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To introduce
the principles and issues involved in a mucositis-related clinical
trial, we will create a hypothetical Phase III trial of a mouth
rinse for oral palliation in patients undergoing chemotherapy
for colorectal cancer. This study will be a multi-center, randomized,
double-blind, placebo-controlled, parallel Phase III clinical
trial. Click here
for a review of clinical trial phases.
The hypothetical
mouth rinse we want to study is alleged to work by adhering to
the cellular components of damaged oral and gastrointestinal mucosa
and acting as a barrier to various damaging by-products of chemotherapy
treatment. It is alleged to alleviate pain and mucosal damage
intraorally and along the gastrointestinal tract. The rinse is
delivered via swish and swallow every 8 hours. Patients are asked
to swish for 2 minutes and swallow thereafter. The medication
is to commence when pain in the throat or mouth develops.
The objectives
of this study are:
- To assess
the efficacy of the rinse in decreasing the severity of mucositis
and mucositis-related mouth pain, and
- To assess
the rinse's safety and tolerability.
Determining
such general objectives is often easier than defining specific
questions to address them. Well-defined objectives allow for better
design. Objectives should be solidified before data collection
begins.
Choosing primary
and secondary endpoints can help to clarify the goals of the study.
Due to the subjective nature of symptoms such as pain and discomfort,
unambiguous endpoints need to be defined. In this study subjective,
objective, and functional outcomes are assessed.
The primary
endpoints are:
- Severity
of mouth and throat pain following the presentation of oral
and throat pain;
- Duration
of mouth and throat pain and narcotic use following treatment
with the rinse;
- Severity
of oral mucositis as determined by the OMAS and WHO scales;
and
- Duration
of narcotic use.
Secondary
endpoints include:
- Ability
to eat solids, liquids, or nothing by mouth;
- Weight
loss/gain; and
- Safety
and tolerability.
Question
8.1
| If
you were performing a Phase I study for this medication, how
would the primary and secondary endpoints differ? |
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They would not differ because you would be interested in the
same outcomes. |
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The primary endpoints would be safety and tolerability. |
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The endpoints of the study would only focus on efficacy of
the medication. |
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