CystinuriaPhase 2ALANCT02910531

Lipoic Acid Supplement for Cystine Stone

A Phase 2 study testing Alpha lipoic acid in people with Cystinuria, run by Thomas Chi, MD at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Cystinuria
What is being tested?: Alpha lipoic acid
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 50 participants
Where: 1 siteUnited States
Study dates: 19 Jun 2017to 18 Dec 2024
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of California, San Francisco
San Francisco, California, 94143

Other trials in United States

About this study

Official title: The Effect of Lipoic Acid Natural Supplement on Cystine Stone Formation

Read the detailed description

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.

Conditions: Cystinuria
Being tested: Alpha lipoic acid, Placebo
Keywords: Cystinuria, Kidney stone, Alpha lipoic acid
Medical subject headings: Cystinuria, Kidney Calculi

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Alpha lipoic acidDietary supplement
Already mentioned in arm/group descriptions.
PlaceboDrug
Already mentioned in arm/group descriptions.

Study groups in NCT02910531
Study group	Role	What they receive
ALA supplement	Experimental	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. Receives: Dietary Supplement: Alpha lipoic acid
Placebo	Placebo comparator	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. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 3 years

Secondary outcomes (1)

Urinary Cystine Level
The secondary endpoints will be quantitative urinary cystine level determined by 24-hour urine collection.
Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

19 Jun 201718 Dec 2024

First posted22 Sep 2016 (estimated)
Study start19 Jun 2017
Primary completion18 Dec 2024
Study completion18 Dec 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (4)Papers the sponsor has linked to this study.
Other trials for CystinuriaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 50 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02910531
Sponsor's protocol ID: 16-20523
Other IDs: 2P20DK100863-03, OPD Grant Number 5716
Acronym: ALA
Status last verified: Jan 2026
First submitted: 20 Sep 2016
Last updated: 15 Apr 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Chillaron J, Font-Llitjos M, Fort J, Zorzano A, Goldfarb DS, Nunes V, Palacin M. Pathophysiology and treatment of cystinuria. Nat Rev Nephrol. 2010 Jul;6(7):424-34. doi: 10.1038/nrneph.2010.69. Epub 2010 Jun 1.
PubMed 20517292
Gomes MB, Negrato CA. Alpha-lipoic acid as a pleiotropic compound with potential therapeutic use in diabetes and other chronic diseases. Diabetol Metab Syndr. 2014 Jul 28;6(1):80. doi: 10.1186/1758-5996-6-80. eCollection 2014.
PubMed 25104975
Ziegler D, Low PA, Litchy WJ, Boulton AJ, Vinik AI, Freeman R, Samigullin R, Tritschler H, Munzel U, Maus J, Schutte K, Dyck PJ. Efficacy and safety of antioxidant treatment with alpha-lipoic acid over 4 years in diabetic polyneuropathy: the NATHAN 1 trial. Diabetes Care. 2011 Sep;34(9):2054-60. doi: 10.2337/dc11-0503. Epub 2011 Jul 20.
PubMed 21775755
Scholich H, Murphy ME, Sies H. Antioxidant activity of dihydrolipoate against microsomal lipid peroxidation and its dependence on alpha-tocopherol. Biochim Biophys Acta. 1989 Feb 20;1001(3):256-61. doi: 10.1016/0005-2760(89)90108-2.
PubMed 2492825

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Prospective Research Rare Kidney Stones (ProRKS)
Hyperoxaluria, Cystinuria
RecruitingObservational study
Rare Kidney Stone Consortium Biobank
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Rare Kidney Stone Consortium Patient Registry
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Monogenic Kidney Stone - Genetic Testing
Rare Kidney Stone Diseases
Active, not recruiting
Randall's Plaque Study: Pathogenesis and Relationship to Nephrolithiasis
Nephrocalcinosis, Renal Calculi

All Cystinuria trials

Study at a glance

From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: The Effect of Lipoic Acid Natural Supplement on Cystine Stone Formation
ClinicalTrials.gov ID: NCT02910531
Acronym / sponsor ID: ALA / 16-20523
Conditions: Cystinuria
Keywords: Cystinuria, Kidney stone, Alpha lipoic acid
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 50 participants (actual)
Lead sponsor: Thomas Chi, MDUniversity, hospital or other
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Study start: 19 Jun 2017 (actual)
Primary completion: 18 Dec 2024 (actual)
Study completion: 18 Dec 2024 (actual)
First posted: 22 Sep 2016 (estimated)
Results: Posted 15 Apr 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
ALA supplement	Experimental	Dietary Supplement: Alpha lipoic acidClinical 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.
Placebo	Placebo comparator	Drug: PlaceboClinical 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.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (2 listed)

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.

Key exclusion criteria (8 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Alpha lipoic acidAlready mentioned in arm/group descriptions.	Dietary supplement	ALA supplement
PlaceboAlready mentioned in arm/group descriptions.	Drug	Placebo

Primary outcome

Cystine Stone Recurrence
Time frame: 3 years
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.

1 secondary outcome

Urinary Cystine Level
Time frame: 3 years

Results and safety

Results posted to the registry on 15 Apr 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
ALA Supplement	25	16
Placebo	25	23

Primary outcome: Cystine Stone Recurrence

Time frame: 3 years. Count of participants. Unit: Participants

Measure	ALA Supplement n = 25	Placebo n = 25
Value	16	23

1 secondary outcome is reported in the full results.

Adverse events

Time frame: 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).. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
ALA Supplement	0 of 25 (0%)	4 of 25 (16%)	4 of 25
Placebo	0 of 25 (0%)	0 of 25 (0%)	0 of 25

Limitations and caveats (from the sponsor): This clinical trial experienced several patient withdrawls and although some were due to adverse events, many cited withdrawal due to the demand of trial participation. Alpha Lipoic Acid is available over-the-counter and easily obtainable without the demands of a clinical trial. This also made it much harder to recruit patients that had not already started self-medicating once the information about this trial was publicly widespread, further compromising an already limited participant pool.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 20517292Chillaron J, Font-Llitjos M, Fort J, Zorzano A, Goldfarb DS, Nunes V, Palacin M. Pathophysiology and treatment of cystinuria. Nat Rev Nephrol. 2010 Jul;6(7):424-34. doi: 10.1038/nrneph.2010.69. Epub 2010 Jun 1.
Background: PubMed 25104975Gomes MB, Negrato CA. Alpha-lipoic acid as a pleiotropic compound with potential therapeutic use in diabetes and other chronic diseases. Diabetol Metab Syndr. 2014 Jul 28;6(1):80. doi: 10.1186/1758-5996-6-80. eCollection 2014.
Background: PubMed 21775755Ziegler D, Low PA, Litchy WJ, Boulton AJ, Vinik AI, Freeman R, Samigullin R, Tritschler H, Munzel U, Maus J, Schutte K, Dyck PJ. Efficacy and safety of antioxidant treatment with alpha-lipoic acid over 4 years in diabetic polyneuropathy: the NATHAN 1 trial. Diabetes Care. 2011 Sep;34(9):2054-60. doi: 10.2337/dc11-0503. Epub 2011 Jul 20.
Background: PubMed 2492825Scholich H, Murphy ME, Sies H. Antioxidant activity of dihydrolipoate against microsomal lipid peroxidation and its dependence on alpha-tocopherol. Biochim Biophys Acta. 1989 Feb 20;1001(3):256-61. doi: 10.1016/0005-2760(89)90108-2.
Individual participant dataSharing planned: No

Related trials for Cystinuria

RecruitingObservational study
Prospective Research Rare Kidney Stones (ProRKS)
Hyperoxaluria, Cystinuria
RecruitingObservational study
Rare Kidney Stone Consortium Biobank
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Rare Kidney Stone Consortium Patient Registry
Primary Hyperoxaluria, Dent Disease
RecruitingObservational study
Monogenic Kidney Stone - Genetic Testing
Rare Kidney Stone Diseases
Active, not recruiting
Randall's Plaque Study: Pathogenesis and Relationship to Nephrolithiasis
Nephrocalcinosis, Renal Calculi

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Thomas Chi, MD principal investigator

University of California, San Francisco

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Alpha lipoic acid in people with Cystinuria, run by Thomas Chi, MD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Cystine Stone Recurrence (time frame: 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02910531, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2026.