Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

Neuromyelitis Optica Spectrum Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05549258
• Other study ID #: VIB0551.P2.S2.NMO
• Status: Recruiting
• Phase: Phase 2
• Start date: August 25, 2022
• Est. completion date: April 13, 2027

Study information

• Verified date: May 2024
• Source: Amgen
• Contact: Amgen Call Center
• Phone: 866-572-6436
• Email: medinfo[@]amgen.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Acquired from Horizon in 2024.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: April 13, 2027
• Est. primary completion date: April 13, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Neuromyelitis Optica
• Neuromyelitis Optica Spectrum Disorder

Intervention

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

Locations

Country	Name	City	State
Netherlands	Erasmus MC Sophia Children's Hospital-Wytemaweg 80	Rotterdam	Zuid-Holland
Sweden	Karolinska Universitetssjukhuset Solna	Stockholm	Stockholms Lan
United Kingdom	Birmingham Women's and Children's NHS Foundation Trust	Birmingham	West Midlands
United Kingdom	Evelina London Children's Hospital	London	London, City Of
United States	Loma Linda University Children's Hospital	Loma Linda	California

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

Countries where clinical trial is conducted

United States,  Netherlands,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Maximum Observed Concentration (Cmax) of inebilizumab		Day 1 to Week 28
Primary	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
Primary	Area Under the Concentration Versus Time Curve of inebilizumab from Time 0 extrapolated to infinity (AUC0-Inf)		Day 1 to Week 80
Primary	Systemic Clearance (CL) of inebilizumab		Day 1 to Week 80
Primary	Terminal Elimination Half-life (t½) of inebilizumab		Day 1 to Week 80
Primary	Volume of Distribution at Steady State (VSS) of inebilizumab		Day 1 to Week 80
Primary	Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell counts		Baseline, Week 1, Week 2, Week 28, Week 80
Primary	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
Primary	Change from Baseline in Serum Chemistry		Baseline, Week 1, Week 2, Week 28, Week 80
Primary	Change from Baseline in Hematology		Baseline, Week 1, Week 2, Week 28, Week 80
Primary	Change from Baseline in Serum Immunoglobulins		Baseline, Week 1, Week 2, Week 28, Week 80
Primary	Change from Baseline in Systolic Blood Pressure		Baseline, Week 1, Week 2, Week 28, Week 80
Primary	Change from Baseline in Diastolic Blood Pressure		Baseline, Week 1, Week 2, Week 28, Week 80
Primary	Change from Baseline in Pulse Rate		Baseline, Week 1, Week 2, Week 28, Week 80
Primary	Change from Baseline in Respiratory Rate		Baseline, Week 1, Week 2, Week 28, Week 80
Primary	Change from Baseline in Body Temperature		Baseline, Week 1, Week 2, Week 28, Week 80
Secondary	Disease Activity: Time to first relapse.		Day 1 to Week 80
Secondary	Disease Activity: Proportion of relapse-free subjects.		Day 1 to Week 80
Secondary	Disease Activity: Annualized relapse rate.		Day 1 to Week 80
Secondary	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.	Day 1 to Week 80
Secondary	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.	Day 1 to Week 80
Secondary	Visual Acuity change from baseline.		Day 1 to Week 80
Secondary	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.	Day 1 to Week 80
Secondary	Anti-drug antibody (ADA) rate.		Day 1 to Week 80

External Links

•   AmgenTrials clinical trials website