euromyelitis Optica Spectrum Disorder Inebilizumab Study in Children and Adolescents

Phase 1

Conditions: euromyelitis optica spectrum disorder (NMOSD
also known as Devic's syndrome and previously known as neuromyelitis optica [NMO])
MedDRA version: 21.1Level: PTClassification code 10077875Term: Neuromyelitis optica spectrum disorderSystem Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2021-003528-33-SE

Lead Sponsor: Horizon Therapeutics Ireland DAC

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 15

Inclusion Criteria

1. Written informed consent and any locally required data privacy authorization obtained from the subject’s legally authorized representative in accordance with regional laws or regulations and the subject’s assent, when applicable, prior to performing any protocol-related procedures.
2. Male or female subjects, minimum body weight of 15 kg, age 2 to < 18 years at the time of screening.
3. Positive serum anti-AQP4-IgG result at screening (verified by the central laboratory) and diagnosed with NMOSD
4. 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.
5. Female subjects of childbearing potential who are sexually active with a nonsterilized male partner must agree to use a highly effective method of contraception from screening until 6 months after the final dose of investigational product (IP)
6. Nonsterilized male subjects who are sexually active with a female partner of childbearing potential must agree to use a male condom from Day 1 until 3 months after the final dose of IP.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Any condition that, in the opinion of the Investigator, would interfere with the evaluation or administration of the IP or interpretation of subject safety or study results
2. 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
3. Females who are breastfeeding, pregnant, or who intend to become pregnant at any time from screening until 6 months after the final dose of IP
4. Known history of allergy or reaction to any component of the IP formulation or history of anaphylaxis following any biologic therapy
5. Evidence of alcohol, drug, or chemical abuse, or a recent history of such abuse < 1 year prior to Day 1
6. Major surgery within 8 weeks prior to screening
7. Spontaneous or induced abortion, still or live birth, or pregnancy = 4 weeks prior to screening
8. Evidence of significant hepatic, renal, or metabolic dysfunction or significant hematological abnormality
9. 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
10. Receipt of intravenous immunoglobulin (IVIG) within one month prior to Day 1
11. Receipt of particular immunosuppressive therapy within 2 months prior to Day 1
12. Receipt of natalizumab (Tysabri®) within 6 months prior to Day 1
13. Severe drug allergic history or anaphylaxis to 2 or more food products or medicine
14. Diagnosed with a concurrent autoimmune disease that is uncontrolled (unless approved by the medical monitor)
15. Receipt of any of the following:
a. Any live or attenuated vaccine within 4 weeks prior to Day 1
b. Bacillus Calmette-Guérin vaccine within one year of screening
c. Blood transfusion within 4 weeks prior to screening or during screening
16. Clinically significant serious active or chronic viral, bacterial, or fungal infection that requires treatment with anti-infectives, within 2 months prior to Day 1
17. Known history of congenital or acquired immunodeficiency that predisposes the subject to infection
18. Positive test for chronic hepatitis B infection at screening
19. Positive test for hepatitis C virus antibody
20. Negative test for varicella zoster virus (VZV)-IgG
21. 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
22. History of active or latent tuberculosis
23. For subjects who may undergo MRI scans: Unable to undergo an MRI scan (eg, 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 unable to tolerate or comply with the MRI procedure.