Study to Evaluate the Safety and Efficacy of Davunetide for the Treatment of Progressive Supranuclear Palsy

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Progressive Supranuclear Palsy

In brief

The purpose of the study is to evaluate the safety and efficacy of davunetide for the treatment of Progressive Supranuclear Palsy.

Key facts

Study ID

NCT01110720

Run by

Allon Therapeutics

People needed

313

Starts

2010-10-01

Expected to finish

2012-12-01

Last updated by the study team

2013-01-17

Who can join

Age: 41 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Probable or possible PSP defined as:
• at least a 12-month history of postural instability or falls during the first 3 years that symptoms are present; and
• at screening, a decreased downward saccade velocity defined as observable eye movement (deviation from the "main sequence" linear relationship between saccade amplitude and saccade velocity) or, supranuclear ophthalmoplegia defined as 50% reduction in upward gaze or 30% reduction in downward gaze; and
• age at symptom onset of 40 to 85 years by history; and
• an akinetic-rigid syndrome with prominent axial rigidity.
• Aged 41 to 85 years at the time of screening.
• Judged by investigator to be able to comply with neuropsychological evaluation at baseline and throughout the study.
• Must have reliable caregiver accompany subject to all study visits. Caregiver must read, understand, and speak local language fluently to ensure comprehension of informed consent form and informant-based assessments of subject. Caregiver must also have frequent contact with subject (at least 3 hours per week at one time or at different times) and be willing to monitor study medication compliance and the subject's health and concomitant medications throughout the study.
• Modified Hachinski score ≤ 3 (Appendix 7). This modified Hachinski will not include the focal neurological signs, symptoms or pseudobulbar affect questions, given the prominence of all 3 in PSP.
• Score ≥ 15 on the mini-mental state examination (MMSE) at screening (Visit 1).
• Written informed consent provided by subject (or legally-appointed representative, as appropriate) and caregiver (if not the legally-appointed representative) who are both fluent local language speakers.
• Subject resides outside a skilled nursing facility or dementia care facility at the time of screening, and admission to such a facility is not planned. Residence in an assisted living facility is allowed.
• If the subject is receiving levodopa/carbidopa, levodopa/benserazide, a dopamine agonist, catechol-o-methyltransferase (COMT) inhibitor, or other Parkinson's medication,with teh exception of Azilect(rasagiline), the dose must have been stable for at least 60 days prior to the screening visit (Visit 1) and must remain stable for the duration of the study. No such medication can be initiated during the study. Subjects receiving rasagiline or CoQ10 must be on a stable dose for at least 90 days prior to the screening visit.
• Able to tolerate the MRI scan during screening with either no sedation or low dose lorazepam.
• Able to ambulate independently or with assistance defined as the ability to take at least 5 steps with a walker (guarding is allowed provided there is no contact) or the ability to take at least 5 steps with the assistance of another person who can only have contact with one upper extremity.
• Presence of symptoms for less than 5 years or the presence of symptoms for more than 5 years with a PSPRS baseline score ≥ 40.
• Stable on all other chronic medications for at least 30 days prior to the screening visit (Visit 1).

You may not qualify if…

• Insufficient fluency in local language to complete neuropsychological and functional assessments.
• A diagnosis of Amyotrophic Lateral Sclerosis or other motor neuron disease.
• Any of the following:
• Abrupt onset of symptoms defined in inclusion criteria 1 associated with ictal events,
• Head trauma related to onset of symptoms defined in inclusion criteria 1,
• Severe amnesia within 6 months of the symptoms defined in inclusion criteria 1,
• Cerebellar ataxia,
• Choreoathetosis,
• Early, symptomatic autonomic dysfunction; or
• Tremor while at rest.
• Presence of other significant neurological or psychiatric disorders including (but not limited to) Alzheimer's disease; dementia with Lewy bodies; prion disease; Parkinson's disease (which has not subsequently been revised to PSP); any psychotic disorder; severe bipolar or unipolar depression; seizure disorder; tumor or other space-occupying lesion; or history of stroke or head injury with loss of consciousness for at least 15 minutes within the past 20 years.
• Within 4 weeks of screening or during the course of the study, concurrent treatment with memantine; acetylcholinesterase inhibitors; antipsychotic agents (other than quetiapine) or mood stabilizers (e.g., valproate, lithium); or benzodiazepines (except as below).
• Low dose lorazepam (not more than 2 mg) may be used for sedation prior to MRI scans for those subjects requiring sedation. Neuropsychological testing may not be performed after lorazepam administration.
• Subjects who take short acting benzodiazepines (only temazepam or zolpidem are allowed) for sleep may continue to do so if they have been on a stable dose for 30 days prior to screening.
• Clonazepam may be used for treatment of dystonia or painful rigidity associated with PSP if the dose has been stable for 90 days prior to screening and is not expected to change during the course of the study.
• Treatment with lithium, methylene blue, tramiprosate, ketone bodies, latrepirdine, or any putative disease-modifying agent directed at tau within 90 days of screening.
• A history of alcohol or substance abuse within 1 year prior to screening and deemed to be clinically significant by the site investigator.
• Any malignancy (other than non-metastatic dermatological conditions) within 5 years of the screening visit (Visit 1) or current clinically significant hematological, endocrine, cardiovascular, renal, hepatic, gastrointestinal, or neurological disease. For the non-cancer conditions, if the condition has been stable for at least one year before the screening visit and is judged by the site investigator not to interfere with the subject's participation in the study, the subject may be included.
• Clinically significant laboratory abnormalities at screening, including creatinine ≥ 2.5 mg/dL, alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 3 times the upper limit of the normal reference range, vitamin B12 below the laboratory normal reference range, or thyroid stimulating hormone TSH above laboratory normal reference range.
• The systolic blood pressure measurement is > 190 or < 85 mm Hg. The diastolic blood pressure measurement is > 105 or < 50 mm Hg at screening.
• Abnormal ECG tracing at screening and judged to be clinically significant by the site investigator.
• Treatment with any investigational drugs or device within 90 days of screening.
• Known history of serum or plasma progranulin level less than one standard deviation below the normal subject mean for the laboratory performing the assay.
• Known presence of known disease-associated mutation in TDP-43, PGRN, CHMPB2, or VCP genes or any other frontotemporal lobar degeneration (FTLD) causative genes not associated with underlying tau pathology (e.g., Chromosome 9 associated FTD).
• History of deep brain stimulator (DBS) surgery other than sham surgery for DBS clinical trial.

Where it is running

• University of Alabama - Birmingham — Birmingham, Alabama, United States
• Muhammed Ali Parkinson Center and Movement Disorders Clinic — Phoenix, Arizona, United States
• Mayo Clinic, AZ — Scottsdale, Arizona, United States
• USC Keck School of Medicine — Los Angeles, California, United States
• David Geffen School of Medicine - UCLA — Los Angeles, California, United States
• UCSD/VA Neurology Service — San Diego, California, United States
• UCSF Memory and Aging Center — San Francisco, California, United States
• Colorado Neurological Institute - Rocky Mountain Movement Disorders Ctr, PC — Englewood, Colorado, United States
• Parkinson's Disease and Movement Disorders Center of Boca Raton — Boca Raton, Florida, United States
• Mayo Clinic, Florida — Jacksonville, Florida, United States
• The Frances J. Zesiewicz Foundation for Parkinson's Disease at USF — Tampa, Florida, United States
• University of Chicago Medical Center — Chicago, Illinois, United States
• University of Kansas Medical Center Parkinson Disease & Movement Disorders Center — Kansas City, Kansas, United States
• University of Louisville Division of Movement Disorders — Louisville, Kentucky, United States
• John Hopkins Hospital — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Lahey Clinic — Burlington, Massachusetts, United States
• University of Michigan Medical Center — Ann Arbor, Michigan, United States
• University of Minnesota Department of Neuology — Minneapolis, Minnesota, United States
• Mayo Clinic, Rochester, MN — Rochester, Minnesota, United States
• UMDNJ - Robert Wood Johnson Medical Center — New Brunswick, New Jersey, United States
• Columbia University — New York, New York, United States
• Univeristy of North Carolina Department of Neurology — Chapel Hill, North Carolina, United States
• University Hospitals Case Medical CenterNI Movement Disorders Center — South Euclid, Ohio, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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