Frontotemporal DementiaPhase 2NCT03987295
A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
A Phase 2 study testing AL001 in people with Frontotemporal Dementia, run by Alector Inc. at 12 sites in 6 countries.
Trial summary
A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Frontotemporal Dementia
- What is being tested?
- AL001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 12 sitesin 6 countries
- Study dates
- 27 Sep 2019to 5 Jun 2024
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- At screening, female participants must be nonpregnant and nonlactating
- In good physical health on the basis of no clinically significant findings from medical history, physical examinations (PEs), laboratory tests, ECGs, and vital signs.
- Participant is a carrier of a loss of function progranulin gene (GRN) mutation or carrier of a hexanucleotide repeat expansion C9orf72 mutation
Who cannot join
- Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
- History of alcohol abuse or substance abuse
- Participant resides in a skilled nursing facility, convalescent home, or long term care facility
Trial locations and contacts
12 sites in 6 countries.
12 sites in 6 countries
United States4 sites
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
The Glenn Biggs Institute for Alzheimer's and Neurodegenerative Diseases UT Health San Antonio
San Antonio, Texas, 78229
UCSF
San Francisco, California, 94158
Canada2 sites
Lawson Health Research Institute, St. Joseph's
London, Ontario, N6A 4V2
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
Germany2 sites
Technical University of Munich
München, 81675
University of Ulm
Ulm, 89081
Netherlands2 sites
Brain Research Center - PPDS
Amsterdam, 1081GN
Erasmus University Medical Center
Rotterdam, 3015 GD
About this study
Official title: A Phase 2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9orf72 Mutations Causative of Frontotemporal Dementia
A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.
Read the detailed description
This is a Phase 2, multicenter, open label study evaluating the safety, tolerability, PK and PD of AL001 administered intravenously in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.
- Conditions
- Frontotemporal Dementia
- Being tested
- AL001
- Medical subject headings
- Frontotemporal Dementia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AL001Drug
60 mg/kg of AL001 every 4 weeks
| Study group | Role | What they receive |
|---|---|---|
| Granulin and C9orf72 | Experimental | IV administration of AL001; 60 mg/kg, every 4 weeks [q4w] Receives: Drug: AL001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Severity of TEAEs
Number of TEAEs categorized by severity
Measured: 197 weeks
Severity of Treatment-Related TEAEs
Number of treatment-related TEAEs categorized by severity
Measured: 197 weeks
Any TEAE Leading to Study Drug Discontinuation
Number of TEAEs leading to study drug discontinuation
Measured: 197 weeks
Immunogenicity Antidrug Antibodies (ADA) Titer
Titer values of antidrug antibodies (ADAs) in participants receiving latozinemab at week 97/Part 1 end of study
Measured: 97 weeks
Confirmatory Immunogenicity Antidrug Antibodies (ADA) Responses
Presence of confirmatory antidrug antibodies (ADAs) in participants who test positive for ADA at week 97 (Part 1 End of Study).
Measured: 97 weeks
Change From Baseline in Sheehan Suicidality Tracking Scale
The Sheehan Suicidality Tracking Scale (S-STS) is a structured assessment tool used to evaluate the presence, severity, and frequency of suicidal ideation and behavior. It includes items that address passive thoughts of death, active suicidal ideation, intent, planning, suicide attempts, and non-suicidal self-injury. The S-STS total score ranges from 0 to 64, based on 16 items each rated from 0 (not at all) to 4 (extremely), with higher scores indicating greater severity of suicidal ideation, intent, or behavior. The total score provides a quantitative measure of suicidality severity and is sensitive to change over time, making it suitable for clinical monitoring and research use.
Measured: 197 weeks
Secondary outcomes (7)
Longitudinal Percent Change From Baseline of CSF PGRN
The percent change from baseline to specified timepoints of PGRN in CSF. The baseline visit is labeled as week 1 and therefore the 96th week is labeled as week 97.
Measured: 97 weeks
Longitudinal Percent Change From Baseline in Plasma PGRN
The percent change from baseline to specified timepoints for PGRN in plasma. The baseline visit is labeled as week 1 and therefore the 96th week is labeled as week 97.
Measured: 97 weeks
Longitudinal Levels of Sortilin in WBCs
The overall change from baseline in Sortilin in WBCs.
Measured: 97 weeks
Latozinemab Concentration in Serum
Serum concentration of Latozinemab at week 97.
Measured: 97 weeks
Cmax of Latozinemab at Specified Timepoints
Maximum observed concentration of Latozinemab at week 96 of treatment.
Measured: 97 weeks
Ctrough of Latozinemab at Specified Timepoints
Trough concentration of Latozinemab at week 96 of treatment
Measured: 97 weeks
ARCmax of Latozinemab
Ratio of latozinemab Cmax at week 61 to the Cmax at week 1
Measured: 61 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2019
- Study start27 Sep 2019
- Primary completion5 Jun 2024
- Study completion5 Jun 2024
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 Frontotemporal DementiaStudies 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
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alector Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- GlaxoSmithKline
- Responsible party
- Sponsor
- Investigators
- Peter Ljubenkov, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03987295
- Sponsor's protocol ID
- AL001-2
- Status last verified
- Dec 2025
- First submitted
- 14 May 2019
- Last updated
- 5 Dec 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 5 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9orf72 Mutations Causative of Frontotemporal Dementia
- ClinicalTrials.gov ID
- NCT03987295
- Acronym / sponsor ID
- AL001-2
- Conditions
- Frontotemporal Dementia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Alector Inc.Industry (pharma, biotech, devices)
- Collaborators
- GlaxoSmithKline
- Study start
- 27 Sep 2019 (actual)
- Primary completion
- 5 Jun 2024 (actual)
- Study completion
- 5 Jun 2024 (actual)
- First posted
- 17 Jun 2019 (actual)
- Results
- Posted 22 Aug 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 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.
Detailed description
This is a Phase 2, multicenter, open label study evaluating the safety, tolerability, PK and PD of AL001 administered intravenously in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Granulin and C9orf72 | Experimental | Drug: AL001IV administration of AL001; 60 mg/kg, every 4 weeks [q4w] |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- At screening, female participants must be nonpregnant and nonlactating
- In good physical health on the basis of no clinically significant findings from medical history, physical examinations (PEs), laboratory tests, ECGs, and vital signs.
- Participant is a carrier of a loss of function progranulin gene (GRN) mutation or carrier of a hexanucleotide repeat expansion C9orf72 mutation
Key exclusion criteria (3 listed)
- Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
- History of alcohol abuse or substance abuse
- Participant resides in a skilled nursing facility, convalescent home, or long term care facility
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 |
|---|---|---|
| AL00160 mg/kg of AL001 every 4 weeks | Drug | Granulin and C9orf72 |
Primary outcome
Severity of TEAEs
Time frame: 197 weeks
Number of TEAEs categorized by severity
Severity of Treatment-Related TEAEs
Time frame: 197 weeks
Number of treatment-related TEAEs categorized by severity
Any TEAE Leading to Study Drug Discontinuation
Time frame: 197 weeks
Number of TEAEs leading to study drug discontinuation
Immunogenicity Antidrug Antibodies (ADA) Titer
Time frame: 97 weeks
Titer values of antidrug antibodies (ADAs) in participants receiving latozinemab at week 97/Part 1 end of study
Confirmatory Immunogenicity Antidrug Antibodies (ADA) Responses
Time frame: 97 weeks
Presence of confirmatory antidrug antibodies (ADAs) in participants who test positive for ADA at week 97 (Part 1 End of Study).
Change From Baseline in Sheehan Suicidality Tracking Scale
Time frame: 197 weeks
The Sheehan Suicidality Tracking Scale (S-STS) is a structured assessment tool used to evaluate the presence, severity, and frequency of suicidal ideation and behavior. It includes items that address passive thoughts of death, active suicidal ideation, intent, planning, suicide attempts, and non-suicidal self-injury. The S-STS total score ranges from 0 to 64, based on 16 items each rated from 0 (not at all) to 4 (extremely), with higher scores indicating greater severity of suicidal ideation, intent, or behavior. The total score provides a quantitative measure of suicidality severity and is sensitive to change over time, making it suitable for clinical monitoring and research use.
7 secondary outcomes
Longitudinal Percent Change From Baseline of CSF PGRN
Time frame: 97 weeks
Longitudinal Percent Change From Baseline in Plasma PGRN
Time frame: 97 weeks
Longitudinal Levels of Sortilin in WBCs
Time frame: 97 weeks
Latozinemab Concentration in Serum
Time frame: 97 weeks
Cmax of Latozinemab at Specified Timepoints
Time frame: 97 weeks
Ctrough of Latozinemab at Specified Timepoints
Time frame: 97 weeks
ARCmax of Latozinemab
Time frame: 61 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 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.
Peter Ljubenkov, MD principal investigator
University of California, San Francisco
12 sites in 6 countries: 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 Frontotemporal Dementia, run by Alector Inc. at 12 sites in 6 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 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 5 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Canada, Germany, Netherlands, Italy and 1 more country. 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?
Yes. Results are posted in the registry (first posted 22 Aug 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?
Severity of TEAEs (time frame: 197 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 5 Dec 2025.