Clinical trial NCT02471339: Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain
Official title: Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain: A Phase III Clinical Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Neurofibromatosis Type 1
Plexiform Neurofibromas
Keywords: Psychological Intervention
Coping
Mindfulness
Randomized
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 2015-06-15
Enrollment: 66
Sex: ALL
Minimum age (years): 16
Maximum age (years): 59
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: Background:

- People with neurofibromatosis type I (NF1) and plexiform neurofibroma (PN) tumors often have chronic pain that his hard to control. People usually take medicines for the pain, but they may not work well and might cause side effects. A new strategy called Acceptance and Commitment Training (ACT) may help these people cope with chronic pain. ACT focuses on things like values and living in the moment.

Objective:

- To see if Acceptance and Commitment Training improves pain coping in people with NF1 pain.

Eligibility:

- People age 16-34 who have NF1, 1 or more PN tumors, and pain that interferes with their daily functioning.

Design:

* Participants will be screened with a physical exam, medical history, and questions about their pain.
* Participants will fill out questionnaires about their pain and feelings. Their heart rate will be measured via electrocardiogram (ECG).
* Participants will be divided into 2 groups randomly. One will wait 8 weeks.
* The other will start training right away.
* Participants will have 2 two-hour sessions with an ACT trainer. They will learn techniques for setting goals based on personal values and other ways to cope with pain. They will get a workbook and a compact disc (CD) to take home for practice.
* Participants will do practice exercises at home between sessions. They will get weekly emails with a practice exercise. They will join video chat sessions via home computer with their trainer.
* All participants will return to National Institutes of Health (NIH) after 8 weeks for questionnaires and an ECG. The wait group will then start training. They will return 8 weeks later for questionnaires and an ECG.
* Six months later, they will complete questionnaires from home by computer.

Detailed description: BACKGROUND:

* Neurofibromatosis type 1 (NF1) is a genetic disorder that affects approximately 1 in 3,500 individuals.
* A number of common clinical manifestations, including plexiform neurofibromas, can cause frequent and significant pain and impact quality of life.
* Often, NF1-related pain is not well-controlled with medication and many of the medications cause significant side effects.
* To our knowledge, only one prior study (by our group) has examined the effectiveness of a psychological intervention for chronic pain in adolescents and young adults (AYA) with NF1.
* Acceptance and Commitment Therapy (ACT), a newer generation of cognitive-behavioral therapy, focuses on encouraging individuals to engage in more adaptive ways of coping with pain.
* The goal of ACT is not to eliminate the person's pain, but to optimize the person's functioning despite their pain

OBJECTIVES:

-To compare pain interference mean score changes from baseline to 8 weeks between the ACT intervention group and the waitlist (WL) group.

ELIGIBILITY:

* Patients 16 - 59 years of age with a confirmed diagnosis of NF1 and greater than or equal to 1 plexiform neurofibroma (PN).
* The patient must obtain a mean score of 2.0 or higher or a score of 3 on three or more items on the Pain Interference Index, and report having pain that interferes with functioning for at least three months.
* The patient must have regular access to a computer or tablet with internet access.

DESIGN:

* This is a psychological intervention study to determine the potential benefit of ACT on pain interference, with patients randomized to the ACT intervention or a wait-list control group.
* Patients will come to the National Institutes of Health (NIH) for a 2-day visit (Time 1). After baseline assessments (questionnaires and ECG) are completed, participants in the ACT group will take part in two 2-hour ACT training sessions. At home, this group will receive weekly emails through week 8, and will participate in further ACT training/educational sessions via video chat at weeks 2, 4, and 6.
* All patients will return to the NIH at week 8 (Time 2) to complete follow-up questionnaires and ECG. At this time, the WL group will cross over and receive the ACT intervention (in-person sessions, weekly emails, and video chat sessions). All patients will complete questionnaires again from home six months following the completion of the intervention.
* To detect a change of .68 standard deviation on the primary outcome measure between the two groups at .80 power, 41 patients per group are needed.

Eligibility criteria: * INCLUSION CRITERIA FOR PARTICIPANT

  1. Patients must be between 16 and 59 years of age at the time of the baseline assessment. Because the research on the effectiveness of Acceptance and Commitment Therapy (ACT) with younger children is still emerging, children 15 and younger will be excluded from the present study.
  2. Diagnosis of Neurofibromatosis Type 1 (NF1) through germline mutation OR clinical diagnosis; for the clinical diagnosis of NF1 all study subjects must have two or more diagnostic criteria for NF1 listed below (National Institutes of Health (NIH) Consensus Conference):

     * Six or more cafe-au-lait spots (greater than or equal to 0.5 cm in prepubertal subjects or greater than or equal to 1.5 cm in postpubertal subjects)
     * Greater than or equal to 2 neurofibromas or 1 plexiform neurofibroma
     * Freckling in the axilla or groin
     * Optic glioma
     * Two or more Lisch nodules
     * A distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex)
     * A first-degree relative with NF1
  3. Participants must have documentation of a plexiform neurofibromas (PN), based on either clinical exam or imaging.
  4. Patient must self-report having chronic pain for at least the past 3 months that has interfered with their daily functioning, as assessed by the Pain Interference Index (must get a mean score of 2.0 or higher, or score a 3 on three or more individual items).
  5. Patients must have regular access to a computer or tablet with internet access.
  6. Ability of subject or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.
  7. No anticipated major changes in their pain treatment regimen (i.e., new class of pain medication starting or change in the class of pain medication) or enrollment on a new treatment study presumed to impact pain in the near future.
  8. Subjects must be able to read and comprehend the English language, since the highly trained ACT therapists are not fluent enough to conduct the trainings in Spanish or other languages.

EXCLUSION CRITERIA FOR PARTICIPANT

1. In the opinion of the Principal Investigator (PI) or an Associate Investigator (AI), the subject has significant cognitive or emotional difficulties that would prevent them from being able to understand and/or participate fully in the intervention or the measures.
2. Subjects who are participating in any other treatment studies, either medical or behavioral, specifically for pain management.
3. Subjects who began a medical intervention for treatment of their disease that has a possible impact on pain (including Mitogen-activated Protein/Extracellular Signal-regulated Kinase Kinase (MEK) trials) will not be eligible until after one year on the medical treatment; at that time, eligibility will be discussed with the PI of the medical study to assess the stability of the patient s pain and whether further pain-related changes due to the medical treatment are likely.
4. Inability to travel to the NIH, for example, due to physical limitations, for the in-person evaluation(s).

No groups in regards to gender, race, or ethnicity are being excluded from participation in the trial.

Primary outcomes:
measure: Mean Pain Interference Mean Score Change From Baseline to 8 Weeks Between the Acceptance and Commitment Therapy Intervention Group and the Waitlist Group
timeFrame: Baseline and 8 weeks
Secondary outcomes:
measure: Change in Quality of Life (QOL) for Physical Functioning
timeFrame: Baseline and 8 weeks
measure: Mean Pain Acceptance Pre- and Post-treatment
timeFrame: Baseline and 8 weeks
measure: Change in Disease-related Pain Severity and Overall Tumor Pain
timeFrame: Baseline and 8 weeks
measure: Change in Quality of Life (QOL) for Daily Activities
timeFrame: Baseline and 8 weeks
measure: Change in Disease-related Depression
timeFrame: Baseline and 8 weeks
measure: Change in Pain-related Anxiety
timeFrame: Baseline and 8 weeks
measure: Change in Heart Rate Variability (HRV)
timeFrame: Baseline and 8 weeks
measure: Pain Interference Patient-Reported Outcomes Measurement Information System (PROMIS) II
timeFrame: Baseline and 8 weeks
measure: Measure of Pain Inflexibility Pre- and Post-treatment
timeFrame: Baseline and 8 weeks
Other outcomes:
measure: Number of Participants With Serious and/or Non-serious Adverse Events
timeFrame: Baseline, and Week 8, 16, 32, and 40.
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center, 9000 Rockville Pike
state: Maryland
Study officials:
affiliation: National Cancer Institute (NCI)
name: Staci M Peron, Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: