Study to assess the effect of AZD3241 in Multiple System Atrophy
Research type
Research Study
Full title
A 12-Week, Multicenter, Randomized, Parallel-Group Study to Assess the Safety, Tolerability, Pharmacokinetics, Biomarker Effects, Efficacy, and Effect on Microglia Activation, as Measured by Positron Emission Tomography, of AZD3241 in Subjects with Multiple System Atrophy
IRAS ID
170514
Contact name
Michele Hu
Contact email
Sponsor organisation
AstraZeneca AB
Eudract number
2014-004902-13
Duration of Study in the UK
1 years, 9 months, 2 days
Research summary
Multiple system atrophy (MSA) is a rare, progressive, neurological disorder that affects adult men and women. The cause of MSA is unknown, however MSA is associated with degeneration of nerve cells or atrophy of nerve cells in areas of the brain which can result in problems with movement, balance and automatic functions of the body such as bladder and blood pressure control.
There is no cure for MSA. Current treatment involves treating specific symptoms which is only modestly effective. Therefore there is a great need for drugs that treat the core area of the disease, as well as for drugs that could slow disease progression in patients with MSA.AZD3241 is a powerful, specific blocker of a special protein molecule called myeloperoxidase (MPO) enzyme. MPO produces toxic agents harmful to the body's cells in MSA. AZD3241 blocks its activity which prevents the production of toxic agents and will slow the progression of neurodegeneration. These effects in the brain are measured with positron emission tomography (PET) scanning, and involve changes in brain cells called microglia.
Astra Zeneca is sponsoring a study to determine the safety and tolerability of AZD3241. This is a randomised, double-blind study meaning neither the patient nor the study doctor will know which medication is being given. Patients will be randomly assigned to receive one of three treatment groups, which will receive twice-daily doses of up to 300 mg AZD3241, up to 600 mg AZD3241, or placebo, depending on the group.
This is a multicentre study which will take place across Europe and North America. Approximately 64 subjects will be recruited worldwide. The total duration of participation is approximately 5 months, including a screening period of up to 49 days, a double blind treatment period of 12 weeks and a follow up visit 14 days after the last dose.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
15/SC/0028
Date of REC Opinion
9 Apr 2015
REC opinion
Further Information Favourable Opinion