cyrilzakka/clinical-trials huggingface dataset:
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

OBJECTIVES:

I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism

II. To assess the safety and tolerability of cholic acid

Investigational Plan:

A Phase III, open label, single arm, nonrandomized, non-comparative, compassionate treatment study of cholic acid in the treatment of defects of bile acid metabolism.

The study was begun with a single study site at Cincinnati Children's Hospital Medical Center (CCHMC), but in 2005 was expanded so that compassionate treatment could be provided to additional patients who had been identified with inborn errors of bile metabolism through the center's screening/diagnostic program.

Patients who were screened were contacted and evaluated with respect to the inclusion/exclusion criteria. Signed informed consent by the patient and/or parents/legal guardian was obtained as soon as it is confirmed that the patient met inclusion/exclusion criteria and the parents/guardian would agree for the child to participate in the study.

The primary interventions for the study were:

1. Administration of study drug.
2. Collection of baseline physical exam, vital signs, blood and urine samples for laboratory tests.
3. Collection of periodic physical exam, vital signs, blood and urine samples for laboratory tests during the period of administration of the study drug.
4. Collection of any adverse event information.

Time and Events Schedule:

Baseline:

1. Confirm eligibility
2. Obtain written informed consent from patient and/or parents/legal guardian
3. Collect demographic data and disease and medication history, including family history

   Baseline and Ongoing:
4. Obtain body weight
5. Record adverse events
6. Obtain blood and urine samples for laboratory tests
7. Initiate study drug therapy \& monitor study drug therapy and adjust dose as needed

Eligibility Criteria:
PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Clinical or biochemical evidence of liver disease, unexplained fat-soluble vitamin malabsorption, or peroxisomal dysfunction that compromises bile acid biosynthesis

Inclusion criteria for enrollment were:

* Infants \< age 3 months
* Children presenting for evaluation of cholestasis defined as a conjugated bilirubin \> 2mg/dl or increased serum bile acids
* Older subjects of any age with cholestatic liver disease if urine screens suggested that they had inborn errors of bile acid metabolism
* Confirmation of a diagnosis of an inborn error of bile acid synthesis based upon urine analysis by FAB-MS to determine whether specific abnormalities in bile acid synthesis are indicated
* The patient and/or parent/legal guardian must have signed the written informed consent document before study start.
* The patient must be willing and able to comply with all study assessments and procedures.
Efficacy of Lower Extremity Mirror Therapy on Balance in Children With Hemiplegic Cerebral Palsy

The study will be conducted to assess the efficacy of mirror therapy on balance in children with hemiplegic cerebral palsy.

Hemiplegic cerebral palsy in the most common type of cerebral palsy that has permanent motor disorders and associated with life-long disability. the aim of study to assess the efficacy of mirror therapy on balance in children with hemiplegic cerebral palsy. Seventy child with hemiplegic cerebral were enrolled in this study and were assessed for eligibility. Their aged ranged from eight to twelve years. The children were assigned randomly into two equal groups. Group (a) control group received traditional physical therapy program. And group (b) study group received the same traditional physical therapy program in addition to mirror therapy three times / weak for three successful months. Biodex balance system was used to assess balance pre and post treatment. All children were assisted before and after three months of intervention.

Eligibility Criteria:
Inclusion Criteria:

* Children's aged ranged from eight to twelve years.
* Children participated in this study will from both sexes.
* Their degree of spasticity will ranged from mild to moderate according to Modified Ashworth Scale.
* Children with stable medical and psychological status.
* Children able to follow the verbal commands or instructions.

Exclusion Criteria:

* children with visual or auditory problems.
* Children with history of epilepsy.
* Children with history of surgical interference in lower limbs less than one year.
* Medically unstable children especially with cardiovascular disorders.
* Mentally retarded children.
* un-cooperative children.
Effect of Nigella Sativa in the Treatment of Palmer Arsenical Keratosis

Daily intake of Nigella sativa for 12 weeks is effective in the treatment of palmer arsenical keratosis

Depending on inclusion and exclusion criteria total 40 patients of palmer arsenical keratosis patients (both male and female; age range 18- 60 years) will be recruited from an arsenic affected area. Study population will be divided randomly into two equal groups: one group will receive vitamin E capsules (200 mg) plus placebo and another group will receive vitamin E capsule plus N. sativa capsules (500 mg) twice daily for 12 weeks. Drinking water (50 mL) and urine (50 mL) samples will be collected before starting the study and estimate the amount of total arsenic in order to confirm the diagnosis. Nail (approx. 1 g) and blood (5 mL) samples will be collected both before and completion of the study to see the efficacy and safety of the drug. Clinical examinations will be done at regular interval (two weeks). Clinical features and adverse effects will be recorded using a structured data collection sheet. Plasma cholesterol, vitamin E, and total antioxidant, serum transaminase, billirubin, creatinine levels and arsenic level in nail will be measured to evaluate the effectiveness of N. sativa on palmer arsenical keratosis patients

Eligibility Criteria:
Inclusion Criteria:

* Moderate palmer arsenical keratosis
* Patient drinking arsenic contaminated water (more than 0.05 mg/L) for more than six months
* Subjects those voluntarily agree to participate

Exclusion Criteria:

* Patient received treatment of arsenicosis for the last three months
* Pregnancy
* Lactating mother
* Eczema
* Psoriasis
* Contact dermatitis
* Tuberculosis
* Diabetes mellitus
* Patients with hepatic and renal impairment
Next Generation Sequencing Detection of Lyme Disease

Next Generation Sequencing is capable of sequencing millions of small strands of DNA from a single blood sample, potentially improving its sensitivity compared to PCR testing, which only detects predetermined larger strands of DNA. We will test the ability of NGS to detect Borrelia burgdorferi DNA in the blood of pediatric patients with Lyme disease. We will conduct an observational study of NGS testing on pediatric patients at all stages of Lyme disease. Study involvement will require a single study visit for clinical data collection and blood draw. We will enroll patients at all phases of suspected Lyme disease, collect clinically relevant information, and test for Lyme disease using Next Generation Sequencing and standard Lyme serologic testing. If the patient has multiple erythema migrans, Lyme meningitis, facial nerve palsy, arthritis, or carditis, a B. burgdorferi serum PCR will also be sent. Enrollment and Next Generation Sequencing blood draw will occur before or up to 24 hours after the first dose of antibiotics is administered. We will also study the impact of antibiotics on NGS testing by running the test 6-24 hours after antibiotics are started among a small subset of patients with a multiple erythema migrans rash. Collected data will be analyzed with basic descriptive statistics.


Eligibility Criteria:
Lyme disease subjects (Cases):

Inclusion criteria:

1. Age 1 to \<18 years old
2. The subject has spent time in a Lyme-endemic area during the previous month
3. The subject has a suspected Lyme disease infection

Exclusion criteria:

1. Past infection with Lyme disease
2. Received oral or IV antibiotics within 1 month prior to presentation Note: Subjects may be enrolled if NGS blood test can be drawn \<24 hours after the first dose of Lyme diseasetargeted antibiotics is administered
Optimisation of Mechanical Insufflation:Exsufflation

Patients with neuromuscular diseases (NMD) can suffer from a range of respiratory problems due to respiratory muscle weakness. Cough muscle weakness means secretion clearance from the airways can be problematic, a source of infection, and importantly a cause of death, in this patient group. Therefore, these patients are often supported with devices to aid clearance, such as mechanical insufflation-exsufflation (MIE). Although evidence supports the use of these devices, the optimal technique or settings on the device are not clear. Increasingly, higher pressures are used during MIE and recent work has demonstrated that there may be a physiological benefit to this. However, higher pressures increase the risk of causing lung collapse and may cause detriment to blood flow back to the heart, which is important as NMD patients frequently have concurrent heart muscle weakness. Further, recent work has demonstrated that higher pressures can cause closure of the throat, which is counter-productive in secretion clearance.

The overall aim of this study is to investigate methods to manipulate MIE to improve secretion clearance in patients with NMD. The questions it seeks to answer are:

(i) how can we maximally improve lung recruitment during inspiration, whilst maintaining patient comfort and lower pressures (ii) what is the smallest pressure difference required in expiration to achieve an improvement in cough (iii) do these proposed changes to MIE also cause throat closure (iv) what factors do patients believe contribute to their adherence to MIE therapy?

Patients with slowly progressive or stable neuromuscular diseases will be included in the study. Participation will involve two visits to the Lane Fox Respiratory Unit, each lasting approximately four hours. Patients will be recruited from specialist neuromuscular respiratory clinics by their clinical teams.


Eligibility Criteria:
Inclusion Criteria:

* Stable or slowly progressive neuromuscular disease
* Respiratory muscle weakness (FVC \<60%, snip \<60%, sleep disordered breathing)
* Clinical evidence of respiratory secretions or cough peak expiratory flow \<270 and history of lower respiratory tract infection
* Documented clinical stability by supervising clinician

Exclusion Criteria:

* Rapidly progressive neuromuscular disease (such as motor neuron disease)
* Decompensated respiratory failure (pH \< 7.35)
* Pregnancy
* Aged \<18
* Change in ventilator settings in preceding 4 weeks
* Significant physical or psychiatric co-morbidity that would prevent compliance with trial protocol
Use of Osteopathic Manipulative Treatment for Non-musculoskeletal Conditions

The goal of this observational study is to learn how clinicians use osteopathic manipulative treatment (OMT) for their patients who have problems only related to their muscles, bones, and joints (ie, musculoskeletal problems) compared to patients whose problems involve other body systems (eg, infections, breathing, bowel function). The main questions it aims to answer are:

* What percentage of patients receiving OMT have a non-musculoskeletal problem?
* Is there a difference in the OMT techniques when the patient's problems include a non-musculoskeletal problem compared to patients with only musculoskeletal problems?

Surveys will be completed by the clinicians about 20 unique adult (age 18 or older) patients who have received OMT.


Eligibility Criteria:
Inclusion Criteria:

* Adult patients receiving osteopathic manipulative treatment from a participating treatment provider

Exclusion Criteria:

-
Nasal Inhalation of Pulmozyme in Patients With Cystic Fibrosis and Chronic Rhinosinusitis

Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections.

The prospective placebo controlled cross-over study aims at the evaluation of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.


Eligibility Criteria:
Inclusion Criteria:

* Subject has a confirmed diagnosis of cystic fibrosis based on: 3 positive sweat chloride tests and/or genetic characterisation
* Subject is 5 years of age or older
* Subject has chronic or recurrent rhinosinusitic disorders
* Subject is able to comply with the procedures scheduled in the protocol
* Women of childbearing potential are only included into the study, if they are using an effective method of birth control during the protocol (e.g. implants, combined oral contraceptives, injectables, some IUDs, sexual abstinence or vasectomised partner)

Exclusion Criteria:

* Subject has a critical condition defined as: FEV1 \< 30% and / or SaO2 \< 93% without O2-substitution; need of O2-substitution
* Subject had an ENT surgery within 6 months prior to study
* Subject shows signs of nasal bleeding
* Subject has an ear drum perforation
* Subject had an acute rhinosinusitis or a pulmonary exacerbation at study entry with need of additional systemic antibiotic therapy
* Subject has a new therapy with nasal topic steroids during treatment interval
* Subject has a new systemic steroid therapy
* Subject is unlikely to comply with the procedures scheduled in the protocol
* Subject has a known allergic reaction to the medication
* Subject is pregnant or breastfeeding
* Patient participates in another clinical trial within 30 days prior to study entry or 30 days after end of the study.
Efficacy of the Administration of Colloidal Silicon Dioxide in Tablet Dosage Form in Patients With Acute Diarrhea

The purpose of the study is to demonstrate the antidiarrheal efficacy of colloidal silicon dioxide in tablet dosage form (Carbowhite) in patients with acute diarrhea.

Background: Diarrhea is a medical condition characterized by frequent stool, more than 3 times (and more than 5-7 times in newborns) within the last 24 hours, and/or loose stool. The fecal matter is liquid, its daily mass exceeds 200 g per day, and water content reaches 95%.

Objectives: To demonstrate the antidiarrheal efficacy of colloidal silicon dioxide in tablet dosage form (Carbowhite) in patients with acute diarrhea.

Methods: A randomized clinical trial, double-blind, placebo-controlled, multi-center.

Eligibility Criteria:
Inclusion Criteria:

* signed Informed Consent Form for patient's study participation
* male and female patients at the ages from 18 to 55 years
* acute diarrhea (more than three episodes of liquid stool a day) within 48 hours or less in patients with usually normal stool
* body temperature of ≤ 38 °C
* patient's ability to adequately cooperate in the process of study

Exclusion Criteria:

* aged of \<18 or \>55 years;
* blood or pus in stool;
* body temperature of \>38 °C;
* episodes of acute diarrhea for the last 30 days;
* administration of antidiarrheal products for the last 24 hours;
* salmonellosis (Salmonella of any serotype different from S. typhi and S. paratyphi; ICD-10: A02.0), dysentery (Shigella dysenteriae, Shigella flexneri, Shigella boydii; ICD-10: A03.0-A03.3), colibacillosis (Escherichia coli; ICD-10: A04.0-A04.4) which require administration of antimicrobial products;
* pregnancy, lactation;
* concomitant decompensated diseases or acute conditions which, according to an investigator, may affect study results
* alcoholism and drug abuse;
* participation in any other clinical study.
Reproducibility and Method Comparison Studies of the Quo-Test™ A1C System and the Quo-Lab A1C Test and CLIA Waiver Study of the Quo-Test™ A1C System

The Quo-Test A1C Analyzer and Reagent Test System (Quo-Test A1C System) are intended for the in-vitro quantitative determination of glycated hemoglobin in whole blood samples obtained from fingerstick or venous samples for point-of-care testing. The Quo-Test A1C System is indicated in the management and treatment of diabetes and for monitoring long term glycemic control by diabetics. It is for multiple patient use. Only auto-disabling, single use lancing devices should be used with this system.


Eligibility Criteria:
Inclusion Criteria:

* Aged 18 years or over
* Able to read English
* Read, understood and signed the Informed Consent Form
* Agrees to participate and does not withdraw\\
* Either healthy (without diabetes) or has Type 1 or Type 2 diabetes

Exlcusion Criteria:

• Declines participation or withdraws before study completion
Immunohistochemical Evaluation of DNA Methylation and Histone Modification in H. Pylori Associated Gastric Carcinoma

The goal of this cross-sectional study is to evaluate epigenetic modifications in gastric carcinogenesis, mainly in H. pylori gastritis vs gastric carcinoma. The main question\[s\] it aims to answer are:

* How does H. pylori infection cause gastric cancer?
* Can DNA methylation and other epigenetic changes predict and affect the development of gastric cancer? Formalin-fixed-paraffin-embedded tissue specimens will be obtained and reviewed for comparison between epigenetic changes in H. pylori gastritis and gastric adenocarcinoma.


Eligibility Criteria:
Inclusion Criteria:

* Resected specimens and endoscopic guided biopsies of intestinal type gastric carcinoma, H. Pylori associated gastritis, and non-H. Pylori gastritis with:
* Available full clinical data: (age, sex, complaint, findings in clinical examination).
* Full data of previous endoscopy and radiology.

Exclusion Criteria:

* Cases of diffuse type gastric carcinoma.
* Any case of gastric cancer, H. Pylori associated gastritis, non-H. Pylori gastritis, benign gastric lesions with missing clinical, endoscopic, radiologic or follow-up data.
* Cases with no available H\&E stained slides or formalin fixed paraffin embedded block.
Effects of MRSA Bactericidal Gel To Promote Healing and Eliminate MRSA in cSSTI Vancogel(TM)

Based on personal experience and the literature it is reasonable expectation that Vancomycin is a viable treatment in a direct contact form to eliminate MRSA from open wounds in order to heal the wounds by conventional means. The key question in my research has been to measure the effectiveness of my Vancomycin Gel by culturing the wound, applying the Gel in a controlled manner and then culturing the wound after one week. The end point to achieve in the process, is a clinical response of accelerated healing and negative culture report. Another question to solve is the duration of potency and stability of the Vancomycin gel over time.

Over a prior preliminary 33 month study I have found negative cultures at the end of one week of treatment and have had microbiological studies establishing the same MIC zones in culture media greater than 15mm over a 33 month time frame. The microbiological and HPLC studies are completed at present and approved. I have started the phase 2 studies on August 1,2011 as authorized by the FDA on June 22,2011.

Eligibility Criteria:
Inclusion Criteria:

* MRSA infected open wounds
* Acute and chronic wounds
* Type of wounds: venous stasis,diabetic, pressure, post surgical, post traumatic, and spontaneous
* Infection criteria: Include a positive culture for MRSA
* Location of ulcers: any place on the body
* Diagnosis of MRSA: Based on tissue cultures of MRSA
* Willing and reliable patients
* Study to include only one ulceration no more than 50 square centimeters
* The study to include stages two and three ulcerations

Exclusion Criteria:

* Non-compliant patients
* Patient must accept all issues in consent form
* Non compliance to include failed appointments
* Wounds greater than 50sq. cm
* No wounds deeper than soft tissue
* Ischemic or vascular disease, dermatitis, immune deficiency,or psoriasis
* Allergy to Vancomycin
* Post irradiation ulceration
* Bleeding disorders
* Skin allergies to adhesives and tape
* Ulcers related to cancers
* Multiple wounds
* Stage 4 ulcerations
* Patients in any other trial
* Patients with any other conditions which, in the opinion of the investigator/doctor, would preclude participation in the study.
Biomarkers for Pain in Spinal Cord Injury (SCI) Patients

The investigators propose to compare plasma protein profiles for SCI patients with/without chronic neuropathic pain in order identify biomarker(s) that are associated with this medical condition. Secondly, the investigators propose to identify a temporal relationship to initial SCI at which these biomarkers manifest.

Our working hypothesis is that sustained alterations in specific inflammatory molecules are associated with chronic neuropathic pain following SCI, and that their plasma levels can serve as biomarkers to identify patients at risk for the development of neuropathic pain.

Additionally the investigators are collecting skin tissue biopsy samples from patients following acute and chronic spinal cord injury to create vector-free human iPS cells from fibroblasts by direct delivery of reprogramming proteins.


Eligibility Criteria:
A. Chronic Patients

Inclusion:

1. Two or more years post traumatic SCI with deficit

Exclusion:

1. \< 18 years of age
2. Evidence of brain injury on computed tomography (CT) (SCI patients need to be able to comply with completing the pain survey)
3. Other medical condition that accounts for chronic pain (i.e. diabetic neuropathy, renal insufficiency, multiple sclerosis, HIV associated neuropathy, alcohol-related neuropathy)
4. Temperature \> 100.5°C
5. History of infection within the last 30 days (i.e. UTI, URI, pressure sore)
6. History of Pulmonary Embolus (PE) or deep vein thrombosis (DVT)
7. Inability to obtain informed consent
8. Psychiatric problems (patients need to be able to complete the pain survey)
9. Diagnosis or treatment of cancer in the last 5 years

B. Longitudinal, Prospective Cohort Patients:

Inclusion:

1. Initial traumatic SCI with deficit

Exclusion:

Same as listed above C. Healthy Volunteers Healthy volunteers include gender, age and race matched volunteers able to provide informed consent who have,

1. No significant medical history (pain free)
2. No recent infections
3. Take no medications
4. Fever free
5. Greater than 18 years old