cyrilzakka/clinical-trials huggingface dataset:
Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients

This study is to compare the efficacy and safety of defibrotide prophylaxis in addition to best supportive care versus best supportive care alone in the prevention of hepatic veno- occlusive disease (VOD) in adult and pediatric patients undergoing hematopoietic stem cell transplant who are at high risk or very high risk of developing VOD.


Eligibility Criteria:
Inclusion Criteria:

1. Patient must be above the age of 1 month as of the start date of study treatment.
2. Patient must be scheduled to undergo allogeneic hematopoietic stem cell transplant (HSCT) (adults or pediatric patients) or autologous HSCT (pediatric patients only) and be at high risk or very high risk of developing veno-occlusive disease (VOD).
3. Female patients (and female partners of male patients) of childbearing potential who are sexually active must agree to use a highly effective method of contraception with their partners during exposure to defibrotide and for 1 week after the last dose of defibrotide.
4. Adult patients must be able to understand and sign a written informed consent. For minor patients, the parent/legal guardian or representative must be able to understand and sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.

Exclusion Criteria:

1. Patient has hemodynamic instability within 24 hours before the start of study treatment.
2. Patient has acute bleeding that is clinically significant within 24 hours before the start of study treatment.
3. Patient used any medication that increases the risk of bleeding within 24 hours before the start of study treatment.
4. Patient is using or plans to use an investigational agent for the prevention or treatment of VOD.
5. Patient, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
6. Patient or parent/legal guardian or representative has a psychiatric illness that would prevent the patient or parent/legal guardian or representative from giving informed consent and/or assent.
7. Patient has a serious active disease or co-morbid medical condition, as judged by the investigator, which would interfere with the conduct of this study.
8. Patient is pregnant or lactating and does not agree to stop breastfeeding.
9. Patient has a known history of hypersensitivity to defibrotide or any of the excipients.
10. Patient or parent/legal guardian or representative lacks the full mental capacity to understand and sign a written informed consent.
11. Patient is receiving or plans to receive other investigational therapy during study.
Transfusion of Pathogen Reduced Cryoprecipitated Fibrinogen to Expedite Product Availability in Perioperative Bleeding

The goal of this quality improvement study is to compare pathogen-reduced cryoprecipitate with traditional cryoprecipitate in liver transplant and cardiovascular patients. The investigators hypothesize that by having immediate access to a readily available thawed blood product that replaces fibrinogen (the main substrate of a blood clot), early bleeding can be treated before it escalates into uncontrolled hemorrhage, and therefore additional blood products, like platelets, plasma and red blood cells can be avoided.

Participants will be given one of the two FDA-approved blood products.

Immediately replacing fibrinogen in perioperative bleeding patients with acquired fibrinogen deficiency improves outcomes. The product that is primarily used for fibrinogen replacement in the US, cryoprecipitate (cryo), must be stored frozen and expires six hours after thawing, resulting in a delay in transfusion of approximately 50 minutes from the time it is ordered, as well as unnecessary transfusion of more readily available but not indicated blood components that are transfused while the patients waits for cryo . A modified version of the product, pathogen reduced (PR) cryo, is now FDA approved and can be thawed and stored for 5 days, allowing the product to be available immediately when needed. In this quality improvement study, the investigators will compare the effect that readily available, pre-thawed PR cryo has on transfusion practice in cardiovascular and liver transplant patients who receive PR cryo versus those who receive traditional cryo by randomizing cryo transfusions in the blood bank by month to all cryo or all PR cryo. All clinical decisions, including the need for cryo, and laboratory testing will occur per standard of care.

Eligibility Criteria:
Inclusion Criteria:

1. Adult patients undergoing cardiovascular surgery or liver transplant who receive cryo during surgery during the two year study period.
2. Cardiovascular surgery includes the following procedures:

   1. coronary artery bypass grafting
   2. valve repair or replacement
   3. open thoracic aortic and thoracoabdominal aortic surgery
   4. atrial or ventricular septal defects
   5. ventricular assist device implantation or revision
   6. or any combination of the above.

Exclusion Criteria:

1. Patients who do not receive any cryo product in the OR
2. Patients who are not cardiovascular surgery or liver transplant patients
3. Cardiac transplantation surgery
4. Patients who receive a product in error within either the cryo time period or the PR cryo time period. For example, PR cryo during a cryo month or cryo during a PR cryo time month.
5. Patients who receive less than 1 pool (5 units) of cryo
6. Pediatric patients (less than 18 years of age).
7. Patients who received both PR cryo and traditional cryo
8. Pregnant women
A Study to Evaluate the Efficacy of Lastacaft® Compared to Pataday™ and Placebo in Patients With Acute Allergic Conjunctivitis

This study will evaluate the efficacy of Lastacaft® (Alcaftadine 0.25%) and Pataday™ (Olopatadine 0.2%) as compared to each other and to placebo in the prevention of ocular itching associated with allergic conjunctivitis.


Eligibility Criteria:
Inclusion Criteria:

* History of ocular allergies within the past 24 months.
* Able to avoid wearing contact lenses for at least 72 hours prior to and during the study trial period.

Exclusion Criteria:

* Any presence of active ocular infection or history of an ocular herpetic infection.
* Ocular surgery within 3 months prior to the first visit or refractive surgery within the past 6 months.
* Have any planned surgery during the study or 30 days after the study.
Non Transplantable Recurrence After Percutaneous Thermal Ablation of HCC

Percutaneous thermal ablation (PTA), resection and liver transplantation (LT) are the standard curative options for hepatocellular carcinoma (HCC). LT yields the best long-term outcomes but is limited by graft shortage. Thus, patients with ≤3cm HCC are mainly treated by PTA although recurrence is frequent and may occur outside transplant criteria. However, data on non transplantable recurrence (NTR) following PTA are lacking. Therefore, the investigators investigated the incidence and predictors of NTR among transplantable patients with ≤3cm HCC(s) treated by PTA, in order to stratify them according to their NTR risk and to improve treatment allocation.

Consecutive patients undergoing PTA for HCC between January 2015 and December 2020 were included. Data were collected from our prospective database. This study was approved by our institutional review board (NCT03428321 \[www.clinicaltrials.gov\]) and written informed consent was obtained from all patients. Inclusion criteria were: HCC diagnosed by histopathology or by EASL imaging criteria, HCC ≤30mm, 1 - 3 tumor nodules, follow-up\<3 months, no prior or combined treatment with intra-arterial therapy, potentially transplantable patient (ie, ≤70yr, AFP-score≤2 (7), no macroscopic portal vein invasion or extra-hepatic metastasis, no major comorbidity precluding LT).

Patient's and liver's characteristics were collected including: age, sex, HCC-naïve status, body mass index (BMI), diabetes mellitus, liver steatosis, cirrhosis, cause for hepatopathy, AFP serum level, Child-Pugh, MELD and Albumin-Bilirubin (ALBI) scores and preablative biological data.

Occurrence of NTR after PTA will be analyzed in a competing risks framework, with transplantation and death as competing events. Covariates associated with NTR were analyzed using Fine-Gray proportional sub-distribution hazards models.

Recurrence-free survival (RFS) will be defined as the time from PTA until the first recurrence, death, or last follow-up. OS will be defined as the interval between PTA and death (any cause) or last follow-up. Survival curves will be estimated using the Kaplan-Meier method and compared with the log-rank test.

Eligibility Criteria:
Inclusion criteria :

* HCC diagnosed by histopathology or by EASL imaging criteria
* HCC ≤30mm, 1 - 3 tumor nodules, follow-up\<3 months
* no prior or combined treatment with intra-arterial therapy
* potentially transplantable patient (ie, ≤70yr, AFP-score≤2 (7), no macroscopic portal vein invasion or extra-hepatic metastasis, no major comorbidity precluding LT).

Exclusion criteria:

- age \< 18 years
A Study of the Pharmacology of Tamiflu in Pregnancy

The purpose of this study is to determine the pharmacokinetics (PK) of the anti-flu medication, Tamiflu during pregnancy and to determine to what extent pregnancy may affect the currently recommended dosage of this medication.

The emergence of the novel H1N1 influenza virus has raised concern among public health officials and the public alike. Although initial reports suggested that the risk of serious disease or death was less than initially feared, continued experience suggests that the disease will have a major impact on the public's health. To address this urgent public health challenge, the centers of the Obstetric-Fetal Pharmacology Research Units Network (OPRU) will recruit a total of 75 pregnant women (25 per trimester with singletons pregnancies) who are receiving oseltamivir for supposed influenza infection. When possible, we will recruit women with twin gestation to determine if fetal number affects the drugs pharmacokinetics. We do not anticipated a significant number of multi-fetal gestations (\< 10) but even limited data might prove useful in this population given the tremendous change in renal drug clearance that is normally seen in this group of women. An additional 25 healthy non-pregnant women will be studied to define pregnancy- related changes in the PK of oseltamivir and the carboxylate.

We will study each woman once during therapy, performing steady state pharmacokinetic analyses. This study will be performed after 3 days of oral therapy to optimize the possibility that steady state concentrations have been achieved.

We will:

1. collect biological fluids (plasma and urine) to further characterize the pharmacology of the drug;
2. collect a check swab for DNA to allow study of the impact of single nucleotide polymorphisms on drug handing and disease expression;
3. develop an assay or partner with others to develop an assay for oseltamivir and the primary metabolite, oseltamivir carboxylate;
4. compare the pharmacokinetics of oseltamivir and its metabolite in each trimester of pregnancy to evaluate whether gestational age has an impact on the drug's pharmacokinetics;
5. assess the impact of fetal number on the drug's pharmacokinetics;
6. ask all participants about GI tolerance to the drug and any side effects they may be experiencing, especially related to mood and psychiatric well being; and
7. on all pregnant subjects studied, when possible, we will obtain at delivery maternal and umbilical cord blood to evaluate placental transport especially in those who deliver proximate (within 3 days) to drug treatment.

Eligibility Criteria:
Inclusion Criteria:

1. Taking or about to start oral oseltamivir for presumed influenza viral infection or for prophylaxis
2. Able to give informed consent
3. Pregnant female with singleton or multifetal gestation or healthy non-pregnant female (ancillary study)

Exclusion Criteria:

1. Unable to take medication orally
2. Renal failure - creatinine \> 1.5
3. Hct \<28
Clinical Study of CM338 in the Treatment of Immunoglobulin A Nephropathy

This study is a multicenter, randomized phase II clinical study to evaluate Efficacy and safety, while observing pharmacokinetic profiles, pharmacodynamic effects, and immunogenicity of CM338 in subjects with Immunoglobulin A(IgA) nephropathy.

In 1968, immunoglobulin A nephropathy (IgAN) was developed by French pathologist Dr. Jean Berger and his colleague Dr. Nicole Hinglais that what is described as glomerular "immunoglobulin A (IgA)-immune globule" - immune globule Protein G (IgG) intercapillary deposition" of kidney disease, the main pathological feature of which is on the glomerulus IgA deposition, usually accompanied by local cell proliferation and stromal dilation. Since there is not enough specificity and spirit Biomarkers of sensitivity, the only way to confirm the diagnosis is renal biopsy.

Eligibility Criteria:
Inclusion Criteria:

* Male or female aged 18-75.
* Understand the nature of the study and sign the Informed Consent Form voluntarily.
* Take effective contraception measures throughout the study period.

Exclusion Criteria:

* Used other investigational drugs within 30 days before the first study administration.
* With previous history of Human immunodeficiency virus(HIV) infection.
* Treponema pallidum antibody positive in screening period.
* May have active Mycobacterium tuberculosis infection.
* Major surgery is planned during the study.
* Other reasons the investigator believes that the subject is not suitable to participate in this study.
Giant Cell Arteritis - Optimization of Diagnostics

Giant cell arteritis - Optimization of diagnostics

A prospective study. Investigating the use of ultrasound, temporal artery biopsy, biomarkers and 18F-fluorodeoxyglucose positron emission tomography-computed tomography for establishing a diagnosis of giant cell (temporal) arteritis.

Eligibility Criteria:
Inclusion Criteria:

Clinical suspicion of giant cell arteritis

Meets the American College of Rheumatology Criteria:

Age at disease onset =\> 50 years New headache Temporal artery abnormality Elevated erythrocyte sedimentation rate \>=50 mm/hour Abnomal artery biopsy

Exclusion Criteria:

Active cancer Use of steroid for more than a week (the prior 6 months)
High Risk Myelodysplasia Treated by Azacytidine : Genetic and Epigenetic (MYRAGE)

Myelodysplastic syndromes (MDS) are the most frequent myeloid neoplasms in Western Countries.They mainly affect patients aged 65 years or older. This is a very heterogenous group of diseases, which prognosis is evaluated with International Prognosis Scoring System. High risk MDS present with high frequency of transformation into acute myeloid leukemia. Treatment of high risk MDS often is based on hypomethylating agents, such as 5'-azacytidine (Azacytidine), with a complete response in approximativel 20% of cases..

This treatment is based on 4-week cycles, with daily injection during the first week and rest during the 3 next weeks of the cycle.

Azacytidine efficacy is commonly evaluated with clinical and biological parameters determined by the International Working Group 2006. These parameters are usually evaluated after at least 6 cycles of treatments.

There is a response with Azacytidine treatment in 60% of cases, including 40% of partial responses and 20% of complete responses. In 40% of patients, there is no response, which means that the disases is stable or in progression under therapy.

In this regard, early evaluation of treatment response is an issue. We want to improve our knowledge about early response criteria in Azacytidine-treated high-risk MDS, focusing on SMD with excess blasts, which represent 30 to 40% of total MDS.

Then, the investigator team want to compare DNA methylation profile at diagnosis and after 3 cycles of Azacytidine treatment.

Main objective :

Identify DNA methylation profiles related to response to Azacytidine therapy, after only 3 cycles of treatment, in high risk MDS with excess blasts.

Secondary objective :

Identify at diagnosis DNA methylation profiles that are predicitive of response to Azacytidin, in high risk MDS with excess blasts.


Eligibility Criteria:
Pre-inclusion Criteria:

* patient benefiting from social welfcare
* patient followed at the University Hospital of Nancy
* patient aged 18 years or older
* patient informed on research organization and having signed an informed pre-inclusion consent
* No personal history of myelodysplastic syndrome
* clinical exam adapted to research
* one or more blood cytopenia

Inclusion Criteria:

* patient benefiting from social welfcare
* patient followed at the University Hospital of Nancy
* patient aged 18 years or older
* patient informed on research organization and having signed an informed inclusion consent
* definitive diagnosis of high risk myelodysplastic syndrome with excess blasts
* eligibility to an Azacytidine therapy as first-line treatment

Exclusion Criteria:

* personal history or current other cancer
* immediate acute myeloid leukemia
* personal history of demethylation treatment
* pregnant or breast feeding women
* life-theatening condition
* guardianship
* imprisoned patients
Phase I Study of High-Dose Cyclophosphamide and Total Body Irradiation With T Lymphocyte-Depleted Autologous Peripheral Blood Stem Cell or Bone Marrow Rescue in Patients With Multiple Sclerosis

OBJECTIVES:

I. Determine the efficacy, in terms of disease progression, of high-dose cyclophosphamide and total body irradiation with T lymphocyte-depleted autologous peripheral blood stem cell or bone marrow rescue in patients with multiple sclerosis.

PROTOCOL OUTLINE: Following an induction course of cyclophosphamide IV, patients receive filgrastim (G-CSF) subcutaneously (SC) daily until the completion of peripheral blood stem cell (PBSC) harvesting. PBSCs are collected over several days. Patients who do not mobilize sufficient cells undergo bone marrow harvest. Harvested PBSCs or bone marrow then undergo T-lymphocyte depletion.

Patients receive cyclophosphamide IV over 1 hour on days -6 and -5 and methylprednisolone IV daily on days -4 to -1. Patients also undergo total body irradiation twice a day on days -4 to -1. Lymphocyte-depleted PBSCs or bone marrow is reinfused on day 0. Patients receive G-CSF SC daily beginning on day 0 and continuing until blood counts have recovered for 3 days.

Patients are followed at 1, 2, 3, 6, and 12 months and then annually for 5 years.

Eligibility Criteria:
PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Diagnosis of multiple sclerosis Kurtzke score of 4.0-7.5 Increase of 1.0 point over the past 12 months More than 3 relapses in 24 months despite conventional disease modifying therapy Failure to stabilize active clinical progression with a 3-day regimen of methylprednisolone IV

--Prior/Concurrent Therapy--

Chemotherapy: No prior cladribine

Radiotherapy: No prior radiotherapy to greater than 10 cm2 of lung tissue No prior craniospinal irradiation No prior total lymphoid irradiation

--Patient Characteristics--

Hematopoietic: Absolute neutrophil count at least 1,000/mm3 Platelet count at least 100,000/mm3 Hemoglobin at least 9.0 g/dL

Hepatic: Hepatitis B antigen negative Bilirubin no greater than 2.0 mg/dL Transaminases no greater than 2 times upper limit of normal

Renal: Creatinine no greater than 2.0 mg/dL Cardiovascular: No history of coronary artery disease Resting LVEF at least 45%

Pulmonary: FEV1/FVC at least 75% predicted DLCO at least 50% predicted

Other:

* Not pregnant
* Negative pregnancy test
* Fertile patients must use effective contraception
* HIV negative
* No uncontrolled diabetes mellitus
* No other concurrent medical illness that would preclude study
* No concurrent psychiatric illness or mental deficiency that would preclude study
* No prior malignancy except localized basal cell or squamous cell skin cancer (malignancies judged to be cured by local surgical therapy such as head and neck cancer or stage I breast cancer, are considered on an individual basis)
* No presence of body shrapnel, metal fragments, or unremovable devices such as a pacemaker or aneurysm clip
Using Shared Decision Making to Improve Family Media Use Planning

Investigators will conduct a pilot randomized controlled trial assessing the efficacy and feasibility of the newly developed Family Media Check-In (FMC).

The clinical trial portion of this study will enroll 150 families (child age 11-17, and at least one parent/guardian). Stratified randomization will be used to assign families to the intervention (FMC) or control (delayed FMC) condition. Families assigned to the intervention condition will be asked to complete the FMC within 2 weeks of randomization and will be sent a second survey one week after completion. Participants in the control condition will complete a condition-specific subset of these questions three weeks after baseline. Participants in both conditions will complete a follow-up survey two months after their second survey.

The primary outcomes will be measured at the first follow-up period and will be related to screen media planning and decision making. Two-month follow-up surveys will explore intervention impact on screen media practices.

Prior to the clinical trial portion of the study, the study team conducted interviews/focus groups, participatory design sessions, and usability testing sessions with a diverse group of families to refine the concept for the intervention and to iteratively develop the intervention itself. Changes in the behavioral targets and components of the intervention and outcomes for the evaluation (relative to the original grant proposal) were driven by family-expressed needs and priorities.

Eligibility Criteria:
Inclusion Criteria:

* Children between the ages of 11 and 17
* Parent/guardian of a child between the ages of 11 and 17
* English speaking

Exclusion Criteria:

* Parent/guardian or child does not want to participate (i.e., dyads only)
* Non-English speaking