An Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects with Neuromyelitis Optica Spectrum Disorder
- Conditions
- Spinal cord and optic nerve disorder10041543
- Registration Number
- NL-OMON53937
- Lead Sponsor
- Horizon Therapeutics Ireland DAC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 2
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.
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Objectives:<br /><br>1. To characterize the PK of inebilizumab administered in pediatric subjects<br /><br>with NMOSD<br /><br>2. To characterize the PD of inebilizumab administered in pediatric subjects<br /><br>with NMOSD<br /><br>3. To assess the safety and tolerability of inebilizumab administered in<br /><br>pediatric subjects with NMOSD<br /><br>Endpoints:<br /><br>1. PK parameters, including maximum observed concentration, area under the<br /><br>concentration-time curve from time 0 to 14 days postdose and from time 0<br /><br>extrapolated to infinity, systemic clearance, terminal elimination half-life,<br /><br>and volume of distribution at steady state<br /><br>2. Cluster of differentiation 20 positive B-cell counts on Days 1, 8, 15, 29,<br /><br>57, 85, 113, 155, and 197<br /><br>3. Safety and tolerability assessments, including incidence of adverse events<br /><br>(AEs), serious AEs, and AEs of special interest, and changes in laboratory<br /><br>parameters and vital signs</p><br>
- Secondary Outcome Measures
Name Time Method