Levoleucovorin Calcium for Autism

Recruiting at 3 trial locations

Overseen By Neurology

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Phoenix Children's Hospital

Must not be taking: Chemotherapy, Antimalarial, Immunosuppressive, Antibiotics

Disqualifiers: Epilepsy, Genetic syndromes, Liver disease, others

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

Compensation: Varies

Number of Visits: 11

80 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that participants keep their current complementary, dietary, traditional, and behavioral treatments the same during the study and for two months before joining. However, if you are taking certain drugs that interact with l-leucovorin, you may need to stop those.

What data supports the effectiveness of the drug Levoleucovorin Calcium for Autism?

Research suggests that d,l-leucovorin calcium (a form of folinic acid) can improve certain symptoms in children with autism, especially those with specific folate metabolism issues. This drug may help bypass a blockage in folate transport to the brain, which is linked to autism symptoms. ^{1 2 3 4 5}

How does the drug Levoleucovorin Calcium differ from other treatments for autism?

Levoleucovorin Calcium is unique because it targets folate metabolism abnormalities in children with autism, particularly those with folate receptor autoantibodies, by bypassing the blockage in folate transport to the brain. This approach is different from other treatments as it addresses a specific underlying pathophysiological mechanism in autism. ^{1 3 5 6 7}

What is the purpose of this trial?

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.

Research Team

Richard E Frye, MD, PhD

Principal Investigator

Rossignol Medical Center

Eligibility Criteria

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.

Inclusion Criteria

English is one of the languages used in raising my child.

I can keep my current non-medical treatments the same during the study.

Your autism has a moderate or higher severity level based on a specific scale.

Exclusion Criteria

You were born very early, before 34 weeks of pregnancy.

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

You are very sick according to a standard rating scale.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment Phase 1

Participants receive either leucovorin calcium or placebo for 12 weeks under double-blind conditions

12 weeks

6 visits (in-person) at Weeks 2, 4, 6, 8, 10, 12

Treatment Phase 2

All participants receive active leucovorin calcium treatment for 12 weeks

12 weeks

4 visits (in-person) at Weeks 16, 20, 24

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Levoleucovorin Calcium
• Placebo

Trial Overview 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.

Participant Groups

Group I: L-leucovorin calcium Experimental Treatment 1 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: Placebo Placebo Group 1 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.

Who Is Running the Clinical Trial?

Phoenix Children's Hospital

Lead Sponsor

Trials

78

Recruited

5,014,000+

Rossignol Medical Center

Lead Sponsor

Trials

6

Recruited

780+

Southwest Autism Research & Resource Center

Lead Sponsor

Trials

9

Recruited

950+

University of Arizona

Collaborator

Trials

545

Recruited

161,000+

New York State Institute for Basic Research

Collaborator

Trials

7

Recruited

960+

State University of New York - Downstate Medical Center

Collaborator

Trials

67

Recruited

12,100+

United States Department of Defense

Collaborator

Trials

940

Recruited

339,000+

References

1. Korea (South) pubmed.ncbi.nlm.nih.gov

Effects of Vitamin D Supplementation on Children with Autism Spectrum Disorder: A Systematic Review and Meta-analysis. [2023]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Ratings of the Effectiveness of Nutraceuticals for Autism Spectrum Disorders: Results of a National Survey. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Treatment of Folate Metabolism Abnormalities in Autism Spectrum Disorder. [2021]

4. England pubmed.ncbi.nlm.nih.gov

The effect of vitamin D supplementation in treatment of children with autism spectrum disorder: a systematic review and meta-analysis of randomized controlled trials. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Lack of effect of vitamin D3 supplementation in autism: a 20-week, placebo-controlled RCT. [2018]

6. England pubmed.ncbi.nlm.nih.gov

Cerebral folate receptor autoantibodies in autism spectrum disorder. [2022]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Immunological Dysfunction in Autism Spectrum Disorder: A Potential Target for Therapy. [2019]