Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS (REFALS)
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|ClinicalTrials.gov Identifier: NCT03505021|
Recruitment Status : Completed
First Posted : April 20, 2018
Results First Posted : August 31, 2021
Last Update Posted : August 31, 2021
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Levosimendan Drug: Placebo for levosimendan||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||496 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS|
|Actual Study Start Date :||June 21, 2018|
|Actual Primary Completion Date :||July 23, 2020|
|Actual Study Completion Date :||July 23, 2020|
Levosimendan 1 mg capsules for oral administration, once to twice a day. The total duration of treatment 48 weeks
Levosimendan 1 mg capsule for oral administration
Other Name: ODM-109
Placebo Comparator: Placebo for levosimendan
Placebo capsule for oral administration, once to twice a day. The total duration of treatment 48 weeks.
Drug: Placebo for levosimendan
Placebo capsule for oral administration
Other Name: Placebo for ODM-109
- Supine Slow Vital Capacity (SVC) [ Time Frame: The change from baseline at 12 weeks ]Change from baseline to 12 weeks, expressed as % of predicted normal.
- Combined Assessment of Function and Survival Through 48 Weeks [ Time Frame: Mean rank at 48 weeks ]Scale: The ALS Functional Rating Scale - Revised. This scale includes 12 items. Each item was scored from 0 to 4. Total score is the sum of the scores of all 12 items. Each subject is ranked according to time-to-death (earlier deaths ranked lower than later deaths). Subjects who survive are ranked more favorably than subjects who died. Among the survivors, subjects are ranked according to change in ALSFRS-R (greater worsening of ALSFRS-R is ranked lower than after deaths). Subjects who survive are ranked more favorably than subjects who died. Among the survivors, subjects are ranked according to change in ALSFRS-R (greater worsening of ALSFRS-R is ranked lower than less worsening or an improvement in ALSFRS-R). The ranked scores range from 001 to 495 (the number of participants assessed for the Outcome Measure) with larger rank score numbers associated with a better outcome.
- Time to Respiratory Event Through 48 Weeks [ Time Frame: Time to event through 48 weeks ]ALSFRS-R scale contains 3 items that relate to respiratory function: severity of dyspnoea, occurrence of orthopnoea (shortness of breath when lying flat) and the use of mechanical ventilation for respiratory insufficiency. A reduction in any one of these items was considered a respiratory event. Not all patients receive ventilatory support, despite respiratory insufficiency: meeting "protocolised" criteria for NIV relates to patients without NIV whose slow vital capacity declined to a level that would ordinarily trigger such treatment.
- Change From the Baseline in Clinical Global Impression CGI at 48 Weeks [ Time Frame: The change from baseline at 48 weeks ]Visual Analogue Scale 0-100 millimeters, rated by study subjects. Score 0 indicates that the subject is completely well without any disability and score 100 indicates the worst possible severity of the condition.
- Change From Baseline in Respiratory Function of ALSFRS-R at 48 Weeks [ Time Frame: Slope of decline at 48 weeks ]ALSFRS-R scale contains 3 items that relate to respiratory function: severity of dyspnoea, occurrence of orthopnoea (shortness of breath when lying flat) and the use of mechanical ventilation for respiratory insufficiency. These are added together to created the respiratory domain with a score range 0-12 (where 12 represents normal function). Although individual items and patients vary, ALSFRS-R typically declines at a relatively constant rate over time. Plotted over time the slope of the line obtained indicates the speed of progression and thus an effective treatment might be expected to reduce the slope of decline.
- Supine Borg Category Ratio 10 Scale at 12 Weeks [ Time Frame: Change from baseline at 12 weeks ]Patients rated their perception of the severity of their dysnoea using the Borg scale. The scale ranges from 0 (no dyspnoea) to 10 (maximal). Each category is numbered and most (not all) have verbal cues. At each assessment the patient scored the category they felt best described their symptoms. The analysis measured change from baseline at 12 weeks, where a negative score indicates improvement and a positive score reflects worsening.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03505021
|Study Director:||Merja Mäkitalo, CSD||Orion Corporation, Orion Pharma|