Predicted Tauopathies, IncludingPhase 1NCT01056965

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

A Phase 1 study testing davunetide (AL-108, NAP) in people with Predicted Tauopathies, Including and Progressive Supranuclear Palsy, run by University of California, San Francisco at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 40 to 85 years
Conditions the study is for: Predicted Tauopathies, Including, Progressive Supranuclear Palsy, Frontotemporal Dementia With Parkinsonism Linked to Chromosome 17
What is being tested?: davunetide (AL-108, NAP)
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 12 participants
Where: 1 siteUnited States
Study dates: Jan 2010to Dec 2012
Phase: Phase 1
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 40 to 85 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

A probable tauopathy defined as:
Probable or possible progressive supranuclear palsy (PSP) defined as:
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;
age at symptom onset ≥ 40 years by history; and

Show all 39Show fewer

an akinetic-rigid syndrome with prominent axial rigidity.
OR,
Progressive nonfluent aphasia (PNFA)defined as:
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
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:
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
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.
Documented age 40-85 years at the time of the onset of symptoms associated with the neurological deficits described in inclusion criterion 1.
Judged by investigator to be able to comply with neuropsychological evaluation at baseline.
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.
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.
MMSE ≥ 15 at Visit 1.
Written informed consent provided by both subject and caregiver who are both fluent English speakers.
Subject resides outside a skilled nursing facility or dementia care facility. Residence in an assisted living facility is allowed.
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.
Able to tolerate MRI scan during screening without use of sedation.
Able to ambulate with or without assistance.

Who cannot join

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,

Show all 31Show fewer

Cerebellar ataxia,
Choreoathetosis,
Early, symptomatic autonomic dysfunction, or
Tremor at rest.
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.
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).
Treatment with lithium, methylene blue, tramiprosate, ketone bodies, Dimebon 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 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.
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.
Systolic blood pressure greater than 180 or less than 90 mm Hg. Diastolic blood pressure greater than 105 or less than 50 mm Hg.
ECG abnormal at screening and judged to be clinically significant by the site investigator.
Treatment with any investigational drugs or device or participation in an investigational drug study within 60 days of screening.
Known history of serum or plasma progranulin level < 110.9 ng/mL.
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).
History of deep brain stimulator surgery other than sham surgery for DBS clinical trial.
History of early, prominent REM behavior disorder.
Women of childbearing potential who are not using at least two forms of medically recognized contraception.
An employee or relative of an employee of Allon Therapeutics
Significant anatomical nasal abnormality (e.g., septal deviation obstructing airflow to at least one nostril or septal perforation) or history of nasal turbinate surgery.
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.
Contraindication to MRI examination for any reason (eg., severe claustrophobia, ferromagnetic metal in body, etc.).
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.
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.
Subject not willing to attempt LP.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of California, San Francisco (UCSF)
San Francisco, California, 94143-1207

Other trials in United States

About this study

Official title: A 12 Week Randomized, Double Blind, Placebo-Controlled Pilot Study of Davunetide (NAP, AL-108) in Predicted Tauopathies

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.

Conditions: Predicted Tauopathies, Including, Progressive Supranuclear Palsy, Frontotemporal Dementia With Parkinsonism Linked to Chromosome 17, Corticobasal Degeneration Syndrome, Progressive Nonfluent Aphasia
Being tested: davunetide (AL-108, NAP), Placebo nasal spray
Keywords: Frontotemporal Dementia, Corticobasal Degeneration, Progressive Nonfluent Aphasia, Progressive Supranuclear Palsy, FTLD, FTD, PNFA, CBD, PSP, Predicted tauopathies, including:, Progressive Supranuclear Palsy (PSP), (CBS), Progressive Nonfluent Aphasia (PNFA)
Medical subject headings: Supranuclear Palsy, Progressive, Primary Progressive Nonfluent Aphasia, Frontotemporal Dementia, Corticobasal Degeneration, Frontotemporal Lobar Degeneration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

davunetide (AL-108, NAP)Drug
Also called AL-108, NAP
Subjects will be randomized 2:1 (drug:placebo). Subjects will receive twice daily treatment with davunetide 15 mg administered intranasally.
Placebo nasal sprayDrug
Subjects will be randomized 2:1 (drug:placebo). Subjects will receive twice daily treatment with placebo administered intranasally.

Study groups in NCT01056965
Study group	Role	What they receive
davunetide (Al-108, NAP) nasal spray	Experimental	Subjects will be randomized 2:1 (drug:placebo). Subjects will receive twice daily treatment with either davunetide 15 mg or placebo. Davunetide and placebo will be administered intranasally with a multi-dispensing, metered nasal spray pump device. Receives: Drug: davunetide (AL-108, NAP)
Placebo nasal spray	Placebo comparator	Not described Receives: Drug: Placebo nasal spray

What is being measured

The primary outcome is the main question the study is designed to answer.

Safety evaluations will be performed by recording clinical adverse events at each study visit. Clinical laboratory, ECGs, physical examinations will be conducted.
Measured: 12 weeks

Secondary outcomes (12)

PSP Rating Scale
Measured: 12 weeks
Clinician's Global Impression (CGI-ds)
Measured: 12 weeks
Schwab and England Activities of Daily Living scale (SEADL)
Measured: 12 weeks
MRI brain ventricular volume
Measured: 12 weeks
Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)
Measured: 12 weeks
Unified Parkinson's Disease Rating Scale (UPDRS)
Measured: 12 weeks
Neuropsychiatric Inventory (NPI)
Measured: 12 weeks
Geriatric Depression Scale (GDS)
Measured: 12 weeks
CSF biomarkers will assess total tau, phosphorylated tau, and amyloid beta peptide (1-42)
Measured: 12 weeks
Saccadic Eye movements - vertical and horizontal total saccade time
Measured: 12 weeks
Clinical Dementia Rating (CDR)
Measured: 12 weeeks
Functional Activities Questionnaire (FAQ)
Measured: 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 2010Dec 2012

First posted26 Jan 2010 (estimated)
Study startJan 2010
Primary completionDec 2012
Study completionDec 2012

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (46)Papers the sponsor has linked to this study.
Other trials for Predicted Tauopathies, IncludingStudies of the same condition, open and under way.
More trials run by University of California, San FranciscoThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 12 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01056965
Sponsor's protocol ID: AL-108 NAP Pilot
Status last verified: Apr 2019
First submitted: 21 Jan 2010
Last updated: 5 Apr 2019
Results posted: No
Data monitoring committee: Yes

Publications

Antoniades 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.
PubMed 17589313
Antoniades 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.
PubMed 20477368
Bassan 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.
PubMed 10037502
Bensimon 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.
PubMed 19029129
Boeve 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.
PubMed 12833363
Brenneman DE, Gozes I. A femtomolar-acting neuroprotective peptide. J Clin Invest. 1996 May 15;97(10):2299-307. doi: 10.1172/JCI118672.
PubMed 8636410
Cairns 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.
PubMed 17579875
Cummings 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.
PubMed 9153155
Detre 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.
PubMed 19491678
Dickson DW. Neuropathologic differentiation of progressive supranuclear palsy and corticobasal degeneration. J Neurol. 1999 Sep;246 Suppl 2:II6-15. doi: 10.1007/BF03161076.
PubMed 10525997
Fahn S, Elton RL, Committee. Unified Parkinson's Disease Rating Scale. In: Fahn S, Marsden CD, Calne D and Goldstein M, editors. Recent Developments in Parkinson's Disease. Vol 2. Florham Park, NJ: Macmillan Health-care Information, 1987: 153-163.
Furman 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.
PubMed 16845437
Garbutt 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.
PubMed 18362099
Giladi E, Hill JM, Dresner E, Stack CM, Gozes I. Vasoactive intestinal peptide (VIP) regulates activity-dependent neuroprotective protein (ADNP) expression in vivo. J Mol Neurosci. 2007;33(3):278-83. doi: 10.1007/s12031-007-9003-0. Epub 2007 Oct 2.
PubMed 17952637
Golbe LI, Ohman-Strickland PA. A clinical rating scale for progressive supranuclear palsy. Brain. 2007 Jun;130(Pt 6):1552-65. doi: 10.1093/brain/awm032. Epub 2007 Apr 2.
PubMed 17405767

Study at a glance

From the registry record, last updated by the sponsor on 5 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A 12 Week Randomized, Double Blind, Placebo-Controlled Pilot Study of Davunetide (NAP, AL-108) in Predicted Tauopathies
ClinicalTrials.gov ID: NCT01056965
Acronym / sponsor ID: AL-108 NAP Pilot
Conditions: Predicted Tauopathies, Including, Progressive Supranuclear Palsy, Frontotemporal Dementia With Parkinsonism Linked to Chromosome 17, Corticobasal Degeneration Syndrome, Progressive Nonfluent Aphasia
Keywords: Frontotemporal Dementia, Corticobasal Degeneration, Progressive Nonfluent Aphasia, Progressive Supranuclear Palsy, FTLD, FTD, PNFA, CBD
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 12 participants (actual)
Lead sponsor: University of California, San FranciscoUniversity, hospital or other
Study start: Jan 2010
Primary completion: Dec 2012 (actual)
Study completion: Dec 2012 (actual)
First posted: 26 Jan 2010 (estimated)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
davunetide (Al-108, NAP) nasal spray	Experimental	Drug: davunetide (AL-108, NAP)Subjects will be randomized 2:1 (drug:placebo). Subjects will receive twice daily treatment with either davunetide 15 mg or placebo. Davunetide and placebo will be administered intranasally with a multi-dispensing, metered nasal spray pump device.
Placebo nasal spray	Placebo comparator	Drug: Placebo nasal spray

Who may qualify

Ages: 40 to 85 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (39 listed)

A probable tauopathy defined as:
OR,
OR,
OR
Documented age 40-85 years at the time of the onset of symptoms associated with the neurological deficits described in inclusion criterion 1.

Key exclusion criteria (31 listed)

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:
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.
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).

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
davunetide (AL-108, NAP)Also: AL-108, NAPSubjects will be randomized 2:1 (drug:placebo). Subjects will receive twice daily treatment with davunetide 15 mg administered intranasally.	Drug	davunetide (Al-108, NAP) nasal spray
Placebo nasal spraySubjects will be randomized 2:1 (drug:placebo). Subjects will receive twice daily treatment with placebo administered intranasally.	Drug	Placebo nasal spray

Primary outcome

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

12 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
Clinical Dementia Rating (CDR)
Time frame: 12 weeeks
Functional Activities Questionnaire (FAQ)
Time frame: 12 weeks

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2019.
Background: PubMed 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.
Background: PubMed 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.
Background: PubMed 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.
Background: PubMed 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.
Background: PubMed 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.
Background: PubMed 8636410Brenneman DE, Gozes I. A femtomolar-acting neuroprotective peptide. J Clin Invest. 1996 May 15;97(10):2299-307. doi: 10.1172/JCI118672.
Background: PubMed 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.
Background: PubMed 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.
Background: PubMed 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.
Background: PubMed 10525997Dickson DW. Neuropathologic differentiation of progressive supranuclear palsy and corticobasal degeneration. J Neurol. 1999 Sep;246 Suppl 2:II6-15. doi: 10.1007/BF03161076.
BackgroundFahn S, Elton RL, Committee. Unified Parkinson's Disease Rating Scale. In: Fahn S, Marsden CD, Calne D and Goldstein M, editors. Recent Developments in Parkinson's Disease. Vol 2. Florham Park, NJ: Macmillan Health-care Information, 1987: 153-163.
Background: PubMed 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.

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Adam L. Boxer, M.D., Ph.D. principal investigator

UCSF Memory and Aging Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 39 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01056965, which also lists 46 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Apr 2019.