Chapter 1 Overview

1.1 Dataset

Extraction

The data contained in Enroll-HD PDS5 was extracted from the Enroll-HD electronic data capture (EDC) database on October 31, 2020, at 23:00 UTC.


Data sources

The PDS5 dataset encompasses data exclusively from Enroll-HD participants, collected from several sources. These sources are the Enroll-HD study, the REGISTRY study, and clinical data collected in adhoc visits outside of the aforementioned studies.

Enroll-HD is an observational cohort study and global clinical research platform designed to facilitate Huntington’s disease (HD) clinical research. It includes participants from North America, Europe, Australasia, and Latin America. The study started in 2012 and is still active and actively recruiting.

REGISTRY isan observational cohort study of HD conducted in Europe. The study started in 2004 and concluded in 2015. As Enroll-HD began, REGISTRY sites and participants began to transition into Enroll-HD. Enroll-HD dataset releases include individuals who initially participated in REGISTRY then consequently enrolled in Enroll-HD and consented to the migration of their REGISTRY data into the Enroll-HD dataset. Registry data are available for a subset of Enroll-HD participants.

Clinical data from additional sources (Ad Hoc data) are available for a subset of Enroll-HD participants. These data were collected at routine clinical visits outside of the Enroll-HD and REGISTRY studies, and comprise HD assessment data (e.g., UHDRS Motor). The date of collection of these data typically pre-date a participant’s enrolment into REGISTRY or Enroll-HD.

Study specific protocols, annotated eCRFs, and data collection guidelines are housed here.


Participant inclusion

To be included in the PDS5 release, participant’sdata had to meet several requirements. Figure 1.1 illustrates the number of participants whose data met each of the predefined inclusion requirement and illustrates how the final sample size of PDS5 was determined.

Participant flow chart for inclusion in PDS5.

Figure 1.1: Participant flow chart for inclusion in PDS5.

Note that as a result of these requirements, not all participants enrolled in Enroll-HD at the time of PDS5 data cut are included in PDS5. Similarly, not all participants included in PDS4 are included in PDS5. Data from 150 participants who were included in PDS4 were not included in the current PDS release. Enroll-HD is an active, longitudinal study. A participant eligible for inclusion for one release may be ineligible the next (e.g., participant data quarantined). Data for PDS4 participants not included in PDS5may be available through specified dataset (SPS) request.

1.2 Sample Size

PDS5 contains data on 21,116 Enroll-HD participants. Sample size by PDS release is presented in Figure 1.2 .

Enroll-HD sample size by PDS release

Figure 1.2: Enroll-HD sample size by PDS release

1.3 Visits

PDS5 contains data from 71,682 visits (baseline and follow-up visits only; all sources). Of these, 55,975 were Enroll-HD visits. The remainder are from Registry (N = 14,737) and ‘Ad Hoc’ sources (N = 970). A breakdown of visits by data source is provided in Table 1.1. Number of Enroll-HD visits only by PDS release are illustrated in Figure 1.3.

Table 1.1: Number of visits in PDS5 by constituent data source. Participant number indicates the number of EnrollHD participants with visit data available for the indicated data source (maximum N = 21,116)
Data source Participants Visits
Enroll-HD 21116 55975
Registry 3 4168 9708
Registry 2 2079 5029
Ad Hoc 302 970
Total 71682
Enroll-HD visitsonly (baseline and follow-up only) by PDS release.

Figure 1.3: Enroll-HD visitsonly (baseline and follow-up only) by PDS release.

Considering baseline and follow-up visits from Enroll-HD only, total number of visits per participant in PDS5 ranges from 1 to 9. In Figure 4, we illustrate participant counts by maximum number of Enroll-HD visits. Each participant is represented once, included in the bar indicative of their maximum number of visits. In Figure 5, we illustrate maximum participant counts for a specific number of visits. This plot is cumulative, the goal being to illustrate largest available sample size for a specific number of visits. For example, the participant with 9 visits is represented in visit bars 1 through 9, the participants with 8 visits are represented in visit bars 1 through 8, and so on.

1.4 Sample Characteristics

The PDS5 sample is characterized below with respect to participant category, sociodemographic variables, and clinical characteristics(Figures 7 to 18).

1.5 Data availability and completeness