cyrilzakka/clinical-trials huggingface dataset:
Ceritinib (LDK378) for Patients Whose Tumors Have Aberrations in ALK or ROS1 (SIGNATURE)

The purpose of this signal seeking study was to determine whether treatment with ceritinib demonstrated sufficient efficacy in select pathway-activated solid tumors and/or hematologic malignancies to warrant further study.

This was an open label study to determine the efficacy and safety of treatment with ceritinib in patients with a diagnosis of solid tumors or hematological malignancies that had been pre-identified (prior to study consent) to have ALK or ROS1 positive mutations, translocations, rearrangements or amplifications and whose disease had progressed on or after standard treatment. The study consisted of a treatment phase where all patients received ceritinib capsules for a total dose of 750 mg daily for up to 8 cycles of 28 days. Disease assessments for clinical benefit were performed every 8 weeks until disease progression or end of treatment. Following discontinuation of treatment for any reason, patients were followed for safety for 30 days. Survival information was collected every 3 months until 2 years after the last patient had enrolled into the study. Study was amended to allow for discontinuation of survival period if primary endpoint was not met.

Study was terminated due to low enrollment.

Eligibility Criteria:
Inclusion Criteria:

* Patient had a confirmed diagnosis of a select solid tumor (except ALK+ NSCLC) or hematological malignancy and was in need of treatment because of radiologic progression or relapse.
* Patient must have been pre-identified as having a tumor with an ALK or ROS1 positive mutation, translocation, rearrangement or amplification. The qualifying alteration must have been assessed and reported by a CLIA-certified laboratory. ALK positivity as assessed by IHC or FISH were allowed.
* Patient must have received at least one prior treatment for recurrent, metastatic and/or locally advanced disease and for whom no standard therapy options were anticipated to result in a durable remission.
* Patient had progressive and measurable disease as per RECIST 1.1 or other appropriate hematological guidelines.
* Patient had an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1.

Exclusion Criteria:

* Patient had received prior treatment with ceritinib.
* Patients with symptomatic CNS metastases who were neurologically unstable or required increasing doses of steroids within the 2 weeks prior to study entry to manage CNS symptoms.
* Patient had received chemotherapy or anticancer therapy ≤ 4 weeks (6 weeks for nitrosourea, monoclonal antibodies or mitomycin-C) prior to starting study drug.
Heart Rate Variability and Hyperthyroidism: Evaluation of the Short-term Effects of Propanolol

Objective: The aim of our study was to evaluate the short term effects of Propanolol on heart rate variability in patients with hyperthyroidism.

Design: In ten patients newly diagnosed as having hyperthyroidism and naïve of all treatment specific for hyperthyroidism, we evaluated the short term effects of Propanolol on heart rate variability (HRV).

Methods: We measured heart rate variability by power spectral analysis during 2 hours of rest and during Ewing's tests before and after the administration of Propanolol 40mg/12 hours orally for 72 hours.

Objective: The aim of our study was to evaluate the short term effects of Propanolol on heart rate variability in patients with hyperthyroidism.

Design: In ten patients newly diagnosed as having hyperthyroidism and naïve of all treatment specific for hyperthyroidism, we evaluated the short term effects of Propanolol on heart rate variability (HRV).

Methods: We measured heart rate variability by power spectral analysis during 2 hours of rest and during Ewing's tests before and after the administration of Propanolol 40mg/12 hours orally for 72 hours. Other outcomes included mean heart rate, mean R-R interval during 2-hour rest.

The study questions the systematic adjunction of Propanolol early in the treatment of hyperthyroidism.

Eligibility Criteria:
Inclusion Criteria:

* consenting patients referred to or diagnosed at the National Centre of Obesity of the Yaounde Central Hospital as having hyperthyroidism
* naïve of all treatment specific for hyperthyroidism.
* aged 18-70 years

Exclusion Criteria:

* Patients already under a specific treatment for hyperthyroidism
* Patients taking beta blockers or any other cardiospecific treatment
* Patients presenting complications including acute thyrotoxic crisis, or symptomatic cardiac failure
* Contraindications to the prescription of beta blockers
Localized Effects of PBM and Exogenous NO on CREST Patients Calcinosis Cutis & Raynaud Phenomenon

Background CREST is an acronym for the cardinal clinical features of the syndrome (Calcinosis, Raynaud phenomenon, Esophageal dysmotility, Sclerodactyly, and Telangiectasia) and part of the heterogeneous group of sclerodermas.

Calcinosis is the pathologic calcification of soft tissues. When symptomatic, they can be tender and painful, ulcerate, and drain a white chalky substance. With time, heterotopic bone formation may occur. Inflammatory reactions also intermittently occur at the site of calcinosis. It has been suggested that TGF-beta3 plays a major role in the pathogenesis of calcinosis.

A variety of medical therapies have been used to try to alleviate patient symptoms. These include pharmacological approaches (e..g., warfarin), surgical curettage or excision, as well as carbon dioxide laser treatments. No consistently reliable pharmacological treatment seems to be available to prevent or eliminate calcinosis. Curettage and excision and carbon dioxide laser of localized painful large deposits can relieve symptoms but recurrence is common. In addition, aggressive curettage or excision can damage deeper neurovascular structures. While calcinosis is associated with significant morbidity its treatment remains a challenge.

Photobiomodulation (PBM) has been shown to promote wound healing, suppress inflammatory reactions and regulate collagen synthesis in a number of in vitro and in vivo studies.

Human skin contains photolabile nitric oxide (NO) derivatives which decompose after UVA irradiation and release vasoactive NO. However, aside from blue light, barely nothing has been reported about the effects of red and NIR wavelengths.

Method A custom-built air tight sleeve which envelopes the forearm of a subject will be used to measure the NO emanating from the skin under photobiomodulation conditions (red \& NIR) and quantified by chemiluminescence detection.

Simultaneously, CREST patient's hands exhibiting calcinosis and/or Raynaud phenomenon will be exposed to exogenous gaseous nitric oxide (INOMAX) to determine the vascular impact of this approach.

This case series will assess Light Emitting Diode (LED) based PBM therapy as a treatment alternative for cutaneous calcinosis and the effects of gaseous NO on calcinosis and/or Raynaud phenomenon in CREST patients.


Eligibility Criteria:
Inclusion Criteria:

* Male or female
* 18-60 years of age
* CREST syndrome with calcinosis cutis.
* CREST syndrome without calcinosis cutis.

Exclusion Criteria:

* Diabetes mellitus
* Acute inflammation
* Arrhythmia
* Acute malignancy
* Renal failure
* Active CVD
* Photodermatosis and/or photosensitivity including skin cancer-prone disease/syndrome (XP and Bloom Syndrome)
* Porphyria and/or hypersensitivity to porphyrins
* Congenital or acquired immunodeficiency.
Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ Electromechanical Device for Growth Hormone Treatment

This is a Chinese, Multicentre, Observational Study to study Adherence and Long Term Outcomes of Therapy in paediatric subjects using easypod™ electromechanical device for growth hormone treatment to assess the level of adherence of subject receiving SAIZEN® via easypod™.

Subjects will be enrolled in a multicenter longitudinal observational study. Parents/subjects will provide their Informed Consent/assent to upload their data for population-based analyses and, ultimately, clinical outcomes. Adherence data will be primarily derived from the easypod™ device combined with physician data entry of outcome measures. Data will be collected retrospectively and prospectively. This will allow the establishment of adherence profiles and subsequent clinical outcomes. Collected data will be also analyzed in a multinational pooled analysis of comparable national studies.

Primary Objective:

* To assess the level of adherence of subjects receiving SAIZEN® via easypod™ Secondary Objectives:
* To describe the impact of adherence on clinical outcomes for subject receiving SAIZEN® via easypod™
* To identify adherence subject profiling
* To asses the impact of adherence on Insulin-like Growth Factor 1 (IGF1) (i.e. above, below or within normal ranges)

Eligibility Criteria:
Inclusion Criteria:

* Over the age of \>2 years
* Under \<18 years of age, or over 18 without fusion of growth plates
* Patients diagnosed with growth hormone deficiency
* Naïve to growth hormone treatment
* Parent's or guardian's written informed consent, given before entering data into the observational study, with the understanding that the subject or parent/guardian may withdraw consent at any time without prejudice to future medical care. If the child is old enough (16 years old) to read and write, a separate assent form will be given as defined in the appropriate jurisdiction of each country.

Exclusion Criteria:

* Subjects taking growth hormone in whom growth plates have fused (i.e. for taking growth hormone for it's metabolic effects)
* Contra-indications to SAIZEN® as defined in the Summary of Product Characteristics (SmPC)
* Use of an investigational drug or participation in another interventional clinical study
Social Determinants of Health, Medication Use, and Quality of Life in Inflammatory Bowel Disease

Optimizing health related-quality of life (HRQoL) for patients with inflammatory bowel disease (IBD), who often experience a relapsing disease course, is an essential component of care. Improving IBD disease control is linked to increased health-related quality of life. Even as many effective pharmacotherapies to promote disease control are available, evidence suggests that Hispanic and Non-Hispanic Black IBD patients may not receive full benefit from these therapies compared to their Non-Hispanic White counterparts. Underlying mechanisms that contribute to observed disparities in the use of IBD medical therapies are likely multifactorial. Adequate access to treatment has been implicated. Hispanic and Non-Hispanic Black IBD patients are more likely to be Medicaid-insured, and Medicaid insurance has been associated with increased emergency room visits, a proxy for sub-optimal IBD control. Medication adherence has also been proposed as a potential mediating factor. IBD therapies can be time-consuming and costly, which can pose a challenge in achieving medication adherence. While previous studies suggest Black IBD patients have lower medication adherence than Non-Hispanic White patients, it is unclear the extent to which social factors contribute to this observation. The purpose of this study is to evaluate the association between social determinants of health, medication adherence, and HRQoL among Hispanic and Non-Hispanic Black IBD patients. Understanding potentially modifiable psychosocial factors that contribute to medication adherence and HRQoL will provide targets for later intervention towards the goal of health equity.


Eligibility Criteria:
Inclusion Criteria:

* clinical diagnosis of Crohn's disease, ulcerative colitis, or indeterminate colitis ≥ 3 months investigator confirmed on the basis of supportive clinical data such as colonoscopy, pathology and/or radiology
* age 18 years or older
* ability to provide informed consent in English or Spanish
* basic computer proficiency (i.e. to complete online survey)

Exclusion Criteria:

* race and ethnicity self-identified as other than Hispanic, Non-Hispanic Black, or Non-Hispanic White
High Dose of Dual Therapy Plus Bismuth for Helicobacter Pylori Treatment

Dual therapy for Helicobacter Pylori including a proton pump inhibitor (PPI) and amoxicillin. Amoxicillin has low resistance rate as well as low percentage of side effects. No trial has examined the the efficacy of high dose of dual therapy plus bismuth for H. pylori treatment.This study is designed to evaluate the efficacy and safety of the addition of bismuth to high dose of dual therapy for H. pylori eradication.


Eligibility Criteria:
Inclusion Criteria:

* Participants with non-ulcer functional dyspepsia or peptic ulcer disease
* Ability and willingness to participate in the study and to sign and give informed consent
* Confirmed H. pylori infection

Exclusion Criteria:

* Less than 18 years old
* With previous gastric surgery
* Major systemic diseases
* Pregnancy or lactation
* Allergy to any of the study drugs Administration of antibiotics, bismuth, antisecretory drugs in 8 weeks prior to inclusion
An Observational Study of Tocilizumab (Actemra) in Participants With Moderate to Severe Rheumatoid Arthritis

This observational study evaluates the use and efficacy of intravenous (IV) tocilizumab in routine clinical practice in participants with moderate to severe rheumatoid arthritis. Eligible participants initiated on tocilizumab treatment in accordance with the local label were followed for 6 months.


Eligibility Criteria:
Inclusion Criteria:

* Adults, \>/= 18 years of age
* Moderate to severe rheumatoid arthritis according to the revised (1987) American College of Rheumatology (ACR) criteria
* Participants in whom the treating physician has made the decision to commence tocilizumab treatment (in accordance with the local label); this can include participants who have received tocilizumab treatment within 8 weeks prior to the enrollment visit

Exclusion Criteria:

* Participants who have received tocilizumab more than 8 weeks prior to the enrollment visit
* Participants who have received treatment with any investigational agent within 4 weeks (or 5 half-lives of the investigational agent, whichever is longer) before starting treatment with tocilizumab
* History of autoimmune disease or any joint inflammatory disease other than rheumatoid arthritis
Quantification, Analysis and Simulation of Facial Mimics Movements

The SIMOVI project is a collaboration between our Department of Maxillofacial Surgery (CHU Amiens) and the Biomechanical bioengineer Research Department and Roberval Labs (Technological University of Compiègne). The question raised is the extent to which a facial mimic can be evaluated objectively. In today's clinical practice, the investigators use grading systems based on muscular scale or testing, in order to evaluate a facial muscular disorder. This assessment remains subjective because the investigators are deducing from surfacing cutaneous deformations the movement quality of the solicited muscle.

This qualitative approach is therefore an approximate approach and deserves to be better thought to plan a surgical treatment which involves mimic facial muscle (as rehabilitation of facial palsy, cleft palate for example), to monitor the results, and to follow the recovery and progress in physiotherapy care.

The aim of this study is to correlate external soft tissue movement (essentially cutaneous) during facial mimic with internal movement (essentially facial mimic muscle) using qualitative and QUANTITATIVE indicators, and to perform a biomechanical model of selected mimic face's movement using the precedent data.


Eligibility Criteria:
Inclusion Criteria:

* Caucasian subject
* Ability to provide informed consent

Exclusion Criteria:

* Facial pathology
* Facial trauma
* Pregnant or breastfeeding.
* Contraindications to MRI
A 28 - 90 Days Study to Evaluate the Safety, Tolerability, and Efficacy of Caspofungin Injection as Empirical Therapy in Indian Adults With Persistent Fever and Neutropenia (0991-053)(COMPLETED)

The purpose of this study is to evaluate the safety, tolerability and efficacy of ertapenem sodium as initial therapy for the treatment of complicated urinary tract infections, including pyelonephritis in indian adults.


Eligibility Criteria:
Inclusion Criteria:

* Patient has a low white cell count (less than 500/mm3) for at least 96 hours
* Patient is indian and is greater than 18 years of age
* Patient received chemotherapy for blood disorders and blood cancers

Exclusion Criteria:

* Patient has an invasive fungal infection
* Patient has a bacterial infection that is not controlled
* Patient has allergy to the class of antifungals of study drug
* Patient is not expected to survive at least 5 days
* Patient is pregnant or breast-feeding
Study to Assess the Effect of Dosing AZD6244 HydSulfate in the Presence and Absence of Food in Patients With Advanced Solid Malignancies

The purpose of this study is to test if the levels of AZD6244 in blood are affected by taking food at the same time as the capsules compared to taking capsules on an empty stomach


Eligibility Criteria:
Inclusion Criteria:

* Able to eat a high fat breakfast within a 30-minute period
* Advanced cancer which is refractory to standard therapies, for which no standard therapies exist
* WHO or ECOG performance status 0-2 (those with performance status 2 must have been stable with no deterioration for over 2 weeks)

Exclusion Criteria:

* Any radiotherapy or chemotherapy within 21 days prior to starting the study treatment (not including palliative radiotherapy at focal sites).
* Any evidence of severe or uncontrolled systemic disease (eg., severe hepatic impairment, severe renal impairment, uncontrolled diabetes, acute uncontrolled infection) or current unstable or uncompensated respiratory or cardiac conditions or peripheral vascular disease including diabetic vasculopathy.
* Refractory nausea and vomiting, chronic GI diseases (eg., inflammatory bowel disease) or significant bowel resection that would preclude adequate absorption.
IN Dexmedetomidine for Procedural Sedation in Pediatric Closed Reductions for Distal Forearm Fractures

The primary objective of this study is to evaluate the sedative, and analgesic effects of intranasal (IN) Dexmedetomidine (DEX) in children presenting to a Pediatric Emergency Department (PED) who undergo conscious sedation for reduction of closed distal forearm fractures when compared to those receiving intravenous (IV) Ketamine. The secondary objective is to compare each sedation technique for safety and procedural outcomes.

Distal Forearm fractures are often are displaced requiring conscious sedation for closed reduction in the Emergency Department. Our institution's current standard of care consists of IN Fentanyl for baseline control of pain, and for those fractures requiring reduction; typically IV Ketamine is utilized. Ketamine is typically well tolerated but is not without concerns including hypertension, vomiting, and the rare but serious complication of laryngospasm. Dexmedetomidine (DEX) offers a possible alternative to IV Ketamine. DEX has been used safely in the critical care setting for both pediatrics and adults. It has been well documented as being quite effective in sedation, amnesia and analgesia. Using IN DEX for PED procedural sedation has the potential to obviate the need for IV placement and may offer a better conscious sedation profile than Ketamine with respect to sedation, analgesia, and adverse outcomes.

Our overall project would be to assess the efficiency of IN DEX in comparison to IV Ketamine, for proper sedation and analgesic coverage for children undergoing closed reduction of distal forearm fractures.

Eligibility Criteria:
Inclusion Criteria:Verbal children aged 2-18 with a single extremity displaced forearm fracture requiring conscious sedation and reduction will be screened for enrollment.

Exclusion Criteria:

1. Under age 2 years old or patients \> 18 years old
2. Multiple Fractures
3. Significant multisystem trauma
4. Glasgow Coma Scale (GCS \< 15)
5. Complex fractures that aren't deemed reducible in ED
6. Reported Allergy to Alpha -2-agonists
7. Pregnancy
8. Intoxication
9. Baseline Hypotension as \< 70mm Hg + 2 x age or \< 90mm Hg for patients \> 11 years of age
10. Patients with prior reductions attempted at outside facilities
11. Aberrant nasal anatomy that precludes IN medications
12. Chronic Health issues that can affect DEX metabolism
13. History of adverse reactions to anesthesia
14. Patients transferred from outside facilities
15. Open fractures
PSMA-RLT in Biochemically Recurrent PCa

Prospective single-center one-arm phase II study in patients with prostate cancer and confirmed biochemical recurrence (BCR) with PSA of ≥ 0.2 ng/ml after radical prostatectomy (RP) or PSA \> nadir + 2ng/ml after radiotherapy (RT) but not radio-morphological local recurrence after primary therapy with curative intent receive systemic therapy with only 2 cycles of highly standardized (3 GBq in first cycle and 6 GBq in the second cycle) PSMA-RLT at 6-week intervals.


Eligibility Criteria:
Inclusion Criteria:

* Patients with biochemical recurrence after radical prostatectomy and radiotherapy with a PSA doubling-time (DT) of ≤ 12 months.
* No hormonal therapy within the last 12 months or recovered testosterone levels.
* PSMA PET negative result for local recurrence; presence of distant metastases is allowed: (cN0, cM0/cM1).
* Eastern Cooperative Oncology Group (ECOG) Performance Status: 0-1
* Patients must have adequate bone marrow reserve: WBC ≥1.5 x 109 /L, Platelets ≥100 x 109 /L and Haemoglobin ≥9 g/dL.
* Patients must have adequate renal function with eGFR ≥ 50mL/min/1.73m2 using the Modification of Diet Renal Disease (MDRD) equation and an Albumin level of ≥2.5 g/dL.
* Patients must be able to sign Informed Consent Form.

Exclusion Criteria:

* Concomitant participation in any other interventional trial.
* Concurrent severe oncological and medical conditions that result in patients not having a life expectancy of longer than one year.
* Presence of clinically relevant somatic or psychiatric diseases that might interfere with the objectives and assessments of the study.
* Complete urinary out-flow obstruction or severe unmanageable urinary incontinence.