SKIP-NMD

Work Plan

This section provides a graphical presentation of the SKIP-NMD workplan, showing the timing of the different workpackages and the main milestones and deliverables planned over the course of the project.

DX.X= project deliverable, i.e. a report showing completion of a specific task within the project

MSX = project milestone, i.e. control points marking successful attainment of a major result or points where decisions are needed with regards to the next stage f the project.

  • Year 1: November 2012-October 2013

Workpackages:
M1 
M2
M3
M4
M5
M6
M7
M8
M9
M10
M11
M12
WP1-Choice of a lead sequence
    D1.1

D1.2

MS1

               
WP2-Preclinical toxicology                        
WP3-Trial design and Regulatory authority submission                       D3.1
WP4-Clinical trial recruitment and execution
                       
WP5-A Natural History study of non-invasive biomarkers in  DMD
                       
WP6-ethics and parent organisations forum
    D6.1
    D6.2
          D6.3
WP7-Dissemination to the academic and parent organisations
                      D7.1
WP8-Project management and administration
    D8.1
                 

  • Year 2: November 2013-October 2014

Workpackages:
M13
M14
M15
M16
M17
M18
M19
M20
M21
M22
M23
M24
WP1-Choice of a lead sequence
                       
WP2-Preclinical toxicology D2.1
      D2.2
D2.3
  D2.5
  D2.4
     
WP3-Trial design and Regulatory authority submission            

D3.2

MS2

 

D3.3

MS3

     
WP4-Clinical trial recruitment and execution
                  D4.1
  D4.2
WP5-A Natural History study of non-invasive biomarkers in  DMD
                  D5.1
   
WP6-Ethics and parent organisations forum
                       
WP7-Dissemination to the academic and parent organisations
                      D7.2
WP8-Project management and administration
                       

  • Year 3: November 2014-October 2015

Workpackages:
M25
M26
M27
M28
M29
M30
M31
M32
M33
M34
M35
M36
WP1-Choice of a lead sequence
                       
WP2-Preclinical toxicology                        
WP3-Trial design and Regulatory authority submission                        
WP4-Clinical trial recruitment and execution
D4.3
MS4
D4.4
         

D4.5

MS5

     
WP5 - A Natural History study of non-invasive biomarkers in  DMD
                D5.2
MS6
   
WP6-Ethics and parent organisations forum
                       
WP7-Dissemination to the academic and parent organisations
                      MS7
WP8-Project management and administration
                       

Description of deliverables

  • D1.1, AOs comparative analysis 1: AOs comparative analysis in cell lines [month 3]
  • D1.2, AOs comparative analysis 2: AOs comparative analysis in humanised DMD mice and choice of lead compound for clinical trial [month 4]
  • D2.1, PMO production: 1 batch for toxicology will be delivered at month 11 and 5 batches for the clinical trial will be delivered at month 13. [month 13]
  • D2.2, Safety pharmacology: GLP Safety pharmacology in cynomolgus monkeys. [month 17]
  • D2.3, Gentoxicity studies report: Genotoxicity studies. [month 17]
  • D2.4, Repeated dose tox report: GLP 12 week repeat dose toxicity study in cynomolgus monkeys. [month 21]
  • D2.5, Interim report in repeated dose toxicology in CM: Four week interim report cynomolgus monkey repeat dose study. [month 19]
  • D3.1, clinical trial design: Definitive clinical trial design, study protocol and statistical plan. Accelerometer device modified to record activity in ambulant . Shipping of devices for functional monitoring [month 12]
  • D3.2, Dossier production for regulatory authorities: Investigational medicinal product dossier and submission to ethics and regulatory authorities; Ethics application for natural history study. [month 19]
  • D3.3, Registration for the trial: Registration of the trial [month 21]
  • D3.4, Submission of protocol for study extension: Submission of protocol for study extension [month 23]
  • D4.1, Initiate recruitment cohort I: Initiate recruitment cohort I [month 22]
  • D4.2, Initiate recruitment cohort II: Initiate recruitment cohort II [month 24]
  • D4.3, Initiate recruitment cohort III: Initiate recruitment cohort III [month 25]
  • D4.4, Initiate cohort extension: Initiate cohort extension [month 27]
  • D4.5, Study report: Study report [month 33]
  • D5.1, Iniatiation of enrolment: natural history patient enrolment [month 22]
  • D5.2, Study report: Study report [month 33]
  • D6.1, Creation of ethics board: Creation of ethics board [month 3]
  • D6.2, Discussion list for ethics board: Discussion list for ethics board and consortium members to raise issues and concerns [month 6]
  • D6.3, Six monthy meetings: Six monthly meetings or 'virtual' web conferences for ethics board [month 12]
  • D7.1, Dissemination and exploitation plan: Dissemination and exploitation plan [month 12]
  • D7.2, Dissemination to the public: Dissemination to the public [month 24]
  • D8.1, A secure website for partners: A web site for partners to deposit their data with secure access [month 3]

Project milestones

  • MS1: Lead compound identification [month 4]
  • MS2: Clinical trial protocol [month 19]
  • MS3: Start of clinical trial [month 22]
  • MS4: Completion of induction phase in all cohorts [month 26]
  • MS5: Completion and follow up of the final patients in the trial [month 33]
  • MS6: Completion of natural history study [month 34]
  • MS7: End of study [month 36]

Back to Top