None listed
Conditions
Brief summary
This study is testing whether cannabidiol (CBD), a non-psychoactive compound from the cannabis plant, can reduce irritability and aggressive behaviour in children, adolescents, and young adults with autism spectrum disorder (ASD). Over the first 12 weeks, participants will be randomly assigned to receive CBD at one of three doses or a placebo (inactive treatment) to compare differences in behaviour, sleep, and anxiety. After this, everyone will receive CBD for another 12 weeks, with some participants also receiving melatonin (to improve sleep) and/or N-acetylcysteine (to support emotional regulation). We believe CBD, alone or in combination with melatonin and N-acetylcysteine, will improve irritability, aggression, sleep, and overall quality of life for people with ASD.
Interventions
The trial consists of two phases:
Randomized, Double-Blind, Placebo-Controlled Phase (Weeks 0–12) with 3 treatment arms:
Arm 1: CBD 20 mg/kg/day (10 mg/kg taken orally twice daily, BD)
Arm 2: CBD 10 mg/kg/day (5 mg/kg taken orally twice daily BD)
Arm 3: CBD 6 mg/kg/day (3 mg/kg taken orally twice daily BD)
Duration: 12 weeks
Open-Label Extension Phase (OLE; Weeks 12–24):
All participants who complete the 12-week randomized phase are intended to proceed into the 12-week Open-Label Extension (Ph
The trial consists of two phases: Randomized, Double-Blind, Placebo-Controlled Phase (Weeks 0–12) with 3 treatment arms: Arm 1: CBD 20 mg/kg/day (10 mg/kg taken orally twice daily, BD) Arm 2: CBD 10 mg/kg/day (5 mg/kg taken orally twice daily BD) Arm 3: CBD 6 mg/kg/day (3 mg/kg taken orally twice daily BD) Duration: 12 weeks Open-Label Extension Phase (OLE; Weeks 12–24): All participants who complete the 12-week randomized phase are intended to proceed into the 12-week Open-Label Extension (Phase 2); this is not a separate cohort. No wash-out period for CBD is required. However, if a participant was using melatonin and/or NAC (non-study) during Phase 1, they must stop these at least 2 weeks before OLE enrolment—i.e., a 2-week wash-out for those adjuncts only. All participants receive CBD. Participants either continue CBD at their prior dose (if they were on CBD in Phase 1) or start CBD at OLE Day 1 if they were on placebo at 7.5 mg/kg taken orally twice daily. Participants are randomized to two adjunct sequences: Sequence A: CBD + NAC (Weeks 12–18), then CBD + NAC + Melatonin (Weeks 18–24) Sequence B: CBD + Melatonin (Weeks 12–18), then CBD + Melatonin + NAC (Weeks 18–24) Adjunctive Treatments (OLE Phase) 1. Melatonin Dose: 2 mg nightly (more than 35 kg) or 4 mg nightly (less than or equal to 35 kg) Route: Oral (capsule or solution, ARTG-registered products) Timing: 30 min before bedtime; no food within 2 hours before administration 2. N-acetylcysteine (NAC) Dose: 60–80 mg/kg/day in two divided doses (BD), max 2700 mg/day Route: Oral (solution or effervescent tablets) Duration: Introduced sequentially for 12 weeks for all participants, as per randomised sequence Dosing is fixed, oral, weight-based, with no escalation; adherence monitored via pill counts, diaries, and optional plasma CBD levels.
Sponsors
NeuroElevate Biosciences
Study design
Allocation
Randomised controlled trial
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
5 Years to 25 Years
Healthy volunteers
No
Inclusion criteria
1. Age: 5 to no older than 25 years. 2. ASD level 2 or level 3 (DSM-5 criteria), as diagnosed by a pediatrician or psychiatrist. 3. ABC-I subscale score more than or equal to 18 and CGI-S score more than or equal to 4. 4. Not currently receiving any oral or intramuscular anti-psychotic medications (typical or atypical). 5. Participants taking melatonin and/or NAC are eligible to participate in the first phase of the trial. However, to be eligible for the OLE phase, participants must cease taking melatonin and NAC at least two weeks prior to enrolment. Participants will still be eligible for the first phase of the study even if they choose not to cease taking melatonin and/or NAC. 6. Stable concomitant medications (more than or equal to 4 weeks) before enrolment, excluding anti-psychotics, which are not permitted at study entry.. 7. Caregiver or participant ability to complete assessments. 8. Willingness to provide informed consent/assent, as appropriate, based on age and legal requirements. 9. Caregiver availability to assist with study requirements, including completing assessments. 10. Body mass index (BMI) within the 5th to 95th percentile for age and gender. 11. No significant abnormalities in screening laboratory values that would preclude participation. 12. No active infections at the time of screening or baseline. 13. Normal or corrected vision to complete required study assessments. 14. Capable of swallowing oral medications without difficulty. 15. Stable housing environment for the duration of the study. 16. Consistent sleep schedule with no significant disruptions during the screening period. 17. Agreement to avoid initiating new behavioural or pharmacological treatments during the study. 18. No prior participation in other interventional trials within the last 30 days. 19. Stable seizure control (if applicable) with no changes in antiepileptic medications in the past 6 months. 20. No history of substance abuse or dependence within the last year. 21. No clinically significant cardiac, renal, hepatic, or respiratory conditions. 22. Demonstrated capacity to adhere to the visit schedule and study requirements.
Exclusion criteria
1. Current use of any anti-psychotic medication (oral or intramuscular, typical or atypical). 2. Severe psychiatric or neurological conditions unrelated to ASD. 3. Uncontrolled seizure disorder or recent antiepileptic medication changes. 4. Known hypersensitivity to CBD or melatonin. Participants with hypersensitivity to melatonin will be excluded from OLE but are eligible to be enrolled in the first phase of the study. 5. Concurrent enrollment in another interventional clinical trial. 6. Pregnant or breastfeeding individuals. 7. Known or suspected history of significant gastrointestinal disorders affecting drug absorption. 8. History of suicidal ideation or behaviour within the last 6 months. 9. Any active autoimmune disorders requiring systemic immunosuppressive therapy. 10. Positive screen for active drug or alcohol abuse. 11. Involvement in any legal or custodial disputes likely to interfere with participation. 12. Diagnosis of severe intellectual disability preventing meaningful participation in assessments. 13. History of severe adverse reactions to cannabinoids or related compounds. 14. Recent history (within 3 months) of major surgery or hospitalization. 15. Clinically significant abnormal findings on screening ECG. 16. Elevated liver transaminases (ALT or AST) exceeding 2.5 times the upper limit of normal. 17. Known active malignancy or history of cancer within the last 5 years (except for localized basal cell carcinoma). 18. Chronic or acute infectious diseases requiring ongoing treatment (e.g., tuberculosis, hepatitis B/C, HIV). 19. Inability to commit to study visits or comply with medication regimen. 20. Concurrent use of prohibited medications that could interact with study treatments. 21. Planned or foreseeable likelihood of initiation of other behaviour-modifying medication during the initial 12-week study phase.
Outcome results
None listed