Kornimate/medical-research-clean huggingface dataset:
sickle cell disease (scd) bone pain study

Conditions: Sickle Cell Disease; Sickle Cell Anemia; Low Bone Density; Osteoporosis; Osteopenia; Vertebral Fracture; Vertebral Compression; Osteonecrosis; Ischemic Necrosis; Avascular Necrosis

Summary: a prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease

the investigators hypothesize that adults with sickle cell disease (scd) and low bone density and/or vertebral compression fractures on a dual x-ray absorptiometry (dxa) scan (adjusted for age, sex, scd genotype, relevant labs, presence of osteonecrosis, and scd-modifying therapies) will report more severe pain than those with normal bone density or no vertebral fractures. the adult sickle cell quality of life measurement system (ascq-me) is a validated patient-reported outcome measure of physical, mental, and social health in adults with scd. this cross-sectional observational study involves obtaining a baseline dxa scan, vertebral fracture analysis (vfa) and pain assessment using ascq-me pain impact scores. the investigators plan to recruit 50 adults with scd followed at university of california davis medical center between nov 2022- dec 2023 and anticipate enrolling up to 4 adults with scd per month. the study endpoints are listed below: * to determine the association between bone density z-scores and ascq-me pain impact scores in a prospective cohort of adults with scd * to study the association between spine deformity index scores (sdi, a proxy for vertebral fracture analysis) and ascq-me pain impact scores in a prospective cohort of adults with scd * to assess the correlation between baseline hematological and biochemical laboratory parameters (including bone biomarkers), bone density, and/or vertebral fractures in a prospective cohort of adults with scd the investigators' goal is to complete primary data analysis by mar 2024. as an exploratory endpoint, 1cc of serum and 5cc of urine will be collected from each study participant once (at baseline), after an overnight fast, for bone biomarker analyses.

Eligibility criteria:
inclusion criteria: * age 18-80 years with scd (any genotype, confirmed by hemoglobin electrophoresis or high performance liquid chromatography) * ability to provide written informed consent * ability to lay on a dxa scanner * negative urine pregnancy test for women of childbearing potential at study entry exclusion criteria: * pregnant women * adults unable to consent * individuals who are not yet adults (infants, children, teenagers) * prisoners * hospitalizations (any cause) within 2 weeks of study entry

Keywords: sickle cell disease sickle cell anemia low bone density osteoporosis osteopenia low bone mass
an extension trial to assess the safety of re-dosing of iron isomaltoside/ferric derisomaltose (monofer®/monoferric®)

Conditions: Iron Deficiency Anemia; Iron Deficiency Anaemia

Summary: evaluate safety and efficacy of intravenous (iv) iron isomaltoside/ferric derisomaltose re-dosing, in subjects who were previously treated with iron isomaltoside/ferric derisomaltose.

among the various formulations of parenteral iron that are currently available, iron isomaltoside/ferric derisomaltose may allow flexibility in terms of high and rapid dosing. up to now, most clinical trials with intravenous (iv) iron treatment were of 4-12 weeks in duration; longer trials are warranted to follow-up on long-term safety. the aim of the trial was to evaluate the safety and efficacy of iv iron isomaltoside/ferric derisomaltose re-dosing in subjects who were previously treated with iron isomaltoside/ferric derisomaltose in lead-in trials. this was a 6-months extension trial lasting 26 weeks. eligible subjects attended 5 visits: screening, baseline (subjects treated with a single iv dose of 1000 mg iron isomaltoside/ferric derisomaltose), and follow-up visits at week 2, 13, and 26 weeks after the iv dose, for safety and efficacy assessments.

Eligibility criteria:
inclusion criteria: 1. completed one of the lead-in trials 2. randomised and dosed with iron isomaltoside/ferric derisomaltose in one of the lead-in trials. 3. haemoglobin (hb) of ≤ 11 g/dl 4. screening serum ferritin (s-ferritin) ≤ 100 ng/ml, or ≤ 300 ng/ml if transferrin saturation (tsat) ≤ 30 % 5. willingness to participate and signing the informed consent form (icf) exclusion criteria: 1. intravenous (iv) iron treatment between the lead-in trial and screening 2. during 30-day period prior to screening or during the trial period; has or will be treated with a red blood cell transfusion, radiotherapy, and/or chemotherapy 3. received an investigational drug within 30 days of screening 4. decompensated liver cirrhosis or active hepatitis 5. pregnant or nursing women. 6. any other laboratory abnormality, medical condition, or psychiatric disorders which, in the opinion of the investigator, will put the subject's disease management at risk or may result in the subject being unable to comply with the trial requirements

Keywords: iron deficiency anemia iron deficiency anaemia ida intravenous iron replacement therapy chronic kidney disease ckd iron isomaltoside ferric derisomaltose monofer monoferric monover monofar monoferro
sort-out vi - randomized clinical comparison of biomatrix flex® and resolute integrity®

Conditions: Coronary Artery Disease; Angina Pectoris

Summary: to perform a randomized comparison between the biomatrix flex™ and the resolute integrity® stents in the treatment of unselected patients with ischemic heart disease.

sort out vi is a randomized, multicenter, all-comer, two-arm, non-inferiority trial comparing the biolimus-eluting biomatrix flex and the zotarolimus-eluting medtronic resolute integrity in treatment of atherosclerotic coronary artery lesions. primary endpoint: target lesion failure within 12 months of stent implantation (combination of cardiac death, myocardial infarction (not index procedure related) not related to other than index lesion or target lesion revascularization). secondary endpoints: individual components of the primary end point comprised the secondary end points and stent thrombosis rate according to the academic research consortium definition (see protocol for further specification of secondary endpoints). clinically driven event detection will be used to avoid study-induced reinterventions. data on mortality, hospital admission, coronary angiography, repeat percutaneous coronary intervention, and coronary bypass surgery will be obtained for all randomly allocated patients from the following national danish administrative and healthcare registries: the civil registration system; the western denmark heart registry; the danish national registry of patients, which maintains records on all hospitalizations in denmark; and the danish registry of causes of death

Eligibility criteria:
inclusion criteria: * patients aged 18 years or over with chronic stable coronary artery disease or acute coronary syndromes, and at least one coronary artery lesion with more than 50% diameter stenosis, requiring treatment with a drug-eluting stent. exclusion criteria: * life expectancy of less than one year * an allergy to aspirin, clopidogrel, ticagrelor, prasugrel, sirolimus, or biolimus * participation in another randomized trial * inability to provide written informed consent

Keywords: percutaneous coronary intervention des angina pectoris stent
remote pulmonary function testing and nurse coaching in als

Conditions: ALS

Summary: comparison of respiratory outcomes in patients receiving telemedicine-guided remote pulmonary function testing (rpft) with or without the additional support of nurse coaching. this is a randomized controlled study which assesses the effects rpft and coaching on respiratory outcomes and quality of life.

this is a two-arm, randomized study to determine 1) whether weekly monitoring of respiratory function can lead to timelier initiation of niv and 2) whether structured nurse coaching leads to improved self-efficacy for managing disease and better maintenance of respiratory health. for enrollees in both arms, standard fvc and mip measurements obtained approximately every three months by the respiratory therapist during als clinic are supplemented with self-administered rpfts performed weekly. enrollees in the rpft+nrhc (nurse respiratory health coaching) arm will additionally receive monthly coaching with the study nurse via telehealth. outcomes include measures of quality of life and self-efficacy for managing disease.

Eligibility criteria:
inclusion criteria: patients: 1. possess a diagnosis of definite, probable, probable laboratory-supported, or possible als by revised el escorial research criteria \[brooks2000\]. 2. be 18 years of age or older. 3. have a caregiver available to participate in the study 4. symptom onset within the last three years. 5. have a computer and home internet service sufficient for engaging in telemedicine sessions. 6. have a second device capable of downloading the spirometer application from an app store (android- or ios-based smartphone or tablet). caregivers: 1. be 18 years of age or older, of either gender. 2. be able and willing to provide informed consent. exclusion criteria: patients: 1. use of niv or diaphragm pacer at time of obtaining informed consent. 2. fvc ≤50% predicted or mip \> -60 cm of water. 3. als functional rating scale (alsfrs-r) \[cedarbaum1999\] score on day of screening of ≥2 on items for speech, swallowing, and salivation. these items are indicators of bulbar dysfunction, which limits the reliability of pft administration. 4. cognitive impairment, as judged by the als clinic neurologist, that prevents participation in the study. caregivers: none

Keywords: telemedicine pulmonary function testing telehealth
binocular amblyopia treatment

Conditions: Amblyopia

Summary: to evaluate the effectiveness of enhanced binocular amblyopia treatments in achieving a more complete and stable recovery.

to date, all studies of contrast-rebalanced binocular amblyopia treatment games used the same 10% per day contrast-increment that was designed for short-term intervention studies lasting 1-4 weeks; i.e., after 18 days of successful game play, both eyes viewed 100% contrast (no contrast-rebalancing).this study will investigate two alternative protocols designed to extend the game treatment period beyond 4 weeks to allow for more complete recovery of visual acuity. the new protocols will be compared with the original 10% contrast-increment game protocol in an 8-week 3-arm rct to determine whether one or both of the new protocols is more effective than the current 10% increment. based on pilot data, we expect about 40% of amblyopic children to achieve normal visual acuity in 8 weeks. in an effort to also promote recovery of binocular vision, children who attain ≤0.4 logmar at 8 weeks will be enrolled in stereoacuity training at the 8-week visit for the next 4 weeks. children with residual amblyopia (bcva \>0.4 logmar) at the 8-week visit will be provided with dichoptic movies for additional amblyopia treatment for another 4 weeks.

Eligibility criteria:
inclusion criteria: * age 4-10 y * male and female * strabismic, anisometropic, or combined mechanism amblyopia * amblyopic eye best-corrected visual acuity 0.3-0.8 logmar * fellow eye best-corrected visual acuity ≤0.1 logmar * interocular visual acuity difference ≥0.3 logmar * wearing glasses (if needed) for 8 weeks or no change visual acuity with glasses at 2 visits * 4 weeks apart. * child's ophthalmologist and family must be willing to forgo patching treatment during the 12-week study period * parent's informed consent * child must demonstrate understanding and ability to play binocular games exclusion criteria: * prematurity ≥8 weeks * coexisting ocular or systemic disease * developmental delay * strabismus \>5pd * any binocular amblyopia treatment in the past 3 months
14 vs 24 weeks hcv treatment to genotype 2/3 patients with rapid virological response

Conditions: Hepatitis C Virus Infection

Summary: patients with hcv genotype 2 or 3 infection who have a rapid virological response to treatment are randomised to either 14 or 24 weeks hcv treatment. our hypothesis is that there is no important difference in effect between the two treatment effect.

patients with hcv genotype 2 or 3 infection are currently recommended 6 months treatment with pegylated interferon alfa (2a or 2b) and ribavirin.approximately 80% obtain sustained virological response (hcv rna undetectable 6 months after treatment) to this approach. however, the treatment is associated with many and sometimes serious side effects. in addition, the treatment is costly also in econimical terms. increasing the treatment duration beyond 6 months does not increase the response rate. shorter treatment has only been assessed in small trials, but the results have been encouraging. in this randomised, open label,multicenter phase 3 trial with acitive controls patients are treated with pegylated interferon alfa 2a (pegintron (r), schering plough nj)(1,5 mcg/kg)and ribavirin (rebetol (r), schering plough, nj) (800-1400mg based on weight)for 4 weeks. those who are hcv rna negative at week 4 (\<50 iu; cobas amplicor monitor test, roche diagnostic) are defined as rapid virological responders and randomised to either an additional 10 or 20 weeks combination treatment. patients who are hcv rna positive are all treated for 20 more weeks. the endpoint is sustained virological response defined as undetectable hcv rna 24 weeks after end of treatment. our hypothesis is that there is no important difference in the effect in the two groups. this is a non-inferiority trial. the smallest difference considered to be clinically important is 10%. thus to state "non-inferiority" the 95% confidence interval of the observed difference between the groups shall not overlap 10%. both intention to treat and and per protocol analyses will be published. conclusion will be conservative and based on the analysis who detect the biggest difference.

Eligibility criteria:
inclusion criteria: hcv rna positive genotype 2 or 3 treatment naive raised alt - exclusion criteria: active substance abuse poorly controlled psychiatric disease decompensated cirrhosis hbsag positive anti-hiv positive suffering from other significant concurrent medical conditions including chronic liver diseases -

Keywords: interferon pegylated interferon pegylated interferon alfa 2b hepatitis c genotype 2 genotype 3 rapid virological response short treatment 14 weeks treatment health related quality of life sick leave injecting drug use
international, multicenter study of a twenty-eight week, open-label, titrated oral lixivaptan administration in patients with chronic hyponatremia: extension to studies ck-lx3401, 3405, and 3430

Conditions: Hyponatremia With Normal Extracellular Fluid Volume; Hyponatremia With Excess Extracellular Fluid Volume

Summary: to evaluate the overall safety and continued efficacy of oral lixivaptan capsules in subjects with euvolemic and hypervolemic hyponatremia

phase i and phase ii clinical trials have demonstrated that lixivaptan may play an important role in treating hyponatremia and the signs and symptoms of water retention associated with hf, lcwa and siadh. lixivaptan was previously evaluated in disease states characterized by hyponatremia with euvolemia (siadh) and hyponatremia combined with fluid overload (hf, lcwa). lixivaptan demonstrated correction in serum sodium concentration together with marked aquaresis in patients with hyponatremia.

Eligibility criteria:
inclusion criteria: 1. men and women 18 years of age or older 2. ability to provide informed consent or assent 3. prior participation in a lixivaptan hyponatremia trial with evidence of continued need or desire for therapy exclusion criteria: 1. a current medical condition where long-term treatment with an aquaretic agent may present an undue risk to the patient 2. hyponatremia which is acute, reversible, artificial or due to conditions not associated with vasopressin excess or likely to respond to aquaretic therapy 3. hyponatremia due to reversible medical condition or therapy 4. conditions associated with an independent imminent risk of morbidity and mortality

Keywords: euvolemic hyponatremia hypervolemic hyponatremia siadh serum sodium
individual patient compassionate use of fedratinib

Conditions: Myelofibrosis

Summary: this is an expanded access program (eap) for eligible participants designed to provide access to fedratinib. expanded access is only available in markets where fedratinib is not yet approved.

this program is being offered on a patient by patient basis and will require company, institutional review board/independent ethics committee and single patient ind approval.

Eligibility criteria:
inclusion criteria: subjects will be considered if they received tg101348, sar302503 or fedratinib as a participant on a clinical trial prior to 31 jan 2018. exclusion criteria: subject is eligible for enrollment in an ongoing clinical trial using fedratinib or has been treated with fedratinib after 31 jan 2018.

Keywords: expanded access compassionate use
clinical outcomes of open wedge high tibial osteotomy with autologous bone marrow or adipose-derived stem cell therapy

Conditions: Osteoarthritis; Genu Varum

Summary: the purpose of this research is to confirm the efficacy of cartilage regeneration (or stem cell transplant) simultaneously performed with high tibial osteotomy. furthermore, as conventional microfracture surgery on injured cartilage has a disadvantage of the replacement by fibrocartilage, this research intends to prove the excellence over the conventional therapy by proving a hyaline cartilage regeneration of injured cartilage by stem cell.

the prevalence rate of arthritis in medial knee joint is increasing with an increase in senile diseases and there are two main types of therapy: total knee replacement surgery and high tibial osteotomy. generally, the elderly prefer total knee replacement surgery and younger people prefer high tibial osteotomy. high tibial osteotomy is a procedure to reduce pain and to prevent the progression of osteoarthritis by evenly dispersing excessive load on medial knee joint attributed to varus deformity on the overall knee joint through the correction of abnormal anatomical axis arising from degenerative changes in articular cartilage. the clinical outcomes of high tibial osteotomy are reported as favorable but this high tibial osteotomy has a limitation in terms of the regeneration of injured cartilage. thus, a research to concurrently perform additional therapy for the regeneration of injured cartilage with the osteotomy was released. microfracture surgery on injured cartilage, autologous chondrocyte implantation, autologous bone marrow injections and adipose derived stem cell injections are typical examples. however, there is not any solid proof for the excellence of concurrent therapy over a therapy using only the osteotomy in the regeneration of injured cartilage. therefore, this research is designed to conduct a research to prove the excellence of the direct transplantation of stem cell into injured cartilage over the existing concurrent therapy in the regeneration of injured cartilage. besides, this research intends to compare the cartilage regeneration capabilities of bone marrow and adipose-derived stem cell.

Eligibility criteria:
inclusion criteria: 15 or more to 65 years of age or younger classification of degenerative arthritis: kellgren-lawrence grade i, iii, iii arthritis lesion limited to medial knee joint varus deformity of knee joint of 15 degrees or less bmi under 35 exclusion criteria: more than 65 years of age classification of degenerative arthritis: kellgren-lawrence grade iv lesions of arthritis (kellgren-lawrence grade iii, iv) found in lateral knee joint varus deformity of knee joint more than 15 degrees bmi above 35 operation history - surgery of microfracture and fracture, and ligament regeneration history of intraarticular injection of steroid and hyaluronic acid in the last 3 months
outcome of 15 versus 5 fractions in adjuvant breast radiotherapy in women over 65 years

Conditions: Breastcancer

Summary: this study will evaluate the effect of highly accelerated external beam radiotherapy (ebrt) in 5 fractions over 10 days compared to 15 fractions over 3 weeks for early as well as locally-advanced stage breast cancer. primary endpoint will be chronic toxicity (breast deformation and retraction). secondary endpoints are acute toxicity, loco-regional and distant tumor control, patient reported qol and cosmetic satisfaction. patients with lymph node irradiation will be closely monitored for radiation induced plexopathy.

over the age of 65, uptake of adjuvant radiotherapy after surgery for breast cancer declines, even in women with poor prognostic stage or adverse tumor characteristics. lowering the number of fractions may reduce reluctancy for radiotherapy in this age group. in a preceding prospective phase i-ii trial, evaluating ebrt over 10 days, results on technical feasibility and acute toxicity were positively evaluated. however, data on the chronic effects of breast irradiation using high doses per fraction are still sparse. this trial is developed to compare standard hypo-fractionation in 15 fractions (if needed with simultaneously integrated boost - sib) with highly accelerated radiotherapy in 5 fractions. patients referred for adjuvant radiotherapy after breast conserving surgery will preferably be randomized, depending on the indication, to a study-arm with whole breast irradiation (wbi) +/- sib or to a study-arm with wbi+/- sib and lymph node irradiation (lni). if patients after breast conserving surgery (bcs) have a strong preference for 5 or 15 fractions, they will be invited to enter the parallel observational arm (patient preference). patients referred for adjuvant radiotherapy after mastectomy +/- lni accepting to participate, can choose a 5 or 15-fraction schedule. no randomization is foreseen in this last observational group.

Eligibility criteria:
inclusion criteria: * breast conserving surgery or mastectomy for breast carcinoma * multidisciplinary decision of adjuvant irradiation * absence of distant metastases * informed consent obtained, signed and dated before specific protocol procedures exclusion criteria: * history of previous radiation treatment to the same region * bilateral breast irradiation * life expectancy of less than 2 years * planned reconstructive breast surgery * condition making toxicity evaluation difficult, e.g. skin disorders, pre-existing brachial plexus injury, ... * inability to respect constraints on skin, heart, lungs, esophagus, ribs, brachial plexus or any other organ at risk * mental condition rendering the patient unable to understand the nature, scope and possible consequences of the study * patient unlikely to comply with the protocol; i.e. uncooperative attitude, inability to return for follow-up visits or unlikely to complete the study

Keywords: accelerated radiotherapy older women hypo-fractonation