cyrilzakka/clinical-trials huggingface dataset:
PTC Study to Evaluate Ataluren in Combination With Ivacaftor

The purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with nonsense mutation cystic fibrosis

Cystic Fibrosis (CF) is a life threatening genetic disorder resulting from mutations found in the CF gene known as the cystic fibrosis transmembrane conductance regulator or CFTR. This defect prevents correct chloride absorption in and out of the cells The purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation

Eligibility Criteria:
Inclusion Criteria:

1. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial.
2. Age ≥6 years
3. Body weight ≥16 kg
4. Diagnosis of cystic fibrosis and documentation of the presence of a nonsense mutations of the CFTR gene, as determined by historical genotyping
5. Ability to perform a valid, reproducible spirometry with demonstration of a forced expiratory volume in 1second (FEV1) ≥30% and ≤90% of predicted for age, gender, and height.
6. If the subject is sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration
7. Willingness and ability to comply with all study procedures and assessments.
8. Currently being administered ivacaftor, either alone (Kalydeco) or in combination with lumacaftor (Orkambi)

Exclusion Criteria:

1. Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 2 weeks prior to screening.
2. Ongoing participation in any other therapeutic clinical trial.
3. Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 2 weeks prior to screening.
4. Ongoing inhaled tobramycin therapy.
5. Ongoing immunosuppressive therapy (other than corticosteroids up to 10mg/d equivalent of prednisone)
6. Ongoing warfarin, phenytoin, or tolbutamide therapy.
7. History of solid organ or hematological transplantation.
8. A history of positive hepatitis B surface antigen test, hepatitis C antibody test, or human immunodeficiency
9. Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 4 weeks prior to screening.
10. Pregnancy or breast-feeding.
11. Current smoker or a smoking history of ≥10 pack-years (number of cigarette packs/day × number of years smoked).
12. Prior or ongoing medical condition (eg, renal failure, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.

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Post-transplantation Cyclophosphamide for Haploidentical Transplant From Maternal or Collateral Donors

In "beijing model" for haploidentical transplant, the rate of acute graft-versus-host disease (GVHD) from maternal or collateral donors was significantly higher than that from other kins. To reduce the GVHD incidence from maternal or collateral donors, post-transplantation cyclophosphamide is planned to be added into the protocol in "beijing model".


Eligibility Criteria:
Inclusion Criteria:

* haploidentical patients from maternal or collateral donors

Exclusion Criteria:

* uncontrolled infection before transplant
Clinical Trial of Fei Re Qing Granules in the Treatment of Elderly Community-Acquired Pneumonia

This study aims to evaluate the clinical efficacy of traditional Chinese medicine formula granules (Fei Re Qing Granules) in the treatment of elderly community-acquired pneumonia, establish a treatment protocol, and obtain high-level clinical evidence.

This study evaluates the efficacy and safety of Fei Re Qing Granules for elderly community-acquired pneumonia (CAP) through a multicenter, randomized, double-blind, placebo-controlled trial.

Key elements of the trial include:

Design: Conducted across multiple centers, comparing treatment and placebo groups.

Participants: Patients aged 65-80 years diagnosed with CAP according to both Western and Traditional Chinese Medicine (TCM) diagnostic criteria.

Intervention:

Test group: Fei Re Qing Granules administered alongside standard CAP treatments.

Control group: Placebo granules matching the appearance and composition of the active granules, plus standard treatments.

Outcomes:

Primary efficacy indicator: Time to clinical stability. Secondary indicators: Symptom improvement, hospital stay length, and imaging-based inflammation resolution.

TCM-specific indicators assess syndrome improvements and integrate modern scoring methods (e.g., CURB-65).

Study Duration: Two 7-day treatment cycles with 12-week follow-ups. This trial seeks to establish robust evidence for integrating Fei Re Qing Granules into TCM-based CAP management strategies

Eligibility Criteria:
Inclusion Criteria:

* Inpatients diagnosed with community-acquired pneumonia (CAP) according to the diagnostic criteria.
* Diagnosed with Phlegm-Heat Obstructing the Lungs syndrome based on Traditional Chinese Medicine (TCM).
* Aged ≥65 and ≤80 years, regardless of gender.
* Onset of illness within 72 hours.
* Clinical symptoms must include at least two of the following four primary。symptoms: cough, sputum production, dyspnea, and pleuritic chest pain.
* Voluntary participation and signed informed consent.

Exclusion Criteria:

* Patients with altered mental status, consciousness disorders, dementia, or psychiatric disorders.
* Patients requiring invasive mechanical ventilation or endotracheal intubation.
* Patients with conditions such as pleural effusion, bronchiectasis, active pulmonary tuberculosis, pulmonary abscess, lung cancer, asthma, or chronic obstructive pulmonary disease (COPD).
* Patients with neuromuscular diseases or those bedridden for long periods, posing a risk of aspiration.
* Patients with cancer, severe cardiovascular diseases (acute myocardial infarction, heart failure stage III or above), or severe liver and kidney diseases (e.g., cirrhosis, portal hypertension with variceal bleeding, dialysis, or kidney transplantation).
* Patients participating in other drug clinical trials or known to be allergic to the study medication.
Use of Activated Recombinant Human Factor VII in Cardiac Surgery

This trial is conducted in Africa, Asia, Europe, South America and the United States of America (USA).

The trial is planned to investigate the safety and efficacy of NovoSeven® in the management of post-operative bleeding in patients following cardiac surgery.


Eligibility Criteria:
Inclusion Criteria:

* Post-operative bleeding according to pre-defined criteria for critical bleeding
Bioavailability Study of 2 Oral Formulations of ALXN1840

The study will assess the relative bioavailability of 2 different formulations of ALXN1840 in healthy participants.

This is a two-way crossover study consisting of 2 dosing periods assessing a test and reference formulation of ALXN1840. A dose-proportionality parallel group design extension period will be conducted following completion of the two-way crossover period of the study and will assess 5 different ascending doses of ALXN1840. There will be at least a 14-day washout following doses between Periods 1 and 2 and also at least a 14-day washout following the dose in Period 2 and the following dose in the Dose-Proportionality Extension Period.

Safety will be assessed throughout the study.

Eligibility Criteria:
Inclusion Criteria:

* No clinically significant history or presence of electrocardiogram findings
* Body weight ≥50 to ≤100 kilograms (kg) and body mass index 18 to \<32 kg/meter squared for all participants
* Willing and able to follow protocol-specified contraception requirements

Exclusion Criteria:

* History or presence of clinical and/or laboratory disorders
* Abnormal blood pressure, defined as supine blood pressure ≤90/60 millimeters of mercury (mmHg) or \>140/90 mmHg
* Lymphoma, leukemia, or any malignancy within the past 5 years
* Alanine aminotransferase, aspartate aminotransferase, or total bilirubin \> upper limit of normal
* Serum copper or serum ceruloplasmin below lower limit of normal
* Hemoglobin \<130 grams (g)/liter (L) for males and hemoglobin \<115 g/L for females
* Significant allergies
* Smoker
Expanded Natural Killer Cells Following Haploidentical HSCT for AML/MDS

The study examines the application of expanded natural killer cells (NK cells) following haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) for AML or MDS. Haplo-HSCT is a preferred treatment option for patients with AML or MDS without a HLA-matched donor. With administration of cyclophosphamide post-transplant , the safety of the procedure is similar to a HSCT from a HLA-identical donor. Relapse of AML/MDS represents a serious problem following haplo-HSCT. NK cells are immune cells able to destroy tumor cells. Their potency has been established particularly in the setting of a haplo-HSCT. In the current study, study participants undergoing haplo-HSCT will receive expanded NK cells from their respective stem-cell donors following haplo-HSCT. The primary goal of the study is to establish the safety and feasibility of this approach. In addition, the activity of the NK cells will be examined.


Eligibility Criteria:
Inclusion Criteria:

Patient:

* \>18 years of age
* No HLA-matched related or unrelated donor available
* AML or MDS-EB with indication for a haplo-HSCT according to the guidelines of the University Hospital Basel Stem Cell Transplant Team
* Judged by the transplant physicians to have adequate organ function and no contraindications to haplo-HSCT
* Available related haploidentical donor
* Written informed consent

Donor:

* \>18 years old, haploidentical parent, sibling or other relative
* Donor suitable for cell donation and apheresis according to standard criteria
* Written informed consent

Exclusion Criteria:

Patient:

* APL diagnosis
* Presence of relevant (mean fluorescence intensity \>2000) donor-specific anti-HLA antibodies
* Pregnancy
* Necessity of immunosuppression apart from GvHD prophylaxis

Exclusion Criteria:

Donor:

• Pregnancy
A Study of Baricitinib in Participants With Rheumatoid Arthritis

This post-marketing study is designed to compare the safety of baricitinib versus tumor necrosis factor (TNF) inhibitors with respect to venous thromboembolic events (VTEs) when given to participants with rheumatoid arthritis (RA).


Eligibility Criteria:
Inclusion Criteria:

* Participants must have at least one of the following characteristics:

  * Documented evidence of a VTE prior to this study
  * At least 60 years of age
  * A body mass index (BMI) greater than or equal to 30 kilograms per meter squared (kg/m²), or
  * Age 50 to less than 60 years and BMI 25 to less than 30 kg/m²
* Participants must have an inadequate response or intolerance to at least 1 disease-modifying antirheumatic drugs (DMARD) (synthetic or biologic)

Exclusion Criteria:

* Participant must not have prior use of a Janus kinase (JAK) inhibitor or have received more than 1 prior TNF inhibitor that was:

  * discontinued for IR (lack or loss of efficacy) for RA, or
  * discontinued for intolerance (AE) when used for any indication
* Participants must not be pregnant or breastfeeding
* Participants must not have had more than one VTE
* Participants must not have cancer
* Participants must not have active herpes zoster, serious infection, active tuberculosis, or any other serious illness
* Participants must not have had a live vaccine within four weeks of study start
* Participants must not have participated in any other clinical trial within four weeks of study start
* Participants must not have a history of IV drug use, other illicit drug abuse, or chronic alcohol abuse in the past year
Investigational Agent AG-013736 In Combinations With Standard Of Care Treatments For Patient's With Advanced Solid Tumor

To determine the best dose of this investigational agent AG-013736 in combination with various standard of care treatments for advanced solid tumors.


Eligibility Criteria:
Inclusion Criteria:

* Advanced solid tumors suitable for treatment with Taxanes, with or without carboplatin, or treatment with Capecitabine, Gemcitabine/Cisplatin. or Pemetrexed/Cisplatin

Exclusion Criteria:

* Tumors abutting or providing support for blood vessels
* Any significant gastrointestinal abnormalities or active bleeding.
Comparing Inhalation and Total Intravenous Anesthesia Methods

comparison of Anesthesia by Target-Controlled Infusion and Inhalation Methods on Pediatric Patients Undergoing Inguinal Hernia Repair: Effects on Patient Outcomes

COMPARİSON OF THE EFFECT OF ANESTHESİA APPLİED BY TARGET CONTROLLED INFUSİON METHOD AND INHALATİON ANESTHESİA METHOD ON PATİENT OUTCOMES İN PEDİATRİC PATİENTS UNDERGOİNG INGUİNAL HERNİA REPAİR

Eligibility Criteria:
Inclusion Criteria:

* Children aged 3-12 years
* Patients weighing over 10 kilograms
* Patients who will undergo inguinal hernia surgery
* ASA (American Society of Anesthesiologists) classification I and II pediatric patients.

Exclusion Criteria:

* Absence of systemic illness.
* ASA (American Society of Anesthesiologists) classification III pediatric patients.
Impact of Noninvasive Targeted Pulsed Electromagnetic Field (tPEMF) on Opioid Use, Pain, and Joint Function Following Total Knee Arthroplasty

The purpose of this study is to determine the effectiveness of pulsed radio frequency (PRF), a type of electromagnetic field treatment with the Ivivi Torino II for reducing post-operative narcotic use in patients recovering from Total Knee Arthroplasty.

Surgeons are continually looking for means to minimize postoperative pain following TKA in order to reduce or eliminate the need for opioid pain medications. Reductions in pain and edema along with improvements in range of motion encourage quicker recovery of joint function and normal activities. Pulsed radio frequency (PRF), a type of electromagnetic field treatment is proven to effectively modulate postoperative pain and edema in plastic surgery. Therefore, PRF has potential as an adjunct therapy for patients recovering from TKA.

The purpose of the current double-blind, randomized, sham-controlled clinical trial is to determine the effectiveness and safety of the Ivivi Torino II, a device that delivers pulsed radio frequency (PRF), a type of electromagnetic field treatment, in patients recovering from TKA for treatment of severe knee OA.

Eligibility Criteria:
Inclusion Criteria:

* Age 40 to 80 years
* Primary diagnosis of knee OA
* Appropriate candidate for TKA
* Ability of the participant to comprehend the full nature and purpose of the study including possible risks and side effects
* Consent to the study and willing to comply with study product and methods including follow-up

Exclusion Criteria:

* Body mass index \> 40 kg/m2
* Scheduled for bilateral TKA
* Advanced hip, spine, or ankle osteoarthritis that significantly limits ambulation
* Subjects who have any implanted metallic leads, wires, or systems (e.g. pacemaker, implantable cardioverter-defibrillator)
* Use of narcotic pain medication for any condition in the last 1 month prior to surgery
* Self-reported diagnosis of post-traumatic or inflammatory arthritis (e.g. rheumatoid or psoriatic)
* Pregnant or lactating female
* Participation in any clinical trial in the past 30 days
* Vulnerable populations including prisoners and nursing home residents
* Any medical condition that, in the opinion of the investigator, may compromise patient safety or confound the assessment of treatment effectiveness
ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE

The primary objective of this study is to assess the efficacy of a single dose of ravulizumab IV compared with placebo in reducing the risk of the clinical consequences of AKI (MAKE) at 90 days in adult participants with CKD who undergo non-emergent cardiac surgery with CPB.


Eligibility Criteria:
Inclusion Criteria:

* Participant weighs ≥ 30 kg
* Planned non-emergent sternotomy with CPB procedure for the following surgeries:
* Multi-vessel CABG
* Valve replacement or repair; ascending aorta surgery permitted if combined with aortic valve replacement/repair
* Combined CABG and valve surgery; inclusion of single-vessel CABG when combined with valve replacement/repair is permitted
* Known CKD for at least 90 days (CKD Stage 3A, 3B, or 4)

Exclusion Criteria:

* Emergency or salvage cardiac surgery is expected at screening or randomization, as assessed by the investigator.
* Single-vessel CABG without valve surgery is planned.
* Off-pump surgery is planned (eg, surgery without CPB).
* Recipient of a solid organ or bone marrow transplantation.
* Cardiogenic shock, hemodynamic instability, use of intra-aortic balloon pump, extracorporeal membrane oxygenation, or left ventricular assist device within 72 hours of randomization.
* Active systemic bacterial, viral, or fungal infection within 14 days prior to randomization.
* History of unexplained, recurrent infection.
* Any use of KRT or presence of AKI within 30 days of randomization
* Use of any complement inhibitors, or plasmapheresis or plasma exchange within the year prior to Screening, or planned use during the course of the study.
* Participant is not willing to be vaccinated against N meningitidis or is unwilling to receive prophylactic treatment with appropriate antibiotics , if needed
* History of or unresolved N meningitidis infection.
Use of Low Cost Prostheses to Improve Upper Extremity Function in Children With Cerebral Palsy

The purpose of the study is to assess the effectiveness of an upper extremity prosthesis in improving the upper extremity function of children with cerebral palsy who have limited use of their hands. Twelve children, aged 4-17 years, who have cerebral palsy and limitations in their ability to use their hands, will be enrolled. All participants will be fitted with a 3D printed arm/hand prosthesis and receive 8 occupational therapy sessions. Each subject will be evaluated pre-treatment, post-occupational therapy sessions and at 6 months follow-up. The evaluation will include assessment of (1)passive and active arm/hand movement and (2)functional hand skills using several standardized tests. The results from the pre-treatment and the two post-treatment evaluations will be compared.


Eligibility Criteria:
Inclusion Criteria:

* Diagnosis: cerebral palsy
* 4-17 years old
* MACS levels III - V
* Active movement of wrist or elbow

Exclusion Criteria:

* MACS levels I, II
* Botox or orthopedic surgery in past 6 months
* Severe contractures
* Lack of voluntary arm motion
Anaesthesiological Routine Care for Thrombectomy in Cerebral Ischaemia

Endovascular thrombectomy is the standard of care for acute ischaemic stroke due to large-vessel occlusion. Current guidelines for periprocedural anaesthesiological care give gross recommendations on management of stroke patients during recanalization, but lack detailed information.

To determine how anaesthesiologists support endovascular thrombectomy with regard to anaesthetic technique, choice of substances, haemodynamic management, and ventilation. With a multivariate analysis, the investigators will look for the factors of anesthetic management that are independently correlated with a good or bad outcome.

see brief summary

Eligibility Criteria:
Inclusion Criteria:

* Endovascular stroke treatment involving anaesthesia care

Exclusion Criteria:

* In-hospital onset of stroke
* Inclusion in an interventional study concerning the anaesthesia protocol
* Age under 18 years
Therapeutic Lifestyle Modifications to Reduce Burden of Chronic Disease

The investigators previously established that there was a high burden of disease among all categories of staff at the University of the West Indies, St. Augustine and in many ways, this group is typical of the working class of the Caribbean population. Many had poor lifestyle behaviors, 23.8% had Metabolic Syndrome (MS) with many having one or more MS components. Since most time is spent at work, the workplace is understandably the most logical location to address issues pertaining to health, disease prevention and wellness and on-site programs have yielded many health and other benefits to employees and employers alike. Onwards of the 1980s, numerous studies have been done on work-site intervention programs and these have shown that if a company is able to identify employees at risk for developing health problems and then intervene, health risks and the resulting medical problems are reduced. Such a program, if tailored to meet the needs of participants, may yield similar benefits among the staff of the University of the West Indies.

To conduct a randomized controlled intervention of lifestyle modifications among individuals with a high burden of metabolic syndrome components such as high body mass index, high fasting blood glucose and abnormal lipid profile.

A 6-month intervention is designed for implementation to detect a 20% reduction in MS components with 90% confidence. Participants (n=148) had 2 or more MS components and will be randomly assigned to intervention or control group. Intervention will comprise individual diet counseling, an exercise plan and monthly lifestyle workshops. Controls will receive printed material on healthy eating and lifestyle modification.

Eligibility Criteria:
Inclusion Criteria:

* having 2 or more metabolic syndrome components

Exclusion Criteria:

* having less than 2 metabolic syndrome components
Trial of Etoricoxib (Arcoxia) Taken Prophylactically to Prevent Ramadan Headache

The investigators intend to study whether the use of the pain medication etoricoxib (Arcoxia) taken just before the Ramadan fast will prevent or lessen headache that some people get while fasting. The investigators hypothesize that etoricoxib will reduce the number of people getting headache, more than placebo. The investigators will do this by giving participants in the study either real medication or placebo (sugar pill) and comparing the results. The investigators will study this over two weeks. The first week one group will get the medicine and the other the placebo. The second the groups will switch. Neither the subjects nor the investigators will know who is in which group.


Eligibility Criteria:
Inclusion Criteria:

* Age 18 -65.
* Patients will be enrolled if they declared that they intended to complete the dawn to dusk Ramadan fast, and had demonstrated their commitment by doing so for the previous three years (unless medically prevented.)
* Patient states that he/she typically suffered from headache during fasting in the past.

Exclusion Criteria:

* Patients with a history of severe cardiovascular disease, diabetes, asthma, COPD, hypertension, or a history of bleeding, particularly in the gastrointestinal tract
* Patients who have experienced bronchospasm, acute rhinitis, nasal polyps, angioneurotic oedema, urticaria, or allergic-type reactions after taking acetylsalicylic acid or NSAIDs including COX-2 (cyclooxygenase-2) inhibitors
* Pregnant or lactating patients. Sexually active women of childbearing age will be included only if using birth control
* Severe hepatic dysfunction
* Estimated renal creatinine clearance \<30 ml/min
* History of peptic ulcer, erosive gastritis, esophagitis or inflammatory bowel disease
* Congestive heart failure
* Patients with hypertension whose blood pressure is persistently elevated above 140/90mmHg and has not been adequately controlled Established ischaemic heart disease, peripheral arterial disease, and/or cerebrovascular disease
* Cancer or any other malignant disease
* History of medication overuse. Overuse of analgesics or antimigraine drugs according to the IHS criteria (chapter 8)