cyrilzakka/clinical-trials huggingface dataset:
Mitochondrial Membrane Protein Neurodegeneration (MPAN)

The purpose of this case study was to investigate the effects of therapeutic intervention in 9- and 12-year-old brothers with MPAN. The main hypothesis it aims to answer is:

-A 6-week balance and coordination focused rehabilitation program improves independence and gait parameters in patients with MPAN.

Neurodegeneration with brain iron accumulation (NBIA) comprises a heterogeneous group of disorders with accumulation of iron in the brain, mostly basal ganglia. Mutations within C19orf12 have recently been identified in patients with NBIA. This C19orf12 gene -located in mitochondria and endoplasmic reticulum- encodes a mitochondrial membrane protein and the abbreviation MPAN. Pathogenic mutations in this gene are postulated to cause dysfunction of lipid homeostasis in mitochondria.

Patients with MPAN typically present with gait disturbance due to lower limb spasticity and dystonia, parkinsonism with subsequent spastic paresis, dysarthria, cerebellar ataxia, behavioral problems, dementia, psychomotor delay, peripheral neuropathy, visual changes like optic atrophy as well as bowel/bladder incontinence; however, MPAN has a greater variability compared with other NBIA cases in terms of age of onset (age three to 30 years) and speed of progression.

This study aims to investigate the effects of therapeutic intervention in male brothers, age 9 and 12, with MPAN. In particular, this study tested the following hypotheses after 6 weeks of rehabilitation by measuring muscle strength, muscle tone, range of motion , balance and coordination: (1) After the training period, muscle strenght, muscle tone and range of motion would be improved, (2) these developments in body function can lead to increase balance and coordination, and (3) due to the these improvements, functional independency may increase.

Eligibility Criteria:
Inclusion Criteria:

* Being diagnosed with MPAN

Exclusion Criteria:

* being involved in any other rehabilitation program
EndoBarrier Register Deutschland-Safety and Efficacy of the Endoscopic Duodenal-Jejunal Bypass Sleeve

The purpose of this study ist to determine long-term safety and efficacy in weight reduction and improvement of diabetes mellitus of the temporary, endoscopic duodenal-jejunal bypass-sleeve EndoBarrier® in a national registry.

Long-term data are collected by approximately 30 German centers. An electronic Case-Report-Form (eCRF) was designed to collect relevant pre-specified items by the Clinical Trial Center North (CTC). All German sites that have implanted EndoBarrier® since 2010 were invited to provide patient results. Data-acquisition is projected to a period of 5 years and a maximum of 1000 patients. An extension of data-acquisition is planned.

Eligibility Criteria:
Inclusion Criteria:

* clinical decision for treatment with EndoBarrier
* informed consent for registry participation

Exclusion Criteria:

* Patients not meeting the inclusion criteria
* age \< 18
Mechanical Ventilation Practices in Uganda's Intensive Care Units

A prospective cohort study carried out in four ICUs in Uganda. All patients, 18 years and above initiated on mechanical ventilation will be recruited. Patients' data will be collected from the files and charts at initiation of mechanical ventilation. Patients will then be followed up for death / discharge within 28 days in ICU.

Background: The need for mechanical ventilation is one of the commonest indication for admission to the ICU. Despite the clear benefits of mechanical ventilation, it may be detrimental to the patient if poorly carried out. This has led to a renewed impetus in employing lung protective ventilation strategies, Ventilator Associated Events (VAE) prevention strategies, and safe and appropriate weaning methods to reduce mortality among mechanically ventilated patients. However, there is paucity of data regarding these practices in low-income settings.

Objective: To describe the mechanical ventilation practices, mortality rate and associated factors among mechanically ventilated patients in Uganda's intensive care units.

Methodology: A prospective cohort study carried out in four ICUs in Uganda. All patients, 18 years and above initiated on mechanical ventilation will be recruited. Patients' data will be collected from the files and charts at initiation of mechanical ventilation. Patients will then be followed up for death / discharge within 28 days in ICU.

Data analysis: The primary outcome is all-cause mortality during ICU stay. Data will be summarised into means and standard deviations, medians and interquartile ranges and presented in tables, charts, cross-tabulations and graphs. Univariate and multivariate logistic regression will be used to determine factors associated with mortality.

Utility of results: The results from this study will be disseminated to the Ministry of Health and Makerere University in order to plan, standardise and form guidelines regarding mechanical ventilation practices in ICUs in Uganda.

Eligibility Criteria:
Inclusion Criteria:

* All adult patients 18 years and above, initiated on mechanical ventilation, admitted into study ICU during the study period

Exclusion Criteria:

* Patients ventilated in another ICU for more than 24 hours and transferred to study ICUs
Short Course Radiotherapy in Complicated Bone Metastases Palliation

The study wants to define the maximum tolerated dose (MTD), safety and efficacy of a short course radiation treatment in patients with complicated bone metastases.

The study wants to define the maximum tolerated dose (MTD) of a conformal short course radiation treatment delivered in twice daily fractions and 2 consecutive days, and the feasibility of this fractionation in term of safety and efficacy for symptomatic palliation of complicated bone metastases.

Eligibility Criteria:
Inclusion Criteria:

* radiologically proven complicated bone metastasis
* age \> 18 years
* Eastern Cooperative Oncology Group (ECOG) performance status \< 3

Exclusion Criteria:

* prior RT to the same region
Evaluation of the Efficacy of Different Drugs in the Treatment of the Pain in Patients With Temporomandibular Disorder

The most important step in the treatment of TMJ (temporomandibular joint) disease in clinical dentistry is diagnosis and planning. The clinical findings, drug requirement and biological risk of the patients determine the examination methods to be used in the Treatment of Temporomandibular Joint Disorders. In this context, it is thought that studies in the field of dentistry should be increased in order to increase the success in the treatment of TMJ Disorders. The aim of the study is to evaluate the efficacy of of different pain relievers as Dexketoprofen Trometamol + Thiocolchicoside, dexketoprofen trometamol + paracetamol and Dexketoprofen Trometamol in the treatment of pain in TMJ disorders.

The study was carried out on 200 patients aged 18 and over, who applied to Van Yuzuncu Yıl University, Faculty of Dentistry, Department of Oral and Maxillofacial Surgery, with complaints of pain due to temporomandibular disease. When patients first applied to the clinic, their anamnesis were taken and they were directed to MR (Magnetic Resonance) imaging after clinical evaluation. Patients who meet the inclusion criteria and volunteer were included in the study. Patients were randomly divided into 4 groups. Medications were given to patients by auxiliary staff and the study was double-blind. Medicines were given as follows. Dexketoprofen Trometamol + Thiocolchicoside (25mg + 4mg, 2x1) in the 1st group, Dexketoprofen Trometamol + Paracetamol (25mg + 300mg, 2x1) in the 2nd group, only Dexketoprofen Trometamol (25mg, 2x1) in the 3rd group and paracetamol 500 mg in the 4th control group (4x1) were given. In addition, patients in each group used occlusal plate for a minimum of 8 hours per day (at night). Medicines were used regularly for 2 weeks. Paracetamol 500 mg was used only if needed for the next 2 weeks. The pain values of the patients was evaluated with the VAS scale, and the amount of mouth opening was evaluated by measuring the distance between the incisal edges of the lower and upper central teeth. Evaluations have been done at the beginning of the treatment and at the 1st week, 2nd week, 1st month and the 3rd month and the findings were recorded. After the 1st month, the patients were followed up to the 3rd month and the treatment of those who need additional treatment were continued. Obtained data will be analyzed and evaluated statistically.

Eligibility Criteria:
Inclusion Criteria:

1. Individuals aged 18 and over
2. Individuals without any systemic disease
3. Individuals without missing teeth
4. Individuals who have not used any medication in the last week
5. Individuals with normal TMJ MR imaging
6. Patients who applied to our clinic and had pain due to temporomandibular disease.

Exclusion Criteria:

1. Individuals who do not come to their check-ups regularly
2. Individuals allergic to any of the study drugs
3. Pregnant and lactating individuals
Posture and Spinal Mobility in Anorexia Nervosa

The primary objective of the study is to compare the results of the postural and mobility analysis obtained with the use of the Idiag M360 instrument in adult women with anorexia nervosa and in a control group represented by age-matched normal-weight women.

Secondary objectives will be to correlate the results obtained from previous postural analyses with anthropometric parameters, the duration and onset of the disease, and the presence/date of onset of secondary amenorrhea.


Eligibility Criteria:
Anorexia Nervosa:

Inclusion Criteria:

* Female sex
* Age 18 years and older
* Anorexia nervosa clinically diagnosed based on the DSM-5 criteria
* Hospitalized in the Division of Eating and Nutrition Disorders (END), Istituto Auxologico Italiano, IRCCS, Piancavallo (VB), Italy

Exclusion Criteria:

* Absence of signed informed consent
* Musculoskeletal or neurological conditions (leg length discrepancy, spinal deformities, and physical disabilities caused by surgery)
* Treatment with anti-inflammatory drugs

Normal Weight:

Inclusion Criteria:

* Female sex
* Age 18 years and older
* BMI: 18.5-24.9 kg/m2
* Recruited from the medical/nursing and research staff

Exclusion Criteria:

* Absence of signed informed consent
* Musculoskeletal or neurological conditions (leg length discrepancy, spinal deformities, and physical disabilities caused by surgery)
* Treatment with anti-inflammatory drugs
Radiomics and Radiogenomics of Non-small Cell Lung Cancer

In this study, the radiomic characteristics and a broad range of genetic aberrations in lung adenocarcinomas will be evaluated. Investigators will assess changes in the radiomic and genetic profiles during targeted therapies in a subset of patients harboring treatable mutations. Patients undergoing targeted therapies will also be evaluated for variations in genomic profile and radiomic signature during follow-up

Non-small cell lung cancers (NSCLC) are the first cause of cancer-related deaths.

Recent guidelines suggest the key role of a wide molecular profile because knowledge of the tumor genotype can enable patients with NSCLC to be treated with targeted therapies.

In this study, a first group of patients with lung adenocarcinoma (study group), tested for the presence of genetic alterations in EGFR, ALK, and KRAS, and with available Computer Tomography (CT) scans, will be evaluated to create a radiomic signature and assess its association with mutational status and prognosis.

Then, a second group of patients (validation group) will be enrolled to validate the radiomic signature and the status of the genetic alterations and their association with prognosis. Radiomic signature and liquid biopsies will be evaluated in patients positive for genetic alterations at each follow-up step to verify their association with prognosis and response to targeted therapies.

Eligibility Criteria:
Inclusion Criteria:

* patients with diagnosis of NSCLC
* patients candidates for mutational status assessment

Exclusion Criteria:

- patients with age \<18 years
Pulse Oximetry Measurement and Transfusions

The primary purpose of this observational study was to determine if pRBC transfusions decrease the frequency of intermittent hypoxia events in very low birth weight infants (VLBW) during the first six weeks of life. The impact on non-pRBC transfusions on the frequency of intermittent hypoxia was also assessed.


Eligibility Criteria:
Inclusion Criteria:

* 23+0 to 28+6 weeks gestational age at birth

Exclusion Criteria:

* Infants with life-limiting diagnoses
* Cyanotic heart disease
* In utero fetal transfusion
* Twin to twin transfusion syndrome
* Isoimmune hemolytic disease
* Severe acute hemorrhage
* Acute shock
* Sepsis with coagulopathy
* Need for perioperative transfusion
* Parents opposed to transfusion of blood
* Parents with hemoglobinopathy or congenital anemia
Efficacy and Safety of Sesame Oil Cake Extract on Improvement of Cognitive Function

This study was the efficacy and safety of Sesame oil cake extract on improvement of Cognitive Function

This study was a 12 weeks, randomized, double-blind, placebo-controlled human trial to evaluate. 70 subjects were participated in Sesame Oil Cake Extract or placebo group. To evaluate the changes of the evaluation items when Sesame Oil Cake Extract was taken three times a day compared with placebo.

Eligibility Criteria:
Inclusion Criteria:

* Those who are at least 60 years of age at screening
* Those who word list memory / recollection/ recognition test scores of CERAD -K decrease 1-2 SD number of normal value
* Those who agree to voluntary participation and to comply with the Notice after fully hearing and understanding the details of this human trial

Exclusion Criteria:

* Those with a past history of treatment with Axis I disorder in SCID(Structured Clinical Interview for DSM-IV) which is a structured clinical interview in the Diagnostic and Statistical Manual of Mental Disorders at Screening or who have been treated within the last 3 years
* Those with alcohol abuse or dependence within the last 3 months
* Those who have clinically significant following severe illness (Epilepsy, mental retardation, brain nervous system diseases, endocrine diseases, blood / malignant tumors, cardiovascular diseases, Crohn's disease, etc.)
* Those who shows the following results in the Laboratory test

  * AST, ALT \> 3 times upper limit of normal range
  * Other significant laboratory test opinion
* Those who take any prescription medicines or herbal medicines within 2 weeks before the first ingestion date or take any OTC or vitamin preparations within 1 week
* Those who participate in other human trial within 2 months before the first ingestion
* Those who whole blood donation within 1 months before the first ingestion or Component blood donation within 2 weeks
* Those who is deemed unsuitable for participating in the human trial due to other reasons
TGR-1202, a PI3K Delta Inhibitor, in Combination With Obinutuzumab and Chlorambucil in Patients With Chronic Lymphocytic Leukemia (CLL)

The purpose of this study is to evaluate the safety and effectiveness of TGR-1202 in combination with obinutuzumab (Gazyva) and chlorambucil in patients with chronic lymphocytic leukemia.


Eligibility Criteria:
Inclusion Criteria:

* Confirmed Chronic Lymphocytic Leukemia (CLL)
* Eastern Cooperative Oncology Group (ECOG) score of 0 to 2
* Ability to swallow oral medication

Exclusion Criteria:

* Known hepatitis B virus, hepatitis C virus or HIV infection
* Primary central nervous system lymphoma or known intracranial involvement
* Autologous hematologic stem cell transplant within 3 months of study entry or Allogeneic hematologic stem cell transplant within 12 months
Efficacy Study of Sorafenib and Cyclophosphamide to Treat Neuroendocrine Tumors

This is a phase II clinical trial to assess the efficacy of the combination of metronomic cyclophosphamide and tailored sorafenib dosing in advanced, progressive NET. NET are highly vascular tumors, and high VEGF expression has been correlated with worse clinical and pathological characteristics as well as poor prognosis. A novel antiangiogenic approach relies on targeting not only the endothelial cells but also rendering them more sensitive to VEGFR blockade by achieving pericyte detachment. In this study, the dose of sorafenib will be titrated up to a maximum of 800mg BID based on patients' toxicity and on a novel pharmacodynamic assay that measures inhibition of molecular target(PDGFR) in patients' peripheral blood mononuclear cells. Dual VEGFR targeting is achieved by administering sorafenib plus metronomic low dose cyclophosphamide.


Eligibility Criteria:
Inclusion Criteria:

* Histologically or cytologically confirmed neuroendocrine tumors
* Progressive and measurable metastatic disease
* Patients must not have disease that is currently amenable to surgery
* Life expectancy of greater than 3 months
* ECOG performance status ≤2
* Patients must have normal organ and marrow function
* Negative pregnancy test; agreement to use adequate birth control

Exclusion Criteria:

* Patients receiving chemotherapy or radiotherapy within last 4 weeks
* Patients that had received Sorafenib for advanced NET(neuroendocrine tumors) are not allowed
* Any other investigational agents within 4 weeks of study
* Patients with known brain metastases
* History of allergic reactions to compounds of similar chemical/biologic composition to sorafenib or cyclophosphamide
* Concurrent cancer from another primary site requiring treatment within the past 3 years
* Uncontrolled intercurrent illness
* Pregnant women and women who are breastfeeding
* HIV-positive patients receiving combination anti-retroviral therapy
Chronic Obstructive Pulmonary Disease: CT Features of Severe Exacerbation

To describe Computed Tomography (CT) features associated with severe exacerbations of Chronic Obstructive Pulmonary Disease (COPD).

The natural course of COPD is associated with episodes of exacerbation which are clinically defined as acute events characterized by a worsening of the patient's respiratory symptoms that is beyond normal day-to-day variations and which leads to a change in medication. Moreover, these episodes can be classified into mild, moderate, or severe exacerbations, following patient's need for medical environment. While the cause of about one-third of severe exacerbation episodes cannot be identified, two-thirds are associated with certain conditions such as respiratory tract infections (viral or bacterial), air pollution, as well as pulmonary embolism (PE), pulmonary edema, cardiac arrhythmia, pneumothorax, or pleural effusion. As COPD exacerbations are associated with increased morbidity and mortality, as well as with increased healthcare costs, their prevention and treatment are two major objectives in COPD management with subsequent requirement for appropriate assessment tools. As imaging tool, chest radiography is limited to the detection of pneumonia and pleural abnormalities, and only leads to change in managements in a marginal proportion of patients. However, while Computed Tomography (CT) scans allows detecting more chest abnormalities, the knowledge of CT features at the time of exacerbation is a pre-requisite for determining possible role of CT in routine work-up of exacerbation. Nevertheless these features remain widely unknown, previous studies having focused on the prevalence of PE. The aim of our study was therefore to describe these features by comparing CT scans performed at severe exacerbation with control scans.

Eligibility Criteria:
Inclusion Criteria:

* Severe exacerbation episode of COPD, requiring hospital admission

Exclusion Criteria:

* No exclusion criteria
Pilot Trial of Colchicine for Graft Failure in CABG

The goal of this pilot trial is to to evaluate the preliminary effect of oral colchicine therapy on graft outcomes in patients underwent primary isolated CABG.

The main questions it aims to answer is:

1. Whether the oral colchicine therapy may reduce the failure outcome of grafts after CABG.
2. Whether it is feasible to construct a muticenter powered trial to test the superiority hypothesis.

Researchers will compare colchicine to none to see if colchicine works.

Participants will

1. Take oral colchicine (0.5mg daily) therapy for 12 months after CABG.
2. Clinical follow-up at Month 1, 6, and 12 after CABG.
3. Protocol-driven CCTA at Week 1 and Month 12 after CABG.


Eligibility Criteria:
Inclusion Criteria:

* Age≥18 years old，
* Any sex，
* Signed informed consent，
* Within 3 days after a successful isolated CABG

Exclusion Criteria:

* Allergy,
* Hematopoietic dysfunction,
* Moderate to severe hepatic dysfunction,
* Moderate to severe renal dysfunction.
Fludarabine and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Metastatic Kidney Cancer That Cannot Be Removed By Surgery

RATIONALE: Giving low doses of chemotherapy, such as fludarabine, and radiation therapy before a donor stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening.

PURPOSE: This phase II trial is studying how well giving fludarabine together with total-body irradiation works in treating patients who are undergoing a donor stem cell transplant for metastatic kidney cancer that cannot be removed by surgery.

OBJECTIVES:

* Determine the efficacy of nonmyeloablative conditioning comprising fludarabine and total-body irradiation followed by allogeneic hematopoietic stem cell transplantation, in terms of 6-month and 12-month response rate, in patients with unresectable metastatic renal cell carcinoma.

OUTLINE:

* Nonmyeloablative conditioning regimen: Patients receive fludarabine IV on days -4 to -2 and total-body irradiation (TBI) on day 0.
* Allogeneic hematopoietic stem cell transplantation (AHSCT): After TBI, patients undergo AHSCT on day 0.
* Immunosuppression: Patients receive oral cyclosporine twice daily on days -3 to 56 followed by a taper until day 81. Patients also receive oral mycophenolate mofetil twice daily on days 0-27 (if patient has a related donor) OR three times daily on days 0-29 and then twice daily on days 30-149 followed by additional tapering until day 180 (if patient has an unrelated donor).

PROJECTED ACCRUAL: A total of 15 patients will be accrued for this study.

Eligibility Criteria:
DISEASE CHARACTERISTICS:

* Histologically confirmed renal cell carcinoma, including 1 of the following subtypes:

  * Clear cell
  * Papillary
  * Medullary
* Metastatic disease
* Not amenable to curative surgery
* No CNS metastases

PATIENT CHARACTERISTICS:

Performance status

* Karnofsky 70-100%

Life expectancy

* More than 6 months

Hematopoietic

* Not specified

Hepatic

* Bilirubin \< 2 times upper limit of normal (ULN)
* AST or ALT \< 4 times ULN

Renal

* Creatinine clearance \> 50 mL/min

Cardiovascular

* LVEF \> 35%
* No symptomatic congestive heart failure

Pulmonary

* DLCO \> 40% of predicted OR
* Total lung capacity or FEV_1 \> 30% of predicted

PRIOR CONCURRENT THERAPY:

Biologic therapy

* More than 30 days since prior biologic therapy

Chemotherapy

* More than 30 days since prior chemotherapy

Radiotherapy

* More than 30 days since prior radiotherapy