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.
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
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 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.
- 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)
Sponsor and investigators
- Lead sponsor
- Alector Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sabrina Paganoni, MD, Principal investigator, Massachusetts General Hospital
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
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
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.
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
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
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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?
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.