Kornimate/medical-research-clean huggingface dataset:
expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload

Conditions: Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Myelofibrosis

Summary: this is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.


Eligibility criteria:
inclusion criteria: * male or female patients greater than or equal to 2 years of age * documented congenital disorder of red blood cells (e.g., β-thalassemia major, sickle cell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions * cannot be adequately treated with a locally approved iron chelator due to one of the following reasons: * documented non-compliance, defined as having taken less than 50% of the prescribed chelation therapy doses in the 12 months prior to study entry * contraindications, unacceptable toxicities and/or documented poor response to locally approved iron chelators despite proper compliance * history of at least 20 blood transfusions (equivalent to 100 ml/kg of packed red blood cells (prbc\]) * serum ferritin value greater than or equal to 1000 µg/l * ability to comply with all study-related procedures, medications, and evaluations exclusion criteria: * ongoing treatment with another iron chelator (any other iron chelation therapy must be discontinued at least 24 hours prior to study entry.) * patients who meet the eligibility criteria for any other ongoing novartis sponsored clinical study protocol with deferasirox and who have geographic access to these sites * patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with deferasirox * serum creatinine above the upper limit of normal at screening. * patients with alt ≥ 500 u/l at screening. * evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline * pregnancy (as indicated by serum β-hcg pregnancy test at screening for all female patients with the potential to become pregnant) and patients who are breastfeeding * patients treated with systemic investigational drug within 4 weeks prior to or with topical investigational drug within 7 days prior to the baseline visit other protocol-defined inclusion/exclusion criteria may apply.

Keywords: deferasirox congenital anemias anemias red blood cell disorders chronic iron overload transfusional iron overload iron chelators oral iron chelators thalassemia sickle cell disease diamond blackfan anemia myelofibrosis icl670a
a study to assess the safety and effectiveness of tacrolimus cream in the treatment of psoriasis

Conditions: Psoriasis

Summary: a study to assess the safety and effectiveness of tacrolimus cream in the treatment of psoriasis.


Eligibility criteria:
inclusion criteria: * clinical diagnosis of plaque psoriasis exclusion criteria: * skin disorder other than plaque psoriasis in the areas to be treated. * disease on only elbows, knees, and scalp

Keywords: treatment outcome tacrolimus safety psoriasis
efficacy, safety, and tolerability of eluxadoline in the treatment of participants with diarrhea-predominant irritable bowel syndrome (ibs-d)

Conditions: Irritable Bowel Syndrome

Summary: the purpose of this study is to determine the efficacy, safety, and tolerability of different doses of eluxadoline (jnj-27018966) compared with placebo in the treatment of participants with diarrhea-predominant irritable bowel syndrome.


Eligibility criteria:
inclusion criteria: 1. participant has a diagnosis of irritable bowel syndrome (ibs) with a subtype of diarrhea defined by the rome iii criteria. 2. participant has had a colonoscopy performed: * within 10 years prior to prescreening if participant is at least 50 years of age (sigmoidoscopy, double contrast barium enema, or computed tomography (ct) colonography within the past 5 years is acceptable) * since the onset (if applicable) of any of the following alarm features for participants of any age: * participant has documented weight loss within the past 6 months * participant has nocturnal symptoms * participant has a familial history of first-degree relatives with colon cancer or * participant has blood mixed with their stool (excluding blood from hemorrhoids). 3. female participants must be: * postmenopausal, defined as 52 years or older and amenorrheic for at least 2 years at prescreening, * surgically sterile (have had a hysterectomy or bilateral oophorectomy, tubal ligation, or otherwise be incapable of pregnancy), * abstinent, or * if sexually active, be practicing an effective method of birth control. exclusion criteria: 1. participant has a diagnosis of ibs with a subtype of constipation, mixed ibs, or unsubtyped ibs by the rome iii criteria. 2. participant has a history of inflammatory or immune-mediated gastrointestinal (gi) disorders including inflammatory bowel disease (ie, crohn's disease, ulcerative colitis) and celiac disease. 3. participant has a history of diverticulitis within 3 months prior to prescreening. 4. participant has a history of intestinal obstruction, stricture, toxic megacolon, gi perforation, fecal impaction, gastric banding, bariatric surgery, adhesions, ischemic colitis, or impaired intestinal circulation (eg, aortoiliac disease). 5. participant has any of the following surgical history: * cholecystectomy with any history of post cholecystectomy biliary tract pain * any abdominal surgery within the 3 months prior to prescreening * participant has a history of major gastric, hepatic, pancreatic, or intestinal surgery (appendectomy, hemorrhoidectomy, or polypectomy greater than 3 months post surgery are allowed) other protocol-specific eligibility criteria may apply.
periperal blood lymphocytes during prostate rt

Conditions: Prostate Cancer

Summary: right now it is not possible to predict which patients are going to react more to rt. many things could affect patient's response to radiation. one of the factors is whether the person can repair damage to the genetic material (dna/rna) caused by the rt. there is some evidence that a certain protein called gamma h2ax is involved in dna damage and repair and this can be used to predict the sensitivity of individual patients. the investigators will be studying this protein: gamma h2ax) in the blood samples of prostate cancer patients exposed to both computed tomography (ct) scan and rt treatment. this will help us to understand why different individuals show differences in side effects following rt.


Eligibility criteria:
inclusion criteria: * intermediate-risk prostate cancer patients (t1/t2n0m0 with either gs \< 7 and psa between 10-20 ng/ml or gs 7 and psa less than 20 ng/ml). * planned for radical rt to the prostate with imrt planning (79gy/39fractions or 60 gy/20 fractions). * no contraindications to diagnostic ct scanning. * prostate volumes will be less than 80 cc to control the planning dose volume histogram (dvh) as much as possible in terms of rectal and bladder volumes. * no known dna repair disorders (e.g. family history of at, brca1/2 or li fraumeni syndrome) or contraindications to radical pelvic rt. exclusion criteria: * inability to provide informed consent. * patient having contraindications to diagnostic ct scan. * patients who have unobtainable data regarding previous therapy and their clinical outcome. * patients should not have any diagnostic x-rays or cat scans 2 weeks prior to their enrollment into the study.

Keywords: prostate cancer
prospective study to determine the effect of subconjunctival bevacizumab (avastin) in corneal neovascularization

Conditions: Corneal Neovascularization

Summary: to determine the effect of subconjunctival bevacizumab in corneal neovascularization

corneal transplantation is the most commonly performed transplant surgery in the world today. immunologic rejection is the leading cause of graft failure, with about 25% of graft recipients experiencing at least one episode of rejection. of these episodes, about 20% are irreversible. the rate of corneal graft rejection in high-risk eyes, such as corneal neovascularization, has been reported to be 50% to 70%. vascularized corneas have a much higher rate of graft rejection than avascular corneas. whereas the normal cornea is devoid of blood and lymphatic vessels, both can invade the cornea secondary to a variety of corneal diseases and after surgery. this not only reduces visual acuity, but also renders such a cornea high-risk, if subsequent corneal transplantation is performed.anti-angiogenesis, the pharmacologic inhibition of new blood vessel growth and formation, is a new treatment strategy under active and vigorous investigation. multiple growth factors have been shown to contribute to the molecular events involved in the regulation of blood vessel growth similarly, it is assumed that angiogenic growth factors such as vascular endothelial growth factor (vegf), considered a major pro-angiogenic factor, could play a role in the pathogenesis of neovascularization. several approaches can be taken to neutralize vegf. bevacizumab (avastin) is a full-length humanized murine monoclonal antibody against the vegf molecule.it binds to and inhibits the biologic activity of human vegf preventing the interaction of this molecule to its receptors on the surface of endothelial cells. the interaction of vegf with its receptors leads to endothelial cell proliferation and new vessel formation. there is evidence that triamcinolone acetonide (ta) inhibits vasogenic edema and inflammation, decreases vascular leakage, reduces the secretion of vegf by pigment epithelial cells during oxidative stress and, down-regulates the expression of the vegf gene in vascular smooth muscle cells furthermore, ta decreases the paracellular permeability of cultured epithelial cells and down-regulates the inflammatory expression of endothelial adhesion molecules.

Eligibility criteria:
inclusion criteria: 1. presence of vessels in minimum one quadrant 2. vessels that penetrate more than 0.5 mm of the limb, in any depth 3. who had signed the informed consent 4. those that could attend to frequent ophthalmologic revisions after treatment and could wait for 6 months before the surgical procedure. exclusion criteria: 1. patients with urgent need of a penetrating keratoplasty, pregnancy or lactancy 2. patient that may need an additional procedure to penetrating keratoplasty.

Keywords: corneal neovascularization bevacizumab (avastin) immunologic rejection vascular endothelial growth factor
a study of genetic variation influencing pain and response to opioid medications

Conditions: Pain

Summary: the investigators will be collecting saliva dna samples from chronic back pain patients. the investigators hope to find candidate genes associated with response to opioid medication by correlating molecular genetics data with pain measurement and opioid responsiveness data including opioid hyperalgesia and opioid analgesic tolerance.

the investigators hope to find a genetic association with various physiologic responses to opioid medication in patients who suffer from chronic pain (e.g. oih vs. analgesic tolerance, baseline pain sensitivity, etc.). this has never been done before, and if it proves successful, it could provide physicians a greater understanding of why some chronic opioid users continue needing increased doses of opioids. this data may also help predict which patients will do well with chronic opioid therapy and which ones may not. initial data with oprm1 gene analysis in humans already implicates certain snps with opioid responsiveness and there have been suggestions for screening patients for oprm1 prior to initiating opioid therapy in order to optimize their treatment response (reynolds et al., 2008). clin lab med. 2008 dec;28(4):581-98. the value of cyp2d6 and oprm1 pharmacogenetic testing for opioid therapy. reynolds kk, ramey-hartung b, jortani sa.

Eligibility criteria:
inclusion criteria: 1. adult patient, aged 18-71 2. recipient of chronic opioid therapy 3. patient is able to produce a saliva sample 4. if a past study participant, patient will have agreed to be contacted for future studies. exclusion criteria: a. not meeting inclusion criteria

Keywords: chronic pain, genetic variation, opioid medications, pain
related haplo-donor haematopoietic stem cell transplantation for adults with severe sickle cell disease

Conditions: Sickle Cell Disease

Summary: the purpose of this clinical trial is to evaluate the clinical and cost effectiveness of haploidentical stem cell transplantation (sct) for adults with severe sickle cell disease (scd), who have failed other therapies or are intolerant of existing therapies or require chronic transfusions to prevent on-going complications of scd.


Eligibility criteria:
inclusion criteria: 1. adult patients age ≥ 18 years 2. confirmed haploidentical donor 3. severe scd phenotype who are at high risk for morbidity and mortality. severe scd is defined by at least one of the following: i. clinically significant neurologic event (stroke) or deficit lasting \> 24 hours. ii. history of ≥2 acute chest syndromes in a 2-year period preceding enrolment despite optimum treatment, e.g. with hydroxycarbamide (hc). iii. history of ≥3 severe pain crises per year in a 2-year period preceding enrolment despite the institution of supportive care measures (e.g. optimum treatment with hc). iv. administration of regular transfusion therapy (=8 packed red blood transfusions per year for 1 year to prevent vaso-occlusive complications). v. patients assessed as requiring transfusion but with red cell allo-antibodies/very rare blood type, rendering it difficult to continue/commence chronic transfusion. vi. patients requiring hc/transfusion for treatment of scd complications who cannot tolerate either therapy due to significant adverse reactions. vii. established end organ damage relating to scd, including but not limited to progressive sickle vasculopathy and hepatopathy. end-organ sufficient for entry to this trial shall be ratified at the uk nhp. d) patients must be fit to proceed to haploidentical sct as defined below: i. karnofsky score ≥60 ii. cardiac function: lvef ≥45% or shortening fraction ≥25% iii. lung function: fev1, fvc and tlco ≥50% iv. renal function: edta gfr ≥40 ml/min/1.73m2 v. hepatic function: alt \<x3 uln and bilirubin \<x2 the upper limit of normal, those with hyperbilirubinemia due to sickle related haemolysis will not be excluded. no radiological evidence of cirrhosis. e) written informed consent. exclusion criteria: 1. fully matched sibling donor. 2. previous bone marrow transplant. 3. pregnancy or breast feeding. 4. participants able to conceive a child that are unprepared to use effective contraception. 5. clinically significant donor specific hla antibodies. 6. hiv infection or active hepatitis b or c. 7. uncontrolled infection including bacterial, fungal and viral. 8. participation in another interventional trial in the last three months. 9. pre-existing condition deemed to significantly increase the risk of haploidentical sct by the local principal investigator.

Keywords: sickle haploidentical stem cell transplant
study to evaluate the efficacy and safety of once-weekly oral nbi-1065846 in the treatment of anhedonia in mdd

Conditions: Anhedonia; Major Depressive Disorder

Summary: to evaluate the efficacy of nbi-1065846 compared with placebo on improving symptoms of anhedonia in participants with major depressive disorder (mdd).


Eligibility criteria:
key inclusion criteria: participants must meet all of the following key inclusion criteria: 1. completed written informed consent. 2. aged 18 to 65 years, inclusive, at the time of informed consent. 3. primary diagnosis of mdd. 4. participants must meet one of the following criteria: * must have been taking ≥1 antidepressant medication(s) for ≥8 weeks prior to screening. * must have received ≥1 antidepressant medication(s) for ≥8 weeks in the current or most recent episode of depression. 5. snaith hamilton pleasure scale (shaps) score is ≥30 at screening and day 1. key exclusion criteria: participants will be excluded from the study if they meet any of the following key criteria: 1. any psychiatric disorder disallowed per protocol and electroconvulsive therapy (ect) within 6 months prior to screening. comorbid anxiety disorders are not exclusionary. 2. have a significant risk of suicidal or violent behavior. 3. a history of seizure disorder, stroke, alzheimer disease, parkinson disease, multiple sclerosis, head injury associated with loss of consciousness for more than 15 minutes, or other neurodegenerative disorder.

Keywords: anhedonia major depressive disorder mdd nbi-1065846 terpsis neurocrine antidepressant depression tak-041
evaluate pf-00547659 on cerebrospinal fluid lymphocytes in volunteers with crohn's disease or ulcerative colitis who failed or did not tolerate anti-tnfs

Conditions: Crohn's Disease; Ileitis; Ileo-colonic and Colonic Crohn's Disease; Granulomatous Colitis; Regional Enteritis; Ulcerative Colitis

Summary: study is designed to show a lack of effect on white blood cells circulating in the spinal fluid.


Eligibility criteria:
inclusion criteria: * males and females \>=18 and =\<75 years * for cd subjects: hscrp \> 5 mg/l and harvey-bradshaw index \> 8 or when hbi cannot be determined (ie if stoma is present) or hscrp \< 5mg/l then: active lesions on colonoscopy or flexible sigmoidoscopy or active crohn's disease on ct or mr enterography * for uc subjects: diagnosis of uc \> 3 months; must have endoscopy to confirm active disease during screening; total mayo score of 6 to 12 points and moderate to severe disease on endoscopy exclusion criteria: * pregnancy or breastfeeding * tb or active enteric infections * entero vesicular fistulae * prior use of natalizumab or vedolizumab * right or left heart failure including symptomatic diastolic dysfunction or unexplained elevation of troponin i (\>0.05 ng/ml)

Keywords: crohn's disease lumbar puncture anti-madcam monoclonal antibody pf-00547659 ulcerative colitis
effectiveness of the use of the picsi dish (hyaluronan microdot) in the selection of sperm for intracytoplasmic sperm injection (icsi)

Conditions: Male Factor Infertility

Summary: the purpose of this study is to determine if using microdots of hyaluronan (picsi) to select sperm for icsi will result in increased pregnancies especially with men that have a low hyaluronan binding score (hba).


Eligibility criteria:
inclusion criteria: * ivf patients who require the icsi procedure and whose care is being managed by the clinical and scientific staff of the participating groups, will be included in the study. exclusion criteria: * ivf patients who do not require the icsi procedure will be excluded from the study. in addition the following groups of patients will also be excluded: * patients using testicular sperm. * patients using donor sperm. * patients where the female partner is over 40 years of age * patients who have an initial hba score less than or equal to 2% * patients who have a sperm count less than or equal to 10,000 motile sperm per ml. * patients who produce less than 4 metaphase ii oocytes

Keywords: infertility icsi hyaluronan binding sperm sperm selection male factor hyaluronan sperm maturity clinical pregnancy rates hyaluronan binding assay hba picsi
clinical evaluation of bw430c in epilepsy

Conditions: Epilepsy

Summary: to evaluate safety information of bw430c when administered using the lower starting doses and slower dose escalations as recommended global data sheet


Eligibility criteria:
inclusion criteria: * epilepsy with partial seizures * tonic clonic seizures * generalized seizures of lennox-gastaut * subjects whose seizures are easily recognizable at least one seizure per month and counts for 8 consecutive weeks prior to the start of the study drug. * concurrent aeds: subjects taking concurrent vpa. exclusion criteria: * previous participation in a study of lamictal * known hypersensitivity to any drugs * pregnant women * nursing mothers * women who may be pregnant * women contemplating pregnancy during the study period

Keywords: epilepsy incidence of rash safety evaluation for initial dose patients with valproic acid
occipital nerve stimulation to treat chronic headaches

Conditions: Headache Disorders

Summary: the purpose of this study is to evaluate the safety and efficacy of a implantable device, called the bion(r), in the treatment of chronic headache. the bion microstimulator is placed underneath the skin next to the greater occipital nerve. the bion microstimulator then stimulates the nerve by generating small amounts of electrical current.

chronic migraine and chronic cluster headaches are among the most disabling medical illnesses, inflicting severe functional impairment. these headache subtypes are particularly resistant to current treatment modalities, with the majority of chronic migraine patients continuing to experience frequent or near-daily headaches despite aggressive, multidisciplinary, and multimodal therapy. the refractory nature of these illnesses and the level of devastation they inflict on the lives of their victims has motivated the pursuit of novel treatment approaches. direct stimulation of the greater occipital nerve has been shown to alleviate the symptoms of chronic migraine and chronic cluster headaches in a small number of patients.

Eligibility criteria:
inclusion criteria: 1. be diagnosed with chronic migraine, probable chronic migraine, and/or chronic cluster headache according to the 2004 ihs classification criteria (2nd ed.) or be diagnosed with migraine with or without aura 2. be 18 years of age or older 3. be willing and able to follow all study-related procedures during the course of the study; 4. have exhibited refractoriness to two acute medication regimens and two prophylactic medications from two separate preventative classes. exclusion criteria: 1. have received a botulinum toxin (botox) injection for treatment of headache within the last 90 days; 2. have previously undergone destructive ganglionectomy affecting c2/c3 occipital and/or trigeminal distribution or have an occipital blockade currently in effect; 3. have had a previous surgery in the intended implant area; 4. have arnold-chiari malformation; 5. have participated within the last 30 days or plan to participate within the next 4 months in a device or drug trial; 6. be pregnant or planning on becoming pregnant during the study period; 7. have an implanted electrical device (e.g., pacemaker, spinal cord stimulator) that may interfere with this therapy; and 8. currently require, or be likely to require, mri or diathermy.

Keywords: headache chronic headache migraine pain cluster headache
decidual stromal cells to treat graft-vs-host disease after stem cell transplant for myelodysplastic syndrome/myeloproliferative neoplasm

Conditions: Myelodysplastic/Myeloproliferative Neoplasm; Steroid Refractory GVHD; Allogeneic Hematopoietic Cell Transplant

Summary: this is a single participant study of decidual stromal cells (dsc) for the treatment of steroid refractory graft-versus-host disease (gvhd) in a patient with myelodysplastic syndrome/myeloproliferative neoplasm (mds/mpn).


Eligibility criteria:
a single participant with myelodysplastic syndrome/myeloproliferative neoplasm located at princess margaret cancer centre who experienced steroid-refractory graft-vs-host disease after receiving an allogeneic hematopoietic cell transplant and have no standard treatment options available.