Guillain-Barré SyndromePhase 1NCT04035135
A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barré Syndrome (GBS)
A Phase 1 study testing ANX005 and Intravenous immunoglobulin in people with Guillain-Barré Syndrome, run by Annexon, Inc. at 2 sites in 2 countries.
Trial summary
This study is a multi center, open-label, study of ANX005 in combination with IVIg in subjects diagnosed with GBS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Guillain-Barré Syndrome
- What is being tested?
- ANX005 and Intravenous immunoglobulin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 29 Jan 2020to 19 May 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
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
This study is a multi center, open-label, study of ANX005 in combination with IVIg in subjects diagnosed with GBS.
Read the 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.
- Conditions
- Guillain-Barré Syndrome
- Being tested
- ANX005, Intravenous immunoglobulin
- Medical subject headings
- Guillain-Barre Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANX005Drug
Also called Anti C1q Antibody
investigational drug
Intravenous immunoglobulinDrug
Also called IVIg
investigational drug
| Study group | Role | What they receive |
|---|---|---|
| Open Label Treatment Arm | Experimental | One (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days. Receives: Drug: ANX005; Drug: Intravenous immunoglobulin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months
Secondary outcomes (3)
Pharmacokinetics of ANX005 when administered in combination with IVIg
As measured by ANX005 serum concentrations
Measured: 3 months
Pharmacodynamics of ANX005 when administered in combination with IVIg
As measured by CH50 and C1q serum concentrations
Measured: 4 months
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
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2019
- Study start29 Jan 2020
- Primary completion19 May 2021
- Study completion19 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Guillain-Barré SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label combination treatment
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Annexon, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ResearchPoint Global, International Centre for Diarrhoeal Disease Research, Bangladesh
- Responsible party
- Sponsor
- Investigators
- Henk-André Kroon, MD, MBA, Study director, Annexon, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04035135
- Sponsor's protocol ID
- ANX005-GBS-03
- Status last verified
- Aug 2021
- First submitted
- 11 Jul 2019
- Last updated
- 11 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04035135
- Acronym / sponsor ID
- ANX005-GBS-03
- Conditions
- Guillain-Barré Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Annexon, Inc.Industry (pharma, biotech, devices)
- Collaborators
- ResearchPoint Global, International Centre for Diarrhoeal Disease Research, Bangladesh
- Study start
- 29 Jan 2020 (actual)
- Primary completion
- 19 May 2021 (actual)
- Study completion
- 19 May 2021 (actual)
- First posted
- 29 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label combination treatment
| Arm | Type | Receives |
|---|---|---|
| Open Label Treatment Arm | Experimental | Drug: ANX005, Drug: Intravenous immunoglobulinOne (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ANX005Also: Anti C1q Antibodyinvestigational drug | Drug | Open Label Treatment Arm |
| Intravenous immunoglobulinAlso: IVIginvestigational drug | Drug | Open Label Treatment Arm |
Primary outcome
Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's
Time frame: 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.
3 secondary outcomes
Pharmacokinetics of ANX005 when administered in combination with IVIg
Time frame: 3 months
Pharmacodynamics of ANX005 when administered in combination with IVIg
Time frame: 4 months
Values and change from baseline in Guillain-Barré Syndrome Disability Score (GBS-DS) by visit
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Henk-André Kroon, MD, MBA study director
Annexon, Inc.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ANX005 and Intravenous immunoglobulin in people with Guillain-Barré Syndrome, run by Annexon, Inc. at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Bangladesh, Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2021.