cyrilzakka/clinical-trials huggingface dataset:
Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

The purpose of the study is to prospectively assess longitudinal changes in biomarkers (MECP2, potential biomarkers of target engagement and disease activity) in cerebrospinal fluid (CSF) and blood; characterize longitudinal changes in performance on clinical scales (clinician-reported measures of neurodevelopment and functioning) and caregiver-reported outcome assessments (communication, gastrointestinal, social-emotional-adaptive behavioral measures); evaluate longitudinal changes in caregiver-reported health-related quality-of-life measures; and assess the frequency, type, and severity of seizures over time.

This is a multi-center, non-randomized, non-interventional prospective and retrospective study in up to 40 participants with MECP2 duplication syndrome (MDS) who can undergo general anesthesia or conscious sedation to collect fluid biomarkers (CSF and blood), undergo electrophysiological assessments (electroencephalogram \[EEG\], evoked potentials \[EP\], pupillometry), clinical assessments and caregiver reported outcomes measures, to be used in support of the development of therapies for MDS. The study duration for each participant will be approximately 110 weeks.

Eligibility Criteria:
Key Inclusion Criteria

* Participant has a diagnosis of MDS with genetic confirmation of MECP2 duplication (or triplication)
* Participant has a parent or caregiver (CG) ≥ 18 years old capable of providing informed consent (signed and dated), and able to attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol and be able to comply with all study requirements and activities
* Male ≥ 1 month and ≤ 65 years of age
* No contraindications for lumbar puncture (LP)'s, blood draws, sedation (if necessary) or other study activities
* Medically stable to complete the study and will tolerate sedation or general anesthesia and other study activities

Key Exclusion Criteria

* Clinically significant abnormalities in medical history (e.g., clinically significant renal, hepatic, or cardiac abnormalities; major surgery within 3 months of screening) or upon physical examination that could potentially impact the NH of MDS
* Unwillingness or inability to comply with study procedures, including follow up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator
* Treatment with an investigational drug, gene therapy, stem cell therapy, biological agent, or device within 30 days of screening, or 5 half-lives of investigational agent, whichever is longer (participants cannot be concurrently enrolled in NH00006 and ION440-CS1).
Can Blood Panels Provide Early Warning of Bleeding in Patients on ECMO?

This is a prospective, observational study of 60 adult patients anticoagulated with bivalirudin for extracorporeal membrane oxygenation (ECMO). The study aims to identify the most precise early warning blood test or panel of blood tests to predict bleeding in patients on bivalirudin/ECMO. The project will involve comparing currently used blood tests with an extended panel of coagulation and metabolic blood tests with the aim of early warning of impending bleeding to allow intervention in the form of adjusted bivalirudin dose, modification of other risk factors, or transfusion with hemostatic products targeted to documented coagulation test abnormalities.


Eligibility Criteria:
Inclusion Criteria:

* adults 18 - 70 years who are anticoagulation (AC) with bivalirudin and placed on ECMO for cardiac and/or respiratory failure. Surgical and non-surgical patients will be included.

Exclusion Criteria:

* known pre-existing bleeding disorder (due to a factor deficiency that requires factor replacement, or platelet disorder), or a creatinine \> 2.0.
Effect of Traditional Chinese Medicine on GI Function Recovery and Nutrition Support in SAP

With multicenter randomized control clinical evaluation investigations, the effect of traditional Chinese medicine on gastrointestinal function recovery and nutrition support in early stage of severe acute pancreatitis (SAP) was evaluated, and the therapeutic effects, safety and indications assured. Drawing in latest research fruits at home and abroad, a clinical practical guideline would be formed, to be popularized all over China.


Eligibility Criteria:
Inclusion Criteria:

* Adult patients 18 yr-70 yr
* Complying with the diagnostic standard of acute pancretitis (clinical symptoms and signs, elevated blood and urine amylase, and imaging criteria)
* Conforming to 3 or more Ranson's criteria and APACHE-II score larger than 8, Balhazar CT score larger than 5

Exclusion Criteria:

* Pregnancy
* Nursing mother
* Critical cases of advanced tumor
* On the verge of death (estimated to be moral in 12h)
* Severe visceral function disturbance
* Patients undergoing other therapeutic research that might interfere with the present study
The Effect of Intravenous Mannitol Plus Saline on the Prevention of Cisplatin-induced Nephrotoxicity: A Randomized, Double-blind, Placebo Controlled Trial

This study include cancer patients who had received chemotherapy that include cisplatin. Patients were randomly assigned to either a mannitol 20 g intravenous single dose after cisplatin or a placebo (saline). The primary outcome was to compare acute kidney injury (AKI), which was defined as increase creatinine 0.3 mg/dl in 48 hours by KDIGO criteria using serum creatinine and 24 hour urine creatinine to calculated.


Eligibility Criteria:
Inclusion Criteria:

1. Patients who was at least 18 years old.
2. Patients who had been diagnosed with cancer proven by tissue biopsy.
3. Patients who were scheduled to receive cisplatin or combination of cisplatin and other chemotherapy, which the dose of cisplatin was not exceeded 100 mg/m2.
4. Patients were required to have a normal renal function (GFR \> 60 mL/min/1.73m2).
5. Patients with an Eastern Cooperative Oncology Group (ECOG) score ≤ 2.
6. Patients with normal serum sodium and serum potassium level.

Exclusion Criteria:

1. Patients with any acute kidney injury event before randomized into trial not more than 6 months.
2. Patients with chronic kidney disease or hydronephrosis.
3. Patients with history of nephrectomy.
4. Patients who had previously received immunosuppressants for any immune deficiency disease.
5. Patients with who had received chemotherapy which induce nephrotoxicity.
6. Patients with had received drug which is nephrotoxic (amphotericin B, aminoglycoside or non-steroidal anti-inflammatory drug).
7. Patients who had cirrhosis with child pugh score more than 7.
8. Patients with or had a known allergy to cisplatin or mannitol.
9. Patients with chronic heart failure who cannot received fluid more than 1 liter.
10. Patients who were not comfortable to follow up at clinic for long term outcome.
Comparison of Insulin Detemir With NPH Insulin in Type 1 Diabetes

This trial is conducted in Africa, Asia, Europe, Oceania, and South America. The aim of this research study is to compare the efficacy (reduction in HbA1c and in blood glucose levels) of insulin detemir compared to NPH insulin administered as basal insulin for the treatment of type 1 diabetes and to verify the safety of use (number and severity of episodes of hypoglycemia, body weight, and insulin antibodies and side effects).


Eligibility Criteria:
Inclusion Criteria:

* Type 1 diabetes
* Currently on basal-bolus regimen
* BMI = 35.0 kg/m2
* HbA1c =11.0%

Exclusion Criteria:

* Proliferative retinopathy or maculopathy
* Recurrent major hypoglycaemia
* Cardial problems
* Uncontrolled hypertension
* Impaired hepatic or renal function
COVID-19 Personal Protective Equipment for Vaccinated Health Workers and Convalescents

The aim of the trial is to assess the safety of using simplified personal protective equipment (PPE) for vaccinated or COVID-19 convalescent healthcare workers working in COVID-19 ICU or COVID-19 wards.

The trial is designed as a prospective randomized observational trial with volunteer medical staff working in COVID-19 departments aiming to show non-inferiority of simplified PPE vs standard COVID-19 PPE.

Objectives To assess safety of simplified personal protective equipment (PPE) for vaccinated health workers and COVID-19 convalescents - pilot trial.

Methods Prospective randomized observational trial. 60 participants randomized 1:1 to either standard PPE (COVID-19 ward: FFP2 or higher respirator, goggles of face shield, gown, gloves; COVID-19 ICU: FFP3 respirator and tight goggles or full face mask or powered air-purifying respirators, gown and gloves) or simplified PPE (surgical mask, goggles or face shield, gown). 30 days follow up period.

Participants Volunteer medical staff from COVID-19 regular and ICU departments at University Medical Center Ljubljana, Slovenia, with either proven COVID-19 infection within 6 months or at least 7 days after second dose anti COIVD-19 vaccine.

Hypotheses Simplified PPE is non-inferior to standard PPE in vaccinated health workers or convalescents.

Discussion Positive study results may decrease PPE spending cost and positively affect working conditions at COVID-19 departments.

Eligibility Criteria:
Inclusion Criteria:

* age \< 50 years
* prior COVID-19 infection; time from infection \< 6 months.
* prior completed anti COVID-19 immunization; \> 1 week post second dose of immunization.
* signed written consent
* negative nasopharyngeal COVID-19 RT-PCR test

Exclusion Criteria:

* age \> 50 years
* time post COVID-19 infection \> 6 months
* time post COVID-19 immunization \< 1 week.
* immunocompromised patients
* pregnancy
* CKD 3 or more
* COPD or other lung disease
* heart failure of any cause
* ischemic heart disease
* obesity (BMI \> 30)
* DM on therapy
A Phase 2b Study of Modified Vaccinia Virus to Treat Patients Advanced Liver Cancer Who Failed Sorafenib

This study is to determine whether JX-594 (Pexa-Vec) plus best supportive care is more effective in improving survival than best supportive care in patients with advanced Hepatocellular Carcinoma (HCC) who have failed sorafenib.


Eligibility Criteria:
KEY Inclusion Criteria:

* Diagnosis of primary HCC by tissue biopsy (histological/cytological diagnosis), or clinical diagnosis
* Previously treated with sorafenib for ≥ 14 days and has discontinued sorafenib treatment at least 14 days prior to randomization due to either intolerance or radiographic progression NOTE: Sorafenib is NOT required to be the most recent treatment received for HCC
* ECOG performance status 0, 1 or 2
* Child-Pugh Class A; or Child-Pugh Class B7 without clinically significant ascites
* Hematocrit ≥30% or Hemoglobin ≥10 g/dL
* Tumor status: Measurable viable tumor in the liver and injectable under imaging-guidance; At least one tumor in the liver that has not received prior local-regional treatment OR that has exhibited \>25% growth in viable tumor size since prior local-regional treatment.

KEY Exclusion Criteria:

* Received sorafenib within 14 days prior to randomization
* Received systemic anti-cancer therapy other than sorafenib within 28 days of randomization
* Prior treatment with JX-594
* Platelet count \< 50,000 PLT/ mm3
* Total white blood cell count \< 2,000 cells/mm3
* Prior or planned organ transplant
* Known significant immunodeficiency due to underlying illness (e.g. HIV/AIDS) and/or medication
* Severe or unstable cardiac disease
* Viable CNS malignancy associated with clinical symptoms
* Pregnant or nursing an infant
* History of inflammatory skin condition (e.g., eczema requiring previous treatment, atopic dermatitis)
Correlation Between Thoracic Kyphosis Posture and Static Balance

The effects of balanced posture and postural changes have been evaluated in specific populations with different factors that can affect body postures and balance, such as age, musculoskeletal system, and other metabolic diseases. Studies on postural kyphosis and balance involvement are very few in the literature especially in the health population. Of the few studies that have evaluated kyphosis and balance, most are limited by small sample size and the exclusion of men. Many have focused primarily on the geriatric population. The determination that the effect of thoracic kyphosis posture on trunk biomechanical alignment may be related to balance may contribute to new treatment recommendations in this area. The aim of this study is to investigate whether the thoracic kyphosis posture has an effect on static balance.

Background:

Kyphosis is roughly a slight forward curvature of the spine. A slight kyphosis or posterior curvature is normal throughout the human body and is present in every individual. Hyperkyphotic is a kyphotic angle greater than 40° commonly measured on a lateral X-ray measured by the Cobb method between C7 and T12. Postural stability or balance is the ability to keep the center of mass within the boundaries of the support base. Moving the center of mass beyond the boundaries of the support base may cause postural instability and loss of balance. There are studies showing that kyphotic posture affects the center of gravity and affects fall in the elderly, but there are limited studies on the effect of balance in young individuals.

The Aim of This Study Is:

To study the correlation between the balance and severity of thoracic kyphosis will be investigated

Eligibility Criteria:
Inclusion Criteria:

* Adults aged 18 and over willing to attend the study.
* For those who join the thoracic kyphosis group, individuals with a kyphosis angle ≥ 40 degrees.
* Individuals with a kyphosis angle \< 40 degrees for the control group.

Exclusion Criteria:

* Spine trauma, surgery, bone pathology, arthritis etc. have a history of illness Kyphotic deformity types are rounded back, Scheuermann's disease, hunched back, flat back and Dowager hump.
* Any spinal deformity, bone abnormality, postural deformity and disc herniation with / without peripheral symptoms.
* Body mass index (BMI), which is an indicator of obesity, is more than \> 30.
* Complaining of balance problems, coordination problems, other neurological or vestibular diseases that affect body balance and posture.
* Having any orthopedic or neurological disease that affects the body joints or the integrity of the musculoskeletal system.
* Use of any medication that can cause dizziness or drowsiness in the last months.
The Pelvic Floor Function and Sexual Life of the Women After Different Type of Hysterectomy

In this prospective, comparative non-randomized multiple teaching hospitals study, the investigators try to determine the relationship of total hysterectomy and Taiwanese female pelvic floor and sexual function from the view of epidemiology and clinical survey. This is an important issue that related to female autonomy, health care resources and even national health policy. The study results will help to understand whether there is unnecessary part in the current hysterectomy procedures and its potential health hazard.

Women with benign gynecology disease and indicated for laparoscopic hysterectomy (laparoscopic subtotal hysterectomy: LSH, laparoscopic cervical ligament sparing hysterectomy: CLSH, laparoscopic assisted vaginal hysterectomy: LAVH) are invited and explained the details of the protocol by the surgeon. The types of hysterectomy are determined by the patient and the surgeon together after discussion. All participant have to sign the informed consent. The participant will be followed up for two years after surgery. Preoperative evaluation on uterine condition, pelvic floor, questionaires were recorded. The perioperative parameters including surgical time, blood loss, and postoperative parameters such as pain score, hospitalization etc were recorded. The postoperative evaluation on pelvic floor condition, questionaires etc were also recorded at different time points.

Eligibility Criteria:
Inclusion Criteria:

1. Patient who is indicated for hysterectomy due to benign gynecology disease. The participant's husband is also welcomed to join the survey of the sexual life questionnaire but this is not a necessary inclusion criteria.

Exclusion Criteria:

1. Suspect of or diagnosed as cancer patient
2. Age \< 30 or \> 50 years old
3. Menopausal woman or woman who is nulliparous
4. Patient with advanced pelvic floor prolapse (stage 2, 3, 4) including uterine prolapse, cystocele, enterocele, rectocele.
5. Patient with prominent urinary incontinence that affect social activity or suggested to have surgery by gynecologist or urologist.
6. Patient with severe medical diseases such as liver cirrhosis, heart failure, poor control diabetes mellitus etc.
7. Patient with severe pelvic inflammatory disease/tubo-ovarian abcess or pelvic endometriosis or history of severe pelvic adhesions.
8. Psychiatric patients
9. Patient with chronic lung disease such as bronchial asthma, bronchiectasis, chronic obstructive pulmonary disease and interstitial lung disease
WebQuit Online Smoking Cessation Study

Would you like to quit smoking?

Seattle's Fred Hutchinson Cancer Research Center is launching a new study that may be able to help. Participants who enroll in this study will be randomly assigned by computer (like flipping a coin) to one of two online programs. The goal of this study is to learn which of these programs is the most effective at helping people stop smoking. Findings from this study will be used to help build more effective online smoking cessation programs in the future.

Participants in both of the smoking cessation programs used in this study will receive:

* Interactive tools for dealing more effectively with urges to smoke
* Step-by-step guides for quitting smoking
* Personalized plans for quitting and remaining smoke-free
* Electronic links for reaching one-on-one expert help for quitting

Participants will be asked to complete online questionnaires, including a brief three-month follow-up survey.

There is no cost for participating in this study.

To enroll in this study or for more information, please visit www.webquit.com.

This study is being conducted by Seattle's Fred Hutchinson Cancer Research Center (www.fhcrc.org), a world leader in advancing the prevention, diagnosis and treatment of cancer. The principal investigator for the WebQuit smoking cessation study is Dr. Jonathan Bricker, a faculty member at both the Hutchinson Center and at the University of Washington in Seattle.


Eligibility Criteria:
Inclusion Criteria:

* 18 or older
* a current resident of the US
* someone who has smoked daily for at least the past 12 months
* someone who wants to quit smoking in the next 30 days
* willing and able to read in English
* able to connect to the Internet for at least one hour per week for three consecutive months
* able to use a website
* not participating in other smoking cessation interventions
* willing and able to complete a Baseline Survey
* willing to be randomized to either online program

Exclusion Criteria:

* inability to meet inclusion criteria
Study of Safety of RVL-1201 in Treatment of Blepharoptosis

Phase 3 study to evaluate the extended safety of RVL-1201 compared to placebo for treatment of blepharoptosis. Eligible subjects will be randomized to one of 2 treatment arms.


Eligibility Criteria:
Inclusion Criteria:

1. Male or Female 9 years of age or older
2. Females must not be pregnant or planning to get pregnant and must use acceptable form of contraception
3. Must be able to self-administer study medication
4. Must be able to understand and sign an Informed Consent Form (ICF). For minor subjects, the subject's parent or legal guardian must provide permission by signing an ICF on behalf of the subject and the subject should provide assent.

Exclusion Criteria:

1. Congenital ptosis
2. Horner syndrome
3. Myasthenia gravis
4. Mechanical ptosis
5. Previous ptosis surgery
6. Resting heart rate outside the normal range
7. Hypertension with resting diastolic blood pressure
8. Pregnancy or lactation
Music Therapy: Sickle Cell and Pain Crisis

This study will assess whether participants who receive music therapy during inpatient care report a decrease in pain and whether music therapy influences factors such as family satisfaction.

The purpose of this study is to determine if music therapy services can serve as a viable, necessary, and beneficial aspect of inpatient pain management, distraction, and coping for pediatric patients in the acute care hospital setting. The investigator will note the effect of music therapy services in conjunction with pain scores reported by the participant and an increase in self and family reported coping skills during hospitalization.

Participants will be referred by providers from pain management service, oncology/hematology service, or the gastrointestinal (GI) service for functional and abdominal pain. Participants who meet the criteria for inclusion will be selected. The study will be explained to parents and a consent form will be left for the family to review and sign at their convenience. Participants will provide a pain scale level and vitals before completing the music therapy intervention. Once the music therapy intervention is completed, the pain scale and vitals will be repeated.

Eligibility Criteria:
Inclusion Criteria:

* 5-17 years of age
* Admitted for sickle cell crisis, functional abdominal pain, or whom the pediatric pain management team is following.

Exclusion Criteria:

* Non-English speaking
* Under 5 years old and over 17 years old
* Patients who are unable to hear or have a hearing disability
* Developmentally unable to self-report pain levels
Physiology and Therapeutic Management of Neonatal Abstinence Syndrome

The overall purpose of this project is to to quantify the physiology of neonatal drug withdrawal and develop non-pharmacological techniques to help improve the therapeutic management of Neonatal Abstinence Syndrome (NAS).

Novel approaches to diagnosing and treating Neonatal Abstinence Syndrome (NAS) are needed for reducing prolonged pharmacological management, minimizing hospitalization and improving developmental outcomes in drug exposed newborns. This study seeks to examine the physiology and symptoms of drug withdrawal (e.g., irritability marked by movement activity; cardio-respiratory instabilities) in infants exposed to drugs in utero and test whether sensory stimuli (tactile, auditory) reduce dysregulated systems in the withdrawing infant.

Candidates at-risk for NAS due to fetal drug exposure will be identified to investigators by the infant's primary medical caregiver. Investigators will use a modified-consecutive sampling technique, restricted by equipment and personnel availability, for enrolling infants. Participants will be studied throughout their hospitalization. Effects of stimulation will be examined at different stages of withdrawal. Efficacy of stimulation will be examined as a potential complementary treatment of NAS.

Eligibility Criteria:
Inclusion Criteria:

Eligible subjects are infants currently inpatient in the NICU or Newborn Nursery at University of Massachusetts Memorial Hospital and:

* Full-term infants (\>37 wks gestational age) and late preterm infants (34-37 wks gestational age)
* Newborns at risk for NAS due to fetal-drug exposure
* At-risk infants will be infants who present with confirmed meconium and/or urine toxicology report (documented in medical chart review) for opioids (e.g., methadone, buprenorphine/subutex, oxycodone, heroin); may also have prenatal exposure to benzodiazepines, barbiturates, amphetamines, cannabinoids, alcohol, nicotine and/or caffeine.

Exclusion Criteria:

Eligible infants meeting the inclusion criteria above will be excluded from participation in the study if he/she:

* Born less than \<34 weeks.
* Has a congenital abnormality
* Has a fetal anomaly
* Has hydrocephalus or intraventricular hemorrhage \>grade 2
* Has a seizure disorder not related to drug withdrawal
* Has a clinically significant shunt
* Requires mechanical respiratory support