Nervous System Diseases

All Conditions

Behaviors and Mental Disorders

Substance Related Disorders

Epilepsy

Neurodevelopmental Disorders

Marijuana Abuse

Mental Disorders

Psychotic Disorders

For children with epilepsy, the following tools will be added for data collection at each time point:

* Side effects: Pediatric Epilepsy Side Effects Questionnaire \[PESQ\]
* Seizure frequency/severity: Seizure Diary Data Questionnaire

For children with cancer, the following tools will be added for data collection at each time point:

* Cancer symptom burden: Symptom Screening in Pediatrics Tool \[SSPedi\]
* Cachexia: Pediatric Functional Assessment of Anorexia/ Cachexia Treatment \[peds-FAACT\]

There are no specific outcome scales added for this cohort.

Any cannabis product that is available to participants through the Medical Cannabis Access Program is eligible to be included in this study.

Medical Cannabis

Lauren E Kelly, PhD

Lauren Kelly, PhD

Sapna Oberoi, MD

Taylor Lougheed, MD

Evan Lewis, MD

CAN-RWE is an observational study that is following 500 children who have authorizations for medical cannabis for two years from across Canada.

Pediatric patients (3 to 17 years) using medical cannabis (MC) for pain, sleep, mood, behaviour, seizures, treatment of cancer, or to manage symptoms related to cancer or cancer-treatment are eligible to join this 24-month study if they have a valid MC authorization.

Following screening, eligible participants will be contacted by the study coordinator. Following informed consent, study participants will record complete outcome measures on pain, sleep, mood (anxiety, depression and positive affect), behaviour, as well as report their cannabis use and indication-specific outcomes as applicable at 3, 6, 12, and 18 weeks, as well as 6, 12, 18 and 24-months.

Real World Evidence on the Use of Medical Cannabis in Pediatrics

Real World Evidence on the Use of Medical Cannabis in Pediatrics: A Prospective Observational Study

summary level data will be available upon reasonable request with appropriate approval and oversight of a research ethics board.

Describe patterns of cannabis use are described using a standard data collection form including dose and product type completed by caregivers at baseline, 3, 6, 12, and 18-weeks and 6, 12, 18 and 24-months

Longitudinal assessment for benefit signals in epilepsy seizure frequency/severity measure by a seizure diary

Longitudinal assessment for benefit signals including epilepsy related side effects measured using QOLCE

Longitudinal assessment for benefit signals in caregiver reported changes in symptom burden assessed using mini-SSPEDI (under 8 yr) and SSPEDI scales

Longitudinal assessment of cachexia using Peds-FAACT

Cannabis-related adverse events and serious adverse events reported by caregivers using a standard AE data collection tool and categorized according to CTCAE v5.0 at baseline, 3, 6, 12, and 18-weeks and 6, 12, 18 and 24-months

Longitudinal assessment of cachexia using PedsQL

PROMIS short form scales for pediatric anxiety symptoms 8a v2.0

PROMIS short form scales for pediatric depression symptoms 8a v2.0

PROMIS short form pediatric positive affect scale 8a v2.0

PROMIS short form pediatric sleep related impairment scale 8a v2.0

PROMIS short form pediatric pain interference scale 8a v2.0

Parents/Caregivers will be asked to report on if their childs behaviour has improved or worsened and will have an open-ended text box to describe anything they want to tell us about changes in their child's behaviour.

The Canadian Collaborative for Childhood Cannabinoid Therapeutics

Canadian Cancer Society (CCS)

Canadian Institutes of Health Research (CIHR)

University of Manitoba

Symptoms and General Pathology

Neoplasms

Diseases and Abnormalities at or Before Birth

Rare Diseases

Syndrome

Autistic Disorder

Hamartoma

Hamartoma Syndrome, Multiple

Neoplasms, Multiple Primary

Neoplastic Syndromes, Hereditary

Genetic Diseases, Inborn

PTEN Hamartoma Tumor Syndrome

Patients diagnosed with PTEN Hamartoma Tumor Syndrome and a neurodevelopmental disorder including but not limited to autism spectrum disorder, attention-deficit disorder / attention-deficit hyperactivity disorder, speech language disorder(s), intellectual disability, global developmental delay, oppositional defiant disorder, and / or motor or developmental disorders.

Patients diagnosed with PTEN Hamartoma Tumor Syndrome

Siblings related to patients with diagnosis of PTEN Hamartoma Tumor Syndrome

Unrelated participants with no known genetic or neurodevelopmental disorder

Online survey measuring a variety of developmental domains including anxiety, motor, daily living skills, challenging behaviors, social communication, restrictive and repetitive behaviors, ADHD, executive functioning, mood, quality of life, and sleep

The purpose of this study is to develop a survey completed by parents, caregivers, or other informants that evaluates several important domains of functioning relevant to individuals with neurodevelopmental disorders, including autism spectrum disorder and people with genetic syndromes. Participation in this research will include assisting the research team in the development of the survey measures.

With greater understanding of the biology of PTEN Hamartoma Tumor Syndrome (PHTS), including patients with and without neurodevelopmental disorder (NDD) phenotypes, there is potential to develop novel personalized therapeutics. Given the recent observation that many PHTS patients with and without NDD have significant neurobehavioral manifestations, it will be crucial to broadly track a range of neurobehavioral domains as part of future trials. Unfortunately, there are few well-validated measures that would be appropriate for the full range of presentations in the PHTS population. Further, as a rare genetic syndrome, PHTS patients are geographically distributed making frequent data collection during research very difficult. Thus, the overarching aim of this proposal is to develop and validate an online neurobehavioral evaluation tool (NET) with caregiver-report survey scales and patient-completed webcam-collected performance measures that captures key neurobehavioral information. In partnership with patients, caregivers, and scientist-clinician experts, the study will develop, pilot test, and validate NET survey and performance measures. Specifically, at least 240 caregiver-patient pairs will be recruited (patient ages 4-24) from PHTS without NDD, PHTS with NDD, and controls (sibling and unrelated healthy controls). NET data will be collected at baseline, 1-month, and 4-month follow-up. Aim 1 will focus on the development and validation of brief, psychometrically-sound NET survey scales. Aim 2 will conduct group comparisons on NET measures. Aim 3 will use longitudinal data to evaluate short-term stability of NET survey scales and describe neurobehavioral change within and across study groups. The project will also explore the feasibility of performance measure collection, provide initial psychometric evaluation, and conduct a group comparison of performance measures. If the project aims are achieved, and future validation supports research and clinical use, the NET will be an efficient, reliable, valid, co-normed, multi-modal instrument, that, by virtue of online (at-home) administration, will decrease patient and family burden and increase the temporal resolution and power of future longitudinal studies and clinical trials.

Development and Validation of an Online Neurobehavioral Evaluation Tool for PTEN Patients

Development and Validation of an Online Neurobehavioral Evaluation Tool for PTEN Hamartoma Tumor Syndrome (PHTS)

Plan to upload deidentified data to Open Science Framework

Develop survey questions and scales related content areas for Social Communication Interactions. Social communication refers to the emergence of verbal and nonverbal skills, social interaction, and social cognition

Develop questions and survey scales related to content areas of activities of daily living. Activities of daily living are activities related to personal care. They include bathing or showering, dressing, getting in and out of bed or a chair, walking, using the toilet, and eating.

Develop survey and rating scales related to content areas for restrictive and repetitive behaviors. Restricted and repetitive behaviors and interests are among the three core symptoms of autism. They include repetitive movements with objects, repeated body movements such as rocking and hand-flapping, ritualistic behavior, sensory sensitivities and circumscribed interests

Develop survey questions and scales related to content areas for anxiety. Anxiety is what we feel when we are worried, tense or afraid - particularly about things that are about to happen, or which we think could happen in the future

Develop survey questions and scales related to content areas for motor skills.A motor skill is a function that involves specific movements of the body's muscles to perform a certain task. These tasks could include walking, running, or riding a bike. In order to perform this skill, the body's nervous system, muscles, and brain have

Develop survey questions and rating scales related to content areas for challenging behaviors.A person's behaviour can be defined as "challenging" if it puts them or those around them (such as their carer) at risk, or leads to a poorer quality of life. It can also impact their ability to join in everyday activities. Challenging behaviour can include: aggression. self-harm.

Develop survey questions and rating scales related to content areas for ADHD. Refers to the ongoing pattern of inattention and/or hyperactivity-impulsivity that interferes with functioning or development.

Develop survey questions and rating scales related to content areas for executive functioning. Executive functioning and self-regulation skills are the mental processes that enable us to plan, focus attention, remember instructions, and juggle multiple tasks successfully.

Develop survey questions and rating scales related to content areas for mood. Focusing on particular feelings or a state of mind.

Develop survey questions and rating scales related to content areas for quality of life including family, financial, etc.

Develop survey questions and rating scales related to content areas for sleep patterns and behaviors.

University Hospital Southampton NHS Foundation Trust

John Carroll University

Musculoskeletal Diseases

Heart and Blood Diseases

Blood and Lymph Conditions

Gland and Hormone Related Diseases

Skin and Connective Tissue Diseases

Sclerosis

Anxiety Disorders

Hyperkinesis

Autism Spectrum Disorder

Phobia, Social

Impulsive Behavior

Fragile X Syndrome

Tourette Syndrome

Tics

Tic Disorders

Tuberous Sclerosis

Agoraphobia

DiGeorge Syndrome

22q11 Deletion Syndrome

Anxiety, Separation

Phobic Disorders

Child Development Disorders, Pervasive

Developmental Disabilities

Ganglion Cysts

Synovial Cyst

Basal Ganglia Diseases

Brain Diseases

Central Nervous System Diseases

Movement Disorders

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Intellectual Disability

Mental Retardation, X-Linked

Neurobehavioral Manifestations

Neurologic Manifestations

Chromosome Disorders

Sex Chromosome Disorders

Congenital Abnormalities

Genetic Diseases, X-Linked

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Dyskinesias

Craniofacial Abnormalities

Musculoskeletal Abnormalities

Heart Defects, Congenital

Cardiovascular Abnormalities

Heart Diseases

Lymphatic Abnormalities

Lymphatic Diseases

Abnormalities, Multiple

Hypoparathyroidism

Parathyroid Diseases

Endocrine System Diseases

Tuberous Sclerosis Complex

22q11.2 Deletion Syndrome

Lymphatic Malformations

Psychotropic Drugs

All Drugs and Chemicals

Sertraline

Selective Serotonin Reuptake Inhibitors

Serotonin

Antidepressive Agents

Neurotransmitter Agents

Serotonin Receptor Agonists

Placebo

Karly Janisse

Paul Arnold

Francois Bolduc

Megan Thomas

Alessia Greco

Elisabetta Trinari

Ramona Rego

Sarosh Khalid-Khan

Baudouin Forgeot D'Arc

Evdokia Anagnostou

There are currently no approved medications for the treatment of anxiety in children and youth with neurodevelopmental disorders (NDDs), both common and rare. Sertraline, a selective serotonin reuptake inhibitor, has extensive evidence to support its use in children's and youth with anxiety but not within NDDs. More research is needed to confirm whether or not sertraline could help improve anxiety in children and youth with common and rare neurodevelopmental conditions. This is a pilot study, in which we plan to estimate the effect size of reduction in anxiety of sertraline vs. placebo. across rare and common neurodevelopmental disorders, and determine the best measure(s) to be used as a primary transdiagnostic outcome measure of anxiety, as well as diagnosis specific measures in future, larger-scale clinical trials of anxiety in NDDs.

Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders

A Randomized Placebo-Controlled Trial of Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders

The MASC-2 will be used as an additional anxiety measure to examine the effect of sertraline vs. placebo on measures of anxiety. The MASC-2 can be rated by parents or completed as a self-report. The parent rated version will be used and if the participant has adequate language ability, the self-report will also be used. The MASC-2 is a 50-item questionnaire. Each item is rated from 0 (Never) to 3 (Often). A higher score is indicative of higher symptoms of anxiety.

The CGI-S is a clinician reported scale which will be used to examine the effect of sertraline vs. placebo on measures of global function. The CGI-Severity Scale employs a seven point (1 = Normal, not at all ill to 7 = among the most extremely ill patients) to determine the participant's initial level of severity of impairment.

The SCARED is a 41-item measure of anxiety symptoms, with both child and parent versions. The Child version will be used as an exploratory outcome measure. The minimum overall score is 0 while the maximum overall score is 82. A score greater than or equal to 25 may indicate the presence of an Anxiety Disorder. Higher scores indicate a higher instance of anxiety symptoms while a lower score indicates a lower instance of anxiety symptoms.

The CGI-I is a clinician reported scale which will be used to examine the effect of sertraline vs. placebo on measures of global anxiety. The CGI-Improvement Scale employs a seven point (1 = very much improved to 7 = very much worse) to determine the participant's improvement in response to treatment.

The Parent Chief Complaint will be used to examine the effect of sertraline vs. placebo on individualized target symptoms. The objective of this measure is to have the parent describe the frequency and severity of one or two primary concerns of their child's behavior at the baseline visit and again at the end of treatment. Frequency is rated as Very Often (3), Often (2), Sometimes (1), or Rarely (0), and Severity is rated as Mild (1; behaviour occurs and is a mild problem), Moderate (2; behaviour occurs and is a moderate problem), and Severe (3; behaviour occurs and is a severe problem).

The PRAS-ASD will be used to examine the effect of sertraline vs. placebo on measures of anxiety in ASD. It is completed by the parents and includes 25 questions rated on a scale for 0-3 (none-severe problem).

The ADAMS is designed to assess anxiety and depression in children with and without intellectual disability. The ADAMS is a 28 item questionnaire which is rated by the parent. Each item is rated from 0 (behaviour has not occurred, or is not a problem) to 3 (behaviour occurs a lot, or is a severe problem).

The SCARED is a 41-item measure of anxiety symptoms, with both child and parent versions. The parent version will be used as a primary outcome measure. The minimum overall score is 0 while the maximum overall score is 82. A score greater than or equal to 25 may indicate the presence of an Anxiety Disorder. Higher scores indicate a higher instance of anxiety symptoms while a lower score indicates a lower instance of anxiety symptoms.

The CGI-I is a clinician reported scale which will be used to examine the effect of sertraline vs. placebo on measures of global function. The CGI-Improvement Scale employs a seven point (1 = very much improved to 7 = very much worse) to determine the participant's improvement in response to treatment.

The SMURF will be administered by the clinician at each visit to examine the safety and tolerability of sertraline. This rating covers a range of behavioural and physical AEs that are relevant to SSRIs.

The PedsQL will be used to examine the effect of sertraline vs. placebo on measures of quality of life. The PedsQL is measuring health-related quality of life (HRQOL) in 2- to 18-year-olds. The PedsQL 4.0 Generic Core Scales are multidimensional child self-report and parent-proxy report scales that have been used extensively as an outcome measure, including in ASD. We will use the parent-proxy report scale, and optionally, age-appropriate child self-report scale. Each item is is rated between a 0 (Never a problem) to 4 (Almost always a problem).

The HUI-3 will be used to examine the effect of sertraline vs. placebo on measures of general health status and health-related quality of life. The study is using the parent-proxy version of the HUI-3. The HUI-3 is a 15-item questionnaire to classify a subject's health status.

A whole blood serotonin assessment will be completed to examine the effect of sertraline vs. placebo on biomarkers of serotonin. 3mL of blood will be drawn at screening and week 16 visits for the purpose of this assessment. High performance liquid chromatography will be performed using fluorometric detection of 5-HT, tryptophan (TRP), and 5-hydroxyindole acetic acid (5-HIAA), using N-methylserotonin as an internal standard. Using this method, 5-HT intra- and inter- assay coefficients of variation are reliably less than 5% and 10%, respectively.

Azrieli Foundation

Ontario Brain Institute

Holland Bloorview Kids Rehabilitation Hospital

McMaster University

Western University

Queen's University

University of Alberta

Alberta Health services

St. Justine's Hospital

Dalhousie University

Unity Health Toronto

University of Toronto

The Hospital for Sick Children

Anagnostou, Evdokia, M.D.

Immune System Diseases

Multiple Sclerosis

Brain Neoplasms

Autoimmune Diseases

Demyelinating Autoimmune Diseases, CNS

Autoimmune Diseases of the Nervous System

Demyelinating Diseases

Pediatric patients referred to the participating imaging centre for a standard MRI procedure.

Adult patients referred to the participating imaging centre for a standard MRI procedure, with neurological signs and symptoms.

Acquisition of MRI data with technique "Magnetic Resonance Fingerprinting"

MRI exam with Magnetic Resonance Fingerprinting

Magnetic Resonance Imaging (MRI) is an excellent method for diagnosis and staging of brain disease. However, lengthy scan times and sensitivity to patient motion limit its efficacy. To address this, a novel method has recently been demonstrated, called MR Fingerprinting (MRF). The investigators' improved implementation of MRF, featuring fully-quantitative data and a reduced sensitivity to patient motion, can be used to acquire an anatomical exam in less than five minutes at a standard resolution. The potential for wide applicability of this technique, combined with an implied reduction in complexity and cost of MRI exams, has generated wide interest. However, published studies have been limited to demonstrations in healthy volunteers, and the effectiveness of MRF in the clinical practice has not yet been proven. Here, the investigators aim to assess the efficacy of MRF in performing diagnostic exams avoiding sedation in children and for increasing diagnosis rates in challenging adult patients.

Due to its unrivalled diagnostic and prognostic capabilities, as well as the absence of harmful effects from ionizing radiation, MRI is often the radiological method of choice in children. However, radiology units have to face the inability to produce motion-free images especially in young children, even more so in those affected by neurological or neuropsychiatric disorders. These children are often incapable of completely following verbal instructions and cannot remain still in the scanner for a full exam. To avoid uncontrolled motion and the anxiety derived from long examinations, the majority of young children are sedated. However, sedation represents an extra procedure, carrying its own possible adverse effects, procedural complications as well as additional costs. Producing diagnostic, artefact-free images in a short time in children without sedation is thus an unmet need. The investigators' first hypothesis is that MR Fingerprinting can be used in a number of cases to perform a fast and diagnostic exam in children without using sedation.

The advantages of a fast, motion-resistant quantitative scan are not limited to paediatrics but could see wide application.

Disorders of movement such as tremor-dominant Parkinson's disease and severe cognitive impairments such as Alzheimer's disease represent a big challenge when referred to MRI. Although MRI is not a major player in diagnosing such diseases, these patients often require an exam in order to exclude other pathologies with similar clinical manifestations and to diagnose or follow-up other concomitant conditions. Patients with expansive intracranial lesions or requiring frequent follow-up, like those affected by inflammatory disease of the central nervous system, need to undergo long and demanding procedures, and they can eventually get into significant distress. The image quality of standard MRI with these challenging patient groups is often poor, as they cannot lay still or are unable to follow instructions. The investigators' second hypothesis is that MRF can be used to increase diagnosis rates of exams in difficult adult populations, reducing the time spent in the scanner and thus reducing wasted procedures and cost, and increasing patient comfort.

Increasing Diagnosis Rates While Reducing Examination Time: Can MR Fingerprinting Deliver on Its Promise?

Aumentare il Potere Diagnostico Riducendo la Durata Dell'Esame: MR Fingerprinting Soddisfa le Aspettative?

To evaluate diagnosis rates with MRF in pediatric patients and challenging adult populations

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Real World Evidence on the Use of Medical Cannabis in Pediatrics	NCT05863910	Cancer Epilepsy Neurodevelopmen...	Medical Cannabi...	3 Years - 17 Years	University of Manitoba
Development and Validation of an Online Neurobehavioral Evaluation Tool for PTEN Patients	NCT05671107	PTEN Hamartoma ...	NET	3 Years - 45 Years	John Carroll University
Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders	NCT06081348	Neurodevelopmen... Autism Autism Spectrum... Fragile X Syndr... Tuberous Sclero... 22Q11 Deletion ... 22Q11 Deletion ADHD Tic Disorders Tourette Syndro... Tourette Syndro... Tourette Syndro... ADHD - Combined... ADHD Predominan... ADHD, Predomina... Provisional Tic... Anxiety Anxiety Disorde... Agoraphobia Generalized Anx... Generalized Anx... Social Anxiety Social Anxiety ... Unspecified Anx... Other Specified... Separation Anxi...	Sertraline Placebo	8 Years - 17 Years	Anagnostou, Evdokia, M.D.
Increasing Diagnosis Rates While Reducing Examination Time: Can MR Fingerprinting Deliver on Its Promise?	NCT06251830	Neurodevelopmen... Neurodegenerati... Brain Tumor Multiple Sclero... Epilepsy	MRI exam with M...	4 Years -	IRCCS Fondazione Stella Maris
Real World Evidence on the Use of Medical Cannabis in Pediatrics	NCT05863910	Cancer Epilepsy Neurodevelopmen...	Medical Cannabi...	3 Years - 17 Years	University of Manitoba

Spots Global Cancer Trial Database for neurodevelopmental disorders