement
Subjects in this study arm will be taking one supplement tablet containing 1200 mg of alpha lipoic acid orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
Placebo
Subjects in this study arm will be taking one placebo tablet containing 10 mg of sucrose orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
|---|---|---|
|
Overall Study
STARTED
|
25
|
25
|
|
Overall Study
COMPLETED
|
16
|
23
|
|
Overall Study
NOT COMPLETED
|
9
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Lipoic Acid Supplement for Cystine Stone
Baseline characteristics by cohort
| Measure |
ALA Supplement
n=25 Participants
Subjects in this study arm will be taking one supplement tablet containing 1200 mg of alpha lipoic acid orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
Placebo
n=25 Participants
Subjects in this study arm will be taking one placebo tablet containing 10 mg of sucrose orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
Total
n=50 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
18 Participants
n=193 Participants
|
19 Participants
n=193 Participants
|
37 Participants
n=386 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=193 Participants
|
6 Participants
n=193 Participants
|
13 Participants
n=386 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=193 Participants
|
10 Participants
n=193 Participants
|
27 Participants
n=386 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=193 Participants
|
15 Participants
n=193 Participants
|
23 Participants
n=386 Participants
|
|
baseline non-contrast CT scan
|
25 Participants
n=193 Participants
|
25 Participants
n=193 Participants
|
50 Participants
n=386 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=193 Participants
|
1 Participants
n=193 Participants
|
1 Participants
n=386 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
25 Participants
n=193 Participants
|
24 Participants
n=193 Participants
|
49 Participants
n=386 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
2 Participants
n=386 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Race (NIH/OMB)
White
|
23 Participants
n=193 Participants
|
25 Participants
n=193 Participants
|
48 Participants
n=386 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=193 Participants
|
0 Participants
n=193 Participants
|
0 Participants
n=386 Participants
|
PRIMARY outcome
Timeframe: 3 yearsThe primary efficacy endpoint will be assessed in two ways: 1. symptomatic stone recurrences, defined as renal colic, stone passage, or surgical removal of a stone; 2. silent stone recurrences, classified as stone growth or new stones, diagnosed on the basis of renal ultrasound, plain KUB x-ray, or if clinically indicated, computed tomography.
Outcome measures
| Measure |
ALA Supplement
n=25 Participants
Subjects in this study arm will be taking one supplement tablet containing 1200 mg of alpha lipoic acid orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
Placebo
n=25 Participants
Subjects in this study arm will be taking one placebo tablet containing 10 mg of sucrose orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
|---|---|---|
|
Cystine Stone Recurrence
|
16 Participants
|
23 Participants
|
SECONDARY outcome
Timeframe: 3 yearsThe secondary endpoints will be quantitative urinary cystine level determined by 24-hour urine collection.
Outcome measures
| Measure |
ALA Supplement
n=25 Participants
Subjects in this study arm will be taking one supplement tablet containing 1200 mg of alpha lipoic acid orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
Placebo
n=25 Participants
Subjects in this study arm will be taking one placebo tablet containing 10 mg of sucrose orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
|---|---|---|
|
Urinary Cystine Level
|
0.8 mg/d
Standard Deviation 0.7
|
0.6 mg/d
Standard Deviation 0.4
|
Adverse Events
ALA Supplement
Serious events: 0 serious events
Other events: 4 other events
Deaths: 4 deaths
Placebo
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
ALA Supplement
n=25 participants at risk
Subjects in this study arm will be taking one supplement tablet containing 1200 mg of alpha lipoic acid orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
Placebo
n=25 participants at risk
Subjects in this study arm will be taking one placebo tablet containing 10 mg of sucrose orally once daily for three years.
At the end of the three years of study drug treatment, all subjects will undergo a low dose non-contrast CT scan to look for a silent change in stone size.
|
|---|---|---|
|
Gastrointestinal disorders
nausea
|
8.0%
2/25 • From enrollment through the end of follow up and completion of the study for each participant, which is approximately 5 years (2 years on the study drug, 3 years in follow up).
The investigational product is an over-the-counter natural supplement. The risk of adverse events is low.
|
0.00%
0/25 • From enrollment through the end of follow up and completion of the study for each participant, which is approximately 5 years (2 years on the study drug, 3 years in follow up).
The investigational product is an over-the-counter natural supplement. The risk of adverse events is low.
|
|
Skin and subcutaneous tissue disorders
rash
|
4.0%
1/25 • From enrollment through the end of follow up and completion of the study for each participant, which is approximately 5 years (2 years on the study drug, 3 years in follow up).
The investigational product is an over-the-counter natural supplement. The risk of adverse events is low.
|
0.00%
0/25 • From enrollment through the end of follow up and completion of the study for each participant, which is approximately 5 years (2 years on the study drug, 3 years in follow up).
The investigational product is an over-the-counter natural supplement. The risk of adverse events is low.
|
|
Gastrointestinal disorders
reflux
|
4.0%
1/25 • From enrollment through the end of follow up and completion of the study for each participant, which is approximately 5 years (2 years on the study drug, 3 years in follow up).
The investigational product is an over-the-counter natural supplement. The risk of adverse events is low.
|
0.00%
0/25 • From enrollment through the end of follow up and completion of the study for each participant, which is approximately 5 years (2 years on the study drug, 3 years in follow up).
The investigational product is an over-the-counter natural supplement. The risk of adverse events is low.
|
Additional Information
Marshall Stoller, MD
University of California, San Francisco
Phone: 415-353-2200
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place