Trial · NCT05053035
A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
NCT05053035, Phase 2, Terminated, Alector Inc.; results first posted 2025-06-18
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS. Trials, record NCT05053035. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05053035
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
- NCT number
- NCT05053035
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 5 participants, actual
- Lead sponsor
- Alector Inc.
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- AL001, Placebo
- First posted
- 2021-09-22
- Last updated
- 2025-06-18
- Results first posted
- 2025-06-18
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events | 24 weeks | Count of participants with adverse events during the study treatment period |
| Primary | Immunogenicity of AL001 | Week 24 | Count of participants positive for Anti-drug Antibodies (ADAs) to AL001 at week 24 |
| Primary | Pharmacokinetics (PK) of AL001 in Serum | Week 24 | Concentration of AL001 in Serum at week 24 |
| Primary | Pharmacokinetics (PK) of AL001 in CSF | Week 24 | Concentration of AL001 in Cerebrospinal fluid (CSF) at week 24 |
| Primary | Change From Baseline in Plasma Progranulin | 24 weeks | Evaluate the change from baseline to week 24 in plasma progranulin levels |
| Primary | Change From Baseline in CSF Progranulin | 24 weeks | Evaluate the change from baseline to week 24 in Cerebrospinal fluid (CSF) progranulin levels |
| Secondary | Change From Baseline in Plasma Neurofilament Light Chain | 24 weeks | Evaluate the change from baseline to week 24 in plasma neurofilament light chain levels |
| Secondary | Change From Baseline in CSF Neurofilament Light Chain | 24 weeks | Evaluate change from baseline to week 24 in Cerebrospinal fluid (CSF) neurofilament light chain levels |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events | 24 weeks | Safety population | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Immunogenicity of AL001 | Week 24 | Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Pharmacokinetics (PK) of AL001 in Serum | Week 24 | Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed. | ng/mL | MEAN |
| PRIMARY | Pharmacokinetics (PK) of AL001 in CSF | Week 24 | Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed. | ng/mL | MEAN |
| PRIMARY | Change From Baseline in Plasma Progranulin | 24 weeks | Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed. | ng/mL | MEAN |
| PRIMARY | Change From Baseline in CSF Progranulin | 24 weeks | Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed. | ng/mL | MEAN |
| SECONDARY | Change From Baseline in Plasma Neurofilament Light Chain | 24 weeks | Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed. | pg/mL | MEAN |
| SECONDARY | Change From Baseline in CSF Neurofilament Light Chain | 24 weeks | Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed. | pg/mL | MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events | AL001 | 2 | Participants | ||
| Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events | Placebo | 2 | Participants | ||
| Immunogenicity of AL001 | AL001 | 1 | Participants | ||
| Immunogenicity of AL001 | Placebo | 0 | Participants | ||
| Pharmacokinetics (PK) of AL001 in Serum | AL001 | 705792.5 | Standard Deviation 87387.7916 | ng/mL | |
| Pharmacokinetics (PK) of AL001 in CSF | AL001 | 547.5 | Standard Deviation 395.2727 | ng/mL | |
| Change From Baseline in Plasma Progranulin | AL001 | 175 | Standard Deviation 2.8284 | ng/mL | |
| Change From Baseline in Plasma Progranulin | Placebo | -24.4 | Standard Deviation 9.3338 | ng/mL | |
| Change From Baseline in CSF Progranulin | AL001 | 2.865 | Standard Deviation 1.0677 | ng/mL | |
| Change From Baseline in CSF Progranulin | Placebo | -0.385 | Standard Deviation 0.3323 | ng/mL |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | AL001 | 3 | |
| Overall Study | STARTED | Placebo | 2 | |
| Overall Study | COMPLETED | AL001 | 2 | |
| Overall Study | COMPLETED | Placebo | 2 | |
| Overall Study | NOT COMPLETED | AL001 | 1 | |
| Overall Study | NOT COMPLETED | Placebo | 0 |
Source ClinicalTrials.gov
Adverse events48
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Constipation | Gastrointestinal disorders | AL001 | 0 | 3 | |
| other | Constipation | Gastrointestinal disorders | Placebo | 1 | 2 | |
| other | Diarrhea | Gastrointestinal disorders | AL001 | 0 | 3 | |
| other | Diarrhea | Gastrointestinal disorders | Placebo | 1 | 2 | |
| other | Dyspepsia | Gastrointestinal disorders | AL001 | 1 | 3 | |
| other | Dyspepsia | Gastrointestinal disorders | Placebo | 0 | 2 | |
| other | Nausea | Gastrointestinal disorders | AL001 | 0 | 3 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 1 | 2 | |
| other | Catheter site irritation | General disorders | AL001 | 0 | 3 | |
| other | Catheter site irritation | General disorders | Placebo | 1 | 2 |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | AL001 | 0 | 3 |
| Total, all-cause mortality | Placebo | 0 | 2 |
| Total, other adverse events | AL001 | 2 | 3 |
| Total, other adverse events | Placebo | 2 | 2 |
| Total, serious adverse events | AL001 | 0 | 3 |
| Total, serious adverse events | Placebo | 0 | 2 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| AL001 vs Placebo: Immunogenicity of AL001 | Trial comparisons |
| AL001 vs Placebo: Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events | Trial comparisons |
| AL001 vs Placebo: deaths from any cause | Trial comparisons |
| AL001 vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05053035
GET https://conductscience.org/api/trials/v1/NCT05053035/events?page=2