Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

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

Sponsors

Lead Sponsor: Horizon Therapeutics Ireland DAC

Source Horizon Pharma Ireland, Ltd., Dublin Ireland
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.

Overall Status Recruiting
Start Date 2022-08-25
Completion Date 2024-09-13
Primary Completion Date 2024-09-13
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Maximum Observed Concentration (Cmax) of inebilizumab Day 1 to Week 28
Area Under the Concentration Versus Time Curve of inebilizumab from Time 0 to 14 days post-dose (AUC0-14d) 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) Day 1 to Week 80
Systemic Clearance (CL) of inebilizumab Day 1 to Week 80
Terminal Elimination Half-life (t½) of inebilizumab Day 1 to Week 80
Volume of Distribution at Steady State (VSS) of inebilizumab Day 1 to Week 80
Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell counts 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)). Day 1 to Week 80
Change from Baseline in Serum Chemistry Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Hematology Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Serum Immunoglobulins Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Systolic Blood Pressure Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Diastolic Blood Pressure Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Pulse Rate Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Respiratory Rate Baseline, Week 1, Week 2, Week 28, Week 80
Change from Baseline in Body Temperature Baseline, Week 1, Week 2, Week 28, Week 80
Secondary Outcome
Measure Time Frame
Disease Activity: Time to first relapse. Day 1 to Week 80
Disease Activity: Proportion of relapse-free subjects. Day 1 to Week 80
Disease Activity: Annualized relapse rate. Day 1 to Week 80
Health-Related Quality of Life (HRQoL) change from baseline in Euro Quality of Life-5 Dimension Youth score. Day 1 to Week 80
Health-Related Quality of Life (HRQoL) change from baseline in Pediatric Quality of Life Inventory. Day 1 to Week 80
Visual Acuity change from baseline. Day 1 to Week 80
Expanded Disability Status Scale change from baseline. Day 1 to Week 80
Anti-drug antibody (ADA) rate. Day 1 to Week 80
Enrollment 15
Condition
Intervention

Intervention Type: Drug

Intervention Name: Inebilizumab

Description: Inebilizumab administered intravenously (IV) over a total of 28 weeks.

Arm Group Label: Inebilizumab

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: 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

Gender:

All

Minimum Age:

2 Years

Maximum Age:

17 Years

Healthy Volunteers:

No

Overall Official
Last Name Role Affiliation
Nishi Rampal Study Director Horizon Therapeutics
Overall Contact

Last Name: Horizon Therapeutics

Phone: 1-866-479-6742

Email: [email protected]

Location
Facility: Status: Contact: Investigator:
UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building | La Jolla, California, 92037-1337, United States Not yet recruiting Jacqueline Shanley 858-246-2905 [email protected] Jennifer Sharon Graves, MD, PhD Principal Investigator
Massachusetts General Hospital | Lexington, Massachusetts, 02114, United States Not yet recruiting Santiago Pardo 617-726-7565 [email protected] Michael Levy, MD, PhD Principal Investigator
University of Texas Southwestern Medical Center | Dallas, Texas, 78701, United States Not yet recruiting Tricia Plumb 214-456-2464 [email protected] Cynthia Wang, MD Principal Investigator
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan | Ciudad de Buenos Aires, Buenos Aires, C 1245 AAM, Argentina Not yet recruiting Christian Pires 5(491)167606273 [email protected] Silvia Tenembaum, MD Principal Investigator
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS) | Porto Alegre, 90610-000, Brazil Not yet recruiting Aline de Cassia Santos +555192306920 [email protected] Bruna Klein, MD, PhD Principal Investigator
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo | São Paulo, 05403-000, Brazil Not yet recruiting Neusa Sakita +551126618676 [email protected] Jose Paz, MD Principal Investigator
Hospital For Sick Children | Toronto, Ontario, M5G1X8, Canada Not yet recruiting Hareem Ilyas (416) 813-7654 [email protected] Ann Yeh, MD Principal Investigator
Centre Hospitalier Universitaire de Bicêtre | Le Kremlin-Bicêtre, Val-de-Marne, 94275, France Recruiting Milanthika Nanediri +33145213014 [email protected] Deiva Kumaran, MD, PhD Principal Investigator
Uniwersyteckie Centrum Kliniczne | Pomorskie, 80-952, Poland Not yet recruiting Katarzyna Karpinska +48583492331 [email protected] Maria Mazurkiewicz-Beldzinska, MD Principal Investigator
Clinic for Neurology and Psychiatry for Children and Youth | Belgrade, 11000, Serbia Not yet recruiting Biljana Milic 3(816)42444837 [email protected] Jasna Jancic, MD Principal Investigator
Hospital Sant Joan de Deu | Esplugues De Llobregat, Barcelona, 08950, Spain Not yet recruiting Marta Sanchez +34673918086 [email protected] Thais Armengue Salvador, MD, PhD Principal Investigator
Astrid Lindgrens Barnsjukhus | Stockholm, Stockolm, 171 76, Sweden Not yet recruiting Fanny Deak +46 70 409 67 069 [email protected] Ronny Wickström, MD, PhD Principal Investigator
Evelina Children's Hospital | London, SE17EH, United Kingdom Not yet recruiting Shelley Mieres +4402071887188 [email protected] Ming Lim, MD Principal Investigator
Great Ormond Street Hospital for Children | London, WC1N 3JH, United Kingdom Not yet recruiting Christopher Jackson +442074059200 0439 [email protected] Cheryl Hemingway, MD Principal Investigator
Location Countries

Argentina

Brazil

Canada

France

Poland

Serbia

Spain

Sweden

United Kingdom

United States

Verification Date

2022-09-01

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: Inebilizumab

Type: Experimental

Description: Infusion of Inebilizumab

Patient Data No
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

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