Condition
Suggested Conditions
- Anxiety
- Depression
- Alzheimer's Disease
- Weight Loss
- Heart Disease
- Cancer
- Asthma
Location
Search Clinical Trials
Conditions
Locations
Treatment Type
Trial Phase
Trial Status
Paid Participation
Clear All
38 Nf 1 Trials Near You
Power is an online platform that helps thousands of Nf 1 patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Learn More About PowerMEK Inhibitor for Neurofibromatosis
Columbus, Ohio
This trial tests mirdametinib, a medication taken by mouth, for patients with NF1-related tumors that can't be surgically removed. The drug works by blocking specific proteins to stop or shrink the tumors. Mirdametinib has shown significant and lasting decreases in pain and partial responses in NF1-related plexiform neurofibromas.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:2+
Key Eligibility Criteria
Disqualifiers:Liver Disease, Malignancy, Heart Disease, Others
Must Not Be Taking:MEK1/2 Inhibitors
114 Participants Needed
Telaglenastat for Cancer
Columbus, Ohio
This phase II trial studies how well glutaminase inhibitor telaglenastat hydrochloride (CB-839 HCl) works in treating patients with specific genetic mutations and solid tumors or malignant peripheral nerve sheath tumors that have spread to other places in the body (metastatic) or cannot be removed by surgery (unresectable). Glutaminase converts an amino acid (building block of proteins) called glutamine to glutamate, which can support several cellular pathways. Telaglenastat hydrochloride works by blocking glutamine activity needed for the growth of cells. When this activity is blocked, the growth of cancer cells may stop and the cancer cells may then die. Cancer is caused by changes (mutations) to genes that control the way cells function and uncontrolled cell growth may result in tumor formation. Specific genetic mutations studied in this clinical trial are NF1 mutation for malignant peripheral nerve sheath tumors, and NF1, KEAP1/NRF2, or STK11/LKB1 mutation for other solid tumors. Telaglenastat hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Chemotherapy, Radiotherapy, Glioma, Hepatitis, Others
54 Participants Needed
This phase I/II trial is designed to study the side effects, best dose and efficacy of adding hydroxychloroquine to dabrafenib and/or trametinib in children with low grade or high grade brain tumors previously treated with similar drugs that did not respond completely (progressive) or tumors that came back while receiving a similar agent (recurrent). Patients must also have specific genetic mutations including BRAF V600 mutations or BRAF fusion/duplication, with or without neurofibromatosis type 1. Neurofibromatosis type 1 is an inherited genetic condition that causes tumors to grow on nerve tissue. Hydroxychloroquine, works in different ways to stop the growth of tumor cells by killing the cells or stopping them from dividing. Trametinib and dabrafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving hydroxychloroquine with trametinib and/or dabrafenib may lower the chance of brain tumors growing or spreading compared to usual treatments.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:1 - 30
Key Eligibility Criteria
Disqualifiers:Breastfeeding, Significant Illness, Retinopathy, Others
Must Be Taking:RAF/MEK Inhibitors
57 Participants Needed
Trametinib for Juvenile Myelomonocytic Leukemia
Columbus, Ohio
This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory). Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1 - 21
Key Eligibility Criteria
Disqualifiers:Pregnancy, Breastfeeding, Uncontrolled Infection, Others
Must Not Be Taking:Corticosteroids, Investigational Drugs
10 Participants Needed
Binimetinib for Neurofibromatosis
Columbus, Ohio
This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib. The primary objective is to determine if there is an adequate level of disease responsiveness to binimetinib in children and adults with NF1 and inoperable plexiform neurofibromas. The objective response to binimetinib is defined as ≥ 20% decrease in tumor volume reduction by 12 courses.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:1+
Key Eligibility Criteria
Disqualifiers:Not Listed
40 Participants Needed
Selumetinib vs Carboplatin/Vincristine for Brain Cancer
Columbus, Ohio
This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatin/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). Selumetinib is a drug that works by blocking some enzymes that low-grade glioma tumor cells need for their growth. This results in killing tumor cells. Chemotherapy drugs, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether selumetinib works better in treating patients with NF1-associated low-grade glioma compared to standard therapy with carboplatin and vincristine.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2 - 21
Key Eligibility Criteria
Disqualifiers:Concurrent Malignancy, Serious Illness, Pregnancy, Others
Must Not Be Taking:Investigational Agents, Vitamin E
165 Participants Needed
Poly-ICLC for Low-Grade Glioma
Cincinnati, Ohio
This trial is testing a medication called Poly-ICLC that boosts the immune system in children with NF1 who have brain tumors that don't respond to usual treatments. The goal is to see if this medication can help shrink or control the tumors by enhancing the body's natural defenses.
No Placebo Group
Trial Details
Trial Status:Recruiting
Age:< 22
Key Eligibility Criteria
Disqualifiers:Radiation Treatment, Malignant Glioma, Others
Must Not Be Taking:Steroids, Immunosuppressants
20 Participants Needed
Combination Therapy for Sarcoma
Cincinnati, Ohio
This trial tests a combination of three drugs to treat adults with hard-to-treat sarcomas, especially a type called MPNST linked to NF1. The drugs work together to stop cancer growth and boost the immune system. The study aims to find out if this combination is safe and effective. The combination of panobinostat, venetoclax, and anti-CD40 has shown promise in preclinical models for inducing complete tumor remission.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Organ Transplant, Autoimmune Disorders, Others
Must Not Be Taking:Immunosuppressants, Vitamin E
41 Participants Needed
AZD6244 for Neurofibromatosis
Cincinnati, Ohio
Background:
- Plexiform neurofibromas are tumors that grow in and around nerves. The only way to treat them is with surgery. Some of these tumors cannot be completely removed. The tumors may be too large, too numerous, or in a bad location for surgery. An experimental drug called AZD6244 hydrogen sulfate may be able to prevent the tumors from growing, slow down their growth, or shrink them. This drug has been tested in adults with cancer and in children with some types of brain cancer. This study will test how well this drug works with these types of tumors.
Objectives:
- To study the safety and effectiveness of AZD6244 hydrogen sulfate in children and young adults with plexiform neurofibromas that cannot be completely removed by surgery.
Eligibility:
- Children and young adults between 12 and 18 years of age who have plexiform neurofibromas that cannot be completely removed by surgery.
Design:
* Patients will be screened with a physical exam, medical history, blood tests, and imaging studies.
* They will take the study drug twice a day with 8 ounces of water, every day for 28-day cycles of treatment. During study visits, participants will have blood and urine tests and physical exams. They will also have imaging studies to examine the tumor sizes and locations. They will answer questions about their health. They may have other tests as needed.
* Participants will continue to receive the study drug as long as they have no severe side effects and the disease is not getting worse.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:2 - 18
Key Eligibility Criteria
Disqualifiers:Pregnancy, Severe Disease, HIV, Others
Must Not Be Taking:Antiretrovirals, Vitamin E
99 Participants Needed
Dabrafenib + Trametinib for Brain Tumors
Cincinnati, Ohio
This trial is studying the effects of two cancer medications, dabrafenib and trametinib, in children. These drugs work by stopping signals that make cancer cells grow. Dabrafenib and trametinib have shown benefits in various BRAF-mutant tumors, including melanoma, lung cancer, and thyroid cancer. The goal is to see how these treatments affect children over time.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:1 - 99
Key Eligibility Criteria
Disqualifiers:Severe Toxicities, Others
Must Be Taking:Dabrafenib, Trametinib
250 Participants Needed
HLX-1502 for Neurofibromatosis
Cincinnati, Ohio
The trial will be an open label, single arm, phase 2 study in 20 participants. The study will assess the tolerability and efficacy of HLX-1502 in participants with NF1 16 years of age or older with progressive and/or symptomatic PN.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:16+
Key Eligibility Criteria
Disqualifiers:NF1-related Tumors, Cardiovascular Disorders, Others
Must Not Be Taking:MEK-inhibitors, TKI Drugs
20 Participants Needed
Antioxidant Therapy for Neurofibromatosis Type 1
Cincinnati, Ohio
This trial is testing a medication called NAC in children aged 8-16 with a condition called NF1. These children often have problems with movement and behavior, and there is no current treatment for these issues. NAC works by reducing harmful molecules in the brain, which may help improve these symptoms.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:8 - 16
Key Eligibility Criteria
Disqualifiers:Epilepsy, Major Depression, Asthma, Others
Must Not Be Taking:Antidepressants, Dopamine Blockers, Mood Stabilizers
58 Participants Needed
This trial tests N-Acetyl Cysteine (NAC), a common supplement, to see if it can help children with neurofibromatosis type 1 (NF1) who have cognitive, behavioral, and motor issues. NAC works by reducing harmful substances in the brain, potentially improving behavior and motor skills. NAC is a precursor to glutathione (GSH) and has been studied for its neuroprotective and cognitive benefits in various conditions.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:8 - 16
Key Eligibility Criteria
Disqualifiers:Chemotherapy, Epilepsy, Major Depression, Others
Must Not Be Taking:Antidepressants, Non-stimulant ADHD, Mood Stabilizers, Others
5 Participants Needed
Crizotinib for Acoustic Neuroma
Cincinnati, Ohio
This trial tests crizotinib, an oral medication, in patients with NF2 and growing vestibular schwannoma tumors. The drug works by blocking proteins that help the tumor grow.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:6+
Key Eligibility Criteria
Disqualifiers:Cardiac Disease, Severe Infections, Others
Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers
19 Participants Needed
Selumetinib for Pediatric Brain Cancer
Cincinnati, Ohio
This phase I/II trial studies the side effects and the best dose of selumetinib and how well it works in treating or re-treating young patients with low grade glioma that has come back (recurrent) or does not respond to treatment (refractory). Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:3 - 21
Key Eligibility Criteria
Disqualifiers:Uncontrolled Seizures, Significant Organ Dysfunction, Others
Must Not Be Taking:Anticancer Agents, MEK Inhibitors
220 Participants Needed
Trametinib + Azacitidine for Myelomonocytic Leukemia
Cincinnati, Ohio
This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML). Newly diagnosed lower-risk JMML patients will receive trametinib and azacitidine. High-risk JMML patients will receive trametinib, azacitidine, fludarabine, and cytarabine.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:1 - 21
Key Eligibility Criteria
Disqualifiers:Infections, Noonan Syndrome, Down Syndrome, Others
Must Not Be Taking:MEK Inhibitors
58 Participants Needed
Selumetinib Granules for Neurofibromatosis
Akron, Ohio
This trial tests selumetinib granules in children aged 1 to 7 years with inoperable NF1-related tumors. The medication works by blocking signals that make the tumors grow. The study aims to find the right dose and check if it's safe and effective.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:1 - 6
Key Eligibility Criteria
Disqualifiers:Malignant Glioma, Cardiovascular Disease, Others
Must Not Be Taking:MEKi, Herbal Supplements
36 Participants Needed
Selumetinib for Plexiform Neurofibroma
Akron, Ohio
This trial is testing how well selumetinib works when taken with a low-fat meal in adolescents with NF1 who have tumors that can't be removed by surgery. The goal is to see if eating a low-fat meal affects how the body absorbs the medication and if it helps reduce stomach-related side effects. Selumetinib is being investigated for its effectiveness in treating NF1-associated tumors, with previous studies showing promising positive results in patients.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:12 - 17
Key Eligibility Criteria
Disqualifiers:Cancer, Cardiovascular Disease, Liver, Others
Must Not Be Taking:Vitamin E
24 Participants Needed
FCN-159 for Neurofibromatosis
Detroit, Michigan
This trial is testing FCN-159, a new drug taken by mouth, for patients with advanced solid tumors and neurofibromatosis type 1. The drug works by blocking specific proteins that help cancer cells grow. This targeted approach aims to slow down or stop the progression of these diseases.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:2 - 70
Key Eligibility Criteria
Disqualifiers:Not Listed
160 Participants Needed
9-ING-41 for Advanced Cancers
Pittsburgh, Pennsylvania
GSK-3β is a potentially important therapeutic target in human malignancies. The Actuate 1801 Phase 1/2 study is designed to evaluate the safety and efficacy of 9-ING-41, a potent GSK-3β inhibitor, as a single agent and in combination with cytotoxic agents, in patients with refractory cancers.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Pregnancy, Cardiovascular Impairment, Brain Metastases, Others
350 Participants Needed
Why Other Patients Applied
"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."
IZ
Healthy Volunteer PatientAge: 38
"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."
FF
ADHD PatientAge: 31
"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."
AG
Paralysis PatientAge: 50
"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."
WR
Obesity PatientAge: 58
"I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."
ID
Pancreatic Cancer PatientAge: 40
DCC-3084 for Cancer
Buffalo, New York
This is a multicenter, Phase 1/2 clinical trial to evaluate DCC-3084 alone or in combination with other cancer therapies in participants with advanced cancers. Module A will enroll participants with advanced/metastatic solid tumors. Additional modules exploring other cancers may be added to the master protocol at a later date. Each module will be conducted in 2 parts: Part 1 (Dose Escalation) and Part 2 (Dose Expansion).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Pregnancy, Cardiac Issues, Recent Surgery, Others
Must Not Be Taking:BRAF Dimer Inhibitors
140 Participants Needed
PAS-004 for Advanced Solid Tumors
Fairfax, Virginia
The main purpose of this clinical trial is to test PAS-004 in people with advanced solid tumors with rat sarcoma virus (RAS), neurofibromatosis type I (NF1), or rapidly accelerated fibrosarcoma (RAF) mutations. The main questions it aims to answer are:
* How well participants are able tolerate different doses of PAS-004, and
* What side effects PAS-004 might have.
Study participants will have regular visits to the study doctor and be asked to have tests and exams done to check on their health and safety. Everyone participating in the study will take PAS-004 by mouth as a single dose, followed by one week observation, then once a day during the study, in 28-day cycles. Participants will continue on daily PAS-004 for up to 2 years, or until:
* They decide to withdraw from the study, or
* They experience unacceptable side effects, or
* Their disease progresses, or another illness interferes with taking the study drug, or
* The sponsors stops the study.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:CNS Metastases, Cardiac Disease, Infections, Others
Must Not Be Taking:CYP3A Inhibitors, QTc Prolonging
48 Participants Needed
Vinblastine + Tovorafenib for Brain Tumors
Toronto, Ontario
This is a Pilot, multicenter, open-label study of patients less than or equal to 25 years, with recurrent or progressive LGG harboring a CRAF or BRAF alteration, including BRAF V600 mutations and KIAA1549: BRAF fusions. Patients with BRAF or CRAF alterations will be identified through molecular assays as routinely performed at Clinical Laboratory Improvement Amendments (CLIA) of 1988 or other similarly certified laboratories.
The study will be conducted in two sequential phases:
Phase A: A Feasibility (combination dose finding) phase, followed by Phase B: An Efficacy phase. The maximum tolerated dose (MTD)/Recommended Phase 2 Dose (RP2D) of the combination as determined in Phase A would be the dose used in Phase B. The patients on Phase A who were below the MTD/RP2D would be eligible for intra-patient dose escalation to MTD/RP2D subject to criteria outlined later
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:0 - 25
Key Eligibility Criteria
Disqualifiers:Neurofibromatosis Type 1, Cardiovascular Disease, Others
Must Not Be Taking:CYP2C8, CYP3A4 Inhibitors
57 Participants Needed
Trametinib for Pediatric Brain Tumor
Toronto, Ontario
This is a phase 2, open-label, interventional clinical trial that will study the response rate of pediatric glioma and plexiform neurofibroma (PN) to oral administration of trametinib. Patients meeting all inclusion criteria for a given study group will receive the study medication at a daily dose of 0.025 mg/kg up to a total of 18 cycles, in 28-day cycles. A total of 150 patients will be recruited as part of this clinical study.
Patients aged between 1 month (corrected age) and 25 years old will be eligible, in order to include a maximum of patients affected by low-grade glioma (LGG) and PN. This study includes four groups: patients with neurofibromatosis type 1 (NF1) and LGG, NF1 patients with PN, patients with LGG with a B-Raf Serine/Threonine-protein Kinase/Proto-oncogene Encoding B-Raf (BRAF) fusion and patients with glioma of any grade with activation of the Mitogen-activated Protein Kinase/Extracellular Signal-regulated Kinases (MAPK/ERK) pathway. All patients except patients with PN must have failed at least one line of treatment.
The study will also explore the molecular mechanisms behind tumor development, progression and resistance to treatment. Furthermore, this study will also explore important aspects for patients with brain tumors by including assessment of quality of life and neuropsychological evaluation.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1 - 25
Key Eligibility Criteria
Disqualifiers:Other Malignancy, Uncontrollable Disease, HIV, Others
Must Not Be Taking:MEK Inhibitors, Others
114 Participants Needed
Selumetinib for Plexiform Neurofibromas
Toronto, Ontario
This trial is testing a medication called selumetinib to see if it can help adults with a condition called NF1 who have tumors that cause symptoms and cannot be removed by surgery. The medication works by blocking signals that make the tumors grow.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
146 Participants Needed
Osimertinib + BLU-945 for Non-Small Cell Lung Cancer
Toronto, Ontario
This trial is testing BLU-945, a new drug that targets cancer proteins, alone or with osimertinib. It focuses on patients with specific EGFR gene mutations. The drug aims to block a protein that helps cancer cells grow.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
190 Participants Needed
Abemaciclib for Neurofibroma
Bethesda, Maryland
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.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:12+
Key Eligibility Criteria
Disqualifiers:Pregnancy, Serious Medical Conditions, Others
Must Not Be Taking:Strong CYP3A Inhibitors
55 Participants Needed
Selumetinib for Neurofibromatosis
Bethesda, Maryland
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.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Pregnancy, Uncontrolled Illness, Cardiac Disorder, Ophthalmologic Conditions, Others
Must Not Be Taking:Investigational Agents, MEK Inhibitors
36 Participants Needed
AlgometRx Nociometer for Neurofibromatosis
Bethesda, Maryland
Background:
Neurofibromatosis type 1 (NF1) is a genetic condition that causes tumors to grow along the nerves in the skin, brain, and other parts of the body. People with NF1 often have pain and may experience other abnormal sensations like itching, numbness, or tingling. These symptoms can affect their daily life. Researchers want to learn more about these symptoms and find better ways to measure pain in people with NF1.
Objective:
To learn if a device called the AlgometRx Nociometer(Registered trademark) is effective in measuring pain or other abnormal sensations in people with NF1.
Eligibility:
People aged 1 year and older with NF1.
Design:
Individuals can have up to 3 assessments completed in person. Each assessment may last up to 1.0 to 1.5 hours.
Individuals will be screened. They will complete questionnaires about their health and how bad their pain is. If participants are having blood drawn for other reasons, some additional samples may be used in this study.
The AlgometRx Nociometer includes an electrode that will be placed onto a finger or a toe. The electrode will send non-painful electrical signals to activate nerves in the finger or toe. At the same time, a camera will be used to record changes in the pupil of the eye. The test will be done on all 4 of the participant s limbs; however, researchers may skip 1 or more limbs for various reasons. This test takes about 10 seconds to complete with at least a one-minute rest between testing different limbs.
Individuals will be asked to do a 2nd assessment with the AlgometRx Nociometer that may be done 1 hour later but no more than 72 hours after the first assessment. Participants who will be returning for another visit can opt to do a 3rd assessment that will be done at least 4 weeks but not more than 18 months after the 1st....
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Unphased
Age:1+
Key Eligibility Criteria
Disqualifiers:Eye Pathology, Uncontrolled Illness, Others
Must Not Be Taking:Atropine Eye Drops
70 Participants Needed
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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Key Eligibility Criteria
Disqualifiers:Uncontrolled Psychiatric Illness, Cognitive Impairments, Others
Must Not Be Taking:Psychiatric Medications
70 Participants Needed
Know someone looking for new options?
Spread the word
Learn More About Power
We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.
Bask GillCEO at Power
Frequently Asked Questions
How much do Nf 1 clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Nf 1 clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Nf 1 trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Nf 1 is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Nf 1 medical study?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Nf 1 clinical trials?
Most recently, we added Combination Therapy for Sarcoma, Selumetinib + APG-115 for Neurofibromatosis and AlgometRx Nociometer for Neurofibromatosis to the Power online platform.
Popular Searches
By Condition
By Location
Other People Viewed
By Subject
By Trial
Related Searches
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.