PSP Trials

[Faiza]

I found out a couple of days ago that my dad's local hospital is running PSP trials and they are looking for participants. I've emailed the hospital and they are looking into dad's notes to see if he's suitable. I'm quite nervous at the moment as I hope they get to include him. I keep reading the exclusion/inclusion criteria over and over again! But I thinking they won't take him. I think it's my paranoia! Hopefully will find out soon!

[LindanTerry]

Good luck to you and your Faiza.I do hope he is chosen x

[ray-wiffen-1958]

good luck faiza with the psp trials hope your dad

is picked to take part which is your local hospital

that is running these trials,

[Faiza]

Thank you both.

The trials are being carried out at Princess Royal Hospital, Haywards Heath. I've got the email address and telephone number of the relevant person responsible for recruitment if people want me to pass the information on. I also have the inclusion and exclusion criteria for the study which I can send.

I'm just so nervous he wont't get picked. I'll keep you all posted as to the outcome.

[BLANCHE in reply to Faiza]

I was interested to hear about the trials. Could you pls let me have the contact details too. Blanche

[Faiza]

Hi Blanche,

Here's all the details:

Name: Dee Mullan, Neurosciences Research Nurse

Address: Neurological Office, Downsmere Building, Princess Royal Hospital, Lewes Road, Haywards Heath, West Sussex, RH16 4EX

Email: [e-mail]

Tel: [phone] extension 5426 (Monday to Thursday)

Fax: [phone].

I have the inclusion/exclusion criteria too. I'll cut and paste it into a new blog post below

[Faiza]

6.1 Inclusion Criteria

Subjects may be included in the study only if they meet all of the following criteria:

1. Probable or possible PSP defined as:

1) at least a 12-month history of

a) postural instability or falls during the first 3 years that symptoms are present;

and

b) 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.

2) age at symptom onset of 40 to 85 years by history; and

3) an akinetic-rigid syndrome with prominent axial rigidity.

2. Aged 41 to 85 years at the time of screening.

3. Judged by investigator to be able to comply with neuropsychological evaluation at

baseline and throughout the study.

4. 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.

5. 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.

6. Score = 15 on the mini-mental state examination (MMSE) at screening (Visit 1).

7. 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.

8. 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.

9. If the subject is receiving levodopa/carbidopa, levodopa/benserazide, a dopamine

agonist, catechol-o-methyltransferase (COMT) inhibitor, or other Parkinson’s

medication, the dose must have been stable for at least 90 days prior to the screening

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visit (Visit 1) and must remain stable for the duration of the study. No such

medication can be initiated during the study.

10. Able to tolerate the MRI scan during screening without the use of sedation.

11. 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.

12. Presence of symptoms for less than 5 years or the presence of symptoms for more

than 5 years with a PSPRS baseline score = 40.

13. Stable on all other chronic medications for at least 30 days prior to the screening visit

(Visit 1).

6.2 Exclusion Criteria

Subjects will be excluded from the study for any of the following reasons:

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:

a. Abrupt onset of symptoms defined in inclusion criteria 1 associated with ictal

events,

b. Head trauma related to onset of symptoms defined in inclusion criteria 1,

c. Severe amnesia within 6 months of the symptoms defined in inclusion criteria 1,

d. Cerebellar ataxia,

e. Choreoathetosis,

f. Early, symptomatic autonomic dysfunction; or

g. Tremor while at rest.

4. 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 spaceoccupying

lesion; or history of stroke or head injury with loss of consciousness for at

least 15 minutes within the past 20 years.

5. 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 (other

than temazepam or zolpidem).

6. Treatment with lithium, methylene blue, tramiprosate, ketone bodies, latrepirdine, 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.

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8. 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.

9. 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.

10. The systolic blood pressure measurement is > 190 or < 85 mm Hg. The diastolic

blood pressure measurement is > 105 or < 50 mm Hg at screening.

11. Abnormal ECG tracing at screening and judged to be clinically significant by the site

investigator.

12. Treatment with any investigational drugs or device within 90 days of screening.

13. Known history of serum or plasma progranulin level less than one standard deviation

below the normal subject mean for the laboratory performing the assay.

14. 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).

15. History of deep brain stimulator (DBS) surgery other than sham surgery for DBS

clinical trial.

16. History of early, prominent rapid eye movement (REM) sleep behavior disorder.

17. Women who are pregnant or lactating and women of childbearing potential who are

not using at least two different forms of medically recognized and highly effective

methods of birth control, resulting in a low failure rate when used consistently and

correctly such as implants, injectables, combined oral contraceptives, some IUDs,

sexual abstinence or vasectomised partner.

18. An employee or relative of an employee of the Sponsor, a clinical site, or CRO

participating in the study.

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 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 (e.g., severe claustrophobia,

ferromagnetic metal in body).

22. Structural abnormality on MRI that precludes diagnosis of PSP, such as cortical

infarct in brain region that might account for subject’s symptoms.

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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 with normal functioning following overnight withdrawal of the anti-

Parkinson medication.

24. Known hypersensitivity to davunetide or any ingredient of the formulation.

[Fiona_McL]

Interesting information Faiza. Thanks for sharing that

[ray-wiffen-1958]

thanks for the information faiza

seems very much over the top.

[jillannf6]

hi faiza

i could not be accepted for the davunetide trial as i already have to use a nasal spray for allergic rhinitis

go good luck and go for it wiht your dad

jill x

[jillannf6]

hi faiza and all

SALFORD are also runnnig the trials if anyone in the manchewster area is intterested

jillx

[DianneH]

How did it go? Has anyone had any results or improvements? How encouraging that there are trials going on.