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Trial record 1 of 9 for:    AL001
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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03987295
Recruitment Status : Active, not recruiting
First Posted : June 17, 2019
Last Update Posted : July 1, 2022
Sponsor:
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: Non-Randomized
Intervention Model: Parallel 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 : January 28, 2026
Estimated Study Completion Date : June 2, 2026


Arm Intervention/treatment
Experimental: Granulin
IV administration of AL001; 60 mg/kg, every 4 weeks [q4w]
Drug: AL001
60 mg/kg of AL001 every 4 weeks

Experimental: C9orf72
IV administration of AL001; 60 mg/kg, every 4 weeks [q4w]
Drug: AL001
60 mg/kg of AL001 every 4 weeks




Primary Outcome Measures :
  1. Part 1: Evaluation of safety and efficacy of AL001 as measured by the CDR® plus NACC FTLD-SB [ Time Frame: 96 weeks ]
    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).


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

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

  3. Pharmacokinetics (PK) of AL001 [ Time Frame: 96 weeks ]
    Concentration of AL001 at specified time points


Other Outcome Measures:
  1. Part 2: Assess the long-term safety and tolerability of IV administration of AL001 as measured by the CDR® plus NACC FTLD-SB [ Time Frame: 96 Weeks ]
    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



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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
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

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 ClinicalTrials.gov identifier (NCT number): NCT03987295


Locations
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United States, California
UCSF
San Francisco, California, United States, 94158
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
The Glenn Biggs Institute for Alzheimer's and Neurodegenerative Diseases UT Health San Antonio
San Antonio, Texas, United States, 78229
Canada, Ontario
Lawson Health Research Institute, St. Joseph's
London, Ontario, Canada, N6A 4V2
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada, M4N 3M5
Germany
Technical University of Munich
Munchen, Germany, 81675
University of Ulm
Ulm, Germany, 89081
Italy
University of Brescia
Brescia, Italy, 25123
Netherlands
Brain Research Center - PPDS
Amsterdam, Netherlands, 1081GN
Erasmus University Medical Center
Rotterdam, Netherlands, 3015 GD
United Kingdom
University College London
London, United Kingdom, WC1N 3BG
Sponsors and Collaborators
Alector Inc.
Investigators
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Principal Investigator: Peter Ljubenkov, MD University of California, San Francisco
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Responsible Party: Alector Inc.
ClinicalTrials.gov Identifier: NCT03987295    
Other Study ID Numbers: AL001-2
First Posted: June 17, 2019    Key Record Dates
Last Update Posted: July 1, 2022
Last Verified: June 2022

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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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Dementia
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
Aphasia
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations