Clinical Research Directory

Sleep Quality and Caregiver Burden in Children With Cerebral Palsy

Cerebral palsy (CP) is a lifelong condition affecting movement and posture, often requiring substantial care from family members. Children with CP frequently experience sleep disturbances associated with motor impairment, pain, seizures, and other clinical factors. Sleep difficulties may also be associated with poor sleep quality, psychological symptoms, and increased burden among caregivers. This single-center, observational, cross-sectional study evaluated sleep characteristics in children with CP, caregiver sleep quality, and caregiver burden, as well as their associations with functional status and caregiver psychological symptoms. The study included 61 children with CP and their primary caregivers. Eligible children were aged 2-18 years. Children's sleep characteristics were assessed using a 33-item parent-reported questionnaire adapted from the Children's Sleep Habits Questionnaire (CSHQ), which was not identical to the validated Turkish CSHQ. Children's functional characteristics were evaluated using GMFCS, MACS, and CFCS. Caregiver sleep quality, caregiver burden, and psychological symptoms were assessed using the Pittsburgh Sleep Quality Index (PSQI), Zarit Burden Interview (ZBI), and Hospital Anxiety and Depression Scale (HADS), respectively. The study was designed to examine associations among child sleep characteristics, caregiver sleep quality, caregiver burden, and psychological well-being.

Feasibility of Vision-Language Models for Detecting Physical Assistance as an Observable Criterion Defining GMFCS Levels in Children Aged 2 to 6 Years With Cerebral Palsy

The GMFCS sorts children with cerebral palsy into five levels of gross motor function. What mainly separates one level from the next is two things an observer can see: whether another person has to physically help the child move, and whether the child has to bear weight on something external, such as a walker, a support stand, or furniture. Scoring these takes an experienced clinician, and it is hardest between the ages of 2 and 6. This study asks a narrow question. Can a vision-language model, meaning an artificial-intelligence model that looks at images and answers questions about them, detect those two things from short video clips of a child moving? Twenty-six children with cerebral palsy aged 2 to 6 years were filmed at two hospitals. Each child performed everyday movements, including walking, crawling, rolling sideways, rising from the floor to standing, and lowering back to the floor, and up to three cameras recorded every attempt at the same time. Sixteen still frames were taken from each recorded attempt, always sixteen, and shown to the model without the child's identity, the name of the movement, or the child's GMFCS level. The model answers two yes-or-no questions and nothing else: did an adult touch the child, and did the child bear weight on an external object. A fixed rule, not the model, turns those answers into one determination per attempt, namely whether the child performed the movement unaided. These determinations are compared with what a human annotator recorded for the same clips while blind to clinical information. All rating is done with open-weight models running on hardware the investigators control, and every reported figure is tied to the specific model version that produced it. The study does not assign a GMFCS level, and it is not built to. It tests whether the two observations the GMFCS itself relies on can be read from video reliably. If they can, they can be placed in front of a clinician as evidence, which is where later work on supporting GMFCS assessment would start.

Changes in Land-Based Motor Function After Routine Hydrotherapy in Children With Cerebral Palsy

This retrospective observational study aims to describe changes in land-based motor function recorded before and after routine hydrotherapy among children with cerebral palsy. Existing clinical records from a special education and rehabilitation center in Safranbolu, Türkiye, covering September 1, 2021 through August 31, 2026, will be reviewed after the required ethics approval and institutional authorization. The study will include children younger than 18 years at the start of a documented hydrotherapy course who have comparable assessments using the Gross Motor Function Measure-66 (GMFM-66) before and after that course. No new intervention, examination, or participant contact is planned for research purposes. Changes in motor performance and their associations with age, Gross Motor Function Classification System level, baseline motor function, and recorded hydrotherapy dose will be evaluated when data permit. As this is an uncontrolled retrospective study, observed changes will not be interpreted as the causal effect of hydrotherapy alone. Approximately 45 eligible children are expected. Record abstraction is planned to begin on November 1, 2026; completion is estimated for April 30, 2027.

Short-Burst Interval Treadmill Training Cerebral Palsy

Ambulatory children with cerebral palsy (CP) walk predominately in low intensity stride rates with little variability, thus limiting their walking activity and ability to participate in daily life. In contrast, typically developing (TD) children engage in short bursts of intense walking activity interspersed with varying intervals of low intensity walking within daily life. In order to optimize motor learning, active participation, task-specific training and multiple repetitions or massed practice is required to learn new motor skills. Short bursts of vigorous intensity locomotor treadmill training (SBLTT) alternating with low/moderate intensity was specifically designed to mimic activity patterns of TD children in a massed practice format. Pilot data suggests that SBLTT is feasible and enhances walking capacity and performance in daily life for children with CP. This project will examine the effect of SBLTT versus an equivalent dosage of traditional locomotor treadmill training (TLTT) on the primary outcomes of walking capacity and performance in children with CP and whether the effects of SBLTT on walking capacity and performance are mediated by improvements in in muscle power generation. The scientific premise is that SBLTT, that approximates the walking intensity patterns of typically developing (TD) children through a home-based massed practice protocol, will be more effective than TLTT in improving walking capacity and performance. We hypothesize that SBLTT strategies for children with CP modeled on walking patterns of TD children, will be positively mediated by muscle power generation and subsequently improve walking capacity and community walking performance and mobility. Specific aims. Aim #1. Determine the immediate and retention effects of short-burst interval LTT (SBLTT) on walking capacity in ambulatory children with CP. Aim #2. Examine the effects of treatment on community-based walking activity performance and mobility. Aim #3. Explore whether the effects of SBLTT on walking capacity and performance are mediated by muscle power generation. The proposed research will be the first step in a continuum of research that is expected to direct locomotor training protocols and rehab strategies across pediatric disabilities and positively effecting the community walking performance and mobility for children with CP.

Combined Popliteal and Adductor Canal Blocks for Postoperative Analgesia in Children With Cerebral Palsy

This prospective, randomized, controlled trial aims to evaluate the postoperative analgesic efficacy of combined ultrasound-guided popliteal sciatic nerve block and adductor canal block in children with cerebral palsy undergoing elective lower extremity surgery. Children aged 3-18 years with American Society of Anesthesiologists (ASA) physical status II-III will be randomized into two groups. Participants in the intervention group will receive combined popliteal sciatic nerve block and adductor canal block in addition to general anesthesia, whereas participants in the control group will receive general anesthesia with intravenous analgesia without peripheral nerve block. Postoperative pain will be assessed using the revised Face, Legs, Activity, Cry, Consolability (r-FLACC) scale at 1, 2, 6, 12, and 24 hours after surgery by an anesthesiologist blinded to group allocation.

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.

Functional Progressive Resistive Training and Cerebral Palsy

Functional Progressive Resistive Exercise (FPRE) is a strengthening approach that combines functional movement activities with progressively increased resistance. It involves performing task-oriented exercises, such as sit-to-stand, stair climbing, and walking-related activities, while gradually increasing the load to enhance muscle strength, endurance, and functional performance. FPRE is widely used in rehabilitation programs to improve mobility and the ability to perform activities of daily living in various populations, including children with neurological and musculoskeletal disorders.

Turkish Translation, Cross-Cultural Adaptation, Validity, and Reliability of the ABILHAND-Kids Young CP Scale

The purpose of this study is to perform the Turkish translation, cross-cultural adaptation, validity, and reliability of the ABILHAND-Kids Young CP scale. This methodological and cross-sectional study aims to provide a culturally adapted and psychometrically sound assessment tool to evaluate manual ability in children aged 2 to 7 years with cerebral palsy, based on parent or primary caregiver reports.

Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders

Background: People with cerebral palsy, spina bifida, muscular dystrophy, or spinal cord injury often have muscle weakness and problems controlling how their legs move. This can affect how they walk. The NIH has designed a robotic device (exoskeleton) that can be worn on the legs while walking. The wearable robot offers a new form of gait training. Objective: To learn whether a robotic device worn on the legs can improve walking ability in those with a gait disorder. Eligibility: People aged 3 to 17 years with a gait disorder involving the knee joint. Design: Participants will be screened. They will have a physical exam. Their walking ability will be tested. Participants will have markers taped on their body; they will walk while cameras record their movements. They will undergo other tests of their motor function and muscle strength. The study will be split into three 12-week phases. During 1 phase, participants will continue with their standard therapy. During another phase, participants will work with the exoskeleton in a lab setting. Their legs will be scanned to create an exoskeleton with a customized fit. The exoskeleton operates in different modes: in exercise mode, it applies force that makes it difficult to take steps; in assistance mode, it applies force meant to aid walking; in combination mode, it alternates between these two approaches. During the third phase, participants may take the exoskeleton home. They will walk in the device at least 1 hour per day, 5 days per week, for 12 weeks. Participants walking ability will be retested after each phase....

Functional Benefits of Multisite Upper Limb Surgery in Children With Unilateral Cerebral Palsy (RETRO-CHIRc)

This retrospective study evaluates the functional impact of individualized multisite neuro-orthopedic upper limb surgery in children with unilateral cerebral palsy (hemiplegic CP). Using a before-and-after design in which each patient serves as their own control, the study analyzes data collected in routine clinical care up to 3-5 years before and 1-2 years after surgery. The primary outcome is upper limb function as measured by the Modified Melbourne Assessment (MA2), which assesses range of motion and dexterity. Secondary outcomes include unimanual precision and fluidity (MA2), global manual dexterity (Box and Block Test), bimanual performance (Assisting Hand Assessment), and patient/parent satisfaction. Approximately 70 patients operated at the Centre Médico-Chirurgical de Réadaptation des Massues between January 2012 and June 2025 will be included. The study also aims to identify factors influencing surgical outcomes, such as age at surgery, associated cognitive or sensory impairments, and adherence to post-operative orthotic protocols.

Evaluation of Effectiveness of Child-oriented Goal-setting in Paediatric Rehabilitation (the ENGAGE Approach)

Children with disabilities often access rehabilitation services to improve their abilities to participate in everyday activities. Goal-directed therapy is considered an important therapeutic strategy to achieve outcomes that are meaningful to families. Not a lot is known about the effects of goal setting on rehabilitation outcomes. Strategies to help children participate in the goal-setting process are rarely used in clinical practice. The aim of this project is to test the effects of a child-focussed goal setting approach, Enhancing Child Engagement in Goal Setting (ENGAGE), on therapy outcomes. Service use and the cost vs. benefits of the ENGAGE approach compared to usual practice will also be examined. Children with neurodevelopmental disabilities aged 5-12 years old (n=96) who access paediatric rehabilitation services at six rehabilitation sites will participate. Therapists (n=24) at participating sites in Alberta, Canada will be randomized into 1) the ENGAGE intervention group or 2) the usual therapy practice control group. Children will participate in the ENGAGE approach to goal setting or usual practice based on the allocation of their therapist. This study will determine if the ENGAGE approach to goal setting affects child goal performance, satisfaction with goal performance, functional abilities, participation, and parent and child quality of life. The investigators will also evaluate differences in parent and child quality of life in relation to parent costs (e.g., absenteeism, presenteeism, travel costs) and compare amount of therapy time between the two groups to see which approach is more cost-effective and efficient. After the study, children, parents and therapists will be asked to discuss aspects that influenced effective implementation of the ENGAGE approach. This study could provide evidence to improve meaningful child and family outcomes in paediatric rehabilitation and improve efficiency of paediatric rehabilitation services.

Repetitive Transcranial Magnetic Stimulation on Motor Function and Meta-Plasticity in Cerebral Palsy: TMS-EEG Study

This project examines the use of repetitive transcranial magnetic stimulation (rTMS) as a therapeutic approach to improve motor function in children with cerebral palsy (CP). By applying 6-Hz primed low and high-frequency rTMS and measuring brain responses through TMS-EEG, the study aims to enhance neural plasticity and motor recovery. The goal is to promote faster rehabilitation and reduce long-term healthcare needs.

Expanded Access Protocol: Umbilical Cord Blood Infusions for Children With Brain Injuries

This protocol is designed to enable access to intravenous infusions of banked umbilical cord blood (CB), that is thawed and not more than minimally manipulated, for children with various brain disorders. Children with cerebral palsy, congenital hydrocephalus, apraxia, stroke, hypoxic brain injury and related conditions will be eligible if they have normal immune function and do not qualify for, have previously participated in, or are unable to participate in an active cell therapy clinical trial at Duke Medicine. For the purpose of this protocol the term children refers to patients less than 26 years of age. Cord blood is administered as a cellular infusion without prior treatment with chemotherapy or immunosuppression. The mechanism of action is through paracrine signaling of cord blood monocytes inducing endogenous cells to repair existing damage.

Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cell Intravenous Infusion for CP

This trial will study the safety and efficacy of intravenous infusion of cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cells for the treatment of Cerebral Palsy

Knotless Versus Conventional Sutures for Wound Closure in Cerebral Palsy Multilevel Surgery

This study compares two standard ways of closing surgical incisions in children and adults with cerebral palsy who are having multilevel surgery on both legs (single-event multilevel surgery, or SEMLS). Each participant serves as their own control: all study incisions on one leg, chosen at random, are closed with knotless barbed sutures, and the matching incisions on the other leg are closed with conventional knotted sutures. All sutures used have an antibacterial (triclosan) coating. The main question is whether knotless barbed sutures shorten the time needed to close each incision. The study also compares wound complications, such as infection and wound opening, and scar appearance, rated from photographs by masked reviewers and by patients or caregivers, over the 6 months after surgery.

Nutritional Assessment in Children With Cerebral Palsy

This cross-sectional study aims to assess the current practice of nutritional assessment and management in children with cerebral palsy and to determine the frequency of undernutrition among them.

Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders

Background: \- Cerebral palsy (CP) is the most common motor disorder in children. CP often causes crouch gait, an abnormal way of walking. Knee crouch has many causes, so no single device or approach works best for everybody. This study s adjustable brace provides many types of walking assistance. Researchers will evaluate brace options to find the best solution for each participant, and whether one solution works best for the group. Objective: \- To evaluate a new brace to improve crouch gait in children with CP. Eligibility: * Children 5 17 years old with CP. * Healthy volunteers 5 17 years old. Design: * All participants will be screened with medical history and physical exam. * Healthy volunteers will have 1 visit. They will do motion analysis, EMG, and EEG described below. * Participants with CP will have 6 visits. * Visit 1: \<TAB\>1. Motion analysis: Balls will be taped to participants skin. This helps cameras follow their movement. \<TAB\>2. EMG: Metal discs will be taped to participants skin. They measure electrical muscle activity. \<TAB\>3. Participants knee movement will be tested. \<TAB\>4. Participants will walk 50 meters. \<TAB\>5. Participants legs will be cast to make custom braces. * Visit 2: * Participants will wear their new braces and have them adjusted. * Steps 1 3 will be repeated. * EEG: Small metal discs will be placed on the participants scalp. They record brain waves. * Participants will have electrical stimulation of their knees and practice extending them. * Participants will take several walks with the braces in different settings. * Visits 3 5: participants will repeat the walking and some other steps from visit 2. * Visit 6 will repeat visit 2.

Robot Asissted Training on Neurodevelopmental Alterations

The aim of this randomized crossover clinical trial is to determine the effect of robot assisted gait training combined with physiotherapy on body composition and functional variables in a pediatric population with neurodevelopmental disorders.The main questions to answer are: * Does this treatment improve body composition parameters? * Does the treatment help maintaining functionality and without causing discomfort or pain? Participants will perform 2 weekly physiotherapy sessions for 8 weeks, then there will be 4 weeks of washout, afer which each patient will cross over to the other group for 8 more weeks.

Safety of Cultured Umbilical or Placental Derived Mesenchymal Stem Cell Intravenous Infusion for CP

This trial will study the safety and efficacy of intravenous infusion of cultured umbilical or placental derived mesenchymal stem cells for the treatment of Cerebral Palsy

The Impact of Community-based Wellness Programs on The Triple Aim

People with disabilities experience a staggering incidence of secondary conditions that can result in death or negatively impact their health, participation in the community, and quality of life. Many of these chronic secondary conditions are preventable. The Institute for Healthcare Improvement has advocated for optimizing care through programs that simultaneously improve health and the patient experience of care, while reducing cost, called the "Triple Aim." Studies have shown that the Triple Aim can be achieved through programs that facilitate community integration; however the U.S. healthcare system lacks a paradigm of care for individuals with disabilities that promotes community integration. In order to identify potential models of healthcare delivery for individuals with disabilities that are effective in achieving the Triple Aim, we will conduct a rigorous research project to evaluate the impact of two different models of care on the Triple Aim: 1) a community-based care management program delivered by a non-profit organization through waiver funds, and 2) the Program for All-inclusive Care for the Elderly (PACE) applied to younger individuals with disabilities between ages 55-64.

Dynamic, Real-world Learning, Innovative Mobility, and Versatile Exploration (DRIVE): Evaluating a Dose-matched Developmental Powered Mobility Intervention for Young Children With Cerebral Palsy

The goal of this clinical trial is to understand the effects of developmental powered mobility intervention on global development, gross motor function, and activity performance outcomes in 48 young children (ages 1-2 years) with cerebral palsy (CP). The main question it aims to answer is: Does the developmental powered mobility intervention produce greater improvements in global development and activity performance outcomes in young children with CP? How will the developmental powered mobility intervention effect gross motor function? Researchers will compare the developmental powered mobility (DPM) intervention to usual care physical therapy focused on movement, orientation, repetition, and exercise (MORE-PT) to see if DPM produces greater improvements in outcomes without impeding gross motor function. Child Participants will: Receive either DPM or MORE-PT twice weekly for 12 weeks in the home setting. Complete assessments with the interventionist before and after the 12-week intervention and at a 6-month follow-up visit. Caregiver participants will: Track outside therapies and any outside assistive device use completed during the intervention, in a weekly log.

The Effects of Protocolized Breathing Exercises on Pulmonary Function in Adolescents With Cerebral Palsy.

This multicenter randomized controlled pilot trial evaluates whether an 8-week protocolized breathing exercise program improves pulmonary function and quality of life in adolescents and young adults with cerebral palsy. Participants are randomized to either a structured breathing exercise program added to usual pedagogical rehabilitation care or sham breathing exercises plus usual care. The study assesses changes in spirometric outcomes (FVC, FEV₁, PEF) and patient-reported quality of life and emotional well-being. Participants complete respiratory assessments and questionnaires at baseline, post-intervention, and follow-up.

Tranexamic Acid to Reduce Blood Loss After Varus Derotation Osteotomy

TABLO (Tranexamic Acid to reduce Blood Loss after varus derotation Osteotomy) is a clinical trial of postoperative tranexamic acid vs placebo in non-ambulatory children with cerebral palsy (CP) undergoing reconstructive hip surgery. Improving surgical outcomes is a high priority in this patient population given the high risk of bleeding and the diminished capacity for these children to withstand substantial blood loss. Preliminary data from the study institution indicates that approximately one third of these patients receive transfusion of blood products in the postoperative period. There is growing evidence that hidden blood loss occurring in the postoperative period is substantial and can potentially be attenuated with the administration of Tranexamic Acid (TXA). However, trials on postoperative TXA have been carried out exclusively in adult surgical populations.

Evaluation of the NEUROSWING Orthosis on Walking Speed in a Population With Dorsal and/or Plantar Flexor Deficits, With a View to a Reimbursement Application

The aim of this randomized, cross-over, multi-center, intention-to-treat, open-label study study is to determine the benefits of the NEUROSWING device on gait speed in patients with dorsal and/or plantar flexor deficits, compared with the use of a carbon orthosis. Each patient will benefit in random order from a 14-day investigation period with the NEUROSWING device and a 14-day control period with a carbon orthosis. These two 2-week periods will be separated by one week, during which no orthosis will be worn and no evaluation will be carried out. Patients will be randomized to determine which phase they will start with. The primary endpoint is walking speed, which will be measured by comparing the results of the 10-meter walk test. For the secondary endpoints, patients will be assessed using the Functional Gait Assessment (FGA), the 6-minute test and an analysis system that will analyze other gait parameters (duration of the different phases of the gait cycle, cadence and step length). In order to carry out the assessments, each patient included will have 12 visits spread over 5 weeks.