Clinical trial NCT06075797: The PediQUEST Response to Pain Of Children With Neurologic Disability Pilot Randomized Controlled Trial
Official title: Feasibility of The PediQUEST Response to Pain Of Children With Neurologic Disability (PQ-ResPOND) Intervention: a Pilot Randomized Controlled Trial (RCT)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Cerebral Palsy Infantile
Disabilities Multiple
Pain
Keywords: Disabled children
pain
palliative care
electronic patient reported outcomes measures
Severe neurologic impairment
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: National Institute of Nursing Research (NINR)
name: Boston Children's Hospital
name: Dana-Farber Cancer Institute
name: University of Alabama at Birmingham
name: Deakin University
First posted: 2023-10-10
Enrollment: 60
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 2:1 parallel RCT
Primary purpose: OTHER

Brief summary: The goal of this pilot randomized controlled trial is to test the feasibility of running a full scale randomized controlled trial that compares the effect of the PQ-ResPOND intervention versus usual care to improve recurrent pain in children, adolescents, and young adults with severe neurologic impairment. The main questions it aims to answer are:

* Is the study feasible and acceptable for participants?
* Does PQ-ResPOND have a potential to be effective?

Participants will:

* answer surveys (their parents will) telling us about the child's pain, symptoms, and use of complementary therapies, and about their own psychological distress and satisfaction with care.
* a group will receive the PQ-ResPOND intervention which consists of:

  * activating parents and providers by using the PediQUEST system, a web platform that administers surveys and generates feedback reports alerting parents and providers about the child's experience, AND
  * responding to child pain or discomfort by incorporating the Response team (members of the hospital's palliative care team) into the child's care to privde a standardized approach to managing recurrent pain.

Researchers will use a comparison (control) group consisting of participants who will answer surveys and receive usual care (no feedback reports or consult with palliative care in this group) to see if a randomized design is feasible.

Detailed description: Background: In the US, tens of thousands of children, adolescents, and young adults (AYA) are estimated to live with severe neurologic impairment (SNI). Over 60% of these children endure uncontrolled and often persistent pain, which frequently goes unrecognized and untreated. The impact extends to their parents who commonly experience unremitting physical and psychological distress. There has been a lack of rigorous studies that focus on interventions to alleviate recurrent pain in children with SNI. This gap might be attributed to the stigmatization of these children, who are often from historically marginalized backgrounds, resulting in a low prioritization in research endeavors. To address this inequity and using the ORBIT Model for intervention development, the investigators have adapted and are refining an intervention previously applied in children with cancer. The PediQUEST Response to Pain Of children with Neurologic Disability (PQ-ResPOND) will respond to child distress by using "reciprocal activation" strategies to overcome normalization-the acceptance among parents and clinicians that symptom distress is an unavoidable outcome of serious illness-and integration of a specialty pediatric palliative care team (PPC). The overall objective of the study is to evaluate the feasibility of running a full-scale RCT that compares the effect of the PediQUEST ResPOND intervention vs. usual care in children and AYA with SNI.

Specific Aims are to 1. assess study's feasibility and acceptability by evaluating recruitment (rates and suitability of screening/tracking), adherence to the intervention (by study participants and interventionists), and retention (dropout rates, reasons, and demographics); and, 2. explore potential for intervention's efficacy by estimating effect sizes and variability of the outcomes proposed for the full-scale RCT (child recurrent pain and symptom burden, and parent outcomes) and assessing intervention's acceptability through semi-structured interviews.

Methods: Researchers will conduct a single site Phase II Pilot 2:1 RCT of PQ-ResPOND (intervention, n=30) vs. usual care (control, n=15) in 45 children ≥1 year-old \& AYA with SNI and moderate to severe recurrent pain behaviors. After randomization, participants will answer weekly PQ-ResPOND surveys and monthly outcome surveys over 12 weeks via the PQ App, and a semi-structured exit interview. Intervention: participants assigned to this arm will receive the PQ-ResPOND intervention which combines (i) parent-mediated activation through weekly collection and feedback to parents and clinicians, via our PQ App, of parent reported child pain (Non-Communicating Children's Pain Checklist-Revised scale (NCCPC-R)) and other symptoms (PediQUEST-Memorial Symptom Assessment Scale adapted for children with SNI), with (ii) PPC team-mediated activation of primary clinicians and parents: through PPC consultation, and (iii) PPC team activation through training in a standardized approach to recurrent pain using the PQ-ResPOND Checklist (adapted by the study's interprofessional expert panel). Usual care: participants in this arm will continue receiving care as usual.

Outcomes: Investigators will use quantitative and qualitative outcomes to study feasibility and acceptability including recruitment and retention rates, intermittent attrition, adherence to intervention delivery by clinicians, and participant and clinician's acceptability of the intervention. To preliminarily estimate effect sizes and variability, the corresponding scores for child's pain and symptom burden, and parent distress and activation measures will be estimated.

Analysis: For feasibility and acceptability quantitative outcomes investigators will use descriptive statistics. Demographics and reasons for attrition will be reported to explore potential for selection bias. A focused thematic analysis will be used to analyze qualitative variables. To estimate effect sizes and variability of trial outcomes, mixed linear regression models will be estimated. A predefined set of progression criteria are in place.

Discussion: Results will inform whether a full-scale Phase III RCT is warranted, and will help with study design and sample size calculation, development of a manual of procedures and fidelity monitoring strategies.

Eligibility criteria: Inclusion Criteria:

* Parent-child dyads will be included based on the following criteria:

Child participants will be selected from the base population of patients who are:

* ≥ 1 year old,
* receiving routine (ongoing) care at Boston Children's Hospital, AND
* followed by the Neurology, Cerebral Palsy or Complex Care services.

All patients from the base population will be screened to include any patient who:

* has severe neurological impairment, defined as:

  * a Central Nervous System disorder resulting in motor and cognitive impairment and an inability to communicate either verbally or through sign language, AND
  * has complete caregiver dependency for activities of daily living AND
* date of diagnosis of SNI is ≥ 4 months prior to the date of screening

Exclusion Criteria:

* Parent-child dyads will be excluded if any of the following apply:

  * the child,

    * is already followed by the palliative care team, OR
    * is not expected to survive at least 2 months after enrollment; OR
    * does not have "moderate to severe recurrent pain", as measured through the baseline Child Pain Survey (PPP). Specifically, we will exclude participants whose:
* "Good days" AND "Difficult days" PPP scores are \<14, AND
* "Most troublesome pain" PPP score is \<30, AND
* Number of "difficult days" are \<8 in past month, AND
* Number of days with "most troublesome pain" are \<4 in past month; OR • both parents,

  * do not have legal guardianship, OR
  * are unable to read, write, and speak English OR Spanish
  * are unable to understand and complete surveys.

Primary outcomes:
measure: Recruitment rate
timeFrame: 16 months
measure: Recruitment/month
timeFrame: 16 months
measure: Randomization rate
timeFrame: 16 months
measure: Suitability of screening/tracking
timeFrame: 16 months
measure: Sample diversity
timeFrame: 16 months
measure: Refusal reasons
timeFrame: 16 months
measure: Dropout rate
timeFrame: 16 months
measure: Demographics of dropouts
timeFrame: 16 months
measure: Dropout reasons
timeFrame: 16 months
measure: Intermittent attrition
timeFrame: 16 months
measure: Adherence with answering surveys
timeFrame: 16 months
measure: Feasibility of intervention delivery
timeFrame: 16 months
measure: Participant's acceptability of the intervention
timeFrame: At week 12
measure: Likelihood that participants would recommend study to others
timeFrame: At week 12
measure: Participant's acceptability of intervention (qualitative)
timeFrame: At week 12
measure: Clinician's acceptability of intervention (qualitative)
timeFrame: 16 months
measure: Participant's overall satisfaction with the study
timeFrame: At study exit
Secondary outcomes:
measure: Child pain
timeFrame: 12 weeks
measure: Child symptom burden
timeFrame: 12 weeks
measure: Parent Anxiety
timeFrame: 12 weeks
measure: Parent Depression
timeFrame: 12 weeks
measure: Parent Pain-related Stress
timeFrame: 12 weeks
measure: Parent overall health quality
timeFrame: 12 weeks
measure: Parent sleep quality - total sleep time
timeFrame: 12 weeks
measure: Parent sleep quality - refreshing sleep
timeFrame: 12 weeks
measure: Use of Complementary Therapies
timeFrame: 12 weeks
measure: Parent Coping - Active coping
timeFrame: 12 weeks
measure: Parent Coping - Planning
timeFrame: 12 weeks
measure: Parent Coping - Instrumental support
timeFrame: 12 weeks
measure: Parent Coping - Acceptance
timeFrame: 12 weeks
measure: Parent Coping - Self-blame
timeFrame: 12 weeks
Other outcomes:
measure: Progression criteria: Recruitment rate
timeFrame: 16 months
measure: Progression criteria: Recruitment/month
timeFrame: 16 months
measure: Progression criteria: Retention rate
timeFrame: 16 months
measure: Progression criteria: Feasibility of intervention delivery
timeFrame: 16 months
measure: Progression criteria: Participant's acceptability of intervention
timeFrame: 16 months
Locations:
city: Birmingham
country: United States
facility: University of Alabama, Birmingham
state: Alabama
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: Burwood
country: Australia
facility: Deakin University
state: Victoria
Study officials:
affiliation: Massachusetts General Hospital
name: Joanne Wolfe, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: