cyrilzakka/clinical-trials huggingface dataset:
Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)

The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.

The study design includes ANB-002 dose escalation in at three cohorts. In Cohort 1, the subject is treated with a single dose of ANB-002 (dose 1) administered as an intravenous infusion. Follow-up for the assessment of dose-limiting toxicity (DLT) will be carried out for 28 days. If no DLT events are observed in the subject of Cohort 1, the following subjects will be included in Cohort 1.

In Cohort 2, the subjects are treated with a single dose of ANB-002 (dose 2). In Cohort 3, the subject are treated with a single dose of ANB-002 (dose 3).

The decision to continue enrolling in cohorts will be made at the Independent Data Monitoring Committee (IDMC) meeting. After the IDMC makes a decision regarding the dosing subjects, further enrolment to the cohort will be carried out.

Based on the data from the follow-up period of subjects included in Сohorts 1-3, a potential therapeutic dose for further study will be determined and additional patients will be included to recieve this dose.

In exploratory Cohort 4 patients with anti-AAV5 antibodies and/or hepatitis B in anamnesis will be included. These subjects will recieve the dose 3 of ANB-002.

The total duration of participation of one subjects in the study will be 5 years.

Eligibility Criteria:
Inclusion Criteria:

1. Male with hemophilia B.
2. Age ≥18 years.
3. FIX activity at screening ≤2% without FIX inhibitor.
4. ≥150 previous exposure days of treatment with FIX concentrates.

Exclusion Criteria:

1. Previous gene therapy.
2. Other blood or hematopoietic disorders.
3. Positive Anti-AAV5 antibodies (for Cohorts 1-3).
4. Diagnosed HIV-infection, not controlled with anti-viral therapy.
5. Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.
6. Any active systemic infections or recurrent infections requiring systemic therapy at screening.
7. Any other disorders associated with severe immunodeficiency.
8. Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
9. Malignancies with remission \<5 years.
Healthcare Decisions on Dietary and Environmental Allergen Exposure

Design of this study is to collect data and review Principal Investigators standard operating procedures on allergy testing and changes made to their specific SOP.

The purpose of this minimal risk observational study is to increase surveillance for dietary and environmental allergens exposure and to also determine targeted risk reducing and preventative strategies.

Eligibility Criteria:
Inclusion Criteria:

* Must be a medical practitioner Medical Doctor (MD) Doctor of Osteopathic (DO) Physician Assistant (PA) Advanced Practice Registered Nurse (APRN) Nurse Practioner (NP) Must have a current standard operating procedure that includes obtaining/reviewing medical history and family medical history.

Exclusion Criteria:

* Government funded insurance data cannot be included in the study
Trial Comparing Pegfilgrastim With Filgrastim as an Adjunct to Chemotherapy for Acute Myeloid Leukaemia (AML)

The purpose of this study is to determine if a single dose of pegfilgrastim is able to reduce the time of severe neutropenia in patients receiving induction and consolidation myelosuppressive chemotherapy for de novo acute myeloid leukemia similar to filgrastim.


Eligibility Criteria:
Inclusion

* Histologically confirmed de novo AML as evidenced by absence of an antecedent hematologic disease of \> 1 months duration, prior chemotherapy, prior radiation therapy or myelodysplastic cytogenetics (as per exclusion criteria)
* Life expectancy, with treatment, \> 3 months
* Age \> 18 years
* ECOG performance status 0, 1 or 2
* Adequate organ function to receive protocol specified chemotherapy

Exclusion

* Subjects in blast transformation of chronic myeloid leukaemia (CML)
* Patients with secondary AML (Received previous chemotherapy or radiotherapy)
* Previous treatment for AML
* Patients with AML FAB type M3 (Acute Promyelocytic Leukemia, APL) or M7
* High risk (Unfavourable) cytogenetics \[(-5/del(5q), -7/del(7q), t(8;21) with del (9q) or complex karyotype, inv(3q), abn 11q23, 20q, 21q, del(9q), t(6;9), t(9;22), abn 17p, complex karyotypes(\>3 abnormalities)\]
Synergistic Effect of Vitamin E & D in Reducing Risk of Effects Associated With Atypical Anti-psychotics

Atypical antipsychotic drugs are commonly used to treat psychiatric illnesses but they are significantly associated with side effects including acute dystonia, akathisia, parkinsonism (rigidity and tremor), tardive dyskinesia, bradycardia, hypotension, impotence, sleepiness, seizures, severe dreams or nightmares, and hyperprolactinaemia. Vitamin D and E, have been the focus of much research in the past fifteen years, which has revealed multiple roles in the development and function of the body. According to mounting data from the domains of epidemiology and neuroscience, vitamin D and E deficiency have been related to a number of neuropsychiatric issues as well as neurodegenerative diseases. Additionally, antioxidants like vitamin E help to prevent inflammation and highly reactive oxygen molecules from damaging normal cells. The use of vitamin E and D supplements has been suggested to improve the overall outcomes of psychiatric illnesses and neurological diseases. However, the synergistic effect of vitamins E and D in reducing the risk of the adverse effects associated with atypical antipsychotics and improvement in psychiatric illness is not well understood. Therefore, this study was designed to investigate the potential synergistic effect of vitamin E and D supplements for reducing the adverse effects associated with atypical antipsychotics.

Inclusion Criteria:

1. Patients using antipsychotic medications such as quetiapine, olanzapine, or risperidone 3. Participants who are between the ages of 20 - 70 years 3. Both gender patients male and female 4. Patients with no other chronic morbidity

Eligibility Criteria:
Inclusion Criteria:

1. Those using antipsychotic medications such as quetiapine, olanzapine, or risperidone.
2. Participants who are between the ages of 20 - 70 years are both sex male and female
3. participants who are taking a combination of one or two antipsychotics.
4. Participants who were under antipsychotic therapy and not diagnosed with type 2 diabetes mellitus.

Exclusion Criteria:

1. Patients at mental hospitals are mostly women who are either pregnant or nursing.
2. Patients who were taking anticonvulsants, ketoconazole, or corticosteroids, or who had a history of other mental or neurologic illnesses, as well as those who used phosphor, calcium, vitamin D supplements or teriparatide, were not included in the study.
3. Participants were also ruled out if they had preexisting conditions including renal or hepatic failure or a parathyroid disease

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Pentoxifylline as an Adjuvant Therapy in the Treatment of Pediatric Pneumonia; A New Perspective of an Old Drug

Pentoxifylline is a xanthine-derived, commercially produced drug approved for the management of intermittent claudication in patients suffering from a chronic occlusive arterial disease of the limbs. Pentoxifylline has been documented to display anti-inflammatory and immunomodulatory effects, as well as some antithrombotic and antiviral effects. This drug has also been shown to reduce lung fibrosis in patients with COVID-19, as well as to prevent thromboembolic events. This work aims to assess the benefit of oral Pentoxifyllin supplementation, in addition to standard antibiotic and other supportive therapy, in the management of hospitalized children with community-acquired pneumonia

This is a randomized clinical trial study that will be carried out on 100 children with Community-acquired pneumonia admitted to the Pulmonology Unit, Pediatric department.

Group 1: Pentoxifylline group, and group 2: Control group

Eligibility Criteria:
Inclusion Criteria:

* All children aged 2 months -18 years with community-acquired pneumonia diagnosed by signs and symptoms of CAP including; chest pain, dyspnea, tachypnea, or abnormal auscultatory findings plus focal findings on chest x-ray indicating community-acquired pneumonia

Exclusion Criteria:

* Children with immunodeficiency, chronic lung disease, malignancy, congenital lung anomalies, underlying disorders impacting respiration i.e. genetic, metabolic, neuromuscular disorders, and children with CHD affecting the pulmonary blood flow.
Food-effect Study on Uracil and Dihydrouracil Levels as a Diagnostic Marker of Dihydropyrimidine Dehydrogenase Activity

Healthy volunteer food-effect study to determine the effect of food on uracil and dihydrouracil levels in plasma


Eligibility Criteria:
Inclusion Criteria:

1. Healthy volunteer; not known with cancer or current treatment for cancer
2. Age at least 18 years
3. Able and willing to give written informed consent
4. Able and willing to consume the prescribed breakfast
5. Able and willing to undergo blood sampling

Exclusion Criteria:

1. Any treatment with investigational drugs within 30 days before the start of the study
2. Any condition that may interfere with the study protocol
3. Women who are pregnant
4. Allergies or intolerance for components of the prescribed breakfast
Photo-Narrative Study

The objective of this study is to conduct a pilot randomized controlled trial (RCT) of a photo-narrative communication intervention developed by our study team with patients/parents of children with severe neurological impairment (SNI) and their pediatric intensive care unit (PICU) clinicians to assess feasibility, acceptability, and early efficacy.


Eligibility Criteria:
Patients

Inclusion Criteria:

* ages 3 months-25 years
* severe neurological impairment (including congenital/chromosomal, chronic central nervous system static or progressive conditions, or child \>6 months after severe traumatic brain injury
* admitted to the pediatric ICU at Seattle Children's and with medical team permission to approach
* expected length of stay \>1 week
* life expectancy \>4 weeks
* previous discharge home

Parents

Inclusion Criteria:

* parent/legal guardian
* English and/or Spanish speaking (most common languages spoken at SCH making up 95% of patients and families)
* \>18 years of age

Clinicians

Inclusion Criteria:

* licensed physicians, fellows, residents, advanced practice nurse practitioners, nurses, or respiratory therapists
* working in the pediatric ICU at Seattle Children's Hospital
Acceptability and Feasibility of Medical Abortion in Singapore

To test the hypothesis that a regimen of 200 mg oral mifepristone, with the option of home administration, followed by 800 μg buccal misoprostol 24 hours later for abortion through 70 days LMP will be feasible and acceptable in Singapore.


Eligibility Criteria:
Inclusion Criteria:

* Have an intrauterine pregnancy consistent wit gestational age ≤ 70 days
* Be 21 years of age or over
* Be willing and able to sign consent forms
* Be eligible for abortion according to current hospital guidelines
* Be able to return to the clinic and able to contact study staff or emergency medical services if needed
* Be willing to provide an address, email and/or telephone number for purposes of follow-up
* Agree to comply with the study procedures and visit schedule

Exclusion Criteria:

* confirmed or suspected ectopic pregnancy or undiagnosed adnexal mass
* chronic renal failure
* concurrent long-term corticosteroid therapy
* history of inherited porphyrias
* IUD in place (must be removed after mifepristone is administered).
"Endoscopy First" or "Laparoscopic Cholecystectomy First" for Patients With Intermediate Risk of Choledocholithiasis

The study compares two different methods to evaluate extrahepatic bile ducts for possible stones for patients with cholecystolithiasis and intermediate risk for choledocholithiasis when laparoscopic cholecystectomy is indicated.

Endosonoscopic evaluation of bile ducts and endoscopic retrograde cholangiography (ERCP) on demand are performed before laparoscopic cholecystectomy for one arm. Intraoperative cholangiography during laparoscopic cholecystectomy and postoperative ERCP on demand are administered in another arm.

Use of ERCP as a diagnostic tool should be minimized as it carries considerable risk (5 to 10%) of post-procedural complications. It is noticed that adverse events occur more often to patients with low risk of choledocholithiasis. Therefore the best possible patient selection for ERCP procedure is needed.

At the Centre of Abdominal Surgery of Vilnius University Hospital Santaros klinikos an original prognostic index (Vilnius University Hospital index (VUHI)) is used for evaluation of risk of choledocholithiasis. It is calculated by formula VUHI = A/30 + 0.4×B, where A - total bilirubin concentration (µmol/l), B - common bile duct (CBD) diameter measured by ultrasound exam. A retrospective study evaluated its accuracy and determined threshold values for low, intermediate and high risk groups. The intermediate risk group (risk for choledocholithiasis 25-75%) would benefit from additional examination before ERCP. Endoscopic ultrasound (EUS) and intraoperative cholangiography are less invasive procedures with high accuracy identifying common bile duct stones. Main hypothesis of the trial is that intraoperative cholangiography with ERCP on demand can shorten the duration and costs of treatment and avoid diagnostic ERCPs.

Eligibility Criteria:
Inclusion Criteria:

* patients with cholecystolithiasis when laparoscopic cholecystectomy is indicated
* intermediate risk for choledocholithiasis (VUHI 2,6 - 6,9 and one of the predictors: dilated common bile duct, elevated total bilirubin or suspected stone in CBD on ultrasound)

Exclusion Criteria:

* pregnancy;
* acute cholangitis;
* biliary pancreatitis;
* acute cholecystitis, degree II-III by Tokyo guidelines 2013;
* anastomosis in upper gastrointestinal tract;
* other known cholestatic hepatopancreatobiliary disease;
* known or suspected hepatitis of another origin (viral, toxic, etc.);
* contraindications for general anaesthesia or surgery;
* IV-VI class of American Society of Anesthesiologists physical status classification;
* morbid obesity (body mass index \> 40);
* patient's refusal to participate in the study.
Eribulin as a Second-line Treatment in Triple-negative Advanced Breast Cancer

Multicenter, retrospective and prospective, cohort, observational study evaluating the clinical efficacy and tolerability of Eribulin as second-line treatment in accordance with the indications authorized by AIFA in patients with triple negative advanced breast cancer in a real world setting.

The study, multicenter, retrospective and prospective, cohort, observational, describes the modalities of treatment with Eribulin as a second line therapy for triple negative breast cancer and "clinical outcomes" in a population real-world, evaluating any differences with the results obtained in clinical trials. Therapeutic sequences will also be evaluated with the aim of providing a photograph of the choices made in clinical practice in this historical moment characterized by rapid evolution of new molecules available to clinicians.

A total of at least 200 patients with triple negative advanced breast cancer treated with Eribulin as second line will be enrolled in the retrospective or prospective cohort. Enrollment period will last 2 years.

Pseudoanonymized data will be collected in a electroctronic database (RedCap Cloud); here the list of the main variables collected:

* Patient Registration
* Demography
* Pregnancy test
* Past history and habits of the patient
* Previous antineoplastic therapy
* Anamnesis close to entering the study
* Vital signs and ECOG performance status (WHO)
* Blood chemistry tests
* RECIST 1.1 Baseline and re-evaluations
* 12 Lead ECG evaluation
* Eribulin cycles
* Adverse events
* Previous and concomitant medications
* End of study

The retrospective analysis of the choices made in clinical practice and the benefits obtained from the second therapeutic lines could provide important data to favor the definition of prospective randomized studies, and guide the clinician towards a better therapeutic path.

Eligibility Criteria:
Inclusion Criteria:

* Performance status according to ECOG equal to 0-2
* Locally advanced or triple negative metastatic breast cancer (HR- and HER2-) confirmed histologically
* Progressing after first-line chemotherapy for advanced disease
* Previous anthracyclines and taxanes therapy (in an adjuvant, neoadjuvant or metastatic), unless the patient is ineligible to receive such treatments
* Treatment with Eribulin mesylate since 2017, in accordance with AIFA indications
* Adequate haematological, renal and hepatic function, as per clinical practice
* Written informed consent

Exclusion Criteria:

* Breast cancer HER2 + or HR +
* Treatment with Eribulin in the context of clinical studies
* Patients unsuitable for treatment with Eribulin
* Diagnosis of other malignancies in the two years prior to enrollment, with one exception of adequately treated localized basal cell or squamous cell carcinomas of the skin o cervical carcinomas undergoing curative treatment
Measuring Outcomes of Maternal COVID-19-related Prenatal Exposure

The rapidly spreading coronavirus Covid-19 pandemic is a major concern worldwide and Italy is at the forefront of this emergency. At the present moment, the effects on the offspring of Covid-19 disease in pregnant women and/or the indirect effect of COVID-19-related prenatal maternal stress are poorly understood. Epigenetic mechanisms - and especially DNA methylation - are involved in the embedding of prenatal exposures to large-scale disasters into the epigenome and phenotypic outcomes of the offspring. Specific target genes include SLC6A4, NR3C1, OXTR, BDNF, and FKBP5. The central hypothesis of this project is that the exposure to the COVID-19-related prenatal maternal stress may affect infants' outcomes from birth to 12 months partially through increased methylation of target genes involved in stress regulation. The main goal is to investigate the association between Covid-19 disease in pregnancy or COVID-19-related PMS in women pregnant during the first 9 months of the year 2020 and the emotional, social, and cognitive developmental outcomes in their infants from birth to 12 months. Additionally, the role of target genes methylation in mediating this association will be investigated. Mothers and their infants will be enrolled from the delivery units of eleven neonatal facilities located in Northern Italy's COVID-19 contagion hotspot. One group will be enrolled from April to October 2020 (COVID-Exposure Pregnancy, CEP), a second group will be enrolled from April to October 2021 (Non-Exposure Pregnancy, NEP). Moreover, the CEP group will include mothers positive or exposed to COVID-19-related MPS during different trimesters of pregnancy, allowing within-group comparisons on developmentally sensitive time windows. Within 48 hours from baby delivery, saliva samples will be obtained from CEP and NEP newborns, immediately stored at -20°C and analyzed for epigenetic analyses using Next-Generation Sequencing. At 3 and 6 months, mothers will be asked to fill-in validated and reliable questionnaires on the emotional and social-behavioral development of their infants. At 12 months, infants' stress regulation and maternal sensitivity will be evaluated through video recording of mother-infant interactions. This study will provide unprecedented relevant insights on the biochemical mechanisms underlying the impact of Covid-19 disease and the related PMS on human offspring's developmental outcomes.


Eligibility Criteria:
Inclusion Criteria:

* Italian nationality or full mastery of Italian language to access on-line questionnaires without support
* No prenatal pathologies or perinatal risks
* Maternal age higher than 18 years
* Newborns' gestational age equal or higher than 37 weeks

Exclusion Criteria:

* Single-parent mothers
* Medically assisted conception
* Infants' genetic or metabolic comorbidities
* Perinatal injuries or sensorial deficits
Efficacy and Safety of Tongluo-Kaibi Tablet in Patients With Fibromyalgia Syndrome

This study is a multi-center, randomized, double-blinded, controlled trial with two parallel arms. The aim of the study is to evaluate efficacy and safety of Tongluo-Kaibi tablets in patients with Fibromyalgia Syndrome.


Eligibility Criteria:
Inclusion Criteria:

* Those who meet the classification criteria for Fibromyalgia formulated by the American Rheumatology Association in 2016.
* Pain VAS score ≥ 4 points.
* The variety and dosage of the medication used to treat the disease should be stable for at least 2 weeks.

Exclusion Criteria:

* Severe cardiovascular and cerebrovascular diseases.
* Malignant tumors, hematological diseases, inflammatory arthritis, or other serious or progressive systemic diseases.
* ALT and AST are more than 2 times the upper limit of normal.
* Cr is more than 1.2 times the upper limit of normal.
* Allergic constitution or allergic to experimental drugs Tongluo-Kaibi tablet, pregabalin capsules, excipients or similar ingredients.
* Pregnant, lactating or recently planned pregnancy.
Sarcopenia And Balance In Postmenopausal Osteoporosis

The study was performed on 53 postmenopausal osteoporosis patients who presented to our osteoporosis outpatient clinic. Following written informed consent, patients were evaluated for sarcopenia according to the sarcopenia algorithm proposed by the European Working Group on Sarcopenia in Older People (EWGSOP). Algorithm parameters were walking speed, muscle strength and muscle mass measurements. We used Timed Up and Go Test (TUG) for evaluation of walking speed, Jamar Hand Dynamometer for muscle strength assessment, and calf circumference for muscle mass measurement. Balances of the patients were evaluated with Berg Balance Scale (BBS) and functional skills were evaluated with the Timed Up and Go Test (TUG). Other clinical parameters (age, body mass index (BMI), 25OHvitD level, menopause age, fall within the last year, fracture history, history of fracture in mother) were questioned.

The study was performed on 53 postmenopausal osteoporosis patients who presented to our osteoporosis outpatient clinic. Following written informed consent, patients were evaluated for sarcopenia according to the sarcopenia algorithm proposed by the European Working Group on Sarcopenia in Older People (EWGSOP). Algorithm parameters were walking speed, muscle strength and muscle mass measurements. We used Timed Up and Go Test (TUG) for evaluation of walking speed, Jamar Hand Dynamometer for muscle strength assessment, and calf circumference for muscle mass measurement. Balances of the patients were evaluated with Berg Balance Scale (BBS) and functional skills were evaluated with the Timed Up and Go Test (TUG). Other clinical parameters (age, body mass index (BMI), 25OHvitD level, menopause age, fall within the last year, fracture history, history of fracture in mother) were questioned. SPSS 22.0 program will be used for statistical evaluation and correlation analysis will be done.

Eligibility Criteria:
Inclusion Criteria:

* Having postmenopausal osteoporosis
* No other disease that can cause balance disorder
* Volunteer participation in the study

Exclusion Criteria:

* To have a disease that will cause balance disorder,
* Having understanding problem to prevent communication with the patient,
* The lack of motor function to the balance device.