Trial | NCT03636204  Report issue

Title

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

  • Phase

    Phase 1

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Intervention/Treatment

    AL001 ...

  • Study Participants

    64
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
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 Started
Sep 14
2018
Primary Completion
Dec 31
2019
Study Completion
Dec 31
2019
Last Update
Jul 27
2020

Biological AL001

Active dose of AL001

Other Placebo

Saline solution administered as a single infusion as palcebo.

AL001 Experimental

Up to six single ascending doses of AL001

Saline Solution Placebo Comparator

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

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.
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
No Results Posted