Amyotrophic Lateral SclerosisPhase 2NCT04569435
Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing ANX005 in people with Amyotrophic Lateral Sclerosis, run by Annexon, Inc. at 8 sites in 2 countries.
Trial summary
This study is a multi-center, open-label study of intravenous (IV) ANX005 in participants with ALS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- ANX005
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 15 Jan 2021to 23 Apr 2024
- Phase
- Phase 2
- 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 ALS according to the World Federation of Neurology revised EI Escorial criteria.
- Onset of weakness within 3 years prior to Day 1 visit.
- Slow Vital Capacity ≥ 50% of predicted normal adjusted for sex, age, and height (from the sitting position).
- ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 at the Screening visit (Week -2).
- If female, must be postmenopausal, surgically sterilized, or childbearing potential must agree to use highly effective methods of contraception from Screening until 3 months after the last infusion with study medication.
- Males with a woman partner of childbearing potential must agree to use highly effective methods of contraception from Screening until Week until 3 months after the last infusion with study medication.
- Documented history of vaccinations within 5 years prior to Screening visit against encapsulated bacterial pathogens or willing to undergo vaccinations.
Who cannot join
- Clinically significant intercurrent illness, medical condition, or medical history that would jeopardize the safety of the participant, limit participation, or compromise the interpretation of the data derived from the participant.
- Participants with body weight > 150 kilograms.
- Antinuclear antibodies (ANA) titer ≥ 1:160 (for either of the 2 ANA results a minimum of 2 weeks apart) during the Screening Period.
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States4 sites
Annexon Investigational Site 02
Gainesville, Florida, 32608
Annexon Investigational Site 01
Orange, California, 9492868
Annexon Investigational Site 04
Phoenix, Arizona, 85013
Annexon Investigational Site 03
Tampa, Florida, 33612
Canada4 sites
Annexon Investigational Site 10
Edmonton, Alberta, T6G 2R7
Annexon Investigational Site 07
Montreal, Quebec, H2X 0A9
Annexon Investigational Site 08
Montreal, Quebec, H3A 2B4
Annexon Investigational Site 09
Toronto, Ontario, M4N 3M5
About this study
Official title: Phase 2a Safety, Tolerability, Pharmacokinetic (PK), and Pharmacodynamic (PD) Study of Intravenous ANX005 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
This study is a multi-center, open-label study of intravenous (IV) ANX005 in participants with ALS.
Read the detailed description
In this Phase 2a, multi-center, open label, proof-of-biology study, ANX005 will be administered to participants with ALS.
The study consists of approximately 22 weeks of treatment and approximately 14 weeks of follow-up. All participants will be contacted by phone 6 months after study completion.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- ANX005
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANX005Drug
IV Infusion
| Study group | Role | What they receive |
|---|---|---|
| ANX005 | Experimental | Participants will receive induction dosing of ANX005 on Days 1 and 5 or 6, followed by maintenance doses of ANX005 every 2 weeks up to Week 22. Receives: Drug: ANX005 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Experienced Treatment-Emergent Adverse Events
Measured: Up to Week 36
Secondary outcomes (4)
Serum Concentrations of ANX005
Measured: Up to Week 36
Maximum Observed Serum Concentration (Cmax) of ANX005
Measured: Up to Week 36
Area Under the ANX005 Serum Concentration-Time Curve
Measured: Day 1 and Week 22
Change from Baseline in Free Complement (C) 1q Serum Concentrations
Measured: Baseline, up to Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2020
- Study start15 Jan 2021
- Primary completion10 Jan 2024
- Study completion23 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Annexon, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Benjamin Hoehn, MD, PhD, Study director, Annexon, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04569435
- Sponsor's protocol ID
- ANX005-ALS-01
- Status last verified
- Jan 2025
- First submitted
- 23 Sep 2020
- Last updated
- 6 Jan 2025
- 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 6 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2a Safety, Tolerability, Pharmacokinetic (PK), and Pharmacodynamic (PD) Study of Intravenous ANX005 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT04569435
- Acronym / sponsor ID
- ANX005-ALS-01
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Annexon, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Jan 2021 (actual)
- Primary completion
- 10 Jan 2024 (actual)
- Study completion
- 23 Apr 2024 (actual)
- First posted
- 29 Sep 2020 (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 intravenous (IV) ANX005 in participants with ALS.
Detailed description
In this Phase 2a, multi-center, open label, proof-of-biology study, ANX005 will be administered to participants with ALS.
The study consists of approximately 22 weeks of treatment and approximately 14 weeks of follow-up. All participants will be contacted by phone 6 months after study completion.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ANX005 | Experimental | Drug: ANX005Participants will receive induction dosing of ANX005 on Days 1 and 5 or 6, followed by maintenance doses of ANX005 every 2 weeks up to Week 22. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of ALS according to the World Federation of Neurology revised EI Escorial criteria.
- Onset of weakness within 3 years prior to Day 1 visit.
- Slow Vital Capacity ≥ 50% of predicted normal adjusted for sex, age, and height (from the sitting position).
- ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 at the Screening visit (Week -2).
- If female, must be postmenopausal, surgically sterilized, or childbearing potential must agree to use highly effective methods of contraception from Screening until 3 months after the last infusion with study medication.
Key exclusion criteria (3 listed)
- Clinically significant intercurrent illness, medical condition, or medical history that would jeopardize the safety of the participant, limit participation, or compromise the interpretation of the data derived from the participant.
- Participants with body weight > 150 kilograms.
- Antinuclear antibodies (ANA) titer ≥ 1:160 (for either of the 2 ANA results a minimum of 2 weeks apart) during the Screening Period.
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 |
|---|---|---|
| ANX005IV Infusion | Drug | ANX005 |
Primary outcome
Number of Participants Who Experienced Treatment-Emergent Adverse Events
Time frame: Up to Week 36
4 secondary outcomes
Serum Concentrations of ANX005
Time frame: Up to Week 36
Maximum Observed Serum Concentration (Cmax) of ANX005
Time frame: Up to Week 36
Area Under the ANX005 Serum Concentration-Time Curve
Time frame: Day 1 and Week 22
Change from Baseline in Free Complement (C) 1q Serum Concentrations
Time frame: Baseline, up to Week 36
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 6 Jan 2025.
- 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.
Benjamin Hoehn, MD, PhD study director
Annexon, Inc.
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ANX005 in people with Amyotrophic Lateral Sclerosis, run by Annexon, Inc. at 8 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 7 inclusion and 3 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 6 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Number of Participants Who Experienced Treatment-Emergent Adverse Events (time frame: Up to Week 36). 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 6 Jan 2025.