arbaclofen (STX209) / Allos Pharma - LARVOL DELTA

Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion (clinicaltrials.gov) - P2 | N=60 | Active, not recruiting | Sponsor: Clinical Research Associates, LLC | Recruiting ➔ Active, not recruiting

Enrollment closed

Retinal GABAergic alterations in adults with autism spectrum disorder. (PubMed, J Neurosci) - "Participants were administered an oral dose of placebo, 15 or 30 mg of arbaclofen (STX209, GABAB agonist) in a randomized, double-blind, cross-over order before the test...The extent of this differential GABAergic function correlated with the extent of autistic traits captured using the Autism Quotient. Thus, sensory processing differences in autism appear to be upstream of more complex autistic traits and the ERG from the retina is a potentially useful proxy for cross-domain brain GABA function and target engagement."

Clinical • Journal • Autism Spectrum Disorder • CNS Disorders • Genetic Disorders

Exploratory evidence for differences in GABAergic regulation of auditory processing in autism spectrum disorder. (PubMed, Transl Psychiatry) - "Neurophysiological responses (temporal and frequency domains) to repetitive standard tones and novel deviants presented in an oddball paradigm were compared after double-blind, randomized administration of placebo, 15 or 30 mg of arbaclofen (STX209), a GABA type B (GABA) receptor agonist...Thus, our results confirm: GABAergic dysfunction contributes to the neurophysiology of auditory sensory processing alterations in ASD, and can be modulated by targeting GABA activity. These GABA-dependent sensory differences may be upstream of more complex autistic phenotypes."

Journal • Autism Spectrum Disorder • Genetic Disorders

Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: Clinical Research Associates, LLC | Trial primary completion date: Aug 2024 ➔ Dec 2024

Trial primary completion date

Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: Clinical Research Associates, LLC | Trial completion date: May 2023 ➔ Dec 2024 | Trial primary completion date: Mar 2023 ➔ Aug 2024

Trial completion date • Trial primary completion date

AIMS2-CT1: Arbaclofen in Children and Adolescents With ASD (clinicaltrials.gov) - P2 | N=124 | Completed | Sponsor: Celso Arango, MD, PhD | Recruiting ➔ Completed

Trial completion • Autism Spectrum Disorder • Genetic Disorders

Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: Clinical Research Associates, LLC | Suspended ➔ Recruiting

Enrollment open

AIMS2-CT1: Arbaclofen in Children and Adolescents With ASD (clinicaltrials.gov) - P2 | N=130 | Recruiting | Sponsor: Celso Arango, MD, PhD | Trial completion date: Aug 2022 ➔ Dec 2022 | Trial primary completion date: Aug 2022 ➔ Dec 2022

Trial completion date • Trial primary completion date • Autism Spectrum Disorder • Genetic Disorders

Modulation of the Brain Excitatory/Inhibitory (E/I) Balance in Autism Spectrum Disorder (ASD) (clinicaltrials.gov) - P=N/A | N=87 | Completed | Sponsor: King's College London | Recruiting ➔ Completed

Trial completion • Autism Spectrum Disorder • Genetic Disorders

The GABAB receptor agonist STX209 reverses the autism‑like behaviour in an animal model of autism induced by prenatal exposure to valproic acid. (PubMed, Mol Med Rep) - "However, expression of glutamic acid decarboxylase 65/67 in the hippocampus were not altered by STX209. The present results demonstrated that STX209 administration ameliorated autism‑like symptoms in mice exposed to VPA prenatally, suggesting that autism‑like symptoms in children with a history of prenatal VPA exposure may also benefit from treatment with the GABABR2 agonist STX209."

Journal • Preclinical • Autism Spectrum Disorder • Genetic Disorders

Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion (clinicaltrials.gov) - P2; N=60; Suspended; Sponsor: Clinical Research Associates, LLC; Trial completion date: May 2022 ➔ May 2023; Not yet recruiting ➔ Suspended; Trial primary completion date: Mar 2022 ➔ Mar 2023

Clinical • Trial completion date • Trial primary completion date • Trial suspension

AIMS2-CT1: Arbaclofen in Children and Adolescents With ASD (clinicaltrials.gov) - P2; N=130; Recruiting; Sponsor: Celso Arango, MD, PhD; Trial completion date: Aug 2021 ➔ Aug 2022; Trial primary completion date: Aug 2021 ➔ Aug 2022

Clinical • Trial completion date • Trial primary completion date • Autism Spectrum Disorder • Genetic Disorders

Allos Pharma secures exclusive license on arbaclofen in Fragile X Syndrome (PRNewswire) - "Allos Pharma Inc...has announced the exclusive license rights on arbaclofen in Fragile X Syndrome (FXS). The company is preparing the regulatory path for market authorization....'NFXF supports the future work of Allos Pharma and looks forward to working in partnership to better the lives of individuals with Fragile X Syndrome and their families'...'We are delighted that Drs. Bear and Carpenter and the rest of the Allos team are moving ahead with arbaclofen to treat core symptoms in children with Fragile X.'"

Licensing / partnership • Fragile X Syndrome

AIMS2-CT1: Arbaclofen in Children and Adolescents With ASD (clinicaltrials.gov) - P2; N=130; Recruiting; Sponsor: Inge Winter; Trial completion date: Jan 2021 ➔ Aug 2021; Trial primary completion date: Jan 2021 ➔ Aug 2021

Clinical • Trial completion date • Trial primary completion date • Autism Spectrum Disorder • Genetic Disorders

Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion (clinicaltrials.gov) - P2; N=60; Not yet recruiting; Sponsor: Clinical Research Associates, LLC

Clinical • New P2 trial

A MEG Study of Acute Arbaclofen (STX-209) Administration. (PubMed, Front Integr Neurosci) - "Whether these subjects have a greater trend towards clinical benefit remains to be established. Finally, findings also provide preliminary support for the use of objective electrophysiological measures upon which to base inclusion for optimal enrichment of populations to be included in full-scale clinical trials of arbaclofen."

Journal

MEG Study of Acute STX209 Effects in ASD (clinicaltrials.gov) - P1; N=25; Completed; Sponsor: Timothy Roberts; Active, not recruiting ➔ Completed; N=40 ➔ 25

Enrollment change • Trial completion

AIMS2-CT1: Arbaclofen in Children and Adolescents With ASD (clinicaltrials.gov) - P2; N=130; Recruiting; Sponsor: Inge Winter; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

AIMS2-CT1: Arbaclofen in Children and Adolescents With ASD (clinicaltrials.gov) - P2; N=130; Not yet recruiting; Sponsor: Inge Winter; Initiation date: Oct 2018 ➔ Jun 2019

Clinical • Trial initiation date

MEG Study of STX209 (clinicaltrials.gov) - P1; N=40; Active, not recruiting; Sponsor: Timothy Roberts; Trial primary completion date: Jan 2019 ➔ Jul 2019

Trial primary completion date