cyrilzakka/clinical-trials huggingface dataset:
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

A Phase IIIb (Three b), Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). Carbaglu® clinical experience in Organic Acidemia (OA) is limited to a non-comparative retrospective collection of data from patients who had received Carbaglu® for 1 to 15 days.

There is no current evidence supporting the use of carglumic acid for the chronic management of patients with OA. The investigators are proposing a randomized multicentre prospective clinical trial to evaluate long-term effects of the use of Carbaglu® (50mg/kg/day) combined with standard chronic therapy in patients with PA and MMA compared to standard chronic therapy alone.


Eligibility Criteria:
Inclusion Criteria:

* Male or female Children of 15 years old or less
* Parent or legal guardian agree to participate and to sign the (Institutional Review Board) IRB approved Informed Consent Form (assent forms will be waived due to the mental disability of those children)
* Not participating in any other clinical trial in the previous 30 days
* PA or MMA confirmed using the following criteria:

  * PA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of propionyl Co-A carboxylase in leukocytes or cultured fibroblasts or by DNA molecular testing of PCCA (Propionyl CoA Carboxylase, Alpha Polypeptide) or PCCB (propionyl CoA carboxylase, beta polypeptide) gene
  * MMA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of methymalonyl Co-A mutase in culture fibroblasts or DNA molecular testing of mutgene.
* Expected survival of at least 6 months, for the purpose of this study Survival expectance will be defined as patient not admitted to the Pediatric Intensive Care Unit (PICU) due to hyperammonemia more than 2 times per year or asymptomatic patients diagnosed by newborn screening program or stable chronic patients who are followed up at outpatient clinic.

Exclusion Criteria:

* Patients with other organic acidemia or any other cause of hyperammonemia
* Patient receiving other investigational therapy for PA or MMA
* Past history of hypersensitivity or drug allergy to Carbaglu®
* Patient with PA or MMA and other inherited genetic conditions or congenital anomalies
Mass Balance Study of [14C] TAS-205 in Healthy Volunteers

To evaluate the pharmacokinetics, mass balance recovery, metabolite profile and metabolite identification of \[14C\]TAS-205 following oral single doses.


Eligibility Criteria:
Key Inclusion Criteria:

1. Healthy adult male who provided written informed consent to participate in the study
2. Aged 20 years or older and younger than 40 years at the time of informed consent
3. Capable of oral intake.
4. Body weight of 50 kg or more and body mass index of 18.5 or more and less than 25.0

Key Exclusion Criteria

1. Had current or previous hypersensitivity or allergy to drugs
2. Had current or previous drug abuse (including use of illicit drugs) or alcoholism
3. Had any concurrent disease (including symptoms and signs; however, diseases that do not affect valuations in the study such as asymptomatic pollinosis and wart are excluded)
4. Consumed foods or beverages containing the St. John's Wort within 28 days before study drug administration
5. Had blood pressure, pulse rate, and body temperature meeting any of the following at screening:

Systolic blood pressure: \<90 mmHg or ≥140 mmHg, Diastolic blood pressure: \<40 mmHg or ≥90 mmHg, Pulse rate: \<40 beats per minute (bpm) or ≥100 bp, Body temperature: 35.0°C or ≥37.1°C
Evaluation of A Morning Dosing Of A New Medicine And Its Effects On Sleep At Bedtime In Subjects With Primary Insomnia

This study is designed to determine whether morning doses of GW679769, taken daily for 1 to 9 days, will promote sleep during the following night without significant post-dose thinking impairment and drowsiness in subjects with primary insomnia.


Eligibility Criteria:
Inclusion criteria:

* Difficulty going to sleep and/or staying asleep during at least the past 3 months.
* Insomnia must result in significant distress or impairment in functioning at home, socially or at work.
* Otherwise good health with no significant or unstable medical disorder including psychiatric, neurological, endocrine, heart, lung or gastrointestinal disorders, drug/alcohol abuse, cognitive impairment, cancer or chronic pain conditions such as arthritis.

Exclusion criteria:

* History of other sleep disorders such as sleep apnea or restless leg syndrome; regular sleep habits, including bedtime between 9 PM and midnight, nightshift/rotating shift work, frequent napping or planned travel across \>2 time zones.
* Use to moderate use of nicotine, caffeine and alcoholic products.
RECAB-ASA; Treatment of Recurrent Abortion With Aspirin

The aim of this study is to examine whether treatment with 75 mg aspirin daily compared with placebo could reduce the risk for a new miscarriage. The treatment starts when the pregnancy is detected on transvaginal ultrasound (around gestational week 6+) and continues to week 35/36. The study is a single center, randomized, placebo-controlled, double blind and stratified for age. 400 participants with the diagnosis idiopathic recurrent abortion are enrolled, 200 in each arm aspirin / placebo.


Eligibility Criteria:
Inclusion Criteria:

* recurrent abortion (≤ 3 consecutive abortions in first trimester)
* unknown etiology after work-up
* willingness to be randomized

Exclusion Criteria:

* previous participation in the study
* known cause of recurrent abortion, requiring specific management
* age ≥ 40
* BMI above 35
* IVF pregnancy if the reason for IVF was recurrent spontaneous abortion
* ongoing treatment with aspirin for other reason
* Sjoegren syndrome
Genetic Findings in a Chinese Family With Axenfeld-Rieger Syndrom

Axenfeld-Rieger syndrome (ARS) is a rare autosomal dominant disorder, characterized by anterior segment abnormalities and systemic abnormalities. To date,two major genes, forkhead box C1 (FOXC1) on chromosome 6p25 and pituitary homeobox 2 (PITX2) on chromosome 4q25, have been demonstrated to cause ARS. In this study, we performed complete ophthalmologic examinations and analysis of FOXC1 and PITX2 of a Chinese family with ARS.

Two genetions of a Chinese family with ARS were recruited to Aier Eye Hospital of Changsha.

Clinical evaluations: We performed full ophthalmologic examinations of all subjects, including: visual acuity, intraocular pressure measurements (Goldman), slit lamp, anterior segment photography, visual field test (Humphrey 750, Carl Zeiss, Germany), Anterior segment OCT (Carl Zeiss, Germany). If the refractive medium is clear, we also performed funduscopy, gonioscopic and retinal nerve fiber layer (RNFL) thickness measurements (Carl Zeiss, Germany).

Mutation analysis: About 2 ml of venous blood sampled from each subject and collected in Vacutainer tubes (Sanjiu Medical Technology Co., Ltd., Liuyang, China) containing EDTA. Genomic DNA was extracted from each blood using a genomic DNA mini kit for blood (Life Technologies), All coding exons, with flanking intronic regions, of FOXC1 and PITX2 were amplified using PCR with primers. The amplifed DNA was purifed by agarose gel electrophoresis and sequenced on a 3730/3700xl automated DNA sequencer (Applied Biosystems).

Eligibility Criteria:
Inclusion Criteria:

* a Chinese family with ARS

Exclusion Criteria:

-
Medical ANtiarrhythmic Treatment or Radiofrequency Ablation in Ischemic Ventricular Tachyarrhythmias

The study evaluates whether catheter based radiofrequency ablation is superior to optimized antiarrhythmic medical therapy in preventing ventricular tachyarrhythmia relapses in patients with ischemic heart disease and implantable cardioverter defibrillator.

The study is designed to evaluate whether catheter based radiofrequency ablation is superior to optimized antiarrhythmic medical therapy in preventing ventricular tachyarrhythmia relapses among patients with prior myocardial infarction and implantable cardioverter defibrillator (ICD). The patients have not been using using chronic antiarrhythmic medication. Primary end point is number of ICD therapies (defibrillations, cardioversions and antitachycardia pacing) and otherwise documented sustained ventricular tachycardia (duration more than 30 s or hemodynamically unstable ) or ventricular fibrillation episodes during 12 months follow-up period. Secondary end points include mortality, hospitalization for cardiac reason, health economics, quality of life and several ICD and arrhythmia related issues. Total length of the follow-up is at least two years.

Eligibility Criteria:
Inclusion Criteria:

* Patients 18-80 years of age with prior myocardial infarction and ICD (single chamber, dual chamber ICD or ICD with biventricular pacing capability (CRT-D)) for primary or secondary prevention of sudden cardiac death (SCD), who have had at least two documented episodes of sustained VT or VF and no chronic amiodarone treatment for ventricular tachyarrhythmias

Exclusion Criteria:

* Age less than 18 years or more than 80 years
* Non-ischemic cardiomyopathy
* Ongoing chronic treatment of ventricular tachyarrhythmias with amiodarone, intolerance/contraindication to all class III antiarrhythmic drugs (i.e., intolerance/contraindication to one class III agents is not excluding the patient if another one can be used)
* Contraindication to endocardial catheter ablation (e.g., intracavitary thrombi, contraindication to perioperative anticoagulation)
* Previous VT/VF ablation
* Open heart surgery within 3 months
* Prosthetic heart valve
* Planned revascularization (PCI or CABG)
* Surgery for structural heart disease or heart transplantation
* Pregnancy or planned pregnancy within the follow-up period
* Secondary cause for VT/VF (e.g., acute myocardial infarction)
* Patient does not want to participate
* Life expectancy less than 12 months
Serum Tryptase Levels During Cardiac Surgery, Diagnosis and Treatment Decisions for Allergic Reactions

The investigators aim to assess tryptase test efficacy for measuring the allergic response to protamine during cardiac bypass surgery. Additionally, the investigators aim to establish a differential diagnosis on the basis of potential allergens or clinical causes.

Venous blood samples from cardiac surgery cases will be obtained for tryptase measurement upon admission to the operating room (OR) and immediately prior to and 30 min after protamine infusion initiation. A rapid effect-response-based clinical assessment will be made using clinical and laboratory monitoring data for diagnosis and treatment decisions during protamine infusion, which may complicate the recovery from surgery.

Eligibility Criteria:
Inclusion Criteria:

* Cardiac by-pass cases

Exclusion Criteria:

* Insulin dependent diabetes
* Atopy
* Drug allergy history
Risk Factor of Anterior Cruciate Ligament Rupture After Ligamentoplasty : What is the Importance of a Relative Deficit of Contralateral Hamstrings Assessed by Isokinetic in Postoperative ?

Anterior cruciate ligament (ACL) is a common pathology (37 000 operations in 2006, nearly 43 000 in 2012 in France) justifying more and more operating indications in younger and younger patients. 70-80% of ACL ruptures occur without contact, which makes it a major public health interest because of its frequency and accessibility in terms of prevention.

The place of isokinetic assessment is important pre and postoperatively so that it has become systematic.

The main risk factors for known ACL lesions are female gender, pivotal sports, neuromuscular deficits, proprioceptive, hormonal, morphological deficits ... Moreover, there is also an increase in the number of contralateral fractures in patients who had a ligamentoplasty. There are many articles on the ACL pathology but unequal on the potential risk factors. The only proven risk factor for contralateral rupture is the age of the first episode; the female sex also seems to be important in some studies but remains more controversial. However, many factors have been studied: the intensity of the sport, the sex, the operative technique of ligamentoplasty, the operating duration, the duration of recovery of the sports activity, the level of recovery (of this sporting activity ) ...

Isokinetics is used to measure the peak of strength of quadriceps and hamstrings, in concentric or eccentric, at slow and fast speed and to determine a hamstring / quadriceps ratio to highlight a deficit or imbalance.

Eligibility Criteria:
Inclusion Criteria:

* Athletic patients all levels
* victims of ACL rupture during sports practice,
* operated by the same surgeon
* having been evaluated in isokinetic postoperative in the Sports Medicine Department of Rennes University Hospital
* having resumed a sporting activity
* with the first ACL rupture between 01/01/1994 and 12/31/2015

Exclusion Criteria:

* Patients with ACL rupture outside of a sporting context
* not having resumed post-operative sports activity on the maximum follow-up period of 15 years
* not evaluated for isokinetic activity in the sports medicine department
* minors at the time of inclusion
Evaluation of the Relationship Between Vaginal and Lower Urinary Tract Microbiomes and Infection After Hysterectomy

The purpose of this study is to learn more about the microbes (bacteria) that live in the vagina and the bladder. The investigators are doing this research study to understand the relationship between microbes (the microbiome) and the occurrence of urinary tract infection following surgical removal of the uterus and pelvic organ prolapse repair. The investigators expect Lactobacillus and Gardnerella will be the dominant organisms for most women. Non-Lactobacillus dominant microbiome communities will be more common in women who ultimately develop postoperative urinary tract infection.

The goal of this pilot study is to characterize the presence and stability of the microbial community in the vagina and lower urinary tract in a cohort of 20 postmenopausal women undergoing transvaginal hysterectomy with pelvic reconstruction for pelvic organ prolapse. The investigators intend to collect longitudinal samples in the preoperative, intraoperative, and postoperative time period in order to understand the pervasiveness of the microbes in the vagina and lower urinary tract, and to identify, in a very preliminary way, particular microbes that may be associated with postoperative urinary tract infection.

Eligibility Criteria:
Inclusion Criteria:

* Postmenopausal females (defined by cessation of menses for one full year)
* Planned surgical correction of pelvic organ prolapse with transvaginal hysterectomy and concomitant pelvic reconstruction for uterovaginal prolapse
* Scheduled surgery date within 4 weeks of study consent
* Physically able to self-collect vaginal swabs and clean-catch urine samples

Exclusion Criteria:

* Women who are premenopausal, pregnant or nursing
* Currently taking or have taken antibiotics in the past 2 weeks
* History of recurrent urinary tract infections
* History of mesh complications, including erosion/extrusion
* Non-vaginal approach to hysterectomy or prolapse repair
Tandem Occlusion After Emergent Stenting in Acute Ischemic Stroke

In acute ischemic stroke due to tandem occlusion, the emergent stenting has recently become an endovascular treatment option combining with mechanical thrombectomy to achieve recanalization. However, the data on the beneficial endovascular management of tandem occlusion in two circulations is still limited. The purpose of our study was to compare the improvement of clinical outcome between two circulations after emergent stenting at 3 months.

The posterior circulation (about 20 - 25%) is less frequent than the anterior one in the acute ischemic stroke, but high mortality and morbidity in the acute vertebrobasilar thrombosis even successful recanalization are revealed. Besides, tandem occlusion is one of the complex lesions in large vessel occlusions relating to the poor outcome, particularly in the posterior circulation. Recent studies have suggested that emergent stenting could be used as an additional treatment to achieve permanent recanalization together with mechanical thrombectomy in the intracranial segments. Permanent recanalization is one of the most important factors that impact patient outcomes after acute ischemic stroke. However, although there are various approaches for this lesion, the comparision of the effectiveness in the two circulations remains unclear. Thus, the aims of our study were to compare baseline characteristics and clinical outcome of tandem occlusions between anterior and posterior circulation after emergent stenting in extracranial arteries.

Eligibility Criteria:
Inclusion Criteria:

* Age \> 18 years old
* Premorbid mRS \< 2
* NIHSS \> 5
* ASPECTS ≥ 5 or pc-ASPECTS ≥ 5

Exclusion Criteria:

* Onset to treatment time \> 24 hours
* Loss to follow up after discharge
Comparison of Sunlight Exposure and Vitamin D Supplementation on Serum 25-hydroxyvitamin D Levels

This study aimed to compare the effects between sunlight exposure and oral vitamin D supplementation on serum 25-hydroxyvitamin D concentration and metabolic markers in Korean young adults.


Eligibility Criteria:
Inclusion Criteria:

* Serum 25(OH)vitamin D \< 12ng/mL
* age between 18 and 39 years
* Korean men and women

Exclusion Criteria:

* Vitamin D supplementation within 2 months
* Photosensitivity or UV allergy
* History of hyperparathyroidism/hypercalciuria/kidney stone/skin cancer and other cancer
* Under medication for hypertension, dyslipidemia, and diabetes
* Intake of photosensitive medicine during study period
* Exposure of strong UV (e.g., beach, sun tanning) during study period
* Pregnant/breast-feeding women
A Study to Evaluate EMB 001 in Subjects With Cocaine Use Disorder

EMB-001 is a combination of 2 drugs: the cortisol synthesis inhibitor, metyrapone (Metopirone®), and the benzodiazepine receptor agonist, oxazepam (original trade name Serax®; now marketed as oxazepam (generic) only).

This is a Phase 2 study in approximately 80 adult subjects with moderate-to-severe Cocaine Use Disorder (CUD).

This study is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, study to compare the safety and efficacy of EMB-001 with a placebo control in approximately 80 subjects with moderate-to-severe CUD. Subjects will receive investigational medicinal product (IMP) during a 12-week, double-blind Treatment Period (Week 2 through Week 13) and a 1-week, double-blind Taper Period (Week 14).

After undergoing study procedures during the Screening and Baseline Periods, subjects who meet inclusion and exclusion criteria will then be randomized in a 1:1 ratio (n=40/arm) to one of the following for the Treatment Period (weeks 2 - 13) on Study Day 8:

* EMB-001 720 mg metyrapone/24 mg oxazepam mg BID, for a total daily dose of 1440 mg metyrapone and 48 mg oxazepam (Active Group)
* Placebo BID, (Placebo Group)

During the Taper Period (week 14), subjects in the Active Group will receive EMB-001 240/8 mg BID; and the Placebo Group will continue to receive placebo. Both groups will change from taking three capsules BID (twice daily) to one capsule BID (twice daily).

There will be a follow-up visit for safety assessments at Week 18.

Eligibility Criteria:
Key Inclusion Criteria:

* Provide written informed consent prior to any study procedures
* 18 to 65 years of age
* DSM-5 diagnosis of moderate-to-severe CUD
* Seeking treatment for CUD. Subjects with past rehabilitation attempts are eligible if the most recent rehabilitation attempt ended at least 30 days prior to Screening
* Female subjects must be of non-childbearing potential
* Male subjects must agree to use accepted contraceptive regimens during the study and for at least 90 days after the last dose of the study drug

Key Exclusion Criteria:

* Any significant current medical conditions
* Known hypersensitivity to or intolerance of oxazepam, metyrapone, or any benzodiazepine, or severe hypersensitivity reaction (e.g., angioedema) to any drug
* Heightened likelihood of having adrenal insufficiency in the investigator's or designees' opinion
* Current court-mandated treatment requirement for a substance-use disorder
* Current DSM-5 moderate-to-severe substance use disorder, other than CUD, tobacco or caffeine
* Current DSM-5 opioid or benzodiazepine use disorder of any severity
IG-VMAT for Localized Prostate Cancer

To determine if hypofractionated IG-VMAT (70 Gy in 28 fractions over 5.6 weeks) will result in disease-free survival (DFS) that is no worse than DFS following conventionally fractionated IG-VMAT (80Gy in 40 fractions over 8 weeks) in patients treated for localized prostate cancer. Analysis the local progression, disease-specific survival (DFS), freedom from biochemical recurrence (FFBR), and overall survival (OS) of two groups. Observe the incidence of GI and GU toxicity.


Eligibility Criteria:
Inclusion Criteria:

* Age 50-79
* Histologically confirmed prostate adenocarcinoma
* Clinical stage T1-3N0M0 according to the AJCC 6th edition
* Gleason score must be \>5
* KPS \>70
* No radical surgery or cryosurgery for prostate cancer

Exclusion Criteria:

* Prior or concurrent invasive malignancy (except non-melanomatous skin cancer) or lymphomatous/hematogenous malignancy unless continually disease free for a minimum of 5 years. (For example, carcinoma in situ of the bladder or oral cavity is permissible)
* Evidence of distant metastases
* Previous radical surgery (prostatectomy) or cryosurgery for prostate cancer
* Previous pelvic irradiation, prostate brachytherapy, or bilateral orchiectomy
* Previous or concurrent cytotoxic chemotherapy for prostate cancer
Puerarin Versus Atorvastatin in Treating Metabolism Syndrome in Patients With Chronic Rheumatic Diseases

To evaluate the Effect of Puerarin tablets versus statins in treating metabolism syndrome in patients with chronic rheumatic diseases


Eligibility Criteria:
Inclusion Criteria:

* patients with a definite diagnose with rheumatic disease
* patients with metabolic Syndrome
* without conflict to the written, informed consent signed prior to the enrollment
* no severe hepatic or renal disorders
* no known carotid artery stenosis
* no coagulation disorders
* no hypertension

Exclusion Criteria:

* being in pregnancy, lactation period or under a pregnancy plan
* being allergic to the test drug
* not compatible for the trial medication
* without full legal capacity
PD, PK, and Safety of ALTO-203 in Patients With MDD

The goal of this clinical trial is to determine the pharmacodynamic effects of ALTO-203 in patients with MDD in a randomized, placebo-controlled, single-dose crossover treatment period. Additionally, safety, tolerability, and PK will be assessed in a subsequent randomized placebo-controlled multi-dose parallel-group treatment period of 28 days.

Participants will complete subjective response questionnaires and perform cognitive tasks during the single-dose period, in which participants will receive ALTO-203 25 μg and 75 μg, as well as placebo. During the multiple-dose period, participants will receive either ALTO-203 25 μg, 75 μg , or placebo. Safety will be assessed over the single dose and 28-day multiple dose periods.


Eligibility Criteria:
Inclusion Criteria:

* Have a diagnosis of moderate major depressive disorder (MDD)
* Presence of anhedonia symptoms
* Not taking antidepressant at Screening Visit 2
* Willing to comply with all study assessments and procedures

Exclusion Criteria:

* Evidence of unstable medical condition
* Diagnosed bipolar disorder, psychotic disorder, or dementia
* Concurrent use of prohibited medications
* Current moderate or severe substance use disorder
* Concurrent or recent participation in another clinical trial for mental illness involving an investigational product or device