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21 Neurofibromatosis Trials
Power is an online platform that helps thousands of Neurofibromatosis 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.
Multiple Targeted Therapies for Meningioma
Kingman, KansasThis trial studies four drugs to treat patients with worsening meningioma. These drugs work by blocking enzymes that the tumor cells need to grow. The trial focuses on patients whose tumors have specific genetic mutations.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Uncontrolled Hypertension, Diabetes, Hepatitis, Others
Must Not Be Taking:CYP3A4 Inhibitors, CYP3A Inducers
124 Participants Needed
Telaglenastat for Cancer
Salina, KansasThis 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
Age:18+
Sex:All
50 Participants Needed
Selumetinib vs Carboplatin/Vincristine for Brain Cancer
Oklahoma City, OklahomaThis 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. Drugs used as chemotherapy, 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
Prior Safety Data
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 21
Sex:All
Key Eligibility Criteria
Disqualifiers:Concurrent Malignancy, Serious Illness, Pregnancy, Others
Must Not Be Taking:Investigational Agents, Vitamin E
165 Participants Needed
Trametinib for Juvenile Myelomonocytic Leukemia
Oklahoma City, OklahomaThis 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
Sex:All
10 Participants Needed
MEK Inhibitor for Neurofibromatosis
Oklahoma City, OklahomaThis 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+
Sex:All
114 Participants Needed
REC-2282 for Meningioma
Overland Park, KansasThis trial is testing a new drug called REC-2282 to see if it can help treat specific brain tumors in patients who haven't responded to other treatments. The study will check if the drug can slow down or stop the growth of these tumors.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2, 3
Age:12+
Sex:All
Key Eligibility Criteria
Disqualifiers:Active Malignancy, Pregnancy, Others
Must Not Be Taking:Anti-tumor Agents, Investigational Drugs
92 Participants Needed
PAS-004 for Advanced Solid Tumors
Irving, TexasThe 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
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:CNS Metastases, Cardiac Disease, Infections, Others
Must Not Be Taking:CYP3A Inhibitors, QTc Prolonging
48 Participants Needed
Crizotinib for Acoustic Neuroma
Dallas, TexasThis 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+
Sex:All
19 Participants Needed
Poly-ICLC for Low-Grade Glioma
Dallas, TexasThis 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
Trial Phase:Phase 2
Age:< 22
Sex:All
Key Eligibility Criteria
Disqualifiers:Radiation Treatment, Malignant Glioma, Others
Must Not Be Taking:Steroids, Immunosuppressants
20 Participants Needed
Combination Therapy for Sarcoma
Dallas, TexasThis 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
Age:18 - 99
Sex:All
41 Participants Needed
Selumetinib for Pediatric Brain Cancer
Aurora, ColoradoThis 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
Sex:All
220 Participants Needed
Osimertinib + BLU-945 for Non-Small Cell Lung Cancer
Aurora, ColoradoThis 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
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
190 Participants Needed
Dabrafenib + Trametinib + Hydroxychloroquine for Brain Tumor
Aurora, ColoradoThis 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
Sex:All
57 Participants Needed
DCC-3084 for Cancer
Denver, ColoradoThis 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
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Cardiac Issues, Recent Surgery, Others
Must Not Be Taking:BRAF Dimer Inhibitors
140 Participants Needed
Selumetinib for Plexiform Neurofibromas
Saint Louis, MissouriThis 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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
146 Participants Needed
G207 for Brain Cancer
Saint Louis, MissouriThis study is a clinical trial to determine the safety of inoculating G207 (an experimental virus therapy) into a recurrent or refractory cerebellar brain tumor. The safety of combining G207 with a single low dose of radiation, designed to enhance virus replication, tumor cell killing, and an anti-tumor immune response, will also be tested.
Funding Source- FDA OOPD
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:3 - 21
Sex:All
24 Participants Needed
Binimetinib for Neurofibromatosis
Saint Louis, MissouriThis 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
Trial Phase:Phase 2
Age:1+
Sex:All
40 Participants Needed
Aspirin for Acoustic Neuroma
Iowa City, IowaThis trial is testing whether aspirin can slow tumor growth and improve hearing in patients aged 12+ with vestibular schwannomas. Aspirin's anti-inflammatory properties might help achieve these effects. Aspirin has been studied for its potential to protect against certain types of hearing loss and has shown some protective effects in previous studies.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:12+
Sex:All
Key Eligibility Criteria
Disqualifiers:Severe Asthma, Hydrocephalus, Others
Must Not Be Taking:Aspirin
300 Participants Needed
Dabrafenib + Trametinib for Brain Tumors
Memphis, TennesseeThis 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
Sex:All
164 Participants Needed
Selumetinib for Plexiform Neurofibroma
Rochester, MinnesotaThis 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
Sex:All
24 Participants Needed
Targeted Therapy for Neurofibromatosis
Rochester, MinnesotaThis is a multi-arm phase II platform-basket screening study designed to test multiple experimental therapies simultaneously in patients with NF2-related schwannomatosis (NF2-SWN, formerly known as neurofibromatosis type 2) with associated progressive tumors of vestibular schwannomas (VS), non-vestibular schwannomas (non-VS), meningiomas, and ependymomas.
This Master Study is being conducted as a "basket" study that may allow people with multiple tumor types associated with NF2-SWN to receive new drugs throughout this study. Embedded within the Master Study are individual drug substudies.
* Investigational Drug Sub-study A: Brigatinib
* Investigational Drug Sub-study B: Neratinib
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:12+
Sex:All
100 Participants Needed
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Frequently Asked Questions
How much do Neurofibromatosis 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 Neurofibromatosis 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 Neurofibromatosis 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 Neurofibromatosis 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 Neurofibromatosis 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 Neurofibromatosis clinical trials ?
Most recently, we added Combination Therapy for Sarcoma, DCC-3084 for Cancer and PAS-004 for Advanced Solid Tumors to the Power online platform.