Effect of Oral Levosimendan (ODM-109) on Respiratory Function in Patients with ALS (REFALS)
Study Purpose:
The purpose of the study is to evaluate safety and the effectiveness of levosimendan in preserving respiratory function in patients with amyotrophic lateral sclerosis (ALS).
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
EnrollingPhase:
Phase IIIStudy Chair(s)/Principal Investigator(s):
Merit Cudkowicz, MD, Massachusetts General Hospital
Clinicaltrials.gov ID (11 digit #):
NCT03505021Neals Affiliated?
YesCoordinating Center Contact Information
Orion Corporation/Orion PharmaMerja Mäkitalo / .(JavaScript must be enabled to view this email address) / +358 10 426 7737
.(JavaScript must be enabled to view this email address) Tengströminkatu 8
Turku , Finland
Full Study Summary:
This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality as measured by the ALSFRS-R scale. In this randomized, double-blind, placebo-controlled, parallel-group, multicenter study, subjects are allocated in a 2:1 ratio to receive either levosimendan (1 -2 mg daily) or placebo for 48 weeks. The primary endpoint is slow vital capacity (SVC) at 12 weeks, with the impact on patient function assessed through 48 weeks, adjusted for patient outcome, using ALSFRS-R (combined assessment of function and survival, CAFS). Other important efficacy measures include time to respiratory events, clinical global impression (CGI), assessment of dyspnea using the Borg scale and sleep scales (Pittsburgh sleep quality index and Epworth sleepiness scale). Patient safety is monitored using conventional methods including adverse events, safety laboratory tests, vital signs and 12-lead EKG. Following screening and baseline visits, patients attend the clinic at 2, 4, 8, 12, 24, 36 and 48 weeks, with telephone assessments conducted at weeks 18, 30 and 42. An end of study visit is performed 14-25 days after the last study treatment administration. The study will be monitored by an independent data and safety monitoring board. A long-term extension study will be available for patients completing the study.
Study Sponsor:
Orion Corporation, Orion PharmaParticipant Duration:
Total study duration for each subject will be 51-52 weeks, including the screening period, 48 weeks treatment and an end-of-study visit.
At screening, the visit length will be approximately 2 – 2.5hrs.
For visits 2, 3 and 8, the visit length will be approximately 3 – 5 hrs.
For visits 4 to 7, the visit length will be approximately 2 – 3hrs.
There will also be three telephone contacts made in-between sites visits that should not last longer than 30 minutes.
Depending on location of sites, the study participant may be required to travel.