Clinical Research Directory

Real World Outcomes of Intranasal MuSE Exosomes and Stem Cells in Neurological Regenerative Therapy

This prospective observational study collects real world data on participants receiving regenerative therapies administered internationally and delivered intranasally via the Kurve Therapeutics ViaNase device. The study does not assign treatment. Participants are enrolled after receiving, or electing to receive, therapy as part of routine clinical care outside the study. Participants are observed in one of three cohorts based on the therapy received: MuSE cell derived exosomes, MuSE stem cells, or combination therapy. The objective is to evaluate safety, tolerability, and changes in inflammatory biomarkers and clinical outcomes over time in a real world setting. The study also evaluates changes in inflammatory biomarkers, including serum tumor necrosis factor alpha (TNF-α), to better understand the biological effects of these therapies.

Spinal Cord Stimulation for Children With Cerebral Palsy

The goal of this clinical trial is to help understand spinal cord excitability in children with cerebral palsy compared to neurologically typical children. The main questions it aims to answer are: * What are the mechanisms of spinal circuit hyperexcitability in CP? * Can transcutaneous spinal cord stimulation reduce spinal circuit hyperexcitability? Participants with cerebral palsy will be asked to complete: * Movement and strength exercises * Mild, non-invasive stimulation on the back and legs that feels like a light tap. * Gentle, non-invasive tests that help us see how the brain and the spinal cord communicate with the muscles

Investigating New Methods to Study Movement in Children and Young Adults With Movement Disorders.

Background: Cerebral palsy (CP) is the most common motor disorder that affects children. People with CP have weak muscles; they may have trouble controlling the movements of their arms and legs. Researchers have been developing braces called robotic exoskeletons for people with CP. These devices can adapt to the person s movements and help them move better. This natural history study will explore new technologies that may tell us more about how people with CP move and improve how these exoskeletons work. Objective: To test new technologies to measure people s movements and brain function while they move with and without a robotic exoskeleton. Eligibility: People aged 5 to 25 years with CP. Healthy volunteers are also needed. Design: Participants will have 3 to 5 clinic visits in 2 months. Participants will be fitted with an exoskeleton that will be worn on one of their legs. At each visit, participants will be asked to move their wrist, ankle, and knee while the following measurements are taken: Ultrasound. A bar will be placed against the skin. It will send soundwaves into the body to take pictures of the muscles. Electroencephalography (EEG). Participants will wear a cap with sensors. Their brain waves will be recorded. Electromyography (EMG). Small metal discs will be taped to the skin. They will measure electrical activity of muscle. Participants will flex and extend each joint (wrist, ankle, or knee) on one side of their body. These movements will be done on their own and while assisted by two devices: Functional electrical stimulation (FES). Small adhesive pads will be placed on the skin and electric. Pulses will stimulate muscles to help move the limb. This will be done for the wrist, ankle and knee. Robotic Exoskeleton. A leg brace will be placed on one limb with a motor that will help move the knee. The exoskeleton can be used with or without FES. Participants will also walk on a treadmill at their own pace. Photographs and videos will record how they move.

Efficacy of a Physical Therapy Intervention Targeting Sitting and Reaching for Young Children With Cerebral Palsy

The purpose of the proposed project is to compare the efficacy of two fully developed physical therapy interventions in 8-24 months olds with or at high risk of having Cerebral Palsy (CP). Sitting Together And Reaching To Play (START-Play) targets sitting, reaching and motor-based problem solving in infancy to improve global development. Usual Care Physical Therapy (UCPT) focuses on advancing motor skills and preventing impairments.

Effects of an HABIT-ILE-Based Intervention in Children With Cerebral Palsy

This interventional study will evaluate the effects of an intensive HABIT-ILE-based camp on cardiorespiratory measures and body composition in children with cerebral palsy. Although intensive motor control-based therapies such as HABIT/HABIT-ILE have shown functional benefits, the novelty of this study is the assessment of energy expenditure during functional tasks and post-intervention changes in body composition. Participants will attend a summer camp-format intervention delivering 90 hours of upper and lower extremity training over 15 days (6 hours/day), supervised by trained rehabilitation staff in a playful, progressive group setting. The program includes bimanual activities, gait and stair tasks, functional strengthening, and whole-body games, with fatigue prevention strategies.Assessments will be performed at baseline and immediately after the camp in July 2027, with follow-up at 6 months (January 2028). Outcomes include functional performance, parent-reported function, low-intensity task-related energy expenditure measured in a physiology laboratory, and body composition measured by DEXA at Universidad Europea de Madrid

Cerebral Palsy and the Study of Brain Activity During Motor Tasks

Background: \- Two ways to study the brain while people are moving are near-infrared spectroscopy (NIRS) and electroencephalography (EEG). NIRS uses light to look at blood flow in the brain when it is active. EEG records electrical activity in the brain. Both have been used safely for many years, even in very young children. NIRS or EEG can be used while a person is moving to show which parts of the brain are the most active. Researchers want to use NIRS and EEG to study brain activity during movement in people with cerebral palsy and healthy volunteers. Learning more about how people with and without cerebral palsy use their brain to control their muscles may lead to new ways of training people with cerebral palsy to move better. Objectives: \- To study how the brain controls body movement in people with and without cerebral palsy. Eligibility: * Individuals at least 5 years of age who have cerebral palsy. * Healthy volunteers at least 5 years of age. Design: * This study has three parts. People with cerebral palsy will be selected for all three. Healthy volunteers will be asked to do only two of them. Everyone who participates will have NIRS and/or EEG exams during movement. People with cerebral palsy may also have biofeedback sessions to train coordination of movement and brain activity. * Participants will be screened with a physical exam and medical history. Urine samples may be collected. * All participants will have at least one session of NIRS and/or EEG imaging studies. Sessions may also include the following tests: * Magnetic resonance imaging to look at the brain * Electromyography to measure electrical activity of the muscles * Motion analysis of specific body parts * Ultrasound to measure activity of the muscles * Motorized, robotic, and electrical stimulation of the muscles * Other clinical tests of muscle movement as needed. * Participants with cerebral palsy will have biofeedback sessions. These sessions will help them learn to coordinate muscle movement and brain activity.

Healing Tiny Minds: Rural Recovery Through Virtual and Lived Experience Care

The goal of this behavioral-interventional study is to learn if the Abecedarian Approach implemented virtually for children ages 0-5 with a history of Hypoxic Ischaemic Encephalopathy (HIE) and/or premature birth produces the same effects as when administered at in-person facilities.

Em-power: Maximizing Functional Independence for Children With Severe Cerebral Palsy

For children with severe cerebral palsy, a powered wheelchair (PWC) may provide their only option for functional mobility and independence. PWC use is often restricted to a small percentage of children who can quickly demonstrate proficient PWC skills within a single 30-minute PWC trial. This 2-arm, parallel group, single blinded, pre-test-post-test randomized controlled trial will test our hypothesis that an 8-week PWC skills training intervention will produce greater improvements in children's PWC skills capacity immediately after the intervention and at an 8-week follow-up assessment.

Metrological Properties of a Digital Motion Analysis Research Application for Assessing the Motor Abilities and Performance of Children With Cerebral Palsy

Cerebral palsy is a common neurological disorder that is the leading cause of motor disability in children. It causes significant motor impairments, affecting various aspects such as fine and gross motor skills, as well as locomotion, which has a major impact on the quality of life of affected children. It is therefore essential to characterize these functional impairments in order to guide clinical decisions and maximize the motor function of affected children. Currently, the assessment of motor function is based on physical examinations and functional scales, such as the Gross Motor Function Classification System, which is specifically designed to assess children with CP. However, movement analysis using optoelectronic systems is considered the gold standard for this assessment, even though these systems have notable limitations (expensive installation, time-consuming use, and use only in a clinical environment, making it difficult to assess motor skills in children's everyday lives). In order to establish a comprehensive and personalized functional diagnosis, it is crucial to evaluate other fundamental motor tasks such as running, jumping, object control, and balance tasks. Recently, innovative markerless motion analysis technologies have been developed that allow analysis using smartphones or tablets outside of healthcare facilities. Therefore, developing an application dedicated to motion analysis for children with cerebral palsy that is easy to access and can be used frequently could yield many significant benefits.

Analysis of the Psychometric Properties of Kinematic Parameters of Locomotion Measured by Inertial Measurement Units. Validation in Healthy Children and Children With Cerebral Palsy

Cerebral palsy (CP) is currently one of the major causes of disability in children. The presence of various disorders (muscle stiffness, architectural bone defects, spasticity) leads to a number of functional impacts, including severe impairment of mobility, particularly locomotion. Locomotion can be assessed using a motion capture system that enables 3-dimensional analysis, in order to help make treatment decisions and quantify them. Although these systems are currently considered to be the gold standard, the fact remains that they cause a certain amount of patient fatigue (long set-up times) and that walking is assessed in a laboratory rather than in real life. Today, technological advances have brought to the fore other gait analysis devices, such as inertial measurement units (IMUs). Various systems incorporating IMUs in the feet, for example, respond to these problems of analysing walking in real-life situations. The IMUs record the movements and orientation of the foot in space; the data is then processed by algorithms to recognise walking steps and calculate the spatio-temporal parameters of locomotion. Additional IMUs positioned on the body can be grafted onto this system to provide a more precise analysis of locomotion, in particular by calculating the movements of the various joints of the lower limb. However, before such devices can be used in a pathological paediatric population, they must be validated in a healthy population. This validation must be conducted using a precise method that has been widely documented in the COSMIN recommendations (Consensus-based Standards for the selection of health Measurement Instruments). The first stage will assess the safety of the IMU devices in a healthy paediatric population, and the validity of the spatio-temporal parameters. If these properties are deemed to be compliant, these same parameters will be assessed in a paediatric population with cerebral palsy in the second stage.

Feasibility and Reliability of the Melbourne Assessment-2 (MA-2) for Telehealth

The goals of this study: 1. Determine if a play based test of arm and hand movements is valid and reliable when conducted through telehealth for children with hemiplegic cerebral palsy. 2. Measure differences in parent, provider and child engagement when an assessment is conducted in-person compared to via telehealth. 3. Rate caregivers' overall impressions of procedures when an assessment is conducted in-person compared to telehealth. Participants will attend two visits, one in person and one through telehealth. During each visit, the child will play with common toys. The sessions will be video recorded and scored using two standardized assessments, the Melbourne Assessment-2 (MA-2) and the Assisting Hand Assessment.

WBV and Serial Casting for Children With Cerebral Palsy

Children with cerebral palsy have increased muscle tone which often results in decreased active and passive movement at the ankle. While many management strategies exist (including passive stretching, WBV, serial casting, Botox, and surgical tendon lengthening), the utilization of WBV in combination with active exercises and serial casting has not been previously explored. Serial casting is the repeated application of a fiberglass cast (such as that applied to manage stable fractures) on a scheduled (usually weekly basis) to gradually increase the range of motion at a specific joint. It is theorized that combining previously established standard of care practices may result in more rapid clinical change, and hence reduce the need for prolonged intervention; ultimately reducing healthcare costs.

Intranasal AAV9-PHP.eB Gene Therapy in Cerebral Palsy (CP) & Hypoxic Ischemic Encephalopathy (HIE)

This international study, organized by Healing Hope International, is an observational registry designed to collect real-world data on participants living with chronic hypoxic ischemic encephalopathy (HIE) who receive an emerging intranasal gene therapy based on the AAV9-PHP.eB viral vector. The investigational therapy delivers a panel of 15 restorative genes that support brain repair, reduce inflammation, promote myelination, and improve neural communication. It is administered intranasally in one or three sessions by participating international clinical teams. Because the therapy is already being offered abroad, this registry does not assign treatment but instead follows participants who have received it as part of their existing medical care. The GEN HOPE Study aims to understand how this gene therapy affects movement, cognition, spasticity, and seizure frequency over time. Families and clinicians will share outcomes such as changes in gross motor function (GMFM-66/88), cognitive assessments (Bayley or WISC tests), and quality-of-life measures. Information on safety, laboratory results, MRI findings, and caregiver-reported experiences will also be collected. By combining data from multiple countries, the registry seeks to evaluate whether this novel gene based approach can meaningfully improve daily function and comfort for participants with chronic HIE. Results will guide future clinical trial development and help define safe and effective standards of care for regenerative neurologic therapies.

Intensive Somatosensory Camp for Manual Function and Participation in Children With Unilateral Cerebral Palsy

This study will evaluate whether an intensive day camp that combines somatosensory and motor training for the affected upper limb improves hand function and participation in children with unilateral cerebral palsy more than an intensive motor-only camp of equal duration and intensity. Children aged 5 to 15 years with unilateral spastic cerebral palsy and Manual Ability Classification System (MACS) levels I-III will be randomly assigned to one of two intervention groups. The experimental group will receive an 8-day, 40-hour camp including structured tactile, proprioceptive and vestibular stimulation integrated into meaningful functional tasks for the upper limb. The control group will receive an 8-day, 40-hour camp focused exclusively on intensive motor training without specific somatosensory stimulation. The primary outcome is the change in spontaneous bimanual performance of the affected hand, measured with the Assisting Hand Assessment (AHA). Secondary outcomes include measures of unilateral upper limb function, somatosensory function, hand grip strength, gross motor function, balance, trunk control, participation in daily activities and objective upper limb activity measured with wrist-worn accelerometers. Outcomes will be assessed at baseline, immediately after the intervention, and at follow-up visits approximately 2 and 3 months after the camp.

A Multicenter Pediatric Deep Brain Stimulation Registry

There is limited data on outcomes for children who have undergone deep brain stimulation (DBS) for movement disorders, and individual centers performing this surgery often lack sufficient cases to power research studies adequately. This study aims to develop a multicenter pediatric DBS registry that allows multiple sites to share clinical pediatric DBS data. The primary goals are to enable large-scale, well-powered analyses of the safety and efficacy of DBS in the pediatric population and to further explore and refine DBS as a therapeutic option for children with dystonia and other hyperkinetic movement disorders. Given the current scarcity of evidence available to clinicians, this centralized multicenter repository of clinical data is critical for addressing key research questions and improving clinical practice for pediatric DBS.

Effects of Treadmill Training and Whole-body Vibration in Children With Cerebral Palsy

Whole-body vibration (WBV) and treadmill training (TT) are commonly-utilized rehabilitation interventions for children with neuromotor disorders. WBV has been shown in the literature to positively affect gait and lower body spasticity in this population. However, the effects of a single session of WBV are generally transient, lasting between ten minutes and two hours. Thus, it may be necessary to combine WBV with another intervention to reinforce improved movement patterns and maximize its potential benefits. Therefore, the aim of this study is to investigate the effects of the addition of a single bout of WBV to a single bout of TT on the lower extremity spasticity and gait parameters of ambulatory children with CP.

Constipation in Children With Cerebral Palsy

Constipation is a common problem in children with cerebral palsy and may negatively affect daily activities, quality of life, and family well-being. Several factors such as nutrition, physical activity level, functional status, and psychological condition of the caregiver may be associated with constipation in this population. The aim of this study is to investigate the presence and severity of constipation in children with cerebral palsy and to examine the factors that may be related to constipation, including dietary intake, fluid consumption, physical activity level, functional status, and caregiver psychological status. This study is an observational cross-sectional study including children with cerebral palsy aged 4-18 years and their caregivers. Data will be collected using questionnaires, clinical classification systems, bowel diaries, nutrition records, and psychological scales. No intervention will be applied to participants.

Paired Non-invasive VNS in Adolescent Cerebral Palsy Rehabilitation

This is an open-label safety and feasibility trial evaluating the use of paired non-invasive vagus nerve stimulation (VNS) and upper extremity (UE) rehabilitation in the motor impairment reduction of adolescent patients with spastic cerebral palsy.

Coaching, Learning, Empowerment in Cerebral Palsy - a 6-Week Integrated PT/OT Caregiver "Primer" Bootcamp for Infants Diagnosed With or at High Risk for Cerebral Palsy

This study focuses on the caregiver's knowledge, comfort, and confidence in interacting with and supporting their infant with Cerebral Palsy (CP) or at high risk for Cerebral Palsy. Four caregivers with their infants will be in one cohort to assess the benefits of group intervention. The intervention is delivered in a group, bootcamp-style educational format led by clinical team including pediatric physiatrist, physical therapist and occupational therapist.

The Effect of Targeting the Plantaris Muscle-tendon in Surgical Correction of Ankle Equinus in Children

Tight ankle muscles can produce ankle equinus (limited ability to pull the foot upward) and occur often in children, significantly impacting their ability to walk. If not treated, children with ankle equinus frequently experience reduced function and long-term foot problems, such as pain. Currently, treatment options include surgery or Botulinum toxin (BoNTA) injection into the large calf muscles that point the foot downwards, aiming to reduce their tightness. However, these treatments can be less effective over time, can create prolonged calf weakness, and may require long-term bracing. Another small muscle in the leg, the plantaris, is believed to have some contribution to equinus in many children. It is sometimes included in treatment plans for equinus but its contribution is poorly understood. It is unclear whether targeting the plantaris alone could lead to better treatment of ankle equinus. Understanding the effect of treatments targeting the plantaris could help clinicians improve the management of ankle equinus. In this study, the investigators will look at the impact of surgical treatment to the plantaris in ankle equinus. The investigators hypothesize that the plantaris is a significant contributor to equinus. In this study, data will be collected from children undergoing surgical correction of ankle equinus, including lengthening of the plantaris and lengthening of the larger muscles producing equinus (the gastrocsoleus mechanism). Children will be randomly assigned to have either their plantaris or the gastrocsoleus lengthening be done first during surgery. All children will have both structures lengthened during surgery, only the order will be varied and all surgical procedures for each patient will be completed in a single setting. In both groups, maximum passive ankle dorsiflexion (upwards bend of the ankle with the knee straight) will be measured before and after each structure is lengthened. The outcome is maximum passive ankle dorsiflexion (upwards bend of the ankle) with the knee straight. The investigators expect that maximum passive ankle dorsiflexion will increase after lengthening of the plantaris. Understanding the contribution of the plantaris muscle in ankle equinus could lead to significant improvements in the treatment of children with tight ankles.

Augmenting Ankle Plantarflexor Function in Cerebral Palsy

The first specific aim is to quantify improvement in ankle muscle function and functional mobility following targeted ankle resistance gait training in ambulatory children with cerebral palsy (CP). The primary hypothesis for the first aim is that targeted ankle resistance training will produce larger improvements in lower-extremity motor control, gait mechanics, and clinical measures of mobility assessed four- and twelve-weeks post intervention compared to standard physical therapy and standard gait training. The second specific aim is to determine the efficacy of adaptive ankle assistance to improve capacity and performance during sustained, high-intensity, and challenging tasks in ambulatory children with CP. The primary hypothesis for the second aim is that adaptive ankle assistance will result in significantly greater capacity and performance during the six-minute-walk-test and graded treadmill and stair stepping protocols compared to walking with ankle foot orthoses and walking with just shoes.

Aquatic Therapy for Children With Neuromotor Deficits

The purpose of this study is to explore the feasibility, fidelity and acceptability of an aquatic therapy assessment and intervention for children ages 3-9 with neuromotor deficits such as cerebral palsy. The intervention takes place in a warm water therapy pool, twice a week for ten weeks and targets swim safety skills, upper extremity function and self care participation and performance.

Evaluation of the Transitional and Lifelong Care Program

The population cared for in the Transitional and Lifelong Care (TLC) clinic is youth and adults with childhood-onset disability, of which the large majority are adults with brain-based, neurodevelopmental conditions such as cerebral palsy, spina bifida and developmental disability. The TLC program was created to address the health inequities that have long existed for this population because of the gaps in care they experience once they transition from pediatric healthcare services to the adult healthcare sector. The TLC program offers coordinated and comprehensive management of co-occurring mental, social and physical health conditions for this group of adults with neurodiverse conditions. The proposed study will provide much needed evaluation of the TLC model as an intervention to provide transitional and lifelong care that reduces the barriers experienced because of the undefined clinic path - potentially more appropriately referred to as a "cliff" by a Freeman et al., (2015) - for these individuals. With appropriate evidence of effectiveness, scaling of the TLC program to other Ontario Health regions and more widely across Canada would improve access healthcare providers who are knowledgeable and competent in the management of physical and mental health conditions for adults with neurodiverse conditions as well as service integration and coordination between social and health sectors. The TLC clinic was co-designed with adults with neurodiverse conditions and health care providers in 2014 and represents a significant and sustainable change in the way healthcare has been delivered for this population in the Ontario Health West region over the last 7 years. More than 750 people have accessed coordinated and comprehensive care from Physiatrists, a Nurse Practitioner, Social Worker, Physiotherapist, Occupational Therapist, Speech Language Pathologist, Registered Dietitian and Rehabilitation Therapist in the TLC program since it began, documenting the effectiveness of this care has the power to re-shape care received for adults with neurodiverse conditions that onset in childhood in Canada.

The EXOPULSE Study - a Database for Routine Follow-up of Clinical Outcomes

The objective is to explore the potential short and long-term impact of the EXOPULSE Mollii Suit and EXOPULSE Suit on subjects with CP, MS, stroke, fibromyalgia or other neurologic disorders which may cause such types of symptoms, and to identify high responders among the sub-categories of the diagnoses. The primary endpoint will be improvements in the Berg/Pediatric Balance Scale (BBS/PBS) as a measurement of balance and risk of falls.