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出境医 / 临床实验 / A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)

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

Study Description
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.
Study Design
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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
Arms and Interventions
Arm Intervention/treatment
Experimental: AL001
AL001 every 4 weeks for up to 96-weeks
 Drug: AL001
administered via intravenous (IV) infusion once a month
Outcome Measures
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


Eligibility Criteria
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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
Contacts and Locations

Contacts
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Contact: Study Lead 650-826-2454 clinicaltrials@alector.com

Locations
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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    hannah.wiest@ucsf.edu   
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Study Coordinator    507-293-5551    Forsberg.leah@mayo.edu   
Contact: Study Coordinator    507-284-9295    Nelson.Kevin1@mayo.edu   
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Study Coordinator       Dahlia.Kamel@pennmedicine.upenn.edu   
Contact: Study Coordinator       Vidya.Chenji@Pennmedicine.upenn.edu   
Canada, Ontario
Lawson Health Research Institute, St. Joseph's Recruiting
London, Ontario, Canada, N6A 4V2
Contact: Study Coordinator    +1-519-685-4292 ext 45635    Rokhsana.Mortuza@sjhc.london.on.ca   
Sunnybrook Health Sciences Centre Recruiting
Toronto, Ontario, Canada, M4N 3M5
Contact: Study Coordinator    416-480-6100 ext 1620    Michael.comishen@sunnybrook.ca   
Germany
Technical University of Munich Recruiting
Munchen, Germany, 81675
Contact: Study Coordinator    49 89 4140 4276    Bianca.Bierschneider@mri.tum.de   
Contact: Study Coordinator    49 89 4140 4276    Julia.Graesel@mri.tum.de   
University of Ulm Recruiting
Ulm, Germany, 89081
Contact: Study Coordinator    0049 731 500 63099    karin.graf@uni-ulm.de   
Contact: Study Coordinator    0049 731 500 63099    sarah.straub@uni-ulm.de   
Italy
University of Brescia Recruiting
Brescia, Italy, 25123
Contact: Study Coordinator    39 345 457 4826    benussialberto@gmail.com   
Netherlands
Brain Research Center - PPDS Recruiting
Amsterdam, Netherlands, 1081GN
Contact: Study Coordinator       m.vandermaas@brainresearchcenter.nl   
Erasmus University Medical Center Recruiting
Rotterdam, Netherlands, 3015 GD
Contact: Study Coordinator    0031-6-50031924    a.eladdouti-elbelhajji@erasmusmc.nl   
Contact: Study Coordinator    0031-6-50008263    w.degroot.1@erasmusmc.nl   
United Kingdom
University College London Recruiting
London, United Kingdom, WC1N 3BG
Contact: Study Coordinator    +44 (0) 20 3448 3105    drctrialenquiries@ucl.ac.uk   
Contact: Study Coordinator    44 203 44 84536    joanna.irish@nhs.net   
Sponsors and Collaborators
Alector Inc.
Investigators
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Principal Investigator: Peter Ljubenkov, MD University of California, San Francisco
Tracking Information
First Submitted Date  ICMJE May 14, 2019
First Posted Date  ICMJE June 17, 2019
Last Update Posted Date April 6, 2021
Actual Study Start Date  ICMJE September 27, 2019
Estimated Primary Completion Date March 30, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 23, 2020) 		Evaluation of safety and tolerability of AL001: Incidence of adverse events [ Time Frame: 96 weeks ]
Incidence of adverse events
Original Primary Outcome Measures  ICMJE
 (submitted: June 13, 2019) 		Evaluation of safety and tolerability of AL001: Incidence of adverse events [ Time Frame: 48 weeks ]
Incidence of adverse events
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 23, 2020)
  • Pharmacokinetics (PK) of AL001 [ Time Frame: 96 weeks ]
    Concentration of AL001 at specified time points
  • Maximum plasma concentration (Cmax) for AL001 [ Time Frame: 96 weeks ]
    Evaluate Cmax for concentration of AL001 at specified time points
  • Area under the curve concentration (AUC) for AL001 [ Time Frame: 96 weeks ]
    Evaluate AUC for concentration of AL001 at specified time points
Original Secondary Outcome Measures  ICMJE
 (submitted: June 13, 2019)
  • Pharmacokinetics (PK) of AL001 [ Time Frame: 48 weeks ]
    Concentration of AL001 at specified time points
  • Maximum plasma concentration (Cmax) for AL001 [ Time Frame: 48 weeks ]
    Evaluate Cmax for concentration of AL001 at specified time points
  • Area under the curve concentration (AUC) for AL001 [ Time Frame: 48 weeks ]
    Evaluate AUC for concentration of AL001 at specified time points
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Frontotemporal Dementia
Intervention  ICMJE Drug: AL001
administered via intravenous (IV) infusion once a month
Study Arms  ICMJE Experimental: AL001
AL001 every 4 weeks for up to 96-weeks
Intervention: Drug: AL001
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 23, 2020) 		40
Original Estimated Enrollment  ICMJE
 (submitted: June 13, 2019) 		32
Estimated Study Completion Date  ICMJE May 30, 2023
Estimated Primary Completion Date March 30, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 85 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Study Lead 650-826-2454 clinicaltrials@alector.com
Listed Location Countries  ICMJE Canada,   Germany,   Italy,   Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03987295
Other Study ID Numbers  ICMJE AL001-2
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alector Inc.
Study Sponsor  ICMJE Alector Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Peter Ljubenkov, MD University of California, San Francisco
PRS Account Alector Inc.
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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