Neurotech International Limited (ASX: NTI) has achieved successful outcomes relating to the safety, tolerability, and efficacy of NTI164 and on key behavioural parameters that impact ASD patients.
NTI164 is one of NTI’s proprietary cannabis strains, exclusively licenced from Dolce Cann Global (Ltd), in respect of neurological applications and is the world’s first full-spectrum medicinal cannabis product (less than 0.3% THC) to be successfully studied in children with Autism Spectrum Disorder (ASD).
The study was conducted by Professor Michael Fahey, Head of Paediatric Neurology at Monash Children’s Hospital in Melbourne.
The Phase I/II study was designed to rigorously assess the safety of NTI164 in a dose-escalation regime and to detect a signal for efficacy on the behaviour, focus and related cognitive parameters using a range of validated neuro-psychological tools.
This study was designed to form the foundation for follow-up studies in therapies relating to the treatment of a wide range of neurological disorders such as Attention Deficit Hyperactivity Disorder (“ADHD”), Multiple Sclerosis, Motor Neuron Disease, Rett Syndrome and Cerebral Palsy.
Overall Study Design and Outline:
- Open-label study.
- The study population: Children aged between eight years through to seventeen years that have a medical diagnosis of Level II and III Autism Spectrum Disorder (ASD) as confirmed by the Autism Diagnostic Observational Schedule (ADOS-2) criteria.
Study Primary Endpoints:
- Safety and tolerability – across dose regime (5mg/kg, 10mg/kg, 15mg/kg and 20 mg/kg).
- Safety was monitored and measured by clinical examination, full blood examinations, liver and renal function tests in addition to parent/carer and physician questionnaires.
Study Behavioural Endpoints:
- Efficacy was measured through parent/carer and physician questionnaires to assess parameters including, but not limited to: – Anxiety – Participation – Irritability – Hyperactivity – Mood, and – Self-stimulation. In total, over 2,250 assessment points were created through the landmark study.
Key Study Results
- The safety data concluded that NTI164 at 5, 10, 15 and 20mg/kg administered in two doses daily, is safe and well-tolerated in this study population
- No changes were observed in patients’ full blood examination, liver function or kidney function tests. There were no changes observed in the patients’ vital signs or weight.
EFFICACY
- Statistical analysis of key assessments, including Clinical Global Impression of Severity of Illness (CGIS) demonstrated statistical significance at 28 days of treatment. Paired t-test: the mean difference of CGI-S between 28 days of treatment and baseline was -0.714, 95% Confidence Interval = -1.332, – 0.097, p value=0.027.
- 93% (13 out of 14 active patients) showed symptom improvement relating to severity of illness after 28 days of daily treatment with NTI164.
- Most importantly, parental/carer observations also indicated consistent improvement in the trial participant’s ‘overall functioning’ when compared to baseline at the commencement of the trial. The average rating for the severity of illness at baseline was 4.4 (out of a score of 7 meaning extremely ill and 1 meaning, not ill) and this score was reduced to 3.6 after 28 days of NTI164 treatment.
