NF1 Clinical Trials

Neurofibromatosis type 1 studies recruiting patients for novel treatments. Filter by phase, distance, and inclusion criteria to find your perfect nf1 clinical trial.

Condition

Location

Distance

Age

Clinical Trials NF1 Clinical Trials

Phase-Based Estimates

OPTIMAL-SSIfor Neurofibromatoses

Children with neurofibromatosis are more likely to have difficulties related to their psychological and neurocognitive functioning (e.g., more likely to have depression, have social difficulties, be diagnosed with ADHD). The purpose of this randomized control study is to determine how effective and useful this study's single session intervention can be in improving psychological and neurocognitive functioning. Enrolled families will consist of one parent/guardian and child. Parents and patients will complete questionnaires and objective tests at baseline, 3 months, and 6 months. Families randomized to the intervention arm will be provided with one single session intervention at Month 1 to learn about their child's testing results and receive psychoeducation and recommendations related to psychological and neurocognitive functioning.

Phase-Based Estimates

ACT Interventionfor Neurofibromatoses

Waitlist Available

Background: RASopathies are a group of genetic diseases that affect a child s development. They cause physical, cognitive, and behavioral symptoms. Caring for a child with a RASopathy can be stressful. Acceptance and Commitment Therapy (ACT) is a therapy that helps people become more aware and accepting of difficult thoughts and feelings. ACT has been found to be helpful for parents with high parenting stress. Objective: To find out if Acceptance and Commitment Therapy (ACT) can help caregivers of children with a RASopathy better cope with parenting stress. Eligibility: People aged 18 years or older who care for a child (younger than 18 years) with a RASopathy. The child must live with the caregiver at least 50% of the time. Design: The study is fully remote. Participants need a mobile device that can play audio and video and connect to the internet. They can borrow an iPod if needed. Participants will download a free app called MetricWire. They will use this app to watch videos and answer questions. The first 8 participants will be in a pilot study. They will receive the ACT intervention starting the first week after they begin the study. After the pilot study, we will start a new phase called the randomized trial. In this phase, participants will have a 50-50 chance of being in the group that will start the intervention right away or the group that will start the intervention after about 2 months. Participants will fill out surveys on 5 random days each week. These surveys have 7 questions and take about 2 minutes. They will also fill out 3 longer questionnaires: once before ACT begins, once just after the 8-week study period, and once about 3 months later. Questions will cover topics including: Parenting stress Life satisfaction Self-compassion Uncomfortable feelings and thoughts Mindfulness Participants will take part in an 8-week ACT intervention. They will have one 75-minute session with an ACT coach in the first week. Participants will watch 9- to 17-minute videos each week. The videos talk about how to practice ACT techniques to cope with parenting stress. Participants will have 20- to 30-minute coaching sessions in weeks 3 and 6. The coach will help them practice exercises and work through any problems.

Phase-Based Estimates

Selumetinib Granule Formulationfor Neurofibromatosis Type 1 (NF1)

Philadelphia, PA

Philadelphia, PA

This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints. The study will inform the benefit risk profile of the granule formulation in children aged ≥ 1 to < 7 years with NF1 related symptomatic, inoperable PN.

Phase-Based Estimates

Selumetinibfor Neurofibroma

Waitlist Available

This study is evaluating whether a low fat meal can be used to reduce the amount of selumetinib needed to treat NF1.

Phase-Based Estimates

FCN-159for Watson Syndrome

Locally Advanced

FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib. FCN-159 is an orally available and highly potent selective inhibitor of MEK1/2, which is expected to be a targeted therapy for the treatment of advanced solid tumors and neurofibromatosis type 1.

Phase-Based Estimates

Selumetinibfor Watson Syndrome

Toronto, Canada

Waitlist Available

Toronto, Canada

This study is evaluating whether selumetinib is effective in treating people with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas.

Phase-Based Estimates

PEERSfor Watson Syndrome

This study is evaluating whether a program which helps teens with neurofibromatosis type 1 make friends may help improve their social skills.

Phase-Based Estimates

Whole-body Magnetic Resonance Imagingfor Watson Syndrome

Los Angeles, CA

Los Angeles, CA

This study is evaluating whether a new type of MRI scan may help detect neurofibromatosis type 1.

Phase-Based Estimates

Abemaciclibfor Watson Syndrome

Background: NF1 is a genetic disease that causes tumors called atypical neurofibromas. These tumors, which arise from nerves, can cause serious medical problems. The only treatment is surgery. Researchers want to see if a drug called abemaciclib can help. Objective: To find a safe, tolerable dose of abemaciclib for treating atypical neurofibromas. Eligibility: People ages 12 and older who have NF1 and have one or more atypical neurofibromas that cannot or will not be removed with surgery Design: Participants will be screened with: Medical history and physical exam Blood, urine, and heart tests MRI: Participants will lie in a machine that takes pictures of the body. A padding or coil will be placed around their head. They may have a contrast agent injected into a vein. Biopsy sample: A small piece of tumor will be removed using a large needle. Participants will have frequent visits during the study. These will include repeats of the screening tests as well as the following: PET scan: Participants will lie in a machine that takes pictures of the body. They will have a contrast agent injected into their arm. Questionnaires about the effects of abemaciclib on pain and quality of life Possible photographs of tumors Participants will take abemaciclib capsules orally twice daily in 28-day cycles. They will take the drug for up to 2 years. Some may be able to take it for longer. Participants will have a follow-up visit about 30 days after their last dose of the study drug. Then they will have visits every 3 months for 1 year.

Phase-Based Estimates

ICanCopefor Watson Syndrome

This study is evaluating whether a new program may help improve pain and other symptoms for individuals with chronic pain.

Phase-Based Estimates

N-Acetyl Cysteinefor Neurofibroma

Locally Advanced

This study is evaluating whether N-acetylcysteine may have positive impacts for individuals with neurofibromatosis type 1.

Phase-Based Estimates

Trametinib Dimethyl Sulfoxidefor Multiple Myeloma

Philadelphia, PA

Waitlist Available

Philadelphia, PA

This study is evaluating whether trametinib can shrink a type of cancer that has a genetic change called NF1 mutation.

Phase-Based Estimates

Mirdametinib (PD-0325901) Oral Capsule Or Dispersible Tabletfor Neurofibroma

Waitlist Available

This study is evaluating whether a drug may help treat neurofibromatosis type 1.

Phase-Based Estimates

Stress And Symptom Management Program 2for Neurofibromatoses

This study is evaluating whether a specific type of therapy may help improve the mental health of adolescents with neurofibromatosis.

Phase-Based Estimates

Whole Body MRIfor Neurofibromatoses

Washington, United States

Waitlist Available

Washington, United States

This study is evaluating whether whole body MRI can be used to identify atypical neurofibromas in neurofibromatosis type 1 patients with high tumor burden.

Phase-Based Estimates

Medication Event Monitoring System (MEMS)for Watson Syndrome

Background: Neurofibromatosis type 1 (NF1) is a genetic disorder. It has a broad variety of effects on the body. Up to half of people with NF1 get plexiform neurofibromas (PNs). These are benign tumors. But they can have serious effects like pain and disfigurement. To treat PNs, a person may have to take medicine every day for a long period of time. Researchers think that it will be important for people to take the medicine regularly for it to work. They want to study how well people with NF1 follow their treatment plan for PNs. Objective: To study how often people with neurofibromatosis type 1 take medicine that has been prescribed to them for treating plexiform neurofibromas. Eligibility: People ages 3-59 already enrolled in an NF1 clinical trial Design: Participants will need access to the internet to do the study activities. Parents or caregivers will do some study activities for child participants. Participants will complete 5 questionnaires. They will take about 20 minutes total. The topics will be: Demographic data Recent life events How much pain interferes with daily life Ability to focus and pay attention to tasks Emotional distress or depression Participants will mark down every time they take a dose of the medicine in their clinical trial. They will use a form the researchers give them. The pill bottles they get in their trial will have a chip in the cap that will record when it is opened. Participants will keep a daily diary of their medicine. Their pills will be counted at clinical trial visits. Participants may have more short questionnaires. They may have interviews by phone or video.

Phase-Based Estimates

Selumetinibfor Malignant Peripheral Nerve Sheath Tumour (MPNST)

Waitlist Available

This study is evaluating whether a drug may help treat a type of cancer that is associated with neurofibromatosis type 1.

Phase-Based Estimates

Binimetinibfor Neurofibromatoses

Los Angeles, CA

Waitlist Available

Los Angeles, CA

This study is evaluating whether a drug called binimetinib can shrink tumors in people with neurofibromatosis type 1.

Phase-Based Estimates

Selumetinib Sulfatefor Neurofibroma

Waitlist Available

This study is evaluating whether a drug may help treat neurofibromatosis type 1.

Phase-Based Estimates

Selumetinibfor Watson Syndrome

Waitlist Available

Background: Neurofibromatosis type 1 (NF1) is a disorder that can cause plexiform neurofibromas (PNs). These are tumors that grow along nerves. Some PNs cause serious health problems. PNs often can t be operated on because of their large size, location, or number. There are no effective treatments known for people with NF1 and PNs. Researchers want to test if the drug selumetinib (AZD6244 hydrogen sulfate) causes PNs to shrink or slows down their growth. Objectives: To test if selumetinib helps treat PNs. To test how the body handles selumetinib and how it affects peoples symptoms. Eligibility: People ages 18 and older with NF1, with an inoperable PN that causes morbidity or is growing Design: Participants will be screened with: Medical history and physical exam Blood, urine, and heart tests Eye exam MRI: They lie in a machine that takes pictures of the body. PN biopsy: A small piece of the tumor is removed by a large needle. Questionnaires Participants will swallow selumetinib capsules every 12 hours for several 28-day cycles. The capsules are taken with a full glass of water on an empty stomach. Participants may have only water for 2 hours before and 1 hour after each dose. Participants will keep a drug diary. They will continue taking the drug as long as they tolerate it and their disease doesn t progress. Participants will have several visits throughout the study. These will include repeats of the screening tests. Participants will have a final visit after they stop taking selumetinib.

Phase-Based Estimates

Turaliofor Watson Syndrome

Background: - Some people with cancer have solid tumors. Others have refractory leukemia. This doesn t go away after treatment. Researchers want to see if a drug called PLX3397 can shrink tumors or stop them from growing. Objectives: - To find the highest safe dose and side effects of PLX3397. To see if it helps treat certain types of cancer. Eligibility: People ages 3 22 with a solid tumor or leukemia that has returned or not responded to cancer therapies. For Phase II, people ages 3 31 with a Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibroma (PN) that cannot be removed with surgery. Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Heart tests Scans or other tests of the tumor Participants will take PLX3397 as a capsule once daily for a 28-day cycle. They can do this for up to 2 years. During the study, participants will have many tests and procedures. They include repeats of the screening tests. Participants will keep a diary of symptoms. Participants with solid tumors will have scans or x-rays. Participants with NF1 PN will have MRI scans. Participants with leukemia will have blood tests. They may have a bone marrow sample taken. Some participants may have a biopsy. When finished taking PLX3397, participants will have follow-up visits. They will repeat the screening tests and note side effects. Phase II will follow the same procedures as Phase I above, but participants will also fill out questionnaires about their pain and quality of life.

Phase-Based Estimates

Cabozantinibfor Neurofibroma, Plexiform

Waitlist Available

This study is evaluating whether a drug called cabozantinib might help shrink tumors in people with neurofibromatosis type 1.

Phase-Based Estimates

AZD6244for Watson Syndrome

Philadelphia, PA

Waitlist Available

Philadelphia, PA

This study is evaluating whether a drug called AZD6244 hydrogen sulfate can shrink tumors in children and young adults with plexiform neurofibromas.

Phase-Based Estimates

Selumetinibfor NF1

Indianapolis, IN

Waitlist Available

Indianapolis, IN

A multi-institutional open-label phase 1/2 trial of selumetinib in combination with AZD5153 and durvalumab in refractory/unresectable sarcomas including MPNST. The phase 1 portion will be separated in two parts and will be open to all patients with refractory/relapsed sarcomas. The phase 2 portion will be for patients with refractory/unresectable NF1-associated MPNST.

Phase-Based Estimates

PEERSfor Neurofibroma

Waitlist Available

This is an open trial of the UCLA PEERS protocol delivered via Telehealth with teens with neurofibromatosis type 1 whose parents report that they have difficulty making and keeping friends.

Phase-Based Estimates

Poly ICLCfor Glioma

This study is evaluating whether a drug may help treat low-grade gliomas in children with neurofibromatosis type 1.

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