Antioxidant Therapy for Cystic Fibrosis

(CF-AOX Trial)

Overseen By Jacob Looney, MS

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Augusta University

Must be taking: Traditional CF-treatment

Must not be taking: Vaso-active

Disqualifiers: Heart disease, PAH, Smoking, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how antioxidant treatments might improve exercise capacity and artery health in people with cystic fibrosis (CF). Researchers believe oxidative stress (damage caused by free radicals) may contribute to artery problems and hinder exercise in those with CF. Participants will receive either an acute antioxidant treatment, a chronic antioxidant treatment, or a placebo to assess the treatments' effectiveness. The trial seeks individuals with CF or healthy volunteers who have been clinically stable for at least two weeks and can undergo basic lung function tests. As an unphased trial, this study offers a unique opportunity to contribute to groundbreaking research that could enhance the quality of life for those with CF.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are on vaso-active medications (like nitrates, beta blockers, or ACE inhibitors).

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that antioxidant treatments, like those tested in this study, are usually easy for people to handle. For short-term antioxidant treatments, studies have found that vitamin C, vitamin E, and alpha-lipoic acid can support heart and blood vessel health and are safe for most people. These antioxidants, often found in supplements, combat damage caused by free radicals.

In long-term antioxidant treatment, similar ingredients are used over a longer period. Research indicates that using vitamin E and vitamin C for an extended period is safe for people with cystic fibrosis (CF). These antioxidants help boost the body's ability to fight oxidative stress, which is often lower in those with CF.

Mitoquinol, another component tested in both treatment groups, is a type of antioxidant designed to protect the energy-producing parts of cells. Studies have examined its potential to shield cells from damage, and it is generally considered safe.

Overall, these antioxidant treatments have a good safety record based on existing research. They are well-tolerated, and serious side effects are rare. However, as with any treatment, discussing participation with healthcare providers is important. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for cystic fibrosis, which mainly include antibiotics, bronchodilators, and mucus thinners, the antioxidant therapies being explored here focus on reducing oxidative stress in the body. These treatments are unique because they utilize a cocktail of antioxidants such as Vitamin C, Vitamin E, Alpha Lipoic Acid, Resveratrol, and Mitoquinol, which are believed to help improve lung function and exercise capacity. Researchers are excited about these options because they offer a novel approach by targeting the underlying oxidative damage in cystic fibrosis, potentially providing a new avenue for symptom management and improving the quality of life for patients.

What evidence suggests that this trial's antioxidant treatments could be effective for cystic fibrosis?

This trial evaluates the effects of different antioxidant treatments on people with cystic fibrosis (CF). Participants will join different treatment arms. One arm receives an acute antioxidant treatment, which includes a single dose of an antioxidant cocktail (vitamins C and E, and Alpha Lipoic Acid), Resveratrol, or Mitoquinol. Another arm receives a chronic antioxidant treatment, where the same antioxidants are taken daily over several weeks. Research suggests that these antioxidant treatments might improve blood vessel function and reduce damage from free radicals in CF patients. While these findings are encouraging, more research is needed to understand their impact on exercise ability and lung health in people with CF. ^{1 6 7 8 9}

Who Is on the Research Team?

Ryan Harris, Ph.D.

Principal Investigator

Augusta University

Are You a Good Fit for This Trial?

This trial is for men, women, and children over 7 with cystic fibrosis who can perform lung function tests and have an FEV1 percent predicted over 30%. They must not be currently smoking or pregnant, haven't had a feverish illness recently, no heart disease diagnosis, and not on certain heart medications.

Inclusion Criteria

I have been diagnosed with cystic fibrosis.

I am over 18 years old.

I have been medically stable without needing antibiotics for the past 2 weeks.

Exclusion Criteria

My child is 6 years old or younger.

Your lung function is very low, less than 30% of what's expected.

Currently smoking, pregnant, or nursing

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Acute Antioxidant Treatment

Participants receive a single dose of antioxidant treatment and undergo assessments for blood samples, flow-mediated dilation, lung function, and exercise capacity

1 day

2 visits (in-person)

Chronic Antioxidant Treatment

Participants receive daily antioxidant treatment and undergo assessments at baseline, 4 weeks, 8 weeks, and 12 weeks

12 weeks

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Acute Antioxidant
• Chronic Antioxidant
• Placebo

Trial Overview The study examines the effects of oxidative stress on blood vessel function and exercise capacity in CF patients. Participants will receive either acute antioxidants, chronic antioxidants or a placebo to determine if these treatments improve their condition.

How Is the Trial Designed?

Group I: Chronic Antioxidant Treatment Experimental Treatment 1 Intervention

Following the completion of Arm 1, blood samples, flow-mediated dilation, lung function, and exercise capacity (VO2 peak) will be performed, only in patients with CF, at baseline, 4 weeks, 8 weeks, and 12 weeks following one of the following: 1) an anti-oxidant cocktail (vitamin C 1000 mg, vitamin E 400 IU, and alpha lipoic acid 600 mg) taken once a day, 2) 1500 mg Resveratrol once a day or 3) 10 mg Mitoquinol once a day.

Group II: Acute Antioxidant Treatment Experimental Treatment 2 Interventions

Following an overnight fast, blood samples, flow-mediated dilation, lung function, and exercise capacity (VO2 peak) (post only) will be performed at baseline and 2 hours following either a single dose oral 1) antioxidant cocktail (1000 mg Vitamin C, 600 IU vitamin E, 600 mg Alpha Lipoic Acid) 2) Resveratrol (1500 mg) 3) Mitoquinol (10 mg) or placebo on two days separated by at least 72 hours.

Who Is Running the Clinical Trial?

Augusta University

Lead Sponsor

Trials

219

Recruited

85,900+

Published Research Related to This Trial

This study compiled respiratory adverse event (AE) rates from placebo subjects in inhaled therapy trials for cystic fibrosis, revealing short-term AE rates of 1.1 events per person-week in pediatric and 1.0 in adult subjects, and long-term rates of 1.7 events per person-month in pediatric and 2.2 in adult subjects.

The research identified that factors like forced expiratory volume (FEV1) and age in short-term studies, and FEV1 and gender in long-term studies, were statistically significant in predicting respiratory AE rates, although the models had low predictive power.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Respiratory adverse event profiles in cystic fibrosis placebo subjects in short- and long-term inhaled therapy trials. Sucharew, H., Goss, CH., Millard, SP., et al.[2007]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC2696206/

Antioxidants in cystic fibrosis Conclusions from the CF ... No prospective trials have demonstrated an effect on pulmonary outcomes of vitamin E supplementation in individuals with CF. The level of lipid peroxidation as ...

2. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1155/2019/1629638

Endothelial Dysfunction in Cystic Fibrosis: Role of ... These data demonstrate that ingestion of an antioxidant cocktail can improve vascular endothelial function and improve oxidative stress in ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7264436/

Circulating biomarkers of antioxidant status and oxidative ... This systematic review and meta-analysis found good evidence for reduced antioxidant capacity and elevated oxidative stress in people with clinically-stable CF.

4. pdfs.semanticscholar.org pdfs.semanticscholar.org/3d33/65e534f60c6ac649e5b325d887fa83246a22.pdf

Clinical Study Endothelial Dysfunction in Cystic Fibrosis Therefore, this study sought to test the hypoth- esis that a single dose of an antioxidant cocktail would reduce oxidative stress and improve ...

5. cfdb.eu cfdb.eu/en/topics/detail/code/042

Topic :: A, E, K vitamins supplementation A free access tool to evaluate the current evidences about clinical effectiveness of interventions in cystic fibrosis.

6. sciencedirect.com sciencedirect.com/science/article/pii/S2213231719313679

Circulating biomarkers of antioxidant status and oxidative ... This systematic review and meta-analysis found good evidence for reduced antioxidant capacity and elevated oxidative stress in people with clinically-stable CF.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12073493/

Advances in α-Lipoic Acid for Disease Prevention This review provides a comprehensive overview of the biochemical properties of ALA, including its direct free-radical-scavenging activity, regeneration of ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S2213231719315447

Antioxidant supplements and endurance exercise ROS-mediated vascular function may explain why acute antioxidant treatment (vitamin C, 1000 mg; vitamin E, 600 IU; alpha-lipoic acid, 600 mg) in healthy ...

9. journals.lww.com journals.lww.com/mtmj/fulltext/2022/21010/hepatoprotective_effects_of_alpha_lipoic_acid,.8.aspx

Hepatoprotective Effects of Alpha-Lipoic Acid, Vitamin C... The aim of the current study: To evaluate the effect of ALA and Vitamin C single or concurrent use of them on MTX-induced liver injury.