6 Month Phase II Study With PBT2 in Huntington Patients
MELBOURNE, AUSTRALIA–(Marketwire – Dec 19, 2012) – Prana Biotechnology (NASDAQ: PRAN) (ASX:PBT) today announced that it has completed recruitment in the Reach2HD Phase IIa clinical trial using PBT2 in patients with Huntington Disease (HD).
The 6 month double-blind, placebo-controlled Phase II trial in patients with early to mid-stage Huntington Disease met its enrolment target of 100 patients ahead of schedule. Additional patients may be included in the trial, subject to final procedures this week.
The Principal Investigator on the study, Dr. Ray Dorsey of Johns Hopkins University Medical Center, commented that “interest in PBT2 is high in part because PBT2 offers a novel mechanistic approach to the treatment of HD. We thank the clinical sites and the Huntington community for their wonderful support.” The Reach2HD trial was coordinated in conjunction with the Huntington Study Group (HSG) across 20 clinical sites in the USA and Australia.
Huntington Disease is a complex and severely debilitating genetic, neurodegenerative disease, for which there is no cure. The disease often affects young adults and, whilst associated with severe physical movement symptoms, progressively impacts the mind and emotions as well. The disease causes incapacitation and death about 15-25 years after onset. At this time there is only one marketed drug for the alleviation of some of the involuntary motor symptoms associated with the disease.
Professor Ira Shoulson, Professor of Neurology, Pharmacology and Human Science at Georgetown University (Washington DC) and the Chair of the Executive Committee of the Huntington Study Group said, “PBT2 attracted our attention as an experimental drug with the potential to bring real benefit to Huntington Disease patients who suffer from a range of motor, behavioural and cognitive symptoms. The favourable signals from the PBT2 trial in Alzheimer’s Disease are particularly promising.”
The disease affects 30,000 people in the US and about 70,000 worldwide. There are no drugs in development that have established clinical evidence for treating cognitive decline. In this trial, Prana is studying the safety and tolerability of PBT2 in Huntington patients and investigating potential benefits in cognition, motor coordination, behavioural, functional and psychiatric effects. In addition the trial will pilot biomarker and imaging assessments. The protocol synopsis appears below in Appendix 1.
Appendix 1 – Protocol synopsis
|Title||A randomized, double-blind, placebo-controlled study to assess the safety and tolerability, and efficacy of PBT2 in patients with early to mid-stage Huntington disease (HD)|
|Study Design||Randomised, double-blind, placebo-controlled, parallel group, multi-centre, Phase 2a study.|
To evaluate the safety and tolerability of two dose levels of PBT2 when administered orally once daily over 26 weeks in patients with HD.
|Number of Patients||100 patients (original target)|
|Key Patient Criteria|| — Men and women with Total Functional Capacity (TFC) 6-13, inclusive, and a CAG repeat number of ≥ 36
— Montreal Cognitive Assessment (MoCA) score ≥ 12
|Doses||Placebo (0mg PBT2), 100mg PBT2 and 250mg PBT2, once daily capsules.|
|Per Patient Duration||34 weeks: Four week Screening period, 6 months (26 weeks) treatment period and Follow-up 4 weeks post treatment.|
— Safety and Tolerability assessments.
— Cognition Tests: Cognitive Test Battery (consisting of Category Fluency Test, Trail Making Test parts A and B, Map Search, Symbol Digit Modalities Test and Unified Huntington Disease Rating Scale (UHDRS) Stroop Word Reading). MoCA.
— Motor Function Tests: UHDRS ’99 Motor component; Speeded Tapping Task.
— Behavior: UHDRS Behavioral component.
— Functional Abilities: Total Functional Capacity and Independence Scale from UHDRS ’99; Schwab & England Activities of Daily Living Scale (SEADL).
— Subject and investigator global assessments: Patient Reported Outcomes; Clinical Global Impression – Severity Scale.
— Biomarkers: small molecule markers of metabolic and oxidative stress in blood and urine; blood levels of total and mutant huntingtin; gene expression markers of HD progression; plasma selenium.
— Brain Imaging: volumetric and functional measures.
— Pharmacokinetics: sparse sampling.
|Trial Locations|| — Australia
|Trial Standard||Study being conducted according to ICH GCP|