Multiple System Atrophy Clinical Trial
Official title:
Phase 3 Study of Riluzole in Multiple System Atrophy (MSA) and Progressive Supranuclear Palsy (PSP) (Parkinson's Plus Syndromes)
NNIPPS is a clinical trial of riluzole (a drug previously shown to slow down the rate of progression og amyotrophic lateral sclerosis-ALS; Lou Gehrig's disease) involving nearly 800 people diagnosed with the 'parkinson plus' syndromes of multiple system atrophy (MSA) and progressive supranuclear plasy (PSP). In addition to showing whether riluzole is helpful in MSA and PSP, NNIPPS will improve criteria for making an accurate and early diagnosis, for assessing the rate of progression, and will advance understanding of the biology of these disabling and progressive neurodegenerative diseases.
Multiple System Atrophy (MSA) and Progressive Supranuclear Palsy (PSP) often present as
akinetic-rigid syndromes and in the early stages are difficult to differentiate in the
clinic. Current Consensus Diagnostic Criteria based on retrospective studies have high
specificity but low sensitivity. The NNIPPS study is an EU-funded multinational (France, UK,
Germany) multi-centre academic-led project with four main aims. The first aim is to test the
hypothesis that riluzole, which may have generic neuroprotective properties, reduces the
risk of death and improves function and quality of life (QL) in patients with MSA and PSP-
‘parkinson’s plus syndromes’. The second aim is to identify prognostic factors for survival
and functional deterioration, and to develop and validate functional rating scales
prospectively. The third aim is to investigate MRI, cognitive, pathological and genetic
aspects of these disorders in relation to disease progression and pathogenesis. The fourth
aim is to understand the impact of these diseases on the QL of patients and carers and to
identify the health costs of treatment.
The study is designed as a randomised, stratified, controlled trial of the efficacy and
safety of riluzole (up to 200mg daily) versus placebo in MSA and PSP. The primary outcome
measure is survival at 36 months. Power calculations suggested that we would need to recruit
~400 patients into each stratum (MSA, PSP) in order to detect a reduction in the relative
risk (RR) of death at 36 months with 80% power and two-sided a=0.05. Using modified
consensus criteria (to provide greater sensitivity) we recruited 766 patients (363 PSP, 404
MSA) over 2 years (1999-2001). The first patients recruited are about to enter the
open-label study. The final analysis of the primary efficacy measure is planned for December
2005. Secondary outcome measures include safety, rate of change in UPDRS and other rating
scales including a parkinson’s plus symtoms rating scale (PPSS), changes in cognitive
function assessed using the Mattis Dementia Rating Scale, the Frontal Assessment Battery,
The Bushke Selective Reminding Test, The Neuropsychiatric Inventory, and other tests of
memory and executive function. QL and Health economic data is collected using the SF36 and a
Client Service Receipt Inventory (CSRI). Assessments are made at 6 monthly intervals.
Standardised MRI has been acquired in ~70% of cases at entry and will be repeated at 36
months where possible. DNA has been collected from ~75% of cases. 100 brains have been
donated and are being analysed using a standardised protocol.
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment
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