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

Alector

Status and phase: Completed; Phase 1

Conditions: Healthy; Frontotemporal Dementia

Treatments: Biological: AL001; Other: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03636204

AL001-1

Details and patient eligibility

About

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

Full 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.

Enrollment: 64 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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64 participants in 2 patient groups, including a placebo group

AL001

Experimental group

Description:

Up to six single ascending doses of AL001

Treatment:

Biological: AL001

Saline Solution

Placebo Comparator group

Description:

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

Treatment:

Other: Placebo