cyrilzakka/clinical-trials huggingface dataset:
Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency

The purpose of this prospective study is to characterize the natural history of ENPP1 Deficiency and the early-onset form of ABCC6 Deficiency longitudinally. The study will prospectively gather information about the biochemical, physiological, anatomic, radiographic, and functional manifestations (including patient reported outcomes) of each disease.

Study INZ701-003 is a multicenter, prospective, longitudinal, observational study to evaluate disease presentation, progression, and burden of illness in pediatric subjects aged 2 to \<18 years with ENPP1 Deficiency and the early-onset form of ABCC6 Deficiency. Subjects will receive care available at the clinical site along with additional assessments administered by the study team.

To participate in this study, subjects will give informed assent and parents/guardians will provide informed consent.

Subject participation will consist of a Screening Period and an Observation Period.

Assessments will be performed at each visit as indicated in the Schedule of Events.

During the Screening Period, assessments will be performed to determine eligibility.

Screening and Baseline assessments may be conducted on the same day if the Investigator determines that the patient can successfully complete all study procedures in one day due to their age. If not, sites may schedule additional visits, as needed. A subject will be enrolled into the study if they meet all eligibility criteria.

During the Observation Period, subjects will be assessed for changes in their disease in the following areas: measurements of physiological function (including laboratory testing and anatomical and radiographical assessment of calcification and bone mineralization); performance outcomes; patient, caregiver, and physician reported outcomes; and healthcare utilization.

Eligibility Criteria:
Inclusion Criteria:

Individuals eligible to participate must meet all of the following inclusion criteria:

1. Must provide written consent of the legally authorized representative/caregiver and assent for subjects after the nature of the study has been explained and prior to any research-related procedures, following the policies of the clinical site
2. Genetic confirmation of ENPP1 Deficiency or ABCC6 Deficiency
3. Male or female, aged 2 to \<18 years
4. In the opinion of the Investigator, must be willing and able to complete all aspects of the study
5. Agree to provide access to relevant medical records

Exclusion Criteria:

Individuals who meet the following exclusion criterion will not be eligible to participate:

1. In the opinion of the Investigator and/or Sponsor, presence of any clinically significant disease (outside of those considered associated with the diagnosis of ENPP1 Deficiency or the early-onset form of ABCC6 Deficiency) that precludes study participation or may confound interpretation of study results, such as an unrelated bone, mineral, or muscle disease or genetic connective tissue disease
The Effect of TAP Block on Maternal-Infant Bonding

Cesarean delivery is a common surgical procedure that is typically performed under spinal anesthesia. Postoperative pain management has a significant impact on both the mother's physical recovery and the quality of maternal-infant bonding. The quality of maternal-infant bonding is influenced by various factors, including postoperative pain, early mobilization, initiation of breastfeeding, stress, psychological status, and hormonal balance. Studies have reported that adequate postoperative pain management, early breastfeeding, and early mobilization have positive effects on maternal-infant bonding.

Transversus abdominis plane (TAP) block is a regional anesthesia technique known for its efficacy in controlling postoperative pain. Bilateral TAP block performed after cesarean section has been associated with lower pain scores, reduced analgesic consumption, and shorter hospital stays. However, its impact on maternal-infant bonding has not been fully elucidated.

This study aims to investigate the effect of bilateral TAP block on maternal-infant bonding in women undergoing cesarean section under spinal anesthesia.

This study aims to investigate the effect of TAP block on maternal-infant bonding in women undergoing cesarean section under spinal anesthesia. Participants will be randomly assigned to two groups: one group will receive a TAP block in addition to standard care, while the other group will receive only standard pain management. Maternal-infant bonding will be assessed using the maternal bonding scale and behavioral evaluations at 48 hours and one month postpartum.

The primary outcome is to evaluate whether the TAP block improves early bonding by reducing pain and enhancing maternal comfort. Secondary outcomes include maternal pain scores, mobilization time, and initiation of breastfeeding. This study aims to contribute to the optimization of pain management strategies in cesarean deliveries, ultimately improving both maternal recovery and neonatal outcomes.

Eligibility Criteria:
Inclusion Criteria:

* 18 aged or older patients,
* Agree to participate in the study
* Pregnant women without any maternal or fetal pathology during the pregnancy.

Exclusion Criteria:

* \<18 age
* Deny to participate in the study
* maternal or fetal pathology during the pregnancy
* Allergy to local anesthetics
* local ingection at block side
The Effect of Three Methods Used in Reducing Pain During Dressing Change

This randomized controlled study aims to determine the effect of listening to music (LM), ball squeezing (BS), and performing mathematical operations (MO) in reducing the pain experienced during the first dressing in children aged 8-18 with appendectomy.

The sample of the study consisted of 120 children (LM:30, BS:30, MO:30, control:30). A minute before dressing and during dressing, the children in the LM group listened to music, the children in the SB group squeezed the ball, the children in the MO group performed mathematical operations.According to the child, parent, and nurse assessment, it was determined that the mean pain score of the children in the LM and MO groups was lower than that of the BS group and control groups (p\< .05).

Eligibility Criteria:
Inclusion Criteria:

* Consisted of the child and parent's consent to participate in the study,
* The child and parent's speaking Turkish at the level of native speaker,
* The child's being 8-18 years old and undergoing appendectomy,
* Being the body temperature of the child in the normal level (36.5-37.2⁰C),
* The child's not having intense nausea and vomiting,
* The child's not having any physical, intellectual or neurological disability that would affect his/her participation.

Exclusion Criteria:

* Having a mental or neurological disability
* Children with hearing and vision impairment
Comparative Effectiveness of Intervention in Multi-level Hospitals for Acute Traumatic Brain Injury(Metric-TBI)

A prospective, multicenter, observational cohort study is designed to compare the effectiveness of intervention in multi-grade hospitals for acute traumatic brain injury and to optimize clinical outcomes.

Traumatic brain injury (TBI) is associated with significant morbidity, mortality, and disability, profoundly impacting public health. This study aims to establish a database for acute craniocerebral trauma within the Beijing-Tianjin-Hebei region, and compiles indicators of hospital treatment capabilities and patient data, including neuroimaging, clinical progression, and rehabilitation prognoses.The study is bifurcated into two segments: the initial phase surveys the current status of acute treatment outcomes for TBI inpatients across hospitals in the Beijing-Tianjin-Hebei region and conducts a comparative effectiveness analysis to identify the clinical interventions to optimize. Subsequent phases build on the former by applying optimized treatment strategies to improve efficacy and establish collaborative optimized treatment protocols.

This is a prospective, multicenter, observational study designed to enroll 2,000 patients under the age of 90 years with traumatic brain injury in the presence of clinical symptoms confirmed by computed tomography or magnetic resonance imaging.The primary outcome is the Extended Glasgow Outcome Score within 12 months. Secondary outcomes include the Generalized Anxiety Disorder-7 (GAD-7), Patient Health Questionnaire-9(PHQ-9), Rivermead post-concussion symptoms questionnaire(RPQ), PTSD Checklist-5 Version(PCL-5)，Six-Item Screener (SIS) and 12-Item Short-Form Health Survey version 2(SF-12v2).

The objective of this study is anticipated to the Beijing-Tianjin-Hebei region a 20 percent increase in the number of cases treated by optimized clinical practice guidelines and a 10 percent decrement in mortality and disability rates among TBI patients in the region.

Eligibility Criteria:
Inclusion Criteria:

* Patient (1 years to 89 years) presenting with clinical symptoms of traumatic brain injury
* Traumatic brain injury verified on cranial computed tomography or magnetic resonance imaging
* Written informed consent from patients or their next of kin according to the patient's cognitive status

Exclusion Criteria:

* patients ≥90 years of age
* patients who did not accept follow-up visits or were unable to complete follow-up assessments
* patients with incomplete information
* patients who did not obtain written informed consent
* patients with concomitant cancer or other serious illnesses
Estimation of Kidney Function Through Combination of Renal Biomarkers in Blood and Urine of Healthy Infants and Children.

To characterize the relationship of renal biomarkers (Creatinine, albumin, Cystatin C, NGAL, beta-trace protein, beta-2 microglobulin, and uromodulin) between each other and the variation over age, measured in serum and urine of healthy children. Unused residual blood and urine samples will be used for testing the renal Parameters.

Despite relevant research in renal biomarkers, there is currently no optimal marker available that reliably quantifies kidney function and indicates kidney injury in its early stages. The combination of two or more biomarkers might be a more promising approach than investigating a single parameter. The relationship of renal biomarkers (creatinine, albumin, Cystatin C, NGAL, beta-trace protein, beta-2 microglobulin, and uromodulin) between each other and the variation over age in infants and children (without chronic kidney disease) is investigated.

The biomarkers in urine samples are explored to find less invasive means of quantifying renal health.

Patients between the age of 0 and 12 years undergoing blood with or without urine sampling as part of their diagnostic workup are eligible for the study. Unused residual blood and urine samples will be used for testing the renal parameters.

Eligibility Criteria:
Inclusion Criteria:

* healthy patients for elective surgery, requiring venous access via peripheral venous canula

Exclusion Criteria:

* chronic kidney disease
* acute kidney failure (stage 2 or above as defined by Kidney Disease Improving Global Outcomes (KDIGO) consensus 2012))
* sepsis
* shock
* major haemorrhage
* second or third degree burns
* liver failure
* chronic diseases with effecting the kidney (systemic lupus erythematosus, amyloidosis)
Post-operative Analgesic Effects of Dexamethasone Added to Bupivacaine in Transversusabdominis Plane (TAP) Block

To compare the postoperative analgesic effect of two doses of dexamethasone added to bupivacaine in ultrasound-guided TAP block for inguinal hernia repair.

The transverses abdominis plane block (TAP block) is a regional anesthesia technique that provides analgesia following abdominal surgery . The TAP block significantly reduces pain after open inguinal hernia repair, regardless of whether it is used as a primary anesthesia technique or as a complement to general anesthesia, spinal or local anesthesia .

The investigators designed this study to compare the postoperative analgesic effect of two doses of dexamethasone( 4 mg and 8 mg ) added to bupivacaine in ultrasound-guided TAP block for inguinal hernia repair.

Eligibility Criteria:
Inclusion Criteria:

* Patients' age from 18 to 60 years old,
* physical status ASA I-II (by the American Society of Anesthesiologists),
* Elective unilateral open inguinal hernia repair,
* Under spinal anesthesia

Exclusion Criteria:

* age \< 18 or \> 60,
* American society of anesthesiologist (ASA)class III-IV,
* known allergy to amino amide local anesthetics,
* skin infection and diseases at site of injection.
* coagulation disorders,
* patients with body mass index (BMI) \> 35 kg/m2,
* scrotal hernias,
Pilot Evaluation of Peer-support Coaching to Increase Adherence to Online Self-help for College Mental Health

This study aims to test if coaching can improve program adherence to an online mental health program in college students if delivered by undergraduate peers. Participants will be randomly assigned to either receive phone coaching, text message coaching, or no coaching. It is hypothesized that participants who receive phone coaching will exhibit greater adherence to the provided online mental health program than participants who receive text message coaching or no coaching.

The purpose of this study is to test the feasibility and effectiveness of an innovative peer support coaching model for college students. The primary aim of the coaching model is to increase participants' adherence to ACT Guide, an online mental health program. To test the effects of peer-support coaching on ACT Guide adherence rates and outcomes, the investigators will conduct a randomized controlled trial with three conditions (peer-support phone coaching, peer-support text message coaching, and a no support control group) using a sample of 300 Utah State University undergraduate students. Individuals who indicate interest in participating will complete an automated online workflow through Qualtrics which will include informed consent, baseline assessment, ACT Guide registration, and randomization into one of the three conditions. Participants will then gain access to a research version of ACT Guide, either with no additional support, with concurrent peer-support coaching, or with concurrent peer-support text messaging based on their randomly assigned condition. Coaching will take place over 10 weeks, with the post assessment being administered 10 weeks after baseline assessment. The investigators hypothesize that participants will adhere to coaching (80% completing ≥ 6 coaching calls; 80% responding to texts \> 6 weeks), be satisfied with coaching (M = 5 "agree" on a 6-point self-reported coaching satisfaction scale), and that coaching fidelity will be maintained (80% of audited coaching calls/texts meeting criteria for fidelity). The investigators also hypothesize that participants who receive peer-support phone coaching will complete more ACT Guide modules and will report greater improvements in mental health relative to both the text messaging and no support conditions, and that the text messaging condition will perform greater in this regard than the no support condition.

Eligibility Criteria:
Inclusion Criteria:

* USU undergraduate student
* Fluent in English

Exclusion Criteria:

* Must have not used ACT Guide in the past
Study of Chemoradiotherapy Combined With Cetuximab in Nasopharyngeal Carcinoma

This is an open, multicenter phase Ⅱ clinical trial. The purpose of this study is to evaluate acute toxicity and efficacy of cetuximab (C225) combined with IMRT + neoadjuvant chemotherapy in advanced T stage of nasopharyngeal carcinoma. Besides, to figure out the relationship between patient outcome and EGFR gene copy number, expression and mutation.


Eligibility Criteria:
Inclusion Criteria:

* Histologic diagnosis of nasopharyngeal carcinoma
* Range from 18～69 years old
* T3-4，N0-2，M0 (AJCC 2009)
* KPS ≥ 80
* Nonmetastatic diseases
* WBC count ≥ 4×109/L，Hemoglobin ≥ 100g/L， platelet count ≥ 100×109/L
* ALT or AST \< 1.5×ULN、bilirubin \< 1.5×ULN
* 0Serum creatinine \< 1.5×ULN

Exclusion Criteria:

* Distance metastases
* Previously treated (surgery，chemotherapy, radiation therapy，EGFR targeted therapy or immunotherapy)
* Second malignancy within 5 years
* Precious therapy with an investigational agent
* Uncontrolled seizure disorder or other serious neurologic disease
* ≥ Grade Ш allergic reaction to any drug including in this study
* Clinically significant cardiac or respiratory disease
* Creatinine clearance \< 30ml/min
* Drug or alcohol addition
* Do not have full capacity for civil acts
* Severe complication, active infection
* Concurrent immunotherapy or hormone therapy for other diseases
* Pregnancy or lactation
A Pilot Study of Shockwave Therapy in HSP

This is a pilot study of the efficacy, safety, and tolerability of shockwave therapy for the treatment of spasticity in Hereditary Spastic Paraplegia.

This is a pilot study investigating the efficacy, safety, and tolerability of Extracorporeal Shockwave Therapy (ESWT) for the treatment of lower-extremity spasticity in the setting of Hereditary Spastic Paraplegia (HSP). 8 participants will be treated with shockwave therapy targeting sites of maximal spasticity in the lower extremities for 3 sessions at a rate of once per week. Participants will fill out baseline questionnaires assessing their overall wellbeing and function prior to treatment session 1, and will complete follow-up questionnaires at visit 2, visit 3, a phone call within 7 days of visit 3, and 8 weeks after initial visit. Total time enrolled in the study will be 8 weeks

Eligibility Criteria:
Inclusion Criteria:

1. Over the age of 18
2. Diagnosis of genetically-confirmed HSP
3. Score of 2, 3 or 4 on the PLSFRS walking question
4. 30 days stable dosages of oral spasticity medications prior to screening and throughout study participation
5. Able to come to site for treatment sessions
6. Able to understand all study procedures

Exclusion Criteria:

1. Diagnosis of any other neurological disorder that may impact gait
2. Lower motor neuron (LMN) disease or combined UMN and LMN
3. Non-ambulatory or requiring two-person assistance for any functional ambulation on most days
4. Less than 3 months of symptoms
5. Have received SWT within the past 3 months
6. Prior botulinum toxin injection within 3 months or planning to receive botulinum toxin injections during the study participation for spasticity management
7. Presence of an intra-thecal baclofen pump
8. Women who are pregnant. This is due to the fact that pregnancy will reduce participants activity after the treatment and the safety concerns related to shockwave treatment.
9. Patients with neuropathy affecting sensation to pain
10. Patients with a known underlying cardiac disease that could be affected by shockwave therapy
11. Concomitant intake of another clinical trial or other off-label experimental treatments that might influence study outcomes
12. Presence of unstable psychiatric disease, cognitive impairments, or dementia impairing study participation
13. Presence of other chronic neurological diseases such as Alzheimer's Parkinson's, stroke, or multiple sclerosis
14. Personal history of narcotic overuse for chronic pain or substance abuse or dependence
15. Contraindications for ESWT, including local skin breakdown, infection, malignancy or other soft tissue pathology at sites of planned treatment.
A Study of a New Investigational Medicinal Product to Treat Patients with Advanced or Metastatic Solid Tumors

W0101 combines a cytotoxic compound to a monoclonal antibody targeting a receptor commonly overexpressed in many cancers.

The development of antibody-drug conjugates takes advantage of the specificity of the mAb while augmenting its ability to produce a cytotoxic effect. The expected benefits of antibody-drug conjugation are enhancement of cytotoxicity in target cells and limiting toxicities of cytotoxic drugs in normal tissues.

This is a First In Human study, multicenter, open label study divided into 2 parts: an initial dose escalation phase (I) followed by expansion cohort(s) phase (II).

Eligibility Criteria:
Inclusion Criteria:

Dose escalation phase (cohort A1 and A2)

1. Male or female subjects age ≥ 18 years
2. Subjects with histologically or cytologically confirmed advanced or metastatic solid tumors (excluding lymphoma) , unresponsive to standard treatment or for whom no standard treatment is available or appropriate
3. ECOG performance status 0 or 1
4. Adequate bone marrow, renal, hepatic at screening and at Baseline
5. Subject must have measurable diseases as per RECIST v1.1 criteria

Exclusion Criteria:

1. Symptomatic brain metastases, CNS tumors
2. Symptomatic motor or sensory peripheral neuropathy (≥ grade 2)
3. Subjects having ophthalmologic abnormalities
4. Active serious systemic disease (infection,organic or dysmetabolic desease)
5. Left ventricular ejection fraction (LVEF) \< 45% as determined by MUGA scan or echography at screening
6. QTc \> 470 msec on screening ECG or congenital long QT syndrome
7. Biologic therapy (including ADCs ≤ 4 weeks before first study treatment administration)
Efficacy and Safety of HRS-1780 Tablets and Henagliflozin Proline Tablets in Patients With Chronic Kidney Disease

The objective of this study was to evaluate the efficacy of HRS-1780 tablets or Henagliflozin Proline tablets in patients with chronic kidney disease by evaluating UACR change from baseline to Week 13


Eligibility Criteria:
Inclusion Criteria:

1. Men and women aged 18-75 years old
2. Body mass index (BMI) ≥18.0 and \<50.0 kg/m2 at the screening visit
3. Subjects treated with an angiotensin-converting enzyme inhibitor (ACEI) and/or angiotensin receptor blocker (ARB) for at least 3 months without any adjustments to this therapy for at least 4 weeks prior to the screening visit
4. Diagnosed with chronic kidney disease, and the estimated glomerular filtration rate (eGFR) was ≥30 and \<90 mL/min/1.73 m2 calculated using the CKD-EPI formula at the screening visit
5. Urinary albumin/creatinine ratio (UACR) was ≥300 and \<3000 mg/g for at least 2 times on different days detected by the local laboratory; at the screening visit
6. HbA1c \<9.0% at the screening visit

Exclusion Criteria:

1. A known or suspected allergy to the investigational drug or its components or excipients;
2. Receive a potent inhibitor/inducer of cytochrome P450 3A4 (CYP3A4) within 7 days prior to screening;
3. Within 4 weeks before screening, the following drugs could not maintain the stable regimen: diabetes drugs, hypertension drugs, non-steroidal anti-inflammatory drugs, endothelin receptor antagonists;
4. Receive SGLT-2 or SGLT-1/2 inhibitors within 4 weeks before screening
5. Received glucagon-like peptide-1 receptor agonist (GLP-1RA) within 8 weeks before screening
6. Received systemic glucocorticoid therapy within 3 months before screening
7. Received immunosuppressive drugs or biological agents
8. Received any other study drug treatment within 3 months or 5 half-lives prior to screening
9. Severe hypertension that was not controlled at screening visit or random visit (systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg), or systolic blood pressure \<90 mmHg
10. Have urinary tract infection or/and genital infection at the screening visit or random visit, or have a history of repeated urinary tract infection or/and genital infection
11. Diagnosed or suspected polycystic kidney disease, lupus nephritis, anti-neutrophil cytoplasmic antibody (ANCA) -associated vasculitis, acute glomerulonephritis, bilateral renal artery stenosis;
12. Acute kidney injury or dialysis treatment within 6 months before screening
13. Received kidney transplant, or plan to receive kidney transplant during the trial