World Health Organization site
Skip Navigation Links

Main
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 30 June 2019
Main ID:  EUCTR2018-001887-46-GB
Date of registration: 19/06/2019
Prospective Registration: No
Primary sponsor: Sheffield Teaching Hospitals NHS Foundation Trust
Public title: Trial of Ursodeoxycholic Acid (UDCA) for PD. The 'UP-study'
Scientific title: A Phase II, Placebo Controlled, Double Blind, Randomised Clinical Trial to assess the safety and tolerability Of 30mg/kg daily Ursodeoxycholic Acid (UDCA) in Patients with Parkinson’s Disease (PD) - Trial of Ursodeoxycholic Acid (UDCA) for PD. The 'UP-study'
Date of first enrolment: 08/11/2018
Target sample size: 30
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-001887-46
Study type:  Interventional clinical trial of medicinal product
Study design: 
Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: yes
Parallel group: yes
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: no
Number of treatment arms in the trial: 2
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no
Countries of recruitment
United Kingdom
Contacts
Name: Sarah Moll   
Address:  D54b D Floor, Biomedical Research Centre Office, Royal Hallamshire Hospital S10 2JF Sheffield United Kingdom
Telephone: 01142712563
Email: sarah.moll@sth.nhs.uk
Affiliation:  Sheffield Teaching Hospitals NHS Foundation Trust
Name: Sarah Moll   
Address:  D54b D Floor, Biomedical Research Centre Office, Royal Hallamshire Hospital S10 2JF Sheffield United Kingdom
Telephone: 01142712563
Email: sarah.moll@sth.nhs.uk
Affiliation:  Sheffield Teaching Hospitals NHS Foundation Trust
Key inclusion & exclusion criteria
Inclusion criteria:
Diagnosis of Parkinson’s disease = 3 years ago by a clinician with particular expertise in the diagnosis and treatment of movement disorders
• Subjective improvement of motor impairment on dopaminergic medication, confirmed by PI through personal examination and/or review of medical records
• Hoehn and Yahr stage = 2.5 in the practically defined “ON” medication state. This implies that all patients will be mobile without assistance during their best “ON” medication periods.
• Ability to take the study drug
• Ability to communicate in English
• Age 18-75 yr of any gender
• Documented informed consent to participate.
• Able to comply with study protocol and willing to attend necessary study visits

Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 25
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion criteria:
? Diagnosis or suspicion of other cause of parkinsonism. Patients with clinical features indicating a diagnosis of progressive supranuclear palsy (PSP), multiple systems atrophy (MSA), drug induced-parkinsonism, dystonic tremor or essential tremor will not be recruited.
? Known abnormality on CT or MRI brain imaging considered likely to compromise compliance with trial protocol/31P-MRS acquisition
? Known claustrophobia or other reasons why patient could not tolerate or be suitable for
31P-MRS
? Current or previous exposure to UDCA
? Current or previous diagnosis of liver disease, in particular PBC judged to be significant by the clinical investigator.
? Prior intracerebral surgical intervention for PD (including deep-brain stimulation). Patients who have previously undergone deep brain stimulation, intracerebral administration of growth factors, gene therapies or cell therapies will not be eligible.
? Already actively participating in a trial of a device, drug or surgical treatment for PD
? History of alcoholism
? Women of child-bearing potential (WOCBP)
? Participants who lack the capacity to give informed consent
? Any medical or psychiatric condition which in the investigator’s opinion compromises the potential participant’s ability to participate
? Concurrent dementia defined by a score lower than 25 on the Montreal Cognitive assessment (MoCA). Dementia may affect the ability of potential research participants to give informed consent or follow the study protocol.
? Concurrent severe depression defined by a score > 16 on the Montgomery-Asberg Depression Rating Scale (MADRS)
? Serum transaminases more than 2 times upper
limit of normal
? PD patients who are on ciclosporin, nitrendipine or dapsone for the treatment of concomitant, general medical conditions


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Parkinson’s disease (PD)
MedDRA version: 20.0 Level: PT Classification code 10061536 Term: Parkinson's disease System Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Intervention(s)

Trade Name: Ursonorm
Product Name: Ursonorm
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Ursodeoxycholic acid
CAS Number: 128-13-2
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 30-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: The primary objective is to compare the safety and tolerability of UDCA at 30 mg/kg in PD compared to placebo as indicated by:

• Number of serious adverse events (SAEs)

• Number of adverse Treatment-reactions

• Number of patients completing the study

Primary end point(s): The safety and tolerability of UDCA in Parkinson’s disease (PD) at a dose of 30 mg/kg

Metric:
• Number of serious adverse events (SAE)

• Number of adverse treatment reactions

• Number of patients completing the study



Secondary Objective: The secondary objectives are to assess the effect of UDCA compared with Placebo on disease progression in PD at 48 weeks (assessed as a change from baseline) by:

Clinical assessment (using the Movement Disorders Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) part 3 motor subscale) in the "OFF" medication state.

In vivo parameter estimates of high and low energy metabolite levels (ATP, PCr, Pi) , derived from cranial 31P MRS centred on the basal ganglia and related motor regions (using 31P MRS at 48 weeks)

Sensor-based, objective quantification of motor impairment, using the Optogait and Opals systems (Sheffield patients only) as well as the Dynaport Move monitor (all patients)

Timepoint(s) of evaluation of this end point: Each of the metrics below will be assessed over the whole study period (start of treatment to week 56) and summarised by treatment group.
Secondary Outcome(s)

Secondary end point(s): • Movement Disorders Society Unified Parkinson’s Disease Rating Scale (MDS-
UPDRS) part 3 motor subsection Off medication score

• In vivo parameter estimates of high and low energy metabolite levels (ATP, PCr, Pi) , derived from cranial 31P-MRS centred on the basal ganglia and related motor regions

• Objective quantification of spatio-temporal gait parameters (optogait 5m), postural stability (Opals) and continuous activity monitory (McRoberts)

Timepoint(s) of evaluation of this end point: Baseline, week 48

For each of the secondary outcomes the change from baseline will be summarised within treatment groups using standard summary statistics (n, mean, standard deviation, median, minimum and maximum). The change from baseline will then be compared between treatment groups using a t-test. If there is an imbalance in baseline characteristics between randomised groups further analysis will be explored within the constraints of the small sample size, this may include an analysis of covariance. Scores will be suitably transformed if necessary. If the distributional assumptions for a t-test are not fulfilled an alternative analysis will be performed.
Secondary ID(s)
STH18493
Source(s) of Monetary Support
PRO.MED.CS Praha
The J P Moulton Charitable Foundation
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date:
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history