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.

• ATLAS – 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
• PRECISION MED – Protocol 4800 – A single or multiple visit protocol for collection of DNA/RNA/SERUM/PLASMA/CSF in Amyotrophic Lateral Sclerosis and related disorders. NCT05349721

• GRAND CANYON – 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

• CIPD – 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
• IMMUNOVANT CIPD – IMVT-1401-2401 – A Phase 2b, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study of Batoclimab Treatment in Adult Participants with Active Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). NCT05581199

• NEPTUNIA – 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

• ALKIVIA – 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

• IBM – R3918-OT-2383 – Efficacy and safety of Pozelimab and Cemdisiran combination therapy in patients with sporadic inclusion body myositis-Investigator Initiative Study. NCT06479863

• NIMBLE – R3918-MG-2018 – Efficacy And Safety Of Pozelimab And Cemdisiran Combination Therapy In Patients With Symptomatic Generalized Myasthenia Gravis. NCT05070858
• ADAPT SERON- ARGX-113-2308 – A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod IV in Adult Participants with Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis. NCT06298552
• ADAPT OCULUS- ARGX-113-2315 – A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants with Ocular Myasthenia Gravis. NCT06558279

• RECOVER-AUTONOMIC – A Platform Protocol for Evaluation of Interventions for Autonomic Dysfunction in Post-Acute Sequelae of SARS-CoV-2 Infection (PASC). NCT06305780

• SYNAPSE-CMT- NMD670-02-0003 – A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 Over 21 Days in Ambulatory Adult Patients with Type 1 and Type 2 Charcot-Marie-Tooth Disease. NCT06482437