cyrilzakka/clinical-trials huggingface dataset:
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)

This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.

Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.

Eligibility Criteria:
Inclusion Criteria:

* Parent(s) or legal guardian(s) must provide written informed consent prior to data abstraction, unless all of the following apply:
* The patient is deceased; AND
* The responsible IRB/IEC/REB does not require informed consent per a review of their documented local policies for collecting retrospective data on patients who are deceased; AND
* Written confirmation is received from the responsible IRB/IEC/REB confirming that the abstracted data can be analyzed and used to support regulatory filings by the Sponsor
* Patient must have a documented diagnosis of HPP as indicated by 1 or more of the following:
* Documented ALPL gene mutation(s)
* Serum alkaline phosphatase (ALP) below the age-adjusted normal range and either plasma pyridoxal 5'-phosphate (PLP) or urinary phosphoethanolamine (PEA) above the upper limit of normal
* Serum ALP below the age-adjusted normal range and HPP-related radiographic abnormalities on X-ray
* Patient must have onset of signs of HPP prior to 6 months of age and have documentation of 1 or more of the following characteristics of perinatal and infantile HPP:
* Respiratory compromise (up to and including respiratory failure) requiring institution of respiratory support measure(s), requiring medication(s) for management of symptom(s), and/or associated with other respiratory complications (e.g., pneumonia(s), respiratory tract infection(s))
* Pyridoxine (vitamin B6)-responsive seizures
* Rachitic chest deformity

Exclusion Criteria:

Patients will be excluded from study participation if they have 1 or more of the following exclusion criteria:

* Patient received treatment with asfotase alfa at any time prior to data abstraction
* Patient has clinically significant other disease

Both living and deceased patients will be considered for study participation
Effects of Exergames on Muscle and Lung Function in Elderly

The goal of this study is to examine the effects of 8-weeks exergames program on pulmonary function, muscle strength and cognitive function in elderly.

Participants will:

Play exergame 30min/session, 2 sessions/week or remained regular daily activities for 8 weeks.

Subjects who are older than 60 years will be assigned to exergame group or control group randomly.

Exergame group: Subjects will receive exergame program 30min/session, 2 sessions/week for 8 weeks.

Control group: Subjects remain regular daily activities for 8 weeks. Both groups receive pre- and post- measurements. The measurements included assessment of muscle strength and lung function.

Eligibility Criteria:
Inclusion Criteria:

1. age \> 60 years,
2. sufficient cognitive capacity to provide informed consent,
3. ability to understand and speak Chinese or Taiwanese.

Exclusion Criteria:

1. medical conditions prohibiting physical exercise
2. physical or cognitive disabilities preventing participation in or comprehension of the exergame.
Evaluation of the E/e' Ratio of the Mitral Annulus in Predicting Fluid Responsiveness.

Diastolic function may be evaluated by different measurements on transesophageal echocardiography (TEE). They include mitral inflow velocities obtained by pulsed-wave doppler (PW) : peak early diastolic velocity (E) and late diastolic velocity (A). Mitral annulus velocities, early diastolic (e') and late diastolic (a') are obtained by tissue doppler imaging (TDI). The ratio E/e' reflects left ventricular filling pressure and, as such, might be a predictor of fluid responsiveness.

The aim of this study is to evaluate the predictive value of the mitral valve E/e' ratio for fluid responsiveness among patients undergoing coronary bypass graft surgery. Fluid responsiveness being defined as an increase in stroke volume of ≥ 15%.

After induction of anesthesia, patients will have their diastolic function evaluated by means of E/e' and other measures. They will then be administered an intravenous bolus of 500 mL of Lactate Ringer® along with passive leg raising (PLR). Stroke volume and fluid responsiveness will be assessed by the thermodilution method (Swan-Ganz catheter) and the FloTrac® device. Fluid responders will be compared to non-responders to evaluate the relationship between E/e' ratio and fluid responsiveness.


Eligibility Criteria:
Inclusion Criteria:

* Patients \>18 yrs, undergoing elective coronary artery bypass grafting or surgery will be included.

Exclusion Criteria:

* Significant mitral valvular heart disease (mitral regurgitation ≥ 2/4 or stenosis)
* Significant right sided valvular heart disease (tricuspid regurgitation ≥ 2/4)
* Intracardiac shunts
* Emergency surgery
* Non-sinusal rythme
* Clinical evidence of decompensated heart failure
* Clinical evidence of decompensated pulmonary hypertension
* Renal insufficiency with creatinine clairance ≤ 30 cc/min or dialysis
* Contraindications to TEE, including esophageal disease or unstable cervical spine
* CVP ≥ 15 mm Hg or PCWP ≥ 18 mm Hg immediately before fluid infusion
Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia

To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.

Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.).

Eltrombopag is currently the only TPO receptor agonist with indications for pediatric immune thrombocytopenia. However, at present, the treatment response of pediatric ITP is not good, and a considerable number of patients need to switched to other TPO receptor agonist, such as avatrombopag, because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.

Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of avatrombopag in the treatment of pediatric immune thrombocytopenia in patients who who have been treated with eltrombopag before and switched to avatrobopag because of different reasons.

Eligibility Criteria:
Inclusion Criteria:

* Age 6-18 years old (including both ends), male and female;
* Diagnosed with primary immune thrombocytopenia (ITP);
* Patients who had previously received eltrombopag treatment and then converted to avatrombopag treatment because of ineffectiveness (platelet count \< 30×10\^9/L after eltrombopag treatment, or platelet count increased less than 2 times of the basic value, or bleeding) or large platelet fluctuation or due to patient preference, economic reasons and other reasons;
* Cardiac function of the New York Society of Cardiac Function ≤ 2;
* Understand the study procedure and voluntarily sign the informed consent.

Exclusion Criteria:

* Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
* Subjects with primary disease of important organs (liver, kidney, heart, etc.), or with immune system diseases;
* Subjects who are known to be allergic to avatrombopag or any of its excipients;
* Subjects who had used rituximab within the last 3 months;
* Subjects who underwent splenectomy within the last 3 months；
* Subjects with a history of abnormal platelet aggregation that may affect the reliability of platelet count measurements；
* Any medical history or condition that the investigator deems unsuitable for participation in the study.
Assessing Repeat Fit Testing for CRC

This study will randomly assign 220 men and women to either a social media intervention or a standard reminder system to have a repeat screening test for colo-rectal cancer.

Only persons initially screening by FIT (through a Rural Cancer Prevention Center service program) will be eligible for study participation. The objective is to test a social media-based intervention designed to promote annual repeat colo-rectal cancer screening using the Fecal Immunochemical Test (FIT) among person who have taken the FIT test once before. 220 men and women (50 to 75 years of age) will be recruited and randomly assigned to either the intervention condition (monthly social media contact) or a control condition (standard of care reminder for repeat testing one month before the annual test is due). The dependent variable will be whether volunteers requested and retuned a FIT kit with 60 days of the annual due date (one year from their initial screening).

Eligibility Criteria:
Inclusion Criteria:

* must have completed and returned an initial FIT kit to us as part of a Rural Cancer Prevention Center service program to the community

Exclusion Criteria:

* visible blood in stool or prior diagnosis of cool-rectal cancer
Effect of Calcium Plus Vitamin D Supplementation on Adolescent Mother and Infant Bone Health

Pregnancy and lactation are periods of high calcium requirement. Inadequate maternal calcium intake and vitamin D insufficiency may adversely affect fetal and neonatal growth and maternal bone mass particularly in adolescent mothers. The investigators propose to evaluate the influence of calcium plus vitamin D supplementation during pregnancy on bone mass of lactating adolescent mothers with habitually low calcium and vitamin D, on fetal growth and neonate bone mass. Mothers are randomly assigned to receive daily calcium (600 mg) plus vitamin D3 (200 IU) supplement or placebo from mid gestation until parturition. Fetal growth parameters are assessed by standard ultrasound techniques at mid and late gestation. Maternal bone mass is assessed by dual energy x-ray absorptiometry (DXA) at 2 and 5 months postpartum and 6 months after weaning. Infant bone mass is assessed by DXA at 2 months postpartum.


Eligibility Criteria:
Inclusion Criteria:

* 13-19 y old
* between 23 and 29 wk of gestation
* primiparous
* carrying single fetus
* intending to exclusively or predominantly breastfeed

Exclusion Criteria:

* chronic health problems
* smokers
* users of nutritional supplements, except iron supplements provided during standard prenatal care
Effects of Respiratory Training on Respiratory and Functional Performance in Patients With Stroke

The purpose of the research is to investigate the effects of respiratory training on respiratory and functional performance in patients with stroke.

Stroke ranks third in the global cause of death, behind cancer and coronary heart disease. Stroke is also the third largest cause of death in Taiwan in 2014. Stroke patients in the course of exercise are in addition to muscle weakness and reduced endurance, but usually also accompanied by respiratory damage. From past studies have shown that stroke patients whose respiratory function has significant change, such as decreased respiratory muscle strength, reduced respiratory movement of the affected hemithorax, declined in maximum breathing pressure and decreased amplitude of diaphragmatic movements on the paralyzed side. But whether respiratory training can improve respiratory and functional performance of stroke patients, because of the limited research results could not be confirmed.

Eligibility Criteria:
Inclusion Criteria:

* first episode of unilateral stroke.
* the definition of stroke was according to the World Health Organization (WHO) criteria (Hatano 1976), confirmed by computerized tomography (CT) or magnetic resonance imaging (MRI).
* older than 20 years.
* had maximum inspiratory pressure (MIP) values lower than 90% of those predicted and adjusted for age and sex.
* facial palsy, which could not prevent proper labial occlusion.
* ability to understand and follow simple verbal instructions.
* no receptive aphasia.
* not undergone thoracic or abdominal surgery.

Exclusion Criteria:

* unable to perform the tests.
* impaired level of consciousness and evidence of gross cognitive impairment.
* excluded patients with comorbidities of respiratory system disease (e.g. chronic obstructive pulmonary disease, asthma, cystic fibrosis), or other diseases leading to the impairment of respiratory muscle (e.g. myasthenia gravis).
Analysis of the Effectiveness of Neoadjuvant Chemotherapy in the Treatment of Colon Cancer Locally Advanced (ELECLA)

Effectiveness analysis of neoadjuvant chemotherapy in the treatment of locally advanced colon cancer. ELECLA trial

Participants in this study selected patients according to homogeneous inclusion criteria and will be treated either with a uniform protocol of neoadjuvant chemotherapy, surgery and complementary chemotherapy (intervention group), or with the standard scheme of postoperative surgery and chemotherapy (control group ). All patients will be studied basally through clinical examinations and radiological and endoscopic tests usually used to reach the diagnosis of Locally Advanced Colon Cancer . In the group of patients receiving neoadjuvant treatment, the CT scan will be repeated after the completion of preoperative chemotherapy to restage the disease and quantify the degree of tumor response.

Finally, the project tries to determine whether the neoadjuvant treatment scheme increases disease-free survival (SLE) at 2 and 5 years and overall survival (OS) at 5 years. Likewise, the toxicity derived from chemotherapy treatment and perioperative morbidity and mortality will be analyzed to evaluate the feasibility and safety of the therapeutic procedure. The rate of completion of chemotherapy in both groups will also be compared.

Eligibility Criteria:
Inclusion Criteria:

* CLINICS

  1. Histological confirmation of colon adenocarcinoma.
  2. Patients of both sexes with age over 18 years.
  3. Good general condition of the patient (Karnofsky\> 60% or ECOG (Eastern Cooperative Oncology Group) \<2).
  4. Analytical at the time of inclusion with figures of Hemoglobin\> 10 g / dL; Leukocytes\> 3.0 109 / L, Platelets\> 100,000, Glomerular Filtration\> 50 ml / min and Total Bilirubin \<25 micromol / l.
  5. Absence of contraindication for chemotherapy.
  6. Acceptance and signature of the Informed Consent.
* OF IMAGE

  1. Radiological signs, evaluated by CT, of T4 or T3 tumor infiltration\> 5 mm of transmural invasion.
  2. With or without lymph node involvement by CT.
  3. No metastatic involvement in other organs (M0).
  4. Radiologically resectable disease. REFERENCES TO THE TREATMENT

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  1. That they will undergo elective surgery with curative intent (R0).

Exclusion Criteria:

* 1. Important comorbidity, uncontrolled angina or a history of acute myocardial infarction in the last 6 months.

  2. Personal history of another malignancy in the last 5 years, with the exception of melanoma.

  3. Uncontrolled infection 4. Pregnancy or lactation. 5. Peripheral neuropathy\> grade 1. 6. Rectum cancer (\<15 cm of the anal margin or below peritoneal reflection). 7. Presence of distant metastasis or peritoneal carcinomatosis. 8. Intestinal obstruction. 9. Existence of microsatellite instability 10. Refusal to participate or to give written consent 11. Impossibility, at the investigator's discretion, to understand the purpose of the study or to comply with its procedures.
Using Genetic Polymorphisms to Predict the Efficacy and Toxicity - A Gastric Adenocarcinoma Study

This is an open-label, non-comparative phase II study of sequential capecitabine plus oxaliplatin followed by docetaxel plus capecitabine in patients with unresectable gastric adenocarcinoma.

There are two primary objectives in different steps. In the first step, the primary objective of this study is to investigate the objective response rate in patients receiving sequential capecitabine plus oxaliplatin followed by docetaxel plus capecitabine in patients with unresectable gastric adenocarcinoma.

In the second step, the primary objective of this study is to screen the predictive biomarkers of three different chemotherapeutic drugs and also investigate the objective response rate in patients receiving sequential capecitabine plus oxaliplatin followed by docetaxel plus capecitabine in patients with unresectable gastric adenocarcinoma.

Eligibility Criteria:
Inclusion Criteria:

* Pathologically confirmed gastric adenocarcinoma.
* At least one measurable lesion in a non-irradiated area.
* No prior exposure to systemic chemotherapy for advanced gastric cancer.
* For those have adjuvant chemotherapy after a curative gastrectomy, the last dosing of previous adjuvant chemotherapy should be at least 6 months before the start of this treatment.
* Age \> 20 years old.
* ECOG Performance Status 2.
* Life expectancy greater than 12 weeks.
* Adequate bone marrow function :absolutely neutrophil count 1.5 x 109/L or WBC 4 x 109/L; Hemoglobin \> 9 g/dl;platelet count 100 x 109/L.
* Adequate liver function : ALT \& AST 2.5 x ULN if without liver metastasis or 5 x ULN if with hepatic metastasis. Alkaline phosphatase 2.5 x ULN if without liver metastasis or 5 x ULN, if with hepatic and bone metastasis. Bilirubin \< 2 x ULN
* Adequate renal function :Creatinine \< 1.5 x ULN.
* Patients must be accessible for treatment and follow-up in the participating centers.

Exclusion Criteria:

* Patient who are receiving concurrent radiotherapy, chemotherapy or other experimental therapy.(Previous radiotherapy is allowable if the last dose was given more than 2 weeks before the protocol treatment).
* Major surgery within two weeks prior to entering the study.
* Patients with CNS metastasis, including clinical suspicion.
* Patients who are under active or uncontrolled infections.
* Patients who had cardiac arrhythmia or myocardial infarction history 6 months before entry.
* Patients with clinically detectable peripheral neuropathy \> 2 on the CTC criteria
* Patients with concomitant illness that might be aggravated by chemotherapy.
* Patients who are pregnant or with breast feeding.
* Other concomitant or previously malignancy within 5 yrs except for in situ cervix cancer or squamous cell carcinoma of the skin treated by surgery only.
* Patients with hypersensitivity to any component of the chemotherapeutic regimen.
* Mental status is not fit for clinical trial
* Can not take study medication orally
Effect-site Concentration of Remifentanil for Smooth Induction With Desflurane

Desflurane is a halogenated ether with low solubility, giving rapid induction and emergence from anesthesia This character is suitable for Volatile Induction and Maintenance Anesthesia (VIMA). But desflurane can cause airway irritation when it used as a sole induction agent to adult or children, so there were many studies about reducing the airway reflex combined with desflurane.

Remifentanil is a short acting opioid and is quickly hydrolyzed by nonspecific esterase in the plasma and multiorgan. Several studies suggest the optimal dose of remifentanil for reducing airway reflex or cardiovascular response for intubation combined with desflurane. But these studies were under 1 Minimal Alveolar Concentration (MAC) of desflurane and this point could be a limitation because desflurane concentration over 1 MAC increase airway irritation significantly. This study this study was designed to determine a median effective effect-site concentration (EC50) of remifentanil to prevent airway irritation during desflurane anesthesia over 1 MAC.


Eligibility Criteria:
Inclusion Criteria:

* patients for surgery requiring general anesthesia
* American Society of Anesthesiologists(ASA) classification I, II

Exclusion Criteria:

* history of gastroesophageal reflux, High grade obesity, Predicted difficult airway, Asthma, Chronic obstructive pulmonary disease(COPD), Upper respiratory infection (URI) within 3 weeks
Establishment and Validation of Prediction Model of Simultaneous Chemoradiotherapy for Cervical Cancer Organoids

This is a single-arm, single-center, open-label, Phase II study aimed at assessing the efficacy of organoids in predicting the response to radiotherapy and chemotherapy in cervical cancer

By constructing cervical cancer organoids, establishing a radiochemotherapy prediction model based on organoids, and exploring radiotherapy resistance-related molecules as new therapeutic targets for locally advanced cervical cancer, the feasibility and effectiveness are assessed. Utilizing the constructed model for molecular mechanism discussions and effective drug screening, new avenues for the treatment of cervical cancer patients with radiochemotherapy resistance are sought.

Eligibility Criteria:
Inclusion Criteria:

Able to understand and voluntarily sign written informed consent.

1. Women aged ≥18 years at the time of study entry.
2. Eastern Cooperative Oncology Group Performance Status (ECOG PS) score of 0-2.Life expectancy ≥12 weeks.
3. Histologically confirmed cervical cancer.
4. Histologically-confirmed squamous cell carcinoma, adenocarcinoma, or adenosquamous carcinoma，villous duct carcinoma, invasive mucinous multilayer carcinoma, gastric adenocarcinoma, mesrenal duct carcinoma, clear cell carcinoma, serous carcinoma, endometrioid adenocarcinoma and small cell carcinoma，Intestinal-type adenocarcinoma of the cervix;
5. At least one measurable tumor lesion according to RECIST v1.1 criteria.
6. Available archived tumor tissue samples or recent biopsies.
7. Adequate organ function.
8. For fertile women with negative serum pregnancy and effective contraception within 7 days before administration (until 120 days after the last administration of the study drug and at least 180 days after radiotherapy)

Exclusion Criteria:

1. Subject with other active malignancies within 2 years prior enter the study.
2. Subject who cannot receive brachytherapy.
3. Active or prior documented autoimmune disease that may relapse.
4. History of interstitial lung disease or noninfectious pneumonitis.
5. Subject with the clinically significant cardio-cerebrovascular disease.
6. History of severe hypersensitivity reactions to other mAbs.
7. Prior allogeneic stem cell transplantation or organ transplantation.
8. Subjects who require systemic treatment with glucocorticoid (\>10 mg/day of prednisone or equivalent glucocorticoid) or other immunosuppressive agents within 14 days prior enter the study.
9. Receipt of live attenuated vaccines within 30 days prior to the first dose of the study drug.
10. Prior exposure to any experimental antitumor vaccines, or any agent targeting T-cell costimulation or immune checkpoint pathways (eg, anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CTLA-4, anti-CD137 or anti-OX40 antibody, etc).
11. Any condition that, in the opinion of the Investigator, would interfere with the evaluation of the study drug or interpretation of subject safety or study results.

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