Neuromuscular Research


Expertise:

We offer a robust research program including many Clinical trials and innovative research for patients with ALS, myasthenia gravis, muscular dystrophies, and peripheral neuropathy. We are an active member of the Northeast ALS Consortium (NEALS) and the Muscle Study Group (MSG).  We are currently part of the Healey Platform study in collaboration with Massachusetts General Hospital and NEALS.  We are also members of the CReATe Consortium which is a NIH funded project through the University of Miami for patients with motor neuron disorders.

  • CReATe CAPTURE - ALS

    Caralyne Jackson, MD  

    Principal Investigator:
    Carlayne Jackson, MD

    https://clinicaltrials.gov/study/NCT03489278

    Type of Study:
    Registry/Observational

    Summary
    The purpose of the Clinical Procedures To Support Research (CAPTURE) study is to utilize information collected in the medical record to learn more about a disease called amyotrophic lateral sclerosis (ALS) and related disorders. The study will consent patients with ALS or related disorders that are receiving care at a clinical center in the CReATe consortium that uses Epic as its electronic health record (EHR) system. The study aims to systematically gather a clinical dataset through the EHR using a standardized approach to characterize the natural history of ALS and related diseases.

    Link to Sponsor’s Website:
    https://create.rarediseasesnetwork.org/research-studies

    Randee Kent

    Study Coordinator:
    Randee Kent
    Office: 210-450-0524
    Email: kentbaron@uthscsa.edu

  • HEALEY ALS Platform Regimen G

    Caralyne Jackson, MD  

    Principal Investigator:
    Carlayne Jackson, MD

    https://clinicaltrials.gov/study/NCT05842941

    Type of Study:
    Interventional/Randomized Control

    Summary
    The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683. Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.  Regimen G will evaluate the safety and efficacy of a single study drug, DNL343, in participants with ALS.

    Link to Sponsor’s Website:
    https://www.massgeneral.org/assets/mgh/pdf/neurology/als/generalplatform.june2023.pdf

    Randee Kent

    Study Coordinator:
    Randee Kent
    Office: 210-450-0524
    Email: kentbaron@uthscsa.edu

  • HEALEY ALS Platform Regimen F

    Caralyne Jackson, MD  

    Principal Investigator:
    Carlayne Jackson, MD

    https://clinicaltrials.gov/study/NCT05740813

    Type of Study:
    Interventional / Randomized Control

    Summary
    The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683. Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen. Regimen F will evaluate the safety and efficacy of a single study drug, ABBV-CLS-7262, in participants with ALS.

    Link to Sponsor’s Website:
    https://www.massgeneral.org/assets/mgh/pdf/neurology/als/generalplatform.june2023.pdf

    Randee Kent

    Study Coordinator:
    Randee Kent
    Office: 210-450-0524
    Email: kentbaron@uthscsa.edu

  • MOVE+

    Matthew WIcklund, MD  

    Principal Investigator:
    Matthew Wicklund, MD

    https://clinicaltrials.gov/study/NCT04635891

    Type of Study:
    Observational/Natural History

    Summary
    The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD. Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) will have approximately 450 FSHD participants followed for a minimum of 3 years. A subset of MOVE FSHD participants, approximately 200, will participate in the MOVE+ sub-study which includes whole body MRI and muscle biopsy.

    Link to Sponsor’s Website:
    https://www.fshdsociety.org/2021/07/26/anyone-can-move-for-fshd-research/

    Study Coordinator:
    Marlon Tamayo
    Office: 210-450-7370
    Email: tomayomurada@uthscsa.edu

  • SUMMIT

    Matthew WIcklund, MD  

    Principal Investigator:
    Matthew Wicklund, MD

    https://clinicaltrials.gov/study/NCT06564974

    Type of Study:
    Observational/Registry

    Summary
    The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE® in male patients 2 years of age or older with Duchenne’s Muscular Dystrophy for long term safety and quality of life. Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits (+/- 30 days) for registry assessments. Information on standard of care treatment and procedures for management of DMD will also be collected. Patients and/or their parents/legal guardians will be asked to complete paper QoL questionnaires at enrollment and at each Yearly Follow-up Visit (+/- 30 days)

    Link to Sponsor’s Website:
    https://agamree.com/agamree-summit-study/

    Study Coordinator:
    Marlon Tamayo
    Office: 210-450-7370
    Email: tomayomurada@uthscsa.edu

  • GRASP

    Matthew WIcklund, MD  

    Principal Investigator:
    Matthew Wicklund, MD

    https://clinicaltrials.gov/study/NCT03981289

    Type of Study:
    Observational/Registry

    Summary
    Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

    Link to Sponsor’s Website:
    https://www.mdcrn.com/grasp/network-information

    Study Coordinator:
    Marlon Tamayo
    Office: 210-450-7370
    Email: tomayomurada@uthscsa.edu