{"id":44,"date":"2025-06-19T19:00:08","date_gmt":"2025-06-19T19:00:08","guid":{"rendered":"https:\/\/firdapsehcp-calems.local\/calems\/?page_id=34"},"modified":"2025-12-29T14:09:36","modified_gmt":"2025-12-29T14:09:36","slug":"clinical-trial-results","status":"publish","type":"page","link":"https:\/\/firdapsehcp.com\/ca-lems\/clinical-trial-results\/","title":{"rendered":"Efficacy"},"content":{"rendered":"\n
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MUSCLE STRENGTH MAINTAINED
IN FIRDAPSE-TREATED PATIENTS1<\/sup><\/h1>\n\n\n\n

The differences between those treated with FIRDAPSE and those treated with placebo were shown to be statistically significant based on Quantitative Myasthenia Gravis (QMG) scores in 2 phase 3 studies.1-3<\/sup><\/strong><\/p>\n\n\n\n

Patients treated with FIRDAPSE maintained their muscle strength, while a statistically significant decline was demonstrated in patients in the placebo group.1-3<\/sup><\/p>\n\n\n\n

Coprimary Endpoint<\/strong><\/p>\n\n\n\n

\n
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Study 1: Total QMG Changes From Baseline at Day 141,2<\/sup><\/strong><\/p>\n\n\n\n

\"\"<\/figure><\/div><\/div>\n<\/div>\n\n\n\n
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Study 2: Total QMG Changes From Baseline at Day 41,3<\/sup><\/strong><\/p>\n\n\n\n

\"\"<\/figure><\/div><\/div>\n<\/div>\n<\/div><\/div>\n\n\n\n

The QMG assessment is a 13-item, physician-rated categorical scale assessing muscle weakness. Each assessment is rated 0 to 3, where a score of 0 represents no weakness and a score of 3 represents severe weakness (total score ranges from 0 to 39). Higher scores represent greater impairment.1<\/sup><\/p>\n<\/div><\/div><\/div>\n\n\n\n

PATIENT PERCEPTION OF PHYSICAL WELL-BEING MAINTAINED IN FIRDAPSE-TREATED PATIENTS1-3<\/sup><\/h2>\n\n\n\n

The differences between those treated with FIRDAPSE and those treated with placebo were shown to be statistically significant based on Subject Global Impression (SGI) scores in 2 phase 3 studies.1-3<\/sup><\/strong><\/p>\n\n\n\n

FIRDAPSE-treated subjects reported a statistically significant better sense of well-being compared to those in the placebo group.1-3<\/sup><\/p>\n\n\n\n

Coprimary Endpoint<\/strong><\/p>\n\n\n\n

\n
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Study 1: SGI Changes From Baseline at Day 141,2<\/sup><\/strong><\/p>\n\n\n\n

\"\"<\/figure><\/div><\/div>\n<\/div>\n\n\n\n
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Study 2: SGI Changes From Baseline at Day 41,3<\/sup><\/strong><\/p>\n\n\n\n

\"\"<\/figure><\/div><\/div>\n<\/div>\n<\/div><\/div>\n\n\n\n

The SGI assessment is a 7-point scale on which patients rate their global impression of the study treatment effects on their physical well-being. Lower scores on the SGI represent lower perceived benefit with the study treatment. The 7-point SGI scale: 1=terrible, 2=mostly dissatisfied, 3=mixed, 4=partially satisfied, 5=mostly satisfied, 6=pleased, and 7=delighted.1-3<\/sup><\/p>\n\n\n\n

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CLINICIAN IMPRESSION OF PATIENT WELL-BEING IMPROVED WITH FIRDAPSE1-3<\/sup><\/h2>\n\n\n\n

The differences between those treated with FIRDAPSE and those treated with placebo were shown to be statistically significant based on Clinical Global Impression-Improvement (CGI-I) scores in 2 phase 3 studies.1-3<\/sup><\/strong><\/p>\n\n\n\n

FIRDAPSE-treated subjects demonstrated a statistically significant improvement in symptoms, behavior, and functional ability compared with those in the placebo group.1,3<\/sup><\/p>\n\n\n\n

Secondary Endpoint<\/strong><\/p>\n\n\n\n

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Study 1: CGI-I Scores at Day 141,2<\/sup><\/strong><\/p>\n\n\n\n

\"\"<\/figure><\/div><\/div>\n<\/div>\n\n\n\n
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Study 2: CGI-I Scores at Day 41,3<\/sup><\/strong><\/p>\n\n\n\n

\"\"<\/figure><\/div><\/div>\n<\/div>\n<\/div><\/div>\n\n\n\n

The CGI-I is a subjective, investigator-assessed overall impression of improvement or worsening in symptoms. The 7-point scale is scored from 1 (very much improved) to 7 (very much worse) based on symptoms, behavior, and functional ability. CGI-I scores were collected as a secondary endpoint during this study.26<\/sup><\/p>\n<\/div><\/div><\/div>\n\n\n\n

FIRDAPSE PATIENTS MAINTAINED FUNCTIONAL MOBILITY3<\/sup><\/h2>\n\n\n\n

The Triple-Timed Up-and-Go Test (3TUG) was an exploratory endpoint in a phase 3 study and showed the differences between placebo-treated subjects and those treated with FIRDAPSE.3,27,28<\/sup><\/strong><\/p>\n\n\n\n

The proportion of patients with a \u226520% increase in the 3TUG average time was higher in the placebo group compared with the proportion of patients in the FIRDAPSE group.3<\/sup><\/p>\n\n\n\n

Exploratory Endpoint<\/strong><\/p>\n\n\n\n

Study 2: 3TUG Average Time3<\/sup><\/strong><\/p>\n\n\n\n

\"\"<\/figure><\/div><\/div>\n\n\n\n

The 3TUG is a functional mobility test that requires a patient to stand up from a straight-backed armchair, walk 3 meters, turn around, walk back, and sit down in the chair. Individuals perform the test 3 consecutive times without pause, and the final measurement is the average time required to complete all 3 repetitions. Based on literature reports, a significant change in gait for a similar walk test is an increase in time of >20%, which was incorporated into the endpoint.3<\/sup><\/p>\n\n\n\n

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TRIAL 1 STUDY DESIGN2<\/sup><\/h2>\n\n\n\n
\"\"<\/figure><\/div><\/div>\n\n\n\n

Eligibility1,2<\/sup><\/strong><\/p>\n\n\n\n

Patients were aged \u226518 years, ambulatory, had a confirmed LEMS diagnosis, with documented acquired proximal muscle weakness and at least 1 of the following:<\/p>\n\n\n

    \n
  1. Compound muscle action potential that increased >2-fold after maximum voluntary contraction<\/span><\/li>\n\n\n\n
  2. Positive anti-P\/Q-type VGCC-Ab test 
<\/span><\/li>\n<\/ol>\n\n\n\n

    Open-label phase: Patients received an individualized total daily dosage of FIRDAPSE between 15 and 80 mg\/day, given in 3 to 4 divided doses with a maximum single dose of 20 mg (total of >91 consecutive days). Patients had to receive between 30 and 80 mg\/day to enter the double-blind phase of the study.<\/p>\n<\/div><\/div><\/div>\n\n\n\n

    TRIAL 2 STUDY DESIGN3<\/sup><\/h2>\n\n\n\n
    \"\"<\/figure><\/div><\/div>\n\n\n\n

    Eligibility1,3<\/sup><\/strong><\/p>\n\n\n\n

    Patients were aged >18 years, ambulatory, had a confirmed LEMS diagnosis, with documented acquired proximal muscle weakness and at least 1 of the following: <\/p>\n\n\n

      \n
    1. Compound muscle action potential that increased >2-fold after maximum voluntary contraction<\/span><\/li>\n\n\n\n
    2. Positive anti-P\/Q-type VGCC-Ab test 
<\/span><\/li>\n<\/ol>\n\n\n\n

      Dosing: Patients received an individualized total daily dosage of FIRDAPSE between 30 and 80 mg\/day, given in 3 to 4 divided doses, with a maximum single dose of 20 mg.<\/p>\n\n\n\t\t\t\t

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      \"\"<\/figure><\/div><\/div>\n<\/div>\n\n\n\n
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      Efficacy and Safety Clinical Trial Data of FIRDAPSE in Patients with LEMS<\/strong><\/strong><\/h3>\n\n\n\n

      Take a deeper dive into the study design and clinical findings from the 2 phase 3 studies for FIRDAPSE.<\/p>\n\n\n\n

      \n
      Watch Video<\/span><\/span><\/a><\/div>\n<\/div>\n<\/div>\n<\/div><\/div>\n\n\n<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\n\n\n
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      \n\t\t\t\t\n\t<\/div>\n\n\t
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      Request a Rep<\/h3>\n\t\t\t\t

      Connect with a FIRDAPSE
      \nrepresentative.<\/p>\n\t<\/div>\n\n\t

      \n\t\t\n\t\t\tStart Request\t\t<\/a>\n\t<\/div>\n<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\n\n\n
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      Safety<\/h3>\n\t\t\t\t

      See the results from the clinical trials.<\/p>\n\t<\/div>\n\n\t

      \n\t\t\n\t\t\tGo to Safety\t\t<\/a>\n\t<\/div>\n<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n<\/div><\/div>\n","protected":false},"excerpt":{"rendered":"

      The differences between those treated with FIRDAPSE and those treated with placebo were shown to be statistically significant based on…<\/p>\n","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"em_featured_video_id":0,"footnotes":""},"class_list":["post-44","page","type-page","status-publish","hentry"],"acf":[],"_links":{"self":[{"href":"https:\/\/firdapsehcp.com\/ca-lems\/wp-json\/wp\/v2\/pages\/44","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/firdapsehcp.com\/ca-lems\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/firdapsehcp.com\/ca-lems\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/firdapsehcp.com\/ca-lems\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/firdapsehcp.com\/ca-lems\/wp-json\/wp\/v2\/comments?post=44"}],"version-history":[{"count":0,"href":"https:\/\/firdapsehcp.com\/ca-lems\/wp-json\/wp\/v2\/pages\/44\/revisions"}],"wp:attachment":[{"href":"https:\/\/firdapsehcp.com\/ca-lems\/wp-json\/wp\/v2\/media?parent=44"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}