Primary Four Repeat Tauopathies (4RT)Phase 1TPI-287-4RTNCT02133846

Safety Study of TPI-287 to Treat CBS and PSP

A Phase 1 study testing TPI 287 2 mg/m2, TPI-287 20 mg/m2 and TPI-287 6.3 mg/m2 in people with Primary Four Repeat Tauopathies (4RT) and Corticobasal Syndrome (CBS), run by University of California, San Francisco at 2 sites in United States.

Completed

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

Official record

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Trial summary

The purpose of this study is to determine the safety and tolerability [maximum tolerated dose (MTD) within planned dosing range] of intravenous (IV) infusions of TPI 287 administered once every 3 weeks for 9 weeks (for a total of 4 infusions) in patients with primary four repeat tauopathies (4RT), corticobasal… Read more about this study

Who is it for?: Ages 50 to 85 years
Conditions the study is for: Primary Four Repeat Tauopathies (4RT), Corticobasal Syndrome (CBS), Progressive Supranuclear Palsy (PSP)
What is being tested?: TPI 287 2 mg/m2, TPI-287 20 mg/m2 and TPI-287 6.3 mg/m2
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 44 participants
Where: 2 sitesUnited States
Study dates: May 2014to Sep 2019
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 50 to 85 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Between 50 and 85 years of age (inclusive);
Able to walk 5 steps with minimal assistance (stabilization of one arm or use of cane/walker);
MRI at Screening is consistent with CBS or PSP (≤ 4 microhemorrhages, and no large strokes or severe white matter disease);
MMSE at Screening is between 14 and 30 (inclusive);
FDA-approved AD medications are sometimes prescribed for CBS and PSP subjects, and are allowed as long as the dose is stable for 2 months prior to Screening. Other medications (except those listed under exclusion criteria) are allowed as long as the dose is stable for 30 days prior to Screening;
FDA-approved Parkinson's medications are allowed as long as the dose is stable for 2 months prior to Screening;

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Has a reliable study partner who agrees to accompany the subject to visits, and spends at least 5 hours per week with the subject;
Agrees to 2 lumbar punctures;
Signed and dated written informed consent obtained from the subject and the subject's caregiver in accordance with local IRB regulations;
Males and all WCBP agree to abstain from sex or use an adequate method of contraception for the duration of the study and for 30 days after the last dose of study drug.
Adequate contraceptive methods include those with a low failure rate, i.e., less than 1% per year, when used consistently and correctly, such as complete abstinence from sexual intercourse with a potentially fertile partner, and some double barrier methods (condom with spermicide) in conjunction with use by the partner of an intrauterine device (IUD), diaphragm with spermicide, oral contraceptives, birth control patch or vaginal ring, oral, or injectable or implanted contraceptives.
For this study, a woman who has been surgically sterilized or who has been in a state of amenorrhea for more than two years will be deemed not to be of childbearing potential;
For PSP Only
Meets National Institute of Neurological Disorders and Stroke - Society for Progressive Supranuclear Palsy (NINDS-SPSP) probable or possible PSP criteria (Litvan et al. 1996a), as modified for the Neuroprotection and Natural History in Parkinson Plus Syndromes (NNIPPS) clinical trial (Bensimon et al. 2009).
For CBS Only
11. Meets 2013 consensus criteria for possible or probable corticobasal degeneration, CBS subtype (Armstrong et al. 2013).

Who cannot join

Meets National Institute on Aging-Alzheimer's Association Workgroups criteria for probable AD (McKhann et al. 2011);
Any medical condition other than CBS or PSP that could account for cognitive deficits (e.g., active seizure disorder, stroke, vascular dementia);
A prominent and sustained response to levodopa therapy;
History of significant cardiovascular, hematologic, renal, or hepatic disease (or laboratory evidence thereof);
History of significant peripheral neuropathy;
History of major psychiatric illness or untreated depression;

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Neutrophil count <1,500/mm3, platelets <100,000/mm3, serum creatinine >1.5 x upper limit of normal (ULN), total bilirubin >1.5 x ULN, alanine aminotransferase (ALT) >3 x ULN, aspartate aminotransferase (AST) >3 x ULN, or INR >1.2 at Screening evaluations;
Evidence of any clinically significant findings on Screening or baseline evaluations which, in the opinion of the Investigator would pose a safety risk or interfere with appropriate interpretation of study data;
Current or recent history (within four weeks prior to Screening) of a clinically significant bacterial, fungal, or mycobacterial infection;
Current clinically significant viral infection;
Major surgery within four weeks prior to Screening;
Unable to tolerate MRI scan at Screening;
Any contraindication to or unable to tolerate lumbar puncture at Screening, including use of anti-coagulant medications such as warfarin. Daily administration of 81 mg aspirin will be allowed as long as the dose is stable for 30 days prior to Screening;
Subjects who, in the opinion of the Investigator, are unable or unlikely to comply with the dosing schedule or study evaluations;
Previous exposure to microtubule inhibitors (including TPI 287) within 5 years of Screening. Treatment with microtubule inhibitors other than TPI 287 while on study will not be allowed;
Participation in another interventional clinical trial within 3 months of Screening;
Treatment with another investigational drug within 30 days of Screening. Treatment with investigational drugs other than TPI 287 while on study will not be allowed;
Known hypersensitivity to the inactive ingredients in the study drug;
Pregnant or lactating;
Positive pregnancy test at Screening or Baseline (Day 1);
Cancer within 5 years of Screening, except for non-metastatic skin cancer or non-metastatic prostate cancer not expected to cause significant morbidity or mortality within one year of baseline.
For CBS Only:
History or evidence at Screening of cortical amyloid levels on 18F florbetapir PET scans consistent with underlying AD;
History of serum or plasma progranulin level less than one standard deviation below the normal subject mean for the laboratory performing the assay;
History or evidence at Screening of known disease-associated mutations in GRN or C9ORF72 genes to rule out CBS due to TDP-43 pathology;
History of known disease-associated mutations in ribosomal protein L3 [TDP- 43 gene (TARBP)], chromatin modifying protein 2B (CHMPB2) or valosin containing protein (VCP) genes or any other frontotemporal lobar degeneration (FTLD) causative genes discovered during the course of the trial and not associated with underlying tau pathology.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

University of Alabama
Birmingham, Alabama, 35294
University of California, San Francisco
San Francisco, California, 94158

Other trials in United States

About this study

Official title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Sequential Cohort, Dose-Ranging Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of TPI 287 in Patients With Primary Four Repeat Tauopathies: Corticobasal Syndrome or Progressive Supranuclear Palsy

Read the detailed description

The maximum tolerated dose of TPI-287 will be determined through a planned dose escalation over 3 sequential cohorts, each comprising of 11 participants randomized to either TPI-287 or placebo. TPI-287 or placebo will be administered as an intravenous infusion once every 3 weeks for 9 weeks, for a total of 4 infusions. Participants who successfully complete this phase will have the option of entering into the open label extension phase during which TPI-287 will be administered once every 3 weeks for an additional 6 weeks, for a total of 3 extra infusions.

The dose of TPI 287 will be escalated in sequential cohorts. In the low dose cohort 11 subjects each diagnosis (i.e., separate dose escalations will be performed for CBS and PSP) will be enrolled. The medium and high dose cohorts will be comprised of 11 subjects; combined CBS and PSPdiagnoses. Subjects will be assigned to cohorts in the order of study entry.

Pre-medications of diphenhyramine 25 mg (Benadryl), dexamethasone 10 mg, and famotidine 20 mg (or the H2 blocker ranitidine 50 mg) will be given IV within 60 minutes prior to each study infusion.

Safety and tolerability will be assessed through reporting of adverse events, physical and neurological testing, ECGs, as well as blood and urine analyses. Baseline and end-point measures of cognition and function, MRI brain scans, and cerebrospinal fluid (CSF) biomarker analyses will be used to determine preliminary efficacy of TPI-287 in mild-moderate AD. Pharmacokinetic and pharmacodynamic properties of TPI-287 will be calculated from blood plasma collected after the first infusion, and from CSF collected on the last visit of the placebo-controlled phase.

Conditions: Primary Four Repeat Tauopathies (4RT), Corticobasal Syndrome (CBS), Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD)
Being tested: TPI 287 2 mg/m2, TPI-287 20 mg/m2, Placebo, TPI-287 6.3 mg/m2
Keywords: 4RT, CBS, PSP, CBD, TPI-287
Medical subject headings: Corticobasal Degeneration, Supranuclear Palsy, Progressive, Color Vision Defects

What participants receive

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

TPI 287 2 mg/m2Drug
2 mg/m2 of TPI-287 diluted with 500mL 0.9% sodium chloride. TPI-287 is a microtubule inhibitor belonging to the taxane diterpenoid (taxoid) family, and specifically to the abeotaxane class.Drug: Placebo Drug: Placebo 500mL 0.9% sodium chloride.
TPI-287 20 mg/m2Drug
20 mg/m2 of TPI-287 diluted with 500mL 0.9% sodium chloride. TPI-287 is a microtubule inhibitor belonging to the taxane diterpenoid (taxoid) family, and specifically to the abeotaxane class
PlaceboDrug
500mL 0.9% sodium chloride
TPI-287 6.3 mg/m2Drug
6.3 mg/m2 of TPI-287 diluted with 500mL 0.9% sodium chloride. TPI-287 is a microtubule inhibitor belonging to the taxane diterpenoid (taxoid) family, and specifically to the abeotaxane class

Study groups in NCT02133846
Study group	Role	What they receive
TPI-287 low dose	Experimental	2 mg/m2 of TPI-287 administered as a 1-hour intravenous infusion once every 3 weeks for 9 weeks (for a total of 4 infusions) Receives: Drug: TPI 287 2 mg/m2; Drug: Placebo
TPI-287 moderate dose	Experimental	6.3 mg/m2 of TPI-287 administered as a 1-hour intravenous infusion once every 3 weeks for 9 weeks (for a total of 4 infusions). Receives: Drug: Placebo; Drug: TPI-287 6.3 mg/m2
TPI-287 high dose	Experimental	20 mg/m2 of TPI-287 administered as a 1-hour intravenous infusion once every 3 weeks for 9 weeks (for a total of 4 infusions) Receives: Drug: TPI-287 20 mg/m2; Drug: Placebo
Placebo	Placebo comparator	0.9% sodium chloride as a 1-hour intravenous infusion once every 3 weeks for 9 weeks (for a total of 4 infusions) Receives: Drug: Placebo

What is being measured

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

Maximum tolerated dose of TPI-287 in patients with primary 4RT; CBS or PSP.
To determine the safety and tolerability [maximum tolerated dose (MTD) within planned dosing range] of intravenous (IV) infusions of TPI 287 administered once every 3 weeks for 9 weeks (for a total of 4 infusions) in patients with primary four repeat tauopathies (4RT): corticobasal syndrome (CBS) or progressive supranuclear palsy (PSP).
Measured: 21 months (first subject enrolled to last subject completed)

Secondary outcomes (1)

TPI-287 levels in blood plasma and cerebrospinal fluid
To determine the pharmacokinetic (PK) profile of TPI 287 in plasma after a single IV infusion of TPI 287 and the steady-state cerebrospinal (CSF) concentration of TPI 287 1 week after completion of the fourth infusion.
Measured: 21 months (first subject enrolled to last subject completed)

Timeline

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

May 2014Sep 2019

First posted8 May 2014 (estimated)
Study startMay 2014
Primary completionSep 2019
Study completionSep 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (6)Papers the sponsor has linked to this study.
Other trials for Primary Four Repeat Tauopathies (4RT)Studies 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: 44 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02133846
Sponsor's protocol ID: TPI287-4RT-001
Acronym: TPI-287-4RT
Status last verified: Apr 2020
First submitted: 6 May 2014
Last updated: 15 Apr 2020
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Armstrong MJ, Litvan I, Lang AE, Bak TH, Bhatia KP, Borroni B, Boxer AL, Dickson DW, Grossman M, Hallett M, Josephs KA, Kertesz A, Lee SE, Miller BL, Reich SG, Riley DE, Tolosa E, Troster AI, Vidailhet M, Weiner WJ. Criteria for the diagnosis of corticobasal degeneration. Neurology. 2013 Jan 29;80(5):496-503. doi: 10.1212/WNL.0b013e31827f0fd1.
PubMed 23359374
Litvan I, Agid Y, Calne D, Campbell G, Dubois B, Duvoisin RC, Goetz CG, Golbe LI, Grafman J, Growdon JH, Hallett M, Jankovic J, Quinn NP, Tolosa E, Zee DS. Clinical research criteria for the diagnosis of progressive supranuclear palsy (Steele-Richardson-Olszewski syndrome): report of the NINDS-SPSP international workshop. Neurology. 1996 Jul;47(1):1-9. doi: 10.1212/wnl.47.1.1.
PubMed 8710059
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
McKhann GM, Knopman DS, Chertkow H, Hyman BT, Jack CR Jr, Kawas CH, Klunk WE, Koroshetz WJ, Manly JJ, Mayeux R, Mohs RC, Morris JC, Rossor MN, Scheltens P, Carrillo MC, Thies B, Weintraub S, Phelps CH. The diagnosis of dementia due to Alzheimer's disease: recommendations from the National Institute on Aging-Alzheimer's Association workgroups on diagnostic guidelines for Alzheimer's disease. Alzheimers Dement. 2011 May;7(3):263-9. doi: 10.1016/j.jalz.2011.03.005. Epub 2011 Apr 21.
PubMed 21514250
Brunden KR, Trojanowski JQ, Smith AB 3rd, Lee VM, Ballatore C. Microtubule-stabilizing agents as potential therapeutics for neurodegenerative disease. Bioorg Med Chem. 2014 Sep 15;22(18):5040-9. doi: 10.1016/j.bmc.2013.12.046. Epub 2013 Dec 30.
PubMed 24433963
Tsai RM, Miller Z, Koestler M, Rojas JC, Ljubenkov PA, Rosen HJ, Rabinovici GD, Fagan AM, Cobigo Y, Brown JA, Jung JI, Hare E, Geldmacher DS, Natelson-Love M, McKinley EC, Luong PN, Chuu EL, Powers R, Mumford P, Wolf A, Wang P, Shamloo M, Miller BL, Roberson ED, Boxer AL. Reactions to Multiple Ascending Doses of the Microtubule Stabilizer TPI-287 in Patients With Alzheimer Disease, Progressive Supranuclear Palsy, and Corticobasal Syndrome: A Randomized Clinical Trial. JAMA Neurol. 2020 Feb 1;77(2):215-224. doi: 10.1001/jamaneurol.2019.3812.
PubMed 31710340

Study at a glance

From the registry record, last updated by the sponsor on 15 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Sequential Cohort, Dose-Ranging Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of TPI 287 in Patients With Primary Four Repeat Tauopathies: Corticobasal Syndrome or Progressive Supranuclear Palsy
ClinicalTrials.gov ID: NCT02133846
Acronym / sponsor ID: TPI-287-4RT / TPI287-4RT-001
Conditions: Primary Four Repeat Tauopathies (4RT), Corticobasal Syndrome (CBS), Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD)
Keywords: 4RT, CBS, PSP, CBD, TPI-287
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 44 participants (actual)
Lead sponsor: University of California, San FranciscoUniversity, hospital or other
Collaborators: CBD Solutions, Tau Consortium
Study start: May 2014
Primary completion: Sep 2019 (actual)
Study completion: Sep 2019 (actual)
First posted: 8 May 2014 (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.

Detailed description

Pre-medications of diphenhyramine 25 mg (Benadryl), dexamethasone 10 mg, and famotidine 20 mg (or the H2 blocker ranitidine 50 mg) will be given IV within 60 minutes prior to each study infusion.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 4

Arm	Type	Receives
TPI-287 low dose	Experimental	Drug: TPI 287 2 mg/m2, Drug: Placebo2 mg/m2 of TPI-287 administered as a 1-hour intravenous infusion once every 3 weeks for 9 weeks (for a total of 4 infusions)
TPI-287 moderate dose	Experimental	Drug: Placebo, Drug: TPI-287 6.3 mg/m26.3 mg/m2 of TPI-287 administered as a 1-hour intravenous infusion once every 3 weeks for 9 weeks (for a total of 4 infusions).
TPI-287 high dose	Experimental	Drug: TPI-287 20 mg/m2, Drug: Placebo20 mg/m2 of TPI-287 administered as a 1-hour intravenous infusion once every 3 weeks for 9 weeks (for a total of 4 infusions)
Placebo	Placebo comparator	Drug: Placebo0.9% sodium chloride as a 1-hour intravenous infusion once every 3 weeks for 9 weeks (for a total of 4 infusions)

Who may qualify

Ages: 50 to 85 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (16 listed)

Between 50 and 85 years of age (inclusive);
Able to walk 5 steps with minimal assistance (stabilization of one arm or use of cane/walker);
MRI at Screening is consistent with CBS or PSP (≤ 4 microhemorrhages, and no large strokes or severe white matter disease);
MMSE at Screening is between 14 and 30 (inclusive);
FDA-approved AD medications are sometimes prescribed for CBS and PSP subjects, and are allowed as long as the dose is stable for 2 months prior to Screening. Other medications (except those listed under exclusion criteria) are allowed as long as the dose is stable for 30 days prior to Screening;

Key exclusion criteria (26 listed)

Meets National Institute on Aging-Alzheimer's Association Workgroups criteria for probable AD (McKhann et al. 2011);
Any medical condition other than CBS or PSP that could account for cognitive deficits (e.g., active seizure disorder, stroke, vascular dementia);
A prominent and sustained response to levodopa therapy;
History of significant cardiovascular, hematologic, renal, or hepatic disease (or laboratory evidence thereof);
History of significant peripheral neuropathy;

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
TPI 287 2 mg/m22 mg/m2 of TPI-287 diluted with 500mL 0.9% sodium chloride. TPI-287 is a microtubule inhibitor belonging to the taxane diterpenoid (taxoid) family, and specifically to the abeotaxane class.Drug: Placebo Drug: Placebo 500mL 0.9% sodium chloride.	Drug	TPI-287 low dose
TPI-287 20 mg/m220 mg/m2 of TPI-287 diluted with 500mL 0.9% sodium chloride. TPI-287 is a microtubule inhibitor belonging to the taxane diterpenoid (taxoid) family, and specifically to the abeotaxane class	Drug	TPI-287 high dose
Placebo500mL 0.9% sodium chloride	Drug	Placebo, TPI-287 high dose, TPI-287 low dose, TPI-287 moderate dose
TPI-287 6.3 mg/m26.3 mg/m2 of TPI-287 diluted with 500mL 0.9% sodium chloride. TPI-287 is a microtubule inhibitor belonging to the taxane diterpenoid (taxoid) family, and specifically to the abeotaxane class	Drug	TPI-287 moderate dose

Primary outcome

Maximum tolerated dose of TPI-287 in patients with primary 4RT; CBS or PSP.
Time frame: 21 months (first subject enrolled to last subject completed)
To determine the safety and tolerability [maximum tolerated dose (MTD) within planned dosing range] of intravenous (IV) infusions of TPI 287 administered once every 3 weeks for 9 weeks (for a total of 4 infusions) in patients with primary four repeat tauopathies (4RT): corticobasal syndrome (CBS) or progressive supranuclear palsy (PSP).

1 secondary outcome

TPI-287 levels in blood plasma and cerebrospinal fluid
Time frame: 21 months (first subject enrolled to last subject completed)

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 15 Apr 2020.
Background: PubMed 23359374Armstrong MJ, Litvan I, Lang AE, Bak TH, Bhatia KP, Borroni B, Boxer AL, Dickson DW, Grossman M, Hallett M, Josephs KA, Kertesz A, Lee SE, Miller BL, Reich SG, Riley DE, Tolosa E, Troster AI, Vidailhet M, Weiner WJ. Criteria for the diagnosis of corticobasal degeneration. Neurology. 2013 Jan 29;80(5):496-503. doi: 10.1212/WNL.0b013e31827f0fd1.
Background: PubMed 8710059Litvan I, Agid Y, Calne D, Campbell G, Dubois B, Duvoisin RC, Goetz CG, Golbe LI, Grafman J, Growdon JH, Hallett M, Jankovic J, Quinn NP, Tolosa E, Zee DS. Clinical research criteria for the diagnosis of progressive supranuclear palsy (Steele-Richardson-Olszewski syndrome): report of the NINDS-SPSP international workshop. Neurology. 1996 Jul;47(1):1-9. doi: 10.1212/wnl.47.1.1.
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 21514250McKhann GM, Knopman DS, Chertkow H, Hyman BT, Jack CR Jr, Kawas CH, Klunk WE, Koroshetz WJ, Manly JJ, Mayeux R, Mohs RC, Morris JC, Rossor MN, Scheltens P, Carrillo MC, Thies B, Weintraub S, Phelps CH. The diagnosis of dementia due to Alzheimer's disease: recommendations from the National Institute on Aging-Alzheimer's Association workgroups on diagnostic guidelines for Alzheimer's disease. Alzheimers Dement. 2011 May;7(3):263-9. doi: 10.1016/j.jalz.2011.03.005. Epub 2011 Apr 21.
Background: PubMed 24433963Brunden KR, Trojanowski JQ, Smith AB 3rd, Lee VM, Ballatore C. Microtubule-stabilizing agents as potential therapeutics for neurodegenerative disease. Bioorg Med Chem. 2014 Sep 15;22(18):5040-9. doi: 10.1016/j.bmc.2013.12.046. Epub 2013 Dec 30.
Derived from this study: PubMed 31710340Tsai RM, Miller Z, Koestler M, Rojas JC, Ljubenkov PA, Rosen HJ, Rabinovici GD, Fagan AM, Cobigo Y, Brown JA, Jung JI, Hare E, Geldmacher DS, Natelson-Love M, McKinley EC, Luong PN, Chuu EL, Powers R, Mumford P, Wolf A, Wang P, Shamloo M, Miller BL, Roberson ED, Boxer AL. Reactions to Multiple Ascending Doses of the Microtubule Stabilizer TPI-287 in Patients With Alzheimer Disease, Progressive Supranuclear Palsy, and Corticobasal Syndrome: A Randomized Clinical Trial. JAMA Neurol. 2020 Feb 1;77(2):215-224. doi: 10.1001/jamaneurol.2019.3812.

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 Boxer, MD, PhD principal investigator

UCSF Memory and Aging Center

2 sites 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 16 inclusion and 26 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 15 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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?

Maximum tolerated dose of TPI-287 in patients with primary 4RT; CBS or PSP. (time frame: 21 months (first subject enrolled to last subject completed)). 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 NCT02133846, which also lists 6 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 15 Apr 2020.