cyrilzakka/clinical-trials huggingface dataset:
Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

This is a single-arm, open label, single-dose clinical study to evaluate the safety, tolerability and efficacy of BBM-F101 injection in the pediatric Fabry disease participants up to 52 weeks after infusion, and the long-term safety and efficacy of BBM-F101 injection up to 5 years after infusion.

BBM-F101 injection is an adeno-associated virus (AAV) gene therapy product for the treatment of pediatric Fabry disease.


Eligibility Criteria:
Inclusion Criteria:

1. The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written informed consent; If the participant is \>= 8 years old, the participant must also agree to participate in the study and sign a written informed consent;
2. Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
3. Males or females aged ≥7 years and \<18 years old;
4. Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
5. Participants had at least one of the clinical manifestations for Fabry disease;
6. Acceptable capsid antibody titers;
7. Acceptable anti α-Gal A antibody titers;
8. Acceptable laboratory values;
9. Participant's legal guardian and participant with good cooperation and compliance;
10. Use of reliable contraception methods during the study for adolescence.

Exclusion Criteria:

1. Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA), positive for hepatitis C virus RNA (HCV-RNA), positive for HIV or syphilis;
2. Have potential liver diseases;
3. Heart failure and severe arrhythmias;
4. Severe allergic reactions for enzyme replacement drugs or other medications;
5. Acute/chronic infections;
6. End-stage renal disease;
7. Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period;
8. Have received gene therapy or used other investigational drugs within four weeks prior to dosing;
9. Other conditions that make the participant not eligible for the study according to the investigator.
An Evaluation of Efficacy of the GlideScope Cobalt Video Baton for Intubation on Children Weighing Less Than 10 Kilograms

The purpose of this study is to collect relevant airway data using the GlideScope Cobalt Video Baton 1 and 2 for intubation in a large cohort of neonates and infants weighing 10 kg or less.

Neonates and infants requiring endotracheal intubation and in which the attending anesthesiologist decided to use the CGS-CVB will be included in this observational study. Data will be collected by one of the investigators or the research assistant/coordinator and recorded on a data sheet The data from these forms will be then entered by the investigator or research assistant/coordinator into a database maintained only for this study.

The decision of using the GS-CVB is at the discretion of the attending anesthesiologist. Consequently if consent would be required the anesthesiologist might feel compelled or biased to use the device against his will. This will also require that all possible patients's should be consented which will put a serious strain on the study personal.

Eligibility Criteria:
Inclusion Criteria:

* Neonates, infants and children scheduled for surgical procedures under general anesthesia and requiring endotracheal intubation
* Patients age 0 - 2 years
* Weight up to 10 kg
* American Society of Anesthesiologists physical status I-III

Exclusion Criteria:

* Patients at risk of pulmonary aspiration,
* Increased intracranial pressure
* Those with congenital cardiac diseases or
* Those with hemodynamic instability
Observational Study to Assess Natural History in Cockayne Syndrome Patients

This is an Observational Study of children under the age of 11 diagnosed with Cockayne Syndrome to assess the natural progression of Cockayne Syndrome disease, with special attention to hearing and physical changes in length or height, weight, head circumference, and arm span during standard treatment.

The primary analytical objective is to determine the rate of linear growth over a 6-month period in children \< 2 years of age and over a 12-month period in children ≥ 2 years of age.


Eligibility Criteria:
Inclusion Criteria:

* Pediatric patients with a documented diagnosis of CS, as suggested by clinical features and possible confirmation by genetic consultation and analysis
* Age of participation:

  * At least 12 months of age at the time of signing Informed Consent/Assent
  * Female patient's age will not be greater than 10 years of age at the time of signing Informed Consent/Assent
  * Male patient's age will not be greater than 11 years of age at the time of signing Informed Consent/Assent

Exclusion Criteria:

* Severe contractures or physical deformities that in the opinion of the investigator would prevent accurate measurement of height, length and ulna length
* Patients that have taken growth hormone or growth hormone related medications within 12 months prior to the date of Informed Consent/Assent
* Known history of inborn error of hyperprolinemia (Type I or Type II)
* Clinical features present at the time of initial screening that are associated with the terminal phases of the natural progression of CS suggesting safe travel and completion of the study and its assessments to be unlikely as judged by the Investigator, including any of the following:

  * Continuous or intermittent dependence on supplemental oxygen at home during the prior six months
  * Two or more hospitalizations for pneumonia during the prior 12 months;
  * A documented net weight loss of at least 10%, which has not been recovered and which includes a significant net weight loss (beyond the estimated error of the measurement) over the most recent 6 months, despite intensive nutritional support including the use of gastrostomy tube feedings
* Presence of scoliosis with a Cobb's angle of 30º or greater
A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Hereditary Transthyretin Amyloidosis using data that already exist in patients' medical records.


Eligibility Criteria:
Inclusion Criteria:

* Age ≥18 years at diagnosis
* Diagnosed with ATTRv-CM, mixed phenotype
* Treated with tafamidis (VYNDAQEL 80 mg \[four 20-mg tafamidis meglumine capsules\] orally once daily or VYNDAMAX 61 mg \[one 61-mg tafamidis capsule\] orally once daily) for ≥6 months
* Have had ≥1 pre- and ≥2 post-treatment neurologic assessments

Exclusion Criteria:

* History of organ transplant
* Wild-type TTR genotype
* Individuals who are non-ambulatory
* Prior treatment with any disease-modifying therapy (investigational or approved) alone or in combination, except tafamidis, as either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally once daily or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally once daily
* Peripheral neuropathy attributed to causes other than ATTR amyloidosis (e.g., diabetes mellitus, B12 deficiency, HIV infection)
Evaluation of Contact Lens Saline Solutions With Scleral GP Contact Lens Wearers Experiencing Fogging

Evaluation of saline solutions used with scleral rigid gas permeable contact lens wearers experiencing debris in the post lens tear reservoir

One-month, approximately 36 subjects enrolled (\~12/site), randomized, double-masked crossover trial.

Existing scleral rigid gas permeable (RGP) contact lens wearing subjects are randomly assigned (block) to different arms of the trial and receive different saline solutions for use sequentially for 2-week periods. The subjects will continue to wear their existing scleral GP lenses if lens fit is acceptable following an initial evaluation. Subjects will agree to complete a survey administered daily (via email) that will address contact lens wear time and subjective symptoms.

Eligibility Criteria:
Inclusion Criteria:•

* Daily wear large diameter (scleral) rigid gas permeable contact lens wearers in design with acceptable fit
* 18 years old or above.
* Agree to and sign Informed Consent.
* Correctable to at least 20/40 distance visual acuity in each eye.
* Presence of debris or "fogging" in post lens tear film reservoir (PLTR).

Exclusion Criteria:

* Extended wear contact lens wearers
* Require concurrent ocular medication.
* Grade 2 or more slit lamp findings (does not include fogging assessment).
* Eye injury or surgery within twelve weeks immediately prior to enrolment for this trial.
* Currently enrolled in an ophthalmic clinical trial.
* Pregnant or lactating
* Evidence of active ophthalmic disease or abnormality including glaucoma, chorioretinitis, central retinal artery or vein occlusion, intraocular or ocular infection.
Characterizing Orthodontic Tooth Movement With Photographic Scans

Characterizing orthodontic tooth movements in real time by using photographic scans to monitor teeth movement. The photographic scans will also be compared to 3D model scans. Canine retraction over the course of 1 orthodontic visit will be measured.

The accuracy and reliability of Dental Monitoring scans is be evaluated and compared to that of iTero digital scans during maxillary canine retraction. The magnitude and type of tooth movement occurring in a one month period in patients undergoing maxillary canine retraction will be measured. Patients undergoing orthodontic treatment in the Center for Orthodontic Care at UConn Health will be evaluated for the inclusion criteria. A total of twenty patients whose orthodontic treatment plan required extraction of at least one premolar in the maxillary arch will be recruited. The patient will be required to take photographs and video scans as dictated by Dental Monitoring (DM) on the first and final days of the study period (four weeks), while iTero scans are taken by the provider in clinic on these same days. Patient will also be periodically taking photographic scans at home using Dental Monitoring. The DM scans taken by the patient will be compared to the iTero digital scans taken by the provider. This can be done by superimposing the stereolithographic (STL) files created from each scan and assessing the amount of deviation between the 3D models. The types of tooth movement occurring during canine retraction in buccolingual, mesiodistal, and incisogingival planes will be quantified using Doctor Dashboard software available in DM. DM's 3D matching technology allows superimposition of teeth from each 3D model created per scan. Each tooth and its movements are graphed in a detailed manner to provide precise information about its type of movement.

Eligibility Criteria:
Inclusion Criteria:

* Patients with fixed orthodontic appliances
* Treatment plan requiring extraction of at least one maxillary premolar
* Male and female
* Permanent dentition from first molar to first molar in the maxilla
* Teeth with normal crown morphology
* Fully erupted canines
* Above 11 years of age
* Patients with any cellular phone with a camera

Exclusion Criteria:

* Lack of extraction in orthodontic treatment plan
* Syndromic patients
* Cleft lip/palate patients
* Removable appliances
The Efficacy and Safety of Batroxobin Combined With Anticoagulation in Cerebral Venous Sinus Thrombosis

Our previous clinical case observations showed that batroxobin combined with anticoagulation therapy can improve the sinus recanalization rate in patients with CVST, shorten the hospital stay, and increase the neurological score of patients. Its main mechanism is to inhibit thrombosis after reducing fibrinogen, and to dissolve thrombus.

To further explore the safety of batroxobin combined with anticoagulation therapy for CVST, an open-label, randomized controlled (1: 1), single-center, prospective study was used. Further study on the safety and effectiveness of batroxobin combined with anticoagulation for CVST.


Eligibility Criteria:
Inclusion Criteria:

1. Cerebral venous thrombosis, confirmed by cerebral angiography (with intra-arterial contrast injection), magnetic resonance venography or computed tomographic venography.
2. Severe form of CVST with a high chance of incomplete recovery, as defined by the presence of one or more of the following risk factors

   1. Intracerebral hemorrhagic lesion due to CVST
   2. Mental status disorder
   3. Coma (Glasgow coma scale \< 9)
   4. Thrombosis of the deep cerebral venous system
3. Uncertainty by the treating physician if ET or standard heparin therapy is the optimal therapy for the patient.

Exclusion Criteria:

1. Conditions associated with increased risk of bleeding
2. Any thrombolytic therapy within last 7 days
3. Cerebellar venous thrombosis with 4th ventricle compression and hydrocephalus, which requires surgery
4. Contraindication for anti-coagulant or batroxobin treatment 1)documented generalized bleeding disorder 2)concurrent thrombocytopenia (\<100 x 10E9/L) 3)Fibrinogen below 100mg /dl 4)documented severe hepatic or renal dysfunction, that interferes with normal coagulation 5)uncontrolled severe hypertension (diastolic \> 120 mm Hg) 6)known recent (\< 3 months) gastrointestinal tract hemorrhage (not including hemorrhage from rectal hemorrhoids)
5. Any known associated condition (such as terminal cancer) with a poor short term (1 year) prognosis independent of CVST
6. Clinical and radiological signs of impending transtentorial herniation due to large space-occupying lesions (e.g. large cerebral venous infarcts or hemorrhages)
7. Recent (\< 2 weeks) major surgical procedure (does not include lumbar puncture) or severe cranial trauma
8. Previously legally incompetent prior to CVST
9. Severe renal impairment
10. Active liver disease
11. Pregnancy, nursing or planning to become pregnant while in the trial
12. Further exclusion criteria apply
13. No informed consent.
A Study In Healthy Volunteers To Estimate The Pharmacokinetics Of Four Modified-Release Formulations Of Dimebon (Latrepirdine)

This study will evaluate four different modified release formulation to estimate the amount of dimebon available to the body relative to the current dimebon formulation that is given three times a day. The results of this study will help inform and guide further formulation development efforts with the ultimate goal of reducing dose frequency to once-a-day or twice-a-day.


Eligibility Criteria:
Inclusion Criteria:

* Healthy male and/or female subjects between the ages of 18 and 55 years, inclusive.

Body Mass Index (BMI) of 17.5 to 30.5 kg/m2; and a total body weight \>50 kg (110 lbs).

Exclusion Criteria:

* Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing).
* Subjects with any history of a previous seizure (including childhood febrile seizures) or convulsion or significant head trauma.
* Subjects with hypersensitivity reactions to dimebon or other antihistamines.
* Any condition possibly affecting drug absorption (eg, gastrectomy).
* Smokers who use greater than 5 cigarettes per day.
* Use of proton pump inhibitors, antacids, and H2-blockers are prohibited for the duration of the study.
* Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of non-hormonal contraception.
AVAdeno Study: An Observational Study of First-Line Avastin (Bevacizumab) in Patients With Adenocarcinoma of the Lung

This prospective observational study will evaluate the efficacy and safety of first-line Avastin (bevacizumab) in combination with platinum-based chemotherapy in different age groups (\<60, 60-69, 70-79, \>80 years) in patients with inoperable advanced, metastatic or recurrent adenocarcinoma non-small cell lung cancer. Patients will be followed for 18 months from the start of first-line therapy.


Eligibility Criteria:
Inclusion Criteria:

* Adult patients, \>/= 18 years of age
* Histologically confirmed inoperable advanced, metastatic or recurrent pre-dominantly non-squamous adenocarcinoma non-small cell lung cancer

Exclusion Criteria:

* Contraindications to Avastin treatment according to the Summary of Product Characteristics
Study of Chronic Fatigue, Depression, and Anxiety Among Hypothyroid Patients Attending Assuit University Hospitals

* Assessment of the prevalence of chronic fatigue, depression, and anxiety among patients with hypothyroidism in Assuit University Hospital.
* Assess the severity of chronic fatigue, depression, and anxiety according to the duration and control of treatment in patients with hypothyroidism.

Hypothyroidism is a prevalent endocrine illness with a global impact, necessitating treatment in extreme instances. Inadequate management of hypothyroidism has been associated with adverse health outcomes, including neurological and musculoskeletal issues,this condition impacting approximately 1-6% of the general population.

Thyroid hormone is essential for the normal development of many human tissues and regulates the metabolism of virtually all cells and organs of the human body throughout life. Overt hypothyroidism is defined by thyroid-stimulating hormone (TSH) levels above the upper limit of the reference range, while levels of free thyroxine (fT4) are below the lower limit of the reference range. The reference range is typically statistically defined by the 2.5th and 97.5th percentiles of the measured circulating thyroid hormone values in populations defined as healthy.

It has been reported that up to one-third of patients presenting with fatigue have thyroid disease, predominantly in women,and fatigue was the most common noted symptom among patients with hypothyroidism.Also patients with thyroid disorders are more prone to developing depressive symptoms.

There are few previous studies that addressed this topic in the Egyptian population.

Eligibility Criteria:
Inclusion Criteria:

* Patients diagnosed with primary hypothyroidism.
* Age from 18 to 60 years.

Exclusion Criteria:

* Patients who were previously diagnosed with any psychiatric diseases.
* Serious and chronic diseases (e.g. anemia, liver, heart or kidney problems).
* consumption of medications that may interfere with thyroid function (e.g., chemotherapy), patients who had a history of any type of cancer, due to the confounding effects of cancer on fatigue as the outcome of interest.
* Women who were pregnant or had given birth in the last 6 months to exclude postpartum thyroiditis and depression.
Virus Specific Cytotoxic T-Lymphocytes (CTLs) for Refractory Cytomegalovirus (CMV)

CMV cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults (CAYA) with refractory cytomegalovirus (CMV) infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), with primary immunodeficiencies (PID) or post solid organ transplant.

Funding Source: FDA OOPD


Eligibility Criteria:
1. Patients with refractory CMV infection post allogeneic HSCT, with primary immunodeficiencies or post solid organ transplant with either

* Increasing or persistent quantitative qRT-PCR DNA copies despite two weeks of appropriate anti-viral therapy AND/OR
* Medical intolerance to anti-viral therapies including:
* ANC \< 500/mm2 secondary to ganciclovir

  * 2 renal toxicity with foscarnet And/or
* known resistance to ganciclovir and/or foscarnet

Consent: Written informed consent given (by patient or legal representative) prior to any study-related procedures.

Performance Status \> 30% (Lansky \< 16 yrs and Karnofsky \> 16 yrs) Age: 0.1 to 79.99 years Females of childbearing potential with a negative urine pregnancy test

Donor Eligibility Related donor available with a T-cell response to the CMV MACS® GMP PepTivator antigen(s).

a. Third Party Allogeneic Donor: If original donor is not available or does not have a T-cell response: third party related allogeneic donor (family donor \> 1 HLA A, B, DR match to recipient) with IgG positive to CMV and/or a T-cell response to the CMV MACS® GMP PepTivator .

AND Allogeneic donor disease screening is complete similar to hematopoietic stem cell donors (Appendix 1).

AND Obtained informed consents by donor or donor legally authorized representative prior to donor collection.

3 Patient exclusion criteria:

A patient meeting any of the following criteria is not eligible for the present study:

Patient with acute GVHD \> grade 2 or extensive chronic GVHD at the time of CMV CTL infusion Patient receiving steroids (\>0.5 mg/kg prednisone equivalent) at the time of CMV CTL infusion Patient treated with donor lymphocyte infusion (DLI) within 4 weeks prior to CMV CTL infusion Thymoglobulin (ATG), Alemtuzumab or T cell immunosuppressive monoclonal antibodies within 30 days Patient with poor performance status determined by Karnofsky (patients \>16 years) or Lansky (patients ≤16 years) score ≤30% CMV retinitis Concomitant enrollment in another experimental clinical trial investigating the treatment of refractory CMV infection.

Any medical condition which could compromise participation in the study according to the investigator's assessment Known HIV infection Female patient of childbearing age who is pregnant or breast-feeding or not willing to use an effective method of birth control during study treatment.

Known hypersensitivity to iron dextran Patients unwilling or unable to comply with the protocol or unable to give informed consent.

Known human anti-mouse antibodies CMV retinitis, meningitis, encephalitis, and/or cerebritis
Intelligent Needle Tip Tracking Using Ultrasound Imaging for Lumbar Plexus Blocks

In a randomized controlled crossover study design, ultrasound guided lumbar plexus blocks will be performed with and without the aid of a needle tip tracking (NTT) system. Specialists in anaesthesiology with average experience in ultrasound guided peripheral nerve block (PNB) techniques will perform the blocks. 27 volunteers will be included. The primary objective is to investigate the effect of the NTT system on performance time, as a measure for improved block performance. Secondary objectives are the effects of the NTT system on dexterity, peripheral nerve block characteristics, subjective experience, and peripheral block performance.


Eligibility Criteria:
Inclusion Criteria:

* American Society of Anesthesiologists (ASA) physical status 1 or 2
* Volunteers that have given informed written consent

Exclusion Criteria:

* BMI \< 18 kg/m2
* BMI \> 35 kg/m2
* Body weight \> 95 kg
* Volunteers that cannot cooperate during the examination
* Volunteers that do not speak or understand Norwegian language
* Volunteers with neurologic disease, nerve- or vascular impairment
* Volunteers with known coagulopathy
* Volunteers that are allergic to Lidocaine or other local anaesthetic agents
* Medications at the investigators discretion
* Volunteers with concomitant medical treatments interfering with PNB treatment
* Skin disease or infection affecting the whole-body surface or within the area of examination
* Any reason why, in the opinion of the investigators, the volunteer should not participate
* Subject participates in a potentially confounding drug or device trial during the course of the study
* Pregnancy and lactation period
* Women of childbearing potential who do not use an effective and secure method for birth control