Actively Recruiting

Phase 2
Age: 30Months - 60Months
All Genders
NCT04060030

Treatment of Social and Language Deficits With Leucovorin for Young Children With Autism

Led by Southwest Autism Research & Resource Center · Updated on 2026-04-27

80

Participants Needed

2

Research Sites

303 weeks

Total Duration

On this page

Sponsors

S

Southwest Autism Research & Resource Center

Lead Sponsor

A

Autism Speaks

Collaborating Sponsor

AI-Summary

What this Trial Is About

The primary objective of this study is to evaluate the cognitive and behavioral effects of liquid leucovorin calcium on young children with autism spectrum disorder (ASD) and determine whether it improves social communication as well as the core and associated symptoms of ASD. The investigators will enroll 80 children across two sites, between the ages of 2.5 and 5 years, with confirmed ASD and known social and communication delays. Participation will last approximately 26 weeks, from screening visit to end of treatment.

CONDITIONS

Official Title

Treatment of Social and Language Deficits With Leucovorin for Young Children With Autism

Who Can Participate

Age: 30Months - 60Months
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosed with Autism Spectrum Disorder using ADOS-2 or ADI-R
  • Aged between 2 years 6 months and 5 years 2 months at baseline
  • Positive Folate Receptor Alpha Autoantibody status
  • Language impairment indicated by Ages and Stages Questionnaire between -1 and -3 standard deviations for language
  • Child is being raised with English as one of the languages
  • Autism severity is moderate or higher on the 7-item clinical global impression-severity scale
  • Ability to keep all ongoing complementary, dietary, traditional, and behavioral treatments unchanged during the study
  • Complementary, dietary, traditional, and behavioral treatments unchanged for two months before study entry
  • At least 4 months old expressive language ability as assessed by the MSEL Expressive Language Scale
  • Ability to attend to social stimulus and tolerate imaging procedures as judged by the investigator
Not Eligible

You will not qualify if you...

  • Known Folate Receptor Alpha Autoantibody status from clinical tests outside research
  • Mineral or vitamin supplementation exceeding Tolerable Upper Daily Intake Levels
  • Significant self-abusive or violent behavior or suicidal ideation, plan, or behavior
  • Severely affected children with CGI-Severity Score of 7 (Extremely Ill)
  • Severe prematurity before 34 weeks gestation
  • Current uncontrolled gastroesophageal reflux
  • Current or history of liver or kidney disease
  • Presence of genetic syndromes
  • Congenital brain malformations
  • Epilepsy
  • Any medical condition that could risk participant safety or data integrity as determined by the investigator
  • Significant negative reaction to previous blood draws (e.g., fainting, vomiting)
  • Failure to thrive or body mass index or weight below 5th percentile
  • Concurrent treatment with drugs that interact with l-leucovorin (e.g., specific chemotherapy, antimalarial, immune suppressive agents, select antibiotics)
  • Allergy or sensitivity to ingredients in the investigational product or placebo
  • Evaluation with MSEL or BOSCC within 3 months before the study
  • Planned evaluation with MSEL or BOSCC during the study
  • For MEG recording: ferromagnetic implants or products, head circumference over 60 cm, weight over 407 lbs (185 kg)

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

1
2
3
+1

Trial Site Locations

Total: 2 locations

1

Southwestern Research & Resource Center

Phoenix, Arizona, United States, 85016

Not Yet Recruiting

2

State University of New York, Downstate

Brooklyn, New York, United States, 11203

Actively Recruiting

Loading map...

Research Team

R

Richard E Frye, MD, PhD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here