Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

February 22, 2024 updated by: Horizon Therapeutics Ireland DAC

An Open-Label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Approximately 15 subjects to be enrolled and receive Inebilizumab administered intravenously over 28 weeks. The maximum trial duration per participant is approximately 80 weeks, including up to 4 week screening period, 9 visits during a 28 week open-label treatment period, and approximately 4 visits during a 52 week follow-up period. Safety evaluations will be performed regularly throughout the course of the study.

Study Type

Interventional

Enrollment (Estimated)

15

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Argentina
    • Buenos Aires
      • Ciudad de Buenos Aires, Buenos Aires, Argentina, C 1245 AAM
        • Recruiting
        • Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
        • Contact:
        • Principal Investigator:
          • Andrea Giselle Savransky, MD
  • Brazil
      • Porto Alegre, Brazil, 90610-000
        • Recruiting
        • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
        • Contact:
        • Principal Investigator:
          • Bruna Klein, MD, PhD
      • São Paulo, Brazil, 05403-000
        • Recruiting
        • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
        • Contact:
        • Principal Investigator:
          • Barbosa Paolilo, MD
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G1X8
        • Not yet recruiting
        • Hospital for Sick Children
        • Contact:
        • Principal Investigator:
          • Ann Yeh, MD
  • France
    • Val-de-Marne
      • Le Kremlin-Bicêtre, Val-de-Marne, France, 94275
        • Recruiting
        • Centre Hospitalier Universitaire de Bicêtre
        • Contact:
        • Principal Investigator:
          • Deiva Kumaran, MD, PhD
  • Netherlands
    • Zuid Holland
      • Rotterdam, Zuid Holland, Netherlands, 3015 GD
        • Recruiting
        • Eramus MC Sophia Children Hospital
        • Contact:
        • Principal Investigator:
          • Rinze Neuteboom, MD
  • Poland
      • Pomorskie, Poland, 80-952
        • Recruiting
        • Uniwersyteckie Centrum Kliniczne
        • Contact:
        • Principal Investigator:
          • Maria Mazurkiewicz-Beldzinska, MD
  • Serbia
      • Belgrade, Serbia, 11000
        • Recruiting
        • Clinic for Neurology and Psychiatry for Children and Youth
        • Contact:
        • Principal Investigator:
          • Jasna Jancic, MD
  • Spain
    • Barcelona
      • Esplugues De Llobregat, Barcelona, Spain, 08950
        • Recruiting
        • Hospital Sant Joan de Déu
        • Contact:
        • Principal Investigator:
          • Thais Armengue Salvador, MD, PhD
  • Sweden
    • Stockolm
      • Stockholm, Stockolm, Sweden, 171 76
        • Recruiting
        • Astrid Lindgrens Barnsjukhus
        • Contact:
        • Principal Investigator:
          • Ronny Wickström, MD, PhD
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Recruiting
        • Great Ormond Street Hospital for Children
        • Contact:
        • Principal Investigator:
          • Cheryl Hemingway, MD
      • London, United Kingdom, SE17EH
        • Recruiting
        • Evelina Children's Hospital
        • Contact:
        • Principal Investigator:
          • Ming Lim, MD
  • United States
    • California
      • La Jolla, California, United States, 92037-1337
        • Not yet recruiting
        • UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building
        • Contact:
        • Principal Investigator:
          • Jennifer Sharon Graves, MD, PhD
      • Loma Linda, California, United States, 92354
        • Recruiting
        • Loma Linda University Children'S Hospital
        • Contact:
        • Principal Investigator:
          • Gregory Aaen, MD
    • Massachusetts
      • Lexington, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
        • Contact:
        • Principal Investigator:
          • Michael Levy, MD, PhD
    • Texas
      • Dallas, Texas, United States, 78701
        • Not yet recruiting
        • University of Texas Southwestern Medical Center
        • Contact:
        • Principal Investigator:
          • Cynthia Wang, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Inebilizumab
Infusion of Inebilizumab
Drug: Inebilizumab
Inebilizumab administered intravenously (IV) over a total of 28 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Maximum Observed Concentration (Cmax) of inebilizumab
Time Frame: Day 1 to Week 28
Day 1 to Week 28
Area Under the Concentration Versus Time Curve of inebilizumab from Time 0 to 14 days post-dose (AUC0-14d)
Time Frame: Day 1 to pre-dose on Day 15
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)
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Systemic Clearance (CL) of inebilizumab
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Terminal Elimination Half-life (t½) of inebilizumab
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Volume of Distribution at Steady State (VSS) of inebilizumab
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell counts
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80
Number of subjects with of treatment-emergent events (adverse events (TEAEs), serious adverse events (TESAEs), and adverse events of special interest (AESIs)).
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Change from Baseline in Serum Chemistry
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Hematology
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Serum Immunoglobulins
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Systolic Blood Pressure
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Diastolic Blood Pressure
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Pulse Rate
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Respiratory Rate
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Body Temperature
Time Frame: Baseline, Week 1, Week 2, Week 28, Week 80
Baseline, Week 1, Week 2, Week 28, Week 80

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Disease Activity: Time to first relapse.
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Disease Activity: Proportion of relapse-free subjects.
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Disease Activity: Annualized relapse rate.
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Health-Related Quality of Life (HRQoL) change from baseline in Euro Quality of Life-5 Dimension Youth score.
Time Frame: Day 1 to Week 80
Change in baseline for the 5 dimensions: mobility, looking after myself, doing usual activities, having pain or discomfort, and feeling worried, sad, or unhappy. A higher score indicates onset or worsening of an affective disorder.
Day 1 to Week 80
Health-Related Quality of Life (HRQoL) change from baseline in Pediatric Quality of Life Inventory.
Time Frame: Day 1 to Week 80
Change in baseline comprised from 4 generic core scales: Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. A higher score indicates a better quality of life.
Day 1 to Week 80
Visual Acuity change from baseline.
Time Frame: Day 1 to Week 80
Day 1 to Week 80
Expanded Disability Status Scale change from baseline.
Time Frame: Day 1 to Week 80
Change in baseline comprised from results of 7 Functional Systems: Visual, Brainstem, Pryamidal, Cerebellar, Sensory, Bowel, Bladder, and Cerebral. A higher score indicates a higher grade of impairment and disability.
Day 1 to Week 80
Anti-drug antibody (ADA) rate.
Time Frame: Day 1 to Week 80
Day 1 to Week 80

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Horizon Therapeutics Ireland DAC

Investigators

  • Study Director: Nishi Rampal, Horizon Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 25, 2022

Primary Completion (Estimated)

April 13, 2027

Study Completion (Estimated)

April 13, 2027

Study Registration Dates

First Submitted

July 22, 2022

First Submitted That Met QC Criteria

September 19, 2022

First Posted (Actual)

September 22, 2022

Study Record Updates

Last Update Posted (Estimated)

February 26, 2024

Last Update Submitted That Met QC Criteria

February 22, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VIB0551.P2.S2.NMO

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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