- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03636204
A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation
July 23, 2020 updated by: Alector Inc.
A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation
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
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Actual)
64
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Toronto, Canada
- Sunnybrook Health Sciences Centre
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Ontario
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London, Ontario, Canada
- Lawson Health Research Institute, St. Joseph's
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London, United Kingdom, WC1N 3BG
- University College London
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Alabama
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Birmingham, Alabama, United States, 35294
- University of Alabama
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California
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San Francisco, California, United States, 94158
- UCSF
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Florida
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Orlando, Florida, United States, 32806
- Study Site
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Minnesota
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Rochester, Minnesota, United States, 55905
- Mayo Clinic
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- University of Pennsylvania
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 80 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: AL001
Up to six single ascending doses of AL001
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Active dose of AL001
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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
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Saline solution administered as a single infusion as palcebo.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
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Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.
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85 days
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Pharmacokinetics (PK) of AL001
Time Frame: 85 days
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Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points
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85 days
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Maximum plasma concentration (Cmax) for AL001
Time Frame: 85 days
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Evaluate Cmax for serum and CSF concentration of AL001 at specified time points
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85 days
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Area under the curve concentration (AUC) for AL001
Time Frame: 85 days
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Evaluate AUC for serum and CSF concentration of AL001 at specified time points
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85 days
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: George Stoica, Bioclinica Research
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
September 14, 2018
Primary Completion (Actual)
December 31, 2019
Study Completion (Actual)
December 31, 2019
Study Registration Dates
First Submitted
July 12, 2018
First Submitted That Met QC Criteria
August 15, 2018
First Posted (Actual)
August 17, 2018
Study Record Updates
Last Update Posted (Actual)
July 27, 2020
Last Update Submitted That Met QC Criteria
July 23, 2020
Last Verified
July 1, 2020
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Mental Disorders
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Neurocognitive Disorders
- Neurodegenerative Diseases
- TDP-43 Proteinopathies
- Proteostasis Deficiencies
- Language Disorders
- Communication Disorders
- Speech Disorders
- Frontotemporal Lobar Degeneration
- Aphasia
- Dementia
- Frontotemporal Dementia
- Aphasia, Primary Progressive
- Pick Disease of the Brain
Other Study ID Numbers
- AL001-1
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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