IMLIFIDASE for Neuromyelitis optica spectrum disorder (Devic disease)

Inclusion criteria

• {"criterion_text":"- 1.\tSigned lnformed Consent obtained before any study-related procedures.\n- 2.\tWillingness and ability to comply with the protocol.\n- 3.\tMale or female aged 􀀪18 years at the time of screening.\n- 4.\tNMOSD diagnosed according to the Wingerchuck criteria[4] with a positive anti-AQP4 lgG serum test using a cell-based assay at presentation or in medical history.\n- 5.\tOnset of weakness or loss of visual acuity due to the exacerbation of NMOSD is not more than 14 days prior to administration of imlifidase.\n- 6.\tExacerbation of myelitis is associated with an increase in functional system motor score of at least 1 point, and requires at least bilateral assistance to walk; exacerbation of uni­or bilateral optie neuritis is associated with an increase in functional system visual score of at least 1 point, and results in a visual acuity of 20/60 to 20/99 (0.33-0.21) or worse.\n- 7.\tAcute steroid treatment is indicated.\n- 8.\tIncident cases or prevalent cases treated with maintenance/ prophylactic therapies including azathioprine, mycophenolate mofetil/mycophenol acid, and rituximab, or no maintenance treatment.\n- 9.\tNegative serological screening test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus.\n- 10.\tWamen of child-bearing potential willing or able to use at least one highly effective contraceptive method from the day of treatment until at least 6 months after the dose of imlifidase if not abstinent. In the context of this study, an effective method is defined as those which result in low failure rate (i.e. less than 1 % per year) when used consistently and correctly\n- 11.\tMen willing to use double-barrier contraception from the day of treatment until at least 2 months after the dose of imlifidase if not abstinent."}

Exclusion criteria

• {"criterion_text":"- 1.\tPrevious treatment with imlifidase\n- 2.\tSubjects who are already on plasma exchange.\n- 3.\tlntravenous immunoglobulin (IVlg) treatment S28 days prior to administration of imlifidase\n- 4.\tWamen of child-bearing potential unwilling or unable to use at least one highly effective contraceptive method from the screening visit until at least 180 days following imlifidase dosing.\n- 5.\tSigns or symptoms suggestive of Thrombotic Thrombocytopenic Purpura (TTP).\n- 6.\tHypersensitivity to IVlg or to any of the excipients.\n- 7.\tSubject known to have a severe concurrent disease, e.g. malignancy, severe cardiovascular disease and severe chronic obstructive pulmonary disease.\n- 8.\tAny condition that in the opinion of the investigator could increase the subject's risk by participating in the study or confound the outcome of the study.\n- 9.\tKnown mental incapacity or language barriers precluding adequate understanding of the lnformed Consent information and the study activities.\n- 10.\tSubjects with clinical signs of ongoing infectious diseases that requires treatment.\n- 11.\tSubjects with active SARS-CoV-2 (COVID-19) infection as shown by PCR\n- 12.\tSubjects should not have received other investigational drugs within 5 half-lives prior to imlifidase dosing."}

Primary endpoints

• {"endpoint_text":"- The proportion of participants with a depletion of circulating pathogenie anti-AQP4 lgG antibodies, below detection limits as measured with a state-of-the-arts cell-based assay in the timeframe within 6h after treatment.","definition_or_measurement_approach":"Measured as the proportion with anti-AQP4 IgG antibodies below detection limits using a state-of-the-art cell-based assay within 6 hours after imlifidase treatment."}

Netherlands

Earliest CTIS Part Ii Submission Date

04-09-2024

Latest Decision Or Authorization Date

10-09-2024

Processing Time Days

6

Number Of Sites

1

Number Of Participants

5

Primary sponsor

Full Name

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands