EfgaDem
A double-blinded, randomized, placebo-controlled, multi-center trial of Efgartigimod in autoimmune dementia
Allgemeines
This clinical trial is a multicenter, interventional, randomized, double-blind, placebo-controlled study in parallel design phase IIa. The trial is being led by Prof. Dr. Harald Prüß, DZNE and Charité Berlin, and is being conducted at three trial centers in Germany. The financing is provided by the drug manufacturer argenx BV.
Hintergrund und Ziele
Patients with autoimmune dementia suffer from subacute, progressive cognitive decline that impairs memory, activities of daily living and personal interactions. In contrast to “classic” neurodegenerative dementias (such as Alzheimer's disease without antibody detection), the suspected cause is immune activation in the brain with autoantibodies and immune cells directed against neuronal proteins.
Autoimmune dementia is an increasingly recognized clinical condition that can mimic Alzheimer's disease (AD), frontotemporal dementia (FTD) and other neurodegenerative memory disorders. In “atypical dementia”, the link with autoimmunity appears to be particularly strong. The symptoms lead to massive changes in behavior, memory, orientation and many neuropsychological areas, affecting normal life and personal relationships. In addition, the care of dementia patients is very complex and expensive due to the long course of the disease and the heavy involvement of families and professional caregivers.
Although there are no causal treatment options for Alzheimer's and FTD, patients with autoimmune dementia can benefit from immunotherapy, e.g. by depleting the associated autoantibodies using therapeutic apheresis or immunosuppression. Case reports and small series show that significant clinical improvement is possible in selected patients. Currently, the presence of disease-related autoantibodies is not systematically investigated in routine clinical practice in memory clinics. It is therefore likely that patients with treatable dementia are overlooked and do not receive immunomodulatory treatment that is available and regularly administered in other neurological diseases.
The aim is to investigate whether treatment with the FcRn inhibitor efgartigimod leads to stabilization or improvement or less progression of cognitive impairment compared to placebo by analyzing the change in MoCA from baseline to 52 weeks after the first dose of study drug in the two study arms.
Inclusion criteria:
- Male and female subjects aged ≥ 40 years
- Specialist diagnosis of progressive cognitive impairment or dementia for more than 3 months
- Presence of anti-brain autoantibodies
Übersicht / Ablauf der Studie
The clinical trial will include 66 subjects. Subjects will be randomized in a 2:1 ratio to receive either the active drug (efgartigimod alfa PH20) or placebo.
The study participants will take part in the clinical trial for a total of 57 weeks (13 months). Over a period of 52 weeks, they will receive the investigational drug or the placebo 32 times as subcutaneous injections. In the first 12 weeks, the injections are administered weekly and from week 13 onwards at two-weekly intervals.
The study includes 1 screening visit, 7 treatment visits and 1 end-of-study visit.
Twenty-five additional treatment visits may be conducted either at the study center or at the patient's home.
Study Participation
If you would like to participate in this study, please contact our central study office:
+49 30 450 660 655
studie-neuroimmunologie(at)charite.de
Principal Investigator: Prof. Dr. Harald Prüß
Start of the study: in preparation
Status: in preparation
Projektmanagement:
Klinische Forschungsplattform des DZNE