cyrilzakka/clinical-trials huggingface dataset:
Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

The purpose of this study is to find out more about some of the unusual chemicals (called oligosaccharides) that can occur naturally as a result of processes in the body. Researchers want to look at how these chemicals change with time and how they change between different patients with MPSIVA. These unusual chemicals were recently discovered in the urine from patients with MPSIVA. The investigators would like to study these chemicals before a specific enzyme replacement therapy is used. If the investigators understand how these chemicals change, the investigators may be able to use them to monitor this condition in the near future as well as help doctors know whether certain therapies work well in their patients.

This is not a clinical trial and there is no outcome measurement. The biomarkers in this study are oligosaccharides that secreted in the urine from patients with MPSIVa. In this study, we will try to define the variability of these oligosaccharides in the same patient at different time points, including different month of a year and different collections during the day as well as variations of these oligosaccharide in different affected patients.

Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of Mucopolysaccharidosis IVA

Exclusion Criteria:

* Patients receiving enzyme replacement therapy.
Effect of Speech Enhancer on Listening Effort With EEG

In previous studies, Speech Enhancer has been shown to improve Listening Effort using a subjective method. To overcome the weakness of subjective testing, this study uses an objective measurement that has shown convincing results in previous studies addressing Listening Effort.

The Speech Enhancer (SE) provides better speech intelligibility for soft speech while maintaining the same sound quality for loud input level.

Better speech intelligibility may also result in less listening effort in some circumstances. So there was the hope that the SE provides better speech intelligibility and less listening effort for situation with soft speech.

The SE has already been intensely and successfully investigated in early studies.

The cognitive performance on a neurophiosiological level has already been successfully investigated in a previous studies.

Eligibility Criteria:
Inclusion Criteria:

* Experienced (minimum use duration 6 months)
* Adult (minimum age: 18 years) hearing aid users,
* Symmetrical hearing loss (difference between ears \<= 10 dB for 1kHz-6kHz),
* Sensorineural hearing loss,
* Air Conduction - bone conduction gap \<= 10 dB,
* Written and spoken German,
* Ability to understand instruction,
* Ability to describe listening experiences,
* Ability to attend to the appointments,
* Healthy outer ear (without previous surgical proce-dures),
* Hearing loss within the fitting ranges of the investiga-tional product,
* Informed consent as documented by signature.

Exclusion Criteria:

* Clinical contraindications deformity of the ear (closed ear canal or absence of pinna),
* Known hypersensitivity or allergy,
* Not willing to wear the hearing aid,
* Fluctuating hearing that could influence the results,
* Limited mobility and not in the position to attend all appointments,
* Inability to produce a reliable hearing test result,
* Massively limited dexterity,
* Known psychological problems,
* Known central hearing disorders.
Atopic Dermatitis: Early Gene Expression Changes as Predictors of Therapeutic Response to Narrow-band UVB Treatment

Phototherapy with narrow band (nb) ultraviolet B (UVB) is a safe and effective but time consuming treatment option for patients with widespread eczema. Despite efficacy we know little about how it works, and even less why some patients fail to respond. Tintle et al showed that nb-UVB induced strong suppression of the Th2 and Th22 axes in patients with atopic dermatitis (AD), and also normalized the epidermal barrier function. We want to map the very early changes in gene expression after UVB-treatment in order to shed light on disease mechanisms, which eventually could lead to better treatment options.

Patients will be scored with Patient-Oriented Eczema Measure (POEM), Eczema Area and Severity Index (EASI), SCORAD (SCORing Atopic Dermatitis) and Dermatology Life Quality Index (DLQI) to assess disease severity. The investigators will measure trans epidermal water loss (TEWL), take bacterial swabs for analysis of the microbiome and culture for Staphylococcus aureus and test for mutations in the filaggrin-gene by sputum samples. Blood serum samples will be taken to measure serum (s) -Immunoglobulin E, s-vitamin D status, full blood count including white cell differential and different cytokines. Any history of allergic asthma, allergic rhinitis or food allergy will be noted as well as any recent history of sun exposure, use of tanning beds or UVB-therapy.

The suberythemal dose of UVB for each patient will be established. In each patient two contralateral areas (e.g. both underarms) with eczema will be chosen, which are as similar as possible regarding distribution and severity. One area will be biopsied with a 4mm punch followed by locally treatment with nb-UVB (311nm) three times. In this way the untreated side will serve as an internal control. Both sides will be biopsied after these initial three rounds of treatment. The patients will then undergo 24 standard full-body nb-UV-treatment sessions followed by a new biopsy of the same area. Skin from non-lesional sun-protected skin from nates will also be samples prior to and after UVB-treatment.

Eligibility Criteria:
Inclusion Criteria:

* Atopic dermatitis

Exclusion Criteria:

* Other inflammatory skin disease
* Concurrent illness
Building the Path to Resilience in Preterm Infants: Mindfulness-based Intervention

Yearly 15 million babies worldwide are born too soon. 10% of these preterm births occur very early before 32 weeks of gestation and these newborns are at high risk for neurodevelopmental disorders later in life. Neurocognitive disorders now touch 27% of the European population, and 5% or 3.3 million children suffer from social and learning difficulties, including attention-deficit hyperactivity disorders and autism, whose rates are increasing and prematurity contributes to this rise. Cognition, and socio-emotional competence are based on intact brain structure and functions that are formed early in development, both pre- and post-natally, and are heavily influenced by environment. Ramon y Cajal in his studies on the making of the brain clearly stated: "The total arborisation of a neuron represents the graphic history of conflicts suffered during its developmental life". Understanding how environment affects early brain development and defining timing and mode of early interventions to enhance brain development in high risk populations, such as preterm infants, is currently acknowledged as a fundamental endeavor for the scientific community (see guidelines of the National Scientific Council for the Developing Child). Interventions to improve and maintain cognitive and socio-emotional skills are to become an essential tool of medical care for high-risk infants. The goal of this study is to test the impact of a Mindfulness-based intervention - considered to target brain networks previously described as affected by prematurity and improve socio-emotional and executive functions. Mindfulness based intervention (intentional self-regulation of attention) will be performed in 10-13 year old preterm children, both from our prior studied preterm cohorts. Overall, our planned research will fill an important gap in our theoretical understanding of the brain vulnerability linked to prematurity. Even more importantly, the compelling issue of how to build cognitive and emotional resilience in preterm children will be addressed by preventing the onset of difficulties and reducing them with appropriate interventions.


Eligibility Criteria:
Inclusion Criteria:

* born before 32 gestational weeks

Exclusion Criteria:

* severe sensory or physical disabilities (cerebral palsy, blindness, hearing loss)
* intelligence quotient below 70
* not French speaking
German Multicenter Trial for the Treatment of Newly Diagnosed T-lymphoblastic Lymphoma in Adults

This study evaluates the efficacy and tolerability of treatment for T-lymphoblastic lymphoma (T-LBL) according to a protocol for acute lymphoblastic leukemia. Patients receive one year of intensive cyclical chemotherapy with additional prophylaxis for central nervous system (CNS) relapse by intrathecal therapy and cranial irradiation and mediastinal irradiation after induction chemotherapy.


Eligibility Criteria:
Inclusion Criteria:

* T-lymphoblastic lymphoma (bone marrow \[BM\] involvement \< 25%)
* Aged 15-65 years (55-65 years if biologically younger)
* Written informed consent

Exclusion Criteria:

* Severe complications due to lymphoma or secondary disease
* T-LBL as second malignancy or other active second malignancy
* Cytostatic pretreatment of LBL (exception of emergency treatments)
* Pregnancy
* Severe psychiatric illness or other circumstances which may compromise cooperation of the patient or informed consent
* Participation in other study interfering with study treatment
Sensory Stimulation Effect on Movement Speed in Patients With Parkinson Disease

Movement slowness (bradykinesia) is one of the main motor symptoms in Parkinson Disease (PD). Several studies have shown that patients with PD exhibit slowness because they are unable to modulate, in an optimal way, the velocity of voluntary motor acts not induced by external stimulation. Indeed, these patients have difficulties to integrate multi-sensorial information, mainly proprioception.

The investigators investigated changes in shoulder velocity during pointing movements by patients with PD after stimulation of soft tissues (aponeurosis) of upper limb muscles. The stimulation consisted of manipulating, with a hook (the diacutaneous fibrolysis method), the aponeurotic tissues enrobing the heads of the upper limb muscles. This technique has previously been shown to decrease passive tension and the tendon reflex response of the manipulated muscle group. The investigators hypothesis is that aponeurotic manipulation of shoulder muscles therefore creates a modification in the proprioceptive information, which in return temporarily decreases the bradykinesia of shoulder movements.


Eligibility Criteria:
Inclusion Criteria:

* Clinical diagnosis of Parkinson Disease

Exclusion Criteria:

* Patients with a limitation in the shoulder range of motion necessary to perform pointing movements
Effect of Rapamycin to Improve Cardiac Function in Frail Older Adults

The purpose of this research is to see if the drug rapamycin will improve the heart's ability to pump by improving your oxygen consumption.

This will be a pilot, proof-of-concept study. Patients 60 years or older with invasively proven HFpEF or have definite HFpEF as determined by the 2 established scores and frailty (positive screen for computable phenotype), will be approached for enrollment. Subjects meeting screening criteria following written informed consent will be randomized to receive rapamycin or matching placebo (study drug). Efficacy outcomes will be measured at baseline and at 6 months. Safety will be assessed by in person or remote video monitoring weekly for 1 month, then monthly for 5 months till the end of the study. We will also measure trough rapamycin levels (≤4ng/mL) twice-a-month for the first month and then monthly for 5 months.

Eligibility Criteria:
Inclusion Criteria:

* Age ≥60 years
* Diagnosis of HFpEF by either H2FEPF score of 6-9 or HFA-PEFF score of 5-6, or invasively confirmed diagnosis of HFpEF (resting pulmonary capillary wedge pressure ≥15mmHg or ≥25mmHg with exercise).
* Diagnosis of computable phenotype of frailty rests on screening questions about ability to climb two flights of stairs, activities of daily living, living environment, wearing of dentures/hearing aids, and whether the patient uses assistive devices.

Exclusion Criteria:

* Reduced ejection fraction (≤50%) with or without prior history of myocardial infarction
* Acute coronary syndrome \<3 months
* Uncontrolled diabetes mellitus (HbA1C\>8)
* Uncontrolled hypertension (\>160/100mmHg, despite 3 antihypertensive medications)
* Nephrotic syndrome or eGFR \<30mL/min/1.73m2
* Cirrhosis
* Hepatitis B/C positive
* Elevated lived enzymes (AST/ALT\>3ULN
* Prior malignancy other than basal cell carcinoma
* History of ongoing, chronic or recurrent infectious disease
* Suspected/proven immunocompromised state
* Uncontrolled hyperlipidemia (fasting TG \>400 mg/dL (\>4.5 mmol/L) or total cholesterol \>300 mg/dL (\>7.8 mmol/L) despite maximum lipid lowering therapy
* Class IV HF symptoms
Prediction Model of Vitamin D Deficiency

Many studies have pointed out that patients with vitamin D deficiency have a longer stay in the intensive care unit and a poor prognosis. Previous multi- center prospective observational study in Taiwan reveals that the prevalence of vitamin D deficiency in critically ill patients in northern Taiwan is 59%, and the prevalence of severe vitamin D deficiency is 18%. Several prediction models of vitamin D deficiency had been built for the general population but not patients admitted in intensive care units.

This multi-center retrospective study aims to develop and validate a score-based prediction model for severe vitamin D deficiency in critically ill patients. Investigators will review the data of previous multicenter, prospective, observational study. For temporal validation, the data will be divided into a derivation cohort (first 80% of the data set based on chronology) and a validation cohort (the remaining data set). The development and validation of the models will be carried out following the recommendations established in the Transparency Reporting of a multivariable prediction model for Individual Prognosis or Diagnosis (TRIPOD) initiative.


Eligibility Criteria:
Inclusion Criteria:

* Patients admitted to intensive care units

Exclusion Criteria:

* aged \< 20 years
* admitted to intensive care unit longer than 28 days
* body max index \< 18 kg/m2
* receive vitamin D supplement \> 3000 IU/day
* previously admitted to intensive care unit within 3 months
* have parathyroid disease, rickets, or severe liver cirrhosis \[Child C\]
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an allogeneic transplant.

This 2-year open-label pilot study of laronidase includes patients (age 5-13 years) who are at least 2 years post-hematopoietic cell transplantation (HCT) and donor engrafted. Outcomes are assessed semi-annually and compared to historic controls. Eligible patients will receive Laronidase as an infusion over several hours once a week at a local site. The dosing of enzyme will be the standard doses recommended by Genzyme.

The findings of this Pilot Study will be used to assess whether a subsequent larger study can be conducted.

Eligibility Criteria:
Inclusion Criteria:

* Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant \>2 years previously
* Age \<14 years old
* \>10% engrafted based on recent testing (\<4 months prior to enrollment)
* Willing to commit to traveling to the University of Minnesota every 6 months
* Written informed consent prior to the performance of any study related procedures

Exclusion Criteria:

* Previous administration of Laronidase enzyme \> 3 months post transplantation
* Anticipated survival less than 2 years
* History of cardiac or pulmonary insufficiency, including an ejection fraction (EF) \< 40% or those requiring continuous supplemental oxygen
Prognostic Value of Pediatric GCS-Pupil Score in Pediatric Patients With Traumatic Brain Injury

There are studies reporting that the survival rate in pediatric patients with abnormal pupillary response is 23% and that pupillary response is effective on mortality and patient outcomes. Studies in the literature examining the effect of pupillary response on mortality and outcome in pediatric patients with traumatic brain injury reported that bilaterally dilated pupils were associated with a higher mortality rate.

Considering the literature results, we believe that obtaining the GCS-Pupil score by combining GCS and pupillary reaction in pediatric patients with traumatic brain injury will be effective in predicting patient outcomes.

Obtaining a GCS-Pupil score using the total GCS score: 0 points are given for pupillary reaction if both pupils react to light, 1 point if one pupil does not react to light, and 2 points if both pupils do not react to light. Total GCS-P score is calculated by the formula of Eye Response + Verbal Response + Motor Response - Pupil Reaction and scores between 1-15.

In the study, the worst GCS and current pupillary light reaction score in the first 24 hours will be evaluated.

Eligibility Criteria:
Inclusion Criteria:

* Be under the age of 18,
* Not having mental and motor developmental retardation,
* Having had a traumatic brain injury,
* Alive at admission and followed up for at least 24 hours

Exclusion Criteria:

* Eye trauma or severe facial trauma that will affect the assessment,
* Those under deep sedation,
* Those who have been administered drugs (Atropine and mydriatic) that will affect the pupillary response.
National Register of Moderate and Severe Adult Atopic Dermatitis

Collect clinical history and treatment data of AD in adulthood;

Atopic dermatitis is a chronic-recurrent inflammatory skin disease with high prevalence in children and frequently presenting clinical remission during or after childhood; however, it may persist and/or recur, even after long and transient remission, adulthood. In addition, in some adult patients, DA is not present in pediatric age, but onset in adulthood: the investigators speak, in these cases, of adult-onset DA. The prevalence of DA is increasing all over the world and the cases in which clinical manifestations appear or persist during adolescence and/or adulthood are increased especially in industrialized countries. are not currently available specific laboratory markers for diagnosis of DA and, therefore, diagnosis is eminently clinical. In fact, at the present time, there are no validated diagnostic criteria, nor is there a clear consensus on diagnostic-therapeutic pathway to be followed when evaluating patients with adult DA. For these reasons, the collection of data related to various aspects of the DA such as those epidemiological, medical and clinical history, as well as the impact of the disease on the quality of life, results of crucial importance for estimating the real prevalence of this disease in Italy and also, possibly, to outline possible diagnostic criteria specific to adult DA, so that to facilitate and improve the diagnostic-therapeutic path of the patient. The intention of the project, therefore, is to create and use a National Register dedicated to DA patients in adulthood regardless of the period of onset of their pathology. aim to place the Italian Register as a supranational reference for international studies conducted on the adult DA.

Eligibility Criteria:
Inclusion Criteria:

1. Age 18 years
2. Signature of informed consent
3. Diagnosis of moderate and/or severe DA by a dermatologist specialist defined on the basis of following criteria (10):

   * EASI 16
   * EASI \<16 but with at least one of the following conditions:

     1. location in at least one of the following "critical" locations: face, hands, genitals
     2. QID \> 10
     3. VAS itching \> 7
     4. VAS sleep \> 7

Exclusion Criteria:

1. Patient unable to provide informed consent prior to any collection procedures data on the study;
2. unable to complete the procedures required for the study;
3. a patient already participating in another follow-up register of the same disease.
Naltrexone and Patch for Smokers

The aim of this study is to investigate the effects of naltrexone, alone and combined with nicotine patch, on responses of smokers to smoking cues after 10 hours of tobacco deprivation. Smokers who are not seeking treatment will be assigned to one of six conditions: They will receive either 50 mg of naltrexone or a placebo pill, and also will wear a nicotine patch that has 0, 21, or 42 mg of nicotine during the tobacco deprivation period. Both the day before the medication and deprivation and at the end of 10 hours of deprivation all will be exposed to lit cigarette cues in the laboratory. Effects of the medications will be assessed on withdrawal measures, urge to smoke, psychophysiological measures, and the topography of smoking three test cigarettes. Studies such as these can help to identify potential interventions for tobacco cessation or withdrawal, and thereby could result in less suffering and mortality.


Eligibility Criteria:
Adults who have smoked at least 25 cigarettes per day for at least a year. They are not using any method to quit smoking and are not using naltrexone. Must weigh at least 100lbs with no medical contraindications for naltrexone or transdermal nicotine. No contraindicated medications, no recent opiate use or history
The Effect of an Oral Supplement,TK3 (Tryptophan and Thiamine) on the Quality of Life and Chemotherapy Tolerance in Cancer Patients With Advanced Disease.

The product under investigation relates to a pharmaceutical composition containing a pyrimidine nitrogen base, thymine, and the essential amino acid tryptophan. This product seems to have effect on quality of life and enhance adverse affects of chemotherapy in cancer patients.

The primary objective of this study is to evaluate the impact of TK3 on the quality of life of patients receiving anti-neoplastic chemotherapy. The secondary objectives are to evaluate the influence of TK3 on the side effects caused by anti-neoplastic chemotherapy, tolerance to this treatment and the safety of the use of the TK3 product in cancer patients, accompanying the appearance of non-characteristic effects of the existing cancer type .

Eligibility Criteria:
Inclusion Criteria:

* Patients who agree to study procedures and sign the informed consent
* Patients 18 years of age or older;
* Patients undergoing oncologic treatment for advanced neoplasms. It is understood by advanced patient disease patients with unresectable or with distant metastases.
* Participants should present at least one (1) of the following clinical side effects that can be assessed according to CTCAE (Version 4.0): fatigue, nausea, vomiting, diarrhea, or mucositis.
* Patients with programming to receive at least 3 cycles of anti-neoplastic chemotherapy;
* Patients may be on radiotherapy

Exclusion Criteria:

* Use of vitamin complex or concomitant food supplement;
* Current use or programming of parenteral diet use;
* Hypersensitivity to the components of the formula;
* Participation in another clinical study or use of any investigational product within 28 days prior to commencement of treatment in this clinical trial, unless the Investigator deems there to be a clinical benefit therefore;