cyrilzakka/clinical-trials huggingface dataset:
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia

This proof-of-mechanism study is being performed to investigate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of multiple oral doses of bitopertin in adults with NTD beta-thalassemia.

This study consists of two parts:

Part 1 - The main study - 16 weeks in total: Participants will undergo a 6-week dose-escalation period followed by 10 weeks of treatment at the attained target dose.

Part 2 - Open Label Extension (OLE) - up to an additional 12 months. Participants will be given the option to enroll into the OLE once the 16-week treatment of Part 1 has been completed. Participants who decide not to enroll in the OLE, at the end of Part 1 will enter a 6-week follow-up period.


Eligibility Criteria:
Inclusion Criteria:

* Confirmed diagnosis of beta-thalassemia
* Clinically defined non-transfusion-dependent anemia (Part 1 only), defined as Hb concentrations \>7.5 grams per deciliter (g/dL) and \<9.5 g/dL, less than or equal to 4 transfusions of red blood cell units within 1 year prior to study enrollment, and no transfusion within 12 weeks prior to study enrollment
* Completion of 16 weeks of treatment with bitopertin in Part 1 of this study with more than 80% compliance from expected use of study medication (based on patient diary and study drug accountability; Part 2 only)
* A favorable benefit-risk ratio from treatment with bitopertin as assessed by the Investigator (Part 2 only)

Exclusion Criteria:

* Any history of gene therapy
* History of hemolytic anemia except for beta-thalassemia
* Severe symptomatic splenomegaly and/or hepatomegaly with hypersplenism (Part 1 only)
* Any use of an erythropoiesis-stimulating agent within 24 weeks prior to enrollment.
* Initiation of iron chelation therapy or hydroxyurea within 24 weeks prior to enrollment (Part 1 only)
* Depression, treatment with anti-depressants, or other psychiatric illnesses and/or drug abuse
* Clinically significant/uncontrolled comorbid disease
* Pregnant or breastfeeding females
* Use of cytochrome P450 (CYP) 3A4 inhibitors within 2 weeks or CYP3A4 inducers within 4 weeks prior to study drug
* Active hepatitis B or C or known positive human immunodeficiency virus (HIV) test result
* Diagnosis of cancer within previous 5 years unless treatment has resulted in complete freedom from disease for at least 2 years
* Any major illness within 1 month or febrile illness within 1 week prior to study drug
* Pulmonary hypertension requiring oxygen therapy (Part 1 only)
Phase I Study of the BBP-398 in Patients With Advance Solid Tumors

This is an open label, dose escalation and expansion, two-part Phase I study for SHP-2 inhibitor BBP-398 to evaluate the safety, tolerability, pharmacokinetics, determine MTD and/or RP2D, and preliminary anti- cancer activity in Chinese subjects with advanced solid tumors and in Chinese subjects with advanced or metastatic EGFR-mutant NSCLC.

The Part A of this phase I trial is an abbreviated dose escalation study of BBP-398 following the USA mono dose escalation study (Study NAV- 1001, clinicaltrials.gov ID NCT04528836). The purpose of this part is to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-cancer activity in Chinese subjects with advanced solid tumors. The Part B of this study is to explore the safety, tolerability and efficacy of BBP-398 in Chinese subjects with advanced or metastatic EGFR- mutant NSCLC at MTD and/or RP2D. This Phase I study will provide supportive data to enable Chinese patients to join the combo dose escalation and expansion studies and/or other clinical trials of BBP-398.

Eligibility Criteria:
Inclusion Criteria:

1. Patients must have the ability to understand and the willingness to sign a written informed consent document 2 Patients must be willing and able to comply with the scheduled visits, treatment plan, laboratory tests and other specified study procedures 3. Age ≥18, male or female 4.Dose escalation: locally advanced or metastatic solid tumors Dose expansion: Advanced or metastatic EGFR-mutant NSCLC 5. Patients must have measurable disease by RECIST v1.1. 6. Patients must have an ECOG performance status (PS) ≤2 7.Patients with a life expectancy of ≥12 weeks. 8. Patients must have adequate organ function

Exclusion Criteria:

1. Patients with a known additional malignancy that is progressing or requires active treatment
2. Patients who have previously received a SHP-2 inhibitor
3. Patients who are hypersensitivity to SHP-2 inhibitor or any ingredients
4. Treatment with any of the related anti-cancer therapies prior to the first dose of BBP-398 within the stated timeframes
5. Patients with known active Hepatitis B, Hepatitis C infection, or HIV infection.
6. Patients with any of the cardiac-related issues or findings
7. Patients with a history of CVA, myocardial infarction or unstable angina within the previous 6 months before starting therapy.
8. Patients with known central nervous system (CNS) tumors
9. Patients with known active CNS metastases and/or carcinomatous meningitis.
10. Patients with persisting toxicity related to prior therapy.
11. Patients who have undergone major surgery within 4 weeks prior to study enrollment.
12. Pregnant or breastfeeding female patients.
13. Patients with inability to swallow oral medications or with gastrointestinal illness that would preclude the absorption of an oral agent.
Haploidentical Bone Marrow Stem Cell Transplantation in Patients With Multiple Myeloma

The aim of this phase 2 study is to demonstrate that KIR-ligand mismatched haploBMT with post-transplant cyclophosphamide will improve progression free survival in poor risk multiple myeloma patients.

The goal of this study is to evaluate the effectiveness of a new treatment modality, the KIR-ligand mismatched haploidentical stem cell transplantation (haploBMT), for poor risk multiple myeloma (MM) patients. MM is a malignancy of plasma cells that usually resides in the bone marrow. Despite new treatment modalities that have been introduced in the last years, MM is still an incurable disease for most patients and median survival for the younger patients (\<65) is about 5 years. MM can be treated by several disease modifiers - classical chemotherapy, high dose chemotherapy and autologous stem cell transplantation (ASCT), immunomodulators like thalidomide and lenalidomide, and drugs like bortezomib that interact with relevant intracellular pathways of malignant plasma cells. Though these treatment modalities have improved overall survival and quality of life, patients are not cured.

Eligibility Criteria:
Inclusion Criteria:

* Patients with MM \<60 years.
* Poor prognosis MM patients, permissive for KIR-ligand mismatch and with a KIR-ligand mismatched haploidentical donor. Poor prognosis is based on:

  * Patients with early disease recurrence (within 12 months after first ASCT) or
  * Patients after a minimum of three lines of chemotherapy (including high dose therapy followed by ASCT rescue therapy) or
  * Poor risk based on the cytogenetic profile.
* Written informed consent
* No HLA identical related or 10/10 matched unrelated donor
* Permissive for KIR-ligand mismatch
* Responsive after reinduction therapy
* Measurable disease

Exclusion Criteria:

* - Patients with an full matched (10/10) donor, who will enroll in the HOVON 96 study
* Active uncontrolled infections
* Uncontrolled CNS involvement by the malignant disease
* Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or symptomatic ischemic heart disease)
* Severe pulmonary dysfunction (CTCAE grade III-IV)
* Severe neurological or psychiatric disease
* Significant hepatic dysfunction (serum bilirubin or transaminases ≥ 3 times upper limit of normal)
* Significant renal dysfunction (creatinine clearance \< 30 ml/min after rehydration)
* History of active malignancy during the past 5 years with the exception of basal carcinoma of the skin or stage 0 cervical carcinoma
* Any psychological, familial, lingual, sociological and geographical condition potentially hampering compliance with the study protocol and follow-up schedule
* Breast-feeding female patients.
* Concurrent severe and/or uncontrolled medical condition (DM, hypertension, cancer).
Aquatic Exercises for Patients With Chronic Low Back Pain

The goal of this study is to compare the effect of aquatic exercises (AEG) to the aquatic exercises plus aerobic training (deep-water running) (AEDWRG) on functional status and pain in patients with specific chronic low back pain.

In general, the population will have at least one episode of low back pain during their lifetime, with recurrence in most cases. Several treatments have been proposed for these patients and the evidence is still not clear about the effectiveness of aquatic exercises when compared to other modalities of exercises in the water. This study will be a randomized controlled trial following the Consort-Statement principles. The subjects, aged between 20 and 50 years, after signing the informed consent will be randomly allocated to one group treatment: aquatic exercises (AEG) or aquatic exercises associated to deep-water running (AEDWRG). The duration of the protocol will be set at 12 weeks, 3x-week.

Eligibility Criteria:
Inclusion Criteria:

* Exclusive diagnosis of chronic low back pain (more than 12 weeks)

Exclusion Criteria:

* Central nervous system disease
* Pregnant
* Fear of water
An Infraclavicular Landmark-based Approach to the Axillary Vein

The central line placement is the widespread procedure performed in the intensive care and emergency medicine. Indications for this procedure are chiefly lack of peripheral catheters, administration some medications, renal replacement therapy, parenteral nutrition and hemodynamic monitoring.

The procedure is performed by percutaneous puncture of large vein of the neck and the thorax - internal jugular vein or subclavian vein, then insertion of guidewire through the needle and placement of the catheter over the guidewire. The tip of the catheter is situated in the superior vena cava.

There are two techniques of catheterizations: landmark-based and ultrasound-guided. The most frequently cannulated veins in landmark-based approach are internal jugular and subclavian vein. The cannulation of the axillary vein is not common procedure in the intensive care unit, mainly due its complicated original technique.

The primary intention of this study was to describe and assess usefulness and safety of the new landmark-based technique of catheterization of the axillary vein in patients admitted to the intensive care unit.


Eligibility Criteria:
Inclusion Criteria:

* patients admitted to the intensive care unit with indications for central venous catheterization

Exclusion Criteria:

* chest wall deformities at the cannulations side
* major hemostasis disorders
* infections at the catheterization site
* age less than 18 years
* lack of patients consent
Effect of Osteopathic Techiniques on Intraocular Pressure

The purpose of this study is to assess the effects osteopathic techniques at the upper cervical vertebrae or at the sphenopalatine ganglia on the intraocular pressure.

The 7 volunteers will be randomized into 3 groups and there will be an experimental group 1 that will receive a technique of manipulation of the upper cervical, an experimental group 2 that will receive a technique of manipulation of the sphenopalatine ganglion and a control group that will not receive any intervention. Each volunteer will participate in the 3 groups. There will be 4 assessments, one before the procedure and one just after each procedure, including the control.

Eligibility Criteria:
Inclusion Criteria:

* Intraocular pressure below 21mmHg

Exclusion Criteria:

* Positive Klein test
* Use of medications that affect the circulatory system in up to one month before the procedures;
* Caffeine use 24 hours before procedures;
* Presence of ophthalmic diseases;
* History of hypertension or diabetes;
* Blindness;
* Tumor in the head;
* Skull or cervical fractures that occurred less than 6 months ago.
A Community Health Worker Intervention to Address Adverse Pregnancy Outcomes

United States maternal mortality and preterm birth rates are among the highest among high-income countries due in part to a combination of racial, regional and socioeconomic disparities in access to care and overall health. The research proposed focuses on adapting and expanding a perinatal community health worker intervention for Black postpartum patients with preeclampsia (PE) and other adverse pregnancy outcomes (APOs). Investigators will partner with a community-based organization that trains and deploys community health workers. Investigators will test an intervention for urban and rural Black postpartum patients with APOs to 1) enhance blood pressure control postpartum and 2) promote long-term cardiovascular disease prevention for this underserved population. This pilot study will determine if randomizing and implementing a community health worker intervention tailored to pregnant people experiencing preeclampsia is feasible and found to be acceptable by participants.

The investigators will adapt a current perinatal community health worker intervention, lead by a leading community health worker organization, Connection Health, to the unique needs of Black postpartum patients with preeclampsia and other adverse pregnancy outcomes. The investigators will conduct a pilot trial to assess the feasibility and acceptability of the intervention. The hypothesis is that it is possible to randomize eligible patients to the feasibility trial, and the intervention will be acceptable to participants. The investigators will randomize Black postpartum patients with preeclampsia or other adverse pregnancy outcomes to either 1) usual care - cardiovascular disease-prevention education before discharge, or 2) intervention - cardiovascular disease prevention education before discharge plus an adapted Connection Health community health worker intervention. Following the completion of this project, the investigators intend to conduct a larger postpartum community health worker intervention trial in patients with preeclampsia or other adverse pregnancy outcomes powered to detect a difference in clinically meaningful outcomes, as informed by our existing community advisory board.

Eligibility Criteria:
Inclusion Criteria:

* Self Identifies as Black
* Between 16-56 years old
* Experiencing adverse pregnancy outcomes defined as hypertensive disorders of pregnancy, preterm birth, placental abruption, pregnancy loss (loss at greater or equal to 14 weeks gestation), gestational diabetes, delivering a small for gestational age infant
* Planning to deliver at UAB Hospital
* Speaks and writes in English

Exclusion Criteria:

* Declines Randomization
* Speaks or writes in languages other than English
* Currently incarcerated
* Previously enrolled in P3OPPY
Energy Expenditure in Breast and Bottle Feeding Preterm Infants Fed Their Mother's Breast Milk

OBJECTIVE. Neonatologists consider early feeding at the breast to be too tiring for preterm infants, although there is no evidence that this is actually the case. It is also not known whether the method of feeding affects energy expenditure. We hypothesized that resting energy expenditure (REE) would be higher after breastfeeding than after bottle feeding.

PATIENTS AND METHODS. preterm ( \>32 weeks GA) stable infants who are nourished entirely by their mothers' breast milk will be studied when fed expressed breast milk either by bottle or at the breast. REE will be measured for 20 minutes after feeding. Breast milk quantity is evaluated by pre- and post feeding weighing.

Most VLBW infants cannot be fed at the breast upon birth, and expressed breast milk by gastric tube is thus recommended. Sucking skills mature at around 34 weeks when nipple-feeding is introduced. In the absence of evidence-based data to decide the best timing to introduce breastfeeding, many clinicians use empiric criteria, such as the infant's weight, gestational age, and the ability of infants to bottle feed as proxies of readiness to breastfeed. Despite some evidence of physiological benefits of preterm infants feeding at the breast, many neonatologists consider direct breastfeeding to be too fatiguing for preterm infants. It is not known, however, whether preterm infants who are breastfed expend more energy than bottle-fed infants

Eligibility Criteria:
Inclusion Criteria:

* Thermally stable infants were nursed in an open and unwarmed bassinet.
* All fed solely by their mothers' breast milk equivalent to at least 150 cc/kg/day divided into eight meals and growing steadily

Exclusion Criteria:

* Infants with congenital anomalies and infants who had either more than five daily episodes of apnea of prematurity or any apnea requiring assistance or methylxanthine therapy
Exploring the Visual Sensitivity for Topological Properties in Newborn Infants

The topological perception theory proposed a precise description for how one organizes the visual world that eliminates the vagueness of subjective phenomenology. In addition, the topological theory challenges the dominant computational view on the part-to-whole hierarchy of visual information processing. Lines of evidence from adult psychophysics, brain imaging data, and even honeybee's behavior have supported the notion that the global topological properties are the very primitives of visual representation. However, the question of how the sensitivity to topological properties originates during development has not been explored much. In a previous study, the investigators found that 2- to 6 month old infants could reliably discriminate stimuli based on topological differences, but failed to do so based on geometric differences. Using familiarization/novelty preference procedure, the present study intends to explore the visual sensitivity for topological properties in newborn infants. Experiment 1 focuses on whether neonates can discriminate a disk (no hole) and a ring (with a hole) that are topologically different, and/or a disk (no hole) and a triangle (no hole) that are geometrically different. Experiment 2 focuses on whether neonates can detect a change in the number of holes and/or the size of the hole. If newborn infants are only sensitive to topological properties and not to geometric properties, this will be a strong proof for the claim that topological property is the very "primitive" visual representation at empirical as well as theoretical level.


Eligibility Criteria:
Inclusion Criteria:

* full-term and healthy newborns

Exclusion Criteria:

* Eye illnesses or other physical problems
Short Term Corticosteroids in SARS-CoV2 Patients

The investigators reviewed the charts of SARS-CoV-2 patients with pneumonia and moderate to severely elevated CRP and worsening hypoxemia who were treated with early, short-term dexamethasone.


Eligibility Criteria:
Inclusion Criteria:

1. Moderate disease with at least a 30% increase in CRP within 36 hours of admission, and with increasing oxygen requirements.
2. All patients with severe disease with evidence of escalating oxygen requirements
3. The presence of secondary bacterial infections as a probable cause of increasing CRP levels was excluded in all selected patients.
4. Pulmonary embolism and /or cardiac dysfunction were excluded as probable causes of worsening hypoxia in all selected patients.

Exclusion Criteria:

1. All patients on other treatment modalities- Remdesivir and/or Convalescent plasma who showed evidence of clinical improvement as per decrease in CRP levels and/or oxygen requirements were excluded.
2. Patient with associated COPD exacerbation who would benefit from the use of steroids.
3. Patients with Diabetic ketoacidosis, hyperglycemic hyperosmolar state, active concurrent bacterial infections.
4. Patients with history of steroid-induced mania and/or psychosis.
Hyperthermy Endocavitary Treatment in Pelvic Pain

* Treatment at pelvic floor with hyperactivity with hyperthermy electrode MJS
* MJS electrode is effective for transvaginal treatment at pelvic pain patients
* Hyperthermy treatment with MJS electrode is more effective to usual drugs treatment

This study analyzed the evolution of chronic pelvic pain, defined by the different map of myofascial trigger points assets presents the patient with medical treatment compared to treatment with radiofrequency endocavitary electrode MJS. The initial evaluator analyzed by the NIH-CPSI Scale questionnaire and symptoms of chronic pelvic pain Stanford Protocol for female or male quality of life of the patient before the doctor or radio frequency and a month since treatment. the patient to the Principal Investigator, specialist physiotherapist, who appreciates tenderness the presence of a taut band with a map of pain in endopelvic and exopelvic muscles, determining the activation of myofascial trigger points in each is derived. The Principal Investigator proceeds to address each active myofascial trigger points with the MJS electrode four 20-minute sessions twice a week. The MJS has a curved end pressure which favors the compression point plus the location and accurate approach Radiofrequency pathological point as outstanding feature. The high specificity of myofascial treatment offered by the MJS electrode involves no danger, besides having the computer screen conductivity meter and a temperature sensor on the head to control both parameters in real time during the treatment session

Eligibility Criteria:
Inclusion Criteria:

* Women with chronic pelvic pain

Exclusion Criteria:

* chronic pelvic pain or neurological degenerative etiology
* Presence of pathology or infectious processes genitourinary level
* possibility of pregnancy or lactating.
* Lack of informed consent, mental disorders, dementia, dependent people who need tutor.
Cancer Distress Coach Mobile App Trial

The Cancer Distress Coach (CDC) mobile app is a symptom management tool for posttraumatic stress disorder (PTSD) in cancer patients and caregivers. Based largely on "PTSD Coach" from the National Center for PTSD, the CDC app was redesigned for both iOS and Android platforms and is tailored specifically to individuals impacted by cancer.

This study aims to evaluate whether CDC app usage influences symptoms of PTSD through a randomized controlled trial (RCT) where subjects are placed into either the intervention group or the wait-listed control group for a period of 8 weeks. The CDC app is available as a free download on the App Store® and on Google Play™.


Eligibility Criteria:
Inclusion Criteria:

* Have been diagnosed with cancer or are/were a caregiver to someone with cancer
* Resident of the United States of America
* Able to read and write English
* Age ≥ 18 years

Exclusion Criteria:

* None