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Rady Children's Specialists

Research and Clinical Trials

The Neurology division’s research focuses on a wide range of neurological disorders. Please contact our office and reference the physician involved in the study if you are interested in learning more or have questions about our research initiatives and clinical trials.

Clinical Trials


1. Momentum 1: A Multicenter, Double-blinded, Randomized, Placebo-Controlled, Parallel Group Study with Open Label Extension Phase of Lorcaserin as Adjunctive Treatment In Subjects with Dravet Syndrome. (Drs. Olivia Kim-McManusMark Nespeca)
The primary purpose of the study is to demonstrate that lorcaserin has superior efficacy compared to placebo on percent change in frequency of convulsive seizures per 28 days in participants with Dravet syndrome. Participants will be randomized to receive lorcaserin administered as an oral suspension, twice daily for 14 weeks during the core treatment period. Dose will be based on body weight as follows: target dose for participants weighing 10 to less than (<) 20, 20 to <40, and greater than or equal to (>=) 40 kilogram (kg) will be 5, 10, and 20 milligram per day (mg/day) respectively. Based on clinical response and tolerability and within 2 weeks of treatment, dose can be increased up to 10, 20 mg/day for participants weighing 10 to <20, 20 to <40 kg respectively. Participants completing the core treatment period will enter a 12-week extension phase and will receive lorcaserin.

See full study at

2. Efficacy and Safety of Vatiquinone for the Treatment of Mitochondrial Disease Subjects with Refractory Epilepsy (MIT-E) (Drs. Dr. Richard Haas, Olivia Kim-McManus)
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone.

See full study at

3. Network Analysis of Intracranial Electroencephalography (EEG) in People with Epilepsy. Dr. Olivia Kim-McManus (UC Reliance Site Principal Investigator, UC San Diego, Rady Children’s Hospital; in collaboration with Dr. Robert Knight (UC San Francisco, UC Berkeley) and Dr. Jack Lin (UC Davis, Principal Investigator).
EEG network analysis in medically refractory epilepsy patients ages 7 years and above with non-lesional brain MRIs admitted for epilepsy surgery with intracranial monitoring with stereotactic depth electrodes and/or grids.

4. An Evaluation of the Use of Noninvasive Transcranial Magnetic Stimulation (TMS) in Patients Experiencing Hemiparesis Due to Stroke, Perinatal Injury, Epilepsy Surgery or Tumor Resection (Drs. Neggy Rismanchi, Shifteh Sattar, Johnathan Bui)

Rett Syndrome

(Drs. Richard Haas, Natalie Guido-Estrada)

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women with Rett Syndrome (phase 3)

This multisite study will investigate the efficacy of treatment with oral Trofinetide versus placebo in girls and women with Rett syndrome. There are 184 subjects who are expected to be randomized (with a minimum of 12 subjects randomized for each age stratum [5-10 years old, 11-15 years old, and 16-20 years old]) with a total of 92 subjects per treatment. Subjects will receive an oral dose of Trofinetide or placebo, for up to 12 weeks. Dose will be based on weight. There is also an open-label extension of this study available.

See full study at

Duchenne Muscular Dystrophy, Spinal Muscular Atrophy 

(Dr. Chamindra Konersman)

1. Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients with Duchenne Muscular Dystrophy

A total of 213 male ambulant subjects will be randomised 2:1 (givinostat:placebo). Subjects will be stratified for their concomitant use of steroids in four strata: 1) Deflazacort daily regimen; 2) Deflazacort intermittent regimen; 3) Other steroids daily regimen; 4) Other steroids intermittent regimen. The study duration is planned for 19 months.

See full study at

2. Open-Label Extension for the Above Project

3. AVXS-101-RG-001 A Prospective Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases.Until recently, the mainstay of treatment for these patients was supportive medical care. However, advances in medical treatment focusing on gene replacement, gene enhancement, motor neuron protection and muscle enhancement is likely to change the management and prognosis of these patients in the future. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options.

See full study at

Multiple Sclerosis and Neuroinflammatory Disorders

(Dr. Jennifer Graves)

1. CHARGE (Biogen 105MS306)
This is an open-label, randomized, multicenter, active-controlled, parallel-group study to evaluate the safety, tolerability and efficacy of BIIB017 in pediatric subjects age 10 to 18 years for the treatment of relapsing/remitting multiple sclerosis.

See full study at

For any questions, please reach out to our Neuroimmunology and MS Research Program coordinator at, or see our website at

2.  Eyetracker Study
The visual system provides an opportune and elegant window into the inflammatory and degenerative aspects of MS. We can identify precise relationships between structural damage and functional outcomes that are difficult to achieve in other aspects of nervous system injury. In this study use technologically advanced eye tracking technology to analyze eye movement in MS patients and healthy controls, to learn more about the aspects of eye movement that cannot be seen with the naked eye, and how they are related to diseases of the central nervous system.

For more information please reach out to our research coordinator at, or check out our website at

3. BioAging Study
Our BioAging study investigates the association of biological aging of senescent cells and aging in multiple sclerosis patients ages 14 and up. This study aims to identify potential pathways through which relapsing forms of MS advance into progressive forms of MS. This study consists of a single blood draw, and is now actively recruiting both MS and healthy control participants.

For more information, please reach out to us at, or find our website at

Neurotransmitter Disorders

(Dr. Jennifer Friedman)

1. iNTD (“International Network on Neurotransmitter related Disorders”) Registry
Major aims of the study:

  • To describe the natural history and outcome of the six neurotransmitter disorders, five BH4 deficiencies and two cerebral folate deficiencies (long-term organ-specific complications, survival rate, genotype/phenotype correlation, differences in the disease course relating to the genetic background / ethnic origin/ gender effects)
  • To describe and evaluate the efficacy and safety of current treatment strategies
  • To compare the diagnosis, treatment and management of affected individuals in different European countries.
  • To identify the major impact of a rare inherited disease for patients and their families regarding the quality of life, school education, professional career and social life

Stroke, Tuberous Sclerosis Complex, Autism 

(Dr. Doris Trauner)

1. Perinatal Arterial Stroke: A Multisite RCT of Intensive Infant Rehabilitation (I-ACQUIRE) 

This is an NIH StrokeNet-funded study to test the effectiveness of constraint-induced movement therapy on improving hand us in toddlers with hemiplegia caused by perinatal stroke. Children between the ages of 8-24 months are randomized to one of 3 groups, with either 3 hours/day or 6 hours/day of intensive occupational therapy for 4 weeks or “usual and customary” treatment.

See full study at

2. Dose-Ranging Efficacy and Safety Study of Rapamycin: A Phase 2/3, Multicenter, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of Rapamycin for the Treatment of Facial Angiofibromas (FA) Associated with Tuberous Sclerosis Complex (TSC) in patients 6 years of age and older

Funded by AFT Pharmaceuticals

This is a double-blind study of a new formulation of rapamycin cream to treat facial adenomas associated with tuberous sclerosis. It consists of three arms, rapamycin in one of two doses or placebo. Study participants 4 years and older are included.

See full study at

3. A Double-Blind, Crossover Trial of Cannabidiol to Treat Severe Behavior Problems in Children with Autism

This is a double-blind, cross-over design trial for boys ages 7-14 years with autism and severe behavioral problems. The two treatment arms are cannabidiol (CBD) and placebo. Each child will receive both placebo and CBD in different arms. Each arm is 8 weeks long with a 4-week washout period between arms.

Neurodevelopmental Impairment

(Dr. Dillon Chen)

1. Understanding the Roles of Polysialic Acid and Sialic Acid-Ninding Immunoglobulin-Type Lectins (Siglecs) in the Developing Brain and  How they Affect Neuro-Injury and Neuro-Inflammation 

Perinatal and neonatal brain injury often leads to long-term morbidity including neurodevelopmental impairment. Understanding the effects of injury during these critical periods of brain development may result in improved morbidity and mortality for infants with brain injury. One of the most fundamental building blocks of the brain is the sialic acid and a major protein-bound sialic acid in the brain is polysialic acid (polySia). The goals of the project are to characterize and explore the functions and expression levels of polySia, Siglec-11 and Siglec-16 in the developing brain, especially in the setting of neural injury.

2. A Simple Method to Detect Neurologic Disease in Neonates

Tics and Tourette Syndrome

(Deanna J. Greene, Ph.D.)

The purpose of this study is to learn about the brain in tic disorders. The study is seeking participants ages 7-17 years with tics, as well as those without tics, and involves four to 10 MRI scan visits along with questionnaires and interviews.

Observational Studies

1. PAW
The PAW study aims to investigate physical activity and wellness in pediatric neuroimmunological diseases through the use of physical activity questionnaires. This study will continue over the coming few years and consists of a few questionnaires completed at the time of a regularly scheduled neurology visit. PAW is currently actively recruiting pediatric subjects.

2. Diet and Relapses
The Diet and Relapses study explores the connection between rate of relapse and diet through the gut microbiome and amino acids found in the digestive tract. We hope to uncover the role that 25 (OH) vitamin D levels, fat and vegetable intake, and other nutrients take in the pediatric disease course, and to investigate the biological pathways relevant to this association.

Optional: By filling out a set of questionnaires, and a small blood draw, patients can help us to further understand pediatric MS.

3. PeMSDD Database Study
PeMSDD is a database registry study linked to the Diet and Relapses study. This registry strives to describe the number and characteristics of patients with suspected early onset demyelinating disases. This database will help investigators allied with the National Pediatric MS Center to support hypothesis generation, and study design development.

The PERCEPTION study is an observational, multisite pediatric research collaboration that investigates tests in ocular neuroimmunology. This study aims to analyze current methods of ocular testing in pediatric neuroimmunology, in order to determine better physiological markers and early identifiers of neuroimmunological diseases in children, as well as to better understand the progression of these diseases. PERCEPTION is currently actively recruiting pediatric subjects.

5. NEOS:
A two-year randomized, three-arm, double-blind, non-inferiority study comparing the efficacy and safety of ofatumumab and siponimod versus fingolimod in pediatric patients with multiple sclerosis followed by an open-label extension: The main purpose of the study is to find out if ofatumumab and siponimod versus fingolimod are safe and beneficial to use in children/adolescents with MS aged 10 to 17 at the start of the study. This placebo controlled study is 2 years during the double blinded part of the study (core part) and up to 5 years in the open-label-extension part of the study. It involves MRIs, blood draws, functional testing and additional visits to monitor patients.

6. The CONNECT Registry (CONquering Neuroimmunology and Epilepsies ConsorTium)
A multicenter prospective cohort study of the clinical features and biomarker identification for autoimmune encephalitis, Landau-Kleffner Syndrome and Rasmussen encephalitis.