A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)

Frontotemporal Dementia Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03987295
• Other study ID #: AL001-2
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: September 27, 2019
• Est. completion date: June 2, 2026

Study information

• Verified date: July 2023
• Source: Alector Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 40
• Est. completion date: June 2, 2026
• Est. primary completion date: January 28, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

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

Related Conditions & MeSH terms

• Aphasia, Primary Progressive
• Dementia
• Frontotemporal Dementia
• Pick Disease of the Brain

Intervention

Drug:
• AL001
60 mg/kg of AL001 every 4 weeks

Locations

Country	Name	City	State
Canada	Lawson Health Research Institute, St. Joseph's	London	Ontario
Canada	Sunnybrook Health Sciences Centre	Toronto	Ontario
Germany	Technical University of Munich	Munchen
Germany	University of Ulm	Ulm
Italy	University of Brescia	Brescia
Netherlands	Brain Research Center - PPDS	Amsterdam
Netherlands	Erasmus University Medical Center	Rotterdam
United Kingdom	University College London	London
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Mayo Clinic	Rochester	Minnesota
United States	The Glenn Biggs Institute for Alzheimer's and Neurodegenerative Diseases UT Health San Antonio	San Antonio	Texas
United States	UCSF	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Alector Inc.

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Italy,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Part 2: Assess the long-term safety and tolerability of IV administration of AL001 as measured by the CDR® plus NACC FTLD-SB	The primary objective of the OLE period of the study is to assess the long term safety and tolerability of AL001 in participants who have completed 96 weeks of treatment on Part 1 of the study	96 Weeks
Primary	Part 1: Evaluation of safety and efficacy of AL001 as measured by the CDR® plus NACC FTLD-SB	The CDR® plus NACC FTLD assessment is the CDR® plus the Frontotemporal Dementia Behavior and Language Domain scores from the NACC FTLD module. Data for this scale will be captured through completion of the standard CDR and a domain-specific container form labeled Frontotemporal Dementia Behavior and Language Domains (FTD-BLD). The necessary information to rate these domains is obtained through a semi-structured interview of the participant and a reliable informant or collateral source (e.g., a caregiver).	96 weeks
Secondary	Maximum plasma concentration (Cmax) for AL001	Evaluate Cmax for concentration of AL001 at specified time points	96 weeks
Secondary	Area under the curve concentration (AUC) for AL001	Evaluate AUC for concentration of AL001 at specified time points	96 weeks
Secondary	Pharmacokinetics (PK) of AL001	Concentration of AL001 at specified time points	96 weeks