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Leucovorin 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
Folate Receptor Alpha Autoantibody Positive status
Ability to attend to social stimulus and tolerate imaging procedures, as determined at the discretion of the investigator
Timeline
Screening 3 weeks
Treatment Varies
Follow Up screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 16, week 20, week 24
Awards & highlights
Approved for 5 Other Conditions

Summary

This trial tests a liquid form of leucovorin calcium in young children with autism spectrum disorder (ASD) to see if it improves their social communication and behavior. The study focuses on children who have significant social and communication challenges. The treatment aims to enhance brain function, potentially leading to better communication and interaction skills.

Who is the study for?
This trial is for young children aged 2.5 to 5 years with confirmed autism spectrum disorder (ASD) who have social and communication delays, can attend to social stimuli, tolerate imaging procedures, and are being raised with English included in their languages. They must not change any ongoing treatments during the study and should have a moderate or higher level of autism severity.
What is being tested?
The trial is testing liquid leucovorin calcium against a placebo to see if it improves social communication and other symptoms of ASD in children. The study will enroll 80 participants across two sites for about half a year, from screening to end of treatment.
What are the potential side effects?
While specific side effects are not listed here, potential risks may include allergic reactions or sensitivities to ingredients in leucovorin calcium or the placebo. Parents should monitor for any unexpected health changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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You haven't made any changes to your complementary, dietary, traditional, or behavioral treatments for at least two months before starting the study.
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You have significant difficulty understanding or using language, as measured by a specific test.
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If you have autism, it needs to be moderate or severe based on a scale that measures how it affects your daily life.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 16, week 20, week 24
This trial's timeline: 3 weeks for screening, Varies for treatment, and screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 16, week 20, week 24 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 social communication symptoms
Secondary study objectives
Evaluate adverse effect symptoms in young children with ASD
Autistic Disorder
Evaluate change in specific autism symptom severity
+10 more
Other study objectives
Demonstrate changes in neuronal activation and connectivity associated with treatment response using non-invasive neuroimaging methods
Evaluate the effect of L-leucovorin on cellular regulatory pathways known to be implicated in social communication
Test if biomarkers (single nucleotide polymorphisms) predict clinical response to L-leucovorin

Awards & Highlights

Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.

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.

Find a Location

Who is running the clinical trial?

Phoenix Children's HospitalLead Sponsor
76 Previous Clinical Trials
5,013,712 Total Patients Enrolled
5 Trials studying Autism Spectrum Disorder
662 Patients Enrolled for Autism Spectrum Disorder
University of ArizonaOTHER
539 Previous Clinical Trials
161,095 Total Patients Enrolled
2 Trials studying Autism Spectrum Disorder
98 Patients Enrolled for Autism Spectrum Disorder
New York State Institute for Basic ResearchOTHER_GOV
6 Previous Clinical Trials
884 Total Patients Enrolled
4 Trials studying Autism Spectrum Disorder
185 Patients Enrolled for Autism Spectrum Disorder
State University of New York - Downstate Medical CenterOTHER
66 Previous Clinical Trials
12,067 Total Patients Enrolled
3 Trials studying Autism Spectrum Disorder
636 Patients Enrolled for Autism Spectrum Disorder
Autism SpeaksOTHER
35 Previous Clinical Trials
3,748 Total Patients Enrolled
19 Trials studying Autism Spectrum Disorder
2,321 Patients Enrolled for Autism Spectrum Disorder
Southwest Autism Research & Resource CenterLead Sponsor
8 Previous Clinical Trials
872 Total Patients Enrolled
8 Trials studying Autism Spectrum Disorder
872 Patients Enrolled for Autism Spectrum Disorder
Rossignol Medical CenterLead Sponsor
5 Previous Clinical Trials
697 Total Patients Enrolled
5 Trials studying Autism Spectrum Disorder
697 Patients Enrolled for Autism Spectrum Disorder
Richard E Frye, MD, PhdPrincipal InvestigatorPhoenix Children's Hospital
Richard E Frye, MD, PhDPrincipal InvestigatorRossignol Medical Center, Phoenix AZ
5 Previous Clinical Trials
662 Total Patients Enrolled
5 Trials studying Autism Spectrum Disorder
662 Patients Enrolled for Autism Spectrum Disorder

Media Library

Levoleucovorin Calcium (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04060030 — Phase 2
Autism Spectrum Disorder Research Study Groups: Placebo, L-leucovorin calcium
Autism Spectrum Disorder Clinical Trial 2023: Levoleucovorin Calcium Highlights & Side Effects. Trial Name: NCT04060030 — Phase 2
Levoleucovorin Calcium (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04060030 — Phase 2
~0 spots leftby Jan 2025