cyrilzakka/clinical-trials huggingface dataset:
The Relationship Between Brain MRI Phenotypes, Genes and Cognitive Outcome in CHD Adults

The main purpose of this proposal is to perform novel MRI analyses to determine the brain organizational changes associated with altered executive function and the modulating role of variants in neuroresilience and hypoxia response genes in adults with d-transposition of the great arteries (d-TGA).

Aim 1 is structured to determine the relationship between sulcal patterns(measured by MRI) and executive function in adults with d-TGA and if this relationship is modified by (a) presence of neuroresilience gene ApoE ε2 or ε4 alleles, or (b) damaging variants in hypoxia response genes.

Aim 2 is designed to determine the relationship between structural connectivity using rich club (measured by MRI) and executive function in adults with d-TGA and if this relationship is modified by (a) presence of neuroresilience gene ApoE ε2 or ε4 alleles or (b) damaging variants in hypoxia response genes.

In Aim 3, the investigators will determine the relationship between functional connectivity using rich club (measured by MRI) and executive function in adults with d-TGA and if this relationship is modified by (a) presence of neuroresilience gene ApoE ε2or ε4 alleles or (b) damaging variants in hypoxia response genes.

Eligibility Criteria:
Inclusion Criteria:

* Persons who participated in the Boston circulatory arrest, pH and Hematocrit studies at Boston Childrens Hospital as children (n\~300).
* Normally-developed age- and sex-matched young adults who are able to provide informed consent to undergo the MRI portion of the study and limited neuropsychological evaluation (IQ assessment as well as neuropsychological tests for which normative references are not available).

Exclusion Criteria:

* For controls - inability to complete the MRI (implanted metal, claustrophobia, personal history of mental illness, brain injury, prior brain intervention).
* Intellectual impairment precluding completion of the study questionnaires independently
* Unable to speak and read English fluently
Gene Therapy for Wiskott-Aldrich Syndrome

This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.

Wiskott-Aldrich Syndrome (WAS) is an X-linked primary immunodeficiency caused by mutations in the WAS gene which encodes the WAS protein (WASP), a cytoskeletal regulator which is expressed exclusively in hematopoietic cells.

Eligibility Criteria:
Inclusion Criteria:

1. Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:

   * Severe WAS mutation
   * Absence of WASP expression
   * Severe clinical score (Zhu clinical score ≥ 3
2. No HLA-identical sibling donor
3. Negative search for a matched unrelated donor (10/10) or an adequate unrelated cord blood donor (5-6/6) within 4-6 months

   * Patients of \> 5 years of age who are not candidate to unrelated allogeneic transplant based on clinical conditions.
4. Parental/guardian/patient signed informed consent.

Exclusion Criteria:

1. Patients positive for HIV-infection.
2. Patients affected by neoplasia.
3. Patients with cytogenetic alterations typical of MDS/AML.
4. Patients with end-organ functions or any other severe disease which, in the judgement of the investigator, would make the patient inappropriate for entry into this study.
5. Patients who underwent an allogeneic haematopoietic stem cell transplantation in the previous 6 months.
6. Patients who underwent an allogeneic haematopoietic stem cell transplantation with evidence of residual cells of donor origin.
A Clinical Trial of Lurasidone in Treatment of Schizophrenia

This is a randomized, double-blind, double-dummy, parallel- controlled, adjustable dose, non-inferiority and multicentre study designed to evaluate the efficacy and safety of lurasidone on schizophrenia for 6 weeks treatment, and to compare with risperidone.


Eligibility Criteria:
Inclusion Criteria:

* Provide written informed consent and aged between 18 and 65 years of age.
* Meets DSM-IV-TR criteria for a primary diagnosis of schizophrenia, had a PANSS total score ≥ 70 and ≤ 120 at Screening and Baseline, and a score ≥ 4 on the CGI-S at Screening and Baseline.
* Not pregnant, if of reproductive potential agrees to use adequate and reliable contraception for duration of study.
* Able and agrees to remain off prior antipsychotic medication for the duration of study.
* Willing and able to comply with the protocol, including the inpatient requirements and outpatient visits.

Exclusion Criteria:

* Considered by the investigator to be at imminent risk of suicide or injury to self, others or property.
* Any chronic organic disease of the CNS(other than schizophrenia)
* Subjects are participating or participated in other clinical studies including marketed drugs or medical devices within 30 days before signing the informed consent form.
* Clinically significant or history of alcohol abuse/alcoholism or drug abuse/dependence within the last 6 months.
LUMINA Phase III Study Assessing the Efficacy and Safety of Intravitreal Injections of 440 ug DE-109 Sirolimus for the Treatment of Active, Non-Infectious Uveitis of the Posterior Segment of the Eye

This is a Phase III study to assess the efficacy and safety of DE-109 440 µg every 2 months in subjects with active, non-infectious uveitis of the posterior segment of the eye (NIU-PS).

There is a 6-month, single-arm, open-label period after completion of the 6-month double- masked, controlled period allows the evaluation of the efficacy and safety of intravitreal injection of DE-109 440 µg every 2 months for longer duration than appropriate for a placebo or sham control.


Eligibility Criteria:
Inclusion Criteria:

Non-Infectious Active Uveitis of the Posterior Segment

Exclusion Criteria:

Females who are pregnant, nursing, or planning a pregnancy Confirmed or suspected infectious uveitis
Pharmacokinetics, Efficacy and Safety Study of IMMUNATE SD (Human Plasma-Derived Coagulation Factor VIII Concentrate) in Hemophilia A Patients

The purpose of this study is to evaluate whether IMMUNATE S/D is effective and safe in the treatment of hemophilia A patients. The study consists of 3 parts: Part 1 is a pharmacokinetic comparison of IMMUNATE S/D and its predecessor IMMUNATE. Part 2 is an evaluation of efficacy and safety of IMMUNATE S/D. Part 3 is a pharmacokinetic study of IMMUNATE S/D.


Eligibility Criteria:
Inclusion Criteria:

- Plasma factor VIII level as follows: for Parts 1 \& 3: Subjects with severe hemophilia A (plasma baseline factor VIII level \<= 1% measured at time of screening) for Part 2: Subjects with severe (plasma baseline factor VIII level \<= 1% measured at time of screening) or moderately severe hemophilia A (plasma baseline factor VIII level \<= 2% measured at time of screening)

* Males \>= 12 but \<= 65 years of age
* \>= 35 kg body weight
* Previously treated with factor VIII concentrate(s) for a minimum of 150 exposure days (as documented in the subject's medical history)
* Evidence of a protective titer to HAV and HBV at the time of screening
* Immunocompetent as defined by a CD4+ lymphocyte count \>400/mm3 and an absolute neutrophil count (ANC) \>1500
* Signed informed consent obtained from subject or legally authorized representative

Exclusion Criteria:

* Documented history of inhibitor to factor VIII with a titer \>= 0.8 BU
* Current evidence of inhibitor to factor VIII with a titer \>= 0.8 BU, measured at the time of screening
* Abnormal renal function (serum creatinine \> 1.5 mg/dL)
* HIV-seropositive individuals with any of the following at the time of screening:
* CD4+ lymphocyte count \>400/mm3
* AIDS-related complex
* symptomatic AIDS Note: HIV-seropositive subjects with an absolute CD4+ lymphocyte count \> 400/mm3 are eligible to participate. HIV-seropositive subjects receiving highly active anti-retroviral therapy (HAART) regimens are eligible for enrollment if they are not excluded by the above criteria
* Active hepatic disease (ALT and AST levels \> 5 times the upper limit of normal)
* Clinical or laboratory evidence of hepatic cirrhosis including (but not limited to) a recent and persistent INR (international normalized ratio) \> 1.4, the presence of splenomegaly and/or significant spider angiomata on physical exam, and/or a history of esophageal hemorrhage or documented esophageal varices
* Known hypersensitivity to IMMUNATE
* The subject is currently participating in another investigational drug study, or has participated in any clinical study involving an investigational drug within 30 days of study entry
* The subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug other than anti-retroviral chemotherapy (e.g., a-interferon, steroids at a dose greater than 10 mg/day)
* The subject is identified by the investigator as being unable or unwilling to perform study procedures
Prediction Model for Anemia After Bariatric Surgery

To develop and validate a prediction model for estimating the short and long-term risk of anemia after bariatric surgery.

This study aims to develop and validate a prediction model for estimating the probability of anemia for patients with obesity one, three, and five years after sleeve gastrectomy and Roux-en-Y gastric bypass, using preoperative clinical and laboratory data.

Eligibility Criteria:
Inclusion Criteria:

* aged between 16-70 years;
* body mass index (BMI) ≥ 27.5 kg/m2;
* complete preoperative and follow-up data.

Exclusion Criteria:

* vegetarian;
* other bariatric procedures;
* renal failure at baseline;
* postoperative bleeding (it was defined as a drop in hemoglobin levels \> 3 g/dL or a confirmed blood loss requiring treatment after surgery);
* incomplete preoperative and follow-up data.
Patient Positions for Locating the Cricothyroid Membrane

Cricothyroidotomy is an emergency rescue technique to secure the airway in a 'Can't intubate, Can't Oxygenate' (CICO) situation. The cricothyroid membrane (CTM) is the target site of cricothyroidotomy, and accurate identification of the CTM is essential for successful cricothyroidotomy. In this study, the investigators compare patient positions (neck extension vs. modified ramped position) in terms of the success rate for identifying the CTM in obese patients.


Eligibility Criteria:
Inclusion Criteria:

* body mass index \>30 kg/m2

Exclusion Criteria:

* anatomical abnormalities or pathologies of the neck or larynx
* a history of neck surgery.
Skin Tolerance Study of Betamethasone Creams in Atopic Eczema and the Preventative Properties of a Moisturiser

Topical steroid creams as well as moisturizing creams are important parts of the treatment strategy of atopic eczema. This study aims to investigate the tolerance of a new strong steroid cream in comparison to an already marketed reference cream with equal amount of active but with different cream vehicle. The second part of the study will investigate the possible preventative property of a moisturizing cream on skin that has been previously cleared from eczema.


Eligibility Criteria:
Inclusion Criteria:

* Females and males between 18 and 65 years of age
* Caucasian
* AD according to the criteria of Hanifin and Rajka (12) with eczematous lesions corresponding to an Atopic Dermatitis Severity Index (ADSI) (13) score of at least 6 on any of the following areas: arms, legs, chest, abdomen or back
* No serious health conditions that may interfere with the study
* Written informed consent

Exclusion Criteria:

* Eczematous regions exclusively in intertriginous areas or in the face
* Use of topical steroids or any other dermatologic drug therapy (moisturising creams allowed) in the study area or light therapy within the preceding 2 weeks
* Use of oral steroids within 1 month prior to the study
* Use of concurrent medication e.g. medication that may interfere with the study related activities
* Factors suggesting low compliance with study procedures
* Possible allergy to ingredients in the study medications
* Pregnancy or breast feeding
Comparison of Two Methods of Bowel Preparation for Colonoscopy in Hospitalized Patients

Morning-only colonoscopy preparation may improve efficiency by allowing same-day patient preparation and colonoscopy. The aim of the investigators study is to compare the efficacy and tolerability of morning-only Polyethylene Glycol (PEG) to split-dose preparation in hospitalized patients undergoing colonoscopy.

This is a single-center, prospective, endoscopist-blinded study in which hospitalized patients scheduled to undergo diagnostic colonoscopy were randomly assigned to receive one gallon of PEG either the morning of colonoscopy or as a split-dose (evening-morning). The primary end point is efficacy of colonoscopy preparation measured by the Ottawa scale. The investigators pre-specified a non-inferiority margin of 1.5 for the difference in Ottawa scale between arms. Secondary end points are patient compliance and tolerance. N: 120 hospitalized patients scheduled for diagnostic colonoscopy.

Eligibility Criteria:
Inclusion Criteria:

* Adult patients scheduled to undergo diagnostic colonoscopy during hospitalization

Exclusion Criteria:

* Critically ill/Intensive Care Unit patients
* Suspected bowel obstruction/perforation
* History of colon resection
* Patients with altered mental status/inability to provide informed consent
* Incarcerated patients
* Pregnant females
Virtual Psychiatric Care for Perinatal Depression

Perinatal depression occurs in 15% of pregnant women and new mothers, yet as few as 1 in 5 receive adequate treatment. It has a negative impact on the woman herself, as well as a potential for serious consequences for her child. Virtual psychiatric care is a potential model that may provide accessible mental health care during this time, as barriers to in-person care often include unpredictable infant schedules, competing childcare demands, or travel/cost challenges. The Virtual-PND intervention consists of 12-weeks of the option of supplementing in-person psychiatric care with secure, in-home real-time video-visits through the Ontario Telemedicine Network. This pilot RCT will demonstrate the feasibility of proceeding to a future large-scale RCT evaluation of virtual psychiatric care for this population.

This is a two-site pilot randomized controlled trial (RCT) to evaluate the feasibility of a Virtual Psychiatric Care for Perinatal Depression (Virtual-PND) intervention protocol, comparing virtual care to in-person care only. Participants will be pregnant or postpartum women with a major depressive disorder who are referred for care at one of the participating study sites. Each study site is a specialist reproductive psychiatric program where women from the greater Toronto area are referred, and generally treated with in-person care. Those randomized to the virtual care group will be able to receive psychiatric visits with video-based visits. Those randomized to the in-person care group will receive in-person care only, as per usual clinical practice at the study sites. The length of the active treatment phase will be 12 weeks from enrolment. The overall objective of this pilot RCT is to determine the feasibility of conducting a larger RCT to evaluate the efficacy of the treatment model, comparing outcomes among those with and without access to virtual care, and cost-effectiveness.

Eligibility Criteria:
Inclusion Criteria:

* ≥18 years old
* Pregnant or 0-12 months postpartum
* Experiencing clinically significant depressive symptoms (defined as a score of ≥12 on the Edinburgh Postnatal Depressive Scale, EPDS, during eligibility screening)
* Able to access the internet on a device (i.e. mobile phone, tablet, personal computer) with video-visit capacity (including web camera and speakers)
* Comfortable with email communication

Exclusion Criteria:

* Active alcohol/substance use disorder in the past 12 months
* Active suicidal ideation
* Current mania or psychosis
* Unsuitable for virtual care, as per assessment of the treating psychiatrist
Evaluation of Brain Changes in ALL Patients on Therapy

The study is aimed at assessing changes in the brain of patients from 6 to 18 years old with a diagnosis of acute lymphoblastic leukemia during chemotherapy.

Children with acute lymphoblastic leukemia receive long-term combined chemotherapy treatment, one of the main part of this treatment is methotrexate. Methotrexate can cause acute, subacute, and long-term neurotoxicity. Clinical symptoms of neurotoxicity, such as seizures, aphasia, are often associated with leukoencephalopathy. However, leukoencephalopathy may develop asymptomatically in children receiving methotrexate.

The effect of chemotherapy on the neurological status of patients is widely covered in the literature. There are very few studies devoted to the quantitative assessment of MRI parameters, especially in children. MRI is a non-invasive method that does not harm the patient and can be used to assess absolutely all structures of the brain.

Eligibility Criteria:
Inclusion Criteria:

1. patients from 6 to 18 years old
2. diagnosis of acute lymphoblastic leukemia during chemotherapy

Exclusion Criteria:

1. patients less then 6, and older then 18 years old
2. patients who cannot perform the study MRI without general anesthesia
3. Refusal to sign informed consent.
4. The presence of absolute contraindications to MRI studies.
5. Extremely serious condition of the patient.
Genetic Variation and Immune Responses After Injury

Our overall hypothesis is that genetic variations in innate immunity genes predispose patients to varying responses after injury by altering the systemic and local inflammatory responses. In addition, we hypothesize that these genetic differences are associated with different clinical outcomes

The goal of this research proposal is to identify relationships that exist between specific genetic markers, immune responses to injury and infection (sepsis), and post injury clinical outcomes. Specifically, we will investigate the clinical impact of mutations involved in the innate immune response, which likely influence host response. To accomplish this goal we will collect and analyze data from patients with acute thermal injury, the most quantitative inflammatory stimulus experienced by humans. In addition, we propose to further characterize the immunologic response parameters to injury and infection, and their role in complicated sepsis. In this way, we will identify parameters associated with unfavorable clinical outcomes, and determine how these parameters differ among individuals with different genotypes. We propose to 1) evaluate associations between candidate SNPs within the NOD2/RIP2 signaling pathway and clinical outcome following burn injury, 2) evaluate the functional effects of alternate alleles at candidate SNPs; finally 3), we will use genetically engineered animal models to determine whether mutations in the NOD2 or RIP2 genes alter myocardial signal transduction mechanisms shown to play a role in myocardial inflammation/dysfunction after burn trauma. These approaches should allow us to evaluate more extensively clinically relevant interactions between specific genetic polymorphisms, the cellular expression of immune mediators, and burn-induced immune dysfunction. The proposed research should uncover genetic and/or acute immune-inflammatory parameters that identify patients who are at "high risk" and could as a result make possible the targeted design of pharmacologic intervention strategies that will inhibit the toxic effects of LPS and other bacterial pathogen components without paralyzing the host immunity of patients

Eligibility Criteria:
Inclusion Criteria: All burn,trauma, or acute surgery victims admitted to the surgical, burn or trauma units within 24 hours of injury will be considered for inclusion.

Exclusion Criteria:severe immunosuppression, DNR, severe trauma, terminal diseases.

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Pharmacologic Effects of Darifenacin and Tolterodine on Cardiovascular Parameters in Healthy Subjects

This study will evaluate the pharmacologic effects of exposure to darifenacin and tolterodine on cardiovascular parameters in healthy subjects 50 years of age and older


Eligibility Criteria:
Inclusion Criteria:

* Healthy males and females ≥ 50 years
* Body mass index equal to or greater than 18.5 kg/m2 and less than 35.0 kg/m2

Exclusion Criteria:

* Known or suspected allergy to tolterodine ER or darifenacin or their components
* Subjects with irregular day and night patterns such as night shift workers
* Significant medical problems know to affect heart rate (ie., hypertension, hypotension, history of heart failure, history of pulmonary disease, etc.)
* Medication with potential known to affect heart rate
* History of any malignancy within the past 5 years, with the exception of localized basal cell carcinoma of the skin
* Pregnant or nursing women
* Subjects with diseases such as urinary retention, gastric retention, uncontrolled narrow-angle glaucoma, severe renal insufficiency, etc.

Other protocol-defined inclusion/exclusion criteria may apply
Randomized Double Blind Controlled Trial Comparing a Blind Sciatic Nerve Block in the Popliteal Fossa to Intravenous Morphine for Traumatic Severe Acute Pain in the Prehospital Setting

The purpose of the study is to determine the efficacity of a simple blind technic of loco regional anaesthesia of the sciatic nerve compared to the gold standard in emergency medecine : the intravenous morphine for treated the severe pain of leg, ankle or foot trauma in the prehospital setting and mountain rescue.

Severe pain due to leg, ankle or foot trauma do not beneficiated of a simple loco regional anaesthesia technic. Loco regional anesthesia is superior to intravenous morphine for treating severe pain and specially during transportation and mobilization of the patient. The purpose of the study is to assessed a new technic of blind sciatic nerve block in the popliteal fossa in the pre hospital setting compared to the intravenous morphine. The blind technic is a modified technic of the classic sciatic nerve block with nerve stimulation.

Eligibility Criteria:
Inclusion Criteria:

* Severe pain (VAS \> or = 60mm); injuries of the leg, ankle or foot

Exclusion Criteria:

* Hypersensitivity to student agents
* Local infection
* Nerve or vascular pathology in the affected limb, coagulation pathology,
* Chronic use of opoids, use of opoids within 6 hours
* Drug addiction
* Pregnancy
* Systolic blood pressure less than 90 mmHg
* Respiratory rate less than 16 per minute
* Glasgow coma scale \< 14.