Enroll-HD-Protocol-1.0.pdf - Enroll-HD Protocol Final...

Enroll-HD Protocol
Final Version 1.0
09 September 2011
1
Enroll-HD:
A Prospective Registry Study in a Global Huntington’s Disease Cohort
A CHDI Foundation Project
Clinical Study Protocol
Version 1.0
09 September 2011
Change History: Final Version: 09 September 2011
Principal Investigator: G. Bernhard Landwehrmeyer, MD, PhD
University of Ulm, Department of Neurology
Oberer Eselsberg 45/1
89081 Ulm
Germany

Enroll-HD Protocol
Final Version 1.0
09 September 2011
3
INVESTIGATOR OF RECORD SIGNATURE PAGE
Enroll-HD
Final Version 1.0, dated 09 September 2011
A CHDI Foundation Project
I, the Investigator of Record, agree to conduct this study in full accordance with the
provisions of this protocol. I will comply with all requirements regarding the obligations
of investigators as outlined in the International Conference on Harmonization, Good
Clinical Practices (GCP). I will also adhere to all local laws and regulations and as well
as any applicable Safe Harbor privacy principles. I agree to maintain all study
documentation.
I have read and understand the information in the study protocol and will ensure that all
associates, colleagues, and employees assisting in the conduct of the study are informed
about the obligations incurred by their contribution to the study.
__________________________________
Name of Investigator of Record
__________________________________ ____________________________
Signature of Investigator of Record Date

Enroll-HD Protocol
Final Version 1.0
09 September 2011
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T
ABLE OF
C
ONTENTS
Table of Contents ................................................................................................................ 4
Abbreviations ...................................................................................................................... 6
1 Synopsis ....................................................................................................................... 7
2 Background and Rationale ......................................................................................... 16
3 Study Objectives ........................................................................................................ 18
4 Study Description ....................................................................................................... 18
4.1 Study Period ....................................................................................................... 19
4.2 Study Population ................................................................................................ 19
4.2.1 Inclusion Criteria ........................................................................................ 19
4.2.2 Exclusion Criteria ....................................................................................... 20
4.3 Study Enrollment ................................................................................................ 21
4.3.1 Participant Identification, Recruitment ....................................................... 21
4.3.2 Consent Process .......................................................................................... 21
4.3.3 Participant Withdrawal ............................................................................... 23
5 Data Collection and Assessments .............................................................................. 24
5.1 Training .............................................................................................................. 24
5.2 Core Assessments ............................................................................................... 26
5.2.1 Core Motor Assessments ............................................................................ 27
5.2.2 Core Functional Assessments ..................................................................... 27
5.2.3 Core Behavioral Assessments ..................................................................... 27
5.2.4 Core Cognitive Assessments ....................................................................... 27
5.2.5 Research Genotyping .................................................................................. 28
5.3 Extended Assessments ....................................................................................... 28
5.3.1 Extended Behavioral Assessments ............................................................. 29
5.3.2 Extended Cognitive Assessments ............................................................... 29
5.3.3 Physiotherapy Outcome Measures .............................................................. 30
5.3.4 Quality of Life Assessments ....................................................................... 30
5.3.5 Health Economic Assessments ................................................................... 31
5.4 Optional Assessments ........................................................................................ 31
5.4.1 Family History Questionnaire (FHQ) ......................................................... 31
5.4.2 Biospecimens .............................................................................................. 32
5.4.3 Future Contact Regarding Research Studies ............................................... 33
5.4.4 Discussion of Post-Mortem Donation of Biological Materials ................... 33
5.4.5 Transfer of Data from Other HD Studies and Trials ................................... 33
5.4.6 Transfer of Biospecimens from Other HD Studies and Trials .................... 33

Enroll-HD Protocol
Final Version 1.0
09 September 2011
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5.4.7 Participation in Sub-Studies ........................................................................ 34
5.4.8 Ancillary Studies ......................................................................................... 36
5.5 Reportable Event Monitoring ............................................................................. 36
6 Statistical Methods ..................................................................................................... 37
6.1 Sample Size ........................................................................................................ 37
6.2 Data Analysis ..................................................................................................... 37
7 Study Management ..................................................................................................... 38
7.1 Participants Lost To Follow-Up ......................................................................... 38
7.2 Data Entry/Electronic Data Capture System ...................................................... 38
7.3 Source Documents .............................................................................................. 38
7.4 Quality Assurance and Monitoring .................................................................... 39
7.5 HD Identification Number (HDID) .................................................................... 39
7.6 Data storage and security ................................................................................... 40
7.7 Data Management .............................................................................................. 40
7.8 Changes to the Protocol ...................................................................................... 41
7.9 Study Governance .............................................................................................. 41
7.10 Publication Policy .............................................................................................. 42
8 Ethical And Regulatory Considerations ..................................................................... 44
8.1 Participant Confidentiality ................................................................................. 44
8.2 Data Safety Monitoring Committee (DSMC) ....................................................