Neuromyelitis Optica Spectrum Disorder
Conditions
Keywords
Immunoglobulins, B-cell count, inebilizumab, Uplizna
Brief summary
The purpose of this study is to understand the effects of long-term treatment with inebilizumab on circulating levels of immunoglobulins, B-cell counts, and other safety measures, and to further monitor repletion of immunoglobins and B-cell counts in participants with NMOSD who discontinue treatment. The objectives include: 1. To establish the nadir in circulating immunoglobulins (Ig) during chronic treatment with inebilizumab and ascertain the time needed to ensure restoration of pre-treatment baseline serum levels of IgG and IgM after discontinuation of treatment 2. To characterize B-cell counts throughout treatment with inebilizumab and after discontinuation until repletion of Immunoglobulin (Ig levels) 3. To assess long-term safety of inebilizumab 4. To assess other long-term effects of inebilizumab
Detailed description
This study aims to enroll 30 participants who either participated in the open-label period (OLP) of the N-MOmentum study (CD-IA-MEDI-551-1155; NCT02200770), a global registrational study that determined the safety and efficacy of inebilizumab for treatment of NMOSD, or who are newly initiating inebilizumab treatment at the discretion of their physician at participating sites. Acquired from Horizon in 2024.
Interventions
OTHERBlood tests
Participants will have blood tests done at each scheduled visit (approximately every 6 months).
DRUGInebilizumab
Participants with NMOSD who previously enrolled in N-MOmentum study, who participated for at least 2 years in the open label phase (OLP) of the study, and participants newly initiating inebilizumab treatment at the discretion of their treating physician will have hematology, chemistry, B-cell count, serum immunoglobulin (Ig) levels, adverse events, concomitant medications list, NMOSD attacks information, antidrug antibody (ADA) status and titers collected.
Sponsors
Amgen
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age 18 years or above, and able to provide written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act \[HIPAA\] in the United States of America (USA), European Union \[EU\] Data Privacy Directive in the EU) obtained from the subject/legal representative prior to performing any protocol-related procedures, including screening evaluations. 2. Have completed at least 2 years in the open-label period of the N-MOmentum study or are newly initiating inebilizumab treatment at a participating site. 3. Females of childbearing potential who are sexually active with a nonsterilized male partner must use a highly effective method of contraception as outlined in the protocol (subjects in the Czech Republic only must use 1 additional method of contraception) from screening, and must agree to continue using such precautions for 6 months after treatment with inebilizumab; cessation of contraception after this point should be discussed with a responsible physician. 4. Nonsterilized males who are sexually active with a female partner of childbearing potential must use a male condom plus spermicide (subjects in the Czech Republic only must use 1 additional method of contraception) from Day 1 for 3 months after receipt of last treatment with inebilizumab. 5. Sterilized males, without the appropriate post-vasectomy documentation on the absence of sperm in the ejaculate, who are sexually active with a female partner of childbearing potential must use a condom and spermicide from Day 1 for 3 months after receipt of the last treatment with inebilizumab.
Exclusion criteria
1. Have any condition that would place the participant at unacceptable risk of complications, interfere with evaluation of inebilizumab or confound the interpretation of participant safety or study results. 2. Received rituximab or any other B-cell depleting agent after exit from N-MOmentum study, or within the last 12 months prior to screening for non N-MOmentum participants. 3. Known history of allergy or reaction to any component of inebilizumab formulation or history of anaphylaxis following any biologic therapy 4. Have a severe clinically significant infection, including active chronic infection such as hepatitis B 5. Have active or untreated latent tuberculosis 6. Have a history of progressive multifocal leukoencephalopathy (PML) 7. Is severely immunocompromised state 8. Have active malignancies
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in serum Ig levels (total Ig, IgG, IgM, IgA, IgE) over time | Up to 42 months | This will be assessed via serum samples, drawn at the site and processed at a central laboratory, and analyses performed to assess change from baseline over time, as measured by each parameter in mg/dL |
| Change from baseline in peripheral CD20+ B-cell counts over time | Up to 42 months | This will be assessed via serum samples, drawn at the site and processed at a central laboratory, and analyses performed to assess change from baseline over time, as measured by each parameter in cells/mL |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of serious infections | Up to 42 months | — |
| Incidence of viral reactivation | Up to 42 months | — |
| Incidence of progressive multifocal leukoencephalopathy (PML) | Up to 42 months | — |
| Incidence of other opportunistic infections | Up to 42 months | — |
| Change from baseline in hematology over time | Up to 42 months | The lab parameters being assessed as part of the hematology analyses include the following: platelet count, red blood cell count, other indices of red blood cells, including MCV (mean corpuscle volume), MCH (mean corpuscle hemoglobin), % reticulocyte count, and morphology (shape); white blood count with differential, including neutrophil count, Lymphocyte count, monocyte count, eosinophils, and basophils; hemoglobin and hematocrit. |
| Incidence of infusion reactions | Up to 42 months | — |
| Number of protocol-defined NMOSD attacks | Up to 42 months | — |
| Percentage of protocol-defined NMOSD attacks | Up to 42 months | — |
| Incidence of Anti-drug antibody (ADA) directed against Inebilizumab status and titers | Up to 42 months | — |
| Incidence of malignancies | Up to 42 months | — |
| Change from baseline in clinical chemistry over time | Up to 42 months | The lab parameters being assessed as part of clinical chemistry include blood urea nitrogen (BUN), creatinine, aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase. |
Countries
China, Czechia, Hungary, Poland, South Korea, Thailand, United States
Outcome results
None listed