The PediQUEST Response to Pain Of Children With Neurologic Disability Pilot Randomized Controlled Trial

Pain Clinical Trial

— PQ-ResPOND  

Official title:

Feasibility of The PediQUEST Response to Pain Of Children With Neurologic Disability (PQ-ResPOND) Intervention: a Pilot Randomized Controlled Trial (RCT)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06075797
• Other study ID #: 2023p002305
• Secondary ID: 1R21NR020433
• Status: Recruiting
• Phase: N/A
• Start date: March 25, 2024
• Est. completion date: July 15, 2025

Study information

• Verified date: May 2024
• Source: Massachusetts General Hospital
• Contact: Madeline E Avery, MPH
• Phone: 617 643 8846
• Email: meavery[@]mgh.harvard.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 60
• Est. completion date: July 15, 2025
• Est. primary completion date: May 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

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.

Related Conditions & MeSH terms

• Cerebral Palsy
• Cerebral Palsy Infantile
• Disabilities Multiple
• Pain

Intervention

Behavioral:
• PediQUEST ResPOND
PPC teams will have access to feedback reports and the PQ-ResPOND checklist (standardized framework to diagnosis and treatment of recurrent pain behaviors in children with severe neurologic impairment). Integration of the PPC team into care will be achieved through (i) initial consultation (goals: identify treatment goals, and address recurrent pain), and (ii) follow-up: the team will contact or visit the family in response to feedback reports or for treatment monitoring purposes.

Locations

Country	Name	City	State
Australia	Deakin University	Burwood	Victoria
United States	University of Alabama, Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Dana-Farber Cancer Institute	Boston	Massachusetts

Sponsors (6)

Lead Sponsor	Collaborator
Massachusetts General Hospital	Boston Children's Hospital, Dana-Farber Cancer Institute, Deakin University, National Institute of Nursing Research (NINR), University of Alabama at Birmingham

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Progression criteria: Recruitment rate	To decide whether to progress or not to a Phase III trial, we will analyze a subset of primary outcomes using a red, amber, green (RAG) approach for each criteria. For recruitment rate we will proceed with RCT (Go) if the indicator is =60%, Amend (Proceed with changes) if indicator is between 50% and 59%, and Stop (will not proceed to RCT unless changes are possible) if indicator is <50%.	16 months
Other	Progression criteria: Recruitment/month	For recruitment/month we will proceed with RCT (Go) if the indicator is =4, Amend (Proceed with changes) if indicator is between 3 and 4 and Stop (will not proceed to RCT unless changes are possible) if indicator is <3 patients/month.	16 months
Other	Progression criteria: Retention rate	For retention rate we will proceed with RCT (Go) if the indicator is =80%, Amend (Proceed with changes) if indicator is between 70% and 79% and Stop (will not proceed to RCT unless changes are possible) if indicator is <70%.	16 months
Other	Progression criteria: Feasibility of intervention delivery	For feasibility of intervention delivery, a qualitative indicator, we will proceed with RCT (Go) if the indicator is judged as strongly feasible, Amend (Proceed with changes) if indicator is judged as feasible, and Stop (will not proceed to RCT unless changes are possible) if indicator is judged as possibly feasible.	16 months
Other	Progression criteria: Participant's acceptability of intervention	For participant's acceptability of intervention, a qualitative indicator, we will proceed with RCT (Go) if the indicator is judged (by participants) as strongly acceptable, Amend (Proceed with changes) if indicator is judged (by participants) as acceptable, and Stop (will not proceed to RCT unless changes are possible) if indicator is judged (by participants) as fairly acceptable.	16 months
Primary	Recruitment rate	Proportion of contacted potential participants meeting initial inclusion criteria who consent to participate	16 months
Primary	Recruitment/month	Number of participants recruited per month	16 months
Primary	Randomization rate	Proportion of enrolled participants who meet randomization criteria (moderate to severe pain)	16 months
Primary	Suitability of screening/tracking	Qualitative outcome: As reported by research staff and site investigators involved in patient validation	16 months
Primary	Sample diversity	Proportion of enrolled participants by race/ ethnicity	16 months
Primary	Refusal reasons	Qualitative: Description of reasons for refusal mentioned in the consent conversation or non-participation survey	16 months
Primary	Dropout rate	Proportion of enrolled participants who dropout from the study	16 months
Primary	Demographics of dropouts	Proportion of dropout participants by race/ ethnicity	16 months
Primary	Dropout reasons	Description of dropout reasons referred by participants during exit interview or dropout conversation	16 months
Primary	Intermittent attrition	Surveys' response rates and distribution of non-responses will be used to determine participant's adherence with study data collection procedures	16 months
Primary	Adherence with answering surveys	Qualitative: Parent perspectives on survey frequency, length, relevance, and burden	16 months
Primary	Feasibility of intervention delivery	Qualitative: Using information from the PQ system, medical records, and exit interviews we will assess how feasible it is to deliver the different intervention components.	16 months
Primary	Participant's acceptability of the intervention	Satisfaction with the intervention measured through a numerical 0-10 rating score	At week 12
Primary	Likelihood that participants would recommend study to others	Agreement with recommending the study to others using a numeric 0-10 rating score	At week 12
Primary	Participant's acceptability of intervention (qualitative)	Qualitative: We will explore participant's views on the intervention during exit interviews	At week 12
Primary	Clinician's acceptability of intervention (qualitative)	Qualitative: Clinicians views on the intervention will be explored during semi-structured clinician interviews (these will be conducted every 6 months with a subset of clinicians)	16 months
Primary	Participant's overall satisfaction with the study	We will analyze participant's overall satisfaction with the study on a 0 to 10 numeric scale	At study exit
Secondary	Child pain	This is the proposed primary outcome for future Phase III trial. All secondary outcomes will be used to preliminarily estimate effect sizes and help with sample size calculations. We will present estimates of effect, variability, and intraclass correlation coefficients with their 95% Confidence Intervals (95%CI).; Difference between intervention and control arms of the average Pediatric Pain Profile (PPP) most difficult day of last week scores over 12 weeks, measured monthly, as reported by the parent for all enrolled children. Score range: 0-60, higher scores indicate more pain. Cut off point for pain: 14 (>14 indicates pain)	12 weeks
Secondary	Child symptom burden	Difference between intervention and control arms of the average (PediQUEST ResPOND Memorial Symptom Assessment Scale, PQ-ResPOND MSAS) total scores over 12 weeks, as reported weekly by the parent. PQ-ResPOND MSAS total scores are calculated as the average of the 21 individual symptom scores. Individual symptom scores are calculated as the average of the three symptom questions. Individual symptom scores and total scores range from 0-100, 100 indicates higher symptom burden.	12 weeks
Secondary	Parent Anxiety	Difference between trial arms of the average Spielberger's-State Anxiety Inventory-State scores over 12 weeks, measured monthly. Scores range from 20 to 80, with higher scores correlating with greater anxiety.	12 weeks
Secondary	Parent Depression	Difference between trial arms of the mean Center for Epidemiologic Studies Short Depression Scale (CES-D-10) scores over 12 weeks. Scores range from 0 to 30 (Score of 10 or higher indicates the presence of significant depressive symptoms).	12 weeks
Secondary	Parent Pain-related Stress	Difference between trial arms of the mean pain-related stress scores measured with an adapted version of the stress-portion of the Response to Stress Questionnaire-Pain, measured monthly. The tool assesses 12 potential stress sources. Scores range from 1-4 (higher means more stress). Symptom-related stress score calculated as the sum of the 12 item scores.	12 weeks
Secondary	Parent overall health quality	Difference between trial arms of the mean overall health quality score measured monthly with one item of the Short Form (12) Health Survey (SF-12). Uses a 5 point Likert type scale and is scored 0-100, where 100 is excellent overall health.	12 weeks
Secondary	Parent sleep quality - total sleep time	Parent sleep quality will be measured with an adapted version of the Consensus Sleep Diary. Difference between trial arms of the mean overall score of the weekly average total sleep time (in hours), measured monthly. More hours of sleep is better.	12 weeks
Secondary	Parent sleep quality - refreshing sleep	Measured with an adapted version of the Consensus Sleep Diary. Difference between trial arms of the mean overall weekly score of the refreshing sleep item, measured monthly. Response options 1 to 5, where 5 is extremely refreshing.	12 weeks
Secondary	Use of Complementary Therapies	Difference between trial arms of the No. of complementary therapies used measured monthly with the Use of Complementary therapies checklist (developed ad hoc). Continuous variable, where higher numbers indicates more use of complementary therapies.	12 weeks
Secondary	Parent Coping - Active coping	Difference between trial arms of the mean overall active coping score measured monthly with the Short version of the Coping Orientation to Problems Experienced Inventory (Brief COPE).; Scores on each scale (or item) range from 2 to 8, with higher scores indicating greater use of that strategy.	12 weeks
Secondary	Parent Coping - Planning	Difference between trial arms of the mean planning coping score measured monthly with the Short version of the Coping Orientation to Problems Experienced Inventory (Brief COPE). Scores on each scale (or item) range from 2 to 8, with higher scores indicating greater use of that strategy.	12 weeks
Secondary	Parent Coping - Instrumental support	Difference between trial arms of the mean instrumental support coping score measured monthly with the Short version of the Coping Orientation to Problems Experienced Inventory (Brief COPE). Scores on each scale (or item) range from 2 to 8, with higher scores indicating greater use of that strategy.	12 weeks
Secondary	Parent Coping - Acceptance	Difference between trial arms of the mean acceptance coping score measured monthly with the Short version of the Coping Orientation to Problems Experienced Inventory (Brief COPE).; Scores on each scale (or item) range from 2 to 8, with higher scores indicating greater use of that strategy.	12 weeks
Secondary	Parent Coping - Self-blame	Difference between trial arms of the mean self-blame coping score measured monthly with the Short version of the Coping Orientation to Problems Experienced Inventory (Brief COPE). Scores on each scale (or item) range from 2 to 8, with higher scores indicating greater use of that strategy.	12 weeks