Fluconazole for Autism

Not currently recruiting at 2 trial locations

Overseen By Scott Allen

Age: < 18

Sex: Any

Trial Phase: Academic

Sponsor: The Center for Autism and Related Disorders

Must not be taking: Terfenadine, Cisapride, Phenytoin, others

Disqualifiers: Allergy to fluconazole, Uncontrolled epilepsy, others

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how fluconazole, an antifungal medication, affects behavior in children with Autism Spectrum Disorder (ASD). Researchers aim to determine if fluconazole improves autism symptoms more effectively than a placebo (a pill with no active medication). The study includes two groups: one receiving fluconazole and the other a placebo, both for 30 days. Children aged 3 to 10 diagnosed with ASD who haven't used antifungals in the past 3 months qualify as candidates for this trial. As an unphased trial, this study offers participants a unique opportunity to contribute to groundbreaking research that could lead to new treatment options for ASD.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as terfenadine, cisapride, phenytoin, cyclosporine, rifampin, theophylline, astemizole, rifabutin, or tacrolimus. If you are on any of these, you would need to stop them to participate.

Is there any evidence suggesting that fluconazole is likely to be safe for children with ASD?

Research has shown that fluconazole is generally safe for children. Studies involving over 500 children, from newborns to 17-year-olds, found that most tolerate it well. Common mild side effects include diarrhea and nausea, occurring in less than 2% of cases. No major safety issues emerged with lower doses, particularly those under 200 mg. While minor side effects can occur, serious problems with fluconazole remain rare. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Most treatments for autism spectrum disorder focus on managing symptoms through behavioral therapies and medications like antipsychotics or stimulants. However, Fluconazole stands out because it is an antifungal medication, suggesting a novel approach by potentially addressing underlying biological factors related to gut health and microbiome imbalances. Researchers are excited about its potential to provide a new pathway for symptom management, especially for children who might not respond well to traditional therapies. This could open up a new avenue of treatment that targets different aspects of the disorder beyond the usual neurological focus.

What evidence suggests that fluconazole might be an effective treatment for ASD?

Research has shown that fluconazole, which participants in this trial may receive, effectively treats various fungal infections, including those affecting soft tissues and the lungs. Studies have found that fluconazole outperforms other similar treatments for these infections. It also helps prevent the recurrence of yeast infections, such as vulvovaginal candidiasis. However, limited information exists on fluconazole's effectiveness for Autism Spectrum Disorder (ASD) symptoms. The current hypothesis suggests that fluconazole might help with ASD symptoms due to its antifungal effects potentially influencing behavior. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

Inclusion Criteria

Male or female participants, three to ten years of age.

Meets clinical criteria for an autism spectrum disorder.

No antifungal use in the preceding 3 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either fluconazole or placebo daily for 30 days

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Fluconazole

How Is the Trial Designed?

Group I: Fluconazole Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Placebo capsule daily for 30 days

Fluconazole is already approved in European Union, United States, Canada, Japan, China for the following indications:

Approved in European Union as Diflucan for:

• Candidiasis
• Cryptococcosis
• Histoplasmosis
• Coccidioidomycosis

Approved in United States as Diflucan for:

• Vaginal candidiasis
• Oropharyngeal candidiasis
• Esophageal candidiasis
• Cryptococcal meningitis

Approved in Canada as Diflucan for:

• Vaginal candidiasis
• Oropharyngeal candidiasis
• Esophageal candidiasis
• Cryptococcal meningitis

Approved in Japan as Diflucan for:

• Candidiasis
• Cryptococcosis
• Histoplasmosis
• Coccidioidomycosis

Approved in China as Diflucan for:

• Vaginal candidiasis
• Oropharyngeal candidiasis
• Esophageal candidiasis
• Cryptococcal meningitis

Who Is Running the Clinical Trial?

The Center for Autism and Related Disorders

Lead Sponsor

Trials

2

Recruited

90+

Thoughtful House

Collaborator

Trials

2

Recruited

80+

The International Child Development Resource Center

Collaborator

Trials

3

Recruited

100+

Published Research Related to This Trial

The FDA's adverse drug reaction reporting system is designed to identify rare and unexpected reactions to medications, but the data collected is voluntary and spontaneous, leading to potential biases.

Using this spontaneous reporting data to calculate actual adverse reaction rates or to compare the safety of different drugs is inappropriate, as it does not accurately reflect the true incidence of these reactions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An evaluation of spontaneous adverse drug reaction monitoring systems. Sachs, RM., Bortnichak, EA.[2013]

The safety of marketed drugs is a significant concern, as some commonly prescribed medications can lead to serious or life-threatening side effects in patients.

The ChEMBL resource will provide a curated drug safety data set, including toxicity classifications and black box warnings, which will be freely available and regularly updated to aid in drug safety research and discovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug Safety Data Curation and Modeling in ChEMBL: Boxed Warnings and Withdrawn Drugs. Hunter, FMI., Bento, AP., Bosc, N., et al.[2023]

The study analyzed 345,662 spontaneous reports of adverse drug reactions (ADRs) submitted to the German Federal Institute for Drugs and Medical Devices, revealing a steady increase in reports since 1978, primarily due to stricter legal reporting requirements.

The most commonly reported ADRs were linked to drugs for nervous system disorders, with significant differences in reporting patterns between physicians and patients, suggesting a need for better patient education on potential ADRs.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Frequent Adverse Drug Reactions, and Medication Groups under Suspicion. Dubrall, D., Schmid, M., Alešik, E., et al.[2023]

A new computer-based system has been developed for managing reports of adverse drug reactions and serious adverse events, featuring a network of three regional centers and a central database for efficient data handling.

This system is designed to be secure, reliable, and cost-effective, making it a potential model for future clinical health information networks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Putting the pieces together: using "off-the-shelf" software to safely transfer medical data. Swoboda, WJ., Göttler, M., Zinnhobler, K., et al.[2017]

Citations

1. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK537158/

Fluconazole - StatPearls - NCBI Bookshelf Recent studies have proven that fluconazole is more effective at treating soft tissue and pulmonary infections than other azole antifungal ...

2. nejm.org nejm.org/doi/full/10.1056/NEJMoa033114

Maintenance Fluconazole Therapy for Recurrent ... Weekly treatment with fluconazole was effective in preventing symptomatic vulvovaginal candidiasis. The proportions of women who remained ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC2364408/

Comparative Study of the Effectiveness of Oral Fluconazole ... The rates of clinical effectiveness were 92% or 88% in the 6-day oral fluconzaole group, 80% or 76% in the single oral fluconazole group, and 72% or 58% in the ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S1876034111000463

Oral fluconazole 150 mg single dose versus intra-vaginal ... This study showed that single dose oral fluconazole is more effective than conventional topical clotrimazole for seven days in the treatment of acute VVC.

5. go.drugbank.com go.drugbank.com/drugs/DB00196

Fluconazole: Uses, Interactions, Mechanism of Action A 200-400 mg dose of fluconazole showed no clinically relevant effect on steroid levels or on ACTH-stimulated steroid response in healthy males, in one clinical ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC89397/

Safety and Tolerability of Fluconazole in Children - PMC The safety profile of fluconazole was assessed for 562 children (ages, 0 to 17 years) comprising 323 males and 239 females. The data are derived from 12 ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2024/019949s072lbl.pdf

DIFLUCAN® (Fluconazole Tablets) (Fluconazole for Oral ... The safety profile of DIFLUCAN has been studied in 577 pediatric patients from 1 day to. 17 years of age who received doses ranging from 1 to 15 mg/kg/day ...

8. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/10428919/

Safety and tolerability of fluconazole in children The safety profile of fluconazole was assessed for 562 children (ages, 0 to 17 years) comprising 323 males and 239 females. The data are derived from 12 ...

9. tandfonline.com tandfonline.com/doi/full/10.1080/14740338.2025.2490837?src=

Analysis of adverse events induced by fluconazole based ... As illustrated in Figure 2(a) , fluconazole demonstrates a relatively favorable safety profile at doses below 200 mg. However, a notable increase in severe ...

10. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1341321X15000768

Efficacy and safety of a single oral 150 mg dose ... The most common treatment-related adverse events were diarrhea and nausea (1.9% for each). No clinically significant safety issues were reported. A single oral ...