AMX0035 and Progressive Supranuclear Palsy

Overview

About this study

The purpose of this study is to assess the impact of AMX0035 compared to placebo on disease progression rate as measured by the Progressive Supranuclear Palsy (PSP) Rating Scale (PSPRS).

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- Male or female 40 to 80 years of age, inclusive

- Diagnosis of possible or probable PSP Richardson Syndrome

- Presence of PSP symptoms for < 5 years

- Score of < 40 on the total (28-item) Progressive Supranuclear Palsy Rating Scale
(PSPRS)

- Able to walk independently or with minimal assistance

- Minimum score of 24 on the Mini Mental State Examination (MMSE)

- Must reside outside a skilled nursing facility or dementia care facility at the time
of screening. Residence in an assisted living facility is allowed

- Must have a study partner willing to attend study visits and provide information on
participant's status

- Capable of providing informed consent

- Capable and willing to comply with trial procedures including visits to the trial
clinic, visit requirements and treatment schedule, including MRI scans

- Female participants of childbearing potential must agree to use effective birth
control for the duration of the study and for 6 months after last dose of study drug.

- Males must agree to use effective birth control method for the duration of the study
and for 6 months after the last dose of study drug. Men must not plan to donate
sperm..

Exclusion Criteria:

- Require use of a feeding tube

- Evidence of any neurological disorder that could explain signs of PSP

- Evidence of any clinically significant neurological disorder other than PSP, including
significant cerebrovascular abnormalities, vascular dementia, motor neuron disease or
ALS, Huntington's disease, normal pressure hydrocephalus, brain tumor, seizure
disorder, multiple sclerosis, or known structural brain abnormalities.

- History of autosomal dominant PSP due to a Microtubule Associated Protein Tau (MAPT)
mutation

- History of an autosomal dominant mutation associated with Frontotemporal Lobar
Degeneration (FTLD)

- Prior or current diagnosis of schizophrenia, schizoaffective disorder, or bipolar
disorder

- Presence of unstable psychiatric disease, cognitive impairment (e.g., major cognitive
dysfunction), dementia, major depression, or substance abuse that would impair ability
of the participant to provide informed consent and follow instructions

- Abnormal liver function

- Renal insufficiency

- Ongoing anemia

- History of Class III/IV heart failure per New York Heart Association (NYHA)

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 12/29/23. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

James Bower, M.D.

Open for enrollment

Contact information:

Sherry Klingerman CCRP

(507) 284-0451

klingerman.sherry@mayo.edu

Jacksonville, Fla.

Mayo Clinic principal investigator

Zbigniew Wszolek, M.D.

Open for enrollment

Contact information:

Audrey Strongosky C.C.R.C.

Strongosky.Audrey2@mayo.edu

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Erika Driver-Dunckley, M.D.

Open for enrollment

Contact information:

Hannah Henderson CCRP

(480) 301-6091

Henderson.Hannah2@mayo.edu

More information

Publications

Publications are currently not available