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An Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients With Generalized Myasthenia Gravis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03896295
Recruitment Status : Terminated (Study was originally halted due to the COVID-19 pandemic. The study was later terminated prematurely as the participants will have the option to enter into an open-label extension portion of a planned future study. It was not due to safety concerns.)
First Posted : March 29, 2019
Results First Posted : February 16, 2022
Last Update Posted : February 16, 2022
Sponsor:
Information provided by (Responsible Party):
Momenta Pharmaceuticals, Inc.

Study Description
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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)

Condition or disease Intervention/treatment Phase
Generalized Myasthenia Gravis Drug: M281 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 37 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : August 6, 2019
Actual Primary Completion Date : December 9, 2020
Actual Study Completion Date : December 9, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: M281 Drug: M281
M281 injection administered as intravenous infusion


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Up to 257 days post-baseline (Baseline is Day 1) ]
    Number of participants with TEAEs were reported. An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as any AE occurring during or after the initiation of the first infusion of study drug in this study.

  2. Number of Participants With Serious Adverse Events (SAEs) [ Time Frame: Up to 257 days post-baseline ]
    An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. SAE is defined as any AE occurring at any dose that results in any of the following outcomes: death, life-threatening AE, hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect.

  3. Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 257 days post-baseline ]
    Number of participants with treatment-emergent AESIs were reported. Severe infections and hypoalbuminemia (Grade 3 or higher according to the Common Terminology Criteria for Adverse Events [CTCAE] v5.0) were considered as AESIs.

  4. Number of Participants With Treatment-emergent Abnormal Vital Signs [ Time Frame: Up to 257 days post-baseline ]
    Number of participants with treatment-emergent abnormal vital signs including pulse rate (less than or equal to [<=] 50 beats per minutes [bpm] with greater than or equal to [>=] 15 bpm decrease from baseline, >= 120 bpm with >=15 bpm increase from baseline), systolic blood pressure (SBP) (<= 90 millimeters of mercury [mmHg] with >= 20 mmHg decrease from baseline, >= 160 mmHg with >= 20 mmHg increase from baseline) and diastolic blood pressure (DBP) (<= 50 mmHg with >=15 mmHg decrease from baseline, >=100 mmHg with >=15 mmHg decrease from baseline) were reported.

  5. Number of Participants With Abnormalities in Physical Examinations [ Time Frame: Week 12 ]
    Number of participants with abnormalities in physical examinations (abdomen, head, ears, eyes, nose, throat, and sinuses, lungs, neurological, skin, blood and lymphatic system, cardiovascular, chest, gastrointestinal, general appearance and musculoskeletal) were reported.

  6. Change From Baseline in Chemistry Laboratory Parameters: Albumin and Protein [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in chemistry laboratory parameters: albumin and protein were reported.

  7. Change From Baseline in Chemistry Laboratory Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglycerides, Urate and Urea Nitrogen [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in chemistry laboratory parameters: bicarbonate, calcium, chloride, cholesterol, glucose, phosphate, potassium, sodium, triglycerides, urate and urea nitrogen were reported.

  8. Change From Baseline in Chemistry Laboratory Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, Lactate Dehydrogenase [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in chemistry laboratory parameters alanine aminotransferase (ALT), alkaline phosphatase, aspartate aminotransferase (AST), creatine kinase, gamma glutamyl transferase, lactate dehydrogenase were reported.

  9. Change From Baseline in Chemistry Laboratory Parameters: Bilirubin, Creatinine and Direct Bilirubin [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in chemistry laboratory parameters: bilirubin, creatinine and direct bilirubin were reported.

  10. Change From Baseline in Hematology Laboratory Parameter: Erythrocytes (Red Blood Cell) [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in erythrocytes (red blood cells) (hematology laboratory parameter) was reported.

  11. Change From Baseline in Hematology Laboratory Parameters: Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets and Leukocytes [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in hematology laboratory parameters: basophils, eosinophils, lymphocytes, monocytes, neutrophils, platelets and leukocytes were reported.

  12. Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in erythrocytes mean corpuscular hemoglobin (HGB) concentration (hematology laboratory parameter) were reported.

  13. Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB) [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in erythrocytes mean corpuscular HGB (hematology laboratory parameter) was reported.

  14. Change From Baseline in Hematology Laboratory Parameter: Erythrocytes Mean Corpuscular Volume [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in erythrocytes mean corpuscular volume (hematology laboratory parameter) was reported.

  15. Change From Baseline in Hematology Laboratory Parameter: Hematocrit [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in hematocrit (hematology laboratory parameter) was reported.

  16. Change From Baseline in Hematology Laboratory Parameter: Hemoglobin [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in hemoglobin (hematology laboratory parameter) was reported.

  17. Change From Baseline in Urinalysis Laboratory Parameter: pH [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in pH (urinalysis laboratory parameter) was reported.

  18. Change From Baseline in Urinalysis Laboratory Parameter: Specific Gravity [ Time Frame: Baseline up to Week 12 ]
    Change from baseline in specific gravity (urinalysis laboratory parameter) was reported.

  19. Number of Participants With Treatment-emergent Abnormal Electrocardiograms (ECG) Values [ Time Frame: Up to 257 days post-baseline ]
    Number of participants with treatment-emergent abnormal ECG values for variables including mean heart rate (abnormally low refers to less than or equal to [<=] 50 beats per minute [bpm], abnormally high refers greater than or equal to [>=] 120 bpm), PR interval (abnormally low refers to < 120 and abnormally high refers to >200 milliseconds [msec]), RR interval (abnormally low refers to <600 msec and abnormally high refers to >1200 msec) and QRS duration (abnormally > 120) were reported.

  20. Number of Participants With Columbia Suicide Severity Rating Scale (C-SSRS) Scores [ Time Frame: Up to 257 days post-baseline ]
    Number of participants with C-SSRS scores were reported. C-SSRS is a clinician-administered questionnaire designed to solicit occurrence, severity, and frequency of suicide-related ideation and behaviors. Total score ranges from 1 to 10, score of 0 was assigned (0="no event that can be assessed based on C-SSRS"). Higher total scores indicate greater severity. Maximum score assigned for each participant was summarized into one of 3 categories: no suicidal ideation or behavior (0), suicidal ideation (1 to 5): higher score indicates more suicidal ideation, suicidal behavior (6 to 10): higher score indicates more suicidal behavior. Suicidal ideation includes participants who did not have suicidal ideation or behavior at baseline and had suicidal ideation without behavior at some time point post-baseline. Suicidal behavior includes participants who did not have suicidal ideation or behavior at baseline and had suicidal behavior at some time point post-baseline (baseline=Day 1).

  21. Number of Participants With Below/Above Normal Values of Coagulation Laboratory Parameter [ Time Frame: Up to 257 days post-baseline ]
    Number of participants with at least one value above upper limit of normal (>ULN) or below the lower limit of normal (< LLN) value of coagulation parameters (activated partial thromboplastin time [APTT] and prothrombin time [PT]) were reported. The lab reference range for APTT is 25.1 to 36.5 seconds. The lab reference range for PT is 9.4 to 12.5 seconds.


Secondary Outcome Measures :
  1. Change From Baseline in Total Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score Over Time [ Time Frame: 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) ]
    The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities.

  2. 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 [ Time Frame: Weeks 4, 8, 12, 24, End of treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline) ]
    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 were reported. MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities.

  3. Change From Baseline in Total Quantitative Myasthenia Gravis (QMG) Score Over Time [ Time Frame: 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) ]
    The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment.

  4. Change From Baseline in Total Revised Myasthenia Gravis Quality of Life - 15 Scale (MG-QoL15r) Score Over Time [ Time Frame: 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) ]
    The MG-QoL15r was used to assess the participant's limitations related to living with MG. It consists of 15 questions and each of the 15 questions were rated by the participant on a 3-point scale (0= Not at all, 1= somewhat, 2=very much) based on a recall period of "over the past few weeks". The total score is the sum of the 15 question scores and ranges from 0 to 30. Higher scores indicated more limitation.

  5. Change From Baseline in Clinical Global Impression of Severity (CGI-S) Rating Score Over Time [ Time Frame: 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) ]
    The CGI-S scale is the clinician/physician's global assessment of participants illness severity of MG and is rated by answering on 8-point scale. Considering total clinical experience, participant is assessed on severity of illness according to: 0=not performed; 1=normal, not at all ill; 2=borderline illness; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6=severely ill; 7=among most extremely ill patients. Higher scores indicated more severity of illness. Values of 0 (not assessed) were excluded from analysis. CGI-S permits global evaluation of participant's condition at given time.

  6. Number of Participants With Improvement of Illness Over Time Based on Clinical Global Impression of Improvement (CGI-I) Scale Score [ Time Frame: Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline) ]
    Number of participants with improvement of illness based on CGI-I scale score over time were reported. The CGI-I scale is the clinician/physician's global assessment of the change in severity of the patient's generalized myasthenia gravis (gMG) since starting this study. The rating is given on a 7-point scale with lower scores indicating greater improvement (1= Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = No change; 5 =Minimally worse; 6 = Much worse; 7 = Very much worse. Values of 0 (not assessed) were excluded from analysis. Higher score indicates more severity.

  7. Number of Participants With Change From Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification Score Over Time [ Time Frame: Weeks 8, 24 and End of Treatment (EoT) (up to 253 days post-baseline) ]
    Number of participants with change from baseline in MGFA classification score over time were reported. The MGFA was used to assess the participant's MG severity. MGFA classification identifies the subgroup participants with MG who share distinct clinical features or severity of disease: Class I (ocular MG), classes II, III and IV generalized MG with mild, moderate and severe disease, respectively; Class V MG crisis. Separate subclasses under classes II, III and IV are designed: "a" if the predominant weakness is affecting limb/axial weakness or both; subclass "b" if the predominant weakness is affecting oropharyngeal or respiratory muscles or both. In the MGFA classification, lower roman numerals mean less severity. Changes in MGFA classification (regardless of subclass) are categorized as "Improved" (example, III to II), "Same" (example, II to II), or "Worsened" (example, II to III).

  8. Number of Participants With Anti-drug Antibodies (ADA) to Nipocalimab [ Time Frame: Up to 257 days post-baseline ]
    Number of participants with ADA to nipocalimab were reported.

  9. Number of Participants With Neutralizing Antibodies (NAbs) to Nipocalimab [ Time Frame: Up to 257 days post-baseline ]
    Number of participants with NAbs were reported. Samples positive for ADA in this study could not be further analyzed for neutralizing antibodies (NAbs) to nipocalimab due to limited number of participants developed ADA.

  10. Change From Baseline in Serum Immunoglobulin (Ig)G Concentration Over Time [ Time Frame: Baseline to Weeks 2, 4, 8, 12, 24, up to 253 days post-baseline, up to 257 days post-baseline ]
    Change from baseline in serum immunoglobulin (Ig)G concentration over time was reported.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

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): NCT03896295


Locations
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Sponsors and Collaborators
Momenta Pharmaceuticals, Inc.
  Study Documents (Full-Text)

Documents provided by Momenta Pharmaceuticals, Inc.:
Study Protocol  [PDF] August 1, 2019
Statistical Analysis Plan  [PDF] July 7, 2021

More Information
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Responsible Party: Momenta Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03896295    
Other Study ID Numbers: MOM-M281-005
2018-003618-41 ( EudraCT Number )
First Posted: March 29, 2019    Key Record Dates
Results First Posted: February 16, 2022
Last Update Posted: February 16, 2022
Last Verified: January 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Momenta Pharmaceuticals, Inc.:
M281
Generalized Myasthenia Gravis
Additional relevant MeSH terms:
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Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
 Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases