Clinical trial NCT03896295: An Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients With Generalized Myasthenia Gravis
Official title: An Open-label Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients With Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Generalized Myasthenia Gravis
Keywords: M281
Generalized Myasthenia Gravis
Lead sponsor: Momenta Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-03-29
Enrollment: 37
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the long-term safety and tolerability of M281 in participants with generalized myasthenia gravis (gMG)


Eligibility criteria: Participants must be ≥18 years of age with a documented history of Generalized Myasthenia Gravis (gMG) and clinical signs/symptoms of gMG, not pregnant or breastfeeding, previously participated in the MOM-281-004 study, had no major eligibility deviations or other major protocol deviations or not met any of the stopping criteria or discontinued study drug in the MOM-M281-004 study for any reason other than the need for rescue therapy as specified in the MOM-M281-004 study.

Additional, more specific criteria are defined in the protocol.

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: Up to 257 days post-baseline (Baseline is Day 1)
measure: Number of Participants With Serious Adverse Events (SAEs)
timeFrame: Up to 257 days post-baseline
measure: Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESIs)
timeFrame: Up to 257 days post-baseline
measure: Number of Participants With Treatment-emergent Abnormal Vital Signs
timeFrame: Up to 257 days post-baseline
measure: Number of Participants With Abnormalities in Physical Examinations
timeFrame: Week 12
measure: Change From Baseline in Chemistry Laboratory Parameters: Albumin and Protein
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Chemistry Laboratory Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglycerides, Urate and Urea Nitrogen
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Chemistry Laboratory Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, Lactate Dehydrogenase
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Chemistry Laboratory Parameters: Bilirubin, Creatinine and Direct Bilirubin
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Hematology Laboratory Parameter: Erythrocytes (Red Blood Cell)
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Hematology Laboratory Parameters: Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets and Leukocytes
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB)
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Hematology Laboratory Parameter: Erythrocytes Mean Corpuscular Volume
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Hematology Laboratory Parameter: Hematocrit
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Hematology Laboratory Parameter: Hemoglobin
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Urinalysis Laboratory Parameter: pH
timeFrame: Baseline up to Week 12
measure: Change From Baseline in Urinalysis Laboratory Parameter: Specific Gravity
timeFrame: Baseline up to Week 12
measure: Number of Participants With Treatment-emergent Abnormal Electrocardiograms (ECG) Values
timeFrame: Up to 257 days post-baseline
measure: Number of Participants With Columbia Suicide Severity Rating Scale (C-SSRS) Scores
timeFrame: Up to 257 days post-baseline
measure: Number of Participants With Below/Above Normal Values of Coagulation Laboratory Parameter
timeFrame: Up to 257 days post-baseline
Secondary outcomes:
measure: Change From Baseline in Total Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score Over Time
timeFrame: Baseline up to Weeks 4, 8, 12, 24, End of treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)
measure: Number of Participants With a 2-, 3-, 4-, 5-, 6-, 7-, or Greater Than or Equal to (>=) 8-point Improvement in Total MG-ADL Score Over Time
timeFrame: Weeks 4, 8, 12, 24, End of treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)
measure: Change From Baseline in Total Quantitative Myasthenia Gravis (QMG) Score Over Time
timeFrame: Baseline up to Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)
measure: Change From Baseline in Total Revised Myasthenia Gravis Quality of Life - 15 Scale (MG-QoL15r) Score Over Time
timeFrame: Baseline up to Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)
measure: Change From Baseline in Clinical Global Impression of Severity (CGI-S) Rating Score Over Time
timeFrame: Baseline up to Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)
measure: Number of Participants With Improvement of Illness Over Time Based on Clinical Global Impression of Improvement (CGI-I) Scale Score
timeFrame: Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)
measure: Number of Participants With Change From Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification Score Over Time
timeFrame: Weeks 8, 24 and End of Treatment (EoT) (up to 253 days post-baseline)
measure: Number of Participants With Anti-drug Antibodies (ADA) to Nipocalimab
timeFrame: Up to 257 days post-baseline
measure: Number of Participants With Neutralizing Antibodies (NAbs) to Nipocalimab
timeFrame: Up to 257 days post-baseline
measure: Change From Baseline in Serum Immunoglobulin (Ig)G Concentration Over Time
timeFrame: Baseline to Weeks 2, 4, 8, 12, 24, up to 253 days post-baseline, up to 257 days post-baseline
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Momenta Investigational Site
state: Arizona
city: Los Angeles
country: United States
facility: Momenta Investigational Site
state: California
city: Orange
country: United States
facility: Momenta Investigational Site
state: California
city: Stanford
country: United States
facility: Momenta Investigational Site
state: California
city: Aurora
country: United States
facility: Momenta Investigational Site
state: Colorado
city: New Haven
country: United States
facility: Momenta Investigational Site
state: Connecticut
city: Boca Raton
country: United States
facility: Momenta Investigational Site
state: Florida
city: Maitland
country: United States
facility: Momenta Investigational Site
state: Florida
city: Port Charlotte
country: United States
facility: Momenta Investigational Site
state: Florida
city: Saint Petersburg
country: United States
facility: Momenta Investigational Site
state: Florida
city: Augusta
country: United States
facility: Momenta Investigational Site
state: Georgia
city: Fairway
country: United States
facility: Momenta Investigational Site
state: Kansas
city: Boston
country: United States
facility: Momenta Investigational Site
state: Massachusetts
city: Boston
country: United States
facility: Momenta Investigational Site
state: Massachusetts
city: Boston
country: United States
facility: Momenta Investigational Site
state: Massachusetts
city: New Brunswick
country: United States
facility: Momenta Investigational Site
state: New Jersey
city: New York
country: United States
facility: Momenta Investigational Site
state: New York
city: Charlotte
country: United States
facility: Momenta Investigational Site
state: North Carolina
city: Durham
country: United States
facility: Momenta Investigational Site
state: North Carolina
city: Raleigh
country: United States
facility: Momenta Investigational Site
state: North Carolina
city: Cincinnati
country: United States
facility: Momenta Investigational Site
state: Ohio
city: Columbus
country: United States
facility: Momenta Investigational Site
state: Ohio
city: Cordova
country: United States
facility: Momenta Investigational Site
state: Tennessee
city: Austin
country: United States
facility: Momenta Investigational Site
state: Texas
city: Round Rock
country: United States
facility: Momenta Investigational Site
state: Texas
city: Leuven
country: Belgium
facility: Momenta Investigational Site
state: Vlaams Brabant
city: Antwerp
country: Belgium
facility: Momenta Investigational Site
city: Bruxelles
country: Belgium
facility: Momenta Investigational Site
city: Edmonton
country: Canada
facility: Momenta Investigational Site
state: Alberta
city: London
country: Canada
facility: Momenta Investigational Site
state: Ontario
city: Toronto
country: Canada
facility: Momenta Investigational Site
state: Ontario
city: Quebec City
country: Canada
facility: Momenta Investigational Site
state: Quebec
city: Gottingen
country: Germany
facility: Momenta Investigational Site
state: Niedersachsen
city: Dusseldorf
country: Germany
facility: Momenta Investigational Site
city: Gummersbach
country: Germany
facility: Momenta Investigational Site
city: Munster
country: Germany
facility: Momenta Investigational Site
city: Cefalu
country: Italy
facility: Momenta Investigational Site
city: Messina
country: Italy
facility: Momenta Investigational Site
city: Milano
country: Italy
facility: Momenta Investigational Site
city: Krakow
country: Poland
facility: Momenta Investigational Site
city: Lodz
country: Poland
facility: Momenta Investigational Site
city: Warsaw
country: Poland
facility: Momenta Investigational Site
city: Warsaw
country: Poland
facility: Momenta Investigational Site
city: Barcelona
country: Spain
facility: Momenta Investigational Site
state: Catalan
city: Barcelona
country: Spain
facility: Momenta Investigational Site
state: Catalan
city: Barcelona
country: Spain
facility: Momenta Investigational Site
state: Cataluna
city: L'hospitalet De Llobregat
country: Spain
facility: Momenta Investigational Site
state: Cataluna
city: San Sebastian
country: Spain
facility: Momenta Investigational Site
state: Gipuzkoa
city: Madrid
country: Spain
facility: Momenta Investigational Site
city: Madrid
country: Spain
facility: Momenta Investigational Site
city: Sevilla
country: Spain
facility: Momenta Investigational Site
city: Valencia
country: Spain
facility: Momenta Investigational Site
city: Birmingham
country: United Kingdom
facility: Momenta Investigational Site
city: Sheffield
country: United Kingdom
facility: Momenta Investigational Site
Study officials:
References: