Phase 2
N=33
A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
Frontotemporal Dementia
Bottom Line
View on ClinicalTrials.gov: NCT03987295 ↗Enrolled (actual)
33
Serious AEs
18.2%
Results posted
Aug 2025
Primary outcome: Primary: Severity of TEAEs — 43; 58; 84; 4 TEAEs (all)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AL001 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alector Inc.
- Primary completion
- Jun 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Severity of TEAEs |
43; 58; 84; 4; 9; 24 | — |
| PRIMARY Severity of Treatment-Related TEAEs |
5; 1; 12; 1; 1; 5 | — |
| PRIMARY Any TEAE Leading to Study Drug Discontinuation |
0; 1; 1 | — |
| PRIMARY Immunogenicity Antidrug Antibodies (ADA) Titer |
320.0; 20.0; 40.0; 160.0; 160.0; 120.0 | — |
| PRIMARY Confirmatory Immunogenicity Antidrug Antibodies (ADA) Responses |
1; 0; 2; 4; 5; 7 | — |
| PRIMARY Change From Baseline in Sheehan Suicidality Tracking Scale |
0; 0; 0 | — |
| SECONDARY Longitudinal Percent Change From Baseline of CSF PGRN |
198.16; 104.05; 101.61; 189.46; 59.73; 144.73 | — |
| SECONDARY Longitudinal Percent Change From Baseline in Plasma PGRN |
177.39; 151.32; 178.82; 185.37; 188.12; 157.93 | — |
| SECONDARY Longitudinal Levels of Sortilin in WBCs |
— | — |
| SECONDARY Latozinemab Concentration in Serum |
236000; 250000; 265000 | — |
| SECONDARY Cmax of Latozinemab at Specified Timepoints |
1810; 1320; 1510 | — |
| SECONDARY Ctrough of Latozinemab at Specified Timepoints |
236; 250; 252 | — |
| SECONDARY ARCmax of Latozinemab |
1.22; 1.23; 1.39 | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT03987295). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.