Neurocristopathy

This early-phase study tests a new combination of two different immune cells—NK cells and CAR-T cells—for children and young adults with neuroblastoma or osteosarcoma that has returned or not responded to standard treatments. The NK cells are designed to help the CAR-T cells work…

This study tests whether combining two drugs—ASTX727 (a pill) and retifanlimab (given through a vein)—can shrink tumors and control advanced Merkel cell cancer in people whose disease has worsened after standard immunotherapy. About 31 adults with stage III or IV Merkel cell canc…

This phase 2 trial tests whether adding an experimental immune-boosting pill (PRTX007) to standard immunotherapy (pembrolizumab) before surgery can improve outcomes for people with stage III melanoma. About 48 adults with resectable melanoma will take the combination for 9 weeks …

This phase 1 trial tests a radioactive drug called 211At-MABG in 16 adults with advanced neuroendocrine cancers, including pheochromocytoma and paraganglioma. The drug delivers radiation directly to cancer cells that show up on a special scan. The main goal is to see if the treat…

This study tests an experimental oral drug called VMD-102 in people with advanced solid tumors, including liver, lung, kidney, skin (uveal melanoma), and colorectal cancers that have not responded to standard treatments. The drug works by blocking a specific protein (PKC epsilon)…

This study tests whether adding axelopran, a drug that blocks opioid side effects in the gut, to the immunotherapy nivolumab can help people with advanced melanoma that no longer responds to PD-1 inhibitors. The trial includes about 28 adults with unresectable or metastatic cutan…

This study tests a therapy called Perkileucel, made from a patient's own immune cells (tumor-infiltrating lymphocytes). These cells are collected from a surgically removed melanoma tumor, grown in a lab, and then infused back into the patient. Before the infusion, patients receiv…

This study tests whether a common antibiotic for women's UTIs, fosfomycin-trometamol, works well for men with urinary tract infections. Current treatments can have serious side effects and increase antibiotic resistance. The trial will enroll 138 men in primary care and monitor f…

This early-stage study tests a new drug called N-3C01, given alone or with an immunotherapy, in people with advanced solid tumors that have spread or cannot be removed. The main goals are to check safety, find the right dose, and see if the combination shrinks tumors. About 114 a…

This study tests whether giving a Pfizer-BioNTech COVID-19 mRNA vaccine before standard immunotherapy (pembrolizumab) and chemotherapy can improve outcomes for people with stage 4 non-small cell lung cancer. Researchers will enroll 500 adults to see if the vaccine helps the immun…

This early-phase trial is testing whether adding sitagliptin, a diabetes drug, to the immunotherapy pembrolizumab is safe and can help people with advanced kidney cancer or melanoma whose cancer has stopped responding to standard treatment. The study will first find the safest do…

This study tests a drug called ifinatamab deruxtecan (I-DXd) in children with solid tumors that have returned or not responded to treatment. The drug is an antibody-drug conjugate that targets cancer cells directly. The trial aims to check safety and see if tumors shrink in about…

This study tests whether giving two immunotherapy drugs (fianlimab and cemiplimab) before surgery can shrink melanoma tumors in people with stage IIIB to IV melanoma. About 35 adults will receive the drugs, then have surgery to remove any remaining cancer. The main goal is to see…

This study tests a treatment called extracorporeal photopheresis (ECP) for people with advanced melanoma who develop severe colitis or hepatitis from immunotherapy. ECP filters a patient's blood, treats it with a light-activated drug, and returns it to the body. The trial compare…

This phase 2 trial tests a drug called BMS-986504 in people with advanced solid tumors that have lost a gene called MTAP. The drug is given alone or with other cancer medicines. The study aims to see if the drug shrinks tumors and how safe it is. About 260 adults will take part.

This early-phase trial tests whether a topical cream called squaric acid dibutyl ester (SADBE) can safely reduce pigment-producing cells in large congenital moles before they are surgically removed. Thirty adults with these birthmarks will apply the cream or a placebo for several…

This early-stage study tests a new drug called JL19001 in people with advanced solid tumors or a type of lymphoma that has come back or not responded to treatment. The main goal is to find a safe dose and check for side effects. About 14 participants will receive the drug as a sh…

This study tests a new drug, prifetrastat, combined with fulvestrant for people with advanced HR+/HER2- breast cancer that has stopped responding to standard hormone therapy and CDK4/6 inhibitors. About 51 participants will receive the combination to see if it can shrink tumors o…

This study tests a new drug called Bmab1800 to see if it works like the approved drug Keytruda in people with stage IIB, IIC, or III melanoma after their tumor has been removed by surgery. About 138 adults will get either Bmab1800 or Keytruda, and researchers will compare how the…

This early-phase study tests a new mRNA medicine (NCP-IL-22BP mRNA) in 9 people with advanced solid tumors that have not responded to standard treatments. The medicine is injected directly into tumors to help the body fight cancer. The main goal is to check safety and find the ri…

This study looks at whether giving immunotherapy infusions in the morning improves outcomes for people with advanced melanoma. Researchers will compare morning versus afternoon/evening dosing in 108 participants. The goal is to see if aligning treatment with the body's natural im…

This phase 2 trial is testing whether a lower dose of the immunotherapy drug nivolumab, given less often, can still control cancer in people with various advanced or metastatic cancers. The goal is to see if a cheaper, more accessible regimen works as well as the standard high-co…

This early-phase trial tests a virus called DNX-2401, designed to infect and destroy melanoma cells in the brain and body. About 50 adults with stage IV melanoma will receive injections to find the safest dose and number of treatments. The main goal is to check for side effects, …

This study looks back at how well a type of targeted radiation therapy (PRRT) works for people with advanced pheochromocytoma and paraganglioma, rare tumors of the adrenal glands. Researchers will analyze data from about 210 patients treated in Italy over 24 years. The goal is to…

This early study tests a new treatment for advanced skin melanoma that has not responded to standard therapies. Ten participants will receive two injections of gold nanorods directly into their tumors, which are then activated with a special light to generate heat and destroy can…

This early-stage study aims to develop a personalized treatment for children with syndromic craniosynostosis, a group of rare genetic disorders where skull bones fuse too early. The approach uses a nano-engineered gel to deliver custom genetic medicine directly to the affected ar…

This study tests whether adding the drug naxitamab to standard chemotherapy is safe and effective for children and young adults newly diagnosed with high-risk neuroblastoma, an aggressive nerve cell cancer. Ten participants aged 1 to 21 will receive the combination and be monitor…

This Phase 1 trial tests whether HLX18, a biosimilar of the cancer drug OPDIVO, works similarly in people with esophageal, melanoma, or bladder cancer after their tumors have been surgically removed. About 174 participants will receive either HLX18 or OPDIVO to compare how the dr…

This study tests a new robotic system designed to help surgeons perform chest and belly surgeries in children. It will include 64 kids under 18 who need common surgeries like kidney repair or lung removal. The goal is to see if the robot is safe and works as well as standard surg…

This study tests adding a new drug, iberdomide, to standard chemoimmunotherapy for children and young adults (ages 1-30) whose neuroblastoma has returned, not responded, or worsened after prior treatment. The goal is to see if the combination is safe and works better at controlli…

This phase 2 trial tests the drug mirdametinib, alone or with radiation, in people with NF1-related high-grade gliomas (aggressive brain tumors). It includes three groups: those with recurrent sporadic glioblastoma, newly diagnosed NF1-associated glioblastoma, and other NF1-relat…

This early-phase trial tests a new cell therapy called ThINKK in 12 children aged 2–12 who have had a stem cell transplant for leukemia or neuroblastoma. The therapy is designed to boost the body's natural killer (NK) cells to better fight cancer. The main goals are to check if t…

This phase II trial tests two drugs—SHR-1701 (an immunotherapy) and famitinib (which cuts off tumor blood supply)—given before surgery for resectable mucosal melanoma, a rare and aggressive skin cancer. After surgery, treatment is tailored based on how well the tumor responded. T…

This phase 2 trial tests three immune-boosting drugs (cemiplimab, fianlimab, and ipilimumab) given before surgery in 20 people with advanced but removable melanoma. The goal is to see if the combination is safe and can shrink tumors, potentially improving outcomes. Participants m…

This study tests whether a new drug called Bmab1700 works the same as the approved immunotherapy Opdivo in people with melanoma who have had their tumors surgically removed. The trial will involve 120 adults with stage IIB to IV melanoma. Researchers will compare how the drugs mo…

This study tests whether giving three drugs (pucotenlimab, lenvatinib, and temozolomide) before surgery can shrink tumors in people with a specific type of skin cancer called acral melanoma, which is stage IIB or III and can be removed. About 30 adults will receive the drug combi…

This Phase 2 trial tests a new cell therapy called RAPA-201 for people with advanced melanoma that has stopped responding to PD-(L)1 inhibitors. The therapy uses a patient's own immune cells, modified to resist the drug rapamycin, and is given along with chemotherapy. The study a…

This phase 2 trial tests the drug everolimus in 67 people with vascular anomalies (abnormal blood vessels) that haven't responded to standard treatments. The goal is to shrink the lesions and improve symptoms. Participants take everolimus daily for 6 months, with doses adjusted b…

This trial tests AVT32-DRL_PB, a biosimilar (copy) of the cancer drug Keytruda, in 120 people with stage IIB/C or III melanoma whose tumors were fully removed. Half get the biosimilar, half get Keytruda, every 3 weeks. The main goal is to see if drug levels in the blood are simil…

This early-stage trial tests whether two cancer drugs, cabozantinib and selumetinib, can safely shrink plexiform neurofibromas in people aged 16 and older with neurofibromatosis type 1 (NF1). About 30 participants will receive the combination daily. The study first finds the safe…

This study tests a combination of two immunotherapy drugs, nivolumab and relatlimab, in people with advanced melanoma that hasn't been treated before. The goal is to see if the combination is safe and helps shrink tumors. About 30 adults and teens (ages 12 and up) in India will t…

This study tests a combination of chemotherapy drugs (liposomal irinotecan, vincristine, and temozolomide) with or without an additional drug (anlotinib) in children aged 3-18 whose solid tumors have returned or not responded to treatment. The goal is to see if this combination c…

This early-stage study tests a new treatment for people with advanced ovarian cancer or other solid tumors that have come back or not responded to standard therapy. The treatment uses a patient's own immune cells that are genetically modified to better recognize and attack cancer…

This early-phase trial tests whether immune cells (CD8+ T cells) given through a spinal tap are safe for people with melanoma that has spread to the lining of the brain. Only 8 participants will receive the treatment. The main goal is to check for side effects, not to cure the di…

This Phase 2 trial tests whether a personalized cell therapy called HS-IT101 works better than chemotherapy for people with advanced melanoma that has stopped responding to standard treatments. HS-IT101 uses a patient's own immune cells, grown in large numbers in a lab, to fight …

This early-phase trial tests a combination of three drugs—selumetinib, ZEN-3694, and durvalumab—in people with advanced sarcomas, including malignant peripheral nerve sheath tumors (MPNST) linked to NF1. The study first checks safety and dosing, then looks at whether the treatmen…

This early study tests whether a daily enema made of salt water and glycerin can help children with fecal incontinence stay clean. The trial will include 30 children aged 3 to 16 who have bowel accidents at least once a week due to conditions like corrected anorectal malformation…

This early-phase study tests whether a new cell therapy called B7-H3 CAR T cells is safe and can help children and young adults whose solid tumors have returned or stopped responding to standard treatments. About 40 participants will receive their own immune cells that have been …

This study tests a combination of three drugs (pucotenlimab, lenvatinib, and temozolomide) given before and after surgery for people with a rare type of melanoma in the head and neck area. The goal is to see if this approach can shrink tumors and improve outcomes. About 30 adults…

This early-phase trial tests SL-28, a cell therapy made from donor immune cells, in 60 people with advanced solid tumors (like lung, pancreatic, or breast cancer) that haven't responded to standard treatments. The study aims to find a safe dose and see if the therapy can shrink t…

This study tests whether surgically removing the central mole (halo nevus) in people with vitiligo leads to better skin color restoration than using medication alone. Sixty participants aged 6 to 45 will be randomly assigned to either have the mole removed plus standard treatment…

This study compares two surgical techniques—Duhamel and Soave—for treating total colonic Hirschsprung disease, a severe birth defect where nerve cells are missing from the colon. Fifty children who had a first surgery as newborns will receive one of the two procedures. Researcher…

This study tests a combination of two experimental drugs (iparomlimab and tuvoraleimab) plus standard chemotherapy for people with advanced acral or mucosal melanoma, rare and aggressive skin cancers. About 48 adults who have had little or no prior treatment will receive the ther…

This study tests whether Coenzyme I injections can reverse vascular aging in 60 adults aged 40-70 with stiff arteries. Participants receive either the coenzyme or a placebo, and researchers measure changes in blood vessel function and stiffness over 28 days. The goal is to see if…

This Phase 3 trial tests an oral drug called KP-001 in 150 people aged 2 and older with venous malformations, lymphatic malformations, or KTS/CLOVES syndrome. Participants receive either KP-001 or a placebo for 24 weeks, then everyone gets the drug for another 28 weeks. The goal …

This phase 2 trial tests three different combinations of a radioactive drug (131I-MIBG) with other medicines (vorinostat and/or dinutuximab) in children and young adults with relapsed or refractory neuroblastoma. About 118 participants will be randomly assigned to one of three tr…

This early-stage trial tests an experimental drug called ACC-1898, taken as a daily pill, in adults with advanced solid tumors (like liver, kidney, or stomach cancer) that have not responded to standard treatments. The main goals are to find a safe dose and see how the body handl…

This phase 2 trial tests a combination of two drugs (carilizumab and apatinib) plus radiation therapy as a first treatment for advanced mucosal melanoma, a rare and aggressive skin cancer. The study will enroll 30 adults who have not had prior systemic therapy. Researchers will m…

This early-stage trial tests whether immune cells taken from a patient's own tumor (TIL) can be safely given directly into the liver or through a vein to fight metastatic melanoma. 18 adults with advanced uveal or cutaneous melanoma that has spread will receive TIL along with che…

This study tests whether a new type of PET scan (18F-MFBG) can detect neuroblastoma tumors as accurately as the current standard scan (123I-MIBG). About 84 children and adults with newly diagnosed, high-risk neuroblastoma will receive both scans to compare results. If the new sca…

This study will compare a new radioactive tracer, 18F-AmBF3TATE, to the standard tracer 68Ga-DOTATATE for PET scans in people with neuroendocrine tumors. The goal is to see if the new tracer is as good or better at finding tumors. About 51 participants will receive both tracers a…

This phase 3 trial tests whether a magnetic tracer (Magtrace) works as well as the standard radioactive dye plus blue dye for finding sentinel lymph nodes in people with melanoma. About 254 adults having surgery for melanoma will receive both tracers and be randomly assigned to h…

This study tests whether a new MRI contrast agent, gadopiclenol, can detect brain metastases earlier than standard methods in 180 patients with advanced lung, breast, or skin cancer. Participants will receive screening brain MRIs to compare diagnosis timing and accuracy. The goal…

This study tests a new PET imaging agent, 68Ga-cPP-BCH, to detect PD-L1 protein levels in tumors of people with lung cancer, melanoma, or other solid tumors. The goal is to see if this scan can identify who might benefit from anti-PD-1/PD-L1 immunotherapy without needing a tissue…

This study is developing a blood test called NeutroFlow that measures specific immune cells (neutrophils) to predict whether cancer patients will benefit from immunotherapy. Researchers will collect blood samples from 600 patients with several types of cancer before they start tr…

This study aims to develop an artificial intelligence system that can automatically detect potential melanomas using 3D whole-body skin images. Researchers will collect scans from 1,000 adults with many moles and compare the AI's findings to those of three skin specialists. The g…

This study compares a new type of PET/CT scan using 18F-MFBG to the current standard scan (123I-MIBG) in 30 children with relapsed or refractory neuroblastoma. The goal is to see if the new scan works as well or better for detecting tumors. If it does, it could offer a quicker an…

This study tests whether light therapy (photobiomodulation) can prevent mouth sores in children receiving chemotherapy. Fifty children aged 0-30 will receive daily light treatments during hospital stays and at home. Researchers will check if the therapy is easy to use and accepta…

This study tests five different smartphone programs designed to help melanoma survivors reduce unprotected sun exposure. About 150 participants will wear a UV sensor and activity monitor, complete daily surveys, and use the programs for 8 weeks. The goal is to see which programs …

This pilot study tests whether using bright light therapy for an hour each morning, delivered through an iPad app, is tolerable for people with advanced melanoma or non-small cell lung cancer who are about to start immunotherapy. The idea is that synchronizing the body's internal…

This study tests whether an AI-based website that gives personalized diet tips and recipes can help cancer patients who experience taste changes during therapy. About 198 adults with certain cancers (like multiple myeloma, melanoma, or GI cancers) will either get the AI tool or a…

This pilot trial tests whether adding emotional support to a cancer information website helps young adults (ages 20-39) with thyroid, melanoma, or testicular cancer feel less worried and distressed. 142 participants will either get general cancer info or the same info plus person…

This study tests whether having melanoma patients regularly report their symptoms through an electronic tool, combined with quick access to specialist care, can improve the management of side effects from immune checkpoint inhibitors. These drugs can cause immune-related side eff…

This study looks at whether intermittent fasting is safe and doable for people with advanced solid tumors who are starting immunotherapy. About 90 participants will be split into three groups: no fasting, eating only during an 8-hour window each day, or fasting every other day. R…

This early-phase trial tests whether a home-based morning light therapy program can help people with advanced melanoma who are receiving tumor-infiltrating lymphocyte (TIL) therapy. The light therapy is delivered through an iPad app and aims to improve the body's natural sleep-wa…

This study tests whether a digital tool combining weekly symptom surveys and a Fitbit can help monitor patients during immunotherapy for advanced melanoma. Forty participants will be randomly assigned to use the tool or receive usual care. The goal is to see if the system is easy…

This study tests whether adding telephone health coaching to a symptom management handbook helps reduce symptom burden and improve quality of life for people with metastatic breast, GI, or melanoma cancer and their caregivers. About 200 participants will be split into two groups:…

This early-phase study tests whether adding malic acid (a natural fruit acid) to standard immunotherapy is safe for people with solid tumors like colorectal, lung, or melanoma cancer. About 30 participants will take malic acid by mouth while continuing their immunotherapy. The ma…

This pilot study tests whether a personalized exercise and education program, given before and after surgery, can help melanoma patients regain strength and function. Thirty adults with melanoma who are scheduled for surgery after chemotherapy will take part. The main goal is to …

This study tests whether an 8-week physiotherapy program — including breathing exercises, pelvic floor training, and core stabilization — can improve bowel function in 20 children aged 5–18 who have had surgery for Hirschsprung's disease. Participants will keep a bowel diary and …

This study screens up to 1,000 people with personal or family histories of certain cancers to see if they qualify for ongoing genetics research at the National Cancer Institute. Participants fill out a 15-20 minute online survey about their health and family history. No treatment…

This study looks at whether adding a high-fiber diet and short-term fasting can help melanoma patients undergoing immunotherapy. Forty participants will follow a high-fiber diet, with half also doing 72-hour fasts during each treatment cycle, while 20 others serve as a control gr…

This study looks at a rare type of cancer called oral mucosal melanoma. Researchers want to improve how the cancer is staged by measuring how deep it goes into tissue and by studying its genetic makeup. They will follow 1,100 patients over time to see which treatments work best f…

This study tests whether an online training platform called DermLoop Learn can improve how pathologists diagnose melanoma from skin lesion images. 258 pathologists will be randomly assigned to receive the training or not, and their diagnostic accuracy will be compared against a p…

This study tests a paper-based decision aid designed to help people with intermediate-risk melanoma decide whether to have a sentinel lymph node biopsy. The tool provides clear information about the procedure, its risks and benefits, and helps patients think about their personal …

This study looks at whether using genetic markers from a child's skin tumor can help doctors decide how much surgery is needed. About 51 children and young adults up to age 25 with localized melanoma or similar growths will have their tumor tissue reviewed centrally. Based on ris…

This early-phase study tests whether a low-dose immunotherapy injection directly into the tumor can activate the immune system as effectively as standard high-dose treatment through the veins, but with fewer side effects. About 18 adults with advanced melanoma will receive either…

This study looks at how young children with neurofibromatosis type 1 (NF1) walk, to see if simple walking tests can help doctors spot and predict movement problems early. Researchers will measure walking speed and other skills in 56 children under 6 years old. The goal is to crea…

This study will test whether immunotherapy drugs (immune checkpoint inhibitors) change how the liver processes other medications. About 80 adults with various cancers who are starting immunotherapy will take a low-dose cocktail of 7 common drugs. Researchers will measure drug lev…

This study will watch and record information from 120 newborns with serious gut problems admitted to the NICU. Researchers will track what types of emergencies are most common, how long babies stay in the hospital, and how many need surgery. The goal is to better understand these…

This study will enroll 200 patients scheduled for surgery to remove rare adrenal tumors called pheochromocytomas or paragangliomas (PPGL). Before surgery, researchers will use a model to predict the risk of severe blood pressure swings during the operation. During surgery, a real…

Hirschsprung disease is a rare birth defect where nerve cells are missing in part of the colon, causing severe constipation. Surgery to remove the affected section is the main treatment. This study compares two ways to do that surgery: one through the anus only, and another using…

This study looks at whether eating more fiber helps melanoma patients respond better to immunotherapy. Researchers will track the fiber intake of 60 patients using a special questionnaire and see if those who eat more fiber have less cancer recurrence or progression. The goal is …

This study will follow 600 people with early-stage solid cancers (like lung, head and neck, melanoma, or bladder cancer) who are receiving immunotherapy before surgery. Researchers will collect blood and tumor samples at several time points to see how the immune system responds. …

This study is for young adults (ages 21-45) who had certain cancers as teens or young adults. Researchers are adapting a program that helps survivors manage their health after cancer treatment. The goal is to see if this program improves survivors' confidence and ability to handl…

This study looks at whether tiny particles in the blood, called extracellular vesicles, can help doctors predict if immunotherapy will work for people with advanced lung cancer or melanoma. Researchers will collect blood samples from 378 patients before and during standard treatm…

This study is a long-term follow-up for 100 people who completed a previous trial of bel-sar, an experimental treatment for early choroidal melanoma (a type of eye cancer). Participants will not receive any new treatment but will be monitored for about 5 more years to check for s…

This study looks at how fat cells and immune cells interact in melanoma skin cancer to see if this can predict whether the cancer will spread. Researchers will examine tissue samples from 20 patients to check for a specific pattern they noticed in earlier work. The goal is to bet…

This study looks at whether older adults (75+) with early-stage melanoma can safely skip an extra lymph node biopsy after their main surgery. The goal is to see if the simpler surgery alone works just as well and improves overall well-being. About 428 participants will be followe…

This study aims to find biological markers in blood and urine that could explain congenital central hypoventilation syndrome (CCHS), a rare disorder where the brain fails to control breathing during sleep. Researchers will collect samples from 40 adults with CCHS and healthy volu…

This study looks back at medical records of 150 children who had surgery for neuroblastic tumors in the lower chest. Some had a special X-ray (arteriography) before surgery to map a key spinal artery, while others did not. Researchers want to see if the imaging helps prevent spin…