Current partner codePEPTIDESDE
NCT01056965·Phase 1·INTERVENTIONAL

Davunetide (AL-108) in Predicted Tauopathies - Pilot Study

Status

Completed

Phase

Phase 1

Enrollment

12

Locations

1

Results

Not posted

Publications

12

Study summary

What the protocol is testing.

The primary objective of the study is to obtain preliminary safety and tolerability data with davunetide (NAP, AL-108) in patients with a tauopathy (frontotemporal lobar degeneration \[FTLD\] with predicted tau pathology, corticobasal degeneration syndrome \[CBS\] or progressive supranuclear palsy \[PSP\]). The secondary objectives of this study are to obtain preliminary data on short term changes (at 12 weeks) in a variety of clinical, functional and biomarker measurements from baseline, including cerebrospinal fluid (CSF) tau levels, eye movements, and brain MRI measurements.

Interventions

Treatment arms and agents.

DRUG

davunetide (AL-108, NAP)

Subjects will be randomized 2:1 (drug:placebo). Subjects will receive twice daily treatment with davunetide 15 mg administered intranasally.

DRUG

Placebo nasal spray

Subjects will be randomized 2:1 (drug:placebo). Subjects will receive twice daily treatment with placebo administered intranasally.

Timeline

From registration to results.

  1. First posted

    Jan 26, 2010

  2. Study start

    Jan 2010

  3. Primary completion

    Dec 2012

  4. Study completion

    Dec 2012

  5. Results posted

    Not reported

  6. Registry updated

    Apr 5, 2019

Outcomes

What the study measures.

Primary outcomes

Safety evaluations will be performed by recording clinical adverse events at each study visit. Clinical laboratory, ECGs, physical examinations will be conducted.

Time frame · 12 weeks

Secondary outcomes

PSP Rating Scale

Time frame · 12 weeks

Clinician's Global Impression (CGI-ds)

Time frame · 12 weeks

Schwab and England Activities of Daily Living scale (SEADL)

Time frame · 12 weeks

MRI brain ventricular volume

Time frame · 12 weeks

Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)

Time frame · 12 weeks

Unified Parkinson's Disease Rating Scale (UPDRS)

Time frame · 12 weeks

Neuropsychiatric Inventory (NPI)

Time frame · 12 weeks

Geriatric Depression Scale (GDS)

Time frame · 12 weeks

CSF biomarkers will assess total tau, phosphorylated tau, and amyloid beta peptide (1-42)

Time frame · 12 weeks

Saccadic Eye movements - vertical and horizontal total saccade time

Time frame · 12 weeks

Eligibility

Who can take part.

Minimum age
40 Years
Maximum age
85 Years
Sex
ALL
Healthy volunteers
No

Inclusion Criteria: 1. A probable tauopathy defined as: * Probable or possible progressive supranuclear palsy (PSP) defined as: 1. at least a 12-month history of: * postural instability or falls during the first 3 years that symptoms are present and * prominent decreased saccade velocity or supranuclear ophthalmoplegia; 2. age at symptom onset ≥ 40 years by history; and 3. an akinetic-rigid syndrome with prominent axial rigidity. OR, * Progressive nonfluent aphasia (PNFA)defined as: 1. at least a 6-month history of difficulty with expressive speech characterized by at least 3 of the following: * apraxia of speech, * speech hesitancy, * labored speech, * word finding difficulty, or * agrammatism; and 2. the symptoms above are the subject's principal neurological deficit and the symptoms constituted the initial clinical presentation. OR, * Corticobasal Degeneration syndrome (CBS) defined as: 1. at least a 6-month history of progressive cortical dysfunction evidenced by at least one of the following: * ideomotor apraxia, * alien limb phenomenon, * cortical sensory loss, * focal or asymmetric myoclonus, or * apraxia of speech /nonfluent aphasia; and 2. at least a 6-month history of progressive extrapyramidal dysfunction evidenced by at least one of the following: * focal or asymmetrical rigidity (limb or axial) or asymmetrical dystonia (limb or axial); and * lacking prominent and sustained L-dopa response. OR * Frontotemporal Dementia with Parkinsonism linked to Chromosome 17 (FTDP-17): Motor, cognitive or behavioral dysfunction, as defined below associated with a previously demonstrated mutation of the MAPT gene, and meets criteria for PNFA, CBS or PSP as defined above, or CDR-FTLD ≥ 1.0. 2. Documented age 40-85 years at the time of the onset of symptoms associated with the neurological deficits described in inclusion criterion 1. 3. Judged by investigator to be able to comply with neuropsychological evaluation at baseline. 4. Must have reliable caregiver accompany subject to all study visits. Caregiver must read, understand and speak local language fluently in order to ensure comprehension of informed consent form and informant-based assessments of subject. Caregiver must also have frequent contact with subject (at least 3 times per week for one hour) and be willing to monitor study medication compliance and the subject's health and concomitant medications throughout the study. 5. FTLD Modified Hachinski score ≤ 3.(Knopman et al., 2008) This modified Hachinski will not include the focal neurological signs, symptoms or pseudobulbar affect questions, given the prominence of all three in CBS/PSP. 6. MMSE ≥ 15 at Visit 1. 7. Written informed consent provided by both subject and caregiver who are both fluent English speakers. 8. Subject resides outside a skilled nursing facility or dementia care facility. Residence in an assisted living facility is allowed. 9. If the subject is receiving levodopa/carbidopa, a dopamine agonist, COMT inhibitor or other Parkinson's medication the dose must have been stable for at least 120 days prior to Visit 1 and must remain stable for the duration of the study. 10. Able to tolerate MRI scan during screening without use of sedation. 11. Able to ambulate with or without assistance. Exclusion Criteria: 1. Insufficient fluency in local language to complete neuropsychological and functional assessments. 2. A diagnosis of Amyotrophic Lateral Sclerosis or other motor neuron disease. 3. 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 at rest. 4. History of other significant neurological or psychiatric disorders including, but not limited to, Alzheimer's disease, dementia with Lewy bodies, Prion disease, stroke, Parkinson's disease, any psychotic disorder, severe bipolar or unipolar depression, seizure disorder, tumor or other space-occupying lesion, or head injury with loss of consciousness within past 20 years temporally related to onset of symptoms. 5. Within 4 weeks of screening or during the course of the study, concurrent treatment with memantine (stable dose memantine, greater than 6 months is allowed), acetylcholinesterase inhibitors, antipsychotic agents or mood stabilizers (valproate, lithium, etc.) or benzodiazepines (other than temazepam or zolpidem). 6. Treatment with lithium, methylene blue, tramiprosate, ketone bodies, Dimebon or any putative disease-modifying agent directed at tau within 90 days of screening. 7. A history of alcohol or substance abuse within 1 year prior to screening and deemed to be clinically significant by the site investigator. 8. Any malignancy (other than non-metastatic basal cell carcinoma of the skin) within 5 years of 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 the past year and is judged by the site investigator not to interfere with the patient's participation in the study, the patient may be included. 9. Clinically significant lab abnormalities at screening, including creatinine ≥ 2.5 mg/dL, vitamin B12 below laboratory normal reference range, or TSH above laboratory normal reference range. 10. Systolic blood pressure greater than 180 or less than 90 mm Hg. Diastolic blood pressure greater than 105 or less than 50 mm Hg. 11. ECG abnormal at screening and judged to be clinically significant by the site investigator. 12. Treatment with any investigational drugs or device or participation in an investigational drug study within 60 days of screening. 13. Known history of serum or plasma progranulin level \< 110.9 ng/mL. 14. Known presence of known disease-associated mutation in TDP-43, PGRN, CHMPB2 or VCP genes or any other FTLD causative genes not associated with underlying tau pathology (eg. Chr. 9 associated FTD). 15. History of deep brain stimulator surgery other than sham surgery for DBS clinical trial. 16. History of early, prominent REM behavior disorder. 17. Women of childbearing potential who are not using at least two forms of medically recognized contraception. 18. An employee or relative of an employee of Allon Therapeutics 19. Significant anatomical nasal abnormality (e.g., septal deviation obstructing airflow to at least one nostril or septal perforation) or history of nasal turbinate surgery. 20. History of a clinically significant medical condition that that would interfere with the subject's ability to comply with study instructions, would place the subject at increased risk, or might confound the interpretation of the study results. 21. Contraindication to MRI examination for any reason (eg., severe claustrophobia, ferromagnetic metal in body, etc.). 22. Structural abnormality on MRI within 2 years of baseline that precludes diagnosis of PSP, CBS or PNFA, such as cortical infarct in brain region that might account for subject's symptoms. 23. In subjects receiving anti-Parkinson's Disease medication at the time of screening, in the opinion of the investigator substantial worsening of motor signs or symptoms compared to normal functioning following overnight withdrawal of the anti-Parkinson medication. 24. Subject not willing to attempt LP.

Study locations

1 registered sites.

United States. Showing up to 24 locations stored in the fast local snapshot.

University of California, San Francisco (UCSF)

San Francisco, California, United States

Publications

Results and literature.

PMID 17589313Antoniades CA, Altham PM, Mason SL, Barker RA, Carpenter R. Saccadometry: a new tool for evaluating presymptomatic Huntington patients. Neuroreport. 2007 Jul 16;18(11):1133-6. doi: 10.1097/WNR.0b013e32821c560d.PMID 20477368Antoniades CA, Bak TH, Carpenter RH, Hodges JR, Barker RA. Diagnostic potential of saccadometry in progressive supranuclear palsy. Biomark Med. 2007 Dec;1(4):487-90. doi: 10.2217/17520363.1.4.487.PMID 10037502Bassan M, Zamostiano R, Davidson A, Pinhasov A, Giladi E, Perl O, Bassan H, Blat C, Gibney G, Glazner G, Brenneman DE, Gozes I. Complete sequence of a novel protein containing a femtomolar-activity-dependent neuroprotective peptide. J Neurochem. 1999 Mar;72(3):1283-93. doi: 10.1046/j.1471-4159.1999.0721283.x.PMID 19029129Bensimon G, Ludolph A, Agid Y, Vidailhet M, Payan C, Leigh PN; NNIPPS Study Group. Riluzole treatment, survival and diagnostic criteria in Parkinson plus disorders: the NNIPPS study. Brain. 2009 Jan;132(Pt 1):156-71. doi: 10.1093/brain/awn291. Epub 2008 Nov 23.PMID 12833363Boeve BF, Lang AE, Litvan I. Corticobasal degeneration and its relationship to progressive supranuclear palsy and frontotemporal dementia. Ann Neurol. 2003;54 Suppl 5:S15-9. doi: 10.1002/ana.10570. No abstract available.PMID 8636410Brenneman DE, Gozes I. A femtomolar-acting neuroprotective peptide. J Clin Invest. 1996 May 15;97(10):2299-307. doi: 10.1172/JCI118672.PMID 17579875Cairns NJ, Bigio EH, Mackenzie IR, Neumann M, Lee VM, Hatanpaa KJ, White CL 3rd, Schneider JA, Grinberg LT, Halliday G, Duyckaerts C, Lowe JS, Holm IE, Tolnay M, Okamoto K, Yokoo H, Murayama S, Woulfe J, Munoz DG, Dickson DW, Ince PG, Trojanowski JQ, Mann DM; Consortium for Frontotemporal Lobar Degeneration. Neuropathologic diagnostic and nosologic criteria for frontotemporal lobar degeneration: consensus of the Consortium for Frontotemporal Lobar Degeneration. Acta Neuropathol. 2007 Jul;114(1):5-22. doi: 10.1007/s00401-007-0237-2. Epub 2007 Jun 20.PMID 9153155Cummings JL. The Neuropsychiatric Inventory: assessing psychopathology in dementia patients. Neurology. 1997 May;48(5 Suppl 6):S10-6. doi: 10.1212/wnl.48.5_suppl_6.10s.PMID 19491678Detre JA, Wang J, Wang Z, Rao H. Arterial spin-labeled perfusion MRI in basic and clinical neuroscience. Curr Opin Neurol. 2009 Aug;22(4):348-55. doi: 10.1097/WCO.0b013e32832d9505.PMID 10525997Dickson DW. Neuropathologic differentiation of progressive supranuclear palsy and corticobasal degeneration. J Neurol. 1999 Sep;246 Suppl 2:II6-15. doi: 10.1007/BF03161076.PMID 16845437Furman S, Steingart RA, Mandel S, Hauser JM, Brenneman DE, Gozes I. Subcellular localization and secretion of activity-dependent neuroprotective protein in astrocytes. Neuron Glia Biol. 2004 Aug;1(3):193-9. doi: 10.1017/S1740925X05000013.PMID 18362099Garbutt S, Matlin A, Hellmuth J, Schenk AK, Johnson JK, Rosen H, Dean D, Kramer J, Neuhaus J, Miller BL, Lisberger SG, Boxer AL. Oculomotor function in frontotemporal lobar degeneration, related disorders and Alzheimer's disease. Brain. 2008 May;131(Pt 5):1268-81. doi: 10.1093/brain/awn047. Epub 2008 Mar 24.

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