cyrilzakka/clinical-trials huggingface dataset:
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy

The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.

This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of BBM-D101 within 52 weeks after a single intravenous infusion in DMD boys, as well as the long-term safety and efficacy of BBM-D101 for up to 5 years post infusion.

BBM-D101 is gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.

Eligibility Criteria:
Inclusion Criteria:

1. The legal guardian of the subject fully understands the purpose, nature, methods, and possible risks of the study, and signs a written informed consent form;
2. The study includes ambulatory male subjects who are at least 4 years old and less than 8 years old (4 years old ≤ age \&lt; 8 years old) ;
3. Genetically confirmed diagnosis of DMD;
4. Have at least 1 of the following typical clinical signs or laboratory abnormalities of DMD: proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower\&#39;s sign, pterygoid scapula;
5. Ability to cooperate with motor assessment testing, magnetic resonance imaging (MRI) and muscle biopsy according to the requirements of the study.

Exclusion Criteria:

1. Hepatitis B surface antigen (HBsAg) positive, hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥1000U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive or human immunodeficiency virus (HIV) positive;
2. Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.;
3. Left ventricular ejection fraction (LVEF) \&lt;50% or ≥ class III cardiac function defined by New York Heart Association (NYHA);
4. With severe or persistent arrhythmias and congenital heart disease.
5. The subject\&#39;s preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment;
6. With underlying liver disease, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, or hepatic fibrosis ≥ stage 3; or nodules, cysts found by B-ultrasound in the past, or elevated alpha-fetoprotein in laboratory tests during the screening period, etc., and these abnormalities are judged by the investigator to be clinically significant;
Effect of Raised End-Tidal pCo2 on Choroidal Volume

This study is to investigate the relationship between arterial carbon dioxide (CO2) concentration and vitreous pressure on the choroidal volume by integrated intraoperative OCT imaging under eye exams under anesthesia.

Children with normal eyes undergoing an exam under anesthesia or a non-intraocular eye procedure will have one eye examined after research consent is taken. Children have very elastic ocular tissues. When they are having an intraocular procedure positive vitreous pressure can cause the iris to prolapse out of the eye and this can cause intraoperative and postoperative complications. The causes of the positive vitreous pressure have been speculated to be due to an increase in choroidal volume. The Choroid is a layer of blood vessels sitting between the sclera and the retina. Ocular Coherence Tomography (OCT) is an imaging tool that allows an optical section of the retina, choroid and sclera to be examined. The subfoveal choroidal thickness has been shown to be a consistent area that can be measured with reliability that the anatomical site of measurement is consistent. By varying the end tidal CO2 the thickness of the choroid will be measured and at the same time the intraocular pressure taken using a pneumatonometer. The OCT is integrated into the operating microscope and is used to take a scan of the fovea. Three scans will be taken after 5minutes of the particular end tidal pCO2. In order to ensure the choroidal thickness is not being influenced by the axial length of the eye, the axial length will be measured using an ultrasound probe at the beginning of the measurements just once. In order to ensure that the position of the head and body is not influencing the choroidal thickness a spirit level will be used to ensure a flat operating bed at the beginning of the measurements.

Eligibility Criteria:
Inclusion Criteria:

* any child undergoing an eye exam under anesthesia for an ophthalmologic reason.
* children between the ages newborn to 8 years of age.

Exclusion Criteria:

* children who are not having an eye exam under anesthesia.
* children with a known medical diagnosis that affect the eye to be studied
* \* children over the age of 8 years of age.
* \* children with altered cerebral autoregulation, increased intracranial pressure, pulmonary hypertension, or any other condition deemed appropriate by the anesthesiologist.
* children in whom a laryngeal mask airway will not be used
Concurent Chemoradiotherapy in Head and Neck Cancers

To compare the efficacy and toxicities of the combination between weekly docitaxel and cisplatin (every3 week) concurrent with radiation versus the standard concurrent chemoradiotherapy with high dose cisplatin (100mg\\m2) for locally advanced HNSCC

Head and neck cancer is the eighth most common cancer in the United States . For locally advanced HNSCC, concurrent chemoradiotherapy (chemo-RT) is the standard treatment established with multiple randomized trials . The survival benefit of chemo-RT in locally advanced HNSCC has been confirmed by meta-analyses, which revealed a 19% reduction in mortality and an absolute survival benefit of 8% at 5years when chemotherapy was administered concurrently with radiation Despite incremental improvements in local regional control and survival in HNSCC patients treated with chemo-RT, a substantial fraction of patients suffer persistent or recurrent diseases. Combining novel agents with radiation therapy therefore remains of great interest to further improving the treatment outcomes of HNSCC .

Cisplatin is the most commonly used chemotherapeutic agent given concurrently with radiation. However, introduction of additional effective radiosensitizing agents is urgently needed. A significant percentage of patients appropriate for definitive concurrent chemo-RT are not candidates for cisplatin based treatment.

Furthermore, recent data have emerged that cisplatin might not be the most optimal cytotoxic radiosensitizing agent when addition of novel targeted agents to concurrent chemo-RT is evaluated.

Radiation Therapy Oncology Group (RTOG) 0234 is a phase II randomized clinical trial evaluating postoperative radiation plus concurrent docetaxel and cetuximab versus postoperative radiation plus cisplatin and cetuximab for high-risk HNSCC after surgery. The results showed an impressive improvement in overall survival and disease-free survival of the docetaxel arm compared to the cisplatin arm (79% versus 69% and 66% versus 57% respectively . These results are pointing to the possibility that a non-cisplatin based regimen chemo-RT should be explored for further development of novel targeted agents and led to a recently opened phase III randomized RTOG trial, RTOG 1216, which compares postoperative radiation with concurrent cisplatin versus docetaxel versus docetaxel and cetuximab for high-risk HNSCC patients . docetaxel appears to have active antitumor activity in advanced SCCHN . In preclinical studies, docetaxel leads to tumor cell reoxygenation and mitotic arrest with potent radiosensitization.

Eligibility Criteria:
Inclusion Criteria:

* pathologically confirmed squmous cell carcinoma performance status 0-1 adequte renal and hepatic function

Exclusion Criteria:

* poor performance status
* impaired renal or hepatic function

  =Squamous cell carcinoma of salivary gland,parotid and paranasal carcinomas were --0excluded from the study
* patients with ≥ grade 2 pre-existing peripheral neuropathy, history of allergic reactions to the chemotherapeutic agents, and uncontrolled intercurrent diseases as well as HIV positive patients.
Comparison of the Efficacy and Safety of Cresnon® and Crestor® in Patients With Hyperlipidemia

Comparison of the efficacy and safety of Cresnon® and Crestor® in patients with hyperlipidemia

- 8-week, active-controlled, open-label, randomized, parallelgroup, fixed-dose, noninferiority, multicenter, phase 4 design


Eligibility Criteria:
Inclusion Criteria:

* Age ≥ 20
* Persons who have not reached LDL-C targeted level in accordance with the treatment guidelines for dyslipidemia released by Korean Society of Lipidology and Atherosclerosis
Evaluation of the Teen Outreach Program in Chicago Public Schools

The purpose of this study was to determine the impact of TOP® on sexual health behavior.

The cluster-randomized study was conducted in 9th grade classrooms in Chicago Public Schools (CPS) with equal number of schools randomized to intervention and comparison groups for two cohort years. Students were administered a scannable paper survey at three time points: baseline, post-program and 1-year post program. The primary impact analysis used an intent-to-treat approach. Implementation of TOP® was assessed by conducting classroom observations. TOP® was delivered in a variety of classroom settings over the school year by facilitators from CPS and partner organizations.

Eligibility Criteria:
Inclusion Criteria:

* First-time 9th grade student
* Passive parental consent and student assent

Exclusion Criteria:

* Inability to complete survey in languages provided
A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline on their current GC regimen. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.

This is a study that will test the efficacy and safety of Tildacerfont. The first 12-weeks will be a double-blind, placebo controlled, dose ranging study. The following 58-weeks will assess the long term safety of Tildacerfont. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.

Eligibility Criteria:
Inclusion Criteria:

* Male and female subjects over 18 years old, inclusive
* Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented elevated 17-OHP and currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
* Has been on a stable supraphysiologic dose of GC replacement ≥15 mg/day and ≤60 mg/day in HC equivalents
* For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening

Exclusion Criteria:

* Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21 hydroxylase deficiency)
* Has a history that includes bilateral adrenalectomy or hypopituitarism
* Has a history of allergy or hypersensitivity to Tildacerfont, any of its excipients, or any other CRF1 receptor antagonist
* Current treatment with dexamethasone as GC therapy for CAH. Prior treatment with dexamethasone is allowed as long as the transition to an alternative GC regimen (eg, HC, prednisone, or prednisolone) has resulted in a stable dose of GC replacement for ≥1 month before screening.
* Shows clinical signs or symptoms of adrenal insufficiency
A Phase 3 Efficacy Study Of Dimebon In Patients With Moderate To Severe Alzheimer's Disease

No Dimebon clinical data exist yet in patients with disease that has advanced to the moderate-to-severe stage. Therefore, this study evaluates the safety and efficacy of Dimebon in patients with moderate-to-severe AD who are receiving existing background therapy with memantine.

This study was terminated on May 7, 2010 due to modification of the dimebon development plan following the lack of demonstration of efficacy in the completed DIM14 (CONNECTION) Study. The study was not terminated due to any safety findings. Dimebon has been well-tolerated in clinical trials. Demonstration of efficacy for dimebon in Alzheimer's disease is pending completion of the ongoing DIM18 (CONCERT) Study.

Eligibility Criteria:
Inclusion Criteria:

* Are men and women ≥ 50 years of age with a diagnosis of Alzheimers disease.
* Have a Mini-Mental State Exam between 5 and 14 inclusive.
* Have been taking the medication memantine (ie., Namenda) for at least six months prior to this study.
* Must have a caregiver who assists the patient at least five days per week for at least three hours per day, who can accompany patient to study visits, and who has an intimate knowledge of the patient's health states and personal care.

Exclusion Criteria:

* Have taken medicines for Alzheimers disease other than memantine (e.g., donepezil, rivastigmine, galantamine, tacrine) within 2 months prior to this study.
* Dementia other than Alzheimers disease.
* Any medical condition or reason that interferes with the ability of the patient to participate in or complete the trial or places the patient at undue risk, as judged by the study doctor.
Surgical Treatment for the Displaced JakobⅡ Classification Humerus Lateral Condylar Fractures in Children

Multi-center Study on Surgical Treatment for the Displaced JakobⅡ Classification Humerus Lateral Condylar Fractures in Children.

The Displaced JakobⅡ Classification Humerus Lateral Condylar Fractures in Children that were treated with closed or open reduction.

Eligibility Criteria:
Inclusion Criteria:

* Patients with fractures that met these criteria, underwent definitive surgery and had complete clinical and radiographic follow up until fracture union at either institution were included in this study.

Exclusion Criteria:

* Pathologic fractures, and fractures in patients with osteogenesis imperfecta, neuromuscular disorders or any systemic disease that was associated with a predisposition to fractures or altered fracture healing were excluded.
Preoperative Strength Training in Patients With Total Knee Arthroplasty

The purpose of this study is to investigate if preoperative strength training will result in faster recovery and higher level of function six weeks after total knee arthroplasty (TKA).

Reduced strength of the quadriceps muscle is a common clinical finding in patients with Knee Osteoarthritis and this seems to play a central role in the development of the disease. The strength of the quadriceps muscle have been shown to be reduced by 20-40% as compared to age-matched healthy controls and this decrease in muscle strength have been closely associated with a reduced level of function and increase of pain. Furthermore, it has been demonstrated that the preoperative strength of the quadriceps muscle in patients undergoing TKA, is associated with the level of function one year after surgery. Compared to the level before the operation, an additional operation-induced reduction of the strength of the quadriceps muscle up to 60% as has been demonstrated. Presumably, an optimizing of the strength of the quadriceps muscle could counteract the impact of the expected postoperative loss of muscle strength to such an extent that the loss will be of less clinical significance. It is hypothesized that an optimizing of the strength of the quadriceps muscle may have a favorable effect on the postoperative rehabilitation program and shorten the period of convalescence.

Eligibility Criteria:
Inclusion Criteria:

* Planned TKA at the Aarhus University Hospital or Region Hospital Silkeborg,
* Living in Aarhus Municipality,
* Primary knee osteoarthritis or secondary osteoarthritis after meniscectomy or ligament injury

Exclusion Criteria:

* Unstable blood pressure,
* Neuromuscular or neurodegenerative disease,
* Comprehension or dementia problems
Zometa Adjuvant Treatment of Malignant Pleural Effusion Due To Non-Small Cell Lung Cancer

The purpose of this study is to evaluate whether the addition of the bisphosphonate Zometa (zoledronic acid) used along with standard regimens of chemotherapy, will help to control the need for palliative intervention of malignant pleural effusions due to non-small cell lung cancer.

Malignant pleural effusions are common in late stage non-small cell lung cancer and can lead to significantly increased morbidity in this patient population. The majority of patients are symptomatic due to their malignant effusions. The recurrence rates are thought to be quite high overall and may approach 100% without any further treatment.Currently there is no available non-invasive, medical means for controlling the effusions other than systemic chemotherapy.Zoledronic acid has been shown in a mouse model of malignant pleural effusion to decrease fluid accumulation and tumor dissemination while prolonging survival.

Eligibility Criteria:
Inclusion Criteria:

* Histologically documented non-small cell lung cancer, Stages 3B, 4 or recurrent
* Pleural effusion cytologically proven to be malignant
* 0 or 1 prior chemotherapy regimens for non-small cell lung cancer (adjuvant chemotherapy post resection, or concurrent chemo-radiation therapy counts as one regimen regardless of number of agents used.)
* Planning to start chemotherapy for non-small cell lung cancer (treatment regimen at discretion of treating physician but must include one or more of the following agents:cisplatin,carboplatin,docetaxel,paclitaxel, pemetrexed,gemcitabine,vinorelbine) Patients may receive anti-angiogenesis agents (bevacizumab) in addition to chemotherapy, but patients treated solely with tyrosine kinase inhibitors or growth-factor receptor blockers are not eligible.
* Prior radiation therapy is permitted.
* Performance status 0,1,2
* Serum creatinine less than 2.0 or estimated creatinine clearance over 30cc/min by Calcroft/Gault equation
* Estimated life expectancy over 3 months
* Signed informed consent
* Age greater than 18 years
* Patients who have clinical indication for Zometa treatment such as lytic bone metastases or hypercalcemia can be included

Exclusion Criteria:

* Pregnant or lactating
* Patient with concurrent medical or psychiatric illness which would, in the opinion of the investigator, prevent compliance with the study
* Patients who undergo any procedure other than thoracentesis for drainage of effusion.Patients may have had more than one thoracentesis prior to study. Patients who have large bore chest tube placement, permanent transthoracic catheter (Pleurex), medical pleurodesis or thoracoscopy are excluded.
* Current active dental problems including infection of the teeth or jawbone (maxilla or mandibular);dental or fixture trauma, or a current or prior diagnosis of osteonecrosis of the jaw (ONJ), of exposed bone in the mouth, or of slow healing after dental procedures.
* Recent (within 6 weeks)or planned dental or jaw surgery (e.g.extraction, implants).
Investigation of the Efficiency of Spinal Mobilization in Patients With Adolescent Idiopathic Scoliosis

In this study, it was aimed to evaluate the effect of spinal mobilization techniques applied in addition to core stabilization exercises in Adolescent Idiopathic Scoliosis (AIS) patients.

Consecutive adolescents with idiopathic scoliosis, aged 10 to 16 years, without a brace, and with a Cobb curvature of 10 to 25 degrees were randomly divided into two groups using the closed envelope method: Spinal mobilization and core stabilization exercise were applied to the experimental group; Only core stabilization exercises were applied to the control group.

Both groups received interventions twice a week for 10 weeks. Outcome measurements were made before treatment and after 10 weeks of treatment.

This study was a randomized controlled trial in which participants and assessor were blinded. This study was carried out in Amasya University Ruhi Tingiz Physical Therapy and Rehabilitation Hospital Rehabilitation Unit between August 2022 and January 2023. Ethics committee approval was obtained from Amasya University (E-76988455-050.01.04-83314) on 05.08.2022 to conduct the study. Before the study, face-to-face interviews were conducted with the all participants and parents and informed about the subject and purpose of the study. Verbal and written consent was obtained from the all participants and parents.

Sample size: The sample of the study was calculated with the G\*Power 3.1 program, and the effect size (d=1.0) was calculated according to the double-tailed hypothesis method, taking into account the study. (d=1.0; α=0.05; 1-ß=0.80). Confidence interval was determined as 80% and margin of error was 5% (α=0.05). As a result of the calculation, it was determined that there should be 17 adolescent scoliosis patients for the control group, 17 for the experimental group, and a total of 34 adolescent scoliosis patients. Considering the possible data losses of the participants, we planned to include a total of 40 participants, 20 in both groups.

Eligibility Criteria:
Inclusion Criteria:

* Patients diagnosed with AIS
* Age 10-16 years
* Cobb angle was between 10° and 25
* Risser sing did not exceed 4
* Informed consent forms were signed by the parents and children

Exclusion Criteria:

* Used brace,
* Had previous spinal surgery, inferior limb length difference,
* Could not exercise due to another injury/diagnosis,
* Had neuromuscular, psychiatric, cardiovascular, respiratory insufficiency and mental disability