Kornimate/medical-research-clean huggingface dataset:
expanded access program for r007 (probucol) tablets in adults with mitochondrial disease and chronic kidney disease

Conditions: Mitochondrial Disease; Chronic Kidney Disease

Summary: the purpose of this expanded access program is to enable access to r007 (probucol) tablets for the compassionate treatment of adults with mitochondrial disease who also have chronic kidney disease.

the protocol for this expanded access program provides for the compassionate treatment with r007 (probucol) tablets of adults who (i) have serious or life-threatening mitochondrial disease and also have chronic kidney disease stages 3 or 4, (ii) do not have access to a satisfactory alternative therapy to treat their condition, and (iii) are not able to enroll in a clinical trial.

Eligibility criteria:
inclusion criteria a patient must meet all of the following criteria to be eligible: 1. age 18 to 75 years inclusive; 2. diagnosis of genetically-confirmed mitochondrial disease with a pathogenic mutation or defect in their mitochondrial or nuclear dna; 3. diagnosis of chronic kidney disease stage 3 or stage 4; 4. moderate or severe symptomatology of mitochondrial disease; 5. a sexually-active patient agrees to practice acceptable methods of contraception throughout the protocol period; 6. the patient is not eligible for, or does not have access to, a clinical trial; 7. the patient (or guardian/legal representative) provides signed and dated informed consent to be treated with probucol in accordance with the expanded access protocol. exclusion criteria a patient who meets any of the following criteria will not be eligible: 1. a known hypersensitivity or adverse reaction to probucol; 2. a medical condition or laboratory result that, in the opinion of the treating physician, will interfere with the safe completion of the protocol; 3. an active fungal infection, acute blood, lung, or bladder infection, clinically significant hepatic or renal dysfunction, and/or viral infection (past or present) that, in the opinion of the treating physician, may have a clinically significant effect on their treatment; 4. taking an investigational drug other than probucol within one month prior to the first dosing with probucol under the protocol; 5. a medical history or condition that increases the potential for qtc prolongation; 6. a female with a positive pregnancy test result or who is breastfeeding; 7. has received or is receiving kidney dialysis; 8. has received or is scheduled to receive an organ transplant; 9. known or suspected active alcohol and/or substance abuse; 10. a history of or current suicidal ideation, behavior, and/or attempts; or 11. is unable to swallow tablets or requires gastric feeding.

Keywords: mitochondrial disease chronic kidney disease myopathy genetic disease rare disease probucol
impact of oral phosphorus supplements on the 6-month change in fgf23 levels in anorexic adolescents suffering from undernutrition.

Conditions: Malnutrition

Summary: fibroblast growth factor 23 (fgf23) is a recently discovered phosphaturic hormone produced by osteocytes. it is involved in phosphate-calcium metabolism (via its phosphaturic action and inhibition of 1,25-oh vitamin d). there are no published studies on the role of fgf23 in undernourished anorexic adolescents taking oral phosphorus supplements.


Eligibility criteria:
inclusion criteria: * adolescents aged from 11 to 17 * follow-up or hospitalisation for anorexia * overt undernutrition (clinical signs of undernutrition, body mass index and the waterlow index) * social security coverage exclusion criteria: * known pre-existing renal disorders * known constitutional disorders of phosphate-calcium metabolism * adult patients, legal guardianship, incarceration

Keywords: phosphorus supplementation fgf23 anorexic adolescents undernutrition
mindfulness-based behavioural therapy (mibt) versus psychodynamic therapy for patients with major depressive disorder in psychotherapeutic day treatment. a randomised clinical pilot trial

Conditions: Major Depressive Disorder; Personality Disorder

Summary: background: according to the who, major depressive disorder is the second largest healthcare problem worldwide in terms of disability caused by illness. it afflicts an estimated 17% of individuals during their lifetimes at tremendous costs. a number of depressive patients are treated with antidepressant medication. the efficacy of antidepressant medication has been studied in a number of systematic reviews, and in recent years some of these reviews have shown that the efficacy is questionable for many patients. so are there other effective treatments for this serious illness? cognitive- and psychodynamic therapies are probably both significantly more effective for depression than no treatment, but only limited comparisons have been made between the two interventions. a cochrane review shows that cognitive therapy has a preventive effect against recurrent depression, and that this effect may surpass the preventive effect of antidepressant medication. mindfulness training may be an effective technique in preventing relapse in patients who have had at least 3 previous depressive episodes. but efficacy in treating currently depressed patients has not been studied. objective to perform a randomised clinical trial with blinded assessment of efficacy variables in order to study the effects of mindfulness based behavioral therapy (cognitive therapy and mindfulness) versus psychodynamic therapy in depressive patients. methods a randomised clinical trial of 84 consecutive patients diagnosed with major depressive disorder, referred to the day clinic, roskilde psychiatric services. the patients will be randomised to one of two interventions: 1. mibt (mindfulness-based behavioural therapy) 2. pt (psychodynamic therapy)


Eligibility criteria:
inclusion criteria: 1. aged 18 to 65 years 2. major depressive disorder (scid i). 3. bdi ii \> 13. 4. written informed consent. exclusion criteria: 1. current psychosis, diagnosis of schizophrenia or schizotypal personality disorder (dsm iv-tr). 2. alcohol or substance abuse judged to require treatment in preference to depression (assessed during patient conference). 3. commenced or changed psychopharmacological treatment less than six weeks before randomisation. 4. pregnancy. 5. no written informed consent.
use of airtime incentives to improve short message service surveys in colombia and tanzania

Conditions: Surveys and Questionnaires; Noncommunicable Diseases

Summary: this study evaluates the effect of four different airtime incentive amounts on short message service (sms) survey cooperation, response, refusal and contact rates, as compared to control group, in colombia and tanzania.

using random digit dialing (rdd) sampling technique, participants were randomized to one of four airtime incentive amounts contingent on them completing the noncommunicable disease risk factor survey. this mobile phone survey will be sent as a short message service (sms). in sms surveys, participants use their touch tone key pad to answer written questions. (i.e. if you are male, press 1; if you are female, press 2). this study will be conducted in both colombia and tanzania.

Eligibility criteria:
inclusion criteria: * access to a mobile phone * greater or equal to 18 years of age * in colombia, conversant in the spanish language. in tanzania, conversant in the swahili language. exclusion criteria: * less than 18 years of age

Keywords: mobile phone surveys short message service incentive
efficacy and safety of escitalopram doses up to 50mg in treatment of mdd

Conditions: Major Depressive Disorder

Summary: this will be an open label study of escitalopram. patients not responsive to citalopram will be switched directly to escitalopram. patients will receive escalating doses of escitalopram up to a maximum of 50 mg until they either achieve remission (madrs \<9) or fail to tolerate the dose.

patients will receive escalating doses of escitalopram up to a maximum of 50 mg until they either achieve remission (madrs \<9) or fail to tolerate the dose. visit 1 - (initial visit) - escitalopram 10 mg visit 2 - (week 2) - escitalopram 20 mg visit 3 - (week 4) - review visit visit 4 - (week 6) - madrs \ 12 - escitalopram 30 mg visit 5 - (week 8) - madrs \ 8 - escalate dose (20 mg to 30 mg or 30 mg to 35mg) thereafter, patients who have achieved remission will be maintained on the remission dosage and reviewed at four weekly intervals. at any subsequent visit where the madrs is \>8 they will have a dosage increase patients who have not achieved remission will have dosage escalated by 5 mg at two weekly intervals until remission, a maximum dose of 50 mg is achieved or the dosage is intolerable when they will be reduced to the previous tolerable dose. patients will be followed up until eight months from their initial visit.

Eligibility criteria:
inclusion criteria: * written informed consent will be obtained from each patient * aged 18 to 65 inc * suffering from mdd as defined by dsm iv * have been taking citalopram in a dose of at least 20mg for at least six weeks * an inadequate response -- defined as failure to achieve a madrs score of \<12 exclusion criteria: * significant other psychiatric disorder which would interfere with trial assessments. co-morbid generalized anxiety disorder (gad) and panic will be permitted where mdd is considered the primary diagnosis . * history of mania or bipolar disorder * known contraindication for the use of citalopram or escitalopram. * significant bleeding disorder * prominent suicidal ideation (score more than 4 in the madrs "suicidal thoughts" item) * alcohol or substance dependence in the past 6 months * major physical illness * significant liver or renal function abnormality * significant ecg abnormalities * pregnant or lactating females * inadequate contraception

Keywords: mdd escitalopram
a retrospective observational study of patients with early-stage her2-positive breast cancer, treated with neratinib

Conditions: Breast Cancer; Adjuvant Therapy; HER2-positive Breast Cancer

Summary: the purpose of this study is to describe the demographic and clinical profiles of patients with early-stage her2+ breast cancer treated with neratinib as an extended adjuvant therapy as part of the early access program (eap) in europe.


Eligibility criteria:
inclusion criteria: * patients will be eligible for inclusion if they fulfill all of the following criteria: * age ≥ 18 years at neratinib treatment initiation * having received at least one initial dose of neratinib as an extended adjuvant therapy for the treatment of early-stage her2+ breast cancer, in the context of the eap in europe, and between august 01, 2017 and december 31, 2020 * patients (or next of kin/legal representative, if applicable) who provide written informed consent or non-opposition. exclusion criteria: * n/a

Keywords: her2-positive breast cancer adjuvant therapy
evaluation of a mandibular advancement device in the treatment of obstructive sleep apnea syndrome

Conditions: Obstructive Sleep Apnea

Summary: the purpose of this study is to determine if the mandibular advancement by herbst device is effective in the treatment of moderate obstructive sleep apnea patients.


Eligibility criteria:
inclusion criteria: * moderate sleep apnea syndrome (10 \< respiratory disorder index \< 30) * body mass index (bmi) \< 35 kg/m2 * able to give their informed consent exclusion criteria: * pregnant women * professional drivers requiring continuous positive airway pressure (cpap) treatment * psychiatric disorders, and patients unable to realise to study * severe respiratory pathology which could interfere with the study * morpheic epilepsy * benzodiazepines intake * chronic nasal obstruction * ear, nose, and throat (ent) pathology requiring surgery * previous uvulopalatoplasty surgery * temporomandibular joint pathology * gum disease * insufficient number of teeth to apply the oral appliance * mobile teeth

Keywords: prospective randomized polysomnography orthodontic appliance, removable
amx0035 in adult patients with wolfram syndrome

Conditions: Wolfram Syndrome

Summary: this study is an open label phase ii study to evaluate the safety and efficacy of amx0035 in adults with wolfram syndrome.

amx0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (er). this clinical trial is designed to demonstrate that treatment is safe, tolerable, and to evaluate the effect of amx0035 on residual beta cell functions by monitoring c-peptide levels during a 0-240 minute mixed-meal tolerance test. the trial will also assess the effects of amx0035 on changes to diabetic measurements including daily insulin dose, time in good glucose range, and hba1c levels. effect on best-corrected visual acuity in both eyes will also be evaluated.

Eligibility criteria:
key inclusion criteria: * definitive diagnosis of wolfram syndrome * insulin dependent diabetes mellitus due to wolfram syndrome * at least 17 years of age * participant must be willing to wear a cgm device for the duration of the study key exclusion criteria: * presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis) * any history of heart failure per new york heart association (nyha) * history of or family history of breast and/or ovarian cancer * participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the investigator's judgment * received treatment with any investigational drug or device within the 30 days (or 5 half-lives, whichever is longer) prior to first dose at day 1 * previous treatment with gene or cellular therapy

Keywords: wolfram wolfram syndrome
comparative study of the quality of life of patients suffering from ototoxicity due to chemotherapy based on platinum salts fitted with a hearing aid compared to those not fitted.

Conditions: Cancer; Ototoxicity, Drug-Induced; Quality of Life; Hearing Disorders

Summary: there are many undesirable effects associated with platinum-based cancer treatments (renal failure, anaemia, etc.). their administration also leads to neurosensory problems such as ototoxicity, tinnitus and reduced hearing acuity. according to a the french survey (2018), 39.7% of people suffer from hearing problems due to cancer treatments, five years after a cancer diagnosis. improving side effects such as hypoacusis and tinnitus can significantly improve patients' quality of life and adherence to treatment. many clinical trials proposed a medicinal solution to patients receiving platinum-based cancer treatments but none has led to a consensus on management. the aim of the study is to offer patients receiving platinum-based chemotherapy and suffering from hearing problems a hearing aid to improve their quality of life.


Eligibility criteria:
inclusion criteria: * adult patient * patient currently undergoing treatment with platinum-based chemotherapy and suffering from hypoacusia consistent with the treatment or presenting a worsening of already existing hypoacusis consistent with the start of treatment with platinum-based chemotherapy * patient whose hypoacusis is confirmed by the audiometric test * patient able and willing to follow all study procedures in accordance with the protocol. * patient having understood, signed and dated the consent form * patient affiliated to the social security system exclusion criteria: * pregnant or breastfeeding woman * persons deprived of liberty or under guardianship (including curatorship) * impossibility of submitting to medical monitoring of the trial for geographical, social or psychological reasons * patient with a contraindication to wearing hearing aids * patient already fitted * patient already included in a protocol including an experimental molecule * patient who has not started treatment with platinum-based chemotherapy * patient presenting only tinnitus without hearing loss
the use of cryotherapy to prevent paclitaxel-induced peripheral neuropathy and nail changes in women with breast cancer

Conditions: Cryotherapy Effect; Peripheral Neuropathy; Nail Toxicity

Summary: this study will determine the efficacy of cryotherapy to prevent paclitaxel-induced peripheral neuropathy and nail changes in women with breast cancer.

women with breast cancer receiving paclitaxel will complete the brief pain inventory(bpi), neuropathic pain syndrome inventory (npsi)while a member of the research team will conduct an assessment of their nails. once complete, cryotherapy will be administered 15 minutes prior to the paclitaxel infusion, continuously during and then 15 minutes after completion of the paclitaxel for a total of 90 minutes. cyrotherapy will be delivered using elasto-gel mitts and rapid relief slippers. these items will be changed 45min after initiation to ensure coldness. there will be two arms of the study. one arm will include subjects who will wear one slipper/one mitt on their foot/hand while the other foot/hand will serve as a control. the other arm will include subjects who will have cryotherapy to both hands and feet. . this process will be repeated for a total of 12 times in conjunction with 12 doses of paclitaxel. at time point 13, during a routine visit to their oncologist, the subjects nails will be reassessed along with completion of the bpi and npsia total of 25 subjects will be enrolled in each arm.

Eligibility criteria:
inclusion criteria: * females 18 or older * histologically confirmed diagnosis of breast cancer * receiving neo-adjuvant or adjuvant dose-dense anthracycline (ac) plus taxane-based chemotherapy or paclitaxel in combination with trastuzumab and pertuzumab exclusion criteria: * prior taxane therapy * prior oxaliplatin therapy * non-english speaking * history of peripheral neuropathy, i.e., buerger's disease * history of diabetes mellitus * pre-existing peripheral neuropathy from other conditions, e.g., raynaud's disease, guillain-barre, miller fisher syndrome * hand, nail or foot conditions, e.g., hand/foot syndrome, arthritis, hammer toe, carpal tunnel, bunions, deformities * alcohol abuse (history/current) * current medication usage of opioids, anti-depressants, anti-convulsants, glutamines, vitamin b12
vac-stent registry

Conditions: Perforation of the Oesophagus; Perforation of the Colon

Summary: this registry is intended to collect data on quality assurance and use of the vac-stent® and thus verify the suitability of the vac-stent® for sealing leakages in the oesophagus or colon. furthermore, the safety and efficacy of the vac-stent® shall be proven.

for this registry, quality assurance data will be prospectively collected from all patients who receive the vac-stent®. the vac-stent® combines a vacuum sponge, which lies intraluminal and conditions the wound, with a covered stent, which seals the sponge from the lumen and thus ensures passage. due to its design, the new medical device vac-stent® offers a solution for the most important problems of the covered stent, the stent migration and the lack of drainage function, as well as for those of the endoluminal vacuum sponge, the blockage of the gastrointestinal lumen and the difficult application. the vac-stent® is the technical advancement for all indications of the covered stent and for the indication of the endoluminal vacuum sponge, so that a very good effectiveness with reduced complications can be expected for these clinical situations. the vac-stent® therefore offers the prospect of an innovative further development in the endoscopic treatment of leaks in the gastrointestinal tract, without any new and unknown risks being associated with it. the primary aim of this registry is to collect data on the quality assurance and application of the vac-stent® and thus to evaluate the suitability of the vac-stent® for sealing leaks in the oesophagus or colon. in addition, the safety and efficacy of the vac-stent® will be demonstrated.

Eligibility criteria:
inclusion criteria: * written informed consent * treatment with the vac-stent® exclusion criteria: - accommodation in an institution under court or administrative order

Keywords: perforation oesophagus oesophageal anastomotic leak endoscopic vacuum therapy perforation colon
alpha radiation emitters device for the treatment of cutaneous, mucosal or superficial soft tissue neoplasia (dart)

Conditions: Skin Cancer; Mucosal Neoplasm of Oral Cavity; Soft Tissue Neoplasm

Summary: a unique approach for cancer treatment employing intratumoral diffusing alpha radiation emitter device for superficial cutaneous, mucosal or soft tissue neoplasia

this will be a prospective, open label, single arm, controlled study, assessing the safety and efficacy of diffusing alpha emitters radiation therapy (dart) delivered through radioactive seeds inserted into the tumor. this approach combines the advantages of local intratumoral irradiation of the tumor, as used in conventional brachytherapy, with the power of the alpha radiation emitting atoms, that will be introduced in quantities considerably lower than radiation therapy already used in patients. superficial lesions with histopathological confirmation of malignancy will be treated using dart seeds. reduction in tumor size 70 days after dart insertion will be assessed. safety will be assessed by the incidence, severity and frequency of all adverse events (ae).

Eligibility criteria:
inclusion criteria: * subjects with histopathological confirmation of primary or secondary malignant cutaneous neoplastic lesions, or oral cavity mucosal tumors, or superficial soft tissue sarcoma. * subjects with a tumor size ≤ 7 centimeters in the longest diameter. * patients who have either failed first-line treatment, or are medically unfit for standard of care (surgery, external-beam radiation therapy or chemotherapy), or refuse standard of care. * subjects' ecog performance status scale is \< 2. * subjects' life expectancy is more than 6 months. * platelet count ≥100,000/mm3. * international normalized ratio of prothrombin time ≤1.8. * creatinine ≤1.9 mg/dl. * women of childbearing potential (wocbp) will have evidence of negative pregnancy test. * subjects are willing to sign an informed consent form. exclusion criteria: * subject has a tumor of keratoacanthoma histology. * patients with significant comorbidities that the treating physician deems may conflict with the endpoints of the study (e.g., poorly controlled autoimmune diseases, vasculitis, etc.) * patients undergoing systemic immunosuppressive therapy excepting intermittent, brief use of systemic corticosteroids * volunteers participating in another interventional study in the past 30 days which might conflict with the endpoints of this study or the evaluation of response or toxicity of dart. * high probability of protocol non-compliance (in opinion of investigator) * women who are pregnant or breastfeeding.

Keywords: squamous cell carcinoma scc skin cancer skin metastasis hnscc carcinoma, squamous cmn basal cell carcinoma superficial sarcoma kaposi sarcoma alpha radiation cutaneous lesion tongue cancer lip cancer brachytherapy