Study Summary
This trial will study the effects of inebilizumab in children 2-18 years old with NMOSD who have AQP4-IgG antibodies.
Eligible Conditions
- Neuromyelitis Optica Spectrum Disorders
Treatment Effectiveness
Phase-Based Effectiveness
Phase 2
Study Objectives
16 Primary · 8 Secondary · Reporting Duration: Baseline, Week 1, Week 2, Week 28, Week 80
Week 80
Change from Baseline in Body Temperature
Change from Baseline in Diastolic Blood Pressure
Change from Baseline in Hematology
Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell counts
Change from Baseline in Pulse Rate
Respiratory rate
Change from Baseline in Serum Chemistry
Change from Baseline in Serum Immunoglobulins
Change from Baseline in Systolic Blood Pressure
Week 28
Maximum Observed Concentration (Cmax) of inebilizumab
Week 80
Anti-drug antibody (ADA) rate.
Area Under the Concentration Versus Time Curve of inebilizumab from Time 0 extrapolated to infinity (AUC0-Inf)
Disease Activity: Annualized relapse rate.
Disease Activity: Proportion of relapse-free subjects.
Disease Activity: Time to first relapse.
Expanded Disability Status Scale change from baseline.
Health-Related Quality of Life (HRQoL) change from baseline in Euro Quality of Life-5 Dimension Youth score.
Health-Related Quality of Life (HRQoL) change from baseline in Pediatric Quality of Life Inventory.
Therapeutic procedure
Systemic Clearance (CL) of inebilizumab
Terminal Elimination Half-life (t½) of inebilizumab
Visual Acuity change from baseline.
Volume of Distribution at Steady State (VSS) of inebilizumab
Day 15
Area Under the Concentration Versus Time Curve of inebilizumab from Time 0 to 14 days post-dose (AUC0-14d)
Trial Safety
Phase-Based Safety
This is further along than 68% of similar trials
Side Effects for
Inebilizumab/Inebilizumab
23%Urinary tract infection
22%Nasopharyngitis
18%Headache
16%Upper respiratory tract infection
16%Arthralgia
14%Back pain
13%Infusion related reaction
10%Influenza
10%Cough
9%Diarrhoea
9%Nausea
7%Fatigue
7%Bronchitis
7%Hypoaesthesia
7%Paraesthesia
6%Anaemia
6%Fall
6%Insomnia
6%Pain in extremity
5%Vomiting
5%Eye pain
5%Rhinitis
5%Constipation
5%Pyrexia
5%Depression
4%Dizziness
4%Pruritus
4%Rash
3%Cystitis
3%Conjunctivitis
3%Oral herpes
3%Muscle spasms
3%Myalgia
3%Alopecia
3%Hypertension
2%Lymphopenia
2%Hordeolum
2%Peripheral swelling
2%Respiratory tract infection viral
2%Sinusitis
2%Dysaesthesia
2%Musculoskeletal pain
2%Neck pain
2%Nocturia
2%Oropharyngeal pain
2%Pneumonia
2%Neutropenia
2%Influenza like illness
2%Neuromyelitis optica spectrum disorder
2%Dry eye
2%Abdominal pain
2%Gastritis
2%Muscular weakness
2%Eczema
2%Rhinorrhoea
1%Eye pruritus
1%Facial pain
1%Gait disturbance
1%Dry mouth
1%Vision blurred
1%Abdominal discomfort
1%Pancreatitis acute
1%Adrenal insufficiency
1%Abdominal pain lower
1%Hepatic steatosis
1%Eye irritation
1%Enteritis
1%Enterocolitis
1%Chills
1%Hyperthyroidism
1%Vertigo
1%Abdominal pain upper
1%Dyspepsia
1%Irritable bowel syndrome
1%Ileus
1%Chest discomfort
1%Ear pain
1%Non-cardiac chest pain
1%Dental caries
1%Sensitivity of teeth
1%Drug intolerance
1%Eye inflammation
1%Discomfort
1%Hepatic function abnormal
1%Muscle abscess
1%Seasonal allergy
1%Bacterial vaginosis
1%Toothache
1%Pain
1%Asthenia
1%Malaise
1%Abscess limb
1%Breast abscess
1%Oedema peripheral
1%Gastroenteritis viral
1%Rash pustular
1%Periodontitis
1%Conjunctivitis viral
1%Fungal skin infection
1%Blood glucose fluctuation
1%Bacteriuria
1%Paronychia
1%Beta haemolytic streptococcal infection
1%Pharyngitis
1%Pharyngitis streptococcal
1%Tracheitis
1%Gingivitis
1%Viral rhinitis
1%Arthropod bite
1%Femur fracture
1%Ligament injury
1%Laryngitis
1%Respiratory tract infection
1%Tinea cruris
1%Viral infection
1%Blood cholesterol increased
1%Otitis externa
1%Eye contusion
1%Retinitis
1%Soft tissue infection
1%Viral pharyngitis
1%Laceration
1%Ligament sprain
1%Salpingitis
1%Urinary tract infection staphylococcal
1%Viral upper respiratory tract infection
1%Post-traumatic pain
1%Groin pain
1%Muscle spasticity
1%Bacterial test positive
1%Lymphocyte count decreased
1%Arthritis
1%Bone deformity
1%Intercostal neuralgia
1%Abnormal dreams
1%Nuclear magnetic resonance imaging abnormal
1%Road traffic accident
1%Gamma-glutamyltransferase increased
1%Alanine aminotransferase increased
1%Blood folate decreased
1%Eosinophil percentage increased
1%Neutrophil count increased
1%Cognitive disorder
1%Benign prostatic hyperplasia
1%Weight decreased
1%White blood cell count increased
1%Hypertriglyceridaemia
1%Flank pain
1%Limb discomfort
1%Crystal urine present
1%Ligament laxity
1%Spinal pain
1%Balance disorder
1%Myasthenia gravis
1%Facial paralysis
1%Yawning
1%Dry skin
1%Hyperlipidaemia
1%Migraine with aura
1%Restlessness
1%Menorrhagia
1%Chronic obstructive pulmonary disease
1%Blister
1%Coccydynia
1%Musculoskeletal stiffness
1%Allodynia
1%Hypokinesia
1%Migraine
1%Hypercholesterolaemia
1%Tendon pain
1%Pituitary tumour benign
1%Menstrual disorder
1%Epilepsy
1%Hypersomnia
1%Monoparesis
1%Depressed mood
1%Asthma
1%Erythema
1%Night sweats
1%Hiccups
1%Acne
1%Drug eruption
1%Hidradenitis
1%Tooth extraction
1%Hyperhidrosis
1%Psoriasis
1%Rash maculo-papular
1%Skin hyperpigmentation
1%Miliaria
1%Onychoclasis
1%Skin exfoliation
1%Urticaria
1%Leukopenia
1%Myelitis
1%Tinnitus
1%Arrhythmia
1%Neutrophilia
1%Ear pruritus
1%Leukocytosis
1%Skin abrasion
1%Procedural nausea
1%Body temperature increased
1%Liver function test increased
1%Adjustment disorder with depressed mood
1%Anxiety
1%Leukocyturia
1%Fibrocystic breast disease
1%Tension headache
1%Somnolence
1%Post-traumatic headache
1%Syncope
1%Tremor
1%Benign ovarian tumour
1%Peripheral nerve palsy
1%Appendicitis
1%Burns third degree
1%Connective tissue disorder
1%Myelitis transverse
1%Micturition urgency
1%Covid-19 pneumonia
1%Renal abscess
1%International normalised ratio increased
1%Optic neuritis
1%Calculus bladder
1%Deafness
1%Blindness unilateral
1%Atypical pneumonia
1%Cataract
1%Steroid withdrawal syndrome
1%Cholecystitis acute
1%Cholangitis acute
1%Covid-19
1%Cellulitis
1%Umbilical hernia
1%Chorioretinitis
1%Herpes zoster
1%Progressive multifocal leukoencephalopat
1%Bacteraemia
1%Central nervous system infection
1%Overdose
1%Colon cancer
1%Pneumonia bacterial
1%Foot fracture
1%Respiratory failure
1%Wrist fracture
1%Shock
1%Post cardiac arrest syndrome
1%Hypokalaemia
1%Hypotension
Awards & Highlights
No Placebo Group
All patients enrolled in this trial will receive the new treatment.
Trial Design
1 Treatment Group
Inebilizumab
1 of 1
Experimental Treatment
15 Total Participants · 1 Treatment Group
Primary Treatment: Inebilizumab · No Placebo Group · Phase 2
Inebilizumab
Drug
Experimental Group · 1 Intervention: Inebilizumab · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Inebilizumab
2015
Completed Phase 3
~240
Trial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, week 1, week 2, week 28, week 80
Who is running the clinical trial?
Horizon Therapeutics Ireland DACLead Sponsor
14 Previous Clinical Trials
1,499 Total Patients Enrolled
Nishi RampalStudy DirectorHorizon Therapeutics
Eligibility Criteria
Age 2 - 17 · All Participants · 0 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:Who else is applying?
How old are they?
| 18 - 65 | 100.0% |
What site did they apply to?
| UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building | 100.0% |
What portion of applicants met pre-screening criteria?
| Met criteria | 100.0% |
What state do they live in?
| New York | 100.0% |
How responsive is this trial?
Average response time
- < 1 Day
Typically responds via
| 100.0% | |
Most responsive sites:
- UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building: < 24 hours
Frequently Asked Questions
Am I eligible to partake in this investigation?
"The requirements to be accepted into this clinical trial are specific; potential participants must have neuromyelitis optica and fall between the ages of 2 and 17. The maximum capacity for enrolment is 15 individuals." - Anonymous Online Contributor
Unverified Answer
Does this research encompass participants older than two decades in age?
"This medical trial is seeking participants aged between 2 and 17 years old." - Anonymous Online Contributor
Unverified Answer
Is Inebilizumab considered a risk-free treatment option for patients?
"Preclinical research indicates that Inebilizumab is reasonably safe, thus warranting a score of 2. Despite this safety evidence, no clinical studies have been conducted to measure the efficacy of this drug." - Anonymous Online Contributor
Unverified Answer
Is the recruitment phase of this research still ongoing?
"Affirmative. According to clinicaltrials.gov, this medical experiment was posted on August 25th 2022 and is still currently recruiting participants; 15 patients are needed at 4 distinct sites." - Anonymous Online Contributor
Unverified Answer
How many individuals are partaking in this medical study?
"All in all, 15 compliant participants are necessary to keep the trial running. Horizon Therapeutics Ireland DAC will be managing it from several different sites including University of Texas Southwestern Medical Centre and Massachusetts General Hospital respectively located in Dallas and Lexington." - Anonymous Online Contributor
Unverified Answer
Are there extensive facilities that are presently conducting research with this study in North America?
"This study is being conducted at 4 separate medical institutions, such as the University of Texas Southwestern Medical Center in Dallas and Massachusetts General Hospital in Lexington. Additionally, UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building in La Jolla has also been enlisted along with other centres." - Anonymous Online Contributor
Unverified Answer