Early treatment of language impairment in young children with autism using leucovorin calcium

Early Treatment of Language Impairment in Young Children With Autism Spectrum Disorder With Leucovorin Calcium

This study is testing if a liquid treatment called leucovorin calcium can help young children with autism and language difficulties improve their language skills and overall symptoms.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 80 (estimated)
• Ages: 30 Months to 60 Months
• Sex: All
• Sponsor: Southwest Autism Research & Resource Center (other)
• Drugs / interventions: chemotherapy
• Locations: 2 sites (Phoenix, Arizona and 1 other locations)
• Trial ID: NCT04060017 on ClinicalTrials.gov

What this trial studies

This study evaluates the effects of liquid leucovorin calcium on cognitive and behavioral symptoms in young children diagnosed with autism spectrum disorder (ASD) who also have language impairments. The trial will enroll 80 children aged 2.5 to 5 years across two sites, assessing whether leucovorin calcium can improve language skills and core symptoms of ASD over a 26-week period. The study aims to determine the safety and efficacy of this treatment compared to a placebo, while also exploring potential biomarkers that may predict treatment response.

Who should consider this trial

Good fit: Ideal candidates are children aged 2.5 to 5 years with a confirmed diagnosis of autism spectrum disorder and language impairments.

Not a fit: Patients without language impairments or those with mild autism severity may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly enhance language development and reduce core symptoms in young children with autism.

How similar studies have performed: While there is limited research on leucovorin calcium for this specific purpose, the approach of targeting language impairments in ASD is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

1. Autism Spectrum Disorder (as defined below).
2. Between 2 years 6 months and 5 years 2 months of age at baseline
3. Language impairment (Ages and Stage Questionnaire between -1 and -3 SD for Language)
4. 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.
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.

6. Ability to maintain all ongoing complementary, dietary, traditional, and behavioral treatments constant for the study period 7. Unchanged complementary, dietary, traditional, and behavioral treatments for two months prior to study entry

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 5 below)
3. Significant self-abusive or violent behavior or evidence of suicidal ideation, plan or behavior
4. Severely affected 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.) because 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 screening.
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 6 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

Where this trial is running

Phoenix, Arizona and 1 other locations

• Southwestern Autism Research & Resource Center — Phoenix, Arizona, United States (NOT_YET_RECRUITING)
• State University of New York, Downstate — Brooklyn, New York, United States (RECRUITING)

Study contacts

• Principal investigator: Richard E Frye, MD, PhD — Rossignol Medical Center
• Study coordinator: Richard E Frye, MD, PhD

Conditions: Autism Spectrum Disorder, Language Disorders