Could a vitamin help kids with autism talk and connect?
NCT ID NCT04060030
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing whether a liquid form of leucovorin (a type of folate) can improve social communication and language in young children with autism. Researchers will enroll 80 children ages 2.5 to 5 years who have a specific antibody linked to folate transport. The study lasts about 26 weeks and measures changes in social withdrawal and language delays.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 80 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Oct 2020
- Expected to finish
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Jul 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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30 to 60 months
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. 1\. Autism Spectrum Disorder (diagnosed as Autistic Disorder on the ADOS-2 or the ADI-R). 2. Between 2 years 6 months and 5 years 2 months of age at baseline 3. Folate Receptor Alpha Autoantibody Positive status 4. Language impairment (Ages and Stage Questionnaire between -1 and -3 SD for Language) 5. English included in the languages in which the child is being raised 6. Autism severity of moderate or higher (≥4) under the 7-item clinical global impression-severity scale. Moderate level of autism severity (4) is defined by the diagnosis of ASD with language impairment, so fulfilling #1 and #4 fulfills this requirement. 7. Ability to maintain all ongoing complementary, dietary, traditional, and behavioral treatments constant for the study period 8. Unchanged complementary, dietary, traditional, and behavioral treatments for two months prior to study entry 9. Has at least 4 month old expressive language ability as assessed by the MSEL Expressive Language Scale (i.e., Parent answers "yes" to " Voluntary babbling (such as 'bu, bu, bu")" Question #7 on the MSEL Expressive Language Scale. 10. Ability to attend to social stimulus and tolerate imaging procedures, as determined at the discretion of the investigator Exclusion Criteria: 1. Known FRAA status by clinically validated test performed outside of research studies. 2. Mineral or vitamin supplementation that exceeds the Tolerable Upper Daily Intake Levels set by the Institute of Medicine (See Table 6 below) 3. Significant self-abusive or violent behavior or evidence of suicidal ideation, plan or behavior 4. Severely affected children as defined by CGI-Severity Standard Score = 7 (Extremely Ill) 5. Severe prematurity (\<34 weeks gestation) as determined by medical history 6. Current uncontrolled gastroesophageal reflux 7. Current or history of liver or kidney disease as determined by medical history and safety labs 8. Genetic syndromes 9. Congenital brain malformations 10. Epilepsy 11. Any medical condition that the PI determines could jeopardize the safety of the study subject or compromise the integrity of the data 12. Significant negative reaction (i.e. fainting, vomiting, etc.) as a result of a previous blood draw. 13. Failure to thrive or Body Mass Index \< 5%ile or \<5%ile for weight (male \<11.2kg; female \<10.8kg by CDC 2000 growth charts) at the time of the study. 14. Concurrent treatment with drug that would significantly interact with l-leucovorin such as specific chemotherapy agents, antimalarial and immune suppressive agents and select antibiotics (See Table 7 below). 15. Allergy or Sensitivity to ingredients in the investigational product or placebo 16. Evaluation with the MSEL or BOSCC within 3 months of entering the study 17. Planned evaluation with the MSEL or BOSCC during the study 18. Exclusion Criteria for the MEG recording include: 1. Ferromagnetic implants, artificial joints, fixation hardware, dental work or shrapnel (additional screening will be completed to determine MRI eligibility) 2. Ferromagnetic products attached to the body (including hair extensions) 3. Head circumference greater than 60 cm 4. A weight greater than 407 lbs. (185 kg)
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
2 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Southwestern Research & Resource Center
NOT_YET_RECRUITINGPhoenix, Arizona, 85016, United States
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State University of New York, Downstate
RECRUITINGBrooklyn, New York, 11203, United States
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