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

NCT06075797 | Completed DMC

• 2 other identifiers: 2023p0023051R21NR020433
• Study Type: interventional
• Target: 54
• Locations: 2 countries
• Sites: 4

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.

Conditions

Pain; Cerebral Palsy Infantile; Disabilities Multiple

Keywords

Disabled children; pain; palliative care; electronic patient reported outcomes measures; Severe neurologic impairment

Outcome Measures

Primary Outcomes (17)

• Recruitment rate

  Proportion of contacted potential participants meeting initial inclusion criteria who consent to participate

  16 months

• Recruitment/month

  Number of participants recruited per month

  16 months

• Randomization rate

  Proportion of enrolled participants who meet randomization criteria (moderate to severe pain)

  16 months

• Suitability of screening/tracking

  Qualitative outcome: As reported by research staff and site investigators involved in patient validation

  16 months

• Sample diversity

  Proportion of enrolled participants by race/ ethnicity

  16 months

• Refusal reasons

  Qualitative: Description of reasons for refusal mentioned in the consent conversation or non-participation survey

  16 months

• Dropout rate

  Proportion of enrolled participants who dropout from the study

  16 months

• Demographics of dropouts

  Proportion of dropout participants by race/ ethnicity

  16 months

• Dropout reasons

  Description of dropout reasons referred by participants during exit interview or dropout conversation

  16 months

• 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

• Adherence with answering surveys

  Qualitative: Parent perspectives on survey frequency, length, relevance, and burden

  16 months

• 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

• Participant's acceptability of the intervention

  Satisfaction with the intervention measured through a numerical 0-10 rating score

  At week 12

• 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

• Participant's acceptability of intervention (qualitative)

  Qualitative: We will explore participant's views on the intervention during exit interviews

  At week 12

• 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

• 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 Outcomes (14)

• Child pain

  12 weeks

• Child symptom burden

  12 weeks

• Parent Anxiety

  12 weeks

• Parent Depression

  12 weeks

• Parent Pain-related Stress

  12 weeks

Other Outcomes (5)

• Progression criteria: Recruitment rate

  16 months

• Progression criteria: Recruitment/month

  16 months

• Progression criteria: Retention rate

  16 months

Study Arms (2)

PQ-ResPOND (Intervention)

EXPERIMENTAL

Participants in the intervention arm will (i) answer weekly PQ-ResPOND Surveys over 12 weeks, (ii) receive feedback reports (generated by the PediQUEST system to summarize parents answers), and (iii) engage with the pediatric palliative care (PPC) team. The interprofessional PPC team will focus on child's recurrent pain and related symptoms using a standardized approach and work on family activation strategies.

Behavioral: PediQUEST ResPOND

Usual care (Control)

NO INTERVENTION

Participants randomized to the control arm will be assigned weekly PQ-ResPOND Surveys up to week 12. Participants in this arm will not have access to the full PediQUEST system, i.e. no reports will be generated. They will not meet the PPC team but can receive regular palliative care consultations following the site's usual referral procedures. Children in the usual care arm will receive standard care, which at Boston Children's Hospital usually involves several primary clinicians (primarily neurologists or Complex Care Service attending physicians) and access to psychosocial clinician care throughout the illness course. PPC referrals are typically made at the discretion of the primary clinician, often for decision making reasons and/or closer to end-of-life. If a child presents persistent distress, they will be cared through the usual mechanisms. The rationale for collecting weekly surveys is to minimize reporting bias and may increase adherence and retention.

Interventions

PediQUEST ResPOND BEHAVIORAL

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.

Also known as: Intervention Arm

PQ-ResPOND (Intervention)

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• National Institute of Nursing Research (NINR): collaborator
• Boston Children's Hospital: collaborator
• Dana-Farber Cancer Institute: collaborator
• University of Alabama at Birmingham: collaborator
• Deakin University: collaborator

Study Sites (4)

University of Alabama, Birmingham

Birmingham, Alabama, 35294, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Deakin University

Burwood, Victoria, 3125, Australia

Location

MeSH Terms

Conditions

Cerebral Palsy; Pain

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Joanne Wolfe, MD, MPH

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Physician in Chief, Chair, Department of Pediatrics

Model Details: 2:1 parallel RCT

Study Record Dates

• First Submitted: September 26, 2023
• First Posted: October 10, 2023
• Study Start: May 8, 2024
• Primary Completion: July 15, 2025
• Study Completion: July 31, 2025
• Last Updated: January 21, 2026

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); US (3)