Back to results

A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barré Syndrome (GBS)

Primary Purpose

Guillain-Barré Syndrome

Status

Completed

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

ANX005

Intravenous immunoglobulin

About this trial

This is an interventional treatment trial for Guillain-Barré Syndrome

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

National Institute of Neurosciences and Hospital
Aarhus University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open Label Treatment Arm

Arm Description

One (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days.

Outcomes

Primary Outcome Measures

Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's

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 Outcome Measures

Pharmacokinetics of ANX005 when administered in combination with IVIg

As measured by ANX005 serum concentrations

Pharmacodynamics of ANX005 when administered in combination with IVIg

As measured by CH50 and C1q serum concentrations

Values and change from baseline in Guillain-Barré Syndrome Disability Score (GBS-DS) by visit

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

Full Information

NCT ID

NCT04035135

First Posted

July 11, 2019

Last Updated

August 9, 2021

Sponsor

Annexon, Inc.

Collaborators

ResearchPoint Global, International Centre for Diarrhoeal Disease Research, Bangladesh

1. Study Identification

Unique Protocol Identification Number

NCT04035135

Brief Title

A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barré Syndrome (GBS)

Official Title

A Phase 1b Study to Evaluate the Safety, Tolerability and Drug-Drug Interactions of ANX005 and Intravenous Immunoglobulin (IVIg) in Subjects With Guillain Barré Syndrome

Study Type

Interventional

2. Study Status

Record Verification Date

August 2021

Overall Recruitment Status

Completed

Study Start Date

January 29, 2020 (Actual)

Primary Completion Date

May 19, 2021 (Actual)

Study Completion Date

May 19, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Annexon, Inc.

Collaborators

ResearchPoint Global, International Centre for Diarrhoeal Disease Research, Bangladesh

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Guillain-Barré Syndrome

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Model Description

Open label combination treatment

Masking

None (Open Label)

Allocation

N/A

Enrollment

14 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Open Label Treatment Arm

Arm Type

Experimental

Arm Description

One (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days.

Intervention Type

Drug

Intervention Name(s)

ANX005

Other Intervention Name(s)

Anti C1q Antibody

Intervention Description

investigational drug

Intervention Type

Drug

Intervention Name(s)

Intravenous immunoglobulin

Other Intervention Name(s)

IVIg

Intervention Description

investigational drug

Primary Outcome Measure Information:

Title

Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's

Description

Time Frame

6 months

Secondary Outcome Measure Information:

Title

Pharmacokinetics of ANX005 when administered in combination with IVIg

Description

As measured by ANX005 serum concentrations

Time Frame

3 months

Title

Pharmacodynamics of ANX005 when administered in combination with IVIg

Description

As measured by CH50 and C1q serum concentrations

Time Frame

4 months

Title

Values and change from baseline in Guillain-Barré Syndrome Disability Score (GBS-DS) by visit

Description

Time Frame

6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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 ≤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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Henk-André Kroon, MD, MBA

Organizational Affiliation

Annexon, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

National Institute of Neurosciences and Hospital

City

Dhaka

Country

Bangladesh

Facility Name

Aarhus University Hospital

City

Aarhus

Country

Denmark

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barré Syndrome (GBS)

We'll reach out to this number within 24 hrs