cyrilzakka/clinical-trials huggingface dataset:
Analysis of Post-Translational Modifications of a Critical Protein Implicated in Amyotrophic Lateral Sclerosis

The purpose of this research study is to discover and quantitate the differences in post-translational modifications found in the Cu, Zn superoxide dismutase (SOD1) of patients with amyotrophic lateral sclerosis (ALS) as compared to healthy individuals. SOD1 is a known genetic cause of ALS. With certain mutations, SOD1 gains a toxic function which leads to motor neuron death.

Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disease in which mutations in human Cu, Zn Superoxide Dismutase (SOD1) have been identified as a cause of familial ALS (FALS) cases.1-2 It has been shown that mutant SOD1 develops a novel toxic function through experiments demonstrating that many disease mutants maintain enzymatic activity, SOD1-null mice do not exhibit ALS symptoms, and co-expressed wild type protein does not rescue the disease-state.7-11 The majority of cases, however, are not caused directly by mutations of SOD1, instead being caused by a poorly understood interplay of several genes as well as environmental factors, which is often referred to as sporadic ALS (SALS).3 It has been found that FALS and SALS share similar pathology. 4-6 The hSOD1 protein aggregates characteristic of FALS have also been found in SALS patients, furthering the evidence that hSOD1 has an important role in the etiology of ALS in sporadic ALS patients.16-19 The exact mechanism of SOD1-associated toxicity has not yet been elucidated though many disease mutants have been shown to destabilize the SOD1 dimer. In this study we aim to compare the levels of SOD1 post-tra slational modifications in ALS patients to levels in healthy donors and to determine if there are distinct patterns of protein glutathionylation or phosphorylation. Our overall goal is to elucidate a direct mechanism of toxicity in SALS as well as identify potentially critical triggers

Eligibility Criteria:
Inclusion Criteria:

* SALS patients
* SOD1 associated FALS patients
* Healthy control

Exclusion Criteria: none
The Effect of a Web-based Simulation Application

Cardiopulmonary resuscitation (CPR) training for nursing students is important. The importance of using computer-assisted simulation in gaining skills for basic life support applications is increasing. In addition to the use of simulation-based teaching methods in many different fields, it is seen that they are also used for the development of cardiopulmonary resuscitation practices of students and produce effective results.The aim of the study is to evaluate the effect of a web-based simulation application used in basic life support education on the level of knowledge, satisfaction and self-confidence of nursing students.

This research was conducted with 60 nursing student (intervention group: 30, control group: 30). The study is a mixed method type randomized controlled intervention study in which quantitative and qualitative research methods are used together.The quantitative data were obtained by using the ""Basic Life Support Knowledge Form", "Basic Life Support Skill Form", "Student Satisfaction and Self Confidence in Learning Scale, Perceived Learning Scale" , and the qualitative data were collected by using the "Semistructured Interview Form" in focus group sessions.

Eligibility Criteria:
Inclusion Criteria:

Being nursing student

Exclusion Criteria:

no previous training in basic life support not filling out the questionnaires completely
Managed Access Programs for LDK378, Ceritinib

The purpose of this registration is to list Managed Access Programs (MAPs) related to LDK378, Ceritinib.

CLDK378A2401 - No longer available - Managed Access Program (MAP) to provide access to Ceritinib, for ALK Positive Tumors.

CLDK378A2402 - No longer available - An open-label, multi-center, Expanded Treatment Protocol (ETP) of oral LDK378 in adult patients with non-small cell lung cancer (NSCLC) characterized by ALK positivity.

CLDK378A2003M - Available - Managed Access Program (MAP) Cohort Treatment Plan CLDK378A2003M to provide access to LDK378 (ceritinib), for ALK-Positive Tumor Pediatric Patients.

Eligibility Criteria:
Inclusion Criteria:

1. An independent request was received from a licensed physician.
2. The patient has a serious or life-threatening disease or condition and there is no comparable or satisfactory alternative therapy available for diagnosis, monitoring, or treatment.
3. The patient is not eligible or able to enroll in a clinical trial or continue participation in such trial.
4. There is a potential patient benefit to justify the potential risk of the treatment use, and the potential risk is not unreasonable in the context of the disease or condition to be treated.
5. The patient must meet any other medical criteria established by the medical experts responsible for the product or by the health authority in the country of request (as applicable).
6. Provision of the product will not interfere with the initiation, conduct, or completion of a Novartis clinical trial or overall development program.
7. Managed Access provision is allowed per local laws/regulations.
Revisiting the Mechanism of the Anti-inflammatory Effect of Colchicine

We are doing this research study to better understand the mechanism of action of colchicine, a drug commonly prescribed for gout. Precisely, we aim to evaluate the effect of colchicine on a specific protein (GDF15) blood levels at different timepoints after its administration.

This research study will compare GDF15 blood levels after the administration of colchicine or placebo. The placebo looks exactly like colchicine but contains no active drug. During this study, participants may get a placebo instead of colchicine. Placebos are used in research studies to see if the results are due to the study drug or due to other reasons.

This will be a randomized, placebo-controlled, double-blind, pilot study evaluating the effect of a single FDA-approved dose of colchicine in healthy adults on plasma GDF15 levels. Randomization will be stratified by sex as this may be a factor influencing GDF15 levels.

On Day 1, subjects will be admitted on the CCI 9A unit early in the morning, 30 minutes before the study drug administration. They will be asked to fast from midnight before the dose until 2 hours after. Water is allowed during the fasting period. Baseline blood tests will be drawn and a peripheral line will be left in place for further blood draws. A dose of either colchicine or placebo will be administered. Blood samples will be drawn at 2, 4, 6, 8, 10 and 12 hours. The patient will be discharged home after the last blood draw and the removal of the peripheral line. Two other blood draws will be performed using standard procedures at 24 and 48 hours post study drug administration at the research clinic

Eligibility Criteria:
Inclusion Criteria:

* Participant able to understand and sign the informed consent of the study
* Healthy male or female aged from 18 to 50 years.
* Participant considered healthy after evaluation such as medical history and screening laboratories.

Exclusion Criteria:

* Pregnant or lactating women
* Use of colchicine within 28 days prior to study drug administration.
* Renal insufficiency (GFR \< 50 mL/min)
* Use of any drug or product known to interact with colchicine including CYP3A4 and/or P-glycoprotein (P-gp) inhibitors in the 14 days prior study drug administration, such as atazanavir, clarithromycin, indinavir, itraconazole, ketoconazole, nefazodone, nelfinavir, ritonavir, saquinavir, telithromycin, amprenavir, aprepitant, diltiazem, erythromycin, fluconazole, fosamprenavir, verapamil, cyclosporine and ranolazine. Significant consumption of grapefruit juice can also inhibit CYP3A4. Interactions may also be seen with fibrates (e.g., gemfibrozil), HMG-CoA reductase inhibitors (statins) and digoxin.
* History of colchicine allergy.
The Safety of Oka Varicella in Children Prior to Solid Organ Transplantation

This study sought to determine the safety of the varicella vaccine pre- and post-transplantation when given to pediatric patients listed for solid organ transplantation. The study assessed the antibody response to a two-dose vaccine regimen and determined the durability of that antibody response at several intervals in the post-transplant period. As a secondary objective, the relationship between antibody titers and different variables were explored


Eligibility Criteria:
Inclusion Criteria:

* Children \> 9 months of age and adolescents \< 18 years of age.
* Pediatric transplant candidates who are in any of the following categories:

  1. listed to receive kidney, liver, heart, lung or other or solid organ transplantation in a Canadian transplant centre
  2. not yet officially listed, but are regarded by their physicians as transplant candidates by virtue of their underlying diseases
* No clinical history for varicella.
* Seronegative for antibodies to VZV except infants 9 - 12 months of age who may be seropositive due to maternal antibodies.
* Informed consent obtained

Exclusion Criteria:

* Previous immunization with varicella vaccine.
* Any established immune deficiency (underlying disease or drug induced) or any neoplastic disease
* Children on any oral and / or intravenous steroids within 3 months prior to immunization. Children on inhaled corticosteroids in excess of 800 mcg of beclomethasone dipropionate ( or equivalent ) per day.
* Any exposure to varicella or herpes zoster in the previous 4 weeks involving household, playmate or hospital contacts.
* Inability to delay the transplantation for up to 6 weeks following the last varicella immunization.
* Presence of a person at increased risk for varicella infection in direct and unavoidable proximity with the vaccinees ( e.g. an immunocompromised sibling)
* Past history of varicella or known positive antibody titer for varicella except infants 9 - 12 months of ages who may be seropositive due to maternal antibodies
* Known hypersensitivity to any of the components of the vaccine, including neomycin and gelatin
* Patients whose mothers are known to be seronegative and plan to become pregnant in the subsequent three months
* Administration of VZIG or any other blood products in the previous six weeks (packed red blood cells excepted).
* Any significant infection and/or fever at the time of vaccination
* Any patient receiving or planning to receive salicylates in the six weeks after immunization
* Any patient who has received any live vaccine for 6 weeks or killed vaccine for 2 weeks prior to or after the scheduled VARIVAX™ vaccination.
A Study of AbGn-168H in Patients With Steroid Refractory Acute Graft-vs-Host Disease After Donor Stem Cell Transplant

This study is to establish the safety, determine if there is an improvement in steroid refractory acute graft-vs-host disease (aGvHD) compared to historical cohorts, and determine the changes of aGvHD-associated T-cell clones in patients with steroid-refractory aGVHD following allogeneic hematopoietic cell transplantation administered AbGn-168H once weekly for 4 weeks.

AbGn-168H is a humanized monoclonal antibody. This is a dose escalation study using a modified toxicity probability interval method. AbGn-168H will be administered intravenously (IV) once weekly for four weeks, in patients with steroid refractory aGVHD following hematopoietic cell transplant (HCT). After completion of study treatment, patients are followed up for 90 days.The primary objective of this study is to establish the safety, and the secondary objectives of this study are to determine if there is an improvement in disease response at 3 months after diagnosis of steroid refractory aGVHD compared to historical cohorts and to determine the changes in frequency and/or phenotype of aGVHD-associated T cell clones in response to AbGn-168H therapy.

Eligibility Criteria:
Inclusion Criteria:

1. Diagnosis of skin, gut and/or liver steroid-refractory GVHD by clinical assessment of treating physician following allogeneic HCT. Patients who fail to respond to steroids by 7 days are considered steroid-refractory
2. Previously-treated with any conditioning regimen and any GVHD immune suppression prophylaxis and formulation of steroids, except as noted in Exclusion Criteria #2.
3. AbGn-168H (neihulizumab) therapy can begin not more than 14 days after diagnosis of aGvHD
4. Karnofsky Performance Status (KPS) \> 50%
5. No evidence of HCT graft failure or multi-organ failure
6. Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria:

1. Uncontrolled infections not responsive to antimicrobial therapy requiring intensive critical care
2. Progressive malignant disease, including post-transplant lymphoproliferative disease unresponsive to therapy
3. Treatment with investigational GVHD prophylactic agents (eg, CCR5 inhibitors; lenalidomide; and/or bortezomib) within the 7 days prior to the 1st dose of neihulizumab
4. Treatment with other investigational agents within the prior 7 days prior to the 1st dose of AbGn-168H (neihulizumab)
5. CMV PCR \> 500 copies/mL or evidence of end-organ damage due to CMV
6. Pregnant or nursing
7. HIV positivity (NOTE: patients positive for hepatitis B or hepatitis C are not excluded, and may be evaluated on a case-by-case basis)
8. Renal clearance CCR \< 40 mL/min
Effect of Niacin in the Lipoprotein (a) Concentration

Objectives.

* To evaluate the absolute and relative Lp(a) lowering effect of 1g/20 mg and 2 g/40 mg day of Niacin/Laropiprant in subjects with normal Lp(a) (\< 30 mg/dL), high Lp(a) (30-60 mg/dL) and very high Lp(a) (\> 60 mg/dL).
* To evaluate the absolute and relative Lp(a) lowering effect of 1g/20 mg and 2 g/40 mg day of Niacin/Laropiprant depending on the number of kringle IV-2 repeated copies on the apo(a) gene. 2.1.1 Hypotheses.
* The Lp(a) lowering effect of niacin is dependent of the pre-treatment Lp(a) concentration, with higher absolute and relative reduction in Lp(a) in subjects with hyperlipoproteinemia(a).
* Lp(a) size, throughout modifying hepatic synthesis of apo(a), is a major factor related to the lowering effect variability of niacin in human.

Open-label 12-week study, 1g/20 mg day of Niacin/Laropiprant for 4-weeks followed by 8 additional weeks of 2 g/40 mg day. Subjects with normal Lp(a) will be use as comparative group for the other two groups, so no placebo group is required is this study.

Subjects: volunteers from the Lipid Clinic of Hospital Universitario Miguel Servet of Zaragoza, Spain. Subjects were selected according to their previously determined Lp(a)concentration. All volunteers before any study procedure will have to give written inform consent to a protocol previously approved for the Ethical Committees of our institutions.

Biochemical determinations: lipids: total cholesterol and triglycerides; lipoproteins: HDL-cholesterol, Lp(a); apolipoproteins: Apo A1 and apo B and safety biochemical parameters (glucose, uric acid, creatinine, liver and muscle enzymes will be measured at baseline and at the end of the two treatment periods (weeks 4 and 8).

An adverse experience questionnaire will be done in each visit. Genetic analysis: apo(a) genetic polymorphism responsible of the Lp(a) size variability will be analyzed by a PCR-based methodology (Lanktree et al. J Lipid Res 2009; 50: 768-72 ).

Eligibility Criteria:
Inclusion Criteria:

1. Age \>18 and \< 80 years
2. LDL cholesterol between 70 and 190 mg/dL
3. Triglycerides \< 500 mg/dL
4. At least 2 Lp(a) determinations previous to the beginning of the study without differences \>20% or \> 20 mg/dL.
5. No lipid lowering therapy or on stable doses in the last 3 months

Exclusion Criteria:

1. Liver disease or liver enzymes \>2 times higher than reference values
2. Creatinine \> 2 mg/dL
3. Active peptic ulcer
4. Clinical gout in the last year
5. Uncontrolled diabetes (HbA1c \>8%)
6. Enrolment in other drug clinical trial in the previous 3 months.
Blood Pressure After PREeclampsia/HELLP by SELF Monitoring Study

RATIONALE:

Women with a previous history of preeclampsia (PE)/HELLP have a twofold higher risk of cardiovascular disease (CVD) and a fourfold increased risk to develop hypertension at a relative young age. In the latest 2016 ESC guidelines 'CVD prevention', previous PE has been acknowledged as a serious CVD risk factor in women. In clinical practice, however, these women are still underappreciated for their CVD risk. It has not been established yet how adequate lifelong prevention in these potentially high risk women can optimally be achieved from their childbearing years onwards.

OBJECTIVE:

To evaluate feasibility, usability, acceptance and potential obstacles of blood pressure (BP) home monitoring in high-risk women, aged 40-60 years, after a previous PE in pregnancy. In addition to evaluate the effectiveness of BP home monitoring on the occurrence of hypertension, the efficacy of BP treatment, quality of life (QOL), health-related symptoms, work ability and life-style behaviour.

STUDY DESIGN:

Randomized, open label feasibility study to compare eHealth guided self management of BP monitoring and lifestyle advice with 'usual care'.

STUDY POPULATION:

Approximately 200 women, aged ≥ 40 and ≤ 60 years, with a previous PE in pregnancy will be invited to participate (both early and late PE). Patients will be recruited from the departments of obstetrics/gynaecology in 6 centres in the Euregio Rhein-Waal and from the department of cardiology Radboudumc.

INTERVENTION:

eHealth guided measurements of BP at regular intervals and recurrent lifestyle advice (intervention-group) will be compared with "usual care".

PRIMARY OUTCOME:

Feasibility, usability, acceptance and potential obstacles of BP home monitoring in high-risk women after a previous PE in pregnancy at 1 year of follow-up.


Eligibility Criteria:
Inclusion Criteria:

* previous preeclampsia/HELLP syndrome, defined as diastolic BP≥90 mmHg with proteinuria ≥ 0.3gram/24 h, during one or more pregnancies, more than 1 year ago.
* 'early' or 'late' PE/HELLP
* age ≥40 and ≤ 60 years

Exclusion Criteria:

* pregnant women or women wishing for future pregnancie(s)
* inability to perform self BP measurements
* not having a smartphone (Apple or Android)
* already having regular hypertension control by GP or medical specialist
* previous cardiovascular events, such as myocardial infarction , stroke, heart failure and symptomatic ischemic heart disease
Network Intervention to Prevent Vaping

Rates of adolescent vaping are increasing rapidly. Current high school student use of electronic vaping products (EVPs) rose from 1.5% in 2011 to 20.8% in 2018 - an increase from 220,000 to 3.05 million adolescent users. Effective, school-based interventions are urgently needed to protect adolescents from initiating or continuing use of electronic vaping products (EVPs). This study leverages a state-supported prevention initiative to test the effectiveness of a promising intervention that trains 8th-9th grade student peer leaders to deliver school-wide vaping prevention campaigns with ongoing adult mentoring. If study hypotheses are supported, the study will provide the first evidence of a school-based preventive intervention that reduces adolescent vaping behaviors, as well as insight into how peer communications can be harnessed to prevent vaping.

Above the Influence of Vaping (ATI-V) trains peer nominated 8th-9th grade Peer Leader, and adult advisors. Peer Leaders learn skills and implement school-wide prevention campaigns informed by communication science.New York State has provided funds to support schools to implement ATI-V but no funds for efficacy research. With support from New York State and a strong team of investigators, our project has two aims:

Aim 1. Efficacy. The primary aim of this study is to determine ATI-V impact in preventing vaping use (past 30 days any vaping, nicotine vaping, and regular use). Using an RCT design, 20 schools will be assigned to (a) immediate ATI-V, or (b) wait-list for ATI-V training after 24 months. Approximately 3,800 8th graders will be enrolled and followed for assessments in fall 8th grade, spring 8th grade, spring 9th grade, and mid-year 10th grade. We will test for which students ATI-V is most effective and in what school contexts (school climate).

Aim 2. Mechanism. The second aim of this study to test the hypothesized mechanisms of ATI-V impact. To accomplish this aim we will conduct statistical analyses of a mediation model to determine (a) whether ATI-V improves students' perceptions that vaping is unacceptable to their peers (anti-vaping norms), connections to supportive adults to address EVP concerns, and social influence of non-vaping students; and (b) whether the impact of ATI-V on reduced vaping behavior is mediated by these improvements.

Eligibility Criteria:
Inclusion Criteria:

* current 8th grade student of study school at start of school participation
* No person shall be excluded from participation, denied benefits, or discriminated against because of race, color, national origin, sexual orientation, sex, gender identity, age, or ability (i.e., special education status).

Exclusion Criteria:

* non-English speaking students
A Study Comparing Use of Manual and Power Bone Marrow Aspiration and Biopsy Devices in Children

The purpose of this study is to compare use of a powered bone marrow aspiration and biopsy device (OnControl) to use of a standard manual bone marrow aspiration and biopsy device to obtain bone marrow biopsy specimens in children. The hypothesis is that use of the powered device will improve the quantity and quality of bone marrow biopsy specimens when compared to the manual device.

The purpose of this randomized, prospective trial is to compare use of the standard manual bone marrow aspiration and biopsy device to use of the powered bone marrow aspiration and biopsy device (OnControl) to obtain bone marrow biopsy specimens in pediatric patients.

Eligibility Criteria:
Inclusion Criteria:

* Informed consent and assent for minors over the age of seven years must be obtained before any trial-related activities are initiated
* Patients requiring bone marrow aspirations and/or biopsies as part of standard diagnosis and treatment
* Treated at Christus Santa Rosa Children's hospital
* Age 2 to 18 years
* Male or female
* Supportive family with willingness to participate in completing questionnaires
* English or Spanish primary language

Exclusion Criteria:

* Patients who are pregnant
* Patients with hemorrhagic disorders such as congenital coagulation factor deficiencies disseminated intravascular coagulation and concomitant use of anticoagulants
* Patients with a skin infection or recent radiation therapy at the sampling site
* Patients with bone disorders such as osteomyelitis or osteogenesis imperfecta