Study Details
AMX0035 for People with Progressive Supranuclear Palsy
(IRB#: IRB_00172910)
Progressive Supranuclear Palsy (PSP) is a nervous system disease that affects nerve cells in the brain and spinal cord (connects the brain to the rest of the body). The disease worsens over time causing loss of muscle control such as slowing body movement and balance. AMX0035 is a study drug for adults with this disease. The study will test the drug AMX0035 to see if it will help adults with the disease and is safe. AMX0035 is a fixed dose combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the endoplasmic reticulum (ER) and mitochondria. This clinical trial is designed to demonstrate that AMX0035 is safe and tolerable, and to assess its effect on disease progression as measured by the Progressive Supranuclear Palsy (PSP) Rating Scale (PSPRS) over a 52-week double-blind phase. The Phase 2b and Phase 3 study portions are planned to feature an identical design: a randomized, double-blind, placebo-controlled phase that is followed by an optional open-label extension (OLE) phase. The phase 3 portion of ORION may be initiated based on results of the phase 2b Interim Analysis and/or the Primary Analysis and the totality of data from the Phase 2b study portion.
- All genders
- Over 18 years old
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: All genders
Age: Over 18 years old
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria
- Ages 40 to 80 years old
- Diagnosis of possible PSP
- PSP symptoms for less than 5 years
- Able to walk alone or with little help
- Must reside outside a skilled nursing facility or dementia care facility at the time of screening. Residence in an assisted living facility is allowed.
Exclusion Criteria
- Require use of feeding tube
- Other nervous system disorders such as Parkinsons disease and multiple system atrophy
- Prior or current diagnosis of schizophrenia, schizoaffective disorder, or bipolar disorder
- History of autosomal dominant PSP due to a Microtubule Associated Protein Tau (MAPT) mutation
- Abnormal liver function
Will I be paid for my time?
Yes
IRB#: IRB_00172910
PI: Paolo Moretti
Department: NEUROLOGY
Approval Date: 2024-11-14 07:00:00
Study Categories: Brain Health Studies
Specialties: Neurology
I am Interested