The objective of the study is to realize an efficient allocation of resources to the available treatment options in advanced PD, while guaranteeing the highest standard of care.Secondary objectives are 1) to quantify the need for and cost of…
ID
Source
Brief title
Condition
- Movement disorders (incl parkinsonism)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary health economic outcomes of the randomized trial are the costs per
unit on the PDQ-39 and the costs per QALY for the cost-effectiveness and
cost-utility analyses respectively.
Secondary outcome
Secondary clinical outcomes are quality of life (PDQ-39), PD motor symptoms
(UPDRS), dyskinesias (CDRS), 3-day motor symptom diary), adverse effects and
complications, treatment failure, non-motor symptoms such as autonomic
functions and sleep (Non Motor Symptom Checklist, Rotterdam Symptom Checklist),
PD-medication, disability, functional health status (ALDS), patient and
physician preferences, patient satisfaction, caregiver burden,
neuropsychological and psychiatric assessment, stopping treatment, starting
with the alternative than initially started treatment, and medical and
non-medical care costs (iMCQ, iPCQ).
Background summary
Both Continuous intrajejunal Levodopa Infusion (CLI) and Deep Brain Stimulation
(DBS) are accepted therapies for the treatment of advanced Parkinson*s disease
(PD). Neurologists and patients tend to prefer the more expensive CLI although
a scientific rationale is lacking. To determine the optimal treatment in
advanced PD, a comparative study of CLI and DBS is warranted.
Study objective
The objective of the study is to realize an efficient allocation of resources
to the available treatment options in advanced PD, while guaranteeing the
highest standard of care.
Secondary objectives are
1) to quantify the need for and cost of healthcare and the health benefits in
both groups
2) to assess the patients motor and non-motor symptoms, quality of life and
daily functioning in both DBS and CLI
3) to register and compare adverse effects and complications in both groups and
verify patients* and treating physicians* conceptions about the different
treatments
Study design
Prospective, randomized, open label multicenter trial, with two additional
patient preference treatment arms (*patient preference randomized trial*).
Intervention
Patients will be randomized to CLI or DBS. For CLI treatment, a tube will be
placed in the jejunum via a percutaneous endoscopic gastrostomy (PEG). This
tube is connected to an external pump that delivers the levodopa-gel. For DBS
treatment, 2 electrodes will be implanted in the brain. The electrodes are
connected to an implanted pulse generator, which will be placed subcutaneously
in the subclavian area.
Study burden and risks
The study investigates the cost-effectiveness and cost-utility comparing CLI
and DBS. Both treatments are currently available for advanced PD and both have
a small risk of severe side effects. The surplus in burden of study
participation compared to the regular treatment consists of both more and more
detailed assessment procedures. The additional time for these extra procedures
* consisting of questionnaires and motor symptom assessments * is approximately
15 hours, including time to travel. Besides this small burden the study has no
additional risk compared to standard practice, which constitutes a negligible
risk according to the NFU (Nederlandse Federatie van Universitaire Medische
Centra) criteria for human research.
Meibergdreef 9
Amsterdam 1105AZ
NL
Meibergdreef 9
Amsterdam 1105AZ
NL
Listed location countries
Age
Inclusion criteria
- idiopathic Parkinson's Disease with bradykinesia and at least two of the
following signs; resting tremor, rigidity, and asymmetry;
- despite optimal pharmacological treatment, at least one of the following
symptoms: severe response fluctuations, dyskinesias, painful dystonia or
bradykinesia;
-a life expectancy of at least two years.
Exclusion criteria
-age below 18 years
- previous PD-neurosurgery (e.g., DBS, pallidotomy, thalamotomy);
- previous CLI (through a PEG-tube or Nasal Jejuna| tube);
- Hoehn and Yahr stage 5 at the best moment during the day;
- dementia or indication for severe cognitive impairment, such as PD-CRS <65;
- psychosis;
- current depression;
- other, severely disabling condition;
- contraindications for DBS surgery, such as a physical disorder making surgery
hazardous;
- contraindications for PEG surgery such as interposed organs, ascites and
oesophagogastric varices, or for Duodopa;
- pregnancy, breastfeeding, and women of child bearing age not using a reliable
method of contraception;
- No informed consent;
- legally incompetent adults.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2014.001501.32-NL |
| ClinicalTrials.gov | NCT02480803 |
| CCMO | NL51240.018.14 |
| OMON | NL-OMON21505 |