Amyotrophic Lateral SclerosisPhase 2NCT05053035

A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS

A Phase 2 study testing AL001 in people with Amyotrophic Lateral Sclerosis, run by Alector Inc. at 3 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated 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?: AL001
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 5 participants
Where: 3 sitesUnited States
Study dates: 2 Sep 2021to 28 Oct 2022
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Confirmation of C9orf72 mutation
Diagnosis of ALS by revised El Escorial criteria
Time since onset of muscle weakness due to ALS ≤36 months at the time of the Screening Visit
Slow Vital Capacity (VC) ≥50% of predicted capacity at the time of the Screening Visit
If taking riluzole, must be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole naive participants are allowed.
If taking edaravone, must have completed at least one cycle of edaravone prior to the Screening Visit and plan to continue edaravone during the study. Edaravone naive participants are allowed.
Females must not be pregnant, breastfeeding or planning to conceive within the study period. Males must agree to use acceptable contraception
Capable of providing informed consent at the Screening visit and complying with study procedures throughout the study

Who cannot join

Clinically significant, unstable, medical condition (other than ALS)
Clinically significant heart disease, liver disease or kidney disease
Cognitive impairment or dementia
Current uncontrolled hypertension
History of unresolved cancer
Any experimental gene therapy
Any experimental vaccine (any vaccine against COVID-19 either approved or administered under an Emergency Use Authorization is allowed)

Trial locations and contacts

3 sites in 1 country.

United States3 sites

Massachusetts General Hospital
Boston, Massachusetts, 02114
Washington University School of Medicine
St Louis, Missouri, 63110
University of South Florida
Tampa, Florida, 33612

Other trials in United States

About this study

Official title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in C9orf72-Associated Amyotrophic Lateral Sclerosis

A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.

Read the detailed description

This is a phase 2 double-blind, placebo-controlled trial to test the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 in participants with C9orf72-associated Amyotrophic Lateral Sclerosis.

Conditions: Amyotrophic Lateral Sclerosis
Being tested: AL001, Placebo
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.

AL001Drug
Administered via intravenous (IV) infusion
PlaceboDrug
Administered via intravenous (IV) infusion

Study groups in NCT05053035
Study group	Role	What they receive
AL001	Experimental	AL001 every 4 weeks Receives: Drug: AL001
Placebo	Placebo comparator	Placebo every 4 weeks Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events
Count of participants with adverse events during the study treatment period
Measured: 24 weeks
Immunogenicity of AL001
Count of participants positive for Anti-drug Antibodies (ADAs) to AL001 at week 24
Measured: Week 24
Pharmacokinetics (PK) of AL001 in Serum
Concentration of AL001 in Serum at week 24
Measured: Week 24
Pharmacokinetics (PK) of AL001 in CSF
Concentration of AL001 in Cerebrospinal fluid (CSF) at week 24
Measured: Week 24
Change From Baseline in Plasma Progranulin
Evaluate the change from baseline to week 24 in plasma progranulin levels
Measured: 24 weeks
Change From Baseline in CSF Progranulin
Evaluate the change from baseline to week 24 in Cerebrospinal fluid (CSF) progranulin levels
Measured: 24 weeks

Secondary outcomes (2)

Change From Baseline in Plasma Neurofilament Light Chain
Evaluate the change from baseline to week 24 in plasma neurofilament light chain levels
Measured: 24 weeks
Change From Baseline in CSF Neurofilament Light Chain
Evaluate change from baseline to week 24 in Cerebrospinal fluid (CSF) neurofilament light chain levels
Measured: 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

2 Sep 202128 Oct 2022

First posted22 Sep 2021
Study start2 Sep 2021
Primary completion28 Oct 2022
Study completion28 Oct 2022

Why it stopped: The Sponsor is considering a subsequent study in ALS, potentially with different inclusion/exclusion criteria.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
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: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 5 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05053035
Sponsor's protocol ID: AL001-ALS-201
Status last verified: May 2025
First submitted: 1 Sep 2021
Last updated: 18 Jun 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
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All Amyotrophic Lateral Sclerosis trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in C9orf72-Associated Amyotrophic Lateral Sclerosis
ClinicalTrials.gov ID: NCT05053035
Acronym / sponsor ID: AL001-ALS-201
Conditions: Amyotrophic Lateral Sclerosis
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: The Sponsor is considering a subsequent study in ALS, potentially with different inclusion/exclusion criteria.
Enrolment: 5 participants (actual)
Lead sponsor: Alector Inc.Industry (pharma, biotech, devices)
Study start: 2 Sep 2021 (actual)
Primary completion: 28 Oct 2022 (actual)
Study completion: 28 Oct 2022 (actual)
First posted: 22 Sep 2021 (actual)
Results: Posted 18 Jun 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
AL001	Experimental	Drug: AL001AL001 every 4 weeks
Placebo	Placebo comparator	Drug: PlaceboPlacebo every 4 weeks

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

Confirmation of C9orf72 mutation
Diagnosis of ALS by revised El Escorial criteria
Time since onset of muscle weakness due to ALS ≤36 months at the time of the Screening Visit
Slow Vital Capacity (VC) ≥50% of predicted capacity at the time of the Screening Visit
If taking riluzole, must be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole naive participants are allowed.

Key exclusion criteria (7 listed)

Clinically significant, unstable, medical condition (other than ALS)
Clinically significant heart disease, liver disease or kidney disease
Cognitive impairment or dementia
Current uncontrolled hypertension
History of unresolved cancer

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
AL001Administered via intravenous (IV) infusion	Drug	AL001
PlaceboAdministered via intravenous (IV) infusion	Drug	Placebo

Primary outcome

Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events
Time frame: 24 weeks
Count of participants with adverse events during the study treatment period
Immunogenicity of AL001
Time frame: Week 24
Count of participants positive for Anti-drug Antibodies (ADAs) to AL001 at week 24
Pharmacokinetics (PK) of AL001 in Serum
Time frame: Week 24
Concentration of AL001 in Serum at week 24
Pharmacokinetics (PK) of AL001 in CSF
Time frame: Week 24
Concentration of AL001 in Cerebrospinal fluid (CSF) at week 24
Change From Baseline in Plasma Progranulin
Time frame: 24 weeks
Evaluate the change from baseline to week 24 in plasma progranulin levels
Change From Baseline in CSF Progranulin
Time frame: 24 weeks
Evaluate the change from baseline to week 24 in Cerebrospinal fluid (CSF) progranulin levels

2 secondary outcomes

Change From Baseline in Plasma Neurofilament Light Chain
Time frame: 24 weeks
Change From Baseline in CSF Neurofilament Light Chain
Time frame: 24 weeks

Results and safety

Results posted to the registry on 18 Jun 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
AL001	3	2
Placebo	2	2

Primary outcome: Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events

Time frame: 24 weeks. Count of participants. Unit: Participants

Measure	AL001 n = 3	Placebo n = 2
Value	2	2

Population analysed: Safety population

Primary outcome: Immunogenicity of AL001

Time frame: Week 24. Count of participants. Unit: Participants

Measure	AL001 n = 2	Placebo n = 2
Value	1	0

Population analysed: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.

Primary outcome: Pharmacokinetics (PK) of AL001 in Serum

Time frame: Week 24. Mean (Standard Deviation). Unit: ng/mL

Measure	AL001 n = 2	Placebo n = 0
Value	705792.5 (87387.7916)	–

Population analysed: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.

Primary outcome: Pharmacokinetics (PK) of AL001 in CSF

Time frame: Week 24. Mean (Standard Deviation). Unit: ng/mL

Measure	AL001 n = 2	Placebo n = 0
Value	547.5 (395.2727)	–

Population analysed: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: 32 Weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
AL001	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
Placebo	0 of 2 (0%)	2 of 2 (100%)	0 of 2

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Amyotrophic Lateral Sclerosis

RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
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Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
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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.

Sabrina Paganoni, MD principal investigator

Massachusetts General Hospital

3 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AL001 in people with Amyotrophic Lateral Sclerosis, run by Alector Inc. at 3 sites in United States.

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 8 inclusion and 7 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: stopped early, as of the sponsor's last update on 18 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. 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. Some participants in this study may receive a placebo. 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?

Yes. Results are posted in the registry (first posted 18 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events (time frame: 24 weeks). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05053035.

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 18 Jun 2025.