Kornimate/medical-research-clean huggingface dataset:
ulipristal acetate in disease charcot-marie-tooth type of 1a

Conditions: CMT1A

Summary: the disease charcot-marie-tooth disease type 1a (cmt1a) is the most common inherited peripheral neuropathy, for which no treatment has proved its effectiveness. it is autosomal dominant, associated with a duplication of the chromosome 17p11.2 region which leads to overexpression of the gene and the protein-peripheral myelin protein-22 (pmp22), a major component of peripheral myelin. in animals and humans, pmp22 mrna level of glutathione s-transferase theta 2 and cathepsin a (markers of oxidative stress), detected in a skin biopsy are markers that may play a role in the prognosis evolution of the disease. furthermore, several studies have shown that the administration of progesterone increased the expression of pmp22 gene (measured in a skin biopsy) and worsening symptoms. in contrast, anti-progestins reduce the synthesis of pmp22 and improve symptoms in rat cmt1a. the long-term safety of anti-progesterone was evaluated for mifepristone (ru486) ulipristal acetate and (ellaone®). few side effects have been reported including a few cases of endometrial hyperplasia reversible upon discontinuation of treatment. with the ru486, rare cases of adrenal androgen and failure have been observed. however, ellaone® has low antagonistic action on the glucocorticoid receptor and no action on androgen receptors. the investigators therefore believe that it will be well tolerated in humans and will reduce the synthesis of pmp22 and the action of oxidative stress by improving disability of patients.


Eligibility criteria:
inclusion criteria: * male 18-70 years * cmt1a proven genetically (17p11.2 duplication) * symptomatic cmt1a (mrc score \<5 in at least one muscle group) * non severe axonal impairment (amplitude of the motor evoked potential on the median nerve and / or ulnar than 50% of normal) * subject contacted with a valid phone number * subject affiliated to a social security scheme * subject has been informed of the results of the medical examination prior exclusion criteria: * another cause of neuropathy: chronic alcohol intoxication, chemotherapy, diabetes, kidney failure, monoclonal gammopathy, cryoglobulin, b12 deficiency, hepatitis b / c, hiv, lyme or poliomyelitis * liver failure * lapp lactase deficiency, malabsoprtion syndrome glucose / galactose * support long-term drug interacting with the cyp3a4 * patients with indication against xylocaine adrenaline * in the biopsy site: surgery, skin disease or local infection * immunosuppression innate or acquired * hypersensitivity to the active substance / excipient * uncontrolled severe asthma * treatment with vitamin c or vitamin b3 in the four weeks preceding randomization * orthopaedic surgery of the lower limbs in the 6 months prior to randomization or planned * against indication xylocaine adrenaline * malfunction of the innate or acquired coagulation

Keywords: physical medicine and rehabilitation neurology
horizons chronic graft-versus-host-disease study

Conditions: Graft Vs Host Disease; Coping Skills; Quality of Life; Stem Cell Transplant Complications

Summary: this research is being done to evaluate the feasibility of the horizons program, a group-based behavioral intervention, to enhance quality of life in patients with chronic graft-versus-host disease.


Eligibility criteria:
inclusion criteria: * underwent allogeneic hematopoietic stem cell transplant * have moderate to severe chronic graft versus host disease * currently receiving care at the sylvester comprehensive cancer center bone marrow transplant program * able to participate in a group intervention in english or spanish language exclusion criteria: * comorbid conditions or cognitive impairment that the treating clinician believes prohibits informed consent or participation in the intervention * vulnerable patients, defined here as prisoners and pregnant women
radiotherapy of multiple brain metastases using aguix®

Conditions: Brain Metastases, Adult; Radiotherapy

Summary: this is a prospective randomized open blinded endpoint phase ii clinical trial. the study will be adaptive: an interim analysis is planned after enrolment of 20 patients in each arm of treatment (wbrt and aguix® + wbrt), to select and continue the study with group(s) that present the best response rate to the experimental treatment (aguix® + wbrt). the main endpoint will be evaluated by a blinded endpoint committee.

the occurrence of brain metastases is a common event in the history of cancer and negatively affects the life expectancy of patients. their incidence varies between 15 and 50% according to the histologic types. surgery, stereotactic radiosurgery, radiotherapy and chemotherapy are the main treatments currently proposed. for patients with multiple brain metastases, whole brain radiation therapy (wbrt) remains the standard of care. however, the median overall survival is less than 6 months and new approaches need to be developed to improve treatment of these patients. in this context, the weak control of the disease comes from three main factors: the multiplicity of the brain lesions, the radioresistance of certain histologies and the poor distribution of cytotoxic agents in brain metastases. the use of radiosensitizing agents is here of great interest. the radiosensitizing agent chosen in this study is aguix®. it is a gadolinium-chelated polysiloxane based nanoparticle developed by nh theraguix company for its theranostic properties (radiosensitization and diagnosis by multimodal imaging). preclinical studies have demonstrated the radiosensitizing effect of aguix® in vivo in 8 tumor models xenografted in rodents, and particularly in brain tumors.

Eligibility criteria:
inclusion criteria: * patients with brain metastases, from a histologically confirmed solid tumor, eligible for wbrt * at least 18 years old * signed informed consent after informing the patient * ecog (eastern cooperative oncology group) performance status 0-2 * extracranial disease: * complete or partial response or stability under systemic treatment * no extracranial disease * or first line of treatment * life expectancy greater than 6 weeks * effective contraceptive method for all patient of childbearing potential * affiliated to a social security regimen exclusion criteria: * leptomeningeal metastasis * evidence of metastasis with recent large hemorrhage * progressive and threatening extracranial disease under systemic treatment * previous cranial irradiation (except stereotactic irradiation) * known contra-indication, sensitivity or allergy to gadolinium * known contra-indication for magnetic resonance imaging * renal insufficiency (glomerular filtration rate ≤ 50 ml/min/1.73m²) * pregnancy or breastfeeding * subject under administrative or judicial control

Keywords: gadolinium nanoparticles aguix
drospirenone only pills and cervical mucus changes: a pre- and post-bariatric surgery study

Conditions: Contraception

Summary: the purpose of this study is to study the effect of drospirenone on cervical mucus change by modified insler score, pre-bariatric surgery and post-bariatric surgery

after being informed about the study and potential risks, all participants giving written informed consent who meet the eligibility requirements will take drospirenone-only pills (4 mg, once daily). cervical mucus by modified insler score will be evaluated on day 4-7 of drospirenone taking. pre-bariatric surgery results will be compared with post-bariatric surgery results.

Eligibility criteria:
inclusion criteria: * thai women aged 18-45 years who are obese and had an appointment for bariatric surgery at chulalongkorn hospital * need contraception * able to use non-hormonal contraception during the study * giving consent exclusion criteria: * pregnancy or history of giving birth within 3 months * breastfeeding within the 6 months * history of using dmpa within 12 months * history of using other types of hormonal birth control pills within 4 weeks * history of bilateral oophorectomy or hysterectomy * suspected ovarian tumor or pathological ovarian cyst * regular cigarette smoking * contraindications to drospirenone

Keywords: drospirenone only pills obesity bariatric surgery roux-en-y gastric bypass cervical mucus
a pilot study of technetium [99tc] methylene diphosphonate in the treatment of psoriatic arthritis

Conditions: Arthritis, Psoriatic

Summary: this study is aim to evaluate the efficacy and safety of technetium \[99tc\] methylene diphosphonate (99tc-mdp, trade name: yunke) in the treatment of psoriatic arthritis.

this is a single-arm, open label, 24 weeks study. patients with psoriatic arthritis get 99tc-mdp 22mg (5.5mg/set, four sets) was injected intravenously once a day for 7 successive days, one course every 4 weeks until week 24.

Eligibility criteria:
inclusion criteria: * 18-70 years of age; * clinical diagnosis of psa according to the caspar classification criteria * active psa at baseline defined as ≥ 3 tender joints and ≥ 3 swollen joints * if subjects were taking csdmards, the doses of csdmards had to be stable for at least 4 weeks and had to remain the same during the trial * the application of bdmards and tsdmards should meet the following requirements: 1. etanercept and its biological analogues: stop at least 4 weeks prior to their baseline visit; 2. other biological: stop at least 6 months prior to their baseline visit 3. tsdmards: stop at least 8 weeks prior to their baseline visit * if subjects were taking glucocorticoids, the doses of gc(prednisone \< 10mg) had to be stable for at least 4 weeks and had to remain the same during the trial; * non steroidal anti-inflammatory drugs: if applied, the dose was stable one week prior to their baseline visit * negative pregnancy test for child-bearing women at screening and baseline * provide written informed consent exclusion criteria: * patients with severe heart, liver, kidney and other important organ diseases * abnormal liver function (alt or ast is 2 times higher than normal) * white blood cell count less than 4,000/mm\^3 (less than 4 x 10\^9/l) * platelet count less than 100,000/mm\^3 (less than 100 x 10\^9/l) * serum creatinine more than or equal to 1.5 mg/dl (less than or equal to 132.6 μmol/l) * pregnancy or breastfeeding women * contraindications to 99tc-mdp therapy and/or known hypersensitivity to 99tc-mdp * participated in other drugs clinical trials within 4 weeks

Keywords: 99tc-mdp
adductor canal block versus liposomal bupivacaine in tka

Conditions: Total Knee Arthroplasty; Post-operative Pain Control

Summary: this is a randomized prospective study of 60 patients undergoing total knee arthroplasty evaluating the clinical outcomes of liposomal bupivacaine periarticular injections (pai) when compared to single shot adductor canal block using bupivacaine hcl.

total knee arthroplasty(tka) is an effective solution for end stage arthritis of the knee; however, post-surgical pain can be difficult to manage and the optimal peri-operative pain management strategy has not been established. this study serves to evaluate the clinical outcomes of liposomal bupivacaine pai when compared to single shot adductor canal block using bupivacaine hcl in patients undergoing a tka. the investigators hypothesized that peri-articular injection with liposomal bupivacaine would not be inferior to an adductor canal block using bupivacaine hcl. a total of 60 patients were recruited. thirty patients were randomized to receive intraoperative liposomal bupivacaine periarticular injection, and another thirty patients randomized to receive a pre-operative adductor canal block using bupivacaine. both groups otherwise received standard pain management strategies peri-operatively. the primary outcome of interest was post-operative pain, which was measured using the visual analog scale (vas). secondary measures included changes in patient function which was measured using the western ontario and mcmaster universities osteoarthritis index (womac), knee range of motion, post-operative ambulation distance, and hospital length of stay.

Eligibility criteria:
inclusion criteria: 1. patients undergoing unilateral primary total knee arthroplasty for end stage osteoarthritis or rheumatoid arthritis. 2. all adults \> 18 years of age. exclusion criteria: 1. not cleared by pcp and appropriate specialists to undergo tka (check no if patient has been cleared.) 2. adults undergoing bilateral primary total knee arthroplasties. 3. women who are pregnant. 4. women who are breastfeeding. 5. inability to receive successful spinal anesthesia. 6. allergy to amide anesthetics. 7. inability to receive iv tranexamic acid. 8. patients who are unable to speak english. 9. patients who are less than 66kg. 10. patients who are opioid dependent. we define opioid dependency as patients who are on a long-acting narcotic or patients who are taking more than six tablets of hydrocodone 5mg or equivalent.
study of stored tumor samples in young patients with brain tumors

Conditions: Brain and Central Nervous System Tumors

Summary: this laboratory study is looking at stored tumor samples in young patients with brain tumors. studying samples of tumor tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in dna and identify biomarkers related to cancer.

the overall objective of this non-therapeutic protocol is to develop and molecularly characterize patient-derived orthotopic xenografts (pdoxs), organoids and in vitro models derived from medulloblastomas, high grade neuroepithelial tumors (hgnet), cns embryonal tumors, atypical teratoid rhabdoid tumors (atrts), choroid plexus carcinomas (cpcs), ependymomas, and gliomas. the investigators will characterize the genome-wide mutation, expression and epigenetic signatures of these models and compare them with the primary tumors from which they were derived, thus creating a well-characterized and invaluable resource for research on these rare and deadly pediatric brain tumors. this will also provide important insights into intratumoral heterogeneity, and molecular abnormalities that may influence the selective pressures driving evolution, and tumor growth as in pdoxs, organoids or in vitro cultures and define the relationship between these abnormalities and tumor histologic and clinical characteristics. this objective will be achieved by applying state-of-the-art dna, rna and epigenome analysis tools to the study of fresh frozen and/or cryopreserved, fixed and cultured tumor cells, pdoxs and organoids. the establishment of patient-derived orthotopic xenografts, organoids and cell cultures from each tumor sample will also allow for in vitro and in vivo analysis of tumor cell growth, signaling and therapeutic response.

Eligibility criteria:
inclusion criteria * tumor may be primary, progressive or recurrent cns tumor including brain and/or spine. all tumor types will be included with a focus on medulloblastoma, hgnet, cns embryonal tumors, gliomas, ependymoma, cpc and atrt tumors. low grade gliomas are currently very challenging to culture and implant but if techniques mature these will also be included. although rare, patients with atrt may present with a primary renal and cns tumor. in these instances samples will be collected from both the kidney and cns tumor for analysis if available. * tumor may be collected at surgery prior to histologic confirmation * age less than 40 years at the time of initial diagnosis. * enrollment in the current version of the institution's banking protocol exclusion criteria * diagnosis of tumor outside the central nervous system. * age greater than or equal to 40 years at the time of diagnosis

Keywords: childhood atypical teratoid/rhabdoid tumor childhood choroid plexus tumor childhood medulloblastoma recurrent childhood medulloblastoma childhood infratentorial ependymoma childhood supratentorial ependymoma recurrent childhood ependymoma recurrent childhood supratentorial primitive neuroectodermal tumor untreated childhood supratentorial primitive neuroectodermal tumor gliomas
prostate cancer diagnosis by multiparametric ultrasound (wholemount)

Conditions: Prostate Carcinoma

Summary: this phase iii trial investigates if perflutren lipid microspheres with ultrasound can be used to diagnose prostate cancer non-invasively. definity (perflutren lipid microspheres) is an ultrasound contrast agent that is typically used for ultrasound bubble studies that involve the heart. definity appears on ultrasound images as tiny gas-filled microbubbles. these microbubbles are about the size of a red blood cell and do not stay in a patient's body for more than several minutes, where they are excreted from the lungs and exhaled back into the air when breathing. definity may enhance ultrasound images of the prostate and help doctors identify prostate cancer on ultrasound images.

primary objectives: i. to collect multiparametric ultrasound (mp-us) data on 70 patients within 30 days of their radical prostatectomy. ii. to correlate the imaging data with whole mount prostatectomy specimens in order to optimize mp-us with a machine learning approach. outline: patients receive perflutren lipid microspheres intravenously (iv) over 5-6 minutes and undergo ultrasound over 30 minutes. patients may receive up to 2 additional doses of perflutren lipid microspheres during ultrasound. after completion of study, patients are followed up for 30 days.

Eligibility criteria:
inclusion criteria: * subject must be scheduled for radical prostatectomy for treatment of prostate cancer * subject must be able and willing to give written informed consent for a contrast enhanced ultrasound study of the prostate prior to prostatectomy * subject must be a male at least 18 years of age when informed consent is obtained exclusion criteria: * participant in a clinical trial involving an investigational drug within the past 30 days * patients with known or suspected hypersensitivity to perflutren, peg, or any other component of definity * previous treatment for prostate cancer, including hormone therapy * clinically unstable, severely ill, or moribund as per treating physician
cdd plus bortezomib or cddin relapsed or refractory multiple myeloma with extramedullary plasmacytoma

Conditions: Extramedullary Plasmacytoma

Summary: the purpose of this study is to determine whether cyclophosphamide, liposome doxorubicin and dexamethasone（cdd) plus bortezomib might have effective in extramedullary plasmacytoma.


Eligibility criteria:
inclusion criteria: * male or female patients from 18 to 80 * biopsy-proven emp with relapsed or refractory myeloma disease. patients may be proteasome inhibitor-exposed or naive, but cannot be refractory to proteasome inhibitor therapy * disease requiring further treatment * measurable disease such as m protein and objective and measurable of emp * eastern cooperative oncology group (ecog) performance status of less than or equal to 2 * meet the clinical laboratories criteria as specified in the protocol * voluntary written consent exclusion criteria: * female patients who are lactating, breastfeeding or pregnant * evidence of current uncontrolled cardiovascular conditions as specified in study protocol * requirement for other concomitant chemotherapy, immunotherapy, radiotherapy, which would be considered as a treatment of emp. * comorbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens * ongoing or active infection, known hiv positive, active hepatitis b or c infection * psychiatric illness/social situations that would limit compliance with study requirements * known allergy to any of the study medications

Keywords: bortezomib
an open-label extension trial of cvl-865 as adjunctive therapy in the treatment of focal onset seizures

Conditions: Seizures

Summary: the purpose of this study is to assess the long-term safety and tolerability of cvl-865 as adjunctive therapy in participants with focal onset seizures.


Eligibility criteria:
inclusion criteria: * participants who completed treatment in trial cvl-865-sz-001 (nct04244175) * a female participant of childbearing potential who is sexually active with a nonsterilized male partner must agree to use a highly effective method of contraception from signing of informed consent through 30 days post last dose * a male participant with a pregnant or a nonpregnant partner of childbearing potential must agree to use a condom during treatment and until the end of relevant systemic exposure in the male participant for 94 days following the last dose with the investigational medicinal product (imp) * participants who are capable of giving signed informed consent * participants who are able, in the opinion of the investigator, to understand the nature of the trial and comply with protocol requirements, including the prescribed dosage regimens, scheduled visits, laboratory tests, and other trial procedures exclusion criteria: * participants who, in the opinion of the investigator, medical monitor, or sponsor, should not participate in the trial * participants who, in the judgment of the investigator, experienced poor tolerability to the imp during the double-blind trial or whose safety assessments resulted in new concerns that would suggest that the participant may not be appropriate for 57 weeks of treatment with cvl-865 in an extension trial * participants who experienced status epilepticus during trial cvl-865-sz-001 * participants who have demonstrated substantial noncompliance to trial procedures in trial cvl-865-sz-001, based on the investigator's judgment, would not be eligible for this trial * participants who answer "yes" on the c-ssrs suicidal ideation item 4 or item 5 (active suicidal ideation with some intent to act, without specific plan, or active suicidal ideation with specific plan and intent), or participants who answer "yes" on any of the 5 c-ssrs suicidal behavior items (actual attempt, interrupted attempt, aborted attempt, preparatory acts, or behavior), or participants who, in the opinion of the investigator, present a serious risk of suicide * participants with any of the following abnormalities in clinical laboratory tests at visit 1, as assessed by the central laboratory and confirmed by a single repeat measurement, if deemed necessary (females: hemoglobin \<11 gram per deciliter (g/dl); males: hemoglobin \<12 g/dl; white blood cell (wbc) count \<3.0 x 10 power 9 per liter (10\^9/l); neutrophil count \<2.0 x 10\^9/l; platelet count \<150 × 10\^9/l) * participants who would be likely to require the use of prohibited concomitant medications during the trial * female participants who have a positive pregnancy test result

Keywords: cvl-865 anti-epileptic drugs (aeds) pf-06372865 γ-aminobutyric acid (gaba) focal epilepsy partial seizure