cyrilzakka/clinical-trials huggingface dataset:
Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia

This is a multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive.

180 treated naive patients with SAA will be enrolled in the study. The primary objective of the study will be the rate of complete hematologic response at six months.


Eligibility Criteria:
Inclusion Criteria:

1.15 Years to 75 Years (weight greater than 50 kg if Age \< 18 years old). 2.Severe aplastic anemia characterized by Bone marrow cell proliferation less than 25 percent (If ≥25% but \<50%, the remaining hematopoietic cells should be \<30%) AND At least two of the following: Absolute neutrophil count \<0.5×109/L; Platelet count \<20×109/L; Absolute reticulocyte count \<20×109/L.

3.Unsuitable or unwilling to perform hematopoietic stem cell transplantation (HSCT).

4.Signed informed consent.

Exclusion Criteria:

1. Diagnosis of whole blood cell reduction due to other causes or bone marrow hypoproliferative diseases.
2. Subjects who have previously received immunosuppressive therapy with mycophenolate mofetil, sirolimus, high dose cyclophosphamide (≥45mg/kg/d), alemtuzumab, etc；or have treated with thrombopoietin receptor agonist (eg, eltrombopag, romiplostim, rhTPO, etc.) prior to randomization.
3. Previous history of hematopoietic stem cell transplantation.
4. Subjects who is known or suspected of contraindications or hypersensitivity to Hetrombopag's API (Active Pharmaceutical Ingredient).
5. Evidence of clonal cytogenetic abnormalities at the time of screening.
6. Bleeding and/or Infection not adequately responding to appropriate therapy.
7. Any laboratory or clinical evidence for HIV infection. Any clinical history for hepatitis C infection; chronic hepatitis B infection; or any evidence for active hepatitis at the time of subjects screening.
8. ALT\> 2.5 x upper limit of normal (ULN), AST\> 2.5 x upper limit of normal (ULN) DBLI\> 1.5 x upper limit of normal (ULN), Scr\> upper limit of normal (ULN).
9. Subjects with uncontrolled hypertension (\>180/100mmHg), severe arrhythmia (such as complete left bundle branch block, QT interval prolongation (Bazetts formula), torsade ventricular tachycardia, etc.), unstable angina, pulmonary hypertension at the time of screening.
10. Subjects diagnosed with cirrhosis or portal hypertension.
11. Subjects with malignant solid tumors of any organ system within 5 years prior to screening, with or without treatment, metastasis or recurrence, except for local cutaneous basal cell carcinoma; subjects with hematological tumors found previously or during screening.
12. Subjects with deep vein thrombosis, myocardial infarction, stroke or peripheral arterial embolization within 12 months prior to randomization.
13. Female subjects who are nursing or pregnant.
14. Subjects cannot comply with effective contraception.
15. Subjects have participated in other clinical trial within the 3 months prior to study entry.
Flat OGTT (Oral Glucose Tolerance Test) During Pregnancy

The aim of the study is to examine the relationship between "flat" sugar curve on OGTT-glucose tolerance test during pregnancy, neonatal birthweight and other perinatal outcomes.

A retrospective cohort analysis of all women delivered in one university affiliated medical center between January 1st 2018 and December 31th 2019 with documented 100 grams OGTT results.

Eligibility Criteria:
Inclusion Criteria:

* Performing the OGTT test during the pregnancy

Exclusion Criteria:

* lack of documentation or Non-performance of OGTT test during the pregnancy.
PANORAMA Observational Study

To construct a computerized database of national profiles and epidemiological data on patients wearing Medtronic implantable pacemakers and cardioverter defibrillators (both with or without cardiac resynchronization therapy), implantable loop recorders and leads used within their intended use.

Clinical variables will be analyzed in relation to device-based data and diagnostics.

PANORAMA will collect a large database of device behavior, for instance to monitor long term behavior of device features, technical reliability and longevity, as well as to allow studying device performance in different subgroups of patients. Larger databases are often essential for formulating and testing study hypotheses for prospective (randomized) trials. While it may depend on specific objectives for individual devices, reliable assessment of device performance over longer periods and in broad patient populations typically requires a study population from a few hundred to a few thousand patients. PANORAMA collects acute and chronic patient and device data in a large population during routine usage of the device, without specific study interventions, and always within the approved intended use of the device.

In the past, observational studies were designed and conducted specifically on a device-by-device case. PANORAMA avoids the redesign of the same set of data. It establishes a core database which can be expanded with substudies creating more detailed data sets for particular CRDM devices/features.

PANORAMA will be open for inclusion to any patient (to be) implanted with a CRDM device. It shall be suitable for current and upcoming CRDM devices implanted in patients. It will serve as an epidemiological tool designed to stratify morbidity and mortality of all cardiac diseases treated by implantable CRDM devices.

Eligibility Criteria:
Inclusion Criteria:

* (To be) implanted with a Medtronic market-released cardiac device,
* Signed Patient Data Release Form.

Exclusion Criteria:

* Unwillingness or inability to cooperate or give voluntary consent
Universal Cancer Peptide-based Vaccination in Metastatic NSCLC

UCPVAx is a therapeutic vaccine based on the telomerase-derived UCP designed to induce strong TH1 CD4 T cell responses in cancer patients.

Three doses of UCPVax (0,25 mg, 0,5 mg and 1 mg) will be tested in this phase I/II study by using Continuous Reassessment Method (CRML) dose escalation design model.

The phase I is a dose escalation study designed to evaluate safety of use of UCPVax and to estimate its Maximum Tolerated Dose (MTD).

The phase II is a dose deescalation designed to evaluate the immunogenicity of UCPVax according to the dose level.

UCPVax study is a prospective multicenter phase I/II study: 54 patients with metastatic NSCLC will be enrolled in 5 centers in France.

A translational research program will be performed to better define the eligibility criteria and predictive biomarkers needed for randomized phase II and Phase III trials.

Eligibility Criteria:
Inclusion Criteria:

* Written informed consent
* Histologically or cytologically confirmed NSCLC (adenocarcinoma, squamous cell carcinoma, large cell carcinoma, undifferentiated carcinoma or other)
* Stage IIIB not amenable to radiotherapy or stage IV cancer according to the TNM classification (7th edition) or recurrent NSCLC after surgery not amenable to loco-regional therapy.
* Pre-treated with at least 2 or 3 lines of treatment (including immunotherapy). Chemoradiation for stage IIIB disease is considered as one treatment line.
* At least one measurable lesion by CT scan or MRI based on RECIST criteria version 1.1
* Performance status 0 or 1 on the ECOG scale
* Life-expectancy \> 3 months
* Adequate hematological, hepatic, and renal function

Exclusion Criteria:

* Prior history of other malignancy except for: basal cell carcinoma of the skin, cervical intra-epithelial neoplasia and other cancer curatively treated with no evidence of disease for at least 5 years
* Symptomatic brain metastases. Patients with controlled brain metastases after radiation therapy or with asymptomatic brain metastases may be included.
* History of active autoimmune diseases (lupus, rheumatoid arthritis, inflammatory bowel disease...)
* Patients under chronic treatment with systemic corticoids or other immunosuppressive drugs (prednisone or prednisolone ≤ 10 mg/day is allowed) - - Positive serology for Human Immunodeficiency Virus (HIV) or Hepatitis C virus (HCV); presence in the serum of the antigens HBs
* Participation in a clinical study with an investigational product within 4 weeks prior to the start of the study treatment
* Pregnancy or lactating patients.
* Patients with any medical or psychiatric condition or disease,
* Patients under guardianship, curatorship or under the protection of justice.
Study To Investigate Safety And Efficacy Of Sildenafil In The Newborns With Persistent Pulmonary Hypertension (PPHN)

Sildenafil is efficacious in newborns with persistent pulmonary hypertension and its use will reduce the need for inhaled nitric oxide.

Letter to investigator dated 18 June 2012 that study was to be terminated. Study terminated due to evolved and widespread use of standard of care, relevance of study questioned. No safety reasons or issues.

Eligibility Criteria:
Inclusion Criteria:

* 72 hours of age; and \> or = to 34 weeks gestational age.
* Persistent Pulmonary Hypertension of the Newborn or Hypoxic respiratory failure associated with:

  1. Idiopathic PPHN or
  2. Meconium aspiration syndrome or
  3. Sepsis or
  4. Pneumonia
* Oxygenation Index (OI) \>15 and \<60 calculated

Exclusion Criteria:

* Patients already receiving inhaled nitric oxide (iNO) on referral.
* Prior or immediate need for full Cardio Pulmonary Resuscitation or Extracorporeal Membrane Oxygenation (ECMO).
* Life threatening or lethal congenital anomaly.
* Large left to right intracardiac or ductal shunting (diagnosed from echocardiogram on admission to GOSH).
* Clinically significant active seizures as per clinical judgment.
* Bleeding diathesis as per clinical judgment
Intraoperative Hypovolemia and Fluid Therapy

Postoperative organ dysfunction severely affects the prognosis of surgical patients. Despite several trials evaluating restrictive or liberal fluid strategies, the ideal fluid replacement strategy remains controversial. Owing to the risk of altered tissue perfusion, a key trigger of organ dysfunction, the purpose of this study was to compare the influence of restrictive and liberal fluid regimens, using a goal-directed approach, on hypovolemia and postoperative organ dysfunction.

Despite several trials evaluating restrictive or liberal fluid strategies, the ideal fluid replacement strategy remains unanswered. Although recent studies suggest that intraoperative fluid restriction may reduce postoperative morbidity and promote faster recovery, extrapolation to individual patients remains difficult. Indeed, whether fluid overload may expose tissue to oedema, impairing oxygenation and wound healing, fluid restriction may, conversely, expose to hypovolemia, which occurs frequently during abdominal surgery, leading to tissue hypoperfusion and organ dysfunction. To date, the restrictive and liberal fluid substitution strategies have not been compared using a goal-directed approach. In addition, recent data suggest that targeting early indicators of hypoperfusion, such as central venous oxygen saturation (ScvO2), which reflects the oxygen delivery/consumption relationship, may be important in the management of patients undergoing major surgery. No data are available on the effects of intraoperative fluid volume replacement strategy on ScvO2 modifications.

The purpose of this study is first to evaluate the influence of restrictive and liberal fluid replacement strategies on both hypovolemia and postoperative organ dysfunction using an oesophageal doppler goal-directed approach (with goal = peak aortic velocity variation and stroke volume optimization) during major abdominal surgery. The second objective is to investigate the effects of fluid loading on ScvO2 modifications.

Eligibility Criteria:
Inclusion Criteria:

* Major intraabdominal surgery

Exclusion Criteria:

* Age \< 18 years
* Pregnancy
* Body mass index \> 35 kg/m2
* Emergency surgery
* Coagulopathy
* Sepsis or Systemic inflammatory response syndrome (SIRS)
* Hepatic failure (prothrombin ratio \< 50%, factor V \< 50%)
* Contraindication for epidural analgesia
Patient and Physician Perspectives on Non-Alcoholic Fatty Liver Disease

This trial studies patient and physician perspectives on non-alcoholic fatty liver disease. Using questionnaires and interviews, this trial may help researchers understand physicians' knowledge about the diagnosis, prognosis, treatment and management of non-alcoholic fatty liver disease, as well as gain an in-depth understanding of Hispanic patients' perceptions about the disease and investigate how cultural factors may play a role in its diagnosis, treatment and management.

PRIMARY OBJECTIVES:

I. Explore the perceptions of non-alcoholic fatty liver disease (NAFLD), including knowledge, current practices for diagnosis and management, and additional disease-related perceptions amongst both primary care physicians.

II. Explore the perceptions of NAFLD, including knowledge, diagnosis and management, and additional disease-related perceptions amongst Hispanic patients.

OUTLINE:

Patients and physicians complete a questionnaire over 5 minutes and an interview over 30-45 minutes about thoughts on NAFLD, including what they know about NAFLD and its diagnosis, management, and monitoring, and other thoughts on NAFLD.

Eligibility Criteria:
Inclusion Criteria:

* Be a self-reported practicing physician, who is a primary care physician (internal medicine, family medicine, etc.)
* Self-reported Hispanic ethnicity from the Greater Houston Metropolitan Area (patient)
* Have been diagnosed with NAFLD by a doctor (patient)
* Be an adult \>/= 18 (patient)
* Able to speak English (patient)

Exclusion Criteria

-None
Myocardial Dysfunction Evaluation in Lower Extremity Arterial Disease Patients With Deformation Analysis

Single centre observational study to assess lower extremity arterial disease (LEAD) patients' cardiac dysfunction with strain analyses and to assess connections between cardiac dysfunction, metabolomic changes and target organ damage in LEAD.


Eligibility Criteria:
Inclusion Criteria in LEAD group:

* Fontaine I-IV class symptoms
* Confirmed diagnosis of LEAD

Inclusion Criteria in control group:

* Age 35-85
* Has to have at least of the following: hypertension, diabetes, coronary artery disease or the patient smokes

Exclusion Criteria:

* Age \<35 or \>85 years
* BMI ≥40 kg/m²
* Known heart failure in history
* Any acute or chronic autoimmune or rheumatic disease
* Cardiac structural disease in history: inflammatory, infiltrative diseases, nonischemic cardiomyopathies
* Acute coronary syndrome in last 3 months
* Moderate to severe cardiac valvular disease
* Cardiac valvular disease operative/invasive treatment in history
* Cardiac implantable electronic device (CIED)
* Transitory ischemic attack or stroke in last 3 months
* Severe COPD (GOLD C or D)
* Moderate to severe asthma (according to GINA 2022 criteria)
* Untreated hypertension (noninvasive blood pressure measurement ≥180/110 mmHg during recruitment)
* Chronic severe kidney dysfunction (eGFR \<30 ml/min/1,73 m²)
* Malignant tumor with radiation treatment to thorax, oncologic or biologic treatment in history
* Chronic hematologic disease (except for anemia)
* Severe liver dysfunction
* Malignant tumor with \<5 years of remission
* Acute or chronic infectious disease
* Anemia with hemoglobin \<80 g/L
Effectiveness and Cost-effectiveness of the Energetic Program

Fatigue is present in at least 60% of the patients with a muscle disease and can be the most disabling symptom. In combination with other impairments, this often results in low levels of physical activity and decreased social participation, leading to large societal costs. Energetic is a self-management group program aimed at improving participation and physical endurance in these patients. Our hypothesis is that Energetic results in improved participation and improved physical endurance, less experienced fatigue and better quality of life compared to usual care, without increasing health care costs.


Eligibility Criteria:
Inclusion Criteria:

* formal diagnosis of muscular disease, either FascioScapuloHumeral Dystrophy (FSHD), Inclusion Body Myositis (IBM), Mitochondrial Myopathy (MM) or any other;
* being severely fatigued
* being cardiopulmonary stable and capable of aerobic training (as determined using a submaximal cycling exercise test with ECG (electrocardiography) and advice from a consultant cardiologist and pulmonologist);
* being motivated, committed to the program, and 'ready to change' which is discussed with the patient during the screening by a rehabilitation physician and an occupational therapist; to this end, motivational interviewing will be used (including a advantages/disadvantages matrix; a visual analogue scale (VAS) to rate the importance of participation in de program; and a VAS to rate the confidence a patient has that participation in the program is feasible)
* being able to formulate at least 3 individual participation goals during the interview with the occupational therapist.

Exclusion Criteria:

* active depression or other psychiatric disorder as indicated by a consultant psychologist or by the medical history,
* pregnancy,
* severe co-morbid condition,
* having participated in the Energetic program before
* a travel distance perceived as too burdensome
My MS Toolkit: a Symptom Self-management Program for Multiple Sclerosis

My MS Toolkit is a web-based symptom self-management program for persons with multiple sclerosis.

This study is evaluating various aspects of My MS Toolkit and how it impacts symptom self-management in multiple sclerosis. The research team believe that the toolkit will be feasible, acceptable, and beneficial to participants.

Study participants will be asked to use a newly developed web-based symptom self-management program, called the My MS Toolkit, for 12 weeks. Participants will also be asked to complete online surveys throughout the study.

The My MS Toolkit includes 8 modules that describe symptom self-management strategies. Participants will explore the My MS Toolkit on their own, following the prompts and guides built into the program, and are encouraged to practice and apply the skills learned.

The study can be done from participants' homes using a reliable internet-connected device. No travel is required.

Eligibility Criteria:
Inclusion Criteria:

* Multiple Sclerosis
* Have access to a reliable, internet-connected device (e.g. computer, tablet). (Note: this study is conducted electronically and is not location dependent.)
* One or more of the following:

  1. Moderate/moderately severe depressive symptoms
  2. Chronic pain
  3. Presence of significant fatigue symptoms
* Read, speak and understand English.

Exclusion Criteria:

* Significant cognitive impairment
* Current psychotherapy for symptoms
Encouraging Mail Order Pharmacy Use to Improve Outcomes and Reduce Disparities

The investigators propose a randomized encouragement trial to encourage use of the existing mail order pharmacy services among diabetes patients with poor adherence to CVD risk factor medications in 3 health care systems: Kaiser Permanente Northern California, Harvard Pilgrim, and Kaiser Permanente Hawaii.

Adherence to effective cardiovascular disease (CVD) risk factor medications is associated with improved CVD risk factor control, fewer hospitalizations, and lower mortality in patients with diabetes. However, many patients are poorly adherent to medications, and there are persistent racial/ethnic and socioeconomic disparities in medication adherence. Traditional clinical trials of interventions to improve medication adherence are often resource-intensive, and focus exclusively on patient-level barriers to behavior change. Unsurprisingly, these trials have not led to sustainable, cost-effective approaches to improve adherence. Health system-level medication adherence interventions that can be implemented, "scaled up," and sustained across a wide range of health care delivery settings are urgently needed. The investigators propose a randomized encouragement trial to encourage use of existing mail order pharmacy services among diabetes patients with poor adherence to CVD risk factor medications and who only use retail pharmacies in 3 health care systems: Kaiser Permanente Northern California, Harvard Pilgrim, and Kaiser Permanente Hawaii. These combined systems include approximately 300,000 patients with diabetes with diverse racial/ethnic and socioeconomic backgrounds. Patients with no history of mail order pharmacy use will be randomized into 2 arms. In addition to examining the impact of the intervention on medication adherence and CVD risk factor control, the investigators will examine factors affecting the sustainability and dissemination of the intervention, assess the intervention's impact on utilization and health care costs, and determine whether the intervention's impact differs across racial/ethnic and socioeconomic subgroups. This research will provide a foundation for developing sustainable, system-level approaches to addressing medication adherence in diabetes patients that can be widely disseminated and implemented across a diverse array of health care systems.

Eligibility Criteria:
Inclusion Criteria:

* Diabetes patients who are users of CVD risk factor medications (antihypertensive therapies, antihyperlipidemics therapies, and oral antihyperglycemic therapies),
* Diabetes patients considered to be poorly adherent to CVD risk factor medications within the prior 12 months
* Diabetes patients who have not used the mail order pharmacy to fill any prescribed medications at least once in the prior 12 months

Exclusion Criteria:

* Pregnancy or Dementia/Traumatic Brain Injury diagnosis in the prior 12 months
* Hospitalized, in Skilled Nursing Facility, Hospice, or Home Health at the time of randomization.