Back to resultsStudy of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
Primary Purpose
Neuromyelitis Optica Spectrum Disorder
Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Inebilizumab
Sponsored by
About this trial
This is an interventional treatment trial for Neuromyelitis Optica Spectrum Disorder
Eligibility 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:
- Alemtuzumab
- Total lymphoid irradiation
- Bone marrow transplant
- 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:
- Cyclosporine
- Methotrexate
- Mitoxantrone
- Cyclophosphamide
- Tocilizumab
- Satralizumab
- 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
Sites / Locations
- UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building
- Loma Linda University Children's Hospital
- Massachusetts General HospitalRecruiting
- University of Texas Southwestern Medical Center
- Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. GarrahanRecruiting
- Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)Recruiting
- Hospital das Clínicas da Faculdade de Medicina da Universidade de São PauloRecruiting
- Hospital For Sick Children
- Centre Hospitalier Universitaire de BicêtreRecruiting
- Uniwersyteckie Centrum KliniczneRecruiting
- Clinic for Neurology and Psychiatry for Children and YouthRecruiting
- Hospital Sant Joan de DeuRecruiting
- Astrid Lindgrens BarnsjukhusRecruiting
- Evelina Children's HospitalRecruiting
- Great Ormond Street Hospital for ChildrenRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Inebilizumab
Arm Description
Infusion of Inebilizumab
Outcomes
Primary Outcome Measures
Maximum Observed Concentration (Cmax) of inebilizumab
Area Under the Concentration Versus Time Curve of inebilizumab from Time 0 to 14 days post-dose (AUC0-14d)
Area Under the Concentration Versus Time Curve of inebilizumab from Time 0 extrapolated to infinity (AUC0-Inf)
Systemic Clearance (CL) of inebilizumab
Terminal Elimination Half-life (t½) of inebilizumab
Volume of Distribution at Steady State (VSS) of inebilizumab
Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell counts
Number of subjects with of treatment-emergent events (adverse events (TEAEs), serious adverse events (TESAEs), and adverse events of special interest (AESIs)).
Change from Baseline in Serum Chemistry
Change from Baseline in Hematology
Change from Baseline in Serum Immunoglobulins
Change from Baseline in Systolic Blood Pressure
Change from Baseline in Diastolic Blood Pressure
Change from Baseline in Pulse Rate
Change from Baseline in Respiratory Rate
Change from Baseline in Body Temperature
Secondary Outcome Measures
Disease Activity: Time to first relapse.
Disease Activity: Proportion of relapse-free subjects.
Disease Activity: Annualized relapse rate.
Health-Related Quality of Life (HRQoL) change from baseline in Euro Quality of Life-5 Dimension Youth score.
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.
Health-Related Quality of Life (HRQoL) change from baseline in Pediatric Quality of Life Inventory.
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.
Visual Acuity change from baseline.
Expanded Disability Status Scale change from baseline.
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.
Anti-drug antibody (ADA) rate.
Full Information
NCT ID
NCT05549258
First Posted
July 22, 2022
Last Updated
October 3, 2023
Sponsor
Horizon Therapeutics Ireland DAC
1. Study Identification
Unique Protocol Identification Number
NCT05549258
Brief Title
Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
Official Title
An Open-Label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 25, 2022 (Actual)
Primary Completion Date
April 13, 2027 (Anticipated)
Study Completion Date
April 13, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Horizon Therapeutics Ireland DAC
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief 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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuromyelitis Optica Spectrum Disorder
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Inebilizumab
Arm Type
Experimental
Arm Description
Infusion of Inebilizumab
Intervention Type
Drug
Intervention Name(s)
Inebilizumab
Intervention Description
Inebilizumab administered intravenously (IV) over a total of 28 weeks.
Primary Outcome Measure Information:
Title
Maximum Observed Concentration (Cmax) of inebilizumab
Time Frame
Day 1 to Week 28
Title
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
Title
Area Under the Concentration Versus Time Curve of inebilizumab from Time 0 extrapolated to infinity (AUC0-Inf)
Time Frame
Day 1 to Week 80
Title
Systemic Clearance (CL) of inebilizumab
Time Frame
Day 1 to Week 80
Title
Terminal Elimination Half-life (t½) of inebilizumab
Time Frame
Day 1 to Week 80
Title
Volume of Distribution at Steady State (VSS) of inebilizumab
Time Frame
Day 1 to Week 80
Title
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
Title
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
Title
Change from Baseline in Serum Chemistry
Time Frame
Baseline, Week 1, Week 2, Week 28, Week 80
Title
Change from Baseline in Hematology
Time Frame
Baseline, Week 1, Week 2, Week 28, Week 80
Title
Change from Baseline in Serum Immunoglobulins
Time Frame
Baseline, Week 1, Week 2, Week 28, Week 80
Title
Change from Baseline in Systolic Blood Pressure
Time Frame
Baseline, Week 1, Week 2, Week 28, Week 80
Title
Change from Baseline in Diastolic Blood Pressure
Time Frame
Baseline, Week 1, Week 2, Week 28, Week 80
Title
Change from Baseline in Pulse Rate
Time Frame
Baseline, Week 1, Week 2, Week 28, Week 80
Title
Change from Baseline in Respiratory Rate
Time Frame
Baseline, Week 1, Week 2, Week 28, Week 80
Title
Change from Baseline in Body Temperature
Time Frame
Baseline, Week 1, Week 2, Week 28, Week 80
Secondary Outcome Measure Information:
Title
Disease Activity: Time to first relapse.
Time Frame
Day 1 to Week 80
Title
Disease Activity: Proportion of relapse-free subjects.
Time Frame
Day 1 to Week 80
Title
Disease Activity: Annualized relapse rate.
Time Frame
Day 1 to Week 80
Title
Health-Related Quality of Life (HRQoL) change from baseline in Euro Quality of Life-5 Dimension Youth score.
Description
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.
Time Frame
Day 1 to Week 80
Title
Health-Related Quality of Life (HRQoL) change from baseline in Pediatric Quality of Life Inventory.
Description
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.
Time Frame
Day 1 to Week 80
Title
Visual Acuity change from baseline.
Time Frame
Day 1 to Week 80
Title
Expanded Disability Status Scale change from baseline.
Description
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.
Time Frame
Day 1 to Week 80
Title
Anti-drug antibody (ADA) rate.
Time Frame
Day 1 to Week 80
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility 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:
Alemtuzumab
Total lymphoid irradiation
Bone marrow transplant
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:
Cyclosporine
Methotrexate
Mitoxantrone
Cyclophosphamide
Tocilizumab
Satralizumab
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Horizon Therapeutics
Phone
1-866-479-6742
Email
clinicaltrials@horizontherapeutics.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Nishi Rampal
Organizational Affiliation
Horizon Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building
City
La Jolla
State/Province
California
ZIP/Postal Code
92037-1337
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jacqueline Shanley
Phone
858-246-2905
Email
jshanley@health.ucsd.edu
First Name & Middle Initial & Last Name & Degree
Jennifer Sharon Graves, MD, PhD
Facility Name
Loma Linda University Children's Hospital
City
Loma Linda
State/Province
California
ZIP/Postal Code
92354
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jaclyn Lopez
Phone
909-558-5830
Email
janlopez@llu.edu
First Name & Middle Initial & Last Name & Degree
Gregory Aaen, MD
Facility Name
Massachusetts General Hospital
City
Lexington
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Santiago Pardo
Phone
617-726-7565
Email
spardo1@mgh.harvard.edu
First Name & Middle Initial & Last Name & Degree
Michael Levy, MD, PhD
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
78701
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tricia Plumb
Phone
214-456-2464
Email
Patricia.plumb@utsouthwestern.edu
First Name & Middle Initial & Last Name & Degree
Cynthia Wang, MD
Facility Name
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
City
Ciudad de Buenos Aires
State/Province
Buenos Aires
ZIP/Postal Code
C 1245 AAM
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Christian Pires
Phone
5(491)167606273
Email
estudiosclinicos.coordinacion@gmail.com
First Name & Middle Initial & Last Name & Degree
Andrea Giselle Savransky, MD
Facility Name
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
City
Porto Alegre
ZIP/Postal Code
90610-000
Country
Brazil
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aline de Cassia Santos
Phone
+555192306920
Email
alinecassia@insightbt.com.br
First Name & Middle Initial & Last Name & Degree
Bruna Klein, MD, PhD
Facility Name
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
City
São Paulo
ZIP/Postal Code
05403-000
Country
Brazil
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Neusa Sakita
Phone
+551126618676
Email
Neusa.sakita@hc.fm.usp.br
First Name & Middle Initial & Last Name & Degree
Barbosa Paolilo, MD
Facility Name
Hospital For Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G1X8
Country
Canada
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hareem Ilyas
Phone
(416) 813-7654
Email
Hareem.ilyas@sickkids.ca
First Name & Middle Initial & Last Name & Degree
Ann Yeh, MD
Facility Name
Centre Hospitalier Universitaire de Bicêtre
City
Le Kremlin-Bicêtre
State/Province
Val-de-Marne
ZIP/Postal Code
94275
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Milanthika Nanediri
Phone
+33145213014
Email
milanthika.nanediri@aphp.fr
First Name & Middle Initial & Last Name & Degree
Deiva Kumaran, MD, PhD
Facility Name
Uniwersyteckie Centrum Kliniczne
City
Pomorskie
ZIP/Postal Code
80-952
Country
Poland
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Katarzyna Karpinska
Phone
+48583492331
Email
kkarpinska@uck.gda.pl
First Name & Middle Initial & Last Name & Degree
Maria Mazurkiewicz-Beldzinska, MD
Facility Name
Clinic for Neurology and Psychiatry for Children and Youth
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Biljana Milic
Phone
3(816)42444837
Email
biljana.milich@gmail.com
First Name & Middle Initial & Last Name & Degree
Jasna Jancic, MD
Facility Name
Hospital Sant Joan de Deu
City
Esplugues De Llobregat
State/Province
Barcelona
ZIP/Postal Code
08950
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marta Sanchez
Phone
+34673918086
Email
msanchezo@sjd.es
First Name & Middle Initial & Last Name & Degree
Thais Armengue Salvador, MD, PhD
Facility Name
Astrid Lindgrens Barnsjukhus
City
Stockholm
State/Province
Stockolm
ZIP/Postal Code
171 76
Country
Sweden
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Fanny Deak
Phone
+46 70 409 67 069
Email
fanny.deak@regionstockholm.se
First Name & Middle Initial & Last Name & Degree
Ronny Wickström, MD, PhD
Facility Name
Evelina Children's Hospital
City
London
ZIP/Postal Code
SE17EH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Shelley Mieres
Phone
+4402071887188
Email
Shelley.mieres@gstt.nhs.uk
First Name & Middle Initial & Last Name & Degree
Ming Lim, MD
Facility Name
Great Ormond Street Hospital for Children
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Christopher Jackson
Phone
+442074059200
Ext
0439
Email
Christopher.jackson@gosh.nhs.uk
First Name & Middle Initial & Last Name & Degree
Cheryl Hemingway, MD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
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