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22 Nf1 Trials Near You

Power is an online platform that helps thousands of Nf1 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.

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

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+

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

50 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

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

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

99 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

160 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:Recruiting
Trial Phase:Phase 1, 2

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

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

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

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

146 Participants Needed

Selumetinib + APG-115 for Neurofibromatosis

Washington, District of Columbia
This is a phase 0/1/2, multi-site study to evaluate the MEK inhibitor Selumetinib with the MDM2 Inhibitor APG-115 in patients with Neurofibromatosis Type 1 and pre-malignant and malignant peripheral nerve sheath tumors
No Placebo Group
Prior Safety Data

Trial Details

Trial Status:Not Yet Recruiting
Trial Phase:Phase 1, 2

45 Participants Needed

Medication + Reading Tutoring for Learning Disabilities in Neurofibromatosis Type 1

Nashville, Tennessee
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:Active Not Recruiting
Trial Phase:Phase 2
Age:8 - 20

120 Participants Needed

TAS0612 for Advanced or Metastatic Cancer

Nashville, Tennessee
This trial is testing a new drug called TAS0612 to see if it is safe for people with advanced or metastatic solid tumor cancer.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting

100 Participants Needed

Photodynamic Therapy for Neurofibromatosis

Milwaukee, Wisconsin
The 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

30 Participants Needed

Mirdametinib for Neurofibromatosis

Baltimore, Maryland
This is a Phase 1/2a, open-label, non-randomized, multi-dose study of mirdametinib monotherapy in adults with NF1 and cNF. In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at the NF1 and cNF specialty center: Johns Hopkins University.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

24 Participants Needed

DCC-3116 for Solid Tumors

Saint Louis, Missouri
This trial tests a new drug, DCC-3116, alone and with other drugs, in patients with advanced cancers that have specific genetic mutations. The drug works by blocking a pathway that helps cancer cells grow.
No Placebo Group
Prior Safety Data

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2

144 Participants Needed

Trametinib for Cancer With NF1 Genetic Changes

Philadelphia, Pennsylvania
This trial tests trametinib, an oral medication, in patients with advanced cancers having an NF1 mutation. Trametinib works by blocking proteins that help cancer cells grow, aiming to stop or slow down the cancer. Trametinib is approved for treating certain types of advanced cancers.
No Placebo Group
Prior Safety Data

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2

50 Participants Needed

Avutometinib for Solid Tumors

Atlanta, Georgia
The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults. Researchers will look for the highest dose of avutometinib that is safe and cause few or mild side effects.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:3 - 30

24 Participants Needed

Stem Cell Transplantation for Blood Cancers

Minneapolis, Minnesota
This is a phase II, open-label, prospective study of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation for children and adults with hematological malignancies
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 60

150 Participants Needed

Why Other Patients Applied

"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'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

"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 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

"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

Alexandrite Laser for NF1

Boston, Massachusetts
The 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

10 Participants Needed

Educational Letters for Neurofibromatosis

Boston, Massachusetts
The goal of this fully decentralized, randomized controlled trial is to compare the efficacy of two educational interventions for individuals with Neurofibromatosis 1 (NF1). The primary objective of the study is to determine which intervention leads to higher rates of evidenced-based health screenings for NF1 patients in primary care settings. Adults with NF1 and parents/guardians of children with NF1 from across the U.S. who do not go to a specialized NF clinic and who have an upcoming annual wellness visits (e.g. an annual physical, a well-child visit, etc.) scheduled with a primary care provider (PCP) are eligible to enroll in the study. To see if you might be eligible, fill out a prescreening survey here: https://redcap.link/mynfguide
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased

360 Participants Needed

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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?

Frequently Asked Questions

How much do Nf1 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 Nf1 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 Nf1 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 Nf1 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 Nf1 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 Nf1 clinical trials?

Most recently, we added Combination Therapy for Sarcoma, Alexandrite Laser for NF1 and Selumetinib + APG-115 for Neurofibromatosis to the Power online platform.

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By Trial

vDOT + Financial Incentives for Latent Tuberculosis

Progestin Patch for Birth Control

Oral Antibiotics for Prosthetic Joint Infections

Prehabilitation for Head and Neck Cancer Surgery

AAC Technology for Child Hearing Loss

Dazodalibep for Sjögren's Syndrome

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