NCT02910531

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jun 2017

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 20, 2016

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 22, 2016

Completed
9 months until next milestone

Study Start

First participant enrolled

June 19, 2017

Completed
7.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 18, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 18, 2024

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

April 15, 2026

Completed
Last Updated

April 15, 2026

Status Verified

January 1, 2026

Enrollment Period

7.5 years

First QC Date

September 20, 2016

Results QC Date

December 12, 2025

Last Update Submit

April 3, 2026

Conditions

Cystinuria

Keywords

CystinuriaKidney stoneAlpha lipoic acid

Outcome Measures

Primary Outcomes (1)

  • 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.

    3 years

Secondary Outcomes (1)

  • Urinary Cystine Level

    3 years

Study Arms (2)

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.

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.

Drug: Placebo

Interventions

Alpha lipoic acidDIETARY_SUPPLEMENT

Already mentioned in arm/group descriptions.

ALA supplement
PlaceboDRUG

Already mentioned in arm/group descriptions.

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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 may not qualify 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Related Publications (4)

  • 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.

    PMID: 20517292BACKGROUND

  • 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.

    PMID: 25104975BACKGROUND

  • 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.

    PMID: 21775755BACKGROUND

  • 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.

    PMID: 2492825BACKGROUND

MeSH Terms

Conditions

CystinuriaKidney Calculi

Interventions

Thioctic Acid

Condition Hierarchy (Ancestors)

Renal AminoaciduriasRenal Tubular Transport, Inborn ErrorsKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNephrolithiasisUrolithiasisUrinary CalculiCalculiPathological Conditions, AnatomicalPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Carboxylic AcidsOrganic ChemicalsThiophenesSulfur CompoundsCoenzymesEnzymes and CoenzymesFatty AcidsLipids

Limitations and Caveats

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.

Results Point of Contact

Title
Marshall Stoller, MD
Organization
University of California, San Francisco
Marshall.Stoller@ucsf.edu
415-353-2200

Study Officials

  • Thomas Chi, MD

    University of California, San Francisco

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor, Urology

Study Record Dates

First Submitted

September 20, 2016

First Posted

September 22, 2016

Study Start

June 19, 2017

Primary Completion

December 18, 2024

Study Completion

December 18, 2024

Last Updated

April 15, 2026

Results First Posted

April 15, 2026

Record last verified: 2026-01

Data Sharing

IPD Sharing
Will not share

Locations

US(1)