cyrilzakka/clinical-trials huggingface dataset:
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).

This is a multicenter, open, dose-escalation clinical study to evaluate the safety, initial efficacy, and immunogenicity of SKG0201 injection in patients with spinal muscular atrophy type 1 (SMA 1).

Eligibility Criteria:
Inclusion Criteria:

1. Type 1 SMA, defined by bi-allelic mutations in the SMN1 gene.
2. Age 180 days or younger at day of infusion.
3. Clinical history and signs are consistent with type I SMA, that is hypotonia on clinical examination, with delay in motor skills, poor head control, rounded shoulder posture, and joint hypermobility.
4. The legal guardian of the subject understands the purpose of the study, the possible risks and rights of the study, agrees that the subject can participate in the study, complete all research steps, tests and visits, and sign the ICF voluntarily.
5. During the study period, according to the change of the subject's condition, the subject's legal guardian is willing to perform standard treatment requirements as suggested by the researcher.

Exclusion Criteria:

1. Pulse oximetry \< 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.
2. Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards (WHO 2006).
3. Active viral infection with significant signs or symptoms and require systematic hospitalization.
4. In the presence of other severe infections or diseases.
5. Known allergy to prednisolone, other glucocorticoids, or their excipients.
6. Clinically significant abnormal laboratory values prior to administration.
7. Previously used other SMA drugs (such as Spinraza, Evrysdi, Zolgensma, etc.) or participated in clinical studies of other SMA drugs.
8. Had received previous or anticipated major surgical procedures during the study assessment period.
Health ImprOvement Program of Bone in China

Fracture risk factors have long been identified as key factors in osteoporosis and fragility fractures. The WHO recommended Fracture risk assessmenttool (FRAX) applies clinical risk factors to assess the absolute risk of osteoporotic fractures in each individual. Our preliminary study suggests that FRAX estimates may underestimate the risk of fractures in the Chinese population. In order to optimize the risk prediction model of osteoporotic fractures, a treatment threshold and its optimal cutting point were initially established based on the data of the health management Center of the Second Xiangya Hospital of Central South University.


Eligibility Criteria:
Inclusion Criteria:

* Dual-energy X-ray bone mineral density measurement was performed in the health management center of our hospital in the physical examination subjects over 40 years old. Have independent civil capacity and agree to participate, and sign informed consent

Exclusion Criteria:

* Under 40 years of age， Patients who had undergone hip replacement or lumbar surgery and could not perform dual-energy X-ray BMD testing; Patients who have been treated with effective anti-osteoporosis drugs or have a history of malignant tumors.
Quality of Life on Elderly Patients with Low Grade Non-Hodgkin Lymphoma

Aims of this study are to describe the variation of QoL (Quality of Life) during the clinical management of low-grade lymphoma in elderly subjects and to identify the most important factors at diagnosis and during treatment with an impact on QoL (Quality of Life).

This is a prospective, multicenter, non-interventional study aiming at investigating on the changes in QoL (Quality of Life) during the clinical management of low-grade lymphoma in elderly subjects and to possibly identify the most important factors at diagnosis and during treatment with an impact on QoL (Quality of Life).

The study plans to enroll 150 patients with a local histologically confirmed diagnosis of indolent non-Hodgkin lymphoma fulfilling all the inclusion/exclusion criteria who provide written informed consent will be included in the study. Any type of treatment is allowed by the study (immunotherapy, immunochemotherapy at full or reduced dose, radiotherapy).

All patients must undergo Comprehensive Geriatric Assessment (CGA) before starting any protocol treatment.

Baseline, post-treatment and follow-up instrumental assessments and response evaluation is done as part of the routine clinical management for patients with indolent NHL (Non Hodgkin Lymphoma) . Response evaluation and all other assessments planned for the EOT (end of treatment) should be planned also in case of early withdrawal, according to clinical practice.

HRQoL (Health-related Quality of Life) evaluation will be done at time of informed consent form signature prior to treatment start, at the end of the treatment (ranging from 1 month to 6 months according to the duration of the treatment) and after 1 year from the start of the therapy.

QoL (quality of life) assessment includes all scales of the EORTC-QLQ-C30 (European Organisation for Research and Treatment of Cancer Quality of life Questionnaire-Core 30) and the FACT-Lym-LymS (Functional Assessment of Cancer Therapy - Lymphoma - lymphoma-specific symptoms questionnaire).

Eligibility Criteria:
Inclusion Criteria:

* Local histological diagnosis of indolent B cell non-Hodgkin lymphoma: follicular lymphoma, marginal zone lymphoma (nodal, MALT (Mucosa-Associated Lymphoid Tissue), splenic), lymphoplasmacytic lymphoma/ Waldenström macroglobulinemia)
* Age ≥ 70 years
* Patient candidates to receive treatment for lymphoma
* Patient previously untreated for lymphoma
* Evaluation of Comprehensive Geriatric Assessment at baseline
* Ability to provide informed consent: subject understands and voluntarily signs an informed consent form approved by an IEC (Independent Ethics Committee), prior to registration into the study

Note: Patient treated in the context of a clinical trial is admitted

Exclusion Criteria:

* none
Modified Percutaneous Achilles Tendon Lengthening by Triple Hemisection for Achilles Tendon Contracture

This study aims to evaluate the efficacy and safety of this new therapeutic method, which is based on the percutaneous sliding technique with three hemi-cuts in the tendon.

This study aims to provide a new therapeutic theory which bases on the percutaneous sliding technique with three hemi-cuts in the tendon. In this study, Achilles tendon parenchymal at distal level is hemisected through the most distal incision, Achilles tendon parenchymal at proximal level is hemisected through the middle incision, then Achilles tendon lengthening is accomplished by the sliding tendon under forceful dorsiflexion. It can better achieve soft tissue balance and reduce the recurrence of Achilles tendon contracture when Achilles tendon strength in plantarflexion is weakened. Thus in this study, the partial aponeurosis of gastrocnemius is cut off at most proximal incision in order to weaken Achilles tendon strength in plantarflexion.

Eligibility Criteria:
Inclusion Criteria:

* Patients older than three years old;
* Severe Achilles tendon contracture and equinus;
* The time after Achilles tendon contracture more than 6 months ;
* Initial Achilles tendon lengthening.

Exclusion Criteria:

* Patients younger than 3 years;
* The course of Achilles tendon contracture is within 6 months;
* Patients who have suffered from the Achilles tendon lengthening;
* Obvious contracture of other soft tissues occurs in combination, such as compartment syndrome or extensive scar tissues in calf;
* Osteotomy or tendon transposition is needed.
* Ankle joint is damaged obviously, such as Charcot's joint or Hemophilic arthritis;
* Skin soft-tissue infection in the lower limb.
* Initial Achilles tendon lengthening.
Safety Study of iCo-007 Intravitreal Injection to Treat Diabetic Macular Edema

The goal of the study is to evaluate the safety and tolerability of 4 different doses of iCo-007 Intravitreal Injection in patients with diffuse diabetic macular edema.


Eligibility Criteria:
Inclusion Criteria:

1. Men or women 18 years of age or older
2. Confirmed diagnosis of diabetes mellitus type I or II (both, insulin or non-insulin dependent)
3. Subjects who have moderate to severe non-proliferative diabetic retinopathy (NPDR), as defined by ETDRS criteria, and diffuse DME within 300 microns from the center of the macula in the study eye (with or without cystoid changes) confirmed by FA.
4. Retinal thickness must be at least 250 microns in the central subfield, as shown by OCT
5. Best-corrected visual acuity between 60 and 15 letters measured with ETDRS charts (approximately 20/63 to 20/500 with Snellen charts)
6. Woman of child-bearing potential must not be pregnant or lactating, must have a negative pregnancy test at screening (serum). Both men and women are required to practice an adequate method of birth control
7. Able and willing to sign an approved informed consent form and return for all scheduled study visits

Exclusion Criteria:

1. Subjects with macular or perimacular edema secondary to an etiology other than diabetes mellitus
2. Subjects with concurrent retinal diseases or conditions in the study eye (including conditions requiring urgent PRP), glaucoma (or ocular hypertension), retinal vascular occlusion, exudative age-related macular degeneration (drusen permitted), tear (rip) of the retinal pigment epithelium, a vitelliform-like lesion of the outer retina (e.g. pattern dystrophies or basal laminar drusen), idiopathic parafoveal telangiectasis or retinal-lesion anastomosis
3. Subjects who have any additional ocular disease or condition which could compromise treatment safety, visual acuity or interfere with assessment of the macular edema, such as ocular inflammation, amblyopia, anterior ischemic optic neuropathy, media opacity, cataract
4. Subjects who have diffuse DME with severe capillary non-perfusion (avascular zone diameter \>1,000 microns)
5. Allergy to fluorescein dye
6. Subjects who had intraocular surgery, photocoagulation, intravitreal or subfoveal injection, topical or systemic steroids or who received any experimental treatment as part of another clinical trial within the last 3 months of the study start (in case of Anecortave within the last 6 months, any panretinal photocoagulation prior to study start)
7. Subjects with uncontrolled diabetes (glycosylated hemoglobin Hb A1c \>12%)
8. Subjects with systolic blood pressure higher than 180 mm Hg or diastolic above 100 mm Hg, with or without anti-hypertensive treatment
9. Subjects with congestive heart disease or any unstable cardiac condition
10. Subjects with clinically significantly impaired renal function (serum creatinine \>2.0 mg/dL) and/or moderate to severe hepatic impairment (subjects with \>2.5 times the upper limit of the normal range for aspartate transaminase \[AST\], alanine transaminase \[ALT\], or total bilirubin)
Maternal Hyperoxygenation in Congenital Heart Disease

A pilot study investigating the safety and feasibility of chronic maternal hyperoxygenation in the setting of fetal congenital heart disease

The aim of the study is to investigate the safety and feasibility of chronic maternal hyperoxygenation in pregnancies affected by fetal congenital heart disease, specifically those pregnancies in which the fetus has single ventricle physiology with aortic arch obstruction. The treatment has a potentially neuroprotective effect on the fetus. This would be desirable as the neurodevelopmental outcomes of the survivors of this form of congenital heart disease are significantly below normal. However, transplacental oxygen has not been tried in this setting, and so before embarking on a trial, the investigators need to establish that the treatment is safe and feasible. This will be accomplished by recruiting subjects which meet the eligibility criteria and commence treatment at the time of diagnosis, usually in the second trimester, and by comparing outcomes with a historical cohort with CHD diagnoses with no oxygen intervention. The oxygen will be delivered to the mother via nasal prongs continuously at a rate of up to 4 L/min. Oxygen concentrators will be supplied to the subjects' homes, and a range of portable devices will also be provided to allow them to continue with usual activities of daily living. A series of follow up appointments will be arranged to check the status of the mother and fetus. Mothers will be invited to keep a diary of their adherence to the treatment. A range of routine clinical and research data on the condition of the fetus and newborn will be collected.

Eligibility Criteria:
Inclusion Criteria:

* Between 20-32 weeks gestation
* Diagnosis of fetus with a single ventricular ventricular heart
* Delivering at Mount Sinai Hospital
* Written informed consent

Exclusion Criteria:

* Opting for termination of pregnancy/ comfort care
* Normal exclusions for MRI (e.g. claustrophobia, cardiac pacemaker, etc.)
* BMI \>37.0
* Infections/ anemia
* Smoker
* Serious cardiorespiratory co-morbidities
Gene Expression During Chemotherapy in Patients With Newly Diagnosed Acute Myeloid Leukemia Treated With Choline Magnesium Trisalicylate

RATIONALE: Studying samples of blood from patients with cancer in the laboratory may help doctors learn more about changes that occur in cancer cells. It may also help doctors understand how cancer cells respond to treatment with choline magnesium trisalicylate.

PURPOSE: This pilot clinical trial is studying gene expression in cancer cells during chemotherapy and the safety of choline magnesium trisalicylate in treating patients with newly diagnosed acute myeloid leukemia.

OBJECTIVES:

Primary

* Determine temporal changes in leukemic cell NF-kB activity when choline magnesium trisalicylate is administered during induction chemotherapy in patients with newly diagnosed acute myeloid leukemia.
* Determine toxicities of this regimen in these patients.

Secondary

* Determine patterns of leukemic cell gene expression in patients treated with this regimen.
* Determine if NF-kB modulation results in enhanced apoptosis in patients treated with this regimen.

OUTLINE: This is an open-label, pilot study.

Patients receive oral choline magnesium trisalicylate every 8 hours for 48 hours or dexamethasone every 6 hours for 48 hours plus choline magnesium trisalicylate every 8 hours for 48 hours during induction chemotherapy as determined by the primary physician.

Blood is collected at baseline, 24 hours, and 48 hours to assess for changes in NF-kB expression, apoptosis, and gene expression in leukemic cells.

PROJECTED ACCRUAL: A total of 24 patients will be accrued for this study.

Eligibility Criteria:
DISEASE CHARACTERISTICS:

* Histologically confirmed acute myeloid leukemia

  * Newly diagnosed disease
* Presence of cytogenetic abnormalities must be determined by standard cytogenetics with or without FISH studies
* Leukemic blast count \> 5,000/mm³ of peripheral blood
* No acute promyelocytic leukemia (M3)

PATIENT CHARACTERISTICS:

* ECOG performance status 0-3
* Bilirubin \< 2.0 times upper limit of normal (ULN)
* AST \< 3.0 times ULN
* Creatinine \< 1.5 times ULN
* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
* No uncontrolled psychiatric illness that, in the opinion of the principal investigator, would preclude study compliance
* No other concurrent medical condition that would preclude study compliance
* No allergies to any investigational drugs and/or chemotherapeutic agents
* No upper or lower gastrointestinal (GI) related hemorrhage within the past 6 months as determined by endoscopy

  * No clinical diagnosis of GI bleeding requiring blood transfusions

PRIOR CONCURRENT THERAPY:

* No prior induction therapy
* No prior chemotherapy for acute leukemia
* No concurrent medications that would preclude study compliance
The Effect of Red Blood Cells Transfusion in Trauma Patients

We will evaluate the effect of red blood cell (RBC) transfusion on fatal and non fatal events according to baseline risk of death in an international cohort of trauma patients (CRASH-2 trial). The following outcomes will be considered: death from all causes, death due to bleeding, death due to multiorgan failure, death due to vascular occlusive events and non vascular occlusive events. Non fatal outcomes include: myocardial infarction, stroke, deep vein thrombosis and pulmonary embolism.

Aims: Primary objective: Evaluate the effect of RBC transfusion on all cause mortality according to baseline risk of death due to bleeding. Secondary objectives: Evaluate the effect of RBC transfusion on specific causes of death. Evaluate the effect of RBC transfusion on non fatal vascular occlusive events.

Sample: Patients from the Clinical Randomisation of an Antiﬁbrinolytic in Signiﬁcant Haemorrhage (CRASH-2) trial. The trial included 20,127 trauma patients with, or at risk of, significant bleeding, within 8 hours of injury, and was undertaken in 274 hospitals in 40 countries.

Outcomes: Death from all causes, death due to bleeding, death due to multiorgan failure, death due to vascular occlusive events and non vascular occlusive events. Vascular occlusive events include: myocardial infarction, stroke, deep vein thrombosis and pulmonary embolism. All events were measured at 28 days or hospital discharge Interventions and comparisons: We will compare the effect of red cells transfusion versus no red cells transfusion. We will also evaluate the effect of the number of units of red cells transfusion.

Methods: We will stratify patients according to baseline risk of death from all causes into four strata (\<6%, 6%-20%, 21%-50% and \>50%). We will then evaluate the effect of RBC transfusion according to baseline risk on fatal and non fatal outcomes. Formal statistical test to detect heterogeneity will be conducted analyzing baseline risk as a continuous variable.

Eligibility Criteria:
Inclusion Criteria:

* Patients eligible for inclusion in the trial were "adult trauma patients with ongoing significant haemorrhage, within 8 hours of injury."

Exclusion Criteria:
Rapid Maxillary Expansion and Protraction by Expander Differential Opening EDO in Cleft Lip and Palate Patients

maxillary expansion in cleft palate patient with expander and using face mask.

patient with cleft palate and maxillary constriction, deficiency will be treat using expander with differential opening which has two screws on anteriorly and another posteriorly positional in midline palate anteriorly as much as possible.

rapid maxillary expansion will be using one turns in day(two turns in the morning and two turns in the evening).

with petit face mask for maxillary protraction and superimposition CBCT will be taken between before and after treatment .

Eligibility Criteria:
Inclusion Criteria:

* ● Patients of both sexes.

  * Cleft lip and palate patients.
  * All patient age range 8-12 years.
  * All the case showed maxillary arch constriction and deficiency.
  * All patients had maxillary permanent first molars.
  * Primary lip adhesion and palatal closure.

Exclusion Criteria:

* • Absent maxillary permanent first molars.

  * Patients had received any surgical assisted rapid maxillary expansion, maxillary protraction or fixed orthodontics before.
  * Less than two dental unit beside maxillary first molar.
  * Uncooperative patients/parents.
A Study of Ixekizumab (LY2439821) in Participants With Moderate-to-Severe Plaque Psoriasis Naive to Systemic Treatment

The main purpose of this study is to evaluate the efficacy of ixekizumab compared to fumaric acid esters (FAE) and methotrexate (MTX) in participants with moderate-to-severe plaque psoriasis who are naive to systemic treatment.


Eligibility Criteria:
Inclusion Criteria:

* Present with moderate-to-severe chronic plaque psoriasis based on a diagnosis of chronic psoriasis for at least 6 months before baseline.
* Participants who are candidates for systemic therapy and who are naive to systemic treatment for psoriasis.
* Have a (PASI score \>10 or BSA \>10) and DLQI \>10 at screening and at baseline.

Exclusion Criteria:

* Have predominant pattern of pustular, erythrodermic, and/or guttate forms of psoriasis.
* Have received systemic nonbiologic psoriasis therapy.
* Have prior, concurrent, or recent use of ixekizumab or any other biological psoriasis therapy.
* Have any condition or contraindication as addressed in the local labeling for MTX or FAE.
* Presence of significant uncontrolled cerebro-cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematologic, neurologic, or neuropsychiatric disorders or abnormal laboratory values at screening.
* Have severe gastrointestinal disease, oral ulcer, or known, active gastrointestinal ulcer.
* Have had a serious infection or are immunocompromised.
* At screening, participants with significant, present, or early liver disease, e.g., explained by alcohol consumption or hepatic insufficiency.
Trial Comparing Three Different Devices for Kidney Stone Removal During Percutaneous Surgery

The investigators plan to compare three different devices that are used to break up large kidney stones during surgery for removal to see if one is faster or more efficient than the others.

Large kidney stones can be removed by a surgical procedure where a small puncture is made through the back into the kidney (percutaneous nephrolithotomy or PNL) but the stones usually need to be broken into smaller pieces before they can be removed through the small surgical incision. There are a number of FDA approved devices commercially available used to break up the stones including pneumatic, ultrasonic, and a combination of the two. Each technology has advantages and disadvantages.

In the last few years there have been new, improved versions of these devices introduced. These improved versions have addressed previous issues of probes clogging or breaking and cumbersome handpiece design. These include the Cyberwand (Cybersonics, Erie, PA), a dual probe ultrasonic device, Swiss Lithoclast Select (EMS, Switzerland) combining the pneumatic and ultrasonic modalities, and a novel device by LMA (Gland, Switzerland) called the StoneBreakerTM, a portable pneumatic device powered by CO2 cartridges.

We propose to compare each of these devices in a randomized study to see if one is better than another at removing kidney stones quickly and efficiently.

Eligibility Criteria:
Inclusion Criteria:

* Scheduled to undergo percutaneous nephrolithotomy for at least one kidney stone measuring 2 cm or greater
* Stone easily visible/measurable on plain abdominal imaging (KUB) or CT scan preoperatively
* Age 18 years or older
* Able to give informed consent

Exclusion Criteria:

* Size of single largest stone less than 2 cm
* Pregnancy
* Active urinary tract infection
* Extracorporeal shockwave lithotripsy within the last three months
* Complex stone anticipating multiple access sites
* Stones that are not clearly able to be measured on KUB or CT scan
* Inability to give informed consent
* Age less than 18 years