[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"neurofibroma-plexiform\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:neurofibroma-plexiform":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,49,73,106],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":31,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100239104","phase-1-phase-i-trial-of-turalior-pexidartinib-plx3397-in-children-and-young-adults-with-refractory-leukemias-and-refractory-solid-tumors-including-neurofibromatosis-type-1-nf1-associated-plexiform-neurofibromas-pn-and-tenosynovial-giant-cell-tumor--100239104",false,"NCT02390752","Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...","Phase I Trial of TURALIO (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor (TGCT)","* INCLUSION CRITERIA:\n* Diagnosis:\n\n  * Individuals must have recurrent or refractory solid tumors or acute leukemia (limited to AML or ALL) or have been intolerant of prior therapies, confirmed by the Laboratory of Pathology, NCI, e.g., solid tumors including rhabdomyosarcoma, Ewing sarcoma, soft tissue sarcomas. These may include primary neoplasms of the central nervous system, such as high-grade (WHO grade III-IV) glioma. Individuals with diffuse intrinsic pontine glioma (DIPG) or optic pathway glioma are exempt from histologic verification. For DIPG typical MRI findings must be present which include hypo- or isointense on T1-weighted imaging, hyperintense on FLAIR or T2-weighted imaging, epicenter in the pons in the face of a typical clinical presentation. Optic pathway gliomas are located in the optic pathway and are typically hypo- or iso-intense on T1 and hyperintense on T2-weighted images.\n  * In addition, individuals with NF1 and with malignant peripheral nerve sheath tumor (MPNST).\n\n    * Individuals must have relapsed after or be refractory to effective standard therapies. There are no limits on number of prior therapeutic regimens.\n* Disease status: Individuals with refractory solid tumors including patients with NF1 and MPNST must have evaluable disease, patients with leukemia must have measurable or evaluable disease at the time of enrollment, which may include any evidence of disease including minimal residual disease detected by flow cytometry.\n* Age \\>= 3 and \\\u003C= 35 years of age (must have BSA \\>= 0.55 m\\^2):\n* Ability of subject or Legally Authorized Representative \\[LAR\\] (the parent\u002Fguardian if subject is a minor) to understand and the willingness to sign a written informed consent document.\n* Individuals must be able to swallow capsules.\n* Performance Status: Karnofsky \\>= 50% for patients \\> 16 years of age and Lansky \\>= 50% for patients \\\u003C= 16 years of age. Individuals who are wheelchair bound because of paralysis will be considered \"ambulatory\" when they are up in their wheelchair. Individuals have to be able to travel to the NIH for evaluations.\n* Prior therapy:\n\nIndividuals must have fully recovered (to Grade 1) from the acute toxic effects of all prior anti-cancer therapy.\n\n* Myelosuppressive chemotherapy: At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea).\n* Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.\n* Immunotherapy: At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines.\n* Monoclonal antibodies: At least 3 half-lives of the antibody after the last dose of a monoclonal antibody.\n* XRT: At least 7 days after local palliative XRT (small port); At least 150 days must have elapsed if prior TBI or if \\>= 50% radiation of pelvis; \\>= 14 days from whole brain radiation, craniospinal radiation, or targeted radiation to CNS tumors. At least 42 days must have elapsed if other substantial BM radiation.\n* HSCT: \\>= 56 days from stem cell transplant with no evidence of active graft vs. host disease; must be off immunosuppressive therapy for at least 4 weeks and have no active graft-versus-host disease (GVHD) at the time of entry onto this trial.\n* Surgery: \\>= 14 days from surgery\n* Others: \\>= 7 days from last dose of short active hematopoietic growth factors, i.e. filgrastim, \\>= 14 days for long-acting, i.e. pegfilgrastim.\n* Steroids: Individuals with CNS tumors who are managed with steroids are eligible if they have no worsening neurologic deficits and are on a stable or decreasing dose of corticosteroids for greater than or equal to 7 days prior to registration. Individuals with leukemia receiving corticosteroids or hydroxyurea are eligible provided that the corticosteroids are not being used to manage GVHD and there has been no increase in corticosteroid of hydroxyurea dose for 7 days prior to starting TURALIO(R).\n\n  -Individual must have adequate hematologic, hepatic, and renal function, defined by:\n* Absolute neutrophil count \\>= 1.5 x 10\\^9\u002FL\n* Hemoglobin \\> 10 g\u002FdL\n* Platelet count \\>= 100 x 10\\^9\u002FL\n* AST and ALT \\\u003C= upper limit of normal (ULN)\n* TBil and DBil \\\u003C= ULN with an exception of patients with confirmed Gilbert's syndrome. For patients with confirmed Gilberts syndrome, the TBil should be \\\u003C= 1.5 x ULN\n* Serum creatinine \\\u003C= 1.5 x ULN\n* Exceptions:\n\n  * Cytopenias due to underlying disease (i.e. potentially reversible with anti-neoplastic therapy); A subject will not be excluded because of cytopenia due to disease, based on the results of bone marrow studies.\n  * Known active or chronic human immunodeficiency virus (HIV) or hepatitis C virus (HCV) infection, or positive hepatitis B (Hep B) surface antigen. Prior hepatitis infection that has been treated with highly effective therapy with no evidence of residual infection and with normal liver function (ALT, AST, total and direct bilirubin \\\u003C= ULN) is allowed.\n  * Hepatobiliary diseases including biliary tract diseases, autoimmune hepatitis, inflammation, fibrosis, cirrhosis of liver caused by viral, alcohol, or genetic reasons. Gilbert's disease is allowed if TBil is \\\u003C= 1.5 x ULN.\n\n    * Cardiac ejection fraction \\>= 50%, and QTcF \\\u003C 450 ms (male) or \\\u003C470 ms (female) on ECG at Baseline. (Fridericia's Formula: QTcF = (QT)\u002FRR0.33)\n    * Contraception: Women of child-bearing potential must agree to use an effective method of birth control during treatment and for 1 month after receiving their last dose of study drug. Fertile men must also agree to use an acceptable method of birth control while on study drug and for at least one month after last dose.\n\nEXCLUSION CRITERIA:\n\n* Individuals who are pregnant or breast feeding or who become pregnant while enrolled on this trial will be excluded from participation, due to the unknown effects of TURALIO(R) on a growing fetus or newborn child.\n* Ongoing treatment with any other cancer therapy or investigational agent, with the exception of IT chemotherapy for leukemia, when indicated.\n* Individuals who require therapy with warfarin.\n* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n* Active untreated infection.\n* Known active hepatitis A, B, C or HIV infection, chronic Hepatitis B or C, or HIV infection or inactive Hepatitis B carrier.\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to TURALIO(R) or other agents used in study.\n* Individuals with PT and\u002For INR higher than or equal to 1.5 times upper limit of normal, unless patients have lupus anticoagulant in which case they are eligible if cleared by hematology.\n* Drugs that strongly inhibit or potentiate CYP3A4, which includes CYP3A4 inducer, UGT inhibitors and acid reducing agents and avoid concomitant use of PPIs:\n\n  * Individuals who have received these drugs within 14 days or within 5 half-lives of the drug (whichever is longer) prior to study initiation will be excluded.","ALL","3 Years","35 Years",{"count":20,"type":21},54,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","Background:\n\n\\- Some people with cancer have solid tumors. Others have refractory leukemia. This may not go away after treatment. Researchers want to see if a drug called TURALIO(R) can shrink tumors or stop them from growing.\n\nObjectives:\n\n\\- To find the highest safe dose and side effects of TURALIO(R). To see if it helps treat certain types of cancer.\n\nEligibility:\n\n\\- People ages 3-35 with a solid tumor or leukemia that has returned or not responded to cancer therapies.\n\nDesign:\n\n* Individuals will be screened with:\n* Medical history\n* Physical exam\n* Blood and urine tests\n* Heart tests\n* Scans or other tests of the tumor\n* Individuals will take TURALIO(R) as a capsule once daily for a 28-day cycle. They can do this for up to 2 years.\n* During the study, participants will have many tests and procedures. They include repeats of the screening tests. Individuals will keep a diary of symptoms.\n* Individuals with solid tumors will have scans or x-rays.\n* Individuals with leukemia will have blood tests. They may have a bone marrow sample taken.\n* Some individuals may have a biopsy.\n* When finished taking TURALIO(R), individuals will have follow-up visits. They will repeat the screening tests and note side effects.",[27,28,29,30],"Neurofibroma, Plexiform","Precursor Cell Lymphoblastic Leukemia-Lymphoma","Leukemia, Promyelocytic, Acute","Sarcoma",[32,33,34,35],"Maximum Tolerated Dose","Dose Escalation","Acute Lymphocytic Leukemia","Acute Myelogenous Leukemia","RECRUITING","2026-06-27",{"date":39,"type":40},"2026-06-30","ACTUAL",{"date":42,"type":40},"2015-04-29",{"date":44,"type":21},"2028-12-31",{"name":46,"class":47},"National Cancer Institute (NCI)","NIH",1,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":53,"acronym":4,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":58,"phases":4,"briefSummary":59,"conditions":60,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":72},"100543787","real-world-treatment-study-of-koselugo-selumetinib-100543787","NCT06360406","Real-World Treatment Study of Koselugo (Selumetinib)","Inclusion Criteria:\n\n1. Patients treated with Koselugo (Selumetinib) under the approved label in South Korea\n2. Provision of signed and dated written informed consent by the patient or legally acceptable representative\n\nExclusion Criteria:\n\n1. Patients treated with Koselugo (Selumetinib) under the approved label in South Korea\n2. Provision of signed and dated written informed consent by the patient or legally acceptable representative","99 Years",{"count":57,"type":21},200,"OBSERVATIONAL","As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients treated with Koselugo (Selumetinib), an oral selective inhibitor of MAPK kinase (MEK) 1 and 2, by physicians in routine clinical practice settings. This study is designed to assess the known safety profile or identify previously unsuspected adverse reactions and evaluate the effectiveness of Koselugo under conditions of routine daily medical practice in Korea.\n\nThis study will provide information on the Korean patient population that is treated with the study drug.",[61,27],"Neurofibromatosis 1","2026-06-26",{"date":64,"type":40},"2026-06-29",{"date":66,"type":40},"2024-06-15",{"date":68,"type":21},"2031-09-30",{"name":70,"class":71},"AstraZeneca","INDUSTRY",12,{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":78,"acronym":4,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":16,"minAge":80,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":22,"phases":83,"briefSummary":84,"conditions":85,"keywords":90,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":96,"lastUpdatePostDateStruct":97,"startDateStruct":99,"completionDateStruct":101,"leadSponsor":103,"locationsCount":105},"100589988","phase-1-pas-004-in-adults-who-have-neurofibromatosis-type-1-with-plexiform-neurofibromas-100589988","NCT06961565","PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas","A Phase 1\u002F1b Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PAS-004, a MAPK\u002FERK Kinase 1\u002F2 (MEK 1\u002F2) Inhibitor, in Adult Participants With Neurofibromatosis Type 1 (NF1) With Symptomatic and Inoperable, Incompletely Resected, or Recurrent Plexiform Neurofibromas","Inclusion Criteria:\n\n1. Participant is capable of providing informed consent, which includes compliance with the requirements, prohibitions and restrictions listed in the informed consent form.\n2. Participant has been informed both verbally and in writing about the objectives of the clinical study, the methods, the anticipated benefits, the potential risks, and the discomfort to which they may be exposed and has given written consent to participation in the study prior to study start and any study-related procedure.\n3. Participant must be at least 18 years of age at the signing of the informed consent form (ICF).\n4. Participant must be able to swallow oral medication.\n5. Performance status: Participant must have a Karnofsky performance level of ≥70%. Note: Participants who are wheelchair bound because of paralysis secondary to a PN should be considered ambulatory when they are in the wheelchair. Similarly, participants with limited mobility secondary to the need for mechanical support (such as an airway PN requiring tracheostomy or CPAP) will also be considered ambulatory for the purposes of this study.\n6. Participant has been diagnosed with NF1 based upon the following diagnostic criteria:\n\n   a. Clinical and imaging confirmation meeting at least two of the following NF1 diagnostic criteria in accordance with the clinical NIH consensus criteria: i. ≥ Six cafe-au-lait macules \\> 1.5 cm in maximum diameter ii. Axillary and\u002For inguinal freckling iii. ≥ Two neurofibromas of any type, or ≥ 1 plexiform neurofibroma; iv. An optic pathway glioma (prior diagnosis without concurrent disease is acceptable) v. ≥ Five Lisch nodules (iris hamartomas). Note: must be confirmed on slit lamp exam by an ophthalmologist if this is one of only two criteria met for diagnosis vi. A distinctive bony lesion such as dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex vii. Biologic parent with confirmed diagnosis of NF1 viii. Genetic testing demonstrating a pathogenic NF1 germline mutation per CLIA-certified laboratory (or equivalent) testing.\n\n1\\. Note: NF1 germline pathologic mutation positive must either be confirmed by the central laboratory or have documentation of NF1 mutation issued by a CLIA-certified laboratory (or equivalent). 2. No concern by the Investigator that an NF1 mimic, including but not limited to Noonan Syndrome, Legius Syndrome, or schwannomatosis could potentially serve as a more likely diagnosis\n\n7\\. Participants satisfying the NF1 diagnostic criteria outlined in Inclusion Criterion #6 must also meet one of the following criteria:\n\na. Participant has at least one symptomatic PN (\"Target PN\") measuring at least 3 cm on maximal cross-sectional (axial) diameter that is judged by the Investigator to be likely responsible for participant symptoms (such as pain, deformity, or neurologic disability), and unable to be completely resected without causing substantial damage\u002Ffunctional deficit, or unsuitable for surgery with high surgical risks.\n\nb. Participant has an incompletely resected symptomatic PN with a postoperative residual of at least 15% of the primary lesion and measuring at least 3 cm on cross-sectional (axial) diameter.\n\nc. Participant has a recurrent symptomatic PN measuring at least 3 cm in maximal cross-sectional (axial) dimension after prior resection.\n\n8\\. Participants should have a minimum of seven measurable CN measuring 6-15 mm (if participants satisfy Inclusion Criterion #6 and have no CN or less than 7 CN they still might be considered for the study as judged by the Investigator and Sponsor).\n\n1. Measurable is defined as: 1. non-pedunculated (no stalk) 2. surrounded by visually uninvolved skin and not in physical contact with another CN, 3. measuring between 6 and 15 mm in the longest diameter and exophytic on visual exam (not macular).\n2. At least seven CN should be located on the trunk, neck, and\u002For limbs measuring between 6 and 15 mm in maximal diameter, and \"measurable\" as per the definition listed above.\n3. Participants with CN meeting the above criteria will undergo optional resections of at least two CN that meet eligibility criteria. If participant is willing to accept additional CN resections, this is permitted for up to four additional lesions after completion of six PAS-004 treatment cycles or after early withdrawal as long as there are sufficient lesions to allow efficacy assessment.\n\n   9\\. Participant must be able and willing to undergo serial MRI scans as outlined in the study protocol.\n\n   Note: Anxiolytic medication or pain medication as deemed clinically appropriate by the Investigator is permissible for the purposes of managing anxiety, claustrophobia, and pain during MRI.\n\n   10\\. Participant must be able and willing to undergo serial 2-D and where available 3-D photography as well as caliper measurements as outlined in the study protocol.\n\n   11\\. Participant must have an international normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤ 1.5 x ULN.\n\n   12\\. Participant must have adequate organ and bone marrow function at screening as indicated by the following laboratory value ranges:\n\na. Absolute neutrophil count (ANC) ≥ 1.5 × 109\u002FL b. Hemoglobin ≥ 90 g\u002FdL c. Platelets ≥ 100 × 109\u002FL d. Serum total bilirubin ≤ 1.5 × ULN for age (≤ 3.0 × ULN in participants with Gilbert's syndrome) e. Serum total bilirubin ≤ 1.5 × ULN (Serum total bilirubin can be ≤ 3.0 × ULN if participants have hemolysis or congenital hemolytic diseases) f. Aspartate aminotransferase (AST) ≤ 2.0 × ULN g. Alanine aminotransferase (ALT) ≤ 2.0 × ULN h. Albumin ≥ 3 g\u002FdL i. Creatinine clearance ≥ 60 mL\u002Fmin\n\n13\\. Participant must either agree to maintain abstinence (no heterosexual intercourse), or to use one highly effective form of contraception during study treatment and for at least 90 days after the last dose of investigational product (IP). Sperm-producing participants must agree not to donate sperm while receiving IP and for at least 90 days after the last dose of IP.\n\nExclusion Criteria:\n\n1. Participant has participated in another interventional clinical study within 28 days of starting PAS-004.\n2. Participant has received chemotherapy for any indication within 90 days of starting PAS-004.\n3. Participant has ongoing side effects from prior chemotherapy that are worse than mild (except alopecia). (\"Mild\" is defined as Asymptomatic or mild symptoms, clinical or diagnostic observations only, or intervention not indicated.)\n4. Participant has received treatment with any PN-directed drug or biologic therapy within 14 days of starting PAS-004.\n5. Participant has received treatment with a strong CYP3A4 inhibitor or inducer, or moderate inducers for CYP2C8 and CYP2C9 within 14 days of starting PAS-004, or any drug considered a major substrate of the enzymes above with a narrow therapeutic index except for topical skin use, as judged by the Investigator and Sponsor.\n6. Participant has received growth factors to increase the number or function of platelets or white blood cells within 7 days of starting PAS-004.\n7. Participant has received radiotherapy, major surgery, or immunotherapy within 28 days of starting PAS-004.\n8. Participant has malignant tumors associated with NF1 requiring chemotherapy, radiotherapy, or surgery, such as intermediate- to high-grade gliomas or malignant peripheral nerve sheath tumors.\n9. Participant has a current malignancy (excluding cured non-melanomatous skin cancer, breast carcinoma in situ, or cervical cancer in situ) or has history of malignancy requiring active treatment within the past 5 years (excluding cured non-melanomatous skin cancer, breast carcinoma in situ, and cervical cancer in situ). Other tissue-limited low stage cancers can be assessed by the Sponsor for possible inclusion on a per-participant basis.\n10. Participant has uncontrolled hypertension defined as blood pressures \\>150\u002F90 mmHg on repeat examinations despite maximal medical management.\n\n    Note: Participants with controlled hypertension with anti-hypertension therapy are permitted, as judged by the Investigator and Sponsor.\n11. Participant has active dysphagia, digestive system disease, malabsorption syndrome, or other conditions that might affect the absorption of PAS-004.\n12. Participant has previous or current retinal vein occlusion (RVO), retinal pigment epithelial detachments (RPED), clinically active glaucoma, or other significant abnormality in screening ophthalmic examination.\n13. Participant has interstitial pneumonia, NF1-related pulmonary disease, including existing clinically significant radiation pneumonitis.\n14. Participant has impaired cardiac function or cardiac disease as indicated by:\n\n    1. Average QTc interval \\> 480 ms calculated using the Fridericia's QT interval correction formula.\n    2. Grade ≥ 3 congestive heart failure per New York Heart Association (NYHA) guidelines.\n    3. Clinically significant arrhythmias, including but not limited to, complete left bundle branch conduction abnormalities and 2nd degree atrioventricular block.\n    4. Known concurrent clinically significant coronary artery disease, cardiomyopathy, or severe valvular disease.\n    5. Echocardiogram or multi-gated acquisition (MUGA) scan performed during the screening showing impaired left ventricular ejection fraction (LVEF) \\\u003C 45%.\n15. Participant has taken a QTc-prolonging medication within seven days of IP initiation or longer if the half-life of the QTc prolonging medication is such that the drug is not cleared from the body within 7 days (5 half-lives) of IP initiation.\n16. Participant has an uncontrolled bacterial, fungal, or viral infections, including active hepatitis B (hepatitis B virus surface antigen positive and hepatitis B virus DNA \\> 1000 IU\u002Fml or meeting the study site's diagnostic criteria for active hepatitis B infection), hepatitis C (hepatitis C virus RNA positive), or human immunodeficiency virus (HIV) infection with detectable viral load.\n17. Any clinically significant active or known history of liver disease or known hepatobiliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones).\n18. Participant has a known hypersensitivity to PAS-004, its excipients, or another MEK 1\u002F2 inhibitor.\n19. Participant is pregnant or lactating.\n20. Participant has a clinically significant condition that, in the opinion of the investigator, would preclude study participation or compliance with safety requirements.\n21. Participant is unable to attend in-person clinic visits per clinical site guidelines and restrictions.","18 Years",{"count":82,"type":21},56,[24],"The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1). The main questions it aims to answer are:\n\n* How well participants are able tolerate different doses of PAS-004, and\n* What side effects PAS-004 might have.\n\nThis study will have two parts, Part A and Part B. The main goal of Part A of this study is to learn more about how participants tolerate different doses of PAS-004, and what side effects PAS-004 might have. What we learn from Part A of the study will help decide what doses of the study drug (PAS-004) should be used in Part B of the study, and if it is safe.\n\nIn Part B, two different doses from Part A will be tested. The main goal of this part of the study is to keep studying any side effects of PAS-004 at those two dose levels, and to learn more about if the doses picked for this part of the study might have an effect on plexiform neurofibromas.\n\nParticipants in Part A of the study who were taking doses selected for Part B may be able to continue on to Part B and keep taking the same dose of PAS-004 for 6 more months.\n\nStudy participants in both parts will have regular visits to the study doctor and be asked to have tests and exams done to check on their health and safety, including blood draws and MRIs. Everyone participating in the study will take PAS-004 by mouth once a day during the study, in 28-day cycles. Participants will be asked to keep a diary to record their daily dose of study drug.\n\nParticipants will continue on daily PAS-004 for up to 6 months, or until:\n\n* They decide to withdraw from the study, or\n* They experience unacceptable side effects, or\n* Their disease progresses, or another illness interferes with taking the study drug, or\n* The sponsor selects a dose level to study further in the next part of the study, or\n* The sponsor stops the study.",[86,87,27,88,89],"NF1 Mutation","Neurofibroma Plexiform","Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)","Neurofibromatosis Type 1 (NF1)",[91,92,93,94,95],"NF1","neurofibromatosis","plexiform neurofibroma","cutaneous neurofibroma","neurofibromatosis type 1","2025-11-26",{"date":98,"type":40},"2025-12-04",{"date":100,"type":40},"2025-05-30",{"date":102,"type":21},"2027-12",{"name":104,"class":71},"Pasithea Therapeutics Corp.",5,{"id":107,"slug":108,"hasResults":11,"nctId":109,"briefTitle":110,"officialTitle":111,"acronym":112,"eligibilityCriteria":113,"healthyVolunteers":11,"sex":16,"minAge":80,"maxAge":4,"enrollmentInfo":114,"targetDuration":4,"studyType":22,"phases":116,"briefSummary":118,"conditions":119,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":121,"lastUpdatePostDateStruct":122,"startDateStruct":124,"completionDateStruct":126,"leadSponsor":128,"locationsCount":48},"100454579","evaluation-of-percutaneous-cryotherapy-in-the-treatment-of-plexiform-neurofibromas-and-unresectable-neurofibromas-in-neurofibromatosis-type-1-100454579","NCT05199376","Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1","Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1 (NF1)","CryoNF1","Inclusion Criteria:\n\n* Age ≥18 years;\n* Patient with neurofibromatosis type 1 according to NIH criteria ;\n* Patient with a benign neurofibromatous lesion that is painful and\u002For generates functional discomfort and is unresectable or with unacceptable scarring ;\n* Neutrophils \\> 1 G\u002Fl in the 14 days prior to inclusion;\n* Adequate coagulation test with normals values (as judged by the investigator);\n* Signed and dated informed consent document indicating that the patient has been informed of all aspects of the trial prior to enrolment\n* Covered by a medical insurance;\n\nExclusion Criteria:\n\n* Patient treated with concomitant chemotherapy and\u002For targeted therapies ;\n* Any contraindication to a percutaneous cryotherapy procedure, including the need for ice formation within 1 cm of the spinal cord, brain or other critical nerve structures, bowel or bladder (unless active or passive thermal protective maneuvers are performed);\n* Patient with malignant neurofibroma or MPNST ; CT scan without abnormality and if tumor presents a max SUV T\u002FF\\>1.5 ratio, targeted biopsy is required to confirm benign or malignant histology.\n* Patient with neurofibroma in areas at risk for neurological sequelae;\n* Patient with cold urticaria with history of angioedema;\n* Any cognitive impairment or condition that may limit the use of numerical scales and quality of life questionnaires;\n* Patient for whom follow-up does not seem feasible even in the short term;\n* Participation in another clinical trial that may interfere with the evaluation of the primary endpoint;\n* Patient under tutorshio, curatorship or deprived of liberty;\n* Pregnant or breast-feeding woman;\n* Any contraindication to the performance of an MRI\n* Patient with dysplastic neurofibroma",{"count":115,"type":21},30,[117],"NA","Neurofibromatosis type 1 (NF1) is an autosomal dominant disease affecting chromosome 17. It is manifested by a neurogenic tumor proliferation that forms cutaneous, subcutaneous or deep neurofibromas.\n\nNeurofibromas can cause functional discomfort, neurogenic pain that is difficult to treat, and severe cosmetic disorders.\n\nTreatment is essentially surgical. It is sometimes a heavy invasive surgery with complicated postoperative follow-up and significant scarring on the aesthetic level. Currently, no systemic treatment has proven its effectiveness in this pathology.\n\nPercutaneous cryotherapy is a cold thermoablation procedure using fine 17 G needles introduced into the lesion after targeting by imaging. This technique is used in the treatment of soft tissue tumors and desmoid tumors.\n\nThe treatment of neurofibromas with percutaneous cryotherapy is not well known. Encouraging results (unpublished) have been observed in patients with NF1 treated with cryotherapy at the Léon Bérard Center. The beneficial effect was observed in terms of quality of life (in particular, pain) as well as a decrease in tumor size.\n\nOn the basis of this first experience, it appears important to corroborate these preliminary results by a prospective study allowing the use of this technique to treat patients with unresectable or resectable neurofibromas but with mutilating surgery in a NF1 context.",[120,27],"Neurofibroma","2024-02-08",{"date":123,"type":40},"2024-02-09",{"date":125,"type":40},"2022-02-14",{"date":127,"type":21},"2027-02-07",{"name":129,"class":130},"Centre Leon Berard","OTHER"]