Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03636204
Recruitment Status : Recruiting
First Posted : August 17, 2018
Last Update Posted : April 19, 2019
Sponsor:
Information provided by (Responsible Party):
Alector Inc.

Brief Summary:
A first in human phase 1 study in healthy volunteers and participants with Granulin 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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 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
Estimated Primary Completion Date : December 31, 2019
Estimated 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 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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. A
  • 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


Contacts
Layout table for location contacts
Contact: Study Lead 4152315660 ext 328 info@alector.com

Locations
Layout table for location information
United States, Alabama
University of Alabama Not yet recruiting
Birmingham, Alabama, United States, 35294
Contact: Study Coordinator         
United States, California
UCSF Not yet recruiting
San Francisco, California, United States, 94158
Contact: Study Coordinator    415-514-5745    lauren.fisher@ucsf.edu   
Contact: Study Coordinator    415-476-0661    mary.koestler@ucsf.edu   
United States, Florida
Study site Active, not recruiting
Orlando, Florida, United States, 32806
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Study Coordinator    507-293-5551    Forsberg.leah@mayo.edu   
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Study Coordinator    215-349-5863    cigne@pennmedicine.upenn.edu   
Canada, Ontario
Lawson Health Research Institute, St. Joseph's Recruiting
London, Ontario, Canada
Contact: Study Coordinator    +1-519-685-4292    cognitiveneurology@sjhc.london.on.ca   
Canada
Sunnybrook Health Sciences Centre Recruiting
Toronto, Canada
Contact: Study Coordinator    416-480-6100 ext 1620      
United Kingdom
University College London Recruiting
London, United Kingdom, WC1N 3BG
Contact: Study Coordinator    +44 (0) 20 3448 3105    drctrialenquiries@ucl.ac.uk   
Sponsors and Collaborators
Alector Inc.
Investigators
Layout table for investigator information
Principal Investigator: George Stoica Bioclinica Research

Layout table for additonal information
Responsible Party: Alector Inc.
ClinicalTrials.gov Identifier: NCT03636204     History of Changes
Other Study ID Numbers: AL001-1
First Posted: August 17, 2018    Key Record Dates
Last Update Posted: April 19, 2019
Last Verified: September 2018

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
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