Symptoms and General Pathology

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Nervous System Diseases

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Pain

Neurofibromatoses

Neurofibromatosis 1

Neurofibroma

Neurofibroma, Plexiform

Nerve Sheath Neoplasms

Neoplasms, Nerve Tissue

Neoplasms by Histologic Type

Syndrome

Neoplastic Syndromes, Hereditary

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Neurodegenerative Diseases

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Neurofibromatosis

Neurofibromatosis Type 1

Patients with NF1 who have PNs and report experiencing pNF related pain and parents of these patients. (completed)

Patients with NF1 who have pNFs(completed)

Parents of patients in cohort 2 (completed)

Patients with NF1 who have pNFs and recent pNF-related pain

Parents of patients (ages 8-17 years) enrolled in cohort 4

Pamela L Wolters, Ph.D.

National Cancer Institute Referral Office

Background:

People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (pNFs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning.

Objectives:

To examine and improve questionnaires about daily living for people with NF1 and pNFs.

Eligibility:

People ages 5 and older with NF1 and a pNF

Design:

Participants will be screened with medical history.

This study will have 2 phases.

Phase 1 participants will talk about existing pain assessment questionnaires and how pNFs affect their life. They will have group discussions of up to 8 people of a similar age with NF1 and pNFs, or the parents of children with it. These will last about 90 minutes. Children ages 5 to 7 and their parents will have one-on-one meetings instead. These will last about 45 minutes. Discussions will be audiotaped. After the questionnaires have been changed, individual interviews will discuss the new wording, instructions, questions, and electronic format of the new forms.

Phase 2 is now complete.

Phase 1 participants may be invited to Phase 2.

Phase 2 participants will complete the new questionnaires. These may be pen-and-paper or electronic. The questionnaires will take about 30 minutes for adults and teens. Children will work one-on-one with a staff member and may need up to 45 minutes. A small group of participants will be complete the forms twice-in clinic and 1 month later at home. Also, a small group who start a new pain treatment or have a dose increase in their treatment will complete the forms twice-before the treatment change and 1 month later.

Background:

* Neurofibromatosis 1 (NF1) is a genetic disease with multiple clinical manifestations, including plexiform neurofibromas (pNFs) that can cause pain and may significantly impact daily functioning and quality of life (QOL).
* Patient-reported outcomes (PROs) are useful in trials for conditions that are disabling and chronic like NF1, where symptom reduction and improved functioning and QOL currently are important treatment outcomes, which may occur with pNF shrinkage.
* A critical step toward approval of drugs to treat pNFs is to evaluate clinical benefit in conjunction with a reduction in tumor volume as assessed by imaging endpoints.
* The FDA requests the use of PROs in NF1 clinical trials, especially for assessing changes in symptoms, such as pain.
* Currently, no valid PRO measures exist that are specific to the NF1 population to assess pNF pain or its functional impact on an individual s life.

Objectives:

* Phase 1: Qualitative Evaluation - COMPLETE

  --To evaluate current modifications and the need for any additional modifications to existing measures of pain intensity (Numeric Rating Scale; NRS-11) and pain interference (Pain Interference Index; PII) and select the most appropriate items to measure physical functioning (PROMIS Physical Functioning; PROMIS-PF) in NF1 based on qualitative feedback from patients with NF1, pNFs and pain to use as endpoints in clinical trials for individuals with NF1 and pNFs.
* Phase 2: Evaluation of Psychometric Properties and Collection of Normative Data
* To evaluate final versions of the NRS-11 (now called the PAin INtensity Scale for pNF \[PAINS-pNF\]) and PII (now called the Pain Interference Index for pNF \[PIIpNF\]) measures on reliability, validity, and feasibility in individuals with NF1 and pNFs.

Eligibility:

* Patients with documented NF1 either by NIH clinical criteria or molecularly-proven mutation in the NF1 gene will be included in the study. Patients must have at least 1 plexiform neurofibroma (pNF) that is at least 3cm on longest diameter by physical exam (i.e. visual exam, palpation) or 2D MR imaging OR \>=3mL by volumetric MR imaging. Patients must be at least 8 years of age and able to understand, read, and speak English.
* Patients will be ineligible if they were enrolled on a MEK inhibitor trial in the past 12 months or began a new pain treatment regimen (e.g., medication, psychosocial therapy, physical therapy, etc.) within the past three months at the time of recruitment.
* Primary caregivers (i.e. parent, guardian, grandparent) of participants younger than 18 years old are also eligible to participate in order to provide parent report information to further validate the PII-pNF questionnaire for parents.

Design:

* This protocol will be a multi-institutional research study to maximize our ability to assess a large, diverse sample of individuals with NF1.
* This study will consist of 2 phases. During the first phase, we conducted the qualitative portion of the study with individuals with NF1, ages \>=5 years, most with pNF-related pain, using both focus groups and individual interviews. During Phase 2, we will evaluate the final electronic versions of the PAINS-pNF and the PII-pNF using a microlongitudinal design to examine internal consistency, construct validity, and test-retest reliability, and to provide normative data on the study measures for the NF1 population.
* Our goal for phase 2 is to recruit between 14 to 16 patients, with a target of 15 in each of the eight phase 2 age bands for an approximate ceiling of 128 patients \>=8 years old (target = 120) and 48 parents of children 8-17 years old (target = 45). All patients and parents will be asked to complete the measures two weeks in a row to examine test-retest reliability.

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)

.All IPD recorded in the medical record will be shared with intramural investigators upon request.

To evaluate final versions of the NRS-11, PII, and PROMIS-PF measures on reliability, validity, sensitivity to change, and feasibility in individuals with NF1 and PNs.

To evaluate current modifications and the need for any additional modifications to existing measures of pain intensity (Numeric Rating Scale; NRS-11) and pain interference (Pain Interference Index; PII) and select the most appropriate items to measure physical functioning (PROMIS Physical Functioning; PROMIS-PF) in NF1 based on qualitative feedback from patients with NF1, PNs and pain to use as endpoints in clinical trials for individuals with NF1 and PNs. (completed)

To provide normative data on these measures of pain intensity and pain interference in individuals with NF1 and pNFs

Convert the measures (including any changes) into an electronic format.

National Cancer Institute (NCI)

Digestive System Diseases

Colorectal Neoplasms

Intestinal Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Colonic Diseases

Intestinal Diseases

Rectal Diseases

We anticipate enrolling up to a total of 18 patients who have been diagnosed with metastatic colorectal cancer; 12 patients will participate in the qualitative interview, and 6 patients will participate in the cognitive interview.

We anticipate enrolling up to a total of 18 partners of patients who have been diagnosed with metastatic colorectal cancer; 12 partners will participate in the qualitative interview, and 6 paartners will participate in the cognitive interview.

We anticipate enrolling 12 clinicians (including physicians, nurse practitioners, physician assistants, mental health providers, other advanced practice clinicians) who treat patients with colorectal cancer at the University of Colorado to participate in qualitative interviews. We seek to enroll a variety of clinicians from advanced practiced clinical roles (physicians, nurse practitioners, and mental health providers) to capture diverse perspectives.

Qualitative interviews will cover four main goals including: (1) key sources of distress in mCRC patients and partners; (2) perceptions of mindfulness, including benefits, definitions, and applications; (3) barriers and facilitators of participation (e.g. home-based and couple-based skills practice, enrollment); and (4) session format preferences, such as session length and frequency.

Qualitative interview

The objective of the cognitive interviews (60 minutes, led by the PI) is to ensure the comprehensibility and appropriateness of the drafted participant materials. Published guidelines note a sample size of 12 participants (6 mCRC couples) should be sufficient. Participants will be asked to read, review, and mark up the participant materials in order to identify sections that were difficult to understand. They will also be asked to test playing segments of the audio/video recordings to ensure ease of use. A semi-structured interview format will be used to elicit feedback, including a standard verbal survey assessing ease of reading the material, any difficulties experienced, and suggestions they have for improving the ease of material use. Information generated will be used to update materials as necessary.

Cognitive Interviews

Joshua Pasek

Lauren Zimmaro, PhD

Chris Lieu, MD

Jennifer Reese, MD

Jamie Studts, PhD

Lauren Zimmaro

The overall objective of this study is to develop a novel couple-based mindfulness intervention aimed at reducing distress in patients with metastatic colorectal cancer (mCRC) and their partners.

The overall objective of this study is to develop a novel couple-based mindfulness intervention aimed at reducing distress in patients with metastatic colorectal cancer (mCRC) and their partners. The intervention is named "MIND-Together" (Mindfully Navigating Distress Together). MIND-Together will be the first mindfulness-based intervention designed to reduce mCRC couples' distress by targeting their unique psychosocial needs. The intervention is projected to be a 4-session intervention delivered by a trained therapist to individual couples facing mCRC (i.e., a patient and his/her intimate partner) via video-conference (e.g., Zoom).

Couple-Based Mindfulness Intervention for Metastatic Colorectal Cancer

Developing a Couple-Based Mindfulness Intervention for Metastatic Colorectal Cancer: Qualitative Phase

Completed through semi-structured qualitative interviews

analyze these qualitative data to extract key themes and will integrate the findings with preliminary drafts of the MIND-Together protocol (e.g., participant handouts, therapist manual).

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)	NCT02544022	Neurofibromatos... Plexiform Neuro...		5 Years -	National Institutes of Health Clinical Center (CC)
Couple-Based Mindfulness Intervention for Metastatic Colorectal Cancer	NCT05840263	Metastatic Colo...	Qualitative int... Cognitive Inter...	18 Years - 85 Years	University of Colorado, Denver
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)	NCT02544022	Neurofibromatos... Plexiform Neuro...		5 Years -	National Institutes of Health Clinical Center (CC)
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)	NCT02544022	Neurofibromatos... Plexiform Neuro...		5 Years -	National Institutes of Health Clinical Center (CC)
Couple-Based Mindfulness Intervention for Metastatic Colorectal Cancer	NCT05840263	Metastatic Colo...	Qualitative int... Cognitive Inter...	18 Years - 85 Years	University of Colorado, Denver

Spots Global Cancer Trial Database for cognitive interviews