Epilepsy clinical trials at UCSF
24 in progress, 10 open to eligible people
ETX101 in Infants and Children with SCN1A-Positive Dravet Syndrome
open to eligible people ages 6 months to 47 months
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to <36 months (Part 1) and aged ≥6 to <48 months (Part 2). Part 1 follows an open-label, dose-escalation design, and Part 2 is a randomized, double-blind, sham delayed-treatment control, dose-selection study.
San Francisco, California and other locations
EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
open to eligible people ages 2 years and up
The purpose of this study is to evaluate the safety and efficacy of EPX-100 as adjunctive therapy in participants with Dravet syndrome.
San Francisco, California and other locations
Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome
open to eligible people ages 2-56
The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of seizures in children and adults with Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS). Participants will receive their standard anti-seizure therapy, plus tablets of soticlestat. There will be scheduled visits and follow-up phone calls throughout the study.
San Francisco, California and other locations
Pharmacodynamics Of Satralizumab In Patients With Anti-N-Methyl-D-Aspartic Acid Receptor (NMDAR) Or Anti-Leucine-Rich Glioma-Inactivated 1 (LGI1) Encephalitis
open to eligible people ages 12 years and up
The purpose of this study is to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in participants with anti-N-methyl-D-aspartic acid receptor (NMDAR) and anti-leucine-rich glioma-inactivated 1 (LGI1) encephalitis.
San Francisco, California and other locations
FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
open to eligible people ages 18-65
This clinical trial is designed to test whether a single stereotactic intracerebral administration of inhibitory nerve cells into subjects with drug-resistant mesial temporal lobe epilepsy is safe (frequency of adverse events) and effective (seizure frequency).
San Francisco, California and other locations
Medtronic Deep Brain Stimulation (DBS) Therapy for Epilepsy Post-Approval Study (EPAS)
open to eligible people ages 18 years and up
The purpose of this post-approval study is to further evaluate the long-term safety and effectiveness of Medtronic DBS therapy for epilepsy on seizure reduction in newly implanted participants through 3 years of follow-up.
San Francisco, California and other locations
Model-based Electrical Brain Stimulation
open to eligible people ages 18 years and up
Neuropsychiatric disorders are a leading cause of disability worldwide with depressive disorders being one of the most disabling among them. Also, millions of patients do not respond to current medications or psychotherapy, which makes it critical to find an alternative therapy. Applying electrical stimulation at various brain targets has shown promise but there is a critical need to improve efficacy. Given inter- and intra-subject variabilities in neuropsychiatric disorders, this study aims to enable personalizing the stimulation therapy via i) tracking a patient's own symptoms based on their neural activity, and ii) a model of how their neural activity responds to stimulation therapy. The study will develop the modeling elements needed to realize a model-based personalized closed-loop system for electrical brain stimulation to achieve this aim. The study will provide proof-of-concept demonstration in epilepsy patients who already have intracranial electroencephalography (iEEG) electrodes implanted for their standard clinical monitoring unrelated to this study, and who consent to being part of the study.
San Francisco, California and other locations
NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)
open to eligible people ages 2-21
The objective of this study is to assess the efficacy, safety, and pharmacokinetics of NBI-921352 as adjunctive therapy for seizures in subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).
San Francisco, California and other locations
CNKSR2 Natural History Study
open to eligible people ages 6-21
This prospective natural history study is being conducted to define the electroclinical, neurodevelopmental, and behavioral characteristics of CNKSR2 epilepsy aphasia syndrome (EAS) and intellectual disability (ID) in children aged 6 to 21 years old with CNKSR2 mutations. The data collected from this study will serve as an external control to eventual clinical trials examining precision medicine investigational therapeutics that aim to improve the seizure burden and neurodevelopmental outcomes in patients with CNKSR2 EAS/ID.
San Francisco, California
Neonatal Seizure Registry, GEnetics of Post-Neonatal Epilepsy
open to all eligible people
The NSR-GENE study is a longitudinal cohort study of approximately 300 parent-child trios from the Neonatal Seizure Registry and participating site outpatient clinics that aims to evaluate whether and how genes alter the risk of post-neonatal epilepsy among children with acute provoked neonatal seizures. The researchers aim to develop prediction rules to stratify neonates into low, medium, and high risk for post-neonatal epilepsy based on clinical, electroencephalogram (EEG), magnetic resonance imaging (MRI), and genetic risk factors.
San Francisco, California and other locations
STK-001 for Patients With Dravet Syndrome
Sorry, accepting new patients by invitation only
Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of STK-001 in patients with Dravet syndrome who previously participated in studies of STK-001. Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.
San Francisco, California and other locations
Fenfluramine in Children with Dravet Syndrome Under 24 Months of Age
Sorry, not yet accepting patients
Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels. Children with Dravet syndrome classically present in the first year of life with prolonged seizures, often hemiclonic and in the setting of fever or temperature changes such as getting in or out of bath water. Many anti-seizure medications are sodium channel blockers and exacerbate seizures in this patient population. This creates some limitations in medication choices for this patient population. Recently fenfluramine was approved for use in Dravet syndrome for people 2 years and older. Randomized studies demonstrated a 74.9% reduction of convulsive motor seizures compared to 19.2% in the placebo group. Additionally, 16% of children treated with fenfluramine were seizure free. Fenfluramine is likely to be as effective in children under the age of 2 years. The current study has proposed a treatment protocol to allow access to fenfluramine for children under 24 months of age.
San Francisco, California and other locations
Evaluate How Safe and Tolerable NBI-921352 is as an Adjunctive Therapy for Participants With SCN8A-DEE
Sorry, accepting new patients by invitation only
Extension study to evaluate how safe and tolerable the drug NBI-921352 is when used as adjunctive therapy in participants with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE).
San Francisco, California and other locations
Functional Organization of the Superior Temporal Gyrus for Speech Perception
Sorry, accepting new patients by invitation only
The basic mechanisms underlying comprehension of spoken language are still largely unknown. Over the past decade, the study team has gained new insights to how the human brain extracts the most fundamental linguistic elements (consonants and vowels) from a complex and highly variable acoustic signal. However, the next set of questions await pertaining to the sequencing of those auditory elements and how they are integrated with other features, such as, the amplitude envelope of speech. Further investigation of the cortical representation of speech sounds can likely shed light on these fundamental questions. Previous research has implicated the superior temporal cortex in the processing of speech sounds, but little is known about how these sounds are linked together into the perceptual experience of words and continuous speech. The overall goal is to determine how the brain extracts linguistic elements from a complex acoustic speech signal towards better understanding and remediating human language disorders.
San Francisco, California
Neural Mechanisms for Stopping Ongoing Speech Production
Sorry, accepting new patients by invitation only
Speech and communication disorders often result in aberrant control of the timing of speech production, such as making improper stops at places where they should not be. During normal speech, the ability to stop when necessary is important for maintaining turn-taking in a smooth conversation. Existing studies have largely investigated neural circuits that support the preparation and generation of speech sounds. It is believed that activity in the prefrontal and premotor cortical areas facilitates high-level speech control and activity in the ventral part of the sensorimotor cortex controls the articulator (e.g. lip, jaw, tongue) movements. However, little is known about the neural mechanism controlling a sudden and voluntary stop of speech. Traditional view attributes this to a disengagement of motor signals while recent evidence suggested there may be an inhibitory control mechanism. This gap in knowledge limits our understanding of disorders like stuttering and aphasia, where deficits in speech timing control are among the common symptoms. The overall goal of this study is to determine how the brain controls the stopping of ongoing speech production to deepen our understanding of speech and communication in normal and impaired conditions.
San Francisco, California
LP352 in Subjects With Developmental and Epileptic Encephalopathy
Sorry, in progress, not accepting new patients
The objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.
San Francisco, California and other locations
RNS System LGS Feasibility Study
Sorry, in progress, not accepting new patients
To generate preliminary safety and effectiveness data for brain-responsive neurostimulation of thalamocortical networks as an adjunctive therapy in reducing the frequency of generalized seizures in individuals 12 years of age or older with Lennox Gastaut Syndrome (LGS) who are refractory to antiseizure medications. The intent is to determine the feasibility and the optimal design of a subsequent pivotal study in order to expand the indication for use for the RNS System as a treatment for patients with medically intractable LGS.
San Francisco, California and other locations
Spatiotemporal Dynamics of the Human Emotion Network
Sorry, accepting new patients by invitation only
The overall goal of this study is to elucidate how emotion network dynamics relate to the behavioral, autonomic, and experiential changes that accompany emotions and to investigate how emotion network dysfunction relates to affective symptoms. Affective symptoms are a common feature of neuropsychiatric disorders that reflect dysfunction in a distributed brain network that supports emotion. How aberrant functioning in a single emotion network underlies a wide range of affective symptoms, such as depression and anxiety, is not well understood. Anchored by the anterior cingulate cortex and ventral anterior insula, the emotion network responds to numerous affective stimuli. The recording of neural activity directly from the cortical surface from individuals is a promising approach since intracranial electroencephalography (iEEG) can provide direct estimates of neuronal populations to map the spatiotemporal dynamics of the emotion network at a millisecond level resolution. This study will exam how activity within emotion network hubs changes during emotions and how emotion network properties make some individuals more vulnerable to affective symptoms than others. A multidisciplinary approach is critical for understanding the dynamic brain network to advance neuroanatomical models of emotions and for guiding the development of novel treatments for affective symptoms.
San Francisco, California
Neural Coding of Speech Across Human Languages
Sorry, accepting new patients by invitation only
The overall goal of this study is to reveal the fundamental neural mechanisms that underlie comprehension across human spoken languages. An understanding of how speech is coded in the brain has significant implications for the development of new diagnostic and rehabilitative strategies for language disorders (e.g. aphasia, dyslexia, autism, et alia). The basic mechanisms underlying comprehension of spoken language are unknown. Researchers are only beginning to understand how the human brain extracts the most fundamental linguistic elements (consonants and vowels) from a complex and highly variable acoustic signal. Traditional theories have posited a 'universal' phonetic inventory shared by all humans, but this has been challenged by other newer theories that each language has its own unique and specialized code. An investigation of the cortical representation of speech sounds across languages can likely shed light on this fundamental question. Previous research has implicated the superior temporal cortex in the processing of speech sounds. Most of this work has been entirely carried out in English. The recording of neural activity directly from the cortical surface from individuals with different language experience is a promising approach since it can provide both high spatial and temporal resolution. This study will examine the mechanisms of phonetic encoding, by utilizing neurophysiological recordings obtained during neurosurgical procedures. High-density electrode arrays, advanced signal processing, and direct electrocortical stimulation will be utilized to unravel both local and population encoding of speech sounds in the lateral temporal cortex. This study will also examine the neural encoding of speech in patients who are monolingual and bilingual in Mandarin, Spanish, and English, the most common spoken languages worldwide, and feature important contrastive differences of pitch, formant, and temporal envelope. A cross-linguistic approach is critical for a true understanding of language, while also striving to achieve a broader approach of diversity and inclusion in neuroscience of language.
San Francisco, California
Role of Inflammation in Neonatal Epileptogenesis
Sorry, in progress, not accepting new patients
The purpose of this study evaluate the relationship between inflammation and epilepsy in neonates with seizures after birth.
Oakland, California and other locations
Human Epilepsy Project 3
Sorry, in progress, not accepting new patients
By carrying a careful, large-scale and ambitious prospective study of a cohort of participants with generalized epilepsy, the study team hopes to clarify the likelihood of response and remission in this type of epilepsy, and try to explore the underlying biological drivers of treatment response, including novel realms of exploration such as impact of the microbiome, and genetics. The identification of biomarkers that predict the likelihood of disease response would allow epilepsy patients to make more informed decisions about the factors affecting their quality of life, including plans for driving, relationships, pregnancy, schooling, work, and play. In addition to its impact on clinical care, the data and specimens collected in HEP3, including sequential electrophysiology, biochemical profiles and neuroimaging and banked DNA for future genomics studies, have the potential to provide new insights into the biological basis of IGE, thereby advancing the discovery of effective treatments and cures. By enrolling both newly diagnosed subjects (prognosis unknown) as well as subjects with established IGE who are already determined to be treatment resistant or treatment responsive, the study team can immediately test potential biomarkers in a confirmation cohort, which will accelerate identification of predictive biomarkers.
San Francisco, California and other locations
Neonatal Seizure Registry - Developmental Functional EValuation
Sorry, in progress, not accepting new patients
The NSR-DEV study is a longitudinal cohort study of around 280 Neonatal Seizure Registry participants that aims to evaluate childhood outcomes after acute symptomatic neonatal seizures, as well as examine risk factors for developmental disabilities and whether these are modified by parent well-being.
San Francisco, California and other locations
RNS® System Epilepsy PAS
Sorry, in progress, not accepting new patients
The purpose of the study is to follow patients with partial onset seizures prospectively over 5 years in the real-world environment to gather data on the long-term safety and effectiveness of the RNS System at qualified CECs by qualified neurologists, epileptologists, and neurosurgeons trained on the RNS System.
San Francisco, California and other locations
Transforming Research and Clinical Knowledge in Traumatic Brain Injury Epileptogenesis Project (TRACK-TBI EPI)
Sorry, accepting new patients by invitation only
The overarching goal of this study is to improve understanding of the long-range natural history of TBI and post-traumatic epilepsy (PTE) by extending follow-up of a previously enrolled cohort (TRACK-TBI) beyond the first 12 months after injury.
San Francisco, California and other locations
Our lead scientists for Epilepsy research studies include Edward F Chang, MD Ernesto Gonzalez-Giraldo, MD Joseph Sullivan, MD Robert C Knowlton, MD Lingyun Zhao, PhD Adam L Numis, MD Hannah C Glass, MDCM, MAS Virginia Sturm, PhD.
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