None listed
Conditions
Brief summary
This is an open-label, outpatient, multiple ascending dose study of AB-2004 in approximately 35 adolescent subjects diagnosed with ASD accompanied by gastrointestinal symptoms. The primary outcome is to assess the safety and tolerability of AB-2004 administered daily over a period of 8 weeks as assessed by the frequency and severity of adverse events and laboratory abnormalities. Secondary outcomes include assessments of the effects of AB-2004 on intestinal permeability, systemic levels of host and microbially derived metabolites, biomarkers of systemic inflammation, the fecal microbiome profile, gastrointestinal signs and symptoms, core behaviors and affect (particularly anxiety and irritability) and the measures of brain connectivity and white matter integrity.
Interventions
It is now known that communication between the bacteria in your gut and your brain is important to the way in which your brain develops and functions. Additionally, your gut bacteria have been shown to have an impact on behavior. Studies have shown that the bacteria in the gut of children with ASD differs from the bacteria in the gut of typically developing children. The changes seen in the gut bacteria of children with ASD can increase the “leakiness” of the gut, making it easier for chemical s
It is now known that communication between the bacteria in your gut and your brain is important to the way in which your brain develops and functions. Additionally, your gut bacteria have been shown to have an impact on behavior. Studies have shown that the bacteria in the gut of children with ASD differs from the bacteria in the gut of typically developing children. The changes seen in the gut bacteria of children with ASD can increase the “leakiness” of the gut, making it easier for chemical substances produced by the bacteria to reach the brain and potentially affect the function of the brain. Axial Biotherapeutics has shown in animal studies that the chemical substances produced by certain gut bacteria may affect the core and non-core symptoms commonly found in ASD. Based on this research Axial Biotherapeutics has developed this study to be an open-label, outpatient, multiple ascending dose study of AB-2004 in a paediatric population diagnosed with autism spectrum disorder (ASD) accompanied by gastrointestinal symptoms. The primary outcome is to assess the safety and tolerability of AB-2004 administered daily over a period of 8 weeks as assessed by the frequency and severity of adverse events and laboratory abnormalities. Secondary outcomes include assessments of the effects of AB-2004 on intestinal permeability, systemic levels of host and microbially-derived metabolites, biomarkers of systemic inflammation, the fecal microbiome profile, gastrointestinal signs and symptoms, core behaviours and affect (particularly anxiety and irritability) and the measures of brain connectivity and white matter integrity. There will be a single treatment arm (AB-2004) consisting of three dosing periods. Total study duration will be 14-16 weeks. Treatment with AB-2004 administered orally, at approximately the same times each day (three times a day). AB-2004 is a granular, free flowing powder in a sachet that can be mixed into a snack and eaten. Weight is used to determine the starting dose of AB-2004. Participants weighing greater than or equal to 50 kg will receive 0.75 gm TID, while subjects weighing greater than or equal to 30 kg and less than 50 kg will receive 0.5 gm TID as a starting dose. In order to better facilitate adherence to the dosing regimen it is recommended that the either product be mixed with the participant’s favourite soft foods (yogurt).
Sponsors
Axial Biotherapeutics Australia Pty Ltd.
Study design
Allocation
Non-randomised trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
12 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
Participants must meet all inclusion criteria's (and not meet exclusion criteria) to be enrolled in the study. Following are the key Inclusion Criteria - Inclusion Criteria: 1. Clinically diagnosed, documented Autism Spectrum Disorder (DSM-V criteria) confirmed with ADOS-2 at Visit 1 or within 18 months prior to Visit 1. 2. Adolescents greater than or equal to 12 and less than 18 years of age at the time of consent 3. History of gastrointestinal symptoms (diarrhea, constipation, abdominal pain, bloating) confirmed in the e-diary with at least 50% compliance of entry for at least 14 days during the screening period. 4. Male subjects who are post-pubertal must be sterile (surgically or otherwise) for at least 6 months or are using single barrier contraception during the duration of the trial and up until 1 month after the last dose AB-2004. OR Female subjects that are not lactating and have a negative pregnancy test at the Screening and who are surgically sterile for at least 6 months or who agree to use double-barrier contraception, an intrauterine device, or an oral contraceptive over the duration of the trial and at least 4 weeks after the last dose of AB-2004
Exclusion criteria
Participants must not meet any exclusion criteria (and meet all inclusion criteria) to be enrolled in the study. Following are the key Exclusion Criteria - 1. History of inflammatory bowel disease, bowel obstruction, diverticulosis or colon polyps. 2. Oral, injected, inhaled antibiotic within 30 days prior to Screening 3. Currently taking a controlled or extended-release medication 4. History of significant gastric or intestinal surgery (excluding appendectomy).
Outcome results
None listed