cyrilzakka/clinical-trials huggingface dataset:
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).

The primary objective of this study was to evaluate the safety of intravenous (IV) infusions of sebelipase alfa in a more broad population of LAL-D participants than previously studied. Such participants may have been excluded from enrollment in other studies of LAL-D because of age, disease progression, previous treatment by hematopoietic stem cell or liver transplantation, less common disease manifestations, or disease characteristics that would preclude participation in a placebo-controlled study. This open-label study included infants \>8 months, children, and adults. At least 4 participants in the study were to be between the age of 2 and 4 years. Eligible participants received sebelipase alfa at a dose of 1 milligram/kilogram (mg/kg) every other week (qow).

Eligibility Criteria:
Key Inclusion Criteria:

1. Participant was \>8 months of age at the time of dosing.
2. Confirmation of LAL-D diagnosis as determined by the central laboratory or, for participants with prior hematopoietic stem cell transplant or liver transplant, historical enzyme activity or molecular genetic testing confirming a diagnosis of LAL-D.
3. Participants \>8 months but \<4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:

   * Dyslipidemia
   * Elevated transaminases
   * Impaired growth
   * Suspected malabsorption
   * Other clinical manifestation of LAL-D
4. Participants ≥4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:

   * Evidence of advanced liver disease
   * Histologically confirmed disease recurrence in participants with past liver or hematopoietic transplant
   * Persistent dyslipidemia
   * Suspected malabsorption
   * Other clinical manifestation of LAL-D

Key Exclusion Criteria:

1. Participant had known causes of active liver disease other than LAL-D, which had not been adequately treated.
2. Participant received a hematopoietic stem cell or liver transplant \<2 years from the time of dosing.
3. Participant with co-morbidities other than complications due to LAL-D, which were irreversible or associated with a high mortality risk within 6 months or would interfere with study compliance or data interpretation.
Exploratory Metabolomics Study of Exhaled Breath in Pulmonary Embolism

Pulmonary embolism is a frequent and recurrent pathology, especially in the elderly. It is often preventable, with high mortality and morbidity, making it a major public health issue. The clinical presentation of pulmonary embolism is non-specific and very highly variable, ranging from asymptomatic thrombus diagnosed incidentally to sudden death. The current diagnosis of pulmonary embolism is based on several diagnostic techniques, mainly non-invasive, which should be used sequentially.

We propose to sample the volatile organic compounds using a device that allows them to be trapped on polymer cartridges. Sampling will be performed under monitoring of respiratory pressure and a capnograph to collect alveolar and upper respiratory tract air separately without contamination of the oral cavity or sinuses.

This exploratory metabolic analysis will be non-targeted (analysis of all molecules detectable without a priori).

The main objective of the study is to identify specific metabolic profiles to predict the results of ventilation-perfusion pulmonary tomoscintigraphy in subjects undergoing this examination for suspected acute pulmonary embolism.

Secondary purposes :

* To identify metabolic profiles to be used to predict, in combination with clinical probability scores, the results of ventilation-perfusion pulmonary tomoscintigraphy in subjects undergoing this examination for suspected acute pulmonary embolism.;
* To correlate metabolic profiles with the topography of embolized lung territories


Eligibility Criteria:
Inclusion Criteria:

* Male or female, at least 18 years old
* Patients with clinical suspected pulmonary embolism
* Patient willing and able to provide informed consent

Exclusion Criteria:

* Contraindications to pulmonary ventilator-perfusion tomoscintigraphy
* All types of thrombolytic or anticoagulant treatment
* Diagnosis of pulmonary embolism established prior to inclusion in the study
* Massive pulmonary embolism (systolic pressure \< 90 mm Hg)
* Pregnancy
* Breastfeeding
Transcatheter Aortic Valve Replacement for Failed Transcatheter Aortic Valve

Patients with failed transcather aortic valve (stenotic, regurgitant, both) and indication to further transcatheter aortic valve replacement (TAVR).

The purpouse of this study is to evaluate the indication to a second TAVR, the selection criteria of the second transcatheter aortic valve, procedural and clinical outcomes.

The primary endpoint of the study is overall mortality and Cardiovascular mortality at 1 year


Eligibility Criteria:
Inclusion Criteria:

* Patients with failed transcather aortic valve (stenotic, regurgitant, both) and indication to further transcatheter aortic valve replacement (TAVR).

Exclusion Criteria:

-
Application of Spaghetti Knotting Technology in Single-hole Laparoscopic Suturing Instructional Program on the Basis of Multidirectional Stitching Technology.

The surgeon's knot-tying technique plays a crucial role in the recovery of patients after single-port laparoscopy. Knot tying is very challenging in minimally invasive surgery and is a critical skill in advanced surgery. In this study, investigators introduce a new knotting technique, compare it with the traditional knotting technique in many aspects, and also discuss its application in the teaching of single-port laparoscopic knotting.


Eligibility Criteria:
Inclusion Criteria:

1. the habitual hand is the right hand;
2. no practical experience in operating single-hole laparoscopic instruments;
3. no simulator sickness;
4. training and assessment are completed on time and carefully

Exclusion Criteria:

1. have participated in other skills training programs
2. are not interested in single-hole laparoscopy will be excluded
Noninvasive Monitoring of Cerebral Blood Flow Autoregulation

Rheoencephalography (REG) shows promise as a method for noninvasive neuromonitoring, because it reflects cerebrovascular reactivity. This protocol will study clinical and technical conditions required to use REG. Additionally, our goal is to study noninvasive peripheral bioimpedance pulse waveforms in order to substitute invasive SAP. A previous study demonstrated that REG can be used to detect spreading depolarization (SD), the early sign of brain metabolic disturbance. SD can be measured invasively with DC EEG amplifiers only. Our goal is to create an automatic notification function for REG monitoring indicating change of clinical conditions.

Neuromonitoring of patients with severe neurological illness are detailed elsewhere. In the setting of cerebral edema, ICP monitoring is a staple of neurocritical care. Pressure AR is an important hemodynamic mechanism that protects the brain against inappropriate fluctuations in CBF in the face of changing CPP. Both static and dynamic AR have been monitored in neurocritical care to aid prognostication and contribute to individualizing optimal CPP targets in patients. Theoretically, failure of cerebral AR is associated with poor outcomes in various acute neurological diseases. Continuous bedside monitoring of autoregulation is now feasible and should be considered as a part of multimodality monitoring including measurement of pressure reactivity. A previous study documented that REG (REGx) and ICP (PRx) has high correlation in order to detect the lower limit of CBF AR. The fundamental relationships between SAP, vessel tone, cerebral blood volume and ICP form the basis for the pressure reactivity index (PRx). PRx is analogous to other time domain AR indices and is calculated as the continuous correlation between thirty consecutive time-averaged (10 s) SAP and ICP values. A positive index (positive correlation) implies impaired passive CBF AR, while a negative index (inverse correlation) implies intact, active AR. The utility and feasibility of REG as a monitoring modality is previously demonstrated and validated as a reflection of cerebrovascular reactivity. The bioimpedance amplifier was used previously at Walter Reed Army Institute of Research (WRAIR) and Naval Medical Research Center (Silver Spring, MD); and has an FDA safety clearance. It is expected that REG can predict evolving vasospasm and expanding intracranial bleeding amongst several other clinical applications.

Eligibility Criteria:
Inclusion Criteria:

* Intact fronto-temporal area
* Intact lower arm area
* Clinical suspicion of elevated intracranial pressure

Exclusion Criteria:

* N/A
High Flow Therapy vs Hypertonic Saline in Bronchiolitis

The purpose of this study is to demonstrate that heated, humidified, high-flow nasal cannula (HHHFNC) is superior to hypertonic saline solution (HSS) in the treatment of moderate acute viral bronchiolitis in infants in improving respiratory distress and comfort and reducing length of hospital stay (LOS) and admission to Pediatric Intensive Care Unit (PICU).


Eligibility Criteria:
Inclusion Criteria:

* Children aged 6 months or less
* Moderate (Respiratory Distress Assessment Instrument score of 4 or greater) viral bronchiolitis (as defined by McConnockie)
* Meeting Admission criteria

Exclusion Criteria:

* History of prematurity
* Chronic lung disease
* Cystic fibrosis
* Congenital heart disease
* Neuromuscular disease
* Airway anomalies
* Immunodeficiency
* Those requiring immediate intubation and ventilation.
Clinical Extend Study to Evaluate Safety and Effectiveness of TnR Nasal Mesh for Correction of Nasal Septum on Patients

The purpose of this observation study is to evaluate long term safety and efficacy at 2\~3 years after surgery on patients who have completed nasal septal deformity surgery using TnR Nasal Mesh.

Nasal obstruction is a nasal disease commonly seen in the area of otolaryngology, and major symptoms include nasal congestion, rhinitis, and etc.

Nasal septoplasty is a surgery performed to improve above symptoms of nasal obstruction and so far, many surgical technique have been studied. All surgical techniques aimed to improve breathing through the nasal cavity by supporting the end of nose using surgical materials such as autologous bone or nonabsorbable material.

In the case of autologous bone transplantation, safety is ensured, but there is a limitation since it causes deficiency of the donor site, and the nasal septum may be altered by a modification of the implanted tissue.

In the case of nonabsorbable materials, there is a high risk of side effects such as hyposmia, septal perforation, and occurrence of boil.

TnR Nasal Mesh, as a surgical material for nasal septoplasty has a sufficient flexural strength to support the nasal septum and to overcome the force of returning to a deviated state prior to surgery.

Animal tests have shown that TnR Nasal mesh well supports the nasal septum after surgery and the surrounding tissue is well fused between the pores without inflammatory responses. Based on above results, a clinical trial was planned to evaluate long term safety and efficacy of TnR Nasal Mesh as a material for nasal septoplasty.

Eligibility Criteria:
Inclusion Criteria:

1. Patients who have completed nasal septal deformity surgery using TnR Nasal Mesh.
2. Females of childbearing potential must have a negative urine pregnancy test, and must not plan to become pregnant during the course of the study.
3. A person who voluntarily signed a consent form after hearing and understanding the description of this clinical study.

Exclusion Criteria:

1. Pregnancy or lactation

   * Before screening
2. History of surgery in nasal or paranasal sinuses before screening
3. History of radiation treatment at Head and neck
4. History of having participated in other clinical trial of a drug/ a medical device within three months

   * Screening point
5. Patients with untreated nasal bone fraction or trauma of nasal
6. Patients with surgical site infection caused by nasal bone fraction or trauma
7. Patients with inflammation in a nasal cavity
8. Patients with asthma
9. Patients with untreated a perforation of septum, a sinusitis, a nasal cavity polyposis, a nasal cavity sarcoidosis, nasal valve collapse or wegener's granulomatosis
10. Patients with sarcoma or carcinoma in a nasal cavity
11. Patients with untreated palate-facial disfigurements or cleft palate
12. Patients with sepsis
13. Patients with systemic inflammatory disease
14. Patients who are unsuitable for clinical sturdy determined by the researcher.
Building a Renewed ImaGe After Head & Neck Cancer Treatment (BRIGHT) Multi-Site RCT

In this multi-center randomized clinical trial, head and neck cancer (HNC) survivors with clinically significant body image distress (BID) (N=180) will be randomized to BRIGHT (a brief video tele-cognitive behavioral therapy intervention) or Attention Control (AC, a manualized tele-supportive care intervention that controls for professional attention, dose, delivery method, and common factors). HNC survivors will complete IMAGE-HN (a validated patient-reported outcome measure \[PROM\] of HNC-related body image distress \[BID\]; primary endpoint), measures of psychological and social well-being and quality of life (QOL), and measures of theory-derived mechanisms of change underlying BRIGHT (mediators).

We will perform a multi-site randomized clinical trial (RCT) comparing BRIGHT with AC to test our hypotheses that BRIGHT reduces BID and improves psychological and social well-being and QOL at 2, 3, 6, and 9-months post-randomization by enhancing body image coping skills and decreasing unhelpful automatic thoughts. HNC survivors with clinically significant BID (N=180) from multiple sites in the United States will be randomized 1:1 to BRIGHT or AC. We will conduct semi-structured interviews with key stakeholders (n=10/site) and in-depth site visits to assess barriers and facilitators of the adoption of BRIGHT and develop an implementation toolkit to enhance the future adoption of BRIGHT into routine clinical care.

Eligibility Criteria:
Inclusion Criteria:

1. Age \> 18 years on the day of informed consent
2. History of pathologically confirmed squamous cell carcinoma (or histologic variant) of the oral cavity, pharynx, larynx, nose/paranasal sinuses, carcinoma of a major or minor salivary gland, or cutaneous malignancy of the face or neck
3. History of curative intent surgery with or without adjuvant therapy, with or without reconstruction
4. Completion of oncologic treatment within 12 months of study enrollment (but no sooner than 6 weeks post-treatment completion)
5. Cancer-free at the time of accrual

   -patients with known indolent malignancies (e.g., non-melanoma skin cancer, low risk thyroid cancer, untreated prostate cancer, etc) would not exclude a patient from the study
6. No planned significant HNC ablative or reconstructive surgery (defined by a postoperative inpatient stay of at least three days) during the study intervention or follow-up period as determined by the HNC oncologic surgeon at the time of study accrual
7. Willingness to be randomized to either BRIGHT or AC
8. IMAGE-HN score \> 22

Exclusion Criteria:

1. Inability to speak or write English
2. Pre-existing, ongoing CBT services for other disorders and the participant is not willing to discontinue the prior therapy for the duration of the proposed trial
3. Initiation or adjustment (\< 3 months of baseline) of psychotropic medication
4. Severe psychiatric comorbidity (e.g., suicidal ideation, psychosis)
Revisiting Omental Transposition in Locally Advance and Locally Recurrent Breast Cancer

Locally advance breast cancer and extensive locoregional recurrence are still considered as complexity of reconstruction. Although previous studies concluded that omental transposition was considerably indicated in poor vascularity in irradiated tissue, secondary infection and large defect become the obstacles of local tissue flaps. While some centres showed the excellent healing of omental transposition. Meanwhile, many studies showed the preferable outcome in escalating radiotherapy in term of locoregional control. In the investigators' institute, omental transposition in breast cancer has been developed for twenty years. The only institute in Thailand enhanced using this procedure in order to closure of large primary breast cancer and chest wall recurrence. This procedure is undertaken by single surgeon (Dr. K.J.). The investigators' protocol of omental transposition is including preoperative chemotherapy, preoperative accelerated radiation up to 80 Gy, approval of breast cancer centre committee, and meticulous wound care including vacuum assisted wound dressing, non-adhesive dressing. As a result, survival time could be improved.


Eligibility Criteria:
Inclusion Criteria:

* The patients with omental transposition operation within King Chulalongkorn Memorial Hospital
* More than 18 years of age

Exclusion Criteria:

* The patients with refusal
Contingency Management for Smoking Cessation in the Homeless

Smoking cessation services are an unmet need among the homeless, who smoke at rates more than 3 times the national estimate; successful interventions in this underserved population have the potential for improving individual and public health. Contingency management (CM) is a behavioral intervention with efficacy in a number of substance use disorder populations, including smokers. However, no known studies have evaluated the effect of CM in homeless smokers. The investigators plan to examine smoking-related outcomes in homeless treatment-seeking smokers (N = 70) randomized to standard care smoking cessation (transdermal nicotine replacement therapy \[NRT\] + standard counseling + carbon monoxide \[CO\] monitoring) or standard care plus CM (NRT + standard counseling + CO monitoring + CM) conditions. Standard counseling and CO monitoring will occur for the first 4 weeks, with NRT use continuing through week 8. Participants in both conditions will meet with study staff up to twice daily on weekdays for biochemical verification of smoking status. Participants in the CM condition will have the opportunity to earn prizes for negative breath samples (CO ≤ 4 ppm) up to twice daily on weekdays. CO breath samples will be collected at all visits. The investigators expect that participants randomized to CM will have better outcomes compared to those in standard care.


Eligibility Criteria:
Inclusion Criteria:

* age ≥ 18 years
* homeless
* regular smoker

Exclusion Criteria:

* non-English speaking
* contra-indications for transdermal nicotine
* in recovery for pathological gambling
Intraoperative Electrical Stimulation to Improve Nerve Grafting Outcome

A research study to find out if brief intraoperative electrical stimulation therapy improves nerve regeneration and smile outcomes following two-stage cross face nerve graft facial reanimation surgery.

Participants that choose to enroll in this study will be randomly assigned in a 2:1 ratio to a "treatment" or "control" group. In either group, patients will receive the same care as they normally would except for two minor differences. First, patients in both groups undergo nerve biopsies during surgery; these biopsies do not affect the length or outcome of surgery. Second, the treatment group will receive brief electrical stimulation therapy for approximately 10 minutes during the first stage cross-face nerve graft surgery.

The purpose of this study is to evaluate efficacy of an intraoperative dose of brief electrical stimulation (BES) to improve axonal regeneration across a cross-face nerve graft (CFNG) using nerve histology and to evaluate clinical outcomes of treatment and control groups using clinician graded, patient reported, and objective smile metric.

Eligibility Criteria:
Inclusion Criteria:

1. Patients 18-80 years-old with unilateral facial paralysis who are candidates for two- stage CFNG smile reanimation surgery (i.e. CFNG-driven free gracilis muscle transfer (FGMT) or dual innervation FGMT).
2. Agree to participate in the study.

Exclusion Criteria:

1. Bilateral facial paralysis
2. Age less than 18 years-old or older than 80 years-old
3. Comorbid medical condition preventing two-stage CFNG surgery
Interleukin-6 Inhibitors and Drug-drug Interactions in Patients With Rheumatoid Arthritis

With this study the investigators aim to assess if drug metabolism changes in patients with rheumatoid arthritis when an interleukin (IL)-6 inhibitor is initiated.

Patients with rheumatoid arthritis have an increased level of inflammation in the body which can lead to decreased expression and activity of drug metabolizing enzymes in the liver. This will lead to a decreased metabolism and excretion of drugs. The inflammation is driven by a number of proinflammatory cytokines e.g., IL-6. The investigators hypothesize that patients with rheumatoid arthritis initiating treatment with an IL-6-receptor inhibitor (anti-IL-6R) will obtain a normalization of the activated IL-6-pathway resulting in increased expression and activity of drug metabolizing enzymes and hence increased metabolism. Ultimately, this normalization of drug metabolism could lead to insufficient efficacy of a wide variety of drugs.

The investigators will perform a clinical pharmacokinetic trial. The study will include patients with active rheumatoid arthritis and a need to initiate treatment with an IL-6 receptor antibody. Patients will ingest a 6-drug cocktail consisting of probes for specific CYP enzymes. Plasma and urine will be drawn over 6 hours to determine concentrations of the drugs and their metabolites. Patients will then initiate IL-6 receptor antibody treatment and to assess both short- and long-term impact of altered inflammation, the same 6-drug cocktail will be ingested, and concentrations measured, after three weeks and three months. To help understand the mechanism and the putative involvement of inflammation, markers of inflammation such as cytokines, transcription factors, etc. will also be assesses.


Eligibility Criteria:
Inclusion Criteria:

* Active rheumatoid arthritis
* Age 18-75 years
* eGFR \> 30 mL/min
* absolute neutrophil count (ANC) ≥ 2 x 109 /L
* Platelet count \> 150 x 103 /μL (corresponding to \>150 x 109 /L)
* ALAT in the normal range or within 1.5x the upper limit of normal.
* Use of effective contraception (only woman of childbearing potential)
* Negative test for hepatitis and tuberculosis

Exclusion Criteria:

* Known sensitivity to any of the medications used.
* Active severe infections
* Malignancy
* Diverticulitis
* Intake of medications which can influence the safety of the patient or the results of the study. Can include prescription medications, over-the-counter medications, herbal medicines or dietary supplements. Will be assessed by the investigators.
* Participation in other clinical intervention trials.
* Pregnancy or breastfeeding
A Study of TCD601 (Siplizumab) in Newly Diagnosed Adult Amyotrophic Lateral Sclerosis (ALS) Patients

The purpose of this study is to investigate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of TCD601 (siplizumab) in newly diagnosed adult ALS patients.


Eligibility Criteria:
Key Inclusion Criteria:

* Male or female patients ≥ 18 to 80 years of age.
* Diagnosis of ALS by revised El Escorial Criteria, at study entry within 24 months of first symptoms.
* Patients on existing ALS treatment must have been on a stable dose for 28 days.

Key Exclusion Criteria:

* Patient with severe systemic infections, current or within the two weeks prior to randomization.
* Subjects who, in the opinion of the investigator, are not capable of giving informed consent for the study or who are unable or unwilling to adhere to the study requirements outlined in the protocol.
* Use of other investigational products or treatment in another investigational drug study within 30 days of screening
* Pregnant or nursing (lactating) women.