cyrilzakka/clinical-trials huggingface dataset:
Pharmacokinetic/Pharmacodynamic & Safety Study of G3041 and SEVIKAR® Tablet in Healthy Adult Volunteers

Study Design : randomized, open label, single-dose, 2-way cross-over design

Phase : Phase I


Eligibility Criteria:
Inclusion Criteria:

* healthy volunteers between the ages of 19 to 55 years old
* 19 ≤ BMI ≤ 27
* having neither congenital/chronic diseases nor pathological symptoms/findings as results of medical examination
* doctor determines to be suitable as subjects within 4 weeks ago before administration

Exclusion Criteria:

* Hypersensitivity(or history of hypersensitivity) to amlodipine and olmesartan
* Exceed 1.5 times of the upper limit of the reference range of AST, ALT, total bilirubin, γ-GT
* Excessive drinking(exceed alcohol 140g/week)
* Excessive caffeine(exceed 4cups/day) and grape fruit/orange juice(exceed 1cup/day)
* Smoking over 10 cigarettes per day
Increasing Physical Activity for Adults With Multiple Sclerosis (MS)

This study aims to advance the scientific understanding and potential future implementation of physical activity promotion by testing the efficacy of a phone-based app for increasing activity in insufficiently active patients with multiple sclerosis (MS).

This study aims to test the efficacy of ExerciseRx, a free software platform designed to increase physical activity in people with multiple sclerosis (PwMS). Exercise is crucial for managing MS symptoms and overall health, but approximately 80% of PwMS do not get enough physical activity due to barriers like physical limitations, common MS symptoms, and lack of community exercise facilities. ExerciseRx translates clinically relevant activity data collected by personal smart devices into actionable metrics on a dashboard within the Epic electronic health record, allowing healthcare providers to prescribe and monitor tailored physical activity solutions for their patients. This study will test the effects of ExerciseRx in a sample of inactive PwMS.

Eligibility Criteria:
Inclusion Criteria:

* Provider-confirmed diagnosis of MS using revised 2017 McDonald criteria
* \> 18+ years of age
* Patient determined disease steps (PDDS) score \< 3, indicating the potential for some gait disability although typically ambulates without an assistive device
* Insufficiently active, defined as \< 150 minutes of physical activity per week, assessed using the PAVS in the EHR in clinics as part of the routine patient intake process
* Use of an iPhone with software version iOS13+ or an Android phone 4.1+
* Agree to install and use the ExerciseRx app for the entire study period and keep their phone on them during the daytime (e.g., pocket, bags, hands)

Exclusion Criteria:

* Recent (past 4 weeks) or planned surgery during the study period which may impact engaging in step counts
* MS relapse within the last 30 days
* Plans to travel internationally during the study period, which could interfere with server uploads of mobile phone data
* Those at a higher risk of falling or injury from falls or unable to safely exercise due to other medical conditions (e.g. heart conditions, diabetes or conditions made worse by walking or physical activity)
Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.

AMT-162 is an investigational gene therapy that encodes an artificial microribonucleic acid (microRNA or miRNA) targeting the SOD1 gene. This clinical study will test the safety of AMT-162 and explore the hypothesis that it will silence expression of mutant cytosolic SOD1 and thereby ameliorate the course of ALS caused by this mutant gene.

Eligibility Criteria:
Inclusion Criteria:

* Confirmed clinical and genetic diagnosis of SOD1-mediated ALS (SOD1-ALS) experiencing signs and/or symptoms of lower motor neuron dysfunction (weakness, atrophy, cramps, fasciculations), with or without upper motor neuron symptoms (weakness, bring reflexes, spasticity).
* ALSFRS-R score ≥ 25 at Screening.
* Slow vital capacity (SVC) ≥50% of predicted normal value.
* Capable of providing informed consent and complying with trial procedures, including: medically able to undergo lumbar puncture and has a responsible caregiver able to attend all clinic visit with the Participant.

Exclusion Criteria:

* SOD1 pathogenic or likely pathogenic variants in amino acid regions 43-47.
* Pathogenic repeat expansion in the C9orf72 gene
* Any of the following prior or concomitant treatments:

  * Any prior SOD1 suppression therapy with viral microRNA mediators
  * Prior SOD suppression therapy with antisense oligonucleotide (ASO) mediators such as tofersen (QALSODY™). Exception: Patients who previously received tofersen may be enrolled if the last dose of tofersen was received at least 20 weeks prior to the first Screening assessment and if there were no previous tofersen-related SAEs or ongoing tofersen-related adverse events that would increase the risk of receiving AMT-162, per Investigator judgment.
  * Other ALS medications riluzole (RILUTEK®, TIGLUTIK®), edaravone (RADICAVA®), and sodium phenylbutyrate and taururosdiol combination (RELYVRIO) or bioequivalents are allowed if dose is stable for 30 days prior to immunosuppression.
  * Any prior administration of an AAV gene therapy.
* Participants must be willing to forego new ALS treatments through at least 6 months after infusion of AMT-162. After 6 months, Investigators and participants may decide to add new ALS medications or change existing ALS medications.
iKnow: A Prospective Study to Evaluate the Use of Multi-omics in Multi-System, Early Onset Disorders

Prospective observational study to further understand the value that a multi-omic approach has in individuals with a multi system, early onset disorder that does not have a molecular diagnosis by whole genome sequencing.

Understand the value and utilization of integrated multi-omics, in multi-system early onset disorders that have failed to yield findings by whole genome sequencing

Eligibility Criteria:
Inclusion Criteria:

* Undiagnosed probands must meet all of the following:

  1. Must be able to understand and sign an informed consent and speak, read, and write in their native language (if the subject is a minor, their parent must have these abilities)
  2. Proband between the ages of 12 months and 65 years
  3. Study consent and participation of at least two unaffected family members (biological parents preferred. One biological parent and unaffected sibling allowed)
  4. If applicable, unaffected sibling must be between the ages of 12 months and 65 years
  5. A high prior probability of a multi-system early onset undiagnosed genetic disorder based on an expert medical assessment
  6. Clinical WGS that did not yield a definitive diagnosis
  7. It is preferred but not required that ancestry is from an under-represented population in current clinical genetic and translational research data repositories, especially African American, Asian American and Native American
  8. Must be willing to have blood, urine and fecal samples taken to include participating family members

Diagnosed probands must meet all of the following:

1. Must be able to understand and sign an informed consent and speak, read, and write in their native language (if the subject is a minor, its Parent or Legally Authorized Representative must have these abilities).
2. Proband between the ages of 12 months and 65 years
3. Study consent and participation of at least two unaffected family members (biological parents preferred. One biological parent and unaffected sibling allowed)
4. If applicable, unaffected sibling must be between the ages of 12 months and 65 years
5. Known genetic cause(s) of disease, disorder, or phenotypic defect through prior clinical whole genome sequencing
6. It is preferred but not required that ancestry is from an under-represented population in current clinical genetic and translational research data repositories, especially African American, Asian American and Native American
7. Must be willing to have blood, urine and fecal samples taken to include participating family members

Exclusion Criteria:

* Undiagnosed probands must not meet any:

  1. Known non-genetic cause(s) of disease, disorder, or phenotypic defect
  2. Principal Investigator decides that the study is not in the best interest of the proband

Diagnosed probands must not meet any:

1. Principal Investigator decides that the study is not in the best interest of the proband
Effect of Standardized Hibiscus Sabdariffa Tea in Seemingly Healthy Human Volunteers

Hibiscus sabdariffa tea is commonly used all over the world by healthy individual but the tea is also employed by patients in the management of chronic diseases such as hypertension diabetes, high cholesterol, liver disease etc. Several studies in humans and animal have proved the efficacy of Hibiscus sabdariffa tea in lowering blood pressure, blood glucose level and serum total cholesterol. But no study exists on the effect of daily consumption of this tea on blood pressure, blood glucose, total cholesterol and other biochemical and hematological parameters in healthy humans. Hence this study.

Several studies have been carried out on the effect of the water beverage of Hibiscus sabdariffa, most focus on hypertensive patients, diabetic patients and obese patient and some studies investigated the hypolipidemic a effect of the water beverage of Hibiscus sabdariffa as well as its effect on haematological parameters but mice were used for these studies. Little or no investigation has been done to assess the safety of daily consumption of this water beverage of hibiscus sabdariffa on humans.

Hence, this study aims at investigating the safety in the daily consumption of Zobo in humans, monitoring lipid profile, blood pressure, blood glucose, body mass index and haematological parameters such as haematocrit, haemoglobin, total white blood cells and also hepatic indices.

Eligibility Criteria:
Inclusion Criteria:

* Healthy volunteers only
* Not on any medications or herbs
* No disease condition
* Females not pregnant
* Non-smokers

Exclusion Criteria:

* Below 18yrs or above 40 years
* presence of chronic disease
* on medications pregnant females
Cone Beam CT Scanning in Lung and Bladder Cancer.

To study changes in tumor and normal organ size and/or position which occur during a course of radiation treatments.


Eligibility Criteria:
Inclusion Criteria:

* Patient has biopsy proven limited stage SCLC
* receiving radical concurrent chemoradiotherapy
* no prior chest RT
* no prior surgical resection of lung tumor
* no prior chemotherapy
* patients' lung tumors should be centrally-located to minimize the effect of respiratory motion
* patient has biopsy proven TCC bladder
* receiving radical concurrent chemoradiotherapy or RT alone
* no prior chemotherapy
* patient signs study-specific consent form

Exclusion Criteria:

* mixed small cell and non small cell histology
* patient not being treated with concurrent chemoradiotherapy
* chemoradiotherapy given sequentially
* previous chest RT
* previous chemotherapy
* prior surgical resection of lung mass
* patient ineligible for pelvic RT
* patient does not sign study consent
Pulmonary Ultrasound in the Diagnosis of Acute Thoracic Syndrome in Vaso-occlusive Sickle Cell Crisis

ATS (acute thoracic syndrome) refers to acute pulmonary involvement in a sickle cell patient. The diagnosis is based on the association of clinical signs (fever or respiratory symptoms) with a recent pulmonary infiltrate on the chest x-ray.

The main objective of the study is to evaluate the place of the pulmonary ultrasound for the diagnosis of ATS, in comparison with frontal chest x-ray.

The ATS occurs in half of the cases during hospitalization for a VOC (Vaso Occlusive crisis). The morbidity of ATS is important (respiratory insufficiency, duration of hospitalization, stays in intensive care, brain complications, pain, hypoxia, long-term sequelae, etc.) but no study has shown the benefits of curative treatments such as transfusion ( recommended at the time of diagnosis and practiced by some teams) or noninvasive ventilation (practiced by other teams). The first step before studying curative treatments is to have tools for early detection of ATS. Recent studies show non-inferiority of pulmonary ultrasonography compared to radiography and thoracic computed tomography in the diagnosis of pulmonary condensation (in adults and children). In a study performed in sickle cell adults admitted for ATS, pulmonary ultrasound was more sensitive than X-ray in detecting images of pulmonary consolidations visible on the chest CT scan.

Daswani et al have also shown the value of pulmonary ultrasound in comparison with radiography in the detection of consolidation lesions, suggestive of STA, in febrile children or young adults with sickle cell disease. They showed a good sensitivity (87%) and specificity (94%) of the pulmonary ultrasound.

Eligibility Criteria:
Inclusion Criteria:

* Age: ≥12 months and \<18 years
* Has a major sickle cell disease whatever the genotype
* Admission to hospital for a febrile vaso-occlusive crisis (VOC) or not
* Evolving at home for less than 48h
* Concomitant treatment with hydroxycarbamide / hydroxyurea possible
* Signed consent
* Patients affiliated to a French social security scheme

Exclusion Criteria:

* Child presenting an acute thoracic syndrome (ATS) from the outset at the admission
* Child who presented an ATS in the month preceding the inclusion
* Child in regular transfusion program or child who has received a hematopoietic stem cell transplant
* Child hospitalized at least 5 times for VOC in the year preceding inclusion (psychic problems making difficult the evaluation of the pain)
* Child who has already been included in the study during a previous VOC (each child participates in the study only once)
* VOC evolving for more than 48 hours before admission to the emergency room
* Acute splenic sequestration crisis at admission
A Novel Therapeutic Approach to Treat Chronic Pelvic Pain

To collect pilot data and assess the feasibility of a trial employing a single-arm intervention to study the effects of combined self-induced therapeutic tremors plus mindfulness on symptoms of chronic pelvic pain among women diagnosed with chronic pelvic pain.

The estimated prevalence of chronic pelvic pain among women is 2-24%. The etiology of chronic pelvic pain is multifactorial making it very difficult to manage clinically. Evidenced-based therapies are limited and a multidisciplinary approach, including complementary and alternative medicine, to treat women with chronic pelvic pain may be the best approach. This study assess the feasibility of conducting randomized controlled trial with a single arm intervention to treat symptoms of chronic pelvis pain among women diagnosed with chronic pelvic pain. The intervention will consist of a novel combined alternative therapy intervention among women with chronic pelvic pain (self-induced therapeutic tremor plus mindfulness). This is a clinically significant project as lifestyle interventions to treat chronic pelvic pain are urgently needed, given the prevalence and the healthcare costs associated with chronic pelvic pain.

Study Objective: The primary purpose of this project is to collect pilot data and assess the feasibility of a single arm intervention trial to improve symptoms of CPP among women diagnosed with CPP.

Intervention Arm - Combined Alternative Therapy: The novel combined alternative therapy that will be studied consists of self-induced therapeutic tremors (SITT) plus mindfulness. SITT is the intentional induction of the body's innate tremor mechanism to treat hyperarousal and muscle tension, which is characteristic of CPP. SITTs are thought to restore somatic and autonomic nervous system homeostasis, by discharging sympathetic arousal and down-regulating the autonomic nervous system. Previous work has shown that mindfulness training may improve symptoms of CPP, however there are no studies investigating the impact that the combination of mindfulness coupled with SITTs may have on the symptoms of CPP.

Eligibility Criteria:
Inclusion Criteria:

* Women
* Age \>18
* Physical requirements: be able to complete the intervention sessions
* Not seeking other therapeutic modalities, such as acupuncture, PT, during study duration
* Free from surgery for \>3 months
* being treated clinically for chronic pelvic pain and related conditions
* CPP of 6 months or more (to be considered chronic)
* Availability to commit to the 4-week intervention (two 60-minute sessions per week at a specified time, for 4 weeks)
* Internet access and consistent access to phone/email/text communication

Exclusion Criteria:

* Pregnancy
* Illicit substance use (e.g. cocaine, methamphetamines, heroin, street drugs) - marijuana and CBD oil and related supplements will be considered on a case-by-case basis by the principal investigators
* Current psychiatric illness/social situation that would limit compliance with study requirements, as determined by the principal investigators
Effect Of The System In The Enriched Fat Metabolism Of The Child Reached Mitochondrial Cytopathy

The aim of the present study is to compare Resting Energy Expenditure (REE) between normal fat diet (NFD) as a control vs high fat diet (HFD) as a treatment in Mitochondrial cytopathies disorders ( MID's ) patients. Secondary objectives is to compare diet induced thermogenesis (DIT) and body composition between NFD vs HFD.

This study is a randomized cross-over study. Normal fat diet (ie 10 % proteins, 45 % lipids, 45 % carbohydrates) is the imposed diet at baseline, high fat diet (ie 10 % proteins, 30 % lipids, 60 % carbohydrates is used according to the cross-over design trial. Main evaluation criteria is REE and second evaluation criteria is DIT both measured by indirect calorimetry ). 36 included MID subjects will be included in this study. Main evaluation criteria and second evaluation criteria will be measured at baseline, 1 month, 2 month and 3 month.

REE and DIT are measured by indirect calorimetry . The device used is a using an open-circuit ventilated hood system (QUARK RMR®, Cosmed, Pavona; Italy). The respiration quo- tient (RQ) and flow settings are calibrated by reference to alcohol combustion every six months. Before each test, the calorimeter is calibrated with a reference gas mixture (5% CO2, 95% O2 v/v). Expired carbon di- oxide (VCO2) and inspired oxygen (VO2) -flows are recorded, as well as the RQ. EE is calculated every minute from oxygen consumption (VO2 in ml/min) and carbon dioxide production (VCO2 in ml/min) using the Weir formula without protein correction. DIT is measured during 6 hours after a calibrated breakfast (normal fat diet or high fat diet according to the cross-over design) as 30% of theoric energy intake.

Eligibility Criteria:
Inclusion Criteria:

* 5 to 21 years-old children,

  * Children with a proven mitochondrial cytopathy documented on biopsies by histology, histoenzymology, ultrastructure and enzyme assay and/or on body fluids by laboratory markers including lactacidemia, metabolic acidosis, increased CSF lactate, lactate/pyruvate and beta-hydroxybutyrate/acetoacetate ratios, paradoxical postprandial ketonemia, alaninemia, citrullinemia, plasma acylcarnitines.
  * Children on the usual diet (30% lipids, 60% glucides and 10% proteins) for at least 1 month.
  * Informed consent of the 2 parents and from the child when in age to express a consent.
  * Child with a social security cover.

Exclusion Criteria:

* Acute infection (fever \> 38.5°C for more than 6h) within 7 days prior to the study.
* Disability for understanding and following the protocol
* Rejection of the study by the patient or failure to comply to the protocol
A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects

This is a multicenter, randomized, placebo-controlled, dose-escalation study. Enrollment is planned to occur at approximately 14 global sites. Approximately 24 subjects with CF.

This is a multicenter, randomized double-blind, placebo-controlled dose-escalation and parallel-arm, dose-ranging study. Enrollment is planned to occur at approximately 14 global sites. Approximately 24 subjects with CF who are homozygous for the F508del-CFTR mutation will be enrolled in two cohorts.

Eligibility Criteria:
Inclusion Criteria:

* Male or female subjects with a confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis or two CF-causing mutations,documented in the subject's medical record or confirmed at screening.
* Age 18 and above on the date of informed consent.
* Weight ≥40 kg.
* Homozygous for the F508del-CFTR mutation. Genotyping to be confirmed at screening.
* Ability to perform a valid, reproducible spirometry test with demonstration of a forced expiratory volume in 1 sec (FEV1) \>40% of predicted normal for age, sex and height.
* Screening laboratory tests with no clinically significant abnormalities that would interfere with the study assessments (as judged by the Investigator).
* Subjects who are sexually active must agree to follow the study's contraception requirements.

Exclusion Criteria:

* An acute upper or lower respiratory tract infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks prior to Day 1.
* Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to screening.
* Impaired renal function or known portal hypertension.
* History of prolonged QT and/or QTcF (Fridericia's correction) interval (\>450 msec) or QTcF \>450 msec at Screening.
* History of solid organ or hematological transplantation.
* History of alcohol abuse or drug addiction (including cannabis, cocaine and opiates) during the past year, (as judged by the Investigator).
* Use of ivacaftor or lumacaftor, within 4 weeks of Day 1
* Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to Day 1.
* Ongoing immunosuppressive therapy (including systemic corticosteroids).
* Hemoglobin \<10 g/dL.
* Abnormal liver function, at screening.
* Abnormal renal function at screening.
* Ongoing participation in another clinical study or prior participation without appropriate washout (minimum of 10 half- lives or 30 days, whichever is longer) prior to Screening visit.
B-line by Lung US in Patients With Acute Decompensated Heart Failure

The purpose of this study is to evaluate that B-line detected by lung ultrasound before extubation in patients with acute decompensated heart failure can predict future risk of reintubation and clinical outcomes during admission in Korea.


Eligibility Criteria:
Inclusion Criteria:

* mechanical ventilation due to acute heart failure

Exclusion Criteria:

* none
Safety and Efficacy Study to Compare Two Rosacea Treatment Regimens

To compare the safety and efficacy of two treatment regimens: 1) COL-101 and metronidazole gel 1%; 2) Doxycycline hyclate 100 mg and metronidazole gel 1%

Compare the safety and efficacy of two treatment regimens: 1) COL-101 and metronidazole gel 1%; 2) Doxycycline hyclate 100 mg and metronidazole gel 1%

Eligibility Criteria:
Inclusion Criteria:

* 8-40 total lesions
* erythema
* telangiectasia

Exclusion Criteria:

* non-pregnant, non-lactating
* achlorhydric
* gastric by-pass surgery
* allergy to study medications
* drug/alcohol abuse
* use of proton pump inhibitors