cyrilzakka/clinical-trials huggingface dataset:
A Clinical Study of PGN-EDODM1 in People with Myotonic Dystrophy Type 1

The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.


Eligibility Criteria:
Inclusion Criteria:

* Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
* Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
* Presence of myotonia
* Body Mass Index (BMI) of \< 32.0 kg/m\^2

Exclusion Criteria:

* Congenital DM1
* Known history or presence of any clinically significant conditions that may interfere with study safety assessments
* Abnormal laboratory tests at screening considered clinically significant by the Investigator
* Medications specific for the treatment of myotonia within 2 weeks prior to screening
* Percent predicted forced vital capacity (FVC) \<40%
* Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening

Note: Other inclusion and exclusion criteria may apply.
ST2, a Novel Biomarker for Cardiac Remodeling in Adults With Congenital Heart Disease

The remarkable improvement in survival of children with congenital disease has led to a continuously growing number of adults with congenital heart disease in the developed world. Many of these patients had had cardiac surgery at early age, the may live for many years with pressure overload, volume overload, systolic or diastolic dysfunction, cyanosis or any combination of the above. These past and ongoing cardiac insults often result in significant cardiac remodeling. A biomarker for fibrosis and remodeling may have enormous clinical and prognostic value for these patients.

Serum biomarkers are now integrated in many fields in medicine. In cardiology, a number of biomarkers are used. In the last decade, our group has focused on the natriuretic peptides as markers for heart disease in infants and children. This resulted in many studies and more than 25 publications in the medical literature. We believe that the newly discovered cardiac marker, ST2, will emerge as an important addition to cardiac evaluation in the coming years.The aim of this study is to measure ST2 levels in patients with congenital right heart disease and correlate ST2 levels to clinical status, imaging and prognosis.


Eligibility Criteria:
Inclusion Criteria:

* Adults (age\>18 y)
* congenital heart disease

Exclusion Criteria:

* Additional lung diseases, renal disease or inflammatory disease.
* Patients who underwent therapeutic intervention between the MRI and the ST2 - measurements.
* Pregnant women
Comparison Between Lactulose and Lactulose-Paraffin in Cirrhotic Patients

Hepatic encephalopathy (HE) is an important complication of liver cirrhosis. Lactulose is a first line treatment for HE, but the adherence to this treatment is relatively low, due to side effects such as diarrhea, distention, etc.

Lactulose is a first line treatment for HE, but the adherence to this treatment is relatively low due to side effects. Lactulose-paraffin recently available in the market can be used for the treatment of HE, and possibly have fewer side effects.

The aim of this study was to compare the adherence of the treatment of lactulose and lactulose-paraffin.

Eligibility Criteria:
Inclusion Criteria:

* Liver cirrhosis
* History of an event of hepatic encephalopathy.
* Ambulatory patients

Exclusion Criteria:

* Alcoholism with active ingest of alcohol in the last 6 months
* Labour turn-overs
* Pregnancy
* Personal history of surgery in the last 4 weeks
* Spontaneous bacterial Peritonitis
* Use of neuropsychiatric drugs
* Neuropsychiatric disorders (Schizophrenia, bipolar disorder, major depression, dementia and Attention-deficit hyperactivity disorder)
* Thyroid disorders without replacement therapy
* Renal failure
* Hepatic or renal transplant
* Personal history of hepatocellular carcinoma
* Placement of transjugular intrahepatic portosystemic shunt
* Use of a probiotic in the last 6 month
Neurobiology of Posttraumatic Stress Disorder Dysfunction and Recovery Following Cognitive Processing Therapy

Posttraumatic stress disorder (PTSD) is a significant personal and societal burden. The purpose of this study is to examine genetic, epigenetic, behavioral, and brain activity changes related to PTSD dysfunction and recovery before and after treatment with Cognitive Processing Therapy.

Using functional magnetic resonance imaging approaches, the present research aims to examine the relationship between traumatic experience and the neural mechanisms of various PTSD symptoms, and determine how this relationship changes during PTSD extinction. Also, this proposal aims to better understand how the genetic/epigenetic profile of several genes predicts, and perhaps changes, in response to recovery from PTSD along with its neural correlates. This understanding will help identify individuals who will respond most optimally to a specific empirically based PTSD treatment, Cognitive Processing Therapy, while further connecting genetic biomarkers of risk with neural intermediate phenotypes underlying PTSD symptomatology.This study is observational as the investigator does not assign specific interventions to the participants of the study.

Eligibility Criteria:
Inclusion Criteria:

* PTSD diagnosis, as measured by the Clinician Administered PTSD Scale for DSM-5
* history of chronic childhood trauma due to relational abuse
* Ability to understand and sign informed consent
* Enrollment in CPT program at McLean Hospital
* Both genders, all ethnic backgrounds, age between 18 and 55
* Fluent English speakers
* Normal or Corrected Vision

Exclusion Criteria:

* History or current schizophrenia spectrum disorder or other psychotic disorders
* History or current bipolar or other related disorders
* Alcohol and/or substance use disorder within the past month
* Metal implants
* Medical or neurological illness (e.g., seizures, head injury in which they experienced a loss of consciousness)
* Positive pregnancy test
The Impact of Intraoperative Stretching Microbreaks on Otolaryngologists

There have been numerous survey-based and observational studies reporting a high percentage of otolaryngologists suffering from work-related musculoskeletal symptoms, in which more than half attributed to poor ergonomics in the operating room. Interventions have been implemented in the past to alleviate ergonomic hazards in general surgery such as intra-operative stretch breaks. However, there has not yet been an intervention done to alleviate ergonomic hazards in otolaryngologists. Our study aims to explore the efficacy of implementing intraoperative stretch breaks and assess their effect on otolaryngologists' practices and musculoskeletal pain.

The study subjects will be recruited voluntarily from George Washington University Hospital otolaryngology attending physicians, residents, and fellows, as well as Children's otolaryngology faculties. Pre-survey will be sent prior to the intervention to obtain background information and baseline ergonomic assessment conducted by physical therapy students. Interventions will include intra-operative stretch breaks that will be implemented by surgeons at their convenient time during the surgery. Post-survey will be sent out to obtain the subjects' experiences after each intervention.

Each survey will be sent via email using a secured third-party vendor, RedCap. Results will be retrieved only by the research assistants listed on the institutional review board (IRB). The survey will not ask for any identifying information and will not publish identifying information. The raw data will be analyzed by the PI and research assistant for publication. The study should take less than a year for data collection and analysis.

Eligibility Criteria:
Inclusion Criteria:

* Otolaryngologists
* Currently practicing

Exclusion Criteria:

* Non-otolarynglogists
* Currently not practicing
Delay to Diagnosis in Digestive Cancerology by the General Practitioner Related to Covid-19 Pandemic Confinement

Objective: the pre-hospital management of cancers is little known in General Medicine. The first lockdown related to the COVID-19 pandemic led to the closure of health facilities. Investigators were interested in the diagnosis and care pathway of digestive cancers in post-confinement in General Medicine in Nouvelle-Aquitaine.

Method: patient records discussed in Digestive Oncology CPR of Bordeaux University Hospital for the first time from June 1 to August 31, 2020 and over the same period in 2019 were included. A questionnaire on the dates of the first symptoms, first consultation with the general practitioner, imaging examination and access to the specialist was sent to the general practitioner. The primary outcome was time from onset to date of first MDT.

Eligibility Criteria:
Inclusion Criteria:

* residing in New Aquitaine and
* whose general practitioner practiced in New Aquitaine
* malignant tumors of the esophagus, stomach, small intestine, colon, recto-sigmoid junction, rectum, anus and anal canal, liver and intrahepatic bile ducts, extrahepatic bile ducts, gallbladder and pancreas.
* to have had at least one consultation or hospitalization in the digestive pole of the Bordeaux University Hospital within 6 months after the MDT.

Exclusion Criteria:

* Patients under 18 years of age,
* who had already had a MDT for this cancer,
* whose date of first MDT was outside the inclusion dates,
* whose new cancer was a digestive metastasis of a primary of another location,
* who did not have French health insurance
Virtual Reality-based Training in Cerebellar Ataxia

The purpose of this study was to investigate the effects of virtual reality-base training for cerebellar ataxia.


Eligibility Criteria:
Inclusion Criteria:

* diagnosis of SCA 1, 2, 3 and 6 by a neurologist
* ability to walk independently with or without any walking aids
* age ≥ 20
* a score of ≥ 24 on the mini-mental state examination

Exclusion Criteria:

* histories of other neurological, cardiovascular, or orthopedic diseases affecting motor performance and balance
* uncontrolled chronic diseases
Non-invasive and Continuous Blood Pressure Monitoring

The goal of this observational study is to evaluate the accuracy and reliability of the VeriTrack non-invasive blood pressure monitor compared to radial arterial catheterization in adult surgical patients.

The main question it aims to answer is:

How well does VeriTrack measure continuous blood pressure compared to an arterial catheter?

Researchers will compare VeriTrack readings to arterial line measurements to determine if the VeriTrack provides comparable accuracy and clinical utility.

Participants will wear the VeriTrack device during surgery and have a radial arterial catheter placed as part of standard care.


Eligibility Criteria:
Inclusion Criteria:

* Patients whose blood pressure will be monitored using an arterial line during their procedure
* Patients who have a palpable dorsalis pedis pulse

Exclusion Criteria:

* Patients who have non-palpable dorsalis pedis pulses
* Patients whose limbs are inaccessible for placement of Vena Vitals Wearable
* Patients with known allergy to adhesives and/or silicone
Brava and Fat Transfer for Breast Reconstruction

Breast reconstruction is one of the most challenging aspects in plastic surgery. One of the most sophisticated methods of breast reconstruction is using of autologous fat grafting. Brava is a device that invented by Dr Roger Khouri, and is intended to improve the final results of fat grafting.

The purpose of the study is to examine the efficacy of Brava with fat grafting in woman undergoing mastectomy in the institution. Woman who will agree to participate in the study will be instructed how to wear the Brava, and after 200 hours of using the device they will be operated for fat transfer to the breast in order to reconstruct it.

Eligibility Criteria:
Inclusion Criteria:

* woman after mastectomy

Exclusion Criteria:

* smokers
* collagen disease
* low compliance
Cross-Education and Mirror Therapy for Upper Limb in Post-Stroke Patients

This randomized controlled trial will investigate the potential synergistic effects of combining cross-education training with mirror therapy on strength and motor function in the more affected upper limb of post-stroke patients.


Eligibility Criteria:
Inclusion Criteria:

* Post-stroke patients with upper limb motor impairments
* Aged 30 years to 60 years old
* At least 3 months post-stroke
* Able to participate in the intervention

Exclusion Criteria:

* Severe cognitive impairments
* Visual impairments
* Any contraindications to the intervention
Phase 1, Dose-Escalation Study of Oral CNF2024(BIIB021) in CLL

CLL dosing escalating study; daily dosing schedule; PK/PD safety

Phase 1, open-label, accelerated-titration study designed to evaluate the effects of increasing doses of CNF2024 on safety, pharmacokinetics, pharmacodynamic markers, and hematological response

Eligibility Criteria:
Inclusion Criteria:

* Patients with CLL who relapsed following or are intolerant to purine analog -based therapy
* Hemoglobin \>=9 gm/dL (may be post-transfusion)
* Total bilirubin \<2 X ULN, and ALT and AST \<2 x ULN
* Creatinine \<=2 X ULN
* Normal plasma cortisol and ACTH concentrations
* ECOG Performance Status \<=2
* Anticipated survival \>=3 months
* For men and women of child-producing potential, use of effective contraceptive methods during the study and for one month after treatment
* Ability to understand the requirements of the study, provide written informed consent and authorization of use and disclosure of protected health information, and agree to abide by the study restrictions and return for the required assessments

Exclusion Criteria:

* Pregnant or nursing women
* Treatment with chemotherapy, monoclonal antibody, or radiotherapy within 28 days before entering the study
* Participation in any investigational drug study within 28 days before CNF2024 administration
* Patients with secondary malignancy requiring active treatment (except hormonal therapy)
* Active symptomatic bacterial, fungal, or viral infection including active HIV or viral (A, B, or C) hepatitis
* Problems with swallowing or malabsorption
* Diarrhea (excess of 2-3 stools/day above normal frequency in the past month)
* Gastrointestinal diseases including gastritis, ulcerative colitis, Crohn's disease, or hemorrhagic coloproctitis
* Major surgery of the stomach or small intestine
* Adrenal dysfunction
* Patients with life- or function-threatening CLL complications (e.g., cord compression, hemolytic crisis, urinary tract obstruction)
* Any illness or condition that in the opinion of the investigator may affect safety of treatment or evaluation of any the study's endpoints
Expanded Access Neoantigen Vaccine in Solid Tumors

This is an expanded access use study. Safety data will be collected from participants.


Eligibility Criteria:
Inclusion Criteria:

* Histologically or cytologically confirmed diagnosis of a solid tumor
* Pathology review demonstrates tumor cellularity of \>30% in quantities sufficient to complete DNA and RNA library construction
* At least 18 years of age.
* Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
* Able to understand and willing to sign an IRB approved written informed consent document.

Exclusion Criteria:

* Known allergy, or history of serious adverse reaction to vaccines such as anaphylaxis, hives, or respiratory difficulty.
* Acute or chronic, clinically significant hematologic, pulmonary, cardiovascular, hepatic renal, and/or other functional abnormality that would jeopardize the health and safety of the participant as determined by the investigator based on medical history, physical examination, laboratory values, and/or diagnostic studies.
* History of syncopal or vasovagal episode as determined by medical record and history in the 12 month period prior to first vaccination administration.
* Individuals in whom a skinfold measurement of the cutaneous and subcutaneous tissue for eligible injection sites (left and right medial deltoid region) exceeds 40 mm.
* Individuals in whom the ability to observe possible local reactions at the eligible injection sites (deltoid region) is, in the opinion of the investigator, unacceptably obscured due to a physical condition or permanent body art.
* Therapeutic or traumatic metal implant in the skin or muscle of either deltoid region.
* Any chronic or active neurologic disorder, including seizures and epilepsy, excluding a single febrile seizure as a child.
* Current use of any electronic stimulation device, such as cardiac demand pacemakers, automatic implantable cardiac defibrillator, nerve stimulators, or deep brain stimulators.
* Pregnant and/or breastfeeding.
Deep Brain Stimulation Effects in Essential Tremor

Deep Brain Stimulation (DBS) is an effective therapy for patients with medically refractory essential tremor. However, DBS programming is not standardized and multiple clinic visits are frequently required to adequately control symptoms. The investigators aim to longitudinally record brain signals from patients using a novel neurostimulator that can record brain signals. The investigators will correlate brain signals to clinical severity scores to identify pathological rhythms in the absence of DBS, and we will study the effects of DBS on these signals in order to guide clinical programming.

The development of newer technologies has allowed clinicians and researchers to better understand pathophysiological underpinnings of different disorders managed with neuromodulation. The is now the ability to stream brain signals from a newly FDA approved device, the Medtronic Percept. The investigators will study the longitudinal effects of DBS on the brain signals that are found to correlate with tremor severity as measured with wireless wearable sensors.

Eligibility Criteria:
Inclusion Criteria

* Patient gives an informed consent.
* Patient is over 21 years of age.
* Having a diagnosis of a essential tremor confirmed by a trained movement disorders neurologist;
* Having failed or not tolerated conventional medical management, at the discretion of the neurologist managing the patient;

Exclusion Criteria

* Having alternative diagnoses to essential tremor;
* Having comorbid neurodegenerative disorders that may affect mobility or cognition (e.g. comorbid Parkinson's disease or dystonia);
* Having sequelae of prior brain insult (e.g. prior stroke or brain tumor);
* History of prior resective brain surgery (e.g. tumor resection);
* Not being a DBS candidate;
* Receiving unilateral implants
* Having a higher surgical risk that precludes patient from having standard intraoperative mapping.
Duloxetine Versus Placebo in Chronic Low Back Pain

The primary purpose of your participation in this study is to help answer the following research question, and not to provide you treatment for your condition--Whether duloxetine once daily can help patients with Chronic Low Back Pain.


Eligibility Criteria:
Inclusion Criteria: - Male/Female outpatients 18 years of age with chronic low back pain. Exclusion Criteria: - You have a serious or unstable disease of the heart or blood vessels, liver, kidney, lungs, or blood-related illness, problems with decreased blood flow to arms and legs (peripheral vascular disease), or other medical conditions, or psychiatric conditions that, in the opinion of the investigator, would affect your participation or be likely to lead to hospitalization during the course of the study. - Have acute liver injury (such as hepatitis) or severe cirrhosis. - Have had previous exposure to duloxetine. - Have a body mass index (BMI) over 40. - Have major depressive disorder. - Require daily narcotics.
Metabolic Effects of Chronic Ketosis (KETO-CHF Metabolic)

Heart Failure (HF) is a major public health issue because the disease affects 1-2% of the Western population and the lifetime risk of HF is 20%. Despite major improvements in the management and care of patients with HF, the 1-year mortality in patients with HF is 13% and \>50% of HF patients are admitted during a 2.5 year period. Furthermore, patients with HF have markedly decreased physical capacity and quality of life. Thus, there is a need for new treatment modalities in this group of patients.

It is well established that patients with heart failure have metabolic disturbances, including disturbed glucose metabolism with increasing insulin resistance, increased lipolysis, and disturbances in skeletal muscle homeostasis.

Presently there are no data on the clinical metabolic effects of long-term oral ketone-supplementation in patients with chronic HF.

In this study we aim to investigate the effect of 14 days modulation of circulating ketone body levels on endogenous protein, glucose, and fatty acid metabolism in patients with HFrEF.


Eligibility Criteria:
Inclusion Criteria:

* Chronic heart failure with NYHA II-III
* Left ventricular ejection fraction ≤40%
* Negative urine-HCG for women with childbearing potential

Exclusion Criteria:

* Known diabetes or HbA1c ≥48 mmol/mol
* Significant cardiac valve disease
* Severe stable angina pectoris
* Age \<18 years
A Phase III Study of SM-13496 in Patients With Bipolar I Depression.

The study evaluates the efficacy and safety of SM-13496 compared with placebo in patients with Bipolar I Depression.

The primary objective is to compare the efficacy of SM-13496 (20-60 or 80-120 mg/day) monotherapy with that of placebo in patients with depressive symptoms associated with bipolar I disorder by assessing the change from baseline in the MADRS total score at Week 6.

Eligibility Criteria:
Inclusion Criteria:

* Patients who were fully informed of and understand the objectives, procedures, and possible benefits and risks of the study and who provided written voluntary consent to participate in the study.
* Outpatients aged 18 through 74 years at the time of consent
* Patients meets DSM-IV-TR criteria for bipolar I disorder, most recent episode depressed (≥ 4 weeks and less than 12 months) without psychotic features.

Exclusion Criteria:

* Patients with imminent risk of suicide or injury to self, others, or property.
* Patients who had been hospitalized because of a manic or mixed episode within 60 days prior to screening.
* Patients who are otherwise considered ineligible for the study by the investigator.