Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT04295811 |
| Other study ID # |
PR-0138 / EG-CL-102 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
N/A
|
| First received |
|
| Last updated |
|
| Start date |
June 18, 2020 |
| Est. completion date |
December 2023 |
Study information
| Verified date |
January 2023 |
| Source |
PAVmed Inc. |
| Contact |
Alexa Rueda |
| Phone |
9157405766 |
| Email |
AXR[@]pavmed.com |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
The study will assess the performance of the combined system, i.e., the use of the EsoGuard
assay (lab developed test) on cells collected using the EsoCheck (501k cleared device) to
detect Barrett's Esophagus (BE), with or without dysplasia, and esophageal adenocarcinoma
(EAC) as compared to Esophagogastroduodenoscopy (EGD) plus biopsies in both confirmed cases
of BE/EAC and in controls (subjects without a prior diagnosis but undergoing screening for
BE/EAC)
Description:
This is a two phase multicenter study to assess the operating characteristics of the EsoGuard
diagnostic assay panel performed on esophageal mucosal cells collected using the EsoCheck
cell collection device in known "Cases" of disease (i.e., patients with a history of
Barrett's Esophagus (BE) with and without varying degrees of dysplasia or intramucosal
adenocarcinoma [IMC]) and in patients with no known history of these conditions. The latter
are presumed to be "Controls", though this final determination is made as part of study
conduct, not at the time of enrollment.
The study is divided into a Run-In phase and an Efficacy phase. The assignment of a patient
to one or the other of these two phases will be made based on two pieces of information: 1)
the Final Study Diagnosis (as defined below) and 2) the current tally versus the
pre-determined target number of patients already assigned to each of the subgroups (e.g.,
non-dysplastic Barrett's Esophagus (NDBE), high grade dysplasia {HGD]) for each phase.
Patients will first be enrolled into the Run-In phase subgroups. Only once the targeted
enrollment for a given Run-In phase subgroup has been reached, subsequent patients with a
particular diagnosis will be assigned to the appropriate Efficacy Phase subgroup, until the
targeted total number is reached. Run In phase data will be maintained in a separate database
from Efficacy phase data. Study conduct is identical in both phases; however, data from each
phase will be segregated and analysis of each phase will be used solely for its predetermined
purpose without any co-mingling of data. Once assigned, no patient, or their data, will be
re-assigned or moved from being a Run-In phase patient to being an Efficacy phase patient, or
from the Run-In phase database to the Efficacy phase database, or vice versa.
The Run-In phase will enroll the initial 60 short segment NDBE (also known as short segment
Barrett's Esophagus [SSBE]) and 25 long segment NDBE (also known as long segment Barrett's
Esophagus [LSBE]) Cases, the initial 10 low grade dysplasia [LGD] Cases, the initial 3 high
grade dysplasia [HGD] Cases, and the initial 2 intramucosal adenocarcinoma [IMC] Cases, as
well as the initial 100 Controls. The Efficacy phase will enroll 54 Cases each with a Final
Study Diagnosis of NDBE, LGD, HGD, and IMC, and 54 Controls.
Run-In phase data will be used solely to derive the optimal numerical cutoffs by which to
score mVIM and mCCNA1 (which are two genes where the methylated DNA changes are located)
positivity or negativity. These cutoffs serve as the key inputs into an algorithm by which an
overall EsoGuard result of positive versus negative is determined. The setting of these
cutoffs will be done by Sponsor personnel with full access to all Run-In phase data; the goal
will be to optimize overall EsoGuard assay sensitivity and specificity for its intended use
as a screening test in the at-risk population. The assay, once validated and locked, will be
used to analyze patients' distal esophageal cells obtained in both the Efficacy phase of this
Case Control study as well as in a separate Screening study (PR-1039/EG-CL-101) to be
conducted in parallel. Only Run-In phase data will be used to set cutoffs. The mVIM and
mCCNA1 (i.e., genes with methylated DNA changes) cutoffs will be set and then the EsoGuard
assay re validated and "locked", all before any Efficacy phase distal esophageal cells
specimens collected from study patients will undergo EsoGuard analysis.
Sponsor personnel will have open access to all Run-In phase data during the enrollment of the
Run-In phase in order to determine if the data from patients enrolled to date is sufficient
to inform adequately the setting of cutoffs. If Sponsor so determines, it may elect to
terminate enrollment in the Run-In phase early (i.e., prior to enrolling the 100 Cases and
100 Controls listed above). If early termination of the Run-In phase is elected, all patients
enrolled subsequent to the date Sponsor makes this election will be entered into the Efficacy
phase and such subsequent patients will count towards the Efficacy phase enrollment
objectives.
As well, should Sponsor complete the intended Run-In phase enrollment of 100 Cases and 100
Controls but determine, upon its assessment of the resulting data, that data from additional
Cases could improve the setting of cutoffs, Sponsor may decide to enroll up to 100 additional
Run-In phase Cases. These 100 Cases may be in whatever distribution of disease Sponsor elects
(i.e., ranging from NDBE through to IMC). In order to augment the Run-In phase patient
counts, the Sponsor will set updated targets for enrollment of each Run-In phase subgroup and
will assign patients to each Run-In subgroup to be augmented, based on patients' Final Study
Diagnosis and on whether the updated subgroup target has been met. When each updated subgroup
enrollment target has once again been met, subsequent patients with that subgroup diagnosis
will be enrolled into the Efficacy phase.
