EFGARTIGIMOD ALFA for Autoimmune dementia

Stratification factors

• center

Inclusion criteria

• {"criterion_text":"- Male and female subjects ≥ 40 years of age\n- Expert diagnosis of progressive cognitive impairment or dementia for more than 3 months\n- 10 < MoCA ≤ 25\n- Presence of anti-brain autoantibodies in serum and/or CSF\n- Age ≥ 40 years\n- Speaks German at a level that, as determined by the investigator, allows to understand the patient information and to reliably perform the neuropsychological assessments\n- Written consent to participate in the study\n- The patient is capable to attend study visits\n- Vaccination according to STIKO recommendations (incl. COVID-19 vaccination)"}

Exclusion criteria

• {"criterion_text":"- Dysfunction (other than neurodegenerative or immunological) that could distort or obscure the diagnosis of dementia such as vitamin deficiency or hypothyroidism\n- HIV infection\n- Active, severe infections (e.g. sepsis and opportunistic infections)\n- History of chronically active hepatitis including active or chronic hepatitis B, acute or chronic hepatitis C\n- Clinically significant infection involving intravenous administration of antibiotics and hospitalization in the 4 weeks prior to the screening visit\n- Serious impairment of the immune system\n- Severe disease in the renal, cardiovascular or hematological system\n- Any medical condition that, in the opinion of the Investigator, might interfere with the patient’s participation in the trial or poses any added risk for the patient\n- Severe active psychiatric illness\n- Contraindications against MRI examination (e.g. wearers of pacemakers, deep brain stimulators, cerebrospinal fluid shunt valves, very large tattoos, metal splinter injuries and implants containing metal in the body)\n- Participation in any other investigational drug study or exposure to an investigational drug within 5 half-lives of the study drug at baseline\n- MRI findings (macro bleeding, malignant tumors)\n- Treatment with B-cell depleting therapy in the last 6 months\n- History of recurrent or chronic infections or with underlying diseases which may further predispose patients to serious infection\n- Sexually active male and female patients of reproductive potential (female patients/female partners of patients less than 12 months postmenopausal) who do not use highly effective contraception methods (pearl index <1) during treatment\n- Hypersensitivity to the active substance and/or pharmaceutical excipients\n- Known cytokine release syndrome after infusions\n- Pregnancy or lactation"}

Primary endpoints

• {"endpoint_text":"- Cognitive impairment: MoCA: change from baseline to 53 weeks. (dichotomized version of the change of the score ≥0 or <0).","definition_or_measurement_approach":"Change in MoCA score from baseline to 53 weeks; dichotomized as change ≥0 versus <0."}

Secondary endpoints

• {"endpoint_text":"- MoCA: Evaluation of change in continuous score from baseline to week 13, 27 and 53","definition_or_measurement_approach":"Continuous change in MoCA score from baseline to weeks 13, 27 and 53."}
• {"endpoint_text":"- Neuropsychological tests: change from baseline to week 13 and 53","definition_or_measurement_approach":"Change from baseline in neuropsychological test battery at weeks 13 and 53."}
• {"endpoint_text":"- Quality of Life: change from baseline to week 13 and 53","definition_or_measurement_approach":"Change from baseline in quality of life questionnaire at weeks 13 and 53."}
• {"endpoint_text":"- Activities of Daily Living: change from baseline to week 13 and 53","definition_or_measurement_approach":"Change from baseline in activities of daily living assessments at weeks 13 and 53."}
• {"endpoint_text":"- Reduction of autoantibodies from baseline to week 13 and 53","definition_or_measurement_approach":"Measurement of autoantibody levels at baseline, week 13 and week 53; change from baseline evaluated."}
• {"endpoint_text":"- Partial normalization of further laboratory parameters from baseline to week 53","definition_or_measurement_approach":"Laboratory parameter measurements at baseline and week 53 assessing partial normalization."}
• {"endpoint_text":"- Serum and cerebrospinal fluid analyses with cell count, cytology, protein, glucose, lactate and infection markers","definition_or_measurement_approach":"Analyses of serum and CSF including cell count, cytology, protein, glucose, lactate and infection markers at specified timepoints."}

Germany

Earliest CTIS Part Ii Submission Date

02-06-2025

Latest Decision Or Authorization Date

14-04-2026

Processing Time Days

316

Number Of Sites

3

Number Of Participants

69

Primary sponsor

Full Name

Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.

Organisation Type

Laboratory/Research/Testing facility

Country Of Registered Address

Germany

Third parties

• {"country":"Germany","full_name":"Universitaetsklinikum Bonn AöR","duties_or_roles":"10","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Germany","full_name":"Medical Center - University Of Freiburg","duties_or_roles":"1,8","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Germany","full_name":"Charite Universitaetsmedizin Berlin KöR","duties_or_roles":"4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Germany","full_name":"Medios Pharma GmbH","duties_or_roles":"14","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Universitaetsklinikum Bonn AöR","duties_or_roles":"4","organisation_type":"Hospital/Clinic/Other health care facility"}