Clinical trial NCT03636204: A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation
Official title: A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy
Frontotemporal Dementia
Lead sponsor: Alector Inc.
Sponsor class: INDUSTRY
First posted: 2018-08-17
Enrollment: 64
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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

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.

Eligibility 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

Primary outcomes:
measure: Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)
timeFrame: 85 days
Secondary outcomes:
measure: Pharmacokinetics (PK) of AL001
timeFrame: 85 days
measure: Maximum plasma concentration (Cmax) for AL001
timeFrame: 85 days
measure: Area under the curve concentration (AUC) for AL001
timeFrame: 85 days
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: San Francisco
country: United States
facility: UCSF
state: California
city: Orlando
country: United States
facility: Study site
state: Florida
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: London
country: Canada
facility: Lawson Health Research Institute, St. Joseph's
state: Ontario
city: Toronto
country: Canada
facility: Sunnybrook Health Sciences Centre
city: London
country: United Kingdom
facility: University College London
Study officials:
affiliation: Bioclinica Research
name: George Stoica
role: PRINCIPAL_INVESTIGATOR
References: