← Back to Search

Other

Folinic Acid for Language Impairment in Autism Spectrum Disorder

Phase 2
Waitlist Available
Led By Richard 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
Weight ≥ 15 kg
Stable medication (no changes in past 6 weeks and no planned changes for the next 6 months (duration of the study)
Must not have
Significant medical condition by history or by physical examination or lab tests that would be incompatible with the study drug
Presence of serious behavioral problems (tantrums, aggression, self-injury) for which another treatment is warranted
Timeline
Screening 3 weeks
Treatment Varies
Follow Up pre-treatment, 12 weeks(optional), and 24weeks (or early termination)

Summary

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.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While the specific side effects are not listed here, as folinic acid is generally used during cancer treatment to reduce side effects from chemotherapy, it may be well-tolerated. However, potential risks will likely be monitored given its investigational use.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I weigh at least 15 kilograms.
Select...
My medications have been the same for the last 6 weeks and won't change during the study.
Select...
I have been in a stable speech therapy program for at least a month with no changes expected for the next 12 weeks.
Select...
I am between 5 and 17 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I do not have any health conditions that would make it unsafe for me to take the study drug.
Select...
I have serious behavioral issues that need treatment.
Select...
My child is on medication for seizures.
Select...
My child takes more vitamins or minerals than the daily amount recommended.
Select...
I am not taking valproic acid, its derivatives, or lamotrigine.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~pre-treatment, 12 weeks(optional), and 24weeks (or early termination)
This trial's timeline: 3 weeks for screening, Varies for treatment, and pre-treatment, 12 weeks(optional), and 24weeks (or early termination) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in Clinical Evaluation of Language Fundamentals 4 (CELF-4) Score.
Change in Clinical Evaluation of Language Fundamentals Preschool (CELF-P) score
Secondary study objectives
Change in Clinician Global Impression for Improvement (CGI-I) Score
Other study objectives
Change in Aberrant Behavior Checklist (ABC) Score
Change in folate gene expression
Change in in methylation
+3 more

Side effects data

From 2016 Phase 3 trial • 1072 Patients • NCT01183780
60%
Diarrhoea
50%
Nausea
48%
Fatigue
38%
Decreased appetite
35%
Neutropenia
34%
Epistaxis
31%
Stomatitis
30%
Constipation
29%
Alopecia
29%
Vomiting
28%
Hypertension
26%
Neutrophil count decreased
23%
Abdominal pain
22%
Oedema peripheral
18%
Anaemia
18%
Proteinuria
17%
Mucosal inflammation
16%
Pyrexia
15%
Thrombocytopenia
15%
Headache
15%
Platelet count decreased
14%
Asthenia
14%
Weight decreased
13%
Cough
13%
Palmar-plantar erythrodysaesthesia syndrome
11%
Dyspnoea
9%
White blood cell count decreased
9%
Insomnia
8%
Rash
8%
Back pain
8%
Malaise
8%
Dysgeusia
7%
Blood alkaline phosphatase increased
7%
Hypoalbuminaemia
7%
Hypokalaemia
7%
Upper respiratory tract infection
7%
Pain in extremity
7%
Dizziness
6%
Hiccups
6%
Arthralgia
6%
Dehydration
6%
Aspartate aminotransferase increased
6%
Dysphonia
6%
Urinary tract infection
6%
Peripheral sensory neuropathy
5%
Dry skin
5%
Alanine aminotransferase increased
5%
Dyspepsia
5%
Haemorrhoids
5%
Proctalgia
4%
Abdominal distension
3%
Febrile neutropenia
2%
Intestinal obstruction
2%
Pulmonary embolism
1%
Sepsis
1%
International normalised ratio increased
1%
Nephrotic syndrome
1%
Fall
1%
Renal failure acute
1%
Haematemesis
1%
Ileus
1%
Small intestinal obstruction
1%
Blood bilirubin increased
1%
Pneumonia
1%
Large intestinal obstruction
1%
Large intestine perforation
1%
Venous thrombosis
1%
Clostridium difficile colitis
1%
Gastritis
1%
Intestinal perforation
1%
General physical health deterioration
100%
80%
60%
40%
20%
0%
Study treatment Arm
FOLFIRI + Ramucirumab
FOLFIRI + Placebo

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Folinic AcidExperimental Treatment1 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 ControlPlacebo Group1 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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Folinic Acid
2015
Completed Phase 3
~4380

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Autism Spectrum Disorder (ASD) include pharmacological interventions like folinic acid, which is being studied for its potential to improve language problems in children with ASD. Folinic acid, also known as leucovorin, is believed to enhance neurotransmitter function and support metabolic processes that are often disrupted in ASD. Other treatments, such as melatonin, are used to address sleep disturbances by regulating the sleep-wake cycle. Understanding these mechanisms is crucial as it helps tailor treatments to target specific symptoms, potentially improving the quality of life for ASD patients by addressing core deficits and associated challenges.
Folic acid as an adjunct in the treatment of children with the autism fragile-X syndrome (AFRAX).Pharmacological and dietary-supplement treatments for autism spectrum disorder: a systematic review and network meta-analysis.Folinic Acid as Adjunctive Therapy in Treatment of Inappropriate Speech in Children with Autism: A Double-Blind and Placebo-Controlled Randomized Trial.

Find a Location

Who is running the clinical trial?

Phoenix Children's HospitalLead Sponsor
76 Previous Clinical Trials
5,013,736 Total Patients Enrolled
5 Trials studying Autism Spectrum Disorder
686 Patients Enrolled for Autism Spectrum Disorder
AprofolUNKNOWN
State University of New York - Downstate Medical CenterOTHER
66 Previous Clinical Trials
12,101 Total Patients Enrolled
3 Trials studying Autism Spectrum Disorder
660 Patients Enrolled for Autism Spectrum Disorder
Southwest Autism Research & Resource CenterLead Sponsor
8 Previous Clinical Trials
896 Total Patients Enrolled
8 Trials studying Autism Spectrum Disorder
896 Patients Enrolled for Autism Spectrum Disorder
Rossignol Medical CenterLead Sponsor
5 Previous Clinical Trials
721 Total Patients Enrolled
5 Trials studying Autism Spectrum Disorder
721 Patients Enrolled for Autism Spectrum Disorder
Emory UniversityOTHER
1,704 Previous Clinical Trials
2,607,242 Total Patients Enrolled
23 Trials studying Autism Spectrum Disorder
21,531 Patients Enrolled for Autism Spectrum Disorder
Harvard UniversityOTHER
233 Previous Clinical Trials
473,875 Total Patients Enrolled
1 Trials studying Autism Spectrum Disorder
61 Patients Enrolled for Autism Spectrum Disorder
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NIH
2,061 Previous Clinical Trials
2,745,092 Total Patients Enrolled
37 Trials studying Autism Spectrum Disorder
10,219 Patients Enrolled for Autism Spectrum Disorder
Richard Frye, MD, PhDPrincipal InvestigatorPhoenix Children's Hospital
Richard E Frye, MD, PhDPrincipal InvestigatorRossignol Medical Center
5 Previous Clinical Trials
686 Total Patients Enrolled
5 Trials studying Autism Spectrum Disorder
686 Patients Enrolled for Autism Spectrum Disorder

Media Library

Folinic Acid (Other) Clinical Trial Eligibility Overview. Trial Name: NCT02839915 — Phase 2
Autism Spectrum Disorder Research Study Groups: Folinic Acid, Placebo Control
Autism Spectrum Disorder Clinical Trial 2023: Folinic Acid Highlights & Side Effects. Trial Name: NCT02839915 — Phase 2
Folinic Acid (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02839915 — Phase 2
~10 spots leftby Dec 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top