What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) include anti-epileptic medications, glutamatergic, GABAergic, and cholinergic agents, as well as supplements like folinic acid (a form of Vitamin B9). Anti-epileptic medications can have burdensome long-term side effects, including cognitive impairments and behavioral changes. Folinic acid has shown limited efficacy in reducing seizures and improving cognitive function, with no significant adverse effects reported. However, the overall evidence for the efficacy and safety of these treatments in ASD is limited, and side effects can vary depending on the specific medication or supplement used.

What prior FDA approvals exist?

The FDA has approved two medications for the treatment of irritability associated with Autism Spectrum Disorder: risperidone and aripiprazole. These antipsychotic drugs help manage symptoms such as aggression, self-injury, and severe tantrums. While Leucovorin Calcium (Folinic Acid) is being studied for its potential to improve language and core symptoms in young children with ASD, it is not yet FDA-approved for this use. Leucovorin Calcium is a form of Vitamin B9 that can cross the blood-brain barrier and may enhance neurological function, representing a different therapeutic approach compared to the currently approved antipsychotic medications.

What other types of research exist?

One promising alternative to Leucovorin Calcium for ASD treatment is DP-b99, a membrane-activated metal ion chelator with neuroprotective properties. DP-b99 has shown potential in improving neurological function by selectively chelating zinc and calcium ions within cell membranes, which could be beneficial for ASD patients.

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Levoleucovorin Calcium for Autism

Phase 2

Recruiting

Led By Richard E Frye, MD, PhD

Research Sponsored by Phoenix Children's Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Autism Spectrum Disorder between 2 years 6 months and 5 years 2 months of age at baseline

Be younger than 18 years old

Must not have

Mineral or vitamin supplementation that exceeds the Tolerable Upper Daily Intake Levels set by the Institute of Medicine

Genetic syndromes

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 1 year after study completion

Awards & highlights

Summary

This trial is testing a liquid form of leucovorin calcium to see if it can help young children with autism improve their language skills and behavior. The study involves young children who have autism and language delays. The treatment aims to enhance brain function and development. Leucovorin calcium has shown potential benefits in improving verbal communication and behavior in children with autism spectrum disorder (ASD) who test positive for cerebral folate receptor autoantibodies.

Who is the study for?

This trial is for young children aged between 2.5 and 5 years with Autism Spectrum Disorder (ASD) who have language delays or impairments. They must not have changed their treatments for two months before the study and should maintain them throughout the trial. Children with severe prematurity, certain medical conditions, violent behavior, or allergies to the study product cannot participate.

What is being tested?

The trial tests if liquid leucovorin calcium can improve cognitive abilities and behaviors related to ASD in children, focusing on language skills. It involves a comparison between leucovorin calcium and a placebo over approximately six months at two different sites.

What are the potential side effects?

While specific side effects are not listed here, potential risks may include allergic reactions to ingredients in leucovorin calcium or placebo. Parents will be informed about any known side effects prior to participation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My child is diagnosed with Autism Spectrum Disorder and is between 2.5 and 5.2 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not take more vitamins or minerals than recommended daily amounts.

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I have a diagnosed genetic syndrome.

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I have epilepsy.

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My FRAA status has been tested and confirmed outside of research.

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I am not taking any medications that would badly interact with l-leucovorin.

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My weight or BMI is in the lowest 5% for my age and gender.

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I have or had liver or kidney disease.

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I have severe heartburn that isn't controlled by medication.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 year after study completion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 year after study completion

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 year after study completion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Evaluate the change in measures of expressive language

Secondary study objectives

Evaluate change in autism severity of specific symptoms

Autistic Disorder

Evaluate the change in adaptive functioning

+9 more

Other study objectives

Combine presence of FRAA titers and single nucleotide polymorphisms (above) into a predicting equation that will evaluate the probability of positive response to L-leucovorin treatment.

Test if biomarkers (FRAAs) predict clinical response to L-leucovorin

Test if biomarkers (single nucleotide polymorphisms) predict clinical response to L-leucovorin

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: L-leucovorin calciumExperimental Treatment1 Intervention

The liquid form of leucovorin calcium will be dosed by weight, with a target dose of 1mg/kg/day, divided into two daily doses. This product may be taken alone or mixed with liquid. Participants randomized to this arm will receive active treatment for both 12-week phases of the study.

Group II: PlaceboPlacebo Group1 Intervention

The placebo will mimic the experimental treatment in flavor, odor, packaging, and dosing instructions. Participants randomized to this arm will receive placebo for the first 12 weeks of the study, then active treatment for the remaining 12 weeks.

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Leucovorin Calcium (Folinic Acid) is a form of Vitamin B9 that can cross the blood-brain barrier and potentially improve neurological function by addressing cerebral folate deficiency, which may be present in some children with Autism Spectrum Disorder (ASD). By supplementing with folinic acid, it is believed that the treatment can enhance verbal communication, receptive and expressive language, attention, and reduce stereotypical behaviors. This is important for ASD patients as it can significantly improve their quality of life and ability to function in daily activities.

Folic acid as an adjunct in the treatment of children with the autism fragile-X syndrome (AFRAX).Folinic Acid as Adjunctive Therapy in Treatment of Inappropriate Speech in Children with Autism: A Double-Blind and Placebo-Controlled Randomized Trial.Association of Maternal Prenatal Vitamin Use With Risk for Autism Spectrum Disorder Recurrence in Young Siblings.