Cain Labs Clinical Trials and Studies
Clinical Trials
Angelman Syndrome: The HALOS study
A phase 1-2a trial to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecally administered ION582 (an investigational antisense oligonucleotide therapy), in patients with Angelman syndrome, a rare neurological condition caused by the loss of function of the maternally inherited UBE3A gene. This condition typically presents in infancy and is characterized by intellectual disability, balance issues, motor impairment, and debilitating seizures. Some patients are unable to walk or speak. Some symptoms can be managed with existing drugs; however, there are no approved disease modifying therapies.
Lead PI:
Dr. Carlos Bacino, MD, FACMG
Professor and Vice-Chair, Clinical Affairs at Baylor College of Medicine
Chief, Genetics Service at Texas Children's Hospital
Phone: 832-822-4280
Email: cbacino@bcm.edu
Study contact:
Luis Vizcaino, ACRP-CCRC
832-822-3869
Luis.VizcainoRisquet@bcm.edu
Clinical Studies
The Undiagnosed Epilepsy Genetics Initiative
To discover and understand genes for epilepsy and epileptic encephalopathies where the cause is unknown, this study provides trio whole genome sequencing for patients with epilepsy whose origin has remained unknown after genetic workup using use whole-genome sequencing (WGS), which is one of the most advanced genetic testing technologies currently available.
Principal Investigator:
Dr. Hsiao Tuan Chao, MD, PhD
832-825-0373
hsiaotuan.chao@bcm.edu
More information
Texas Children’s Hospital and Baylor College of Medicine clinicians and research investigators have recently launched the STXBP1 Clinical Trial Ready (STARR), a new study focused on developing clinical trial readiness for STXBP1-related disorders, an epileptic and neurodevelopmental condition caused by changes in the STXBP1 gene. STXBP1-related disorders are one of the most common genetic causes of childhood epilepsies.
The STARR study at Texas Children’s Hospital is currently recruiting participants across all age groups. Families interested in participating are invited to complete the STARR study eligibility survey, https://redcap.link/stxbp1survey. For more information, contact chao-lab@bcm.edu
Principal Investigator:
Dr. Hsiao Tuan Chao, MD PhD
hsiaotuan.chao@bcm.edu
PPFIA3-related disorder is a neurodevelopmental disorder caused by genetic changes to the gene PPFIA3. It is characterized by developmental delay, intellectual disability, autism and epilepsy. The purpose of this research study is to determine the effects of PPFIA3 genetic variants on neurodevelopment
Principal Investigators:
Hsiao Tuan Chao, MD, PhD
832-825-0373
hsiaotuan.chao@bcm.edu
More information
The goal of the study is to address the unmet need of treatment-resistant depression patients by identifying brain networks critical for treating depression and to use next-generation precision deep brain stimulation (DBS) with steering capability to engage these targeted networks. The study has three specific goals:
To demonstrate device capability to selectively and predictably engage distinct brain networks
To delineate depression-relevant networks and demonstrate behavioral changes with network-targeted stimulation
To demonstrate that chronic DBS using steered, individualized targeting is feasible and safe for reducing depressive symptoms
Study Contacts:
Name: Sameer Sheth, MD, PhD
Phone:713-798-5060
Email: sameer.sheth@bcm.edu
More information
Depression is one of the most common disorders of mental health, affecting 7-8% of the population and causing tremendous disability to afflicted individuals and economic burden to society. To optimize existing treatments and develop improved ones, the investigators need a deeper understanding of the mechanistic basis of this complex disorder. In this study, investigators combine precise intracranial neural recordings in humans with a suite of new eXplainable Artificial Intelligence (XAI) approaches. Successful completion of this project is expected to enhance understanding of the pathophysiology of depression and improve neuromodulatory treatment strategies. This can also be applied to a host of other neurological and psychiatric disorders, taking an important step towards XAI-guided precision neuroscience.
Study contacts:
Sameer A Sheth, MD, PhD
713-798-5060
sameer.sheth@bcm.edu
More information
The purpose of this protocol is to examine the long-term outcomes after neurosurgical intervention in patients with severe, refractory OCD.
Study Contacts:
Dr. Sameer Sheth and Victoria Pirtle
713-798-5060
sameer.sheth@bcm.edu
The purpose of this study is to create and better test systems for acquiring high resolution neurophysiological data from the human brain through recordings from hundreds of neurons simultaneously. The goal is to analyze these recordings to obtain a better understanding of spontaneous brain activity and the neural activity underlying cognitive processes. This information will be essential to understanding brain dysfunction and developing better therapies for a variety of neurological and psychiatric disorders.
Study Contacts:
Dr. Sameer Sheth and Victoria Pirtle
713-798-5060
sameer.sheth@bcm.edu
To demonstrate that the RNS System for thalamic stimulation is safe and effective as an adjunctive therapy for the reduction of primary generalized seizures in individuals 12 years of age or older who have drug-resistant idiopathic generalized epilepsy. The RNS System is currently approved by the FDA for use in patients 18 years and older with hard-to-treat partial-onset seizures, which is being used in the NAUTILUS Study.
Study contacts:
Dr. Sameer Sheth
713-798-5060
sameer.sheth@bcm.edu
More information
U.S. National Library of Medicine
Click here to learn more about all of Texas Children's Clinical Trial Studies by topic.