AMX0035 and Progressive Supranuclear Palsy
- Conditions
- Progressive Supranuclear PalsyMedDRA version: 21.1Level: PTClassification code: 10036813Term: Progressive supranuclear palsy Class: 100000004852Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2023-505893-14-00
- Lead Sponsor
- Amylyx Pharmaceuticals Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 600
Provide a signed informed consent form (ICF) and have the mental capability to understand it. If participant is unable to sign the ICF, the ICF must be signed by a representative in accordance with local regulatory requirements., Score of =24 on the Mini Mental State Examination (MMSE)., Dosing of antiparkinsonian drugs (coenzyme Q10, levodopa/carbidopa, levodopa/benserazide, fava bean extract, a dopamine agonist, catechol-O-methyltransferase inhibitor, amantadine, or other Parkinson’s disease medications) should be stable for 60 days before enrollment and are anticipated to remain stable for the double-blind phase of the study at the discretion of the Investigator, All female participants must have a negative serum pregnancy test at Screening., Female participants of childbearing potential (women of childbearing potential [WOCBP]) who engage in heterosexual intercourse must have a negative urine pregnancy test on Day 1 prior to the start of the study., WOCBP must agree to abstain from heterosexual intercourse or use a highly effective birth control method for the duration of the study and for 6 months after last dose of study drug. Female participants who are two-years post-menopausal or surgically sterile are not considered to have childbearing potential., Female participants must not be planning to become pregnant for the duration of the study and for 6 months after the last dose of study drug., Female participants must not be planning to or be breastfeeding for duration of the study and 6 months after the last dose of study drug., Male participants must agree to abstain from heterosexual intercourse or use a highly effective birth control method for the duration of the study and for 6 months after the last dose of study drug., Male participants must not be planning to father a child or provide sperm for donation for the duration of the study and for 6 months after the last dose of study drug., Are willing and able to comply with scheduled visits, treatment schedule, laboratory tests, and other requirements of the study, including MRI scans, Participant’s study partner is willing and able to comply with scheduled visits, treatment schedule, laboratory tests, and other requirements of the study. The participant’s study partner is defined as a caregiver, family member, social worker, or friend who has frequent contact with the participant (=10 hours per week), will accompany the participant to study visits to provide information as to the participant’s functional abilities, and will speak the local language fluently to ensure comprehension of informed consent and informant-based assessments of participant., Male or female 40 to 80 years of age, inclusive., Participant must reside outside a skilled nursing facility or dementia care facility at the time of Screening and admission to such a facility must not be planned. Residence in an assisted living facility is allowed., Meets the following criteria for possible or probable PSP (Steele-Richardson-Olszewski Syndrome) according to MDS 2017 criteria: a. Gradually progressive disorder, with age at disease onset = 40 years b. Either or both of the following two items are met: i. Vertical supranuclear gaze palsy OR slow velocity of vertical saccades AND postural instability with repeated unprovoked falls within 3 years OR tendency to fall on the pull-test within 3 years ii. Slow velocity of vertical saccades AND postural instability with more than two steps backward on the pull-test within 3
Participants who have previous exposure to AMX0035 or have a known hypersensitivity to AMX0035, either of its components, any of its excipients, or bile salts, Clinically significant infection, inflammation, or medical condition other than PSP that would pose a risk to the participant if they were to participate or impair their ability to participate in the study, in the judgment of the Investigator, including: a. Acute gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea) at time of Screening or at Day 1 prior to dosing b. Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis) c. Severe salt restriction, where added salt intact due to treatment with study drug would put the participant at risk, 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. Evidence of disease may be provided by MRI., Prior or current diagnosis of schizophrenia, schizoaffective disorder, or bipolar disorder according to Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-V) or International Classification of Diseases (ICD)-10 criteria, 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, Significant suicidal ideation within 1 year prior to Screening as evidenced by answering yes” to questions 4 or 5 on the suicidal ideation portion of the Columbia-Suicide Severity Rating Scale (C-SSRS) at Screening or history of suicidal attempts within the last 2 years, Participant who has participated in any other clinical investigation using an experimental drug within 5 half-lives or within 6 weeks (small molecules) or 6 months (monoclonal antibodies, antisense oligonucleotides, or other biologics), whichever is longer, prior to Day 1., Gene or cell therapy prior to Screening or during study, Exposure to any prohibited therapy within 30 days prior to Screening, Anything that, in the opinion of the Investigator, precludes the participant’s full compliance with or completion of this study, Require use of a feeding tube., Evidence of any neurological disorder that could explain signs of PSP, including: a. Signs of idiopathic Parkinson's disease (e.g., severe asymmetric parkinsonian signs, clinically significant tremor at rest, or prominent and sustained response to levodopa therapy or other medications that are used to treat Parkinson’s disease) b. Signs of multiple system atrophy (MSA) (e.g., prominent early cerebellar limb ataxia or unexplained symptomatic autonomic dysfunction) c. Signs of Lewy body disease (e.g., hallucinations or delusions unrelated to dopaminergic therapy or other illness) d. Probable Alzheimer’s disease according to National Institute of Aging – Alzheimer’s Association (NIA-AA) core clinical criteria for mild cognitive impairment due to Alzheimer’s disease or AD dementia e. History of repeated strokes with stepwise progression of parkinsonian features f. History of major stroke g. History of severe or repeated head injury h. History of encephalitis i. History of neuroleptic use within
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method