cyrilzakka/clinical-trials huggingface dataset:
Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)

The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia


Eligibility Criteria:
Inclusion Criteria:

Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."

1. Patient who has been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
2. Patient who has been diagnosed as HPP
3. Documented diagnosis of HPP as indicated by:

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1. Total serum alkaline phosphatase below the lower limit of normal for age
2. Ultrasonographic features of prenatal, characterized by:

1) severe short extremities (femur length \<-4SD in second and third trimesters) 2) extending into the metaphysis (femur metaphysis length or femur length \>0.33) 3) craniotabes 4) Hypoplastic thorax (Thoracic or abdominal circumference \<0.6) (3) Computed tomographic findings of prenatal, characterized by:

1. Generalized decreased ossification
2. Extreme shortening of tubular bones
3. Hypoplastic thorax (4) Radiographic evidence of HPP, characterized by:

1) Flared and frayed metaphyses 2) Severe, generalized osteopenia 3) Widened growth plates 4) Areas of radiolucency or sclerosis (5) Two or more of the following HPP-related findings:

1. History or presence of:

   - Nontraumatic post-natal fracture

   - Delayed fracture healing
2. Nephrocalcinosis or history of elevated serum calcium
3. Functional craniosynostosis
4. Respiratory compromise or rachitic chest deformity
5. Vitamin B6 dependent seizures
6. Failure to thrive
7. Premature tooth loss (6) Patient who have the mutation of tissue non-specific ALP gene 4. Parent or legal guardian(s) must provide written informed consent prior to any study procedures being performed and must be willing to comply with all study-required procedures

Exclusion Criteria:

1. Current evidence of treatable form of rickets
2. Serum calcium or phosphate levels below the normal range
3. Pregnant women and nursing mothers
4. Patient who cannot enforce suitable contraceptive measures during the clinical trial
5. Prior treatment with bisphosphonates
6. Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
7. Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
8. Clinically significant disease that precludes study participation, in the opinion of the Investigator
Integrated Approach in Frail Older People with Atrial Fibrillation

The study will verify if a structured multidisciplinary approach (called iABC), aimed to improve the appropriate management of elderly AF patients with multimorbidity (the i-ABC group), would provide a clear evidence of an improvement in clinical conditions and quality of life compared to usual clinical care. The i-ABC group in AFFIRMO will follow the ABC pathway, focused on three domains: avoid stroke with anticoagulation (with optimized VKA or label-adherent DOAC use); better symptom management; and optimized management of associated cardiovascular and non cardiovascular comorbidities.

The study will be conducted in Bulgaria, Denmark, Italy, Romania, Serbia and Spain .

Controlled study, testing a structured implementation of an appropriate management of elderly AF patients with multimorbidity in clinical practice (adapting the ABC pathway and integrating the means of CGA) versus usual care; it is designed to provide reliable evidence of an active holistic integrated management approach to a common, multimorbid and 'high risk' clinical condition.

AFFIRMO use a novel platform (iABC) in a cluster randomized trial design, randomizing centres to iABC versus usual care.

Centres will be selected in each participating country (Bulgaria, Denmark, Italy, Romania, Serbia and Spain) under the responsibility of National Coordinators on the basis of a demonstrated interest in managing patients with AF. The 8-10 participating clusters in each country (see study size below) will be randomized in a 1:1 ratio to receive a quality-improvement intervention (iABC, experimental group) or 'usual care' practice (control group). The randomization will occur in each Country once all clusters selected in the Country have obtained IRB approval. The allocation schedule for random assignment of care models (iABC or usual care) to sites will be computer generated at the Central Coordinating Centre. The clusters in each Country will be stratified in 2 groups: Centres with or without interventional electrophysiology laboratory for AF ablation.

Eligibility Criteria:
Inclusion Criteria:

* Outpatients of both sexes with age ≥65 years;
* First diagnosed, paroxysmal, persistent, long-standing persistent or permanent AF, confirmed as per guideline-recommended diagnostic criteria for AF, e.g. with electrocardiogram (ECG) or Holter monitoring;
* ≥1 additional long-term comorbidity, thus fulfilling the definition of multimorbidity: hypertension (treated with at least 2 antihypertensive drugs), coronary artery disease (CAD), peripheral artery disease, heart failure, stroke/TIA, diabetes mellitus, COPD, CKD.

Exclusion Criteria:

* Mechanical prosthetic heart valve or moderate/severe mitral stenosis;
* Patient unwilling to be enrolled and sign written informed consent;
* Patient unable to understand the study and attend the follow-up;
* Serious diseases with a life expectancy inferior to 12 months;
* Patients included in other interventional studies.

Only for sites randomized to the iABC group:

- Patient without a device suitable for iABC use.
Pituitary Down-regulation Before IVF for Women With Endometriosis

Endometriosis is a hormone dependent disease of women, in which endometrial tissue (the cells which line the uterus or womb) are found outside the uterus (womb). Some women with endometriosis may be infertile. However, treatments for infertility such as in vitro fertilization (IVF) or IVF with intracytoplasmic sperm injection (IVF-ICSI) appear to be less successful, i.e. the pregnancy rates are lower, for women with endometriosis than for women who may be infertile for other reasons.

Since endometriosis is hormone dependent, it has been suggested that suppressing the activity of hormones produced by the ovaries (which affect endometrial growth) may inactivate endometriosis and so increase the chances of pregnancy. Recently it has been proposed that taking the oral contraceptive pill (OCP) for 6 to 8 weeks before IVF or IVF-ICSI treatment could be used for this purpose.

For our study, which is a randomised controlled trial, women with endometriosis meeting the study criteria planning to undergo IVF or IVF-ICSI at the Oxford Fertility Unit will be recruited. Study participants will be randomised into 2 arms: experimental group and control group. The experimental group will be instructed to complete an 8 week course of OCP before beginning standard IVF treatment. The control group do not take any study medication before beginning standard IVF treatment. The IVF or IVF-ICSI treatment for both groups is not altered by participation in the study.

The aim of the study will be to determine if pretreatment with OCP improves IVF or IVF-ICSI success rates, such as live birth and pregnancy rates, in patients who suffer from endometriosis. The study is funded by the Oxford Fertility Unit.


Eligibility Criteria:
Inclusion Criteria:

Participant is willing and able to give informed consent for participation in the study.

* Female aged 18 to 39 years old.
* Intending to undergo treatment with a first, second or third cycle of IVF or IVF-ICSI.
* Diagnosed with any degree of endometriosis or endometrioma.
* Participants must meet World Health Organisation Group 1 or 2 eligibility requirements for Combined Oral Contraceptive use.

Exclusion Criteria:

The participant does not understand the English language, or has special communication needs. This is because it is impractical to provide, for this small scale study, information sheets and consent forms in other languages except English.

* The patient has already undergone 3 or more IVF or IVF-ICSI cycles.
* Patients who are already taking any medication to treat endometriosis such as progestins, OCP, Gonadotrophin Releasing Hormone agonists, danazol, mirena, etc. or who have done so within the last 3 months.
* Participants who do not meet World Health Organisation Group 1 or 2 eligibility requirements for Combined Oral Contraceptive use.
dnaJ Peptide for Relieving Rheumatoid Arthritis

A small protein called dnaJ peptide may help people with rheumatoid arthritis (RA) by preventing their immune system cells from attacking their own tissues. The purpose of this study is to determine if small amounts of dnaJ peptide can "re-educate" immune cells in people with RA so that the cells stop attacking joint tissues.

Immune modulation is a promising new approach for the treatment of RA. Studies have shown that immune cells in the joints of people in the early stages of RA react strongly against dnaJ peptides from bacteria. These immune cells may also cross-react with human dnaJ peptides in the joints to cause inflammation. dnaJ may help RA by "re-educating" the immune system and dampening the abnormal inflammatory immune response in RA.

This study will last 7 months. Participants will be randomly assigned to receive either dnaJ or placebo by mouth. At screening, participants will have medical history, physical, and medication assessment. At screening, at 6 study visits every month after the start of treatment, and at 1 month follow-up, participants will have a joint exam, blood and urine collection, and will fill out a questionnaire about their condition.

Eligibility Criteria:
Inclusion Criteria:

* Active rheumatoid arthritis as defined by the revised American College of Rheumatology (ACR) 1987 criteria. Evidence of active disease will be based on at least six swollen or nine tender joints.
* Diagnosis of rheumatoid arthritis of less than 5 years
* Reactivity to dnaJ
* Agree to use acceptable methods of contraception
* Able to understand and sign informed consent

Exclusion Criteria:

* Patients taking more 7.5 mg of prednisone or disease modifying agents other than hydrochloroquine or sulfasalazine (i.e., gold, penicillamine, azathioprine, cyclophosphamide, methotrexate, cyclosporine, or anti-TNF agents)
* Serum creatinine greater than 1.5 mg/dl
* SGOT less than SGPT
* Alkaline phosphatase greater than 2 times age/sex adjusted normal values
* Hematocrit of less than 30
* Platelets less than 130,000
* History of lymphoma
* Any active malignancy or cancer requiring treatment in the last 5 years, except for nonmelanoma skin cancers and carcinoma of the cervix in situ
* Medical or psychiatric condition or active serious infection
* Pregnant or breastfeeding
Operant Conditioning of Spinal Reflexes Training System--Reflex Operant Down Conditioning

The purpose of this study is to validate the capacity of a reflex training system to change the size of the targeted reflex. For this, the researchers are recruiting 25 individuals with chronic incomplete SCI who have spasticity in the leg to participate in the reflex training procedure. The study involves approximately 45 visits with a total study duration of about 6 months.


Eligibility Criteria:
Inclusion Criteria:

1. a clinically stable spinal cord injury (above T11) that occurred at least one year previously
2. the ability to ambulate at least 10 m with or without an assistive device (i.e., walker, crutches, or cane, not parallel bars) within 100 sec (those with 10-meter walking time \>100 sec are excluded because it is unlikely that they are able to participate in and complete our planned locomotion evaluation procedures)
3. clinical signs of spasticity in the plantarflexor muscles at least unilaterally (i.e., hyperreactivity to Achilles tendon tap, and increased muscle tone, score \>1 on Modified Ashworth scale)
4. spastic hyperreflexia reflected in exaggerated H-reflex
5. functionally and medically stable for at least 3 months
6. medical clearance to participate
7. reasonable expectation that current medications (including antispasticity medication such as baclofen, diazepam, and tizanidine) will not change over the conditioning period. Each participant's medication and dosage will be monitored and recorded throughout the study. Once enrolled, the subject will remain enrolled even if medication changes. (Because only neurologically stable subjects will enter this study, medication changes will be unlikely.)

Exclusion Criteria:

1. motoneuron injury;
2. a cardiac condition (history of myocardial infarct, pacemaker use, etc.)
3. an unstable medical condition
4. a pre-existing or confounding neurological condition (e.g., history of Multiple Sclerosis (MS), Traumatic Brain Injury (TBI), Stroke, Parkinson's disease)
5. a condition that prevents lower extremity mobility testing or weight bearing (e.g. fracture, severe sprain/strain, botox muscular injection) (orthotic knee hyperextension is not excluded; in severe cases of knee hyperextension, the brace can be worn during the study sessions)
6. a cognitive impairment that precludes giving informed consent (e.g., severe intellectual disability)
7. use of a functional electrical stimulation (FES) foot-drop stimulator or an FES bicycle on a daily basis (FES applied to the arm is acceptable)
8. deep vein thrombosis within the past 6 months
9. depression (due to potential interference of anti-depressant medication with the intervention and possible reduced participation reliability)
10. pregnancy (due to expected changes in weight and posture and potentially unstable medical condition).
Effects of Early Statin Treatment After Acute Myocardial Infarction (AMI) in Japanese Patients

Statins have been shown to prevent coronary artery disease and to preserve left ventricular function in dilated cardiomyopathy. The investigators hypothesized that the early use of statins would reduce cardiovascular events including heart failure in acute myocardial infarction patients. The purpose of this study is to determine whether early (within 96 hours after onset) use of any available statins are effective to prevent cardiovascular events including heart failure after acute myocardial infarction in Japanese patients.

At least, in Japanese, it was controversial if lipid-lowering therapy to the patients whose total cholesterol levels \<240 mg/dL was needed. Furthermore, in general, a role for early statin therapy in patients with ST-elevated AMI reperfused by primary PCI has not been clearly established. Statins have been shown to prevent coronary artery disease and to preserve left ventricular function in dilated cardiomyopathy. The investigators hypothesized that the early use of statins would reduce cardiovascular events including heart failure in acute myocardial infarction patients. A prospective, randomized, open-label, multicenter trial is conducted in AMI patients with normal total cholesterol levels (180-240 mg/dL). Patients are randomly assigned to receive any available statin within 96 hours of AMI onset or no statin and were followed for up to 24 months. The primary endpoint is a composite of cardiovascular death, nonfatal AMI, recurrent symptomatic myocardial ischemia, congestive heart failure, and stroke.

Eligibility Criteria:
Inclusion Criteria:

* Clinical diagnosis of acute myocardial infarction
* Serum total cholesterol levels on admission ranges ≥180 mg/dL and \<240 mg/dL

Exclusion Criteria:

* Age \< 18 years
* Time from symptom onset to admission \> 96 hours
* Use of lipid-lowering agents within the previous 3 months
* Known familial dyslipidemia
* Severe renal failure
* Known hepatic disease
* Signs and symptoms of severe heart failure (Killip class III or IV)
* A scheduled PCI or coronary artery bypass grafting (CABG)
* A history of previous PCI (within 6 months) or CABG (within 3 months)
* The presence of malignant disease
* The presence of allergy to statins.
Vitamin D and Vascular Health in Children

In this study, we will test the central hypothesis that enhancement of vitamin D status in obese and overweight children will improve their vascular health and their cardiovascular disease (CVD) and metabolic syndrome risk profile.

Our primary objective is to determine, in obese and overweight children aged 10 to 18 years with vitamin D deficiency (defined as serum 25-hydroxyvitamin D \<20 ng/mL), the efficacy of enhanced vitamin D3 supplementation in improving vascular endothelial function, arterial stiffness, insulin sensitivity, and metabolic syndrome risk status; and to assess whether these effects are dose-dependent. As a secondary objective, we will examine the vitamin D supplementation-induced effect on adipokines and inflammatory markers relevant to CVD risk. In a double-masked, controlled trial, we will randomize 252 eligible children to receive either 600 IU (conventional supplementation), or 1000 IU or 2000 IU (enhanced supplementation) of vitamin D3 daily for 6 months.

In terms of reporting of results, the following pre-specified outcomes are included in the primary manuscript (PubMed PMID:31950134 -- see Reference section for citation details)

1. Waist Circumference
2. Serum High Density Lipoprotein (HDL) Cholesterol
3. Serum Triglycerides
4. Inflammatory markers (Plasma TNF-alpha, high-sensitivity C-reactive protein, and IL-6)
5. Adiopkines (Plasma Leptin and Adiponectin)

Plasma nitric oxide metabolites were not measured.

Eligibility Criteria:
Inclusion Criteria:

* Eligible subjects will be 10 to 18 years of age;
* obese or overweight (BMI ≥85th %tile);
* otherwise healthy, and
* have a serum 25(OH)D concentration \<20 ng/mL

Children taking multivitamins should be able to hold off their multivitamins during the course of the study.

Exclusion Criteria:

Children will be excluded if they

* (a) have hepatic or renal disease, metabolic rickets, malabsorptive disorders, primary hyperparathyroidism, hyperthyroidism, or other chronic disorders that could affect vitamin D metabolism;
* (b) are receiving treatment with anticonvulsants, systemic glucocorticoids, pharmacologic doses of vitamin D (≥1000 IU of vitamin D2 or D3 daily), antihypertensives, vasoactive drugs, antilipidemics, metformin, antipsychotics, or other oral insulin regulators;
* (c) have cholelithiasis or urolithiasis;
* (d) have type 1 or type 2 diabetes; or
* (e) have a condition or underlying abnormality that could compromise the safety of the subject.

Post-menarchial girls with a positive pregnancy test at randomization, or subjects found during the screening phase to have hypercalcemia (serum calcium \>10.8 mg/dL) or significant fasting hyperglycemia (fasting blood glucose ≥ 125 mg/dL) will also be excluded.
ExBonAdAsia Study: A Study of Once Monthly Bonviva (Ibandronate) in Women With Post-Menopausal Osteoporosis.

This single arm study will continue drug safety surveillance, and assessment of adherence, in patients with postmenopausal osteoporosis who have received monthly oral Bonviva in ML19930, the BonAdAsia study. All patients completing 6 months in ML19930 will continue to receive Bonviva (150mg po, monthly) for an additional 6 months. The anticipated time on study treatment is 3-12 months, and the target sample size is 500+ individuals.


Eligibility Criteria:
Inclusion Criteria:

* patients responding to oral monthly Bonviva during the BonAdAsia study;
* willing to continue Bonviva treatment for a further 6 months.

Exclusion Criteria:

* none specified.
SElf-SAMpling in Cervical Cancer Screening; SESAM Study

Study aims to support development of evidence based health care in Norway through evaluating recently proposed technological improvements in cervical cancer control before their routine use. SESAM II study evaluates the accuracy of vaginal self-sampling for high risk human papillomavirus (hrHPV) testing compared with a physician-taken sample.

Concept of collecting cervical cancer screening smear in home through self-sampling is new both for target population and medical professionals. Self-sampling increases screening attendance and could be an alternative to recruit more women to cervical cancer screening in Norway. As there are is an implementation ongoing to switch from cytology based screening to HPV based screening in Norway, a reliable self-sampling method for HPV testing should be available. Furthermore, detection of HPV from self-sampled specimen requires laboratory capacity and expertise to comply with quality assurance demands such as internal quality control, external quality assessment and quality improvement. National studies are crucial to obtain knowledge and build expertize among health care providers. This study aims to show non-inferior sensitivity of hrHPV testing on self-sampled vs. clinician-sampled specimens to detect high-grade cervical lesions and cancer (CIN2+). Additionally we will;

* Evaluate overall and hrHPV type specific concordance between self-taken and physician-taken samples.
* Evaluate participants views on feasibility and acceptability of self-sampling (questionnaire)
* Compare participants screening history with the questionnaire to evaluate the reason for not participating in the national screening program (if that is the case).
* Biobank biological material collected from self-sample, physician taken samples, blood and urine, for future analysis on HPV-related diseases and cancer.

Study participants will be recruited from the colposcopy referrals and cancer care units from three different hospitals. Patients with CIN 2 or CIN 3 lesions (n=200) will be recruited from Oslo University Hospital, Ullevål and Østfold Hospital Trust, while cancer patients (n=50) will be recruited from Radiumhospital.

Eligibility Criteria:
Inclusion Criteria:

* Women with histological verified CIN 2 or CIN 3
* Women with histological verified cervical cancer

Exclusion Criteria:

* Women with mild cervical lesions