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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04374136
Other study ID # AL001-3
Secondary ID
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date July 23, 2020
Est. completion date October 11, 2027

Study information

Verified date May 2024
Source Alector Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

A phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene.


Description:

This is a phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 administered intravenously in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene. Study completion marks the end of the open label extension period following the 96-week blinded portion of the study.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 110
Est. completion date October 11, 2027
Est. primary completion date September 1, 2025
Accepts healthy volunteers No
Gender All
Age group 25 Years to 85 Years
Eligibility Inclusion Criteria: - Persons with a progranulin gene mutation and at risk of developing FTD symptoms as evidenced by a biomarker, or persons with a progranulin gene mutation and diagnosed with FTD. - If symptomatic, one or more of the criteria for the diagnosis of possible behavioral variant FTD, or a diagnosis of Primary Progressive Aphasia. - Study partner who consents to study participation and who cares for/visits the participant daily for at least 5 hours per week. - Written informed consent must be obtained and documented (from the participant or, where jurisdictions allow it, from their legal decision maker). Exclusion Criteria: - Dementia due to a condition other than FTD including, but not limited to, Alzheimer's disease, Parkinson's disease, dementia with Lewy bodies, Huntington disease, or vascular dementia. - Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins. - Current uncontrolled hypertension, diabetes mellitus or thyroid disease. Clinically significant heart disease, liver disease or kidney disease. History or evidence of clinically significant brain disease other than FTD. - Females who are pregnant or breastfeeding, or planning to conceive within the study period. - Any experimental vaccine or gene therapy. - History of cancer within the last 5 years. - Current use of anticoagulant medications (e.g., coumadin, heparinoids, apixaban). - Residence in a skilled nursing facility, convalescent home, or long term care facility at screening; or requires continuous nursing care.

Study Design


Intervention

Drug:
AL001
Administered via intravenous (IV) infusion
Placebo
Administered via intravenous (IV) infusion
Open label - AL001
Administered via intravenous (IV) infusion

Locations

Country Name City State
Argentina Fundación Para La Lucha Contra Las Enfermedades Neurológicas de La Infancia Buenos Aires
Argentina CENydET S.R.L. Retiro Ciudad Autónoma De BuenosAires
Argentina INECO Castaño San Juan
Australia Box Hill Hospital Box Hill
Australia Royal Prince Alfred Hospital Camperdown
Australia The Queen Elizabeth Hospital Woodville
Belgium UZ Leuven Leuven Vlaams Brabant
Canada The University of Western Ontario London
Canada Sunnybrook Research Institute - University of Toronto Toronto
France CHU de Bordeaux Bordeaux
France CHRU Lille Lille
France Groupe Hospitalier Pitié Salpétrière Paris
France Hopital Charles Nicolle - Hospital Rouen Seinne-Maritime
France CHU de Toulouse Hopital PURPAN Toulouse Haute-Garonne
Germany Uniklinik Köln Köln
Germany Universitätsklinikum Ulm Ulm
Greece University General Hospital of Alexandroupolis - Department of Neurology Alexandroupoli Evros
Greece Eginitio University General Hospital of Athens - 1st University Neurology Clinic Athens Attica
Italy Nuovo Ospedale Civile S. Agostino-Estense di Baggiovara Baggiovara
Italy IRCCS Istituto delle Scienze Neurologiche di Bologna Bologna
Italy ASST degli Spedali Civili di Brescia - Spedali Civili di Brescia Brescia
Italy IRCCS - Centro S. Giovanni di Dio Fatebenefratelli Brescia
Italy Fondazione Istituto G.Giglio Cefalù Palermo
Italy Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico Milano
Italy Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta Milano
Italy Azienda Unita Sanita Locale (ASL) di Reggio Emilia - IRCCS Reggio Emilia Emilia Romagna
Italy PIA Fondazione Panico Tricase
Netherlands Erasmus MC Rotterdam
Portugal Centro Hospitalar E Universitário de Coimbra EPE Coimbra
Portugal Centro Hospitalar de Lisboa Norte, EPE - Hospital de Santa Maria Lisboa
Portugal Hospital CUF Descobertas Lisboa
Portugal Centro Hospitalar do Porto - Hospital de Santo António Porto
Spain Hospital Clinic de Barcelona Barcelona
Spain Hospital Universitario de Donostia Donostia-san Sebastián
Spain Hospital Clinico San Carlos Madrid
Spain Hospital Universitario de La Princesa Madrid
Spain Hospital Universitario Marques de Valdecilla Santander Cantabria
Sweden Karolinska Universitetssjukhuset Huddinge - PPDS Huddinge
Switzerland Felix Platter Spital Basel
Turkey Istanbul University Medical Faculty Istanbul Fatih
Turkey Dokuz Eylul University Izmir Balcova
United Kingdom University College London London
United States Emory University Atlanta Georgia
United States University of Colorado Aurora Colorado
United States Johns Hopkins University School of Medicine Baltimore Maryland
United States University of North Carolina at Chapel Hill Chapel Hill North Carolina
United States University Of Cincinnati Gardner Neuroscience institute Cincinnati Ohio
United States Case Western Reserve University Cleveland Ohio
United States University of Kansas Alzheimer's Disease Center Fairway Kansas
United States Houston Methodist Institute for Academic Medicine Houston Texas
United States Indiana University Health Neuroscience Center Indianapolis Indiana
United States University of California San Diego La Jolla California
United States University of Miami Medical Center Miami Florida
United States Froedtert and The Medical College of Wisconsin Milwaukee Wisconsin
United States Vanderbilt University Medical Center Nashville Tennessee
United States Irving Institute for Clinical and Translational Research New York New York
United States University of Pennsylvania Philadelphia Pennsylvania
United States Dignity Health Phoenix Arizona
United States Oregon Health and Science University Portland Oregon
United States Mayo Comprehensive Cancer Center - PPDS Rochester Minnesota
United States University of Texas Health Science Center at San Antonio San Antonio Texas

Sponsors (1)

Lead Sponsor Collaborator
Alector Inc.

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Belgium,  Canada,  France,  Germany,  Greece,  Italy,  Netherlands,  Portugal,  Spain,  Sweden,  Switzerland,  Turkey,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Other Optional Open-Label Extension Assess the long-term safety and tolerability of AL001 in participants who have completed 96 week of treatment 96 weeks
Primary Evaluation of efficacy of AL001 as measured by the CDR® plus NACC FTLD-SB The Clinical Dementia Rating Dementia Staging Instrument PLUS National Alzheimer's Disease Coordinating Center frontotemporal lobar degeneration Behavior & Language Domains Sum of Boxes (CDR® plus NACC FTLD-SB) is administered by a healthcare professional and based on individual ratings of the eight domains: memory, orientation, judgment and problem solving, community affairs, home and hobbies, personal care, language and behavior. Impairment is scored on a scale in which none = 0, questionable = 0.5, mild = 1, moderate = 2 and severe = 3. The 8 individual domain ratings, or "box scores", were added together to give the CDR® plus NACC FTLD-SB which ranges from 0-24. Higher score indicates severe impairment. Through study completion, on average up to 96 weeks
Secondary Change in Clinical Global Impression-Severity (CGI-S) Score The CGI-S is used by a clinician to rate the severity of a participant's disease relative to the clinician's past experience with patients who have the same disease using an ordinal scale ranging from 1=normal, not at all ill; 2=borderline ill; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6=severely ill; and 7=among the most extremely ill patients. Higher scores indicate worsening. Baseline to 96 weeks
Secondary Change in Clinical Global Impression-Improvement (CGI-I) Score The CGI-I is used by a clinician to rate how much a participant's disease has improved or worsened relative to baseline using an ordinal scale ranging from 1=very much improved; 2=much improved; 3=minimally improved; 4=no change from baseline; 5=minimally worse; 6= much worse; and 7=very much worse. Higher scores indicate worsening. Baseline to 96 weeks
Secondary Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Score RBANS is 20 to 25 minute battery developed for cognitive assessment, detection, and characterization of dementia. RBANS includes 12 subtests that measure following 5 indices: (1)Attention Index, composed of Digit Span and Coding; (2)Language Index, consisting of Picture Naming and Semantic Fluency subtests; (3)Visuospatial/Construction Index, made up of Figure Copy and Line Orientation subtests; (4)Immediate Memory Index, composed of List Learning and Story Memory subtests, and (5)Delayed Memory Index, consisting of List Recall, List Recognition, Story Recall, and Figure Recall subtests. Completion of RBANS yields 5 index scores based on participant performance on various subtests, as well as a composite Total Index score for battery. Total index scores range from 40 to 160, and are normalized to a mean of 100 and standard deviation (SD) of 15. Higher scores indicate less impairment. Baseline to 96 weeks
Secondary Pharmacodynamic Biomarkers Change in magnetic resonance imaging and blood-based biomarkers and optional CSF biomarkers (neurofilament light chain and progranulin) Baseline to 96 weeks
Secondary Evaluation of safety and tolerability of AL001: Incidence of adverse events Incidence of adverse events Baseline to 96 weeks
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