Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

• Status: Recruiting
• Trial end date: 2024-09-13
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Horizon Therapeutics Ireland DAC

Criteria

Inclusion Criteria:

• Male or female subjects 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 subject 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