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.

• Eidos-AG10-501 – A phase 3 Randomized, Multicenter, Double-Blind, Placebo Controlled Study of Acoramidis for
  Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial). NCT06563895

• SeraTrials-Protocol 11008 – A single or multiple visit protocol for collection of DNA/RNA/SERUM/PLASMA/CSF in Amyotrophic Lateral Sclerosis and related disorders.
• 144126 – HEALEY ALS Platform Trial NCT04297683

• 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
• ARGX-113-2406 – A Phase 4, Open-Label, Single-Group, Multicenter Study in Adult Participants with Chronic Inflammatory Demyelinating Polyneuropathy Who Transition from Treatment with Intravenous Immunoglobulin to Efgartigimod PH20 SC. NCT06637072
• ARGX-117-2401 – A Phase 3, Randomized, Double-Blinded, Double-Dummy Study Evaluating the Efficacy and Safety of Intravenous Empasiprubart Versus Intravenous Immunoglobulin in Adults With Chronic Inflammatory Demyelinating Polyneuropathy NCT06920004
• ARGX-117-2402 (Emnergize) – A Phase 3, Randomized, Double-Blinded, Placebo-Controlled Study Evaluating the Efficacy and Safety of Empasiprubart IV in Adults With Chronic Inflammatory Demyelinating Polyneuropathy NCT07091630
• EFC18156 (Vitalize) – A Phase 3, randomized, double-blind, study evaluating efficacy and safety of riliprubart versus intravenous immunoglobulin (IVIg) in participants with chronic inflammatory demyelinating polyneuropathy NCT06859099
• EFC17236 (Mobilize) – A Phase 3, double-blind, randomized, placebo-controlled study evaluating efficacy and safety of SAR445088 in adults with chronic inflammatory demyelinating polyneuropathy NCT06859099

• RAY902CT – Phase 1b Open Label Basket Trial Of Ray121 To Inhibit Classical Complement Pathway In Immunological Diseases (DM and IMNM) (Rainbow Trial) NCT06371417

• MOVE FSHD – Motor Outcomes to Validate Evaluations in FSHD NCT04635891

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

• PASS- ARGX-113-2208 – A non-interventional, post-authorization safety study of patients treated with
  efgartigimod alfa. NCT06298565
• 20240236 – AMG335 – An Open-label Study to evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in children with gMG. NCT04524273
• ARGX-999-2-MG-2000 (ADAPT Forward) – A Master Protocol for an Exploratory, Phase 2a, Proof-of-Concept Platform Study to Evaluate the Safety, Tolerability, and Efficacy of Multiple Regimens in Participants with Myasthenia Gravis

• EMPASSION- ARGX-117-2302 – A Phase 3, Randomized, Double-Blinded, Double-Dummy Study evaluating the Efficacy and Safety of Empasiprubart Versus Intravenous Immunoglobulin in Adults with Multifocal Motor Neuropathy. NCT06742190