Clinical trial NCT04701164: Efficacy and Safety of ANX005 in Subjects With Guillain-Barré Syndrome
Official title: A Randomized, Double-blind, Placebo-controlled Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ANX005 in Subjects With Guillain-Barré Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Guillain-Barre Syndrome
Lead sponsor: Annexon, Inc.
Sponsor class: INDUSTRY
First posted: 2021-01-08
Enrollment: 242
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is intended to evaluate the efficacy and safety of ANX005 administered by intravenous (IV) infusion to participants recently diagnosed with Guillain-Barré Syndrome (GBS). The total duration of study participation is approximately 6 months.


Eligibility criteria: 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 ≤10 days prior to start of infusion.
* GBS-DS score of 3,4, or 5 at screening and at Day 1 prior to infusion.

Exclusion Criteria:

* Clinically significant findings on the screening electrocardiogram (ECG), laboratory test results, or physical examination that are not specific to GBS that may interfere with the conduct of the study, the interpretation of the data, or increase the participant's risk.
* Body weight \<30 kilograms (kg) or \>150 kg at screening.
* Unresponsive (inexcitable) nerve conduction study results in all nerves tested during screening.
* Previous or intended treatment with either plasma exchange or intravenous immunoglobulin for GBS.
* Diagnosis of a variant of GBS, including Miller Fisher syndrome, Bickerstaff's encephalitis, and overlap syndromes.
* History of prior episode of GBS.
* GBS-related weakness that improved since symptom onset, including an improvement between screening and Day 1 (baseline).

Primary outcomes:
measure: GBS Disability Score (GBS-DS) at Week 8
timeFrame: Week 8
measure: Number of Participants with Adverse Events
timeFrame: Through Month 6
Secondary outcomes:
measure: Medical Research Council (MRC) Sum Score at Week 8
timeFrame: Week 8
measure: MRC Sum Score at Day 8
timeFrame: Day 8
measure: Duration (Days) of Ventilation Support Over 26 Weeks
timeFrame: 26 weeks
measure: GBS Disability Score (GBS-DS)
timeFrame: Week 26
measure: Number of Participants Requiring Intensive Care Unit Stay
timeFrame: 26 weeks
measure: Duration (Days) of Intensive Care Unit Stays
timeFrame: 26 weeks
measure: Patient Global Impression of Change Scores
timeFrame: Week 8 and Week 26
Other outcomes:
Locations:
city: Dhaka
country: Bangladesh
facility: Dhaka Medical Center
city: Dhaka
country: Bangladesh
facility: National Institute of Neurosciences and Hospital (NINS)
city: Baguio
country: Philippines
facility: Baguio General Hospital Medical Center
city: Batangas
country: Philippines
facility: Batangas Medical Center
city: Batangas
country: Philippines
facility: Mary Mediatrix Medical Center
city: Cebu City
country: Philippines
facility: Perpetual Succour Hospital
city: Cotabato
country: Philippines
facility: Cotabato Regional Medical Center
city: Davao
country: Philippines
facility: Southern Philippines Medical Center
city: Manila
country: Philippines
facility: Jose R. Reyes Memorial Medical Center
city: Manila
country: Philippines
facility: West Visayas State University Medical Center
city: Zamboanga
country: Philippines
facility: Zamboanga City Medical Center
Study officials:
affiliation: Annexon, Inc.
name: Clinical Trials
role: STUDY_DIRECTOR
References: