Gene Therapy for APOE4 Homozygote of Alzheimer's Disease (NCT03634007) | Clinical Trial Compass
CompletedPhase 1/2
Gene Therapy for APOE4 Homozygote of Alzheimer's Disease
United States15 participantsStarted 2019-11-06
Plain-language summary
This clinical trial is an open label, dose-ranging study designed to evaluate gene therapy to treat patients who are APOE4 homozygotes with clinical diagnosis varying from mild cognitive impairment due to Alzheimer's, mild dementia due to Alzheimer's disease, and moderate dementia due to Alzheimer's disease.
Who can participate
Age range50 Years
SexALL
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Inclusion Criteria:
* APOE4 homozygotes
* Willing and able to provide informed consent (or consent provided by a legally authorized representative)
* Clinical diagnosis of mild cognitive impairment due to Alzheimer's disease or mild to moderate dementia due to Alzheimer's disease
* Evidence of CSF biomarkers consistent with Alzheimer's disease
* Serum neutralizing anti-AAVrh10 titer \<1:100
* No evidence of active infection of any type, including hepatitis virus (A, B, or C) or human immunodeficiency virus (HIV-1 and HIV-2)
* Fertile or infertile individuals; it will be recommended that fertile individuals utilize barrier birth control measures to prevent pregnancy for the duration of the study
* Individuals not receiving experimental medications or participating in another experimental protocol for at least 4 weeks prior to entry into the study
* Participants who agree not to post their personal data related to the study on social media.
Exclusion Criteria:
* Individuals receiving systemic immunosuppressant or corticosteroid therapy other than protocol-specified, are receiving a monoclonal anti-amyloid therapy (example, Aduhelmâ„¢ (aducanumab), Leqembiâ„¢ (lecanemab-irmb) or unable to wash out from anti-coagulant medications.
* Individuals who do not fit the American Journal of Neuroradiology recommendations for image-guided spinal procedures
* Presence of other significant medical, psychiatric, or neurological conditions may disqualify the participant from participation in t…
What they're measuring
1
Proportion of participants with treatment-emergent adverse events and serious adverse events
Timeframe: 1 year
2
The proportion of participants with treatment-emergent adverse events and serious adverse events at each dosage