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

NCT ID: NCT03636204

Last Updated: 2020-07-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

64 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-09-14

Study Completion Date

2019-12-31

Brief Summary

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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

Detailed Description

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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.

Conditions

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Healthy Frontotemporal Dementia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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AL001

Up to six single ascending doses of AL001

Group Type EXPERIMENTAL

AL001

Intervention Type BIOLOGICAL

Active dose of AL001

Saline Solution

Saline solution will be administered as a single infusion for each cohort in a ratio of 6 active and 2 placebo subjects

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Saline solution administered as a single infusion as palcebo.

Interventions

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AL001

Active dose of AL001

Intervention Type BIOLOGICAL

Placebo

Saline solution administered as a single infusion as palcebo.

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Alector Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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George Stoica

Role: PRINCIPAL_INVESTIGATOR

Bioclinica Research

Locations

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University of Alabama

Birmingham, Alabama, United States

Site Status

UCSF

San Francisco, California, United States

Site Status

Study site

Orlando, Florida, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Lawson Health Research Institute, St. Joseph's

London, Ontario, Canada

Site Status

Sunnybrook Health Sciences Centre

Toronto, , Canada

Site Status

University College London

London, , United Kingdom

Site Status

Countries

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United States Canada United Kingdom

References

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Kurnellas M, Mitra A, Schwabe T, Paul R, Arrant AE, Roberson ED, Ward M, Yeh F, Long H, Rosenthal A. Latozinemab, a novel progranulin-elevating therapy for frontotemporal dementia. J Transl Med. 2023 Jun 15;21(1):387. doi: 10.1186/s12967-023-04251-y.

Reference Type DERIVED
PMID: 37322482 (View on PubMed)

Other Identifiers

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AL001-1

Identifier Type: -

Identifier Source: org_study_id

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