Tag Archives: Clinical Trial

The European Medicines Agency (EMA) has released draft revised guidelines on medicines for the treatment of Alzheimer’s disease and other types of dementias for a six-month public consultation.

EMA follows a multi-stakeholder approach to facilitate research and development of more effective medicines. The revised guidelines take into account comments received at EMA’s workshop on the clinical investigation of medicines for the treatment of Alzheimer’s disease in November 2014. This workshop brought together a wide range of stakeholders, including patient representatives, regulators, pharmaceutical industry and independent experts. The aim of the workshop was to ensure that during the revision of its guidelines, EMA would be able to consider the most up-to-date scientific developments in understanding and treating Alzheimer’s disease and views from experts in the field. The revised guidelines also build on EMA scientific advice provided for a number of specific development plans for Alzheimer’s disease in recent years, as well as the qualification of several biomarkers for the selection of patients in clinical trials.

The revised guideline specifically addresses the:

  • impact of new diagnostic criteria for Alzheimer’s disease, including early and even asymptomatic disease stages, on clinical trial design
  • choice of parameters to measure trial outcomes and the need for distinct assessment tools for the different disease stagesin Alzheimer’s (different signs and symptoms, differences in changes over time, severity)
  • potential use of biomarkers and their temporal relationship with the different phases of Alzheimer’s disease at different stages of medicine development (mechanism of action, use as diagnostic test, enrichment of study populations, stratification of subgroups, safety and efficacy markers etc.)
  • design of long-term efficacy and safety studies

Comments received during the consultation will be taken into account in the finalisation of the guideline.

Stakeholders are invited to send their comments by 31 July 2016. To learn more, visit the EMA website.

Source: EMA

Eli Lilly and Company and AstraZeneca announced the start of recruitment of patients for the Phase II/III AMARANTH clinical trial of an inhibitor of beta secretes cleaving enzyme (BACE), currently under development as a promising therapy for Alzheimer’s disease.

The AMARANTH Phase II/III clinical trial will evaluate the safety and efficacy of oral AZD3293/LY3314814 against placebo and will assess if this drug can be designated as disease-modifying therapy for patients with early Alzheimer’s disease. The state of early Alzheimer’s disease includes patients with mild cognitive impairment (MCI) associated with Alzheimer’s disease and patients with mild Alzheimer’s dementia. This new study will include more than 1,500 patients in 15 countries, and the treatment will be for 2 years.

Source:  Alzheimer’s News Today

The 2CARE study of coenzyme Q for Huntington’s disease was halted early because an analysis of the results to date showed that it was very unlikely to show positive results. The study, called 2CARE, was designed to test whether a treatment called coenzyme Q10 could slow the progression of HD.

The Austrian biotech AFFiRiS AG announced positive results of its Phase I safety trial of a vaccine against alpha-synuclein.

Alpha-synuclein is the sticky protein that clumps in the cells of people with Parkinson’s, and AFFiRiS hopes to stop disease by inducing antibodies against alpha-synuclein accumulation. The Michael J. Fox Foundation funded this work with close to $2M, first with a grant for a pre-clinical study and then $1.5M in 2011 for the Phase I trial. This is the first drug against alpha-synuclein to reach clinical testing.

“A treatment that could slow or stop Parkinson’s progression would be a game changer for the five million worldwide living with this disease and the many more who will become at risk as our population ages,” said MJFF CEO Todd Sherer, PhD. “This trial is one of the most promising efforts toward that goal.”

In two different doses the drug, called PD01A, was safe and tolerable. Half of those vaccinated showed alpha-synuclein antibodies, which is a promising but very early sign. Further trials will test PDO1A’s benefit to patients. The next step is a boost study that will test the safety and effect of a boost vaccination (another dose). MJFF will support that trial, which will take place in Vienna, Austria and start recruiting in September.

Source:  PharmaBiz and Michael J Fox Foundation for Parkinson’s Research