17 results
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.
The primary objective is to test the hypothesis that once daily tadalafil administered orally for 48 weeks lessens the decline in ambulatory ability as measured by the 6MWD compared to placebo in boys with Duchenne muscular dystrophy (DMD). Two…
Primary Objective: Is there reduction in pain using an abduction brace in patients undergoing arthroscopic cuff repair? Secondary Objectives: - Are there differences in function and recovery of the cuff, determined using questionnaires and echo?- Is…
The purpose of this 5-year trial is to determine if the surgical procedure, extended transsternal thymectomy (ETTX), combined with prednisone therapy is more beneficial in treating individuals with non-thymomatous MG than prednisone therapy alone.
Objectives. The goals of the current protocol is therefore the natural follow-up of the objectives achieved with the previous grants and, in particular, of projects designed to discern new models for the successful conduct of clinical trials in…
Main Objective:To evaluate the long-term safety and tolerability of BYM338 in the treatment of sIBM and to further evaluate the effect of three BYM338dose regimens against placebo in increasing the distance traveled as measured by the 6 Minute…
Primary:* To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.Secondary:* To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study,…
This study has been transitioned to CTIS with ID 2023-507146-91-00 check the CTIS register for the current data. Primary Objective:• To evaluate the safety and tolerability of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg…
Primary:* To assess the efficacy of idebenone compared to placebo, in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in Forced Vital Capacity percent predicted (FVC %p) using…
Primary:• To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in ambulant boys ages 4 to <8 years with DMD using the Time to Stand Test (…
This study has been transitioned to CTIS with ID 2023-508284-77-00 check the CTIS register for the current data. The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of gMG in adults with autoantibodies…
This study has been transitioned to CTIS with ID 2024-511852-42-00 check the CTIS register for the current data. Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or…
This study has been transitioned to CTIS with ID 2023-506761-65-00 check the CTIS register for the current data. The purpose of this Phase II/III study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of…
The objective of this study is to investigate whether BTA injection in the pelvic floor muscle is an effective treatment for patients with chronic pelvic pain and pelvic floor hypertonicity.
Primary objective:* To establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects.Secondary objectives:* To assess the safety and tolerability of givinostat versus…
This study has been transitioned to CTIS with ID 2024-511707-42-00 check the CTIS register for the current data. The purpose of this study is to collect long-term follow-up safety and efficacy data on patients with SMA who were treated with OAV101…
Primary Objective:The primary objective of this study is to provide confirmatory data on the beneficial effect of 2.0 g/kg of Octagam 10% given every 4 weeks compared with placebo in subjects with active DM based on the percentage of responders at…