Inebilizumab: Inebilizumab administered intravenously (IV) over a total of 28 weeks.
Study summary
A Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible pediatric participants 2 to \< 18 years of age with recently active neuromyelitis optica spectrum disorder (NMOSD) who are seropositive for autoantibodies against aquaporin-4 (AQP4-immunoglobulin \[Ig\]G).
Eligibility
Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
Inclusion Criteria:
* Male or female participants, minimum body weight of 15 kg, age 2 to \< 18 years at the time of screening.
* Positive serum anti-AQP4-IgG result at screening and diagnosed with NMOSD according to the criteria of Wingerchuk et al, 2015.
* Documented history of one or more NMOSD acute relapses within the last year, or 2 or more NMOSD acute relapses within 2 years prior to screening.
Exclusion Criteria:
* Any condition that, in the opinion of the Investigator, would interfere with the evaluation or administration of the Investigational Product or interpretation of participant safety or study results.
* Concurrent/previous enrollment in another clinical study involving an investigational treatment within 4 weeks or 5 published half-lives of the investigational treatment, whichever is the longer, prior to Day 1.
* Evidence of significant hepatic, renal, or metabolic dysfunction or significant hematological abnormality (one repeat test may be conducted to confirm results within the same screening period).
* B-cell counts \< one-half of the lower limit of normal (LLN) for age according to the central laboratory.
* Receipt of the following at any time prior to Day 1:
1. Alemtuzumab
2. Total lymphoid irradiation
3. Bone marrow transplant
4. T-cell vaccination therapy
* Receipt of rituximab or any experimental B-cell depleting agent within 6 months prior to screening unless B-cell counts have returned to ≥ one-half the LLN.
* Receipt of intravenous immunoglobulin (IVIG) within one month prior to Day 1.
* Receipt of any of the following within 2 months prior to Day 1:
1. Cyclosporine
2. Methotrexate
3. Mitoxantrone
4. Cyclophosphamide
5. Tocilizumab
6. Satralizumab
7. Eculizumab
* Receipt of natalizumab (Tysabri®) within 6 months prior to Day 1.
* Severe drug allergic history or anaphylaxis to 2 or more food products or medicine (including known sensitivity to acetaminophen/paracetamol, diphenhydramine or equivalent antihistamine, and methylprednisolone or equivalent glucocorticoid).
* Diagnosed with a concurrent autoimmune disease that is uncontrolled (unless approved by the medical monitor).
* Recent receipt of live/attenuated vaccine or blood transfusion.
Receipt of any of the following:
1. Any live or attenuated vaccine within 4 weeks prior to Day 1 (administration of killed vaccines and nucleoside-modified mRNA-based vaccines is acceptable; the Sponsor recommends that Investigators ensure all participants are up to date on required vaccinations prior to study entry).
2. Bacillus Calmette Guérin vaccine within one year of screening.
3. Blood transfusion within 4 weeks prior to screening or during screening.
* Clinically significant serious active or chronic viral, bacterial, or fungal infection that requires treatment with anti-infectives, hospitalization, or, in the Investigator's opinion, represents an additional risk to the participant, within 2 months prior to Day 1.
* Known history of congenital or acquired immunodeficiency (e.g., due to human immunodeficiency virus \[HIV\] infection, splenectomy, immunosuppression-related or idiopathic T-cell deficiencies) that predisposes the participant to infection.
* Positive test for chronic hepatitis B infection at screening, defined as either:
a. Positive hepatitis B surface antigen (HBsAg), or b. Positive hepatitis B core (HBc) antibody (anti-HBc) plus negative hepatitis B surface (HBs) antibody (anti-HBs).
* Positive test for hepatitis C virus antibody.
* Negative test for varicella zoster virus (VZV)-IgG.
* History of cancer, apart from squamous cell or basal cell carcinoma of the skin treated with documented success of curative therapy \> 3 months prior to Day 1.
* History of active or latent tuberculosis (TB), or a positive QuantiFERON®-TB Gold test at screening, unless treatment for TB was completed per local guidelines. Participants with latent TB or a positive QuantiFERON®-TB Gold test who are actively on anti-TB treatment can enroll if they have completed at least one month of anti-TB treatment and intend to complete the full course of anti-TB treatment. Participants with an indeterminate QuantiFERON®-TB Gold test result can enroll if a repeat QuantiFERON®-TB Gold test is negative or a tuberculin skin test is negative.
* For participants who may undergo MRI scans:
1. Unable to undergo an MRI scan (e.g., hypersensitivity to Gd-containing MRI contrast agents, implanted pacemakers, defibrillators, or other metallic objects on or inside the body that limit performing MRI scans), or
2. Unable to tolerate or comply with the MRI procedure.
Primary outcome measure(s)
Maximum Observed Concentration (Cmax) of Inebilizumab — Day 1 to Week 28
Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 to 14 Days Post-dose (AUC0-14d) — Day 1 to pre-dose on Day 15
Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 Extrapolated to Infinity (AUC0-Inf) — Day 1 to Week 80
Systemic Clearance (CL) of Inebilizumab — Day 1 to Week 80
Terminal Elimination Half-life (t½) of Inebilizumab — Day 1 to Week 80
Volume of Distribution at Steady State (VSS) of Inebilizumab — Day 1 to Week 80
Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell Counts — Week 1, Week 2, Week 28, Week 80
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs) — Day 1 to Week 80
Change from Baseline in Serum Chemistry — Week 1, Week 2, Week 28, Week 80
Change from Baseline in Hematology — Week 1, Week 2, Week 28, Week 80
Change from Baseline in Serum Immunoglobulins — Week 1, Week 2, Week 28, Week 80
Change from Baseline in Systolic Blood Pressure — Week 1, Week 2, Week 28, Week 80
Change from Baseline in Diastolic Blood Pressure — Week 1, Week 2, Week 28, Week 80
Change from Baseline in Pulse Rate — Week 1, Week 2, Week 28, Week 80
Change from Baseline in Respiratory Rate — Week 1, Week 2, Week 28, Week 80
Change from Baseline in Body Temperature — Week 1, Week 2, Week 28, Week 80
Trial sites (19)
Facility
City
Region
Status
UCSD Altman Clinical and Translational Research Institute Building
La Jolla
California
Recruiting
Loma Linda University Children's Hospital - PIN
Loma Linda
California
Recruiting
Massachusetts General Hospital
Boston
Massachusetts
Recruiting
University of Texas Southwestern Medical Center
Dallas
Texas
Recruiting
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Parque Patricios
Ciudad Autónoma de BuenosAires
Recruiting
Hospital Santa Izabel-Rua Floriano Peixoto 300
Salvador
Estado de Bahia
Recruiting
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
Porto Alegre/RS
Brazil
Recruiting
CPQuali Pesquisa Clínica Sao Paulo
São Paulo
Brazil
Recruiting
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo
Brazil
Recruiting
Hospital For Sick Children
Toronto
Ontario
Recruiting
Centre Hospitalier Universitaire de Bicêtre
Le Kremlin-Bicêtre
Val-de-Marne
Recruiting
Erasmus MC Sophia Children's Hospital-Wytemaweg 80
Rotterdam
South Holland
Recruiting
Uniwersyteckie Centrum Kliniczne w Gdansku - Smoluchowskiego 17
Gdansk
Poland
Recruiting
Clinic for Neurology and Psychiatry for Children and Youth
Belgrade
Belgrade
Recruiting
Hospital Sant Joan de Deu - PIN
Espluges de Llobregat
Barcelona
Recruiting
Karolinska Universitetssjukhuset Solna
Stockholm
Stockholm County
Recruiting
Evelina London Children's Hospital
London
London, City of
Recruiting
Great Ormond Street Hospital - PPDS
London
London, City of
Recruiting
Birmingham Women's and Children's NHS Foundation Trust
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time on our Cookie Policy page. See also our Privacy Policy.