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A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation

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: NCT03636204
Recruitment Status : Completed
First Posted : August 17, 2018
Last Update Posted : July 27, 2020
Sponsor:
Information provided by (Responsible Party):
Alector Inc.

Brief Summary:
A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics

Condition or disease Intervention/treatment Phase
Healthy Frontotemporal Dementia Biological: AL001 Other: Placebo Phase 1

Detailed Description:
This is a first-in-human Phase 1 study designed to investigate the safety, tolerability, PK and PD of intravenously administered AL001 in Healthy Volunteers and FTD-GRN mutation carriers.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation
Actual Study Start Date : September 14, 2018
Actual Primary Completion Date : December 31, 2019
Actual Study Completion Date : December 31, 2019


Arm Intervention/treatment
Experimental: AL001
Up to six single ascending doses of AL001
Biological: AL001
Active dose of AL001

Placebo Comparator: Saline Solution
Saline solution will be administered as a single infusion for each cohort in a ratio of 6 active and 2 placebo subjects
Other: Placebo
Saline solution administered as a single infusion as palcebo.




Primary Outcome Measures :
  1. Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs) [ Time Frame: 85 days ]
    Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.


Secondary Outcome Measures :
  1. Pharmacokinetics (PK) of AL001 [ Time Frame: 85 days ]
    Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points

  2. Maximum plasma concentration (Cmax) for AL001 [ Time Frame: 85 days ]
    Evaluate Cmax for serum and CSF concentration of AL001 at specified time points

  3. Area under the curve concentration (AUC) for AL001 [ Time Frame: 85 days ]
    Evaluate AUC for serum and CSF 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 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • BMI 18.0-35.0 kg/m2
  • 45-120 kg, inclusive
  • At screening, females must be non-pregnant and non-lactating, or of nonchildbearing potential (either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1-year postmenopausal (amenorrhoea duration of 12 consecutive months); nonpregnancy will be confirmed for all females by a pregnancy test conducted at screening, (each) admission, and at follow-up.
  • Female participants of child-bearing potential must agree to use adequate contraception from screening until 90 days after the follow-up visit.
  • In good physical health on the basis of no clinically significant findings from medical history, physical examination (PE), laboratory tests, 12 lead ECG, and vital signs, as judged by the Investigator.
  • Willingness and able to comply with the study protocol, in the investigator's judgement.

Exclusion Criteria:

  • Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
  • Positive drug or alcohol at screening and prior to first dose
  • History of alcohol abuse or substance abuse

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): NCT03636204


Locations
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United States, Alabama
University of Alabama
Birmingham, Alabama, United States, 35294
United States, California
UCSF
San Francisco, California, United States, 94158
United States, Florida
Study site
Orlando, Florida, United States, 32806
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Canada, Ontario
Lawson Health Research Institute, St. Joseph's
London, Ontario, Canada
Canada
Sunnybrook Health Sciences Centre
Toronto, Canada
United Kingdom
University College London
London, United Kingdom, WC1N 3BG
Sponsors and Collaborators
Alector Inc.
Investigators
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Principal Investigator: George Stoica Bioclinica Research
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Responsible Party: Alector Inc.
ClinicalTrials.gov Identifier: NCT03636204    
Other Study ID Numbers: AL001-1
First Posted: August 17, 2018    Key Record Dates
Last Update Posted: July 27, 2020
Last Verified: July 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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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
Signs and Symptoms