Leucovorin for Autism

Recruiting at2 trial locations

Overseen by Richard E Frye, MD, PhD

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Phoenix Children's Hospital

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Research Team

Richard E Frye, MD, PhD

Principal Investigator

Rossignol Medical Center, Phoenix AZ

Eligibility Criteria

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.

Inclusion Criteria

Folate Receptor Alpha Autoantibody Positive status

Ability to attend to social stimulus and tolerate imaging procedures, as determined at the discretion of the investigator

English included in the languages in which the child is being raised

Exclusion Criteria

You have a specific genetic condition or syndrome.

You have a history of hurting yourself or others, or show signs of thinking about or planning to harm yourself.

You have epilepsy, a condition that causes seizures.

Treatment Details

Interventions

• Levoleucovorin Calcium (Other)
• Placebo (Other)

Trial Overview 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.

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+

Robert L. Meyer

Phoenix Children's Hospital

Chief Executive Officer since 2003

Bachelor of Business Administration from the University of Michigan

Blake Bulloch

Phoenix Children's Hospital

Chief Medical Officer since 2024

MD, MBA

Southwest Autism Research & Resource Center

Lead Sponsor

Trials

9

Recruited

950+

Rossignol Medical Center

Lead Sponsor

Trials

6

Recruited

780+

University of Arizona

Collaborator

Trials

545

Recruited

161,000+

Dr. Richard Carmona

University of Arizona

Chief Medical Officer since 2021

MD, University of Arizona

Dr. Evan Unger

University of Arizona

Chief Executive Officer since 2021

PhD in Medical Imaging, University of Arizona

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+

Patricia A. Winston

State University of New York - Downstate Medical Center

Interim Chief Executive Officer

DNP, MS, RN, NEA-BC, FACHE

Mohamed (Rami) Nakeshbandi

State University of New York - Downstate Medical Center

Chief Medical Officer

MD

Autism Speaks

Collaborator

Trials

36

Recruited

3,800+