Product Description
Arimoclomol, taken at a dose of 200 mg three times daily will improve survival as defined by time to death, tracheostomy or permanent assisted ventilation. In addition, it will be safe and well tolerated in subjects with SOD1 positive familial ALS. (Sourced from: https://clinicaltrials.gov/ct2/show/NCT00706147)
Mechanisms of Action: HSP70 Modulator
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Fast Track - Amyotrophic Lateral Sclerosis *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Orphazyme
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: United Kingdom, United States
Active Clinical Trial Count: 2
Highest Development Phases
Phase 2: Myositis, Inclusion Body
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C. | P3 |
Active, not recruiting |
Unknown |
2021-04-19 |
|
| STUDY00002461 | P2 |
Completed |
Myositis, Inclusion Body |
2021-01-11 |