E-Rare-3 JTC 2016 – Full proposal application form
E-Rare Joint Transnational Call for Proposals 2016
"Clinical research for new therapeutic uses of already
existing molecules (repurposing) in rare diseases"
Full-proposal application form
Please note that Type A project (including a pre-clinical study) MUST also
complete the “Preclinical Annex Application Form”
All fields must be completed using "Arial font, size 11" characters single-spaced, margins
of 1.27 cm.
Please note that incomplete full-proposals, proposals using a different format or exceeding
length limitations of any sections will be rejected without further review.
Proposals that do not meet the national eligibility criteria and requirements will be declined
without further review.
All the information requested in this document must be compiled into one single Pdfdocument and uploaded to the electronic submission system.
In case of inconsistency between the information registered in the submission tool and the
information included in the PDF of this application form, the information registered in the
submission tool shall prevail.
CHECKLIST FOR THE COORDINATOR:
In order to make sure that your proposal will be eligible to this call, please collect the information
required to tick all the sections below before starting to complete this application form.
- General conditions:
The project proposal addresses the AIM/S of the call
The project proposal meets the TOPIC/S included in this call
- The composition of the consortium:
The project proposal involves at least 3 eligible research partners from at least 3 different countries
participating in the call.
The project proposal does not include more than two eligible research partners from the same partner
country participating in the call.
The consortium coordinator is eligible to receive funding from the funding organisations participating in
the call.
There are a maximum of 2 partners who secure their own funding and contribute substantially to the
workpackages present in the proposal.
The project proposal involves a maximum of 6 eligible research partners asking for funding. In case of
inclusion of partners from participating Eastern European countries the project involves a maximum of 8
eligible partners asking for funding.
There are a maximum of 10 partners in total in the project proposal
E-Rare-3 JTC 2016 – Full proposal application form
- Eligibility of consortium partners:
I have checked that each partner involved in the project proposal is eligible to receive funding by its
funding agency. Not eligible partners are therefore aware of that fact and a signed statement declaring that
they will run the project with their own resources is enclosed in the proposal.
(if applicable) Italian partners involved in the proposal have submitted a pre-submission eligibility check
form to their national funding organization at least 7 working days before the submission deadline.
E-Rare-3 JTC 2016 – Full proposal application form
Basic project data
Project title
Acronym (max. 20 characters)
Type of Project (select one)
Type A
Type A project MUST also complete the
“Preclinical Annex Application Form”
Type B
Project duration
months
Total requested funding
€
Funding by Patients Organizations:
I agree to be co-funded by a Patient Organization
I disagree to be co-funded by a Patient Organization
Keywords: please identify between three and seven keywords that represent the scientific content
(medical domain, disease, etc), therapeutical approach(es), tools (animal models, OMICS, etc.)
Lay summary: please give a comprehensive and readable summary of the primary aims and methods of
the project (why the research is being suggested, what you aim to achieve, how this may impact on the rest
of the research community and society). This summary will be submitted to patient organisations potentially
interested by co-funding. Please note that if your proposal is selected for funding this abstract could be
used for communication purposes by E-Rare-3 or national funding agencies.
Please remember that the text should be written in an easily readable style. You should use short, clear
sentences broken up into paragraphs for readability, and avoid complex grammatical structures where
possible. You should use everyday English words in place of complex words. (max. 1 page)
E-Rare-3 JTC 2016 – Full proposal application form
Consortium coordinator (Partner 1):
Family Name, first Name
Institution
Department/Division
Position
Address
Zip code, City Country
Phone + Fax
E-mail address
Type of entity
Academia, Clinical or Public Health, SME and Industry
Type of entity
(public/private-forprofit/private-non-for-profit)
Research Partners:
a) Research partners asking for funding:
Research
Partner
No.
(principal
investigator)
2
3
4
5
6
7
8
(only possible with
inclusion of Eastern
European partner)
(only possible with
inclusion of Eastern
European partner)
Institution, Department, full
affiliations (address, zip code,
City, Country, phone + fax)
Email
address
Type of
entity
Academia,
Clinical or
Public
Health, SME
and Industry
Type of entity
(public/privateforprofit/privatenon-for-profit)
E-Rare-3 JTC 2016 – Full proposal application form
b) Associated research partners not asking for funding
Research
Institution, Department,
Zip code,
Partner
full affiliations (address, Email
No. City,
(principal
phone + fax)
address
Country
investigator)
1
2
Type of
entity
Academia,
Clinical or
Public
Health, SME
and Industry
Type of entity
(public/privateforprofit/privatenon-for-profit)
E-Rare-3 JTC 2016 – Full proposal application form
Clinical trial description
Please note that the clinical trial description part has to be fulfilled for Type A and Type B projects.
In the case of Type A projects, as many information as possible have to be presented in this
proposal for a correct evaluation of the project. In the case where a Type A project is funded, it will
be re-evaluated at the end of the first year on the basis of: 1/ scientific results obtained in the
preclinical study and 2/ updated clinical trial description.
1. Clinical trial synopsis
Principal/Coordinating First name, last name, academic title
Institution and department (complete name)
Investigator
Title of clinical trial
Clinical trial type and
phase
Objective(s)
Intervention(s)
Key inclusion and
exclusion criteria
Endpoint(s)
Sample size
Statistical analysis
Trial duration
Participating centres
Key words
Summary
Postal address
Telephone
Fax
E-mail address
Descriptive title identifying the study design, population and interventions
e.g. randomized/non-randomized, type of masking (single, double, observer blind), type
of controls (active/placebo), parallel group/cross-over
Which principal research questions are to be addressed? Specify clearly the primary
hypotheses of the trial that determine sample size calculation.
Experimental intervention :
Control intervention:
Duration of intervention per patient:
Duration of follow-up per patient:
Key inclusion criteria:
Key exclusion criteria:
Primary endpoint(s):
Secondary endpoint(s):
Number of patients to be assessed for eligibility:
Number of patients to be allocated to the trial:
Number of patients to be analysed:
Efficacy:
Description of the primary efficacy analysis and population:
Safety:
Secondary endpoints:
First patient in to last patient out (months):
Recruitment period (months):
Duration of the entire trial (months):
Total number:
Official name of the individual centres and respective location:
(5-7)
(max ½ page)
E-Rare-3 JTC 2016 – Full proposal application form
2. The medical problem (indicating background and rationale; evidence [e.g. give references to any
relevant systematic review(s) and/or (own) pilot studies, feasibility studies, relevant previous/ongoing trials,
case reports/series]; description of the unmet medical and patients’ need that are addressed by the
proposed work; critical analysis of the feasibility studies; critical analysis of the added value and the
limitations) (max. 3 pages)
3. Study design and endpoints
3.1 Description of intervention(s) (describe and justify experimental intervention, control intervention,
scheme of intervention [specify the drug dose and mode of administration, if applicable] and additional
intervention(s))
3.2 Primary and secondary endpoint(s) (Describe the primary and secondary objectives and how
these objectives will be measured as endpoints/outcome measures)
3.3 Relevant guidance documents (References to guidance documents considered to be relevant for
the study: e.g. guidelines from scientific societies (e.g. addressing standard-of-care) or regulatory bodies
(e.g. from the European Medicines Agency EMA) and HTA agencies. For example, for studies addressing
development and optimisation of drug therapies, disease specific, general 'clinical pharmacology and
pharmacokinetics' or methodological EMA 'Scientific guidelines' might have an impact on the later
scientific/regulatory value and applicability of results.)
3.4 Scientific advice / protocol assistance / communication with regulatory / competent
authorities / ethics committees (If scientific advice/protocol assistance from a competent/regulatory
authority has been requested, please provide the full text answer of the authority or a comprehensive
summary in this section of the document. If the answer is not yet available provide an explanation of the
current status. Please also include in this section any other relevant correspondence or minutes of
meetings with regulatory authorities or ethics committees such as requested or granted approvals of clinical
trial applications.
Clearly define the regulatory / ethical status and requirements for the study according to the national and
EU regulations.)
3.5 Orphan designation (If orphan designation has been granted provide the reference of the
Commission Decision. If orphan designation has been requested but not granted, provide an update on the
current status.)
3.6 Subjects/population(s) (Define study population(s) by inclusion and exclusion criteria. Please
discuss the potential inclusion of special populations, especially children and elderly (with defines age
groups). If there populations are excluded, please justify. Define sub-populations if subgroup analysis is
intended.)
3.7 Bias protection (Feasibility of randomisation; prognostic factors to be regarded in the randomisation
scheme and the analysis; proposed practical arrangements for allocating participants to trial groups; trial
E-Rare-3 JTC 2016 – Full proposal application form
site effects be considered in randomisation; feasibility of blinding (if blinding is not possible please explain
why and give details of alternative methods to avoid biased assessment of results (e.g. blinded assessment
of outcome))
3.8 Statistic analysis planning and power calculation (Define and justify (power calculation of)
sample size. Define statistical methods and planning of statistical analysis (proposed strategy of statistical
analysis, strategy for analysing the primary outcome, discussion of the robustness of your results). Please
specify: the number of patients to be assessed for eligibility, to be allocated to trial, to be analysed
(including subgroups if applicable); the expected compliance rate; the expected rate of loss to follow-up.)
(max. 1,5 page)
3.9 Cumulative safety information (Concise information on safety and tolerability of study
interventions: e.g. pre-clinical data from in-vitro or in-vivo studies; data from previous clinical studies; data
from (pharmaco-)vigilance systems or other sources.)
3.10 Agent(s) safety information (Concise information on available data on safety of the studied
Agent(s) in other disease e.g. approved for other therapeutic indication, off label use or well established
use, development up to Phase 2 for other indication has been successful)
4. Conduct of the trial
4.1 Schedule for study conduct including timelines for key study milestones (Please present
in this section a (realistic) planning of the schedule for the study conduct including provisions and timelines
for ethics and further administrative approvals. As a minimum include planning and timing for the key study
milestones below. Dates for key study milestones are defined relative to the starting date of the project (i.e.
month 1, month 6 etc.):
 The project has all administrative and regulatory authorisations
 First Patient (or study subject), First Visit (FPFV)
 The project has included 50% of patients
 Last Patient (or study subject), First Visit
 Last Patient (or study subject), Last Visit
 End of Study (including follow-up and data analysis) )
E-Rare-3 JTC 2016 – Full proposal application form
4.2 Trial timeline flow (Please provide a diagram reflecting preparation, pre-study-visits and initiation of
centres, recruitment, follow-up and date cleaning/analysis. This diagram should include the milestones
described in section 4.1)
4.3 Description of recruitment strategy (Description of the recruitment strategy including realistic
estimates of the expected recruitment rate (subjects per month/per centre) based on available and reliable
data. Please provide the evidence that the intended recruitment rate is achievable and specify whether and
how the collaboration with the partners in the research consortium will facilitate the recruitment. Please
specify the plans for monitoring of recruitment and contingency planning for recruitment problems)
4.4 Study management, data and sample management (Please include a description of planned
strategy for study / trial management; adverse event reporting; data collection and management; sample
management)
4.5 Sponsor, coordinating centre(s) and committees (Please specify the trial sponsor. Specify the role
of the coordinating centre(s) and different committees (as for example Data Safety Monitoring Board, Independent
Data Monitoring Committee, etc.) )
4.6 Study medication (Please provide information on whether manufacturing and / or labelling of the
study medication is required and which plans and / or commitments are in place for this)
5. Ethical, legal and social issues (ELSI) implications (Risks/benefits; Protection of
individuals/patients enrolled in the trial; Informed consent process and forms; Compensation for
individual/patients enrolled in the trial; Confidentiality and data protection; Conflict of interest; Strategies
and plan for data and database management (including location, access and regulatory implications),
dissemination of results) (max. ½ page)
6. Quality assurance and safety (Quality assurance (intervention, monitoring (study monitoring plan
(monitoring visits, level of source data verification, etc.)) and follow-up); Safety; Pharmacy issues (if
applicable)) (max. 1 page)
E-Rare-3 JTC 2016 – Full proposal application form
7. List of participants involved in the trial
Trial sponsor :
Trial management:
Name
Affiliation
Responsibility/Role
Signature
Trial statistician:
Name
Affiliation
Responsibility/Role
Signature
Trial supporting facilities (reference laboratories, pharmacies etc.):
Name
Affiliation
Responsibility/Role
Recruiting centres
Name
Affiliation
(only
institution and city/
country, no complete
address)
No. of
patients
with
condition
relevant to
the trial
seen in last
12 months
No. of
these
patients
fulfilling the
inclusion
criteria
No. of these
patients
which would
appr. agree
to participate
in the trial
per year
Expected
no. of
patients
recruited
for the
complete
trial
Total sum of recruited patients
Data and safety monitoring board:
Name
Affiliation (only institution and city/country, no complete address)
Other participating groups / bodies (e.g. steering committee)
Name
Affiliation
Responsibility/Role
Review of trial protocol (who will review and finalize the protocol?)
Name
Affiliation (only institution and city/country, no complete address)
8. Financial details of the trial

Commercial interest (max. ½ page)
Source of
these
figures
E-Rare-3 JTC 2016 – Full proposal application form

Reimbursement and contractual involvement of patient recruitment sites and trial management (if
applicable) [Please specify whether the reimbursement and contractual involvement will involve full
beneficiaries, “third parties”, subcontractors, or contract/clinical research organisations (CRO)]

Distribution of trial costs between health insurer, hospital and trial sponsor

Involvement and specific contribution of patients’ organisations

Financial summary
Organizational
segment
Institution /
Participant /
Trial Site
No of items / Kind of
equipment/ Explanation
Clinical project
management
Project
management
Data
Management
Biometry
- number of visits per site
(incl. initiation, interim and
close-out visits)
- mean number of days per
visit (incl. preparation/ postprocessing)
- mean travel time per visit
- monitoring costs per day
- total no of visits @ x € each
Quality
Assurance/
Monitoring
IRB and DSMB
Number of
members
Meetings/
Travel
Number of
attendees
Case payment
Reference
centres
no. of meetings @ x €/person
- no. of meetings @ x
€/person
- Monitoring travel costs
€/patient x no. of patients
(assays/examinations/
hospitalisation per patient
plus hours of staff per
patient)
no. of samples @ x €
Consumables
Trial manuals,
files, forms
Trial drug
€/patient
Insurance
€/patient
Fees
Publications
Equipment
< 10.000 €
> 10.000 €
> 50.000 €
Qualification
of staff
Total
months
Total
(€)
E-Rare-3 JTC 2016 – Full proposal application form
Other
TOTAL

Financial plan
Total resources required
Organizational
segment /
activity / task
Explanation / Comments /
Items
Staff
salary group
Other
expenses
Months1)
Partner
No.
TOTAL
TOTAL REQUESTED
FUNDING
1)
2)
please indicate full-time equivalents
please use thousands separator

Equipment

Financial contribution provided by industry and/or other third parties:
€2)
€2)
E-Rare-3 JTC 2016 – Full proposal application form
Project description
9. Added value of the proposed transnational collaboration (max. 1 page)
10. Description of the expected impact and exploitation / dissemination of project results
(max. ½ page)
11. Description of patents and present / future position with regard to intellectual property
rights, both within and outside the consortium (e.g. any barriers to sharing materials or translating the
results into clinical application including freedom to operate-analysis in respect to patent and exploitation strategy
(technical and commercial)) (max. ½ page)
12. Description of ongoing or submitted research grants of each participating partner
related to the present topic (indicating funding sources [include at least: ID number, amount and
duration of funded project; funding agency] and possible overlaps with the project proposed) (max. ½ page
per research partner)
13. Concept for sustainability of instruments initiated by the project (e.g. registries, cohorts,
biobanks, databases etc.) and their possible interaction with European Infrastructure Initiatives (where
applicable, e.g. BBMRI, ELIXIR, EATRIS, ECRIN, EU-Openscreen etc.) (max. 1 page)
14. Description of participation/engagement of Industry and/or patient organizations within
the proposal, including their role and contribution (max. 1 page, only if applicable).
15. Brief CVs for each participating partner leader (with a list of up to five relevant publications
(complete reference required) within the last five years demonstrating the competence to carry out the
research project) (max. 1,5 page per partner leader)
Date and signature of coordinator