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

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

      • Toronto, Canada
        • Sunnybrook Health Sciences Centre
    • Ontario
      • London, Ontario, Canada
        • Lawson Health Research Institute, St. Joseph's
      • London, United Kingdom, WC1N 3BG
        • University College London
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama
    • California
      • San Francisco, California, United States, 94158
        • UCSF
    • Florida
      • Orlando, Florida, United States, 32806
        • Study Site
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania

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
Experimental: AL001
Up to six single ascending doses of 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
Saline solution administered as a single infusion as palcebo.

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
Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.
85 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacokinetics (PK) of AL001
Time Frame: 85 days
Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points
85 days
Maximum plasma concentration (Cmax) for AL001
Time Frame: 85 days
Evaluate Cmax for serum and CSF concentration of AL001 at specified time points
85 days
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
85 days

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

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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