Neuromuscular Disease CRO

Accelerate your neuromuscular disease study by partnering with a global CRO with the necessary expertise and leadership

Accelerate your neuromuscular disease study by partnering with a global CRO with the necessary expertise and leadership

Neuron system

Making the Complex Seamless® in Neuromuscular Clinical Development

Diseases characterized by neuromuscular degeneration are particularly challenging when it comes to study design and endpoint selection. You need a trusted partner with specialized scientific leadership, relevant operational experience, and comprehensive services in place to help drive your compound to approval.

Accelerate your neuromuscular disease study with support from our noted medical and regulatory experts, highly experienced clinical trial management teams, central labs, and imaging core labs. As a full-service and therapeutically-focused clinical research organization (CRO), Medpace specializes in the design and conduct of global neuroscience trials, including neuromuscular disorders, across indications spanning adult and pediatric patients.  

Medpace Neuromuscular Disease Trial Capabilities

Enhance and expedite your program by leveraging our global footprint, strategic medical, regulatory and operational leadership, and fully integrated central labs and core imaging services. Partner with a  global CRO with a cross-functional collaborative model to set your Neuromuscular trial up for success. The Medpace Neuromuscular Disease Team can support your clinical trials through:

  • Extensive expertise in gene editing, gene transfer technology, and other cellular and gene therapies, along with the additional proficiency in submissions required to ensure regulatory compliance
  • Specialized project teams that leverage site relationships and utilize novel endpoints such as actigraphy, pulmonary function testing, physical function testing, and imaging to ensure efficient study execution from study-start up through completion of the clinical study report (CSR)
  • Global regulatory affairs team able to lead, consult, and manage complex regulatory approval processes, including complex pediatric trials, along with an experienced regulatory submissions team to facilitate expedited start-up
  • Partnerships with endpoint training and oversight groups and in-house data management programming to improve trend oversight
  • Integrated bioanalytical lab and phase l unit to streamline development
  • Flexible, highly-customizable ePRO solution that is fully integrated into our proprietary data management system, ClinTrak
  • Experience in complex drug delivery, such as intrathecal/intraparenchymal dose administration and CSF collection processes
Neurologist reviews patient's brain MRI scan

Neurologist and Neurosurgeon Talking

Core Imaging Expertise for Neuromuscular Disease Studies

High-quality image acquisition and interpretation are crucial for the success of trials relying on imaging, including neuroimaging for patient selection or as a primary endpoint. Medpace provides an integrated imaging and ECG core lab with an end-to-end suite of global imaging services, ensuring imaging components are seamlessly incorporated into the complex structure of the overall trial. 

Our comprehensive suite of imaging services enhances and expedites biopharmaceutical and imaging contrast agent development, including a broad spectrum of imaging biomarkers for rare neuromuscular disease trials. Capabilities include advanced quantitative magnetic resonance imaging measures for:

  • Muscle Fat Fraction (MRI-PDFF, MRS)
  • Elastography
  • Muscle cross-sectional area or volume
  • Anatomic and functional brain mapping

Neuromuscular Disease Recruitment Strategies

Our multi-dimensional recruitment model and vast experience recruiting for rare disease trials enable us to implement customized recruitment and retention strategies that identify, recruit, and retain challenging patient populations. Established relationships with key patient advocacy groups such as TREAT-NMD, Parent Project Muscular Dystrophy, and Muscular Dystrophy Association help to facilitate patient recruitment and retention for these rare neuromuscular indications. Additionally, we are a member of many key scientific associations including World Muscle Society and American Academy of Neurology.

Patients with rare neuromuscular diseases face obstacles in their daily lives; minimizing burden for the patient and caregiver is a critical component in recruiting a committed group of patients into studies. Medpace places patients and caregivers at the center of a trial to increase awareness and reach enrollment goals while helping yield successful outcomes in terms of study design, participation, adherence, satisfaction, and data collection. Medpace Patient Concierge Services (PCS) provides global travel support to minimize patient burden, facilitate compliance with study visits and help keep the study on track.

Neuromuscular Disease Experts

Our highly-experienced team of medical, regulatory, operational, and imaging experts understand trial issues from the perspective of Sponsors, clinical investigators, scientific leaders, and reviewers at regulatory agencies and work collaboratively to successfully define and execute clear development plans from beginning to end.

Serving as therapeutic team leaders, in-house neurologists, and child neurologists that are skilled in neuromuscular disease studies provide strategic direction for study design and planning, train operational staff, work with Investigators, provide medical monitoring, and meet with regulatory agencies as well as our global regulatory affairs experts. These physicians are embedded throughout every study to provide strategic guidance on the best pathways to accelerate approvals.

Key Neuromuscular Disease Indications

Our physicians and staff have experience in rare diseases and diseases that impact neuromuscular function such as:

  • Allan-Herndon-Dudley Syndrome (AHDS)
  • Amyotrophic Lateral Sclerosis
  • Charcot Marie Tooth Disease
  • Duchenne Muscular Dystrophy
  • Facioscapulohumeral Muscular Dystrophy
  • Familial Amyloidosis Polyneuropathy
  • Foot Drop
  • Mitochondrial Myopathy
  • Mucopolysaccharidosis
  • Muscular Dystrophies
  • Myasthenia Gravis
  • Myotonic Dystrophy
  • Nonsense Mutation Dystrophinopathy
  • Pompe disease
  • Spinal Muscular Atrophy
  • Spasticity
  • X-Linked Adrenoleukodystrophy (X-ALD)

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