Clinical Research

Study trials (protocols) are based on patient safety first. Most if not all clinical trials are regulated by the FDA and Institutional Review Board (IRB). These organizations have strict guidelines to determine if a study is safe for humans to participate. Typically, the IRB board consists of a group of people such as physicians, scientists, attorneys, and laymen who meet to review the risks and benefits of a protocol before it is out for public knowledge. If the benefits weigh higher than the risks, the study (protocol) is approved.

Pharmaceutical and biopharma companies, government agencies and other organizations can sponsor a clinical trial for physician practices, hospitals, universities to be a participating site.

A participating site must have qualified staff trained in understanding research guidelines through Good Clinical Practice (GCP) compliance. The research team consists of the physician aka principal investigator (PI) and clinical research coordinators. The PI has oversight of the study and team at the site, while the coordinators (CRC), help organize, maintain records, and conduct the study with the PI.

Most studies are monitored or audited by a third-party company called a Clinical Research Organization (CRO), who is hired by the Sponsor to perform monthly quality assurance checks and visit the site often. Monitors help organize all the supplies, training and communication between the sponsor and the site.

Clinical Research is essential and beneficial in helping discover new treatments for disease. People who participate in studies are screened to qualify to enter the study. If they agree to participate, they must sign a consent form. A study is voluntary, and a patient can withdraw at any time.

Not only do clinical trials improve and develop medicine, but they also potentially offer a better-quality life for people affected by disease.

• 233AS303 – A Phase 3 Randomized, Placebo-Controlled Trial with a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults with a Confirmed Superoxide Dismutase 1 Mutation. NCT04856982
• PrecisionMed Protocol 4800 – A single or multiple visit protocol for collection of DNA/RNA/SERUM/PLASMA/CSF in Amyotrophic Lateral Sclerosis and related disorders. NCT05349721

• EDG-5506-201 – A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy.” NCT05291091

• 80202135CDP3001 – Phase 2/3, Multistage, Multicenter, Randomized, Double-Blind, Placebo-Controlled Parallel Group Withdrawal Study to Evaluate the Efficacy and Safety of Nipocalimab Administered to Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). NCT05327114
• IMVT-1401-2401 – A Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants with active CIDP. NCT05581199

• PVT-2201-301 – A Phase 3 Study to Investigate the Efficacy and Safety of Brepocitinib in Adults with Dermatomyositis (VALOR). NCT05437263
• MS200569_0041 – A Phase IIa, Randomized, Parallel, Double-Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of Enpatoran in Dermatomyositis and Polymyositis Participants receiving Standard of Care. NCT05650567

• ARGX-113-2007 – A Phase 2/3, Randomized, Double-Blinded, Placebo Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy. NCT05523167

• ABC008-IBM-201 – A Phase 2/3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body MyositisA Phase 2/3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis. NCT05721573

• R3918-MG-2018 – Efficacy And Safety Of Pozelimab And Cemdisiran Combination Therapy In Patients With Symptomatic Generalized Myasthenia Gravis. NCT05070858
• IMVT – 1401-3101 – A Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants with Generalized Myasthenia Gravis. NCT05403541

• VX22-MDO-801 – A Study to Assess Video Hand Opening Time and Ambulation in Healthy Volunteers and Individuals with Myotonic Dystrophy Type 1