Table 1.
IDs, intervention, main features, and outcomes of selected trial protocols
| Identifier | Intervention | Duration | Placebo | Estimated enrollment | Age | Diagnosis | MMSE at baseline | Primary outcome | Secondary outcome | Status |
|---|---|---|---|---|---|---|---|---|---|---|
| Phase I | ||||||||||
| NCT03056495 | Vorinostat | 4 weeks | N | 44 | ≥ 55 to ≤ 90 | Mild AD | ≥ 22 to ≤ 27 | Maximum-tolerated dose |
Incidence of treatment emergent AEs Pharmacokinetics Pharmacodynamics |
Recruiting |
| NCT02921477 | Bosutinib | 1 year | N | 64 | ≥ 45 to ≤ 89 | MCI to moderate dementia | – | Safety, tolerability | – | Enrolling by invitation |
| NCT02061878 | Bexarotene | 5 days | Y | 12 | ≥ 21 to ≤ 50 | Healthy volunteers with the ApoE ε3/ε3 genotype | – | CSF levels of ApoE and Aβ clearance | Fractional clearance rate of beta-amyloid peptide in CNS | Completed |
| Phase I–II | ||||||||||
| NCT04063124 | Dasatinib (+quercetin) | 12 weeks | N | 5 | ≥ 65 | Clinical diagnosis of AD | – | Brain penetrance after 12 weeks |
CSF-tau, CSF-amyloid beta, CSF-IL-6, CSF-P16, MoCA Electronic gait mapping under single- and dual-task conditions |
Recruiting |
| Phase II | ||||||||||
| NCT04070378 | Daratumumab | 16 weeks | N | 15 | ≥ 55 to ≤ 85 | Mild to moderate AD | ≥ 15 to ≤ 26 | ADAS-Cog/11 |
ADAS-Cog/12, MMSE, ADAS-ADL CDR-SOB, ADCOMS |
Recruiting |
| NCT02947893 | Nilotinib | 1 year | Y | 42 | ≥ 50 | Mild to moderate AD | ≥ 17 to ≤ 24 |
Safety, tolerability Pharmacokinetics |
Abl inhibition to demonstrate CNS target engagement | Active, not recruiting |
| NCT04032626 | Lenalidomide | 12 months of treatment followed by 6 months of washout. The trial will last 20 months in duration. | Y | 30 | ≥ 50 to ≤ 89 | MCI | ≥ 22 to ≤ 28 |
ADAS-Cog ADCS-ADL CDR-SOB MMSE |
AEs and SAEs Change in brain amyloid loads Change in blood inflammatory markers Change in neurodegeneration |
Recruiting |
| NCT01120002 | Tamibarotene | – | Y | 50 | ≥ 55 to ≤ 80 | Mild to moderate AD | ≥ 10 to ≤ 26 | Changes in ADAS-JCog (Japanese version) |
MMSE, ADCS-ADL CIBIC-Plus |
Unknown |
| NCT01782742 | Bexarotene | 4 weeks | Y | 20 | ≥ 50 to ≤ 90 | Probable AD | ≥ 10 to ≤ 20 | Change in the composite amyloid burden of the brain according to ApoE genotype |
MMSE, ADAS-Cog NPI, CDR ADCS-ADL Serum level change of Aβ42 and Aβ40 (all subjects and ApoE-ε4 noncarriers) Change in Aβ42/Aβ40 ratio (all subjects and ApoE-ε4 noncarriers) |
Completed |
| 2016-000429-38 | Pexidartinib | 16 weeks | Y | – | ≥ 55 to ≤ 85 | Mild to moderate AD | ≥ 16 to ≤ 26 | Safety and tolerability |
Effect on microglia inflammation Cognitive and functional measures (tests not reported) Pharmacokinetics (CSF to plasma ratio) CSFR-1 biomarkers in blood |
Prematurely ended |
| NCT00976118 | Masitinib | 24 weeks | Y | 34 | ≥ 50 | Mild to moderate AD | ≥ 12 to ≤ 26 | ADAS-Cog |
CIBIC-Plus CDR MMSE |
Completed |
| NCT01094340 | Thalidomide | 24 weeks | Y | 20 | ≥ 50 to ≤ 90 | Probable AD | ≥ 12 to ≤ 26 | Improve cognition | Improve cognition | Unknown |
| Phase II/III | ||||||||||
| NCT01872598 (2010-021218-50) | Masitinib | Minimum of 6 months | Y | 721 | ≥ 50 | Diagnosis of AD | ≥ 12 to ≤ 25 |
ADAS-Cog ADCS-ADL |
MMSE, CIBIC-Plus | Completed |