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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03636204
Recruitment Status : Completed
First Posted : August 17, 2018
Last Update Posted : July 27, 2020
Sponsor:
Information provided by (Responsible Party):
Alector Inc.

Tracking Information
First Submitted Date  ICMJE July 12, 2018
First Posted Date  ICMJE August 17, 2018
Last Update Posted Date July 27, 2020
Actual Study Start Date  ICMJE September 14, 2018
Actual Primary Completion Date December 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 15, 2018)
Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs) [ Time Frame: 85 days ]
Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 23, 2020)
  • Pharmacokinetics (PK) of AL001 [ Time Frame: 85 days ]
    Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points
  • Maximum plasma concentration (Cmax) for AL001 [ Time Frame: 85 days ]
    Evaluate Cmax for serum and CSF concentration of AL001 at specified time points
  • Area under the curve concentration (AUC) for AL001 [ Time Frame: 85 days ]
    Evaluate AUC for serum and CSF concentration of AL001 at specified time points
Original Secondary Outcome Measures  ICMJE
 (submitted: August 15, 2018)
  • Pharmacokinetics (PK) of AL001 [ Time Frame: 85 days ]
    Serum and CSF concentration of AL001 at specified time points
  • Maximum plasma concentration (Cmax) for AL001 [ Time Frame: 85 days ]
    Evaluate Cmax for serum and CSF concentration of AL001 at specified time points
  • Area under the curve concentration (AUC) for AL001 [ Time Frame: 85 days ]
    Evaluate AUC for serum and CSF concentration of AL001 at specified time points
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation
Official Title  ICMJE A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Healthy
  • Frontotemporal Dementia
Intervention  ICMJE
  • Biological: AL001
    Active dose of AL001
  • Other: Placebo
    Saline solution administered as a single infusion as palcebo.
Study Arms  ICMJE
  • Experimental: AL001
    Up to six single ascending doses of AL001
    Intervention: Biological: AL001
  • Placebo Comparator: Saline Solution
    Saline solution will be administered as a single infusion for each cohort in a ratio of 6 active and 2 placebo subjects
    Intervention: Other: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 23, 2020)
64
Original Estimated Enrollment  ICMJE
 (submitted: August 15, 2018)
60
Actual Study Completion Date  ICMJE December 31, 2019
Actual Primary Completion Date December 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   United Kingdom,   United States
Removed Location Countries Netherlands
 
Administrative Information
NCT Number  ICMJE NCT03636204
Other Study ID Numbers  ICMJE AL001-1
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alector Inc.
Study Sponsor  ICMJE Alector Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: George Stoica Bioclinica Research
PRS Account Alector Inc.
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP