Clinical trial NCT03987295: A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Frontotemporal Dementia
Lead sponsor: Alector Inc.
Sponsor class: INDUSTRY
Collaborators: name: GlaxoSmithKline
First posted: 2019-06-17
Enrollment: 33
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

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.

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

Primary outcomes:
measure: Incidence, nature, and severity of AEs and SAEs
timeFrame: 96 weeks
measure: Incidence of ADAs to AL001
timeFrame: 96 weeks
Secondary outcomes:
measure: Maximum serum concentration (Cmax) for AL001
timeFrame: 96 weeks
measure: ARcmax for AL001
timeFrame: 96 weeks
measure: Trough concentration (Ctrough) of AL001
timeFrame: 96 weeks
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: UCSF
state: California
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: San Antonio
country: United States
facility: The Glenn Biggs Institute for Alzheimer's and Neurodegenerative Diseases UT Health San Antonio
state: Texas
city: London
country: Canada
facility: Lawson Health Research Institute, St. Joseph's
state: Ontario
city: Toronto
country: Canada
facility: Sunnybrook Health Sciences Centre
state: Ontario
city: Munchen
country: Germany
facility: Technical University of Munich
city: Ulm
country: Germany
facility: University of Ulm
city: Brescia
country: Italy
facility: University of Brescia
city: Amsterdam
country: Netherlands
facility: Brain Research Center - PPDS
city: Rotterdam
country: Netherlands
facility: Erasmus University Medical Center
city: London
country: United Kingdom
facility: University College London
Study officials:
affiliation: University of California, San Francisco
name: Peter Ljubenkov, MD
role: PRINCIPAL_INVESTIGATOR
References: