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Why We Started 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
Bask GillCEO at Power
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      Why We Started 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
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      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 Power
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      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

      MEK 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

      Dabrafenib + Trametinib + Hydroxychloroquine for Brain Tumor

      Columbus, Ohio
      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:Active Not Recruiting
      Trial Phase:Phase 4
      Age:1 - 99

      Key Eligibility Criteria

      Disqualifiers:Severe Toxicities, Others
      Must Be Taking:Dabrafenib, Trametinib

      163 Participants Needed

      HLX-1502 for Neurofibromatosis

      Cincinnati, Ohio
      The trial will be an open label, single arm, phase 2 study to assess the tolerability and efficacy of HLX-1502 in participants with NF1 that are 16 years or older in age with progressive and/or symptomatic PN. This study will also investigate the safety and efficacy of HLX-1502 in a small cohort of 12 to 15 year olds.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:12+

      Key Eligibility Criteria

      Disqualifiers:NF1-related Tumors, Cardiovascular Disorders, Others
      Must Not Be Taking:MEK-inhibitors, TKI Drugs

      25 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

      Antioxidant Therapy with N-acetylcysteine for Neurofibromatosis Type 1

      Cincinnati, Ohio
      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

      Selumetinib for Plexiform Neurofibroma

      Cincinnati, Ohio
      Plexiform neurofibromas (PN) are known to cause significant morbidity in children with NF1. The recent FDA approval for selumetinib in children 2 years and older with inoperable symptomatic PN was based on the finding that selumetinib shrinks the majority of PN in children with NF1 and results in clinically meaningful benefit such as improvement in pain or range of motion. However, many morbidities, such as blindness or nerve damage, cannot be fully reversed with PN shrinkage. Therefore, there remains a critical need in this patient population to determine if young participants with PN in high-risk locations may benefit from early medical intervention prior to the development of clinical problems. This study will determine whether participants with asymptomatic PN in high-risk locations can potentially benefit from early treatment with selumetinib.
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:1 - 8

      Key Eligibility Criteria

      Disqualifiers:Cancer, Severe Disease, Cardiac Conditions, Others
      Must Be Taking:Antihypertensives

      200 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

      Why Other Patients Applied

      "My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

      HZ
      Arthritis PatientAge: 78

      "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've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."

      ZS
      Depression PatientAge: 51

      "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
      Match to a Nf 1 Trial

      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

      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:Active Not 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.
      Prior Safety Data
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Malignant Glioma, Cardiovascular Disease, Ophthalmological Conditions, Others
      Must Not Be Taking:MEK Inhibitors

      145 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

      12

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      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.

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      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 Imlygic for Neurofibromatosis, Combination Therapy for Sarcoma and AlgometRx Nociometer for Neurofibromatosis to the Power online platform.