Alpha Lipoic Acid for Cystinuria

(ALA Trial)

Overseen By Victoria Hogue, MA

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of California, San Francisco

Must not be taking: Alpha-lipoic acid

Disqualifiers: Diabetes, Pregnancy, Serious illness, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

Duration: 3 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if daily alpha lipoic acid can reduce the recurrence of cystine kidney stones. Participants will take either a 1200 mg supplement of alpha lipoic acid or a placebo (a pill with no active ingredient) for three years. The trial seeks individuals who have previously had cystine kidney stones and can provide consent to participate. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you are currently taking alpha-lipoic acid, you would need to stop, as the trial excludes those who have taken it within the last year.

Is there any evidence suggesting that alpha lipoic acid is likely to be safe for humans?

Research has shown that alpha lipoic acid is generally safe for people. Studies have found it to be well-tolerated, even in large amounts. For example, animal studies noted that only very high doses caused slight changes in liver enzymes, suggesting normal doses are likely safe for humans. Human trials confirm that alpha lipoic acid is usually well-tolerated and may even offer some health benefits. This evidence suggests that taking alpha lipoic acid is relatively safe for most people. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for cystinuria?

Unlike the standard treatments for cystinuria, which mainly focus on increasing fluid intake and using medications to alkalinize urine or chelate cystine, the use of alpha lipoic acid (ALA) offers a novel approach. ALA is an antioxidant that might reduce stone formation by targeting oxidative stress, which is thought to play a role in stone development. Researchers are excited about this treatment because it not only has a different mechanism of action but also the potential to provide an oral supplement option that could simplify management and improve patient adherence. This could be a game-changer for those seeking an alternative to current strategies.

What evidence suggests that alpha lipoic acid might be an effective treatment for cystinuria?

Research has shown that alpha lipoic acid (ALA), which participants in this trial may receive, might help reduce cystine kidney stones. In one study, 40% of patients taking ALA experienced a decrease in stone size without needing additional treatment. ALA also makes cystine more dissolvable in urine, potentially preventing new stones from forming. In animal studies, ALA stopped existing stones from growing. These findings suggest that ALA might be a helpful option for managing cystine stones, especially when other treatments prove ineffective. ^{1 2 3 6 7}

Who Is on the Research Team?

Thomas Chi, M.D.

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

This trial is for people with cystinuria who've had kidney stones made of cystine. They must have documented evidence of the condition, be willing to consent, and not be pregnant or planning pregnancy during the study. It's not for those with uncontrolled diabetes, current alpha-lipoic acid use, non-English speakers, prisoners, or anyone seriously ill.

Inclusion Criteria

I have had cystine kidney stones in the past.

Being able and willing to provide consent

You have been diagnosed with cystinuria based on a urine test or analysis of kidney stones.

Exclusion Criteria

My diabetes has not been well-managed for over a year.

I have taken alpha-lipoic acid in the past year.

Pregnancy, lactation, or child-bearing age without birth control devices

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either 1200 mg alpha lipoic acid or placebo daily for three years

3 years

Routine clinical visit every 4 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Alpha lipoic acid
• Placebo

Trial Overview The trial tests if taking a daily dose of 1200 mg alpha lipoic acid can prevent cystine kidney stone recurrence over three years compared to a placebo (a pill without active medication). Participants are randomly assigned to one of these two groups.

How Is the Trial Designed?

Group I: ALA supplement Experimental Treatment 1 Intervention

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.

Group II: Placebo Placebo Group 1 Intervention

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.

Who Is Running the Clinical Trial?

University of California, San Francisco

Lead Sponsor

Trials

2,636

Recruited

19,080,000+

Thomas Chi, MD

Lead Sponsor

Trials

1

Recruited

50+

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Collaborator

Trials

2,513

Recruited

4,366,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5656064/

α-Lipoic acid treatment prevents cystine urolithiasis in a ... Further, α-LA was an effective suppressor of cystine stone growth, as Slc3a1-/- mice with existing stones demonstrated decreased stone growth ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02910531

NCT02910531 | Lipoic Acid Supplement for Cystine Stone This study evaluates how daily alpha lipoic acid supplementation affects cystine kidney stone recurrence. Half of the subjects will receive 1200 mg alpha ...

3. auajournals.org auajournals.org/doi/10.1097/01.JU.0001009408.66023.77.18

MP26-18 ALPHA LIPOIC ACID REDUCES STONE ... In a randomized clinical trial, 40% of patients who received ALA demonstrated a decrease in their overall stone burden. This occurred without ...

4. publications.aap.org publications.aap.org/pediatrics/article/145/5/e20192951/36811/Lipoic-Acid-ALA-Improves-Cystine-Solubility-in

α-Lipoic Acid (ALA) Improves Cystine Solubility in Cystinuria Our findings suggest that ALA improves solubility of cystine in urine and prevents stone formation in patients with cystinuria who do not respond to diet and ...

5. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT02910531

Lipoic Acid Supplement for Cystine Stone - UCSF Clinical Trials This study evaluates how daily alpha lipoic acid supplementation affects cystine kidney stone recurrence.

6. nad.com nad.com/news/an-overview-of-alpha-lipoic-acid-efficacy-safety-and-dosage

An Overview of α-Lipoic Acid: Efficacy, Safety, and Dosage Human trials suggest that α-lipoic acid is generally safe and may potentially help with weight management and improve some parameters related ...

7. cystinuria.org cystinuria.org/research/

Cystinuria Research This study evaluates how daily alpha lipoic acid supplementation affects cystine kidney stone recurrence. Half of the subjects will receive ...