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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03987295
Recruitment Status : Recruiting
First Posted : June 17, 2019
Last Update Posted : December 7, 2020
Information provided by (Responsible Party):
Alector Inc.

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.

Condition or disease Intervention/treatment Phase
Frontotemporal Dementia Drug: AL001 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : September 27, 2019
Estimated Primary Completion Date : March 30, 2023
Estimated Study Completion Date : May 30, 2023

Arm Intervention/treatment
Experimental: AL001
AL001 every 4 weeks for up to 96-weeks
Drug: AL001
administered via intravenous (IV) infusion once a month

Primary Outcome Measures :
  1. Evaluation of safety and tolerability of AL001: Incidence of adverse events [ Time Frame: 96 weeks ]
    Incidence of adverse events

Secondary Outcome Measures :
  1. Pharmacokinetics (PK) of AL001 [ Time Frame: 96 weeks ]
    Concentration of AL001 at specified time points

  2. Maximum plasma concentration (Cmax) for AL001 [ Time Frame: 96 weeks ]
    Evaluate Cmax for concentration of AL001 at specified time points

  3. Area under the curve concentration (AUC) for AL001 [ Time Frame: 96 weeks ]
    Evaluate AUC for concentration of AL001 at specified time points

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03987295

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Contact: Study Lead 650-826-2454

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United States, Alabama
University of Alabama Withdrawn
Birmingham, Alabama, United States, 35294
United States, California
UCSF Recruiting
San Francisco, California, United States, 94158
Contact: Study Coordinator    415-476-0671   
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Study Coordinator    507-293-5551   
Contact: Study Coordinator    507-284-9295   
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Study Coordinator    215-662-3596   
Contact: Study Coordinator    215-662-3596   
Canada, Ontario
Lawson Health Research Institute, St. Joseph's Recruiting
London, Ontario, Canada, N6A 4V2
Contact: Study Coordinator    +1-519-685-4292 ext 45635   
Sunnybrook Health Sciences Centre Recruiting
Toronto, Ontario, Canada, M4N 3M5
Contact: Study Coordinator    416-480-6100 ext 1620   
Technical University of Munich Recruiting
Munchen, Germany, 81675
Contact: Study Coordinator    49 89 4140 4276   
Contact: Study Coordinator    49 89 4140 4276   
University of Ulm Recruiting
Ulm, Germany, 89081
Contact: Study Coordinator    0049 731 500 63099   
Contact: Study Coordinator    0049 731 500 63099   
University of Brescia Recruiting
Brescia, Italy, 25123
Contact: Study Coordinator    39 345 457 4826   
Erasmus University Medical Center Recruiting
Rotterdam, Netherlands, 3015 GD
Contact: Study Coordinator    0031-6-50031924   
Contact: Study Coordinator    0031-6-50008263   
United Kingdom
University College London Recruiting
London, United Kingdom, WC1N 3BG
Contact: Study Coordinator    +44 (0) 20 3448 3105   
Contact: Study Coordinator    44 203 44 84536   
Sponsors and Collaborators
Alector Inc.
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Principal Investigator: Peter Ljubenkov, MD University of California, San Francisco
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Responsible Party: Alector Inc. Identifier: NCT03987295    
Other Study ID Numbers: AL001-2
First Posted: June 17, 2019    Key Record Dates
Last Update Posted: December 7, 2020
Last Verified: December 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Frontotemporal Dementia
Aphasia, Primary Progressive
Pick Disease of the Brain
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Neurodegenerative Diseases
Frontotemporal Lobar Degeneration
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations