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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests how a liquid form of levoleucovorin calcium affects young children with autism, focusing on improving language and other autism-related symptoms. The trial compares the effects of the treatment to a placebo, with participants receiving both over 26 weeks. Children aged 2.5 to 5 years with autism and language delays might be suitable candidates. Participants must maintain their current treatments throughout the study period. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group.

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.

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

Research has shown that levoleucovorin calcium has been studied for its safety in treating various conditions. In past studies, patients generally tolerated it well. For instance, improvements in neurological and behavioral symptoms have been noted with doses similar to those used in this trial (0.5-2 mg/kg/day).

Moreover, the FDA has approved levoleucovorin calcium for other uses, indicating a recognized safety profile. While this doesn't guarantee safety for everyone, it offers some reassurance about its general tolerability.

Overall, current data suggest that levoleucovorin calcium is safe, with no major side effects reported in related studies. However, as with any treatment, individual reactions can vary. Researchers will closely monitor participants during the trial to ensure their safety. ^{1 2 3 4 5}

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

Levoleucovorin calcium is unique because it offers a potential new approach for treating autism by providing a form of folate that can be more easily utilized by the body. Unlike many current treatments for autism, which often focus on behavioral therapies or medications targeting specific symptoms, levoleucovorin calcium aims to address underlying metabolic abnormalities that may contribute to the condition. Researchers are excited about this treatment because it targets a different aspect of autism's complex biology, offering hope for improved outcomes in areas like communication and social interaction.

What evidence suggests that levoleucovorin calcium might be an effective treatment for autism?

Research has shown that levoleucovorin calcium, which participants in this trial may receive, might improve language and behavior in children with autism spectrum disorder (ASD). Studies have found that it can enhance verbal communication, especially in children with specific folate (vitamin B9) processing issues. Some children with ASD who received this treatment experienced noticeable improvements in their symptoms. Although more research is needed, early findings suggest that levoleucovorin calcium could be promising for helping children with ASD, particularly those with language delays. ^{1 2 4 6 7}

Who Is on the Research Team?

Richard E Frye, MD, PhD

Principal Investigator

Rossignol Medical Center

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. fda.gov fda.gov/news-events/press-announcements/fda-takes-action-make-treatment-available-autism-symptoms

FDA Takes Action to Make a Treatment Available for ... The U.S. Food and Drug Administration today initiated the approval of leucovorin calcium tablets for patients with cerebral folate ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7477301/

Treatment of Folate Metabolism Abnormalities in Autism ... d,l-Leucovorin Improves Verbal Communication in Children With Autism Spectrum Disorder and Folate Receptor Alpha Autoantibodies. Given the high ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04060030

Study Details | NCT04060030 | Treatment of Social and ... The primary objective of this study is to evaluate the cognitive and behavioral effects of liquid leucovorin calcium on young children with autism spectrum ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39243316/

Efficacy of oral folinic acid supplementation in children with ... Oral folinic acid has shown potential to improve symptoms in children with autism spectrum disorder (ASD). However, randomized controlled trials ...

5. withpower.com withpower.com/trial/phase-3-disease-8-2020-9b108

Levoleucovorin Calcium for Autism Leucovorin calcium has shown potential benefits in improving verbal communication and behavior in children with autism spectrum disorder (ASD) who test positive ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04060017

NCT04060017 | Early Treatment of Language Impairment ... The primary aim of this study is to evaluate the effect of a liquid form of leucovorin calcium on language impairments in very young children with ASD.

7. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/540375

Treatment of Social and Language Deficits With ... The primary objective of this study is to evaluate the cognitive and behavioral effects of liquid leucovorin calcium on young children with autism spectrum ...