Table 3.
Clinical development of other therapies for disease modification in HD according to the proposed mechanism of action. (HTT-based approaches were excluded)
| Compound | Mechanism of action | Name of the study | Registration ID (last date of update) | Study phase | Results |
|---|---|---|---|---|---|
| Huntingtin protein homeostasis | |||||
| PBT2 | Metal-protein attenuating compound | Reach2HD | NCT01590888 (July 2016) | II |
Completed Negative for efficacy [16] |
| Selisistat | Sirtuin-1 inhibition |
Paddington – |
NCT01485952 (November 2015) NCT01521585 (November 2015) |
I II |
Completed |
| Immunomodulation | |||||
| Semaphorin 4AD | Monoclonal antibody | SIGNAL | NCT02481674 (May 2020) | II |
Ongoing Not recruiting |
| Laquinimod |
Nf-kb inhibition Cytokine release |
LEGATO-HD | NCT02215616 (May 2020) | II |
Completed Negative for efficacy [107] |
| Other | |||||
| PF-02545920 | Phosphodiesterase 10A inhibition |
APACHE Amaryllis |
NCT01806896 (December 2017) NCT02197130 (December 2017) |
II |
Completed |
| Nilotinib | Bcr-Abl tyrosine kinase inhibitor | Tasigna HD | NCT03764215 (February 2020) | Ib |
Ongoing Recruiting |
| Cysteamine | Multiple mechanisms | CYST-HD | NCT02101957 (April 2014) | II/III |
Completed Negative for efficacy [108] |
| SBT-20 | Mitochondrial function | CHALLENGE-HD | EUCTR2016-003730-25-NL (February 2018) | Ia/II |
Ongoing Not recruiting |
| Fenofibrate | Transcriptional co-activator (PGC-1α) | – | NCT03515213 (March 2019) | II |
Ongoing Not recruiting |
| Resveratrol | Sirtuin-1, PGC-1α modulator | – | NCT02336633 (February 2020) | – |
Completed No results in public domain |