Grants: The specific objective of this call is to promote the clinical and pre-clinical proof of concept studies for the potential application of medicinal products that are either already marketed or having achieved a significant stage in the development process in rare indications. Projects should aim at discovering new therapeutic uses for Agents whose clinical safety has already been documented to move quickly into proof of concept trials in the selected rare diseases’ patient population.
Two types of projects are eligible for this call:
Type A: Preclinical studies to verify target engagement and to perform additional toxicity testing if necessary (for example in the case of paediatric indications where juvenile animal studies might be warranted) in a disease model for a maximum period of one year followed by the implementation of Phase 1b or Phase 2a clinical trials at the beginning of the second year of the project. For this call, Phase 1b, and Phase 2a trials are defined as follows: Phase 1b trials are defined as studies usually conducted in the target patient population to establish feasibility (e.g., target engagement, pharmacodynamics/pharmacokinetics (PD/PK), initial dosing of the Agent) prior to a Phase 2a trial. Phase 2a clinical trials provide data on the relationship of dosing and response for the particular intended use (including trials on the impact of dose ranging on safety, biomarkers, and proof of concept), typically 150 subjects or less for trials in adults.
Type B: Milestone-driven Phase 2 clinical trials to demonstrate that the Agent modulates the target and has the potential to yield the desired clinical outcome in the proposed disease population for a period up to three years.
Criteria that shall be fulfilled
The following approaches and topics are excluded from the scope of the call:
Timeline: here will be a pre-registration procedure followed by atwo-stage submission/evaluation procedurefor joint applicationsThe call is scheduled as follows:
All the information:
Entidad Financiadora: E-Rare-3
Plazo de Presentación: 03/03/2016
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