Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT03464773 |
| Other study ID # |
H16-03288 |
| Secondary ID |
|
| Status |
Completed |
| Phase |
N/A
|
| First received |
|
| Last updated |
|
| Start date |
April 15, 2018 |
| Est. completion date |
July 31, 2023 |
Study information
| Verified date |
May 2024 |
| Source |
University of British Columbia |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
Children born with severe brain-based developmental disabilities frequently experience
persistent unexplained periods of pain and irritability, often compounded by a limited
capacity to communicate their distress. The investigators call this entity Pain and
Irritability of Unknown Origin (PIUO). The investigators have designed a systematic approach,
called the PIUO Pathway, to address the management of these children's pain and irritability
with the goals of reducing pain symptoms, improving the day-to-day lives of the child and
family, and simplifying treatment options for clinicians.
Description:
The purpose of this project is to develop, test, and disseminate an approach to reducing and
resolving pain in children with developmental brain disorders, specifically those with severe
neurological impairments (SNI). It focuses on the problem of ongoing, unexplained, and
difficult-to-treat pain and irritability that many children with SNI, and their families,
experience over time. The goal is to improve the assessment and treatment of pain and
irritability in children diagnosed with complex health conditions and multiple disabilities,
who have limited communication and cognition.
The plan is to evaluate the effectiveness of an integrated clinical pathway (i.e. a
sequential order of standardized evaluation steps) for managing unexplained pain and
irritability in these vulnerable children.
BACKGROUND Pain is the most common symptom reported by the parents and caregivers of children
with SNI, however, pain is notoriously difficult to identify, and therefore treat, in
non-verbal children because their signals of distress are ambiguous and hard to decode.
Typical distress behaviours include crying, tears, facial grimace, limb withdrawal, arching,
hypertonicity, and decreased sleep. More mobile children may curl up into a ball, fling their
arms or legs, or engage in self-injurious behaviour; conversely they may display decreased
activity.
Children with SNI may experience nociceptive-inflammatory pain as a result of their specific
medical condition (e.g. joint contractures) or from many procedures that they experience
(e.g. injections). Often, however, it is not clear what underlies the pain behavior. Parents
are experts in identifying pain behaviours in their own child, but consider their
interpretation to be a complex and uncertain process. As well, while pain behaviours can be
described by clinicians and parents, the behaviours themselves are subjective, ambiguous, and
can reflect a variety of problems in addition to pain. Unless an obvious
nociceptive-inflammatory trigger such as an injury is witnessed, parents and clinicians find
it very difficult to ascribe all of the pain-like behaviours observed in children with SNI to
pain as defined by the International Association for the Study of Pain (IASP). In this
context, the term "pain" does not serve as a descriptive label. Therefore, the investigators
describe pain behaviour episodes using a less deterministic term, "pain and irritability,"
acknowledging that the behaviour is certainly negative in the eyes of the witnessing
caregiver. The team has defined this entity as Pain and Irritability of Unknown Origin
(PIUO).
HYPOTHESIS The overall hypothesis is that children participating in the PIUO Pathway will
experience improvement or resolution of PIUO from baseline to post-study, compared to
children receiving treatment as usual while on the waitlist.
The goal is to improve PIUO beyond what has been undertaken by the child's usual clinical
teams. The primary outcome is improved pain control for non-verbal children with SNI as shown
in a reduction of pain and irritability episodes and their severity. Secondary outcomes
include decreased pain severity; improved family quality of life and ease of implementation
of the PIUO Pathway for clinicians.
STUDY DESIGN Interventions for resolving PIUO are time- and resource-intensive. They require
a focused approach to assessing all the underlying possibilities for the PIUO and addressing
each potential source of pain one at a time. Tackling PIUO requires systematic,
comprehensive, process-oriented thinking applied by an interdisciplinary team with
flexibility in terms of approach and time. There have been recommendations in the literature
about how to approach the problem of PIUO in children with SNI, but these recommendations
have never been subjected to systemic evaluation as complex interventions, for example by
following an integrated clinical pathway.
Integrated clinical pathways (also called "care pathways" or "critical pathways") are
structured, sequenced approaches guiding the delivery of healthcare, often with a
multidisciplinary perspective. They provide algorithms to be followed in the assessment and
treatment of conditions, but are not intended to be rigidly followed; instead, integrated
clinical pathways delineate a foundation for a clinician's approach to a complex problem,
thereby avoiding inefficiencies in evaluation or resource use. Integrated clinical pathways
are a form of Complex Interventions and have been developed for sickle cell pain, childhood
asthma, appendicitis, autoimmune conditions, in-born errors of metabolism, and cerebrospinal
fluid shunt management. Despite their complexity, integrated clinical pathways are
appropriate interventions for evaluation by Randomized Controlled Trial (RCT). RCTs of
integrated clinical pathways have been proposed and/or completed for complex, multi-factorial
patient problems including lung disease, heart failure, gastrointestinal surgery, stroke, and
hip fractures.
In this study, the investigators will use a waitlist-controlled RCT design, with 120 children
randomized to PIUO Pathway or waitlist (standard care) treatment arms. This design was chosen
strategically, with consideration of both the special pediatric population being studied and
the ethical inappropriateness of randomly assigning children to a placebo group when pain is
the target condition.
