NeurofibromatosisEarly Phase 1NCT01633008
Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain
A Early Phase 1 study testing ACT Workshop in people with Neurofibromatosis, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: Neurofibromatosis type 1 (NF1) is a genetic disorder that can cause frequent and significant pain. This pain can have a severe impact on a person s quality of life. Acceptance and Commitment Therapy (ACT) is a new type of therapy that may help people manage pain. Read more about this study
- Who is it for?
- Ages 12 to 99 yearsHealthy volunteers can take part
- Conditions the study is for
- Neurofibromatosis
- What is being tested?
- ACT Workshop
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jun 2012to 10 Jan 2017
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 99 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Subjects must be between 12 and 21 years of age at the time of the intervention. Because the research on the effectiveness of ACT with children younger than 12 is still emerging, children 11 and younger will be excluded from the present study.
- For the clinical diagnosis of NF1 all study subjects must have two or more diagnostic criteria for NF1 listed below (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 postpuberta subjects)
- Greater than or equal to 2 neurofibromas or 1 plexiform neurofibroma
- Freckling in the axilla or groin
- Optic glioma
Show all 18Show fewer
- 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
- Response of 3 or higher on a pain item (1 5 scale) from a self-report quality of life measure (Impact of Pediatric Illness Scale) assessing the extent to which pain has interfered with the patient s daily functioning over the past month.
- Ability of subject or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.
- Subjects may be participating in other medical interventions for the treatment of their disease, or standard care techniques for pain management.
- No anticipated major changes in their pain regimen or enrollment on a new treatment study in the near future.
- Subjects must be able to read and comprehend the English language.
- Parents must have a child with NF1 who is participating in this protocol as a patient.
- Ability to read and comprehend the English language.
- Ability to understand and the willingness to sign a written informed consent document.
- If a minor patient meets all eligibility criteria but his or her parent does not (i.e., in the case where a parent does not speak English), the patient will not be allowed to participate.
Who cannot join
- In the opinion of the PI or an 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.
- Subjects who are participating in any other treatment studies, either medical or behavioral, for pain management.
- Subjects who are scheduled to begin a new drug protocol for the treatment of their NF1 during the time of their enrollment on the current study.
- Inability to travel to the NIH, for example, due to physical limitations, for the scheduled workshop and evaluations.
- No groups in regards to gender, race, or ethnicity are being excluded from participation in the trial.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Acceptance and Commitment Therapy for Adolescents and Adults With Neurofibromatosis Type 1 and Chronic Pain: A Pilot Study
Background:
- Neurofibromatosis type 1 (NF1) is a genetic disorder that can cause frequent and significant pain. This pain can have a severe impact on a person s quality of life.
- Acceptance and Commitment Therapy (ACT) is a new type of therapy that may help people manage pain. It teaches behavioral techniques that can be used to change how people react to pain. ACT may help people with NF1 cope better with the pain associated with the disorder. Researchers want to test ACT techniques to see if they can improve coping in people with NF1 pain.
Read more
Objectives:
- To see if ACT can help treat chronic pain in adolescents and young adults who have NF1.
Eligibility:
- Adolescents and young adults between 12 and 21 years of age who have chronic pain associated with NF1.
Design:
- Participants will be screened with a physical exam and medical history. They will also complete a set of questionnaires about their pain levels, quality of life, and medications that they are taking.
- Participants will take part in a training workshop for ACT. It will consist of three 2-hour sessions over 2 consecutive days. The workshop will teach techniques for setting goals that reflect personal values, and changing the focus from pain avoidance to pain management. There will be practice exercises between each session.
- At the end of the workshop, participants will receive a workbook with the exercises to continue to practice at home. Participants will be encouraged to spend at least 20 minutes, three times per week, on these practice exercises.
- There will be a followup phone call to check on pain management after the workshop.
- Three months after the first study visit, participants will complete the study questionnaires again from home. They will provide more information about how they are managing their pain. The questionnaires will be returned by mail in an envelope provided by the study team.
Read the 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, scoliosis, and chronic headaches, 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, no studies have examined the effectiveness of behavioral interventions for chronic pain in individuals 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 or distress.
- In helping individuals with pain, the goal of ACT is not to eliminate the person s pain, but to optimize the person s quality of life despite their pain.
Objectives:
- To evaluate whether the Acceptance and Commitment Therapy (ACT) group demonstrates improvement on the Modified Brief Pain Inventory scale from baseline to 3 months in adolescents and young adults with NF1 and chronic pain.
- To examine changes in patient s functional ability, quality of life, pain-related coping, pain-related anxiety, pain severity, and depression from baseline to 3 months
- To examine the effects of ACT on parents psychological well-being, overall stress, and ability to cope with their child s pain
- To explore the relationship between changes in patients pain interference, functional ability, pain-related coping, pain-related anxiety, pain severity, depression and treatment adherence with demographic variables, parent stress, and parent coping
- To collect preliminary data on the value of ACT to guide the potential development of a future phase II trial
Eligibility:
Patients ages 12 to 21 with a confirmed diagnosis of NF1.-Parents of participating patients.-The patient must indicate a score of 3 or higher on a pain item (1 5 scale) from a quality of life measure assessing the extent to which pain has interfered with the patient s daily functioning over the past month.
Design:
- This is a behavioral study to determine the feasibility and potential benefit of an ACT intervention on pain interference from baseline to 3 months post-intervention.
- A sample size of 12 patients is needed to provide 90% power for detecting a change of .77 standard deviations on the total pain interference score of the M-BPI using a 1-sided .1 level test.
- ACT workshops will be scheduled multiple times over approximately five months and will be comprised of three 2-hour sessions held over two consecutive days
- Measures assessing pain interference, pain coping, pain-related anxiety, quality of life, functional disability, depression, and pain severity will be administered to patients prior to the ACT intervention and 3 months later.
- At the same time points, a parent or legal guardian of each patient will complete measures assessing their child s quality of life, functional disability, depression, and pain severity, as well as the parent s own psychological functioning and ability to cope with their child s pain
- Conditions
- Neurofibromatosis
- Being tested
- ACT Workshop
- Keywords
- Modified Brief Pain Inventory, Quality of Life, Pain-Related Coping, Pain-Related Anxiety, Depression
- Medical subject headings
- Neurofibromatoses, Depression
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ACT WorkshopBehavioural
The ACT workshops will consist of a patient program and a corresponding parent program, each focusing on helping participants cope with the patient s pain more effectively using ACT techniques
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Attend three 2-hour sessions held over two consecutive days Receives: Behavioral: ACT Workshop |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pain improvement
Measured: 3 months
Secondary outcomes (4)
Value of the ACT for future Ph2 trial.
Measured: 6 months
Psychological well-being & stress changes in parents
Measured: 3 months
Explore improvements of pain interference, abilities, coping, anxiety,depression, etc
Measured: 3 months
Relationship between pain and treatment adherence
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2012 (estimated)
- Study start15 Jun 2012
- Primary completion1 May 2014
- Study completion10 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for NeurofibromatosisStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Staci M Peron, Ph.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01633008
- Sponsor's protocol ID
- 120155
- Other IDs
- 12-C-0155
- Status last verified
- 10 Jan 2017
- First submitted
- 29 Jun 2012
- Last updated
- 30 Sep 2019
- Results posted
- No
Publications
Babovic-Vuksanovic D, Widemann BC, Dombi E, Gillespie A, Wolters PL, Toledo-Tamula MA, O'Neill BP, Fox E, MacDonald T, Beck H, Packer RJ. Phase I trial of pirfenidone in children with neurofibromatosis 1 and plexiform neurofibromas. Pediatr Neurol. 2007 May;36(5):293-300. doi: 10.1016/j.pediatrneurol.2007.01.009.
PubMed 17509460Bach P, Hayes SC. The use of acceptance and commitment therapy to prevent the rehospitalization of psychotic patients: a randomized controlled trial. J Consult Clin Psychol. 2002 Oct;70(5):1129-39. doi: 10.1037//0022-006x.70.5.1129.
PubMed 12362963Blount RL, Cohen LL, Frank NC, Bachanas PJ, Smith AJ, Manimala MR, Pate JT. The Child-Adult Medical Procedure Interaction Scale-Revised: an assessment of validity. J Pediatr Psychol. 1997 Feb;22(1):73-88. doi: 10.1093/jpepsy/22.1.73.
PubMed 9019049
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Acceptance and Commitment Therapy for Adolescents and Adults With Neurofibromatosis Type 1 and Chronic Pain: A Pilot Study
- ClinicalTrials.gov ID
- NCT01633008
- Acronym / sponsor ID
- 120155
- Conditions
- Neurofibromatosis
- Keywords
- Modified Brief Pain Inventory, Quality of Life, Pain-Related Coping, Pain-Related Anxiety, Depression
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 15 Jun 2012
- Primary completion
- 1 May 2014 (actual)
- Study completion
- 10 Jan 2017 (actual)
- First posted
- 4 Jul 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
- Neurofibromatosis type 1 (NF1) is a genetic disorder that can cause frequent and significant pain. This pain can have a severe impact on a person s quality of life.
- Acceptance and Commitment Therapy (ACT) is a new type of therapy that may help people manage pain. It teaches behavioral techniques that can be used to change how people react to pain. ACT may help people with NF1 cope better with the pain associated with the disorder. Researchers want to test ACT techniques to see if they can improve coping in people with NF1 pain.
Objectives:
- To see if ACT can help treat chronic pain in adolescents and young adults who have NF1.
Eligibility:
- Adolescents and young adults between 12 and 21 years of age who have chronic pain associated with NF1.
Design:
- Participants will be screened with a physical exam and medical history. They will also complete a set of questionnaires about their pain levels, quality of life, and medications that they are taking.
- Participants will take part in a training workshop for ACT. It will consist of three 2-hour sessions over 2 consecutive days. The workshop will teach techniques for setting goals that reflect personal values, and changing the focus from pain avoidance to pain management. There will be practice exercises between each session.
- At the end of the workshop, participants will receive a workbook with the exercises to continue to practice at home. Participants will be encouraged to spend at least 20 minutes, three times per week, on these practice exercises.
- There will be a followup phone call to check on pain management after the workshop.
- Three months after the first study visit, participants will complete the study questionnaires again from home. They will provide more information about how they are managing their pain. The questionnaires will be returned by mail in an envelope provided by the study team.
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, scoliosis, and chronic headaches, 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, no studies have examined the effectiveness of behavioral interventions for chronic pain in individuals 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 or distress.
- In helping individuals with pain, the goal of ACT is not to eliminate the person s pain, but to optimize the person s quality of life despite their pain.
Objectives:
- To evaluate whether the Acceptance and Commitment Therapy (ACT) group demonstrates improvement on the Modified Brief Pain Inventory scale from baseline to 3 months in adolescents and young adults with NF1 and chronic pain.
- To examine changes in patient s functional ability, quality of life, pain-related coping, pain-related anxiety, pain severity, and depression from baseline to 3 months
- To examine the effects of ACT on parents psychological well-being, overall stress, and ability to cope with their child s pain
- To explore the relationship between changes in patients pain interference, functional ability, pain-related coping, pain-related anxiety, pain severity, depression and treatment adherence with demographic variables, parent stress, and parent coping
- To collect preliminary data on the value of ACT to guide the potential development of a future phase II trial
Eligibility:
Patients ages 12 to 21 with a confirmed diagnosis of NF1.-Parents of participating patients.-The patient must indicate a score of 3 or higher on a pain item (1 5 scale) from a quality of life measure assessing the extent to which pain has interfered with the patient s daily functioning over the past month.
Design:
- This is a behavioral study to determine the feasibility and potential benefit of an ACT intervention on pain interference from baseline to 3 months post-intervention.
- A sample size of 12 patients is needed to provide 90% power for detecting a change of .77 standard deviations on the total pain interference score of the M-BPI using a 1-sided .1 level test.
- ACT workshops will be scheduled multiple times over approximately five months and will be comprised of three 2-hour sessions held over two consecutive days
- Measures assessing pain interference, pain coping, pain-related anxiety, quality of life, functional disability, depression, and pain severity will be administered to patients prior to the ACT intervention and 3 months later.
- At the same time points, a parent or legal guardian of each patient will complete measures assessing their child s quality of life, functional disability, depression, and pain severity, as well as the parent s own psychological functioning and ability to cope with their child s pain
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Behavioral: ACT WorkshopAttend three 2-hour sessions held over two consecutive days |
Who may qualify
- Ages
- 12 to 99 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (18 listed)
- Subjects must be between 12 and 21 years of age at the time of the intervention. Because the research on the effectiveness of ACT with children younger than 12 is still emerging, children 11 and younger will be excluded from the present study.
- For the clinical diagnosis of NF1 all study subjects must have two or more diagnostic criteria for NF1 listed below (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 postpuberta subjects)
- Greater than or equal to 2 neurofibromas or 1 plexiform neurofibroma
- Freckling in the axilla or groin
Key exclusion criteria (5 listed)
- In the opinion of the PI or an 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.
- Subjects who are participating in any other treatment studies, either medical or behavioral, for pain management.
- Subjects who are scheduled to begin a new drug protocol for the treatment of their NF1 during the time of their enrollment on the current study.
- Inability to travel to the NIH, for example, due to physical limitations, for the scheduled workshop and evaluations.
- No groups in regards to gender, race, or ethnicity are being excluded from participation in the trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ACT WorkshopThe ACT workshops will consist of a patient program and a corresponding parent program, each focusing on helping participants cope with the patient s pain more effectively using ACT techniques | Behavioural | 1 |
Primary outcome
Pain improvement
Time frame: 3 months
4 secondary outcomes
Value of the ACT for future Ph2 trial.
Time frame: 6 months
Psychological well-being & stress changes in parents
Time frame: 3 months
Explore improvements of pain interference, abilities, coping, anxiety,depression, etc
Time frame: 3 months
Relationship between pain and treatment adherence
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2019.
- Background: PubMed 17509460Babovic-Vuksanovic D, Widemann BC, Dombi E, Gillespie A, Wolters PL, Toledo-Tamula MA, O'Neill BP, Fox E, MacDonald T, Beck H, Packer RJ. Phase I trial of pirfenidone in children with neurofibromatosis 1 and plexiform neurofibromas. Pediatr Neurol. 2007 May;36(5):293-300. doi: 10.1016/j.pediatrneurol.2007.01.009.
- Background: PubMed 12362963Bach P, Hayes SC. The use of acceptance and commitment therapy to prevent the rehospitalization of psychotic patients: a randomized controlled trial. J Consult Clin Psychol. 2002 Oct;70(5):1129-39. doi: 10.1037//0022-006x.70.5.1129.
- Background: PubMed 9019049Blount RL, Cohen LL, Frank NC, Bachanas PJ, Smith AJ, Manimala MR, Pate JT. The Child-Adult Medical Procedure Interaction Scale-Revised: an assessment of validity. J Pediatr Psychol. 1997 Feb;22(1):73-88. doi: 10.1093/jpepsy/22.1.73.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Staci M Peron, Ph.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing ACT Workshop in people with Neurofibromatosis, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pain improvement (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01633008, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2019.