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

Healthy Clinical Trial

Official title:

A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03636204
• Other study ID #: AL001-1
• Status: Completed
• Phase: Phase 1
• Start date: September 14, 2018
• Est. completion date: December 31, 2019

Study information

• Verified date: July 2020
• Source: Alector Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 64
• Est. completion date: December 31, 2019
• Est. primary completion date: December 31, 2019
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Aphasia, Primary Progressive
• Dementia
• Frontotemporal Dementia
• Healthy
• Pick Disease of the Brain

Intervention

Biological:
• AL001
Active dose of AL001

Other:
• Placebo
Saline solution administered as a single infusion as palcebo.

Locations

Country	Name	City	State
Canada	Lawson Health Research Institute, St. Joseph's	London	Ontario
Canada	Sunnybrook Health Sciences Centre	Toronto
United Kingdom	University College London	London
United States	University of Alabama	Birmingham	Alabama
United States	Study site	Orlando	Florida
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Mayo Clinic	Rochester	Minnesota
United States	UCSF	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Alector Inc.

Countries where clinical trial is conducted

United States,  Canada,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)	Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.	85 days
Secondary	Pharmacokinetics (PK) of AL001	Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points	85 days
Secondary	Maximum plasma concentration (Cmax) for AL001	Evaluate Cmax for serum and CSF concentration of AL001 at specified time points	85 days
Secondary	Area under the curve concentration (AUC) for AL001	Evaluate AUC for serum and CSF concentration of AL001 at specified time points	85 days