AST-001 / Astrogen - LARVOL DELTA

miRNA-mediated therapeutic effects of AST-001 through modulation of key marker genes in autism spectrum disorder. (PubMed, Sci Rep) - "These findings suggest a potential mechanism by which AST-001 modulates miRNA-mediated regulation of genes involved in synaptic function and brain development, including DLG4, PAX5, and SNAP23. This post-transcriptional modulation highlights a plausible pathway to help rebalance disrupted dopaminergic and synaptic signaling networks, offering early mechanistic insights into the therapeutic potential of AST-001."

Journal • Autism Spectrum Disorder • Genetic Disorders • DLG4 • MIR128 • MIR483 • PAX5

Safety and efficacy of AST-001 in children with autism spectrum disorder: a 52-week multicenter long-term follow-up study. (PubMed, Front Psychiatry) - "Further large-scale, well-controlled studies are warranted. https://cris.nih.go.kr/cris/search/detailSearch.do?seq=26492&search_page=L&search_lang=&class_yn=, identifier KCT0007519."

Clinical • Journal • Autism Spectrum Disorder • Dental Disorders • Gastrointestinal Disorder • Genetic Disorders • Infectious Disease • Novel Coronavirus Disease • Otorhinolaryngology • Septic Shock

A Study to Evaluate the Efficacy and Safety of AST-001 in ASD Children (clinicaltrials.gov) - P3 | N=169 | Completed | Sponsor: Astrogen, Inc. | Active, not recruiting ➔ Completed

Trial completion • Autism Spectrum Disorder • Genetic Disorders

Correction to "AST-001 versus placebo for social communication in children with autism spectrum disorder: A randomized clinical trial". (PubMed, Psychiatry Clin Neurosci) - No abstract available

Clinical • Autism Spectrum Disorder • Genetic Disorders

A Study to Evaluate the Efficacy and Safety of AST-001 in ASD Children (clinicaltrials.gov) - P3 | N=169 | Active, not recruiting | Sponsor: Astrogen, Inc. | Trial completion date: Dec 2024 ➔ Jun 2025 | Recruiting ➔ Active, not recruiting

Enrollment closed • Trial completion date • Autism Spectrum Disorder • Genetic Disorders

Response to [Examining the feasibility and limitations of AST-001 as a treatment for autism spectrum disorder]. (PubMed, Psychiatry Clin Neurosci) - No abstract available

Journal • Autism Spectrum Disorder • Genetic Disorders

Population pharmacokinetic and pharmacodynamic model guided weight-tiered dose of AST-001 in pediatric patients with autism spectrum disorder. (PubMed, Front Pharmacol) - "These doses are expected to significantly improve ASD symptoms. This study not only proposes an optimized dosing strategy for AST-001 but also provides valuable insights into the PK-PD relationship in pediatric ASD treatment."

Journal • PK/PD data • Autism Spectrum Disorder • Genetic Disorders • Pediatrics

Examining the feasibility and limitations of AST-001 as a treatment for autism spectrum disorder. (PubMed, Psychiatry Clin Neurosci) - No abstract available

Journal • Autism Spectrum Disorder • Genetic Disorders

AST-001 versus placebo for social communication in children with autism spectrum disorder: A randomized clinical trial. (PubMed, Psychiatry Clin Neurosci) - "Our results provide preliminary evidence for the efficacy of AST-001 for the core symptoms of ASD."

Clinical • Journal • Autism Spectrum Disorder • Genetic Disorders

Phase III Clinical Trial to Evaluate the Efficacy and Safey of AST-001 in ASD Children (clinicaltrials.gov) - P3 | N=160 | Recruiting | Sponsor: Astrogen, Inc.

New P3 trial • Autism Spectrum Disorder • Genetic Disorders