A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Confirm the Safety,Tolerability, and Efficacy of Zilucoplan in Subjects with Generalized Myasthenia Gravis

Date Added
January 14th, 2020
PRO Number
Pro00093537
Researcher
Katherine Ruzhansky

List of Studies


Keywords
Autoimmune disease
Summary

Myasthenia gravis is an autoimmune disease; a disease that occurs when the immune system attacks the body's own tissues. In generalized myasthenia gravis (gMG), that attack interrupts the connection between nerves and muscles; called the ‘neuromuscular junction.' This study is to study the safety, tolerability and efficacy of a study drug called Zilucoplan in adult participants with gMG.

Participation in this study will last approximately 16 weeks and will include approximately 7 study visits.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
Maddenka@musc.edu

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis

Date Added
July 9th, 2019
PRO Number
Pro00086756
Researcher
Katherine Ruzhansky

List of Studies


Keywords
Autoimmune disease
Summary

Generalized Myasthenia gravis (gMG) is a rare neuromuscular inflamation disorder that involve all voluntary muscle groups. This study is to evaluate the safety and efficacy of ravulizumab for the treatment of patients with generalized myasthenia gravis (gMG).

There will be 3 periods in this study; a 4 week Screening Period, a 26-week Randomized-Controlled Period consisting of 8 study visits, and an Open-Label Extension Period consisting of 17 visits that will continue for up to 2 years. Meningococcal vaccination is required for all patients prior to intravenous administration of study drug. The overall study duration for an individual participant is estimated to take up to 2.5 years.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
maddenka@musc.edu

An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis

Date Added
June 25th, 2019
PRO Number
Pro00086363
Researcher
Katherine Ruzhansky

List of Studies


Keywords
Autoimmune disease
Summary

The purpose of this study is to evaluate how safe and effective intravenous eculizumab is in pediatric patients 6 to less than 18 years old with generalized Myasthenia gravis (gMG). Eculizumab is already approved for use in adult patients with gMG in the US, Europe and Japan, but currently has not been approved for use in pediatric patients.

The study's duration is approximately 4.7 years with 4 treatment periods consisting of a Screening Period (2 to 4 weeks), Primary Evaluation Treatment Period (26 weeks), Extension Period (up to an additional 208 weeks), and Follow-up Period (8 weeks). All patients who complete Week 26 of Study ECU-MG-303 will continue receiving eculizumab in the Extension Period of this study for up to an additional 208 weeks.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
maddenka@musc.edu

HELIOS-A: A Phase 3 Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Date Added
May 14th, 2019
PRO Number
Pro00088330
Researcher
Katherine Ruzhansky

List of Studies


Keywords
Muscle, Nervous System, Rare Diseases
Summary

This is a global phase 3 open-label study designed to evaluate the efficacy and safety of ALN-TTRSC02 in adult patients (18 - 85 years of age) with hATTR amyloidosis. The estimated time on the study is approximately 3 years, including 42 days of Screening, an 18 month Treatment Period and an 18 month Treatment Extension Period.

Institution
MUSC
Recruitment Contact
John Keller
843-792-2209
kellej@musc.edu

PROMISE-MG: Prospective Multicenter Observational Cohort Study of Comparative Effectiveness of Disease-Modifying Treatments for Myasthenia Gravis

Date Added
September 24th, 2018
PRO Number
Pro00077927
Researcher
Katherine Ruzhansky

List of Studies


Keywords
Muscle, Nervous System
Summary

This is an observational study to develop a research registry to collect information from subjects with Myasthenia Gravis (MG) to evaluate the effects of the treatments they receive and to understand how their medical condition and treatment affects their daily life.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
maddenka@musc.edu



-- OR --