Efficacy and Safety of Altibrain® as an Adjunctive Therapy for Autism Spectrum Disorder: An Open Label Trial Targeting Core Symptoms.

Objective: This study aimed to evaluate the effectiveness and safety of Altibrain^® in combination with standard Autism Spectrum Disorder (ASD) treatment compared to standard ASD treatment alone in individuals diagnosed with ASD.

Methods: A randomized, open-label trial was conducted involving 120 participants aged 3 to 17 years, randomly assigned to either the Standard ASD Treatment group or the Altibrain^® + Standard ASD Treatment group. Sixty patients were randomly allocated to each Standard ASD Treatment group or the Altibrain^® + Standard ASD Treatment group. Participant allocation was done by computer-generated randomization. Participants had confirmed ASD diagnoses based on DSM-V or ICD-11 criteria and demonstrated moderate to severe core ASD symptoms. Informed consent was obtained from caregivers. A total number of 120 subjects were included, consisting of 71 male and 49 female patients. Participants received either standard ASD treatment alone or Altibrain^® in addition to standard ASD treatment orally once daily for 24 weeks. A total of 7 study visits/24 weeks to analyze the intervention efficacy of the Standard ASD Treatment group or the Altibrain ^® + Standard ASD Treatment group. Primary outcomes included changes in core ASD symptoms measured by the Autism Diagnostic Observation Schedule (ADOS) and safety assessments. Secondary outcomes included alterations in social communication skills, reduction in repetitive behaviors, overall functional improvements, safety and tolerability of Altibrain^®.

Results: Altibrain^® significantly improved qualitative deficits in social interaction and repetitive behaviors compared to standard ASD treatment alone (p < 0.0001). The Altibrain^® + Standard ASD Treatment group demonstrated significant improvements in social functioning, social awareness, cognition, communication, and motivation compared to the Standard ASD Treatment group (p < 0.0001). Additionally, Altibrain^® showed superior efficacy in reducing hyperactivity/noncompliance, inappropriate speech, irritability, lethargy/ social withdrawal, stereotypic behavior, and aberrant behavior compared to standard treatment alone (p < 0.0001). Additionally, Altibrain^® exhibited a favorable safety profile as per the 4-week post-treatment safety follow-up.

Conclusion: Overall, Altibrain^® holds promise as a valuable therapeutic option for individuals with ASD and their families. Limitations of the study include neuroimaging and biomarkers analysis and larger cohort studies.