80 Participants Needed

Leucovorin for Autism

Recruiting at 2 trial locations
SM
RE
N
SC
Overseen BySophia Crisler
Age: < 18
Sex: Any
Trial Phase: Phase 2
Sponsor: Phoenix Children's Hospital
Prior Safety DataThis treatment has passed at least one previous human trial

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.

Will I have to stop taking my current medications?

The trial requires that you keep your current treatments, including medications, the same for the study period and that they have not changed for two months before starting the study. However, you cannot take certain drugs that interact with leucovorin, like some chemotherapy agents, antimalarial drugs, immune suppressants, and specific antibiotics.

Is leucovorin safe for use in humans?

Leucovorin has been used safely in combination with other drugs for treating cancer, with common side effects including diarrhea, mouth sores, and nausea. Lower doses of leucovorin tend to have fewer side effects compared to higher doses.12345

How is the drug Levoleucovorin Calcium used in the treatment of autism different from other treatments?

Levoleucovorin Calcium is unique in autism treatment as it is a form of folinic acid, which may help improve symptoms by supporting metabolic processes in the brain, unlike other treatments that often focus on behavioral therapy or medications targeting specific symptoms.678910

Research Team

RE

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
See 7 more

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.
See 8 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Treatment Phase 1

Participants receive either placebo or active treatment for 12 weeks under double-blind conditions

12 weeks
Regular visits for monitoring and assessments

Treatment Phase 2

All participants receive active treatment for an additional 12 weeks

12 weeks
Regular visits for monitoring and assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks
2 visits (in-person)

Treatment Details

Interventions

  • Levoleucovorin Calcium
  • Placebo
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
2Treatment groups
Experimental Treatment
Placebo Group
Group I: L-leucovorin calciumExperimental Treatment1 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: PlaceboPlacebo Group1 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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Phoenix Children's Hospital

Lead Sponsor

Trials
78
Recruited
5,014,000+

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+

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+

Autism Speaks

Collaborator

Trials
36
Recruited
3,800+

Findings from Research

A systematic review of 13 studies found that reducing the dose of leucovorin in chemotherapy regimens combining it with fluorouracil does not significantly impact the efficacy of treatment for colorectal cancer.
Lower doses of leucovorin also do not increase the frequency or severity of adverse effects, and in some cases, higher doses were associated with more side effects, indicating that dose adjustments may be a safe alternative during drug shortages.
High vs. low-dose leucovorin in regimens with fluorouracil in colorectal cancer therapy.Juล™ica, J., ล uverovรก, P., Gonฤ›c, R., et al.[2023]

References

Bioequivalence of two leucovorin calcium tablet formulations. [2019]
2.Czech Republicpubmed.ncbi.nlm.nih.gov
High vs. low-dose leucovorin in regimens with fluorouracil in colorectal cancer therapy. [2023]
Pharmacokinetic and pharmacodynamic comparison of two doses of calcium folinate combined with continuous fluorouracil infusion in patients with advanced colorectal cancer. [2019]
Folate levels measured by LC-MS/MS in patients with colorectal cancer treated with different leucovorin dosages. [2021]
Phase I trial of a 5-day infusion of L-leucovorin plus daily bolus 5-fluorouracil in patients with advanced gastrointestinal malignancies. [2019]
Effects of menatetrenone on the decrease in calcium balance induced by vitamin K-deficient diet and sodium loading in rats. [2019]
Vitamin K(2) (menaquinone 4) reduces serum undercarboxylated osteocalcin level as early as 2 weeks in elderly women with established osteoporosis. [2013]
Bioavailability and Chemical/Functional Aspects of Synthetic MK-7 vs Fermentation-Derived MK-7 in Randomised Controlled Trials. [2019]
[Studies on transfer of vitamin K into human breast milk]. [2012]
The possibility of the native alcoholic drugs as etiological factor of the acquired prothrombin complex deficiency syndrome. [2013]
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