A Phase 2 interventional study of Alpha lipoic acid and Placebo in Cystinuria, sponsored by Thomas Chi, MD. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-04-15.
Sponsored by Thomas Chi, MD · Phase 2, Interventional, and Prevention
This study evaluates how daily alpha lipoic acid supplementation affects cystine kidney stone recurrence. Half of the subjects will receive 1200 mg alpha lipoic acid orally daily for three years, while the other half will receive a placebo. The funding source for this clinical trial is FDA OOPD.
Cystinuria is a rare inherited autosomal recessive disorder of the kidney that is the result of a defect in the dibasic amino acid transporter in the renal proximal tubule and small intestine. Supersaturation of cystine in the urine produces crystals that precipitate and form calculi, which can be a cause of obstruction, infection, and chronic kidney disease (Chillarón 2010).
One potential therapeutic is a thiol-containing compound alpha-lipoic acid (thioctic acid, 5-(1,2-dithiolan-3- yl) pentanoic acid, ALA). It is an over-the-counter supplement with antioxidant property. Once ALA is transported into the cell, it is reduced to dihydrolipoic acid (DHLA). Both ALA and DHLA have direct antioxidant activity (Scholich 1989), and they can regenerate endogenous antioxidants including ascorbic acid and vitamin E. It can also increase intracellular coenzyme Q10 and glutathione levels. ALA and DHLA also have additional biochemical effects as metal chelators, reactive oxygen species scavengers, and modulators of signaling transduction of several pathways (Gomes 2014).
While the potential therapeutic effects of ALA have been studied in a number of diseases including, for example, Alzheimer's disease, obesity, cardiovascular disease, hypertension, and several cancers (Gomes 2014), the efficacy of ALA has been best studied in type 2 diabetic peripheral neuropathy (Ziegler 2011). In our lab, results from a mouse model of cystinuria show that ALA markedly slows the initiation of cystine stone formation as well as the growth of existing stones.
Given this history in clinical medicine and, most importantly, based upon our positive findings of ALA effectiveness in a mouse model of cystinuria, we propose a pilot study on the use of this molecule in cystinuric patients.
21 studies on the registry are indexed under Cystinuria; 5 are open to participants now.
Browse Cystinuria studies →This is the only study on the registry with Thomas Chi, MD as lead sponsor.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinical data including medical history, plain KUB x-ray and renal ultrasound, routine blood work and 24-hour urine collections for all subjects will be collected as part of normal clinical care at routine clinical visit every 4 months. 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.
Dietary Supplement: Alpha lipoic acid
Clinical data including medical history, plain KUB x-ray and renal ultrasound, routine blood work and 24-hour urine collections for all subjects will be collected as part of normal clinical care at routine clinical visit every 4 months. 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.
Drug: Placebo
Already mentioned in arm/group descriptions.
Already mentioned in arm/group descriptions.
Cystine Stone Recurrence
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.
Time frame: 3 years
Urinary Cystine Level
The secondary endpoints will be quantitative urinary cystine level determined by 24-hour urine collection.
Time frame: 3 years
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.
| Milestone | ALA Supplement | Placebo |
|---|---|---|
| Started | 25 | 25 |
| Completed | 16 | 23 |
| Not completed | 9 | 2 |
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.
| Participants | ALA Supplement | Placebo |
|---|---|---|
| Cystine Stone Recurrence | 16 | 23 |
The secondary endpoints will be quantitative urinary cystine level determined by 24-hour urine collection.
| mg/d | ALA Supplement | Placebo |
|---|---|---|
| Urinary Cystine Level | 0.8 ± 0.7 | 0.6 ± 0.4 |
Collected over 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).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| ALA Supplement | 4/25 (16%) | 0/25 (0%) | 4/25 (16%) |
| Placebo | 0/25 (0%) | 0/25 (0%) | 0/25 (0%) |
| Event | ALA Supplement | Placebo |
|---|---|---|
| nauseaGastrointestinal disorders | 2/25 | 0/25 |
| rashSkin and subcutaneous tissue disorders | 1/25 | 0/25 |
| refluxGastrointestinal disorders | 1/25 | 0/25 |
| Age, Categorical(Participants) | ALA Supplement | Placebo | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 18 | 19 | 37 |
| >=65 years | 7 | 6 | 13 |
| Sex: Female, Male(Participants) | ALA Supplement | Placebo | Total |
|---|---|---|---|
| Female | 17 | 10 | 27 |
| Male | 8 | 15 | 23 |
| baseline non-contrast CT scan(Participants) | ALA Supplement | Placebo | Total |
|---|---|---|---|
| Count of participants | 25 | 25 | 50 |
| Ethnicity (NIH/OMB)(Participants) | ALA Supplement | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 1 | 1 |
| Not Hispanic or Latino | 25 | 24 | 49 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | ALA Supplement | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 2 | 0 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 23 | 25 | 48 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Thioctic Acid