Folinic Acid for Language Impairment in Autism Spectrum Disorder

Recruiting at 5 trial locations

Overseen by Richard E Frye, MD, PhD

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Phoenix Children's Hospital

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing folinic acid, a vitamin, to see if it can help children with autism who have language problems. The study focuses on whether this vitamin can improve brain function and language skills. Children with moderate language issues will participate. Folinic acid has been studied for its potential to improve verbal communication in children with autism and language impairment.

Research Team

Richard E Frye, MD, PhD

Principal Investigator

Rossignol Medical Center

Eligibility Criteria

This trial is for children aged 5 to less than 17.5 years with Autism Spectrum Disorder and language impairment, who speak English at home and have an IQ of at least 40 or a mental age of at least 18 months. They must be on stable speech therapy and educational plans, not planning medication changes for the study's duration, and not taking certain medications that affect folate metabolism.

Inclusion Criteria

You have a low score on a language test.

I weigh at least 15 kilograms.

My educational or treatment plan will not change for the next 3 months.

Exclusion Criteria

I have a mental health condition like depression or bipolar disorder.

I do not have any health conditions that would make it unsafe for me to take the study drug.

My child has stopped taking Bactrim for 2 months.

Treatment Details

Interventions

• Folinic Acid (Other)
• Placebo (Other)

Trial Overview The trial tests if folinic acid (leucovorin) can improve language problems in autistic children compared to a placebo. Folinic acid is FDA-approved for reducing chemotherapy side effects but its effectiveness in autism-related language issues is being investigated here over a period of up to 24 weeks.

Participant Groups

Group I: Folinic Acid Experimental Treatment 1 Intervention

Subjects randomized to receive Folinic Acid will take Liquid levo-leucovorin via oral route. The target dose is 1 mg/kg/day with a maximum of 25 mg/day, divided in two daily doses. A two- to four-week supply of 15 ml vials will be dispensed in line with the visit schedule. With the exception of children in the lowest weight group (≥ 15 - \< 20 kg) from days 1-14, parents will administer the prescribed dose twice a day at the same time each day.

Group II: Placebo Control Placebo Group 1 Intervention

Subjects randomized to receive placebo will take placebo twice a day (Exception: children in the lowest weight group ( ≥ 15 - \< 20 kg) will start once a day for Days1-13). The pattern of dose escalation will be the same as the active compound. After 12 weeks, the blind will not be broken and subjects will be offered treatment for a 12-week open-label extension phase.

Folinic Acid is already approved in Canada, Japan for the following indications:

Approved in Canada as Calcium Folinate for:

• Colorectal cancer
• Methotrexate overdose
• Megaloblastic anemia

Approved in Japan as Calcium Folinate for:

• Colorectal cancer
• Methotrexate overdose
• Megaloblastic anemia

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+

Aprofol

Collaborator

Trials

1

Recruited

60+

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

Emory University

Collaborator

Trials

1,735

Recruited

2,605,000+

Dr. R. Donald Harvey

Emory University

Chief Medical Officer

MD from Emory University School of Medicine

Dr. George Painter

Emory University

Chief Executive Officer since 2013

PhD in Synthetic Organic Chemistry from Emory University

Harvard University

Collaborator

Trials

237

Recruited

588,000+

Dr. Christopher Anzalone

Harvard University

Chief Executive Officer since 2007

PhD in Biology from UCLA

Dr. Perry

Harvard University

Chief Medical Officer since 2011

MD from Albert Einstein College of Medicine, MPH from Harvard University

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborator

Trials

2,103

Recruited

2,760,000+

Dr. Diana W. Bianchi

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Chief Executive Officer since 2016

MD from Stanford University

Dr. Alison Cernich

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Chief Medical Officer since 2020

PhD in Clinical Psychology from University of Maryland