E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Autism Spectrum Disorder (ASD) |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Psychiatry and Psychology [F] - Mental Disorders [F03] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10063844 |
E.1.2 | Term | Autism spectrum disorder |
E.1.2 | System Organ Class | 10037175 - Psychiatric disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the efficacy of GWP42003-P, compared with placebo, in reducing symptom severity in children with ASD.
To evaluate the safety of GWP42003-P, compared with placebo, in children with ASD. |
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E.2.2 | Secondary objectives of the trial |
• To evaluate changes in anxiety • To evaluate changes in repetitive behavior • To evaluate the effects in caregiver quality of life • To evaluate the PK/pharmacodynamic relationship of GWP42003-P and its metabolites • To evaluate performance in a standardized interactions task (at selected sites) • To evaluate speech (at selected sites) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
For inclusion in the trial, patients must fulfill ALL of the following criteria: • Male or female aged 6 to 17 years (inclusive). • Patient weight is at least 12 kg. • Patients (if possessing adequate understanding, in the investigator’s opinion) and their parent(s)/legal representative are willing and able to give informed assent and consent for participation in the trial. • Patient and their caregiver are willing and able (in the investigator’s opinion) to comply with all trial requirements. • Patient has a diagnosis of ASD as per DSM-5 criteria for ASD, confirmed by ADOS-2 criteria (conducted within 2 years at the trial site or at screening by a qualified assessor). Note: During special circumstances (e.g., COVID-19 pandemic) where the ADOS-2 cannot be performed due to site restrictions (e.g., mandatory use of face masks) and an ADOS-2 conducted within 2 years at the trial site by a qualified assessor is not available, eligibility can be confirmed using: 1) an ADOS-2 performed within 2 years by a qualified assessor(external to the site); 2) if 1) is not available, eligibility may be confirmed using the ADI-R at screening. • CGI-S > or = 4 (moderately ill) at screening and randomization. • ABC-I subscale score ≥ 15 at screening. • IQ > or = 70 at screening, or measured within 1 year of screening, using WASI-II. • All medications or interventions (including psychosocial interventions, dietary supplements, probiotics, speech therapy, etc.) for ASD related symptoms must have been stable for 4 weeks prior to screening and randomization, and the patient/caregiver should be willing to maintain a stable regimen throughout the trial. • Patients must have the ability to swallow the IMP, provided as a liquid solution. • Patient and/or parent(s)/legal representative is willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law. • Patient and/or parent(s)/legal representative is/are willing to allow the patient’s primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator. |
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E.4 | Principal exclusion criteria |
The patient may not enter the trial if ANY of the following apply: • Current diagnosis of bipolar disorder, psychosis, schizophrenia, schizoaffective disorder, or major depression (patients with depression in remission may be included). • Has a diagnosis other than ASD that dominates the clinical presentation (e.g., ADHD). • Has a progressive neurological condition. • Seizures in the past 24 weeks. • Changes in anticonvulsive therapy within the last 12 weeks. • Currently taking more than 2 AEDs. • Taking sirolimus, everolimus, temsirolimus, or tacrolimus. • Taking clobazam. • Taking omeprazole, lansoprazole, tolbutamide, or warfarin. • Taking repaglinide, pioglitazone, rosiglitazone, montelukast, bupropion, or efavirenz. • Currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®, or Epidiolex®/Epidyolex®) within the 12 weeks prior to screening and is unwilling to abstain for the duration of the trial. Patient has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP, such as sesame oil. • Patient has moderately impaired hepatic function at screening, defined as serum ALT or AST > 2 × ULN or TBL > 2 × ULN’ This criterion can only be confirmed once the laboratory results are available; patients enrolled into the trial who are later found to meet this criterion must be screen-failed. • Patient is male and fertile (i.e., after puberty unless permanently sterile by bilateral orchidectomy) unless willing to ensure that they use male contraception (condom) or remain sexually abstinent during the trial and for 12 weeks thereafter. • Patient is female and of childbearing potential (i.e., following menarche and until becoming postmenopausal for > o = 12 consecutive months unless permanently sterile by hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) unless willing to ensure that they use a highly effective method of birth control (e.g., hormonal contraception, intrauterine device/hormone-releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the trial and for 12 weeks thereafter. • Female patient who is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the trial or within 12 weeks thereafter. • Patient has received an IMP within the 12 weeks prior to the screening visit. • Patient had brain surgery or traumatic brain injury within 1 year of screening. • Patient has any other significant disease or disorder which, in the opinion of the investigator, may either put the patient, other participants, or site staff at risk because of participation in the trial, may influence the result of the trial, or may affect the patient’s ability to take part in the trial. • Any abnormalities identified following a physical examination of the patient that, in the opinion of the investigator, would jeopardize the safety of the patient if they took part in the trial. • Any history of suicidal behavior (lifelong) or any suicidal ideation of type 4 or 5 on the C-SSRS in the last 4 weeks or at screening or randomization. Patient has donated blood during the past 12 weeks and is unwilling to abstain from donation of blood during the trial. • Patient has any known or suspected history of alcohol or substance abuse or positive drugs of abuse test at screening (not justified by a known concurrent medication). Patient has previously been randomized into this trial. • Patient has plans to travel outside their country of residence during the trial, unless the patient has confirmation that the IMP is permitted in the destination country/state. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy Endpoints: Behavior • ABC Subscales Social Communication • VABS-3 Overall Condition • CGI-I • CGI-S
Safety Endpoints: • AEs. • Clinical laboratory parameters. • Vital signs. • Physical examination procedures. • 12-lead ECG. • C-SSRS. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Efficacy Endpoints: ABC Subscales: Visits 1, 2, 5, 7 & 8 VABS-3: Visits 2, 5, 7 & 8 CGI-I: Visits 5, 7 & 8 CGI-S: Visits 1, 2, 5, 7 & 8)
Safety Endpoints: AEs: at each visit Clinical laboratory parameters: Visits 1, 5, 8 (if not tapering) & 9 Vital signs: Visits 1, 2, 5, 7, 8 & 9 Physical examination procedures: Visits 1 & 9 12-lead ECG: Visits 1 & 9 C-SSRS: Visits 1, 2, 5, 7, 8 & 9 |
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E.5.2 | Secondary end point(s) |
Exploratory Endpoints:
• PARS • RBS-R • CarerQoL • Explore potential correlations between plasma exposure of GWP42003-P and its metabolites with markers of efficacy • BOSCC • Speech analytics |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
PARS: Visits 2 & 8 RBS-R: Visits 2, 5, 7 & 8 CarerQoL: Visits 2 & 8 Explore potential correlations between plasma exposure of GWP42003-P and its metabolites with markers of efficacy: At study end. BOSCC: Visits 2 & 8 Speech analytics: Visits 2 & 8 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Canada |
Germany |
Spain |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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Last patient last visit or last contact, whichever occurs last. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |