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Folinic Acid as a Treatment for Autism Spectrum Disorder in Children (FOLIN-A)

1 czerwca 2026 zaktualizowane przez: Wong Chui Mae, KK Women's and Children's Hospital

Folinic Acid as a Treatment for Autism Spectrum Disorder in Children: A Double-blind Randomised Placebo-controlled Trial With Open Label Extension (FOLIN-A Trial)

Autism spectrum disorder (ASD) is a neurodevelopmental condition that affects communication, behavior, and social interaction in children. Current treatments are limited and mainly focus on managing symptoms. This study aims to evaluate whether folinic acid is safe and effective in improving symptoms of autism spectrum disorder in children. Participants will be randomly assigned to receive either folinic acid or a placebo during the double-blind treatment phase. After this phase, all participants will have the option to receive folinic acid in an open-label extension. The results of this study may help determine whether folinic acid could be a potential treatment option for children with autism spectrum disorder.

Przegląd badań

Status

Rekrutacyjny

Warunki

Interwencja / Leczenie

Szczegółowy opis

This study is a double-blind, randomised, placebo-controlled clinical trial designed to evaluate the safety and effectiveness of folinic acid in children with autism spectrum disorder (ASD). Eligible participants will be randomly assigned to receive either 2 mg/kg/day of folinic acid or a matching placebo during a 12-week double-blind randomised controlled trial (RCT) treatment phase. Neither the participants, caregivers, nor study staff will know which treatment is assigned during this period. The primary aim is to assess whether folinic acid improves symptoms of autism spectrum disorder. Following completion of the double-blind phase, participants will enter a 12-week open-label extension, during which all participants will receive 2 mg/kg/day of folinic acid. This extension phase will allow for further evaluation of longer-term safety and potential benefits. The findings from this study may contribute to a better understanding of the role of folinic acid as a potential treatment option for children with autism spectrum disorder.

Typ studiów

Interwencyjne

Zapisy (Szacowany)

184

Faza

  • Faza 2

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Kontakt w sprawie studiów

Lokalizacje studiów

  • Singapur
    • Singapore
      • Singapore, Singapore, Singapur
        • Rekrutacyjny
        • KK Women's and Children's Hospital

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

  • Dziecko

Akceptuje zdrowych ochotników

Nie

Opis

Inclusion Criteria:

  • Children aged 3 years 0 months to 10 years 11 months at the time of enrolment
  • Weight ≥ 10 kg and ≤ 30 kg at the time of enrolment
  • Confirmed diagnosis of Autism Spectrum Disorder (ASD) based on Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition, Text Revision (DSM-IV-TR) or Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria, supported by standardized diagnostic assessments
  • Stable on regular behavioral interventions and any medications for at least 6 months prior to enrolment
  • At least one parent or legal guardian able to understand and complete study questionnaires in English
  • Written informed consent obtained from a parent or legal guardian

Exclusion Criteria:

  • Known allergy or intolerance to folinic acid
  • Current or prior supplementation with folinic acid or other folate preparations (folic acid supplementation permitted)
  • Vitamin B12 (cobalamin) deficiency or pernicious anemia
  • Concurrent use of medications known to interfere with folate metabolism (e.g., methotrexate, trimethoprim, fluorouracil, capecitabine, glucarpidase)
  • Known genetic, metabolic, or chromosomal disorder, or structural brain abnormalities
  • History of epilepsy or seizures (except simple febrile seizures) or current use of antiepileptic or antipsychotic medication
  • Significant sensory (hearing or visual) or motor impairment
  • Participation in another interventional clinical trial for autism within the past 6 months
  • Unable or unwilling to comply with study visits and procedures

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Randomizowane
  • Model interwencyjny: Przydział równoległy
  • Maskowanie: Poczwórny

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: Folinic Acid
Participants in this arm will receive folinic acid during the double-blind randomised controlled trial (RCT) phase of the study. After completion of the double-blind phase, participants will continue into an open-label extension in which folinic acid is provided.
Lek: Folinic Acid
Oral folinic acid administered during the double-blind randomised controlled trial (RCT) phase, followed by open-label folinic acid treatment.
Komparator placebo: Placebo
Participants in this arm will receive a matching placebo during the double-blind randomised controlled trial (RCT) phase of the study. After completion of the double-blind phase, participants will continue into an open-label extension in which folinic acid is provided.
Lek: Placebo
Matching oral placebo administered during the double-blind randomised controlled trial (RCT) phase.

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Change in Pervasive Developmental Disorder-Behavior Inventory (PDDBI) Autism Composite T-Score
Ramy czasowe: Baseline to Week 12
The Pervasive Developmental Disorder-Behavior Inventory (PDDBI) is a validated caregiver-completed instrument used to assess adaptive and maladaptive behaviors in individuals with autism spectrum disorder (ASD). The Autism Composite T-score is a standardized score (mean = 50, standard deviation = 10) that summarizes overall autism symptom severity, with higher scores indicating greater symptom severity. This outcome measure evaluates the change from baseline to Week 12 in PDDBI Autism Composite T-score. Lower T-scores indicate improvement in autism-related symptoms. Changes in scores will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Change from baseline in language function as measured by the Clinical Evaluation of Language Fundamentals (CELF)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in standardized language function scores as assessed using the Clinical Evaluation of Language Fundamentals (CELF), a standardized individually administered assessment of receptive and expressive language skills in children. CELF standardized scores have a mean of 100 and a standard deviation of 15, with higher scores indicating better language performance. Changes in scores will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12
Change from baseline in receptive vocabulary score as measured by the Receptive One-Word Picture Vocabulary Test (ROWPVT)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in standardized receptive vocabulary scores as assessed using the Receptive One-Word Picture Vocabulary Test (ROWPVT), a standardized individually administered assessment of a child's ability to understand spoken words. Scores are standardized with a mean of 100 and a standard deviation of 15, where higher scores indicate better receptive vocabulary ability. Changes in scores will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12
Change from baseline in expressive vocabulary score as measured by the Expressive One-Word Picture Vocabulary Test (EOWPVT)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in standardized expressive vocabulary scores as assessed using the Expressive One-Word Picture Vocabulary Test (EOWPVT), a standardized individually administered assessment of a child's ability to produce spoken words. Scores are standardized with a mean of 100 and a standard deviation of 15, where higher scores indicate better expressive vocabulary ability. Changes in scores will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12
Change from baseline in Clinical Global Impression-Severity and Improvement scores as measured by the Clinical Global Impression Scale (CGI-S and CGI-I)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) and Clinical Global Impression-Improvement (CGI-I) scores, assessed using the Clinical Global Impression Scale, a standardized assessor-rated measure of illness severity and overall clinical improvement. CGI-S scores range from 1 (normal, not at all ill) to 7 (among the most extremely ill), where lower scores indicate better outcomes, and CGI-I scores range from 1 (very much improved) to 7 (very much worse), where lower scores indicate greater improvement. Changes in scores will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12
Change from baseline in Aberrant Behavior Checklist total score as measured by the Aberrant Behavior Checklist (ABC)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in total scores on the Aberrant Behavior Checklist (ABC), a standardized caregiver-completed questionnaire that assesses behavioural symptoms in children across domains such as irritability, lethargy, stereotypy, hyperactivity, and inappropriate speech. Total scores range from 0 to 174, with higher scores indicating greater severity of behavioural problems (worse outcomes). Changes in scores will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12
Change from baseline in total problem score as measured by the Child Behavior Checklist (CBCL)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in total problem scores on the Child Behavior Checklist (CBCL), a standardized caregiver-reported questionnaire that assesses a broad range of emotional and behavioural problems in children. Total scores are standardised T-scores (typically ranging from approximately 30 to 100, with a mean of 50 and standard deviation of 10), where higher scores indicate greater behavioural and emotional problems (worse outcomes). Changes in scores will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12
Change from baseline in adaptive behavior composite score as measured by the Vineland Adaptive Behavior Scales (VABS)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in standardised adaptive behavior scores as assessed using the Vineland Adaptive Behavior Scales (VABS), a standardized caregiver-reported assessment of a child's adaptive functioning across domains such as communication, daily living skills, socialization, and motor skills. Adaptive behavior composite scores are standardised (typically ranging from approximately 20 to 160, with a mean of 100 and standard deviation of 15), where higher scores indicate better adaptive functioning (better outcomes). Changes in scores will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12
Change from baseline in proportion of gaze duration to social stimuli as measured by eye-tracking assessment
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in eye-tracking measures of visual attention, specifically the proportion of total gaze duration directed toward social stimuli (e.g., faces or biological motion) compared with non-social stimuli, assessed using a standardized eye-tracking paradigm. Higher proportions indicate greater relative attention to social stimuli. Changes in proportions will be compared between participants receiving folinic acid and those receiving placebo.
Baseline to Week 12
Incidence of adverse events and serious adverse events as measures of safety and tolerability of folinic acid
Ramy czasowe: Baseline to Week 24
Change from baseline to Week 24 in the incidence of adverse events (AEs) and serious adverse events (SAEs), monitored throughout the study period to assess the safety and tolerability of folinic acid treatment.
Baseline to Week 24

Inne miary wyników

Miara wyniku
Opis środka
Ramy czasowe
Change from baseline in plasma glutathione redox ratio (GSH:GSSG)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in plasma glutathione redox ratio (reduced glutathione:oxidized glutathione; GSH:GSSG), a biomarker of oxidative stress balance. Higher ratios indicate a more reduced (favorable) redox state. Plasma reduced glutathione (GSH, µM) and oxidized glutathione (GSSG, µM) will be measured at baseline (T1) and Week 12 (T2).
Baseline to Week 12
Change from baseline in plasma methylation ratio (S-adenosylmethionine:S-adenosylhomocysteine; SAM:SAH)
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in plasma methylation ratio (S-adenosylmethionine:S-adenosylhomocysteine; SAM:SAH), a biomarker of methylation capacity. Higher ratios indicate greater methylation potential (better outcomes). Additional biomarkers related to one-carbon metabolism, including plasma homocysteine (µmol/L), will measured where available.
Baseline to Week 12
Change in Plasma Ergothioneine Levels
Ramy czasowe: Baseline to Week 12
Change from baseline to Week 12 in plasma ergothioneine concentration (ET, nM). Plasma ergothioneine (ET, nM), hercynine (nM), and ophthalmic acid will be measured at baseline (T1) and Week 12 (T2).
Baseline to Week 12
Change in Folate Status Biomarkers
Ramy czasowe: Baseline to Week 12
Change from baseline in serum and plasma folate status biomarkers. Folate status biomarkers include measures related to one-carbon metabolism and amino acid pathways. Plasma amino acid concentrations (µmol/L) will be interpreted with reference to established age-specific clinical ranges.
Baseline to Week 12
Association of FRAA Status With Treatment Response
Ramy czasowe: Baseline to Week 12
Association between folate receptor autoantibody (FRAA) status and change in autism outcome measures. FRAA status will be classified as positive or negative based on the assay-specific cutoff defined by the testing laboratory: Blocking FRAA (pmol blocked per mL) and binding FRAA (optical density [OD] units per µL).
Baseline to Week 12
Sustained Effects of Folinic Acid on Autism Symptoms
Ramy czasowe: Baseline to Week 24
Change from baseline in autism outcome measures following 24 weeks of folinic acid treatment.
Baseline to Week 24

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

KK Women's and Children's Hospital

Współpracownicy

Nanyang Technological University
Singapore Health Services
National University of Singapore

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

19 maja 2025

Zakończenie podstawowe (Szacowany)

30 czerwca 2027

Ukończenie studiów (Szacowany)

30 czerwca 2027

Daty rejestracji na studia

Pierwszy przesłany

8 maja 2026

Pierwszy przesłany, który spełnia kryteria kontroli jakości

1 czerwca 2026

Pierwszy wysłany (Rzeczywisty)

5 czerwca 2026

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

5 czerwca 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

1 czerwca 2026

Ostatnia weryfikacja

1 maja 2026

Więcej informacji

Terminy związane z tym badaniem

Słowa kluczowe

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • FOLIN-A-01
  • MOH-CIRG24jan-0004 (Inny numer grantu/finansowania: National Medical Research Council (NMRC), Singapore)

Plan dla danych uczestnika indywidualnego (IPD)

Planujesz udostępniać dane poszczególnych uczestników (IPD)?

NIEZDECYDOWANY

Opis planu IPD

At this time, there is no confirmed plan to share individual participant data (IPD) collected in this study. The study team will consider requests for access to de-identified individual participant data, including the analyzable dataset and relevant supporting documentation, on a case-by-case basis following completion of the study. Any data sharing will be subject to applicable institutional review board (IRB) requirements, data protection regulations, and data sharing agreements to ensure participant confidentiality and appropriate use.

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Nie

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

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