Neurofibromatosis 1 (NF1)NCT07869914
Cognitive Intervention for Children and Adolescents With NF1
A study testing Unstuck and On Target! in people with Neurofibromatosis 1 (NF1) and Executive Function (Cognition), run by The Hospital for Sick Children.
Trial summary
Psychological interventions have been shown to be effective for managing cognitive and mental health challenges in different patient populations. The investigators are testing whether such a problem-solving skills program is effective and beneficial for children and teens with NF1 when implemented in a real-world… Read more about this study
- Who is it for?
- Ages 8 to 17 years
- Conditions the study is for
- Neurofibromatosis 1 (NF1), Executive Function (Cognition), Cognitive Intervention
- What is being tested?
- Unstuck and On Target!
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- 28 Sep 2026to Jun 2029
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 8 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Receive subject-informed consent;
- Youth with a diagnosis of NF1 and their parents;
- Youth aged 8-17 years old (inclusive) and their parents
Who cannot join
- Conditions that significantly affect cognition (e.g., intellectual disability diagnosis, active psychosis), hearing loss or vision problems precluding participation in group
- Non-English speaking
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Feasibility Pilot of a Cognitive Intervention for Children and Adolescents With Neurofibromatosis, Type 1
Psychological interventions have been shown to be effective for managing cognitive and mental health challenges in different patient populations. The investigators are testing whether such a problem-solving skills program is effective and beneficial for children and teens with NF1 when implemented in a real-world setting. Unstuck and On Target! (UOT) is a comprehensive manualized intervention program that targets executive functioning (EF), self-determination, autonomy, quality of life, and mental health, as well as impact on parental stress. It has demonstrated effectiveness at improving EF in youth with autism. The investigators are assessing the effectiveness of the program in youth with NF1 and adapting the program for NF1, as needed, based on participant feedback.
- Being tested
- Unstuck and On Target!
- Keywords
- NF1, cognitive intervention, psychological intervention, executive functioning, quality of life, children and adolescents with NF1, youth with NF1, parental stress, problem-solving skills, mental health, unstuck and on target, UOT, EF, self-determination, autonomy
- Medical subject headings
- Neurofibromatosis 1, Psychological Well-Being
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Unstuck and On Target!Behavioural
Unstuck and On Target! (UOT) is an evidence-based executive functioning (EF) intervention that places an emphasis on "real world" application of acquired skills.
Participants can participate in-person or online, and either an in-person or online group format will then be offered. As such, those who are not able to participate in the current iteration of the group will be placed on a waitlist. Screened participants who are willing to participate in the current format will be randomized into those receiving treatment immediately or in 6 months (waitlist control group).
Both parent and youth sessions will be 50- to 60-minutes long and will be administered in-person or over Zoom.
Sessions will also assess participant engagement in the program and learning of program content using "Unstuck Vocabulary" tasks. These measure participant knowledge and use of words used in the program to describe specific principles or concepts. Fidelity will also be measured.
| Study group | Role | What they receive |
|---|---|---|
| UOT program | Experimental | Virtual or in-person weekly group sessions (~50 minutes each) over a 13-week period. Receives: Behavioral: Unstuck and On Target! |
| UOT waitlist control group | Experimental | Waitlist control group. The same intervention program will be administered for this group once they are off the waitlist. Receives: Behavioral: Unstuck and On Target! |
What is being measured
The primary outcome is the main question the study is designed to answer.
Developing and assessing preliminary feasibility and acceptability of an intervention tailored to support NF1 patients
Protocol feasibility will be assessed by consent/recruitment, attendance, and completion rates. Intervention group participants will rate their satisfaction with aspects of the program on a brief survey using 5-point Likert scales (ranging from "dissatisfied" to "extremely satisfied"). Semi-structured interviews administered to participants post-intervention will assess program feasibility and acceptability.
Measured: From enrollment to the end of treatment at 13 weeks
Secondary outcomes (6)
Assessing the intervention's impact on parent stress
Parental stress will be assessed using a 5-point Likert scale, via the Parenting Stress Index (PSI-4), ranging from "Strongly Agree" to "Strongly Disagree", to measure the magnitude of stress in parent-child relationships.
Measured: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's executive functioning
Executive Functioning will be assessed using a 3-point Likert scale ("Never", "Sometimes", "Often"), via the Behavior Rating Inventory of Executive Function (BRIEF-2), to measure everyday executive functioning in children and adolescents.
Measured: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's self-determination
Child's self-determination will be assessed using a 5-point Likert scale (ranging from "Never" to "Always"), via the AIR Self-Determination questionnaire.
Measured: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's autonomy
Child autonomy will be assessed using a 4-point Likert scale (ranging from "Never" to "Almost Always"), via the Adaptive Behavior Assessment System (ABAS-3) questionnaire, which measures everyday functional skills and adaptive behavior.
Measured: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's quality of life
Quality of life will be assessed using a 5-point Likert scale via the World Health Organization - Brief (WHOQOL-BREF) questionnaire.
Measured: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's mental health
Child's mental health will be assessed using a 5-point Likert scale (ranging from "Never" to "Almost Always"), via the PROMIS (Patient-Reported Outcomes Measurement Information System) depression and anxiety questionnaires.
Measured: From enrollment to the end of treatment at 13 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2026
- Study start28 Sep 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionJun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis 1 (NF1)Studies of the same condition, open and under way.
- 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Model description
- * The intervention will be administered, virtually or in-person, by a trained graduate student or postdoctoral fellow in Clinical (Neuro)Psychology, under the supervision of a Clinical Neuropsychologist, to a group of 6-8 NF1 patients at a time (ages 8-17) and their parents. * Participants will complete pre- and post-treatment questionnaires, follow-up questionnaires (6 months post-intervention), as well as a post-treatment interview (shortly after the end of the intervention) to assess social communication skills and parent executive functioning skills, as well as intervention impact on parent stress and children's EF, self-determination, autonomy, quality of life, and mental health. * This will be a waitlist control design study. Participants in the waitlist control group will have an additional data collection time point, 6 months before they begin the intervention. At post-treatment, participants will rate their satisfaction with the program.
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Busi Zapparoli, The Hospital for Sick Children
- Investigators
- Busi Zapparoli, Ph.D., C.Psych., Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07869914
- Sponsor's protocol ID
- 4406
- Status last verified
- Oct 2026
- First submitted
- 21 Sep 2026
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility Pilot of a Cognitive Intervention for Children and Adolescents With Neurofibromatosis, Type 1
- ClinicalTrials.gov ID
- NCT07869914
- Acronym / sponsor ID
- 4406
- Keywords
- NF1, cognitive intervention, psychological intervention, executive functioning, quality of life, children and adolescents with NF1, youth with NF1, parental stress
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Study start
- 28 Sep 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Jun 2029 (estimated)
- First posted
- 13 Oct 2026 (actual)
- 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.
Psychological interventions have been shown to be effective for managing cognitive and mental health challenges in different patient populations. The investigators are testing whether such a problem-solving skills program is effective and beneficial for children and teens with NF1 when implemented in a real-world setting. Unstuck and On Target! (UOT) is a comprehensive manualized intervention program that targets executive functioning (EF), self-determination, autonomy, quality of life, and mental health, as well as impact on parental stress. It has demonstrated effectiveness at improving EF in youth with autism. The investigators are assessing the effectiveness of the program in youth with NF1 and adapting the program for NF1, as needed, based on participant feedback.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- * The intervention will be administered, virtually or in-person, by a trained graduate student or postdoctoral fellow in Clinical (Neuro)Psychology, under the supervision of a Clinical Neuropsychologist, to a group of 6-8 NF1 patients at a time (ages 8-17) and their parents. * Participants will complete pre- and post-treatment questionnaires, follow-up questionnaires (6 months post-intervention), as well as a post-treatment interview (shortly after the end of the intervention) to assess social communication skills and parent executive functioning skills, as well as intervention impact on parent stress and children's EF, self-determination, autonomy, quality of life, and mental health. * This will be a waitlist control design study. Participants in the waitlist control group will have an additional data collection time point, 6 months before they begin the intervention. At post-treatment, participants will rate their satisfaction with the program.
| Arm | Type | Receives |
|---|---|---|
| UOT program | Experimental | Behavioral: Unstuck and On Target!Virtual or in-person weekly group sessions (~50 minutes each) over a 13-week period. |
| UOT waitlist control group | Experimental | Behavioral: Unstuck and On Target!Waitlist control group. The same intervention program will be administered for this group once they are off the waitlist. |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Receive subject-informed consent;
- Youth with a diagnosis of NF1 and their parents;
- Youth aged 8-17 years old (inclusive) and their parents
Key exclusion criteria (2 listed)
- Conditions that significantly affect cognition (e.g., intellectual disability diagnosis, active psychosis), hearing loss or vision problems precluding participation in group
- Non-English speaking
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 |
|---|---|---|
| Unstuck and On Target!Unstuck and On Target! (UOT) is an evidence-based executive functioning (EF) intervention that places an emphasis on "real world" application of acquired skills. Participants can participate in-person or online, and either an in-person or online group format will then be offered. As such, those who are not able to participate in the current iteration of the group will be placed on a waitlist. Screened participants who are willing to participate in the current format will be randomized into those receiving treatment immediately or in 6 months (waitlist control group). Both parent and youth sessions will be 50- to 60-minutes long and will be administered in-person or over Zoom. Sessions will also assess participant engagement in the program and learning of program content using "Unstuck Vocabulary" tasks. These measure participant knowledge and use of words used in the program to describe specific principles or concepts. Fidelity will also be measured. | Behavioural | UOT program, UOT waitlist control group |
Primary outcome
Developing and assessing preliminary feasibility and acceptability of an intervention tailored to support NF1 patients
Time frame: From enrollment to the end of treatment at 13 weeks
Protocol feasibility will be assessed by consent/recruitment, attendance, and completion rates. Intervention group participants will rate their satisfaction with aspects of the program on a brief survey using 5-point Likert scales (ranging from "dissatisfied" to "extremely satisfied"). Semi-structured interviews administered to participants post-intervention will assess program feasibility and acceptability.
6 secondary outcomes
Assessing the intervention's impact on parent stress
Time frame: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's executive functioning
Time frame: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's self-determination
Time frame: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's autonomy
Time frame: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's quality of life
Time frame: From enrollment to the end of treatment at 13 weeks
Assessing the intervention's impact on children's mental health
Time frame: From enrollment to the end of treatment at 13 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Neurofibromatosis 1 (NF1)
RecruitingObservational study
Observational Study of the Real-world Effectiveness and Safety of Selumetinib Treating Adult Patients With Neurofibromatosis Type 1 (NF1) Who Have Symptomatic, Inoperable Plexiform Neurofibroma (PN)
Neurofibromatosis Type 1 (NF1)
Active, not recruitingPhase 1/2
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Neurofibromatosis 1, Neurofibromatosis Type 1
Active, not recruitingPhase 2
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
Not yet recruitingPhase 2
Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma
Neurofibromatosis 1 (NF1)
Active, not recruitingPhase 2
MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
Neurofibromatosis 1 (NF1), Plexiform Neurofibromas (PN)
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.
Busi Zapparoli, Ph.D., C.Psych. principal investigator
The Hospital for Sick Children
Questions about this trial
What is this clinical trial studying?
A study testing Unstuck and On Target! in people with Neurofibromatosis 1 (NF1) and Executive Function (Cognition), run by The Hospital for Sick Children.
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 3 inclusion and 2 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: opening soon, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tima Al-Rubeye, B.Sc. (416-813-7654 ext. 415468, tima.al-rubeye@sickkids.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Developing and assessing preliminary feasibility and acceptability of an intervention tailored to support NF1 patients (time frame: From enrollment to the end of treatment at 13 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Oct 2026; AzTrials last refreshed its copy on 26 Oct 2026.