Therapies in a current clinical trial
Arimoclomol
Sponsoring Organization: Orphzyme
Phase: Completed; not recruiting
Expanded Access: Yes, with restrictions
Contact: Regan Sherman, res@orphazyme.com
Clinical Trial website: https://www.clinicaltrials.gov/ct2/show/NCT02612129?term=orphazyme
ESB1609
Sponsoring Organization: Escape Bio
Phase: Phase 1 Active; not recruiting
Expanded Access: No
Contact: info@escapebio.com
Clinical Trial website:
IB1001
Sponsoring Organization: Intrabio
Phase: Completed; not recruiting
Expanded Access:
Contact: Taylor Fields, tfields@intrabio.com
Clinical Trial website: https://clinicaltrials.gov/ct2/show/NCT03759639?term=N-Acetyl-L-leucine
Trappsol Cyclo
Sponsoring Organization: Cyclo Therapeutics
Phase: Phase 1 Completed; not recruiting
Expanded Access: Yes, with restrictions
Contact: Dr. Sharon Hrynkow,
sharon.hrynkow@cyclodex.com
Clinical Trial website: https://clinicaltrials.gov/ct2/show/NCT03759639?term=N-Acetyl-L-leucine
Therapies in a completed Clinical Trial
Adrabetadex
Sponsoring Organization: Mallinckrodt Pharmaceuticals
Phase: Completed; not recruiting
Expanded Access Available: No
Contact: Sheila Talafous, Sheila.Talafous@mnk.com
Clinical Trial website: https://www.clinicaltrials.gov/ct2/show/study/NCT02534844?term=Vtesse&rank=1
Miglustat
Sponsoring Organization: Actelion, Edenbridge Pharmaceuticals (generic)
Access: Off label
Contact: Consult your clinician
Clinical Trial website: https://clinicaltrials.gov/ct2/show/NCT00517153?cond=Niemann-Pick+Disease%2C+Type+C&draw=3&rank=17