Guillain-Barré Syndrome
Conditions
Brief summary
This study is a multi center, open-label, study of ANX005 in combination with IVIg in subjects diagnosed with GBS.
Detailed description
The study will enroll approximately 12 subjects into one cohort, with subjects receiving 75 mg/kg of ANX005, a dose shown to be safe and well-tolerated as monotherapy and that provides a meaningful level of exposure to be tested in combination with IVIg. Dosing of IVIg will be administered as is the current standard of care (0.4 g/kg/day x 5 days) and ANX005 will be administered concurrently, beginning on Day 1 or 2 of the treatment period. Subjects will be followed for 6 months after treatment for observation and evaluation.
Interventions
DRUGANX005
investigational drug
DRUGIntravenous immunoglobulin
investigational drug
Sponsors
ResearchPoint Global
International Centre for Diarrhoeal Disease Research, Bangladesh
Annexon, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open label combination treatment
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosis of GBS according to the National Institute of Neurological Disorders and Stroke Diagnostic Criteria for Guillain Barré Syndrome * Onset of GBS-related weakness ≤14 days prior to infusion * GBS-DS score of 3, 4, or 5
Exclusion criteria
* Clinically significant findings that may interfere with the conduct of the study or the interpretation of the data * Be at risk of suicide or self-harm * Received previous treatment with plasma exchange for GBS * Any diagnosis of a variant of GBS * Have a history of anaphylaxis or severe systemic response to immunoglobulin * Documented, clinically significant, pre-existing polyneuropathy from another cause * Clinically significant intercurrent illness, medical condition, or medical history * History of chronic use of steroid or immunosuppressant medication * Active alcohol, drug, or substance abuse * Females who are pregnant, breast feeding, or unable or unwilling to use highly effective methods of contraception throughout the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's | 6 months | As measured by incidence of TEAEs, SAEs, AE's related to ANX005, SAE's related to ANX005, Grade 3 or higher AEs, Grade 3 or higher AEs related to ANX005, AEs leading to study or treatment discontinuation. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics of ANX005 when administered in combination with IVIg | 3 months | As measured by ANX005 serum concentrations |
| Pharmacodynamics of ANX005 when administered in combination with IVIg | 4 months | As measured by CH50 and C1q serum concentrations |
| Values and change from baseline in Guillain-Barré Syndrome Disability Score (GBS-DS) by visit | 6 months | The 6-point Guillain-Barré Syndrme Disability Score (GBS-DS) is a widely accepted and easily obtainable scoring system used to assess functional status of GBS subjects. The score is as follows: 0 = Healthy, 1 = Minor symptoms and capable of running, 2 = Able to walk independently 10 meters or more but unable to run, 3=Able to walk more than 10 meters across an open space with help, 4 = Bedridden or chair bound, 5 = Needing mechanical ventilation, 6 = death |
Countries
Bangladesh, Denmark
Outcome results
None listed