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15 Neurofibromatosis Type 1 Trials
Power is an online platform that helps thousands of Neurofibromatosis Type 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.
MEK Inhibitor for Neurofibromatosis
Columbus, OhioThis 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
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
Sex:All
57 Participants Needed
Selumetinib for Pediatric Brain Cancer
Cincinnati, OhioThis 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
HLX-1502 for Neurofibromatosis
Cincinnati, OhioThe 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+
Sex:All
Key Eligibility Criteria
Disqualifiers:NF1-related Tumors, Cardiovascular Disorders, Others
Must Not Be Taking:MEK-inhibitors, TKI Drugs
20 Participants Needed
Selumetinib for Plexiform Neurofibroma
Akron, OhioThis 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
Selumetinib for Plexiform Neurofibromas
Toronto, OntarioThis 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
Trametinib for Pediatric Brain Tumor
Toronto, OntarioThis 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
Sex:All
114 Participants Needed
Osimertinib + BLU-945 for Non-Small Cell Lung Cancer
Toronto, OntarioThis 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
AlgometRx Nociometer for Neurofibromatosis
Bethesda, MarylandBackground:
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 is effective in measuring pain or other abnormal sensations in people with NF1.
Eligibility:
People aged 1 year and older with NF1.
Design:
Participants can have up to 3 assessments completed in person. Each assessment may last up to 1.0 to 1.5 hours.
Participants 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.
Participants 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+
Sex:All
70 Participants Needed
REC-2282 for Meningioma
Washington, District of ColumbiaThis 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
Neurofibromatosis Type 1 (NF1) is a common genetic disorder that is associated with a four times greater risk of learning disabilities, including reading disabilities, and a deficiency of neurofibromin - a protein important in a signaling pathway that regulates learning and memory. Our previous work (NS49096) demonstrated that school-age children with NF+RD can respond to standard phonologically-based reading tutoring originally developed to treat reading disability in the general population. Combining our work with that by other researchers suggesting that a medication (Lovastatin) may counteract the effects of the deficient neurofibromin, and possibly ameliorate learning disabilities in NF1, the investigator propose to examine the synergistic effects of medication plus reading tutoring.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:8 - 20
Sex:All
Key Eligibility Criteria
Disqualifiers:Epilepsy, Metabolic Syndrome, Liver Problems, Others
Must Be Taking:Lovastatin
120 Participants Needed
Photodynamic Therapy for Neurofibromatosis
Milwaukee, WisconsinThe investigators wish to determine the time to disease progression for benign neurofibromas treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) in patients with neurofibromatosis type 1 (NF1).
The investigators also wish to measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:14 - 30
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Porphyria, Photosensitivity, Others
Must Not Be Taking:Chemotherapy
30 Participants Needed
Targeted Therapy for Neurofibromatosis
Baltimore, MarylandThis 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
Alexandrite Laser for NF1
Boston, MassachusettsThe goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of Alexandrite (755 nm) Laser in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs). The main questions it aims to answer are:
Will performing up to 6 months of treatment sessions with alexandrite laser will result in tolerable local skin reactions and reduction in both individual cNF size by \>50% as well as improved cNF appearance in the treated field? If there is a comparison group: Researchers will compare laser treatment with cooling to both laser treatment without cooling and an untreated control see if laser treatments are effective and if cooling makes treatment more tolerable.
Participants will:
* Receive up to 6 monthly laser treatments.
* Complete surveys asking about pain during and after treatments.
* Complete surveys asking about satisfaction with the treatments.
* Undergo 2D photography and 3D imaging of treatment areas.
* Optionally, receive biopsies of up to 6 treated lesions to investigate characteristics of tumors that respond well to treatment as well as non-respondent tumors.
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
10 Participants Needed
AI-Enhanced MRI for Detecting Precancerous Lesions in Neurofibromatosis
Los Angeles, CaliforniaThis trial uses a full-body scan and smart computer software to monitor changes in pediatric patients with neurofibromatosis type 1. The MRI takes detailed images, and the AI analyzes them to track any changes over time.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:5 - 17
Sex:All
Key Eligibility Criteria
Disqualifiers:Sedation, Implants, Claustrophobia, Allergy, Others
15 Participants Needed
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Frequently Asked Questions
How much do Neurofibromatosis Type 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 Neurofibromatosis Type 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 Neurofibromatosis Type 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 Neurofibromatosis Type 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 Neurofibromatosis Type 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 Neurofibromatosis Type 1 clinical trials ?
Most recently, we added Alexandrite Laser for NF1, AlgometRx Nociometer for Neurofibromatosis and HLX-1502 for Neurofibromatosis to the Power online platform.