A double-blind, placebo-controlled study to assess the efficacy and safety of AL001 in frontotemporal dementia
Descrizione riassuntiva dello studio
The study investigates whether the investigational drug AL001, administered as an intravenous infusion, is effective and safe in treating individuals who are carriers of a mutation in the Progranulin gene that causes frontotemporal dementia. These individuals have a mutation in one of the two copies of the Progranulin gene (this is referred to as a heterozygous mutation). Individuals with a heterozygous mutation in the Progranulin gene and symptoms of FTD related to behavior or language may participate in the study, as well as individuals who are carriers of a heterozygous mutation in the Progranulin gene and therefore at risk of developing FTD but who do not yet have symptoms. Approximately 180 individuals at risk of disease or already diagnosed with frontotemporal dementia will participate in the study at about 50 trial centers worldwide. In Switzerland, there is 1 trial center. The investigational drug AL001 is considered experimental and is not approved in Switzerland for the treatment of frontotemporal dementia.
(BASEC)
Intervento studiato
The assignment of study participants to receive AL001 or the placebo (an infusion without active ingredient) is done randomly (like flipping a coin).
This is a double-blind study, meaning that neither the investigator nor the study participant knows which treatment the participant is receiving.
All study participants will receive AL001 or the placebo on the day of treatment initiation and then every 4 weeks until the completion of the study treatment via intravenous administration.
(BASEC)
Malattie studiate
Frontotemporal dementia (FTD) is a neurodegenerative disease characterized by a progressive deterioration of behavior, language, ability to perform activities, and motor functions. Frontotemporal dementia is a rare disease, affecting an estimated 4 to 17 individuals per 100,000 people in the European population. After the onset of symptoms, the disease can progress rapidly in affected individuals.
(BASEC)
1. Study participants with a mutation in the Progranulin gene and a diagnosed FTD OR individuals with a mutation in the Progranulin gene for whom there is a risk of developing FTD symptoms, as demonstrated by a biomarker. Symptomatic individuals must exhibit one or more of the behavioral/cognitive symptoms required for the diagnosis of a possible behavioral variant of FTD (characterized by behavioral change), or a primary progressive aphasia must have been diagnosed (characterized by a gradual impairment of language abilities). 2. The study participant requires a study partner who consents to participate in the study and cares for the study participant daily or visits them daily (for at least 5 hours per week). (BASEC)
Criteri di esclusione
1. Dementia due to another condition other than FTD, such as Alzheimer's, Parkinson's, Huntington's disease, or vascular dementia or another type of dementia. 2. Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins. 3. Currently uncontrolled hypertension (high blood pressure), 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. 4. Women who are pregnant or breastfeeding or who plan to become pregnant during the study period. 5. Any experimental vaccines or gene therapies. 6. History of an unsuccessfully treated cancer. 7. Current use of anticoagulants (e.g., Coumadin, heparinoids, Apixaban). 8. Residence in a professional medical care facility, a rehabilitation home, or a long-term care facility; or need for continuous nursing care. (BASEC)
Luogo dello studio
Basilea
(BASEC)
Sponsor
Alector, Inc. PPD Switzerland GmbH c/o Dufour Treuhand AG
(BASEC)
Contatto per ulteriori informazioni sullo studio
Persona di contatto in Svizzera
PD Dr. Marc Sollberger
+41 61 326 47 73
marc.sollberger@clutterfelixplatter.chUniversitäre Altersmedizin FELIX PLATTER
(BASEC)
Informazioni generali
(ICTRP)
Informazioni scientifiche
(ICTRP)
Nome del comitato etico approvante (per studi multicentrici solo il comitato principale)
Commissione d'etica svizzera nord-ovest/centrale EKNZ
(BASEC)
Data di approvazione del comitato etico
23.10.2020
(BASEC)
ID di studio ICTRP
NCT04374136 (ICTRP)
Titolo ufficiale (approvato dal comitato etico)
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gen (BASEC)
Titolo accademico
A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene (ICTRP)
Titolo pubblico
A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3) (ICTRP)
Malattie studiate
Frontotemporal Dementia (ICTRP)
Intervento studiato
Drug: AL001Drug: PlaceboDrug: Open label - AL001 (ICTRP)
Tipo di studio
Interventional (ICTRP)
Disegno dello studio
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor). (ICTRP)
Criteri di inclusione/esclusione
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. (ICTRP)
non disponibile
Endpoint primari e secondari
Evaluation of efficacy of AL001 as measured by the CDR� plus NACC FTLD-SB (ICTRP)
Change in Clinical Global Impression-Severity (CGI-S) Score;Change in Clinical Global Impression-Improvement (CGI-I) Score;Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Score;Pharmacodynamic Biomarkers;Evaluation of safety and tolerability of AL001: Incidence of adverse events (ICTRP)
Data di registrazione
non disponibile
Inclusione del primo partecipante
non disponibile
Sponsor secondari
GlaxoSmithKline (ICTRP)
Contatti aggiuntivi
TBD TBD (ICTRP)
ID secondari
AL001-3 (ICTRP)
Risultati-Dati individuali dei partecipanti
non disponibile
Ulteriori informazioni sullo studio
https://clinicaltrials.gov/ct2/show/NCT04374136 (ICTRP)
Risultati dello studio
Riepilogo dei risultati
non disponibile
Link ai risultati nel registro primario
non disponibile