for people ages 18 years and up (full criteria)
at San Francisco, California
study started
completion around
Principal Investigator
by Thomas Chi, MD
Headshot of Thomas Chi
Thomas Chi



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.

Official Title

The Effect of Lipoic Acid Natural Supplement on Cystine Stone Formation


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.


Cystinuria, Kidney stone, Alpha lipoic acid, Thioctic Acid, ALA supplement


You can join if…

Open to people ages 18 years and up

  • Documented cystinuria on prior 24-hour urine collection and/or stone analysis; history of previous cystine kidney stones.
  • Being able and willing to provide consent.

You CAN'T join if...

  • Poorly controlled diabetes mellitus (hemoglobin A1C > 8.0% for more than 1 year).
  • Current alpha-lipoic acid administration at the time of screening or within the last year prior to screening.
  • Vulnerable populations including incarceration status.
  • Unable to give informed consent.
  • Non-English primary language.
  • Pregnancy, lactation, or child-bearing age without birth control devices.
  • Anticipation of pregnancy during the study period.
  • Serious illness likely to cause death within the next 5 years.


  • UCSF
    San Francisco California 94143 United States

Lead Scientist at UCSF

  • Thomas Chi, MD
    Professor, Urology, School of Medicine. Authored (or co-authored) 139 research publications. Research interests: Nephrolithiasis · Benign Prostatic Hypertrophy (BPH) · Ultrasound · Minimally Invasive Surgery · Laparoscopy


in progress, not accepting new patients
Start Date
Completion Date
Thomas Chi, MD
Phase 2 Cystinuria Research Study
Study Type
Expecting 50 study participants
Last Updated