NCT03636204

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

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
64

participants targeted

Target at P75+ for phase_1 healthy

Timeline
Completed

Started Sep 2018

Longer than P75 for phase_1 healthy

Geographic Reach
3 countries

8 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 12, 2018

Completed
1 month until next milestone

First Posted

Study publicly available on registry

August 17, 2018

Completed
28 days until next milestone

Study Start

First participant enrolled

September 14, 2018

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2019

Completed
Last Updated

July 27, 2020

Status Verified

July 1, 2020

Enrollment Period

1.3 years

First QC Date

July 12, 2018

Last Update Submit

July 23, 2020

Conditions

HealthyFrontotemporal Dementia

Outcome Measures

Primary Outcomes (1)

  • Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)

    Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.

    85 days

Secondary Outcomes (3)

  • Pharmacokinetics (PK) of AL001

    85 days

  • Maximum plasma concentration (Cmax) for AL001

    85 days

  • Area under the curve concentration (AUC) for AL001

    85 days

Study Arms (2)

AL001

EXPERIMENTAL

Up to six single ascending doses of AL001

Biological: 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

Other: Placebo

Interventions

AL001BIOLOGICAL

Active dose of AL001

AL001
PlaceboOTHER

Saline solution administered as a single infusion as palcebo.

Saline Solution

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • BMI 18.0-35.0 kg/m2
  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

University of Alabama

Birmingham, Alabama, 35294, United States

Location

UCSF

San Francisco, California, 94158, United States

Location

Study site

Orlando, Florida, 32806, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Lawson Health Research Institute, St. Joseph's

London, Ontario, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Canada

Location

University College London

London, WC1N 3BG, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 37322482DERIVED

MeSH Terms

Conditions

Frontotemporal Dementia

Condition Hierarchy (Ancestors)

Frontotemporal Lobar DegenerationDementiaBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesTDP-43 ProteinopathiesNeurodegenerative DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesNeurocognitive DisordersMental Disorders

Study Officials

  • George Stoica

    Bioclinica Research

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 12, 2018

First Posted

August 17, 2018

Study Start

September 14, 2018

Primary Completion

December 31, 2019

Study Completion

December 31, 2019

Last Updated

July 27, 2020

Record last verified: 2020-07

Locations

US(5)CA(2)GB(1)