cyrilzakka/clinical-trials huggingface dataset:
The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

The primary aims of the HOME Study are to:

* Design and implement a natural history study for metachromatic leukodystrophy to serve as a source of external control data, to augment or replace concurrent controls in clinical trials;
* Pilot test and develop guidance on how to design, conduct, and analyze the data from a natural history study to support adaptive trial designs for regulatory use;
* Reduce burden of participation in trials and provide a potential solution to patient recruitment challenges, particularly for RCT's; and
* Design approaches that support remote participation in studies.

The HOME Study is a web-based natural history study for patients with metachromatic leukodystrophy. It is hosted by the National Organization for Rare Disorders (NORD); an independent non-profit patient advocacy organization dedicated to individuals with rare diseases and the organizations who serve them.

The study collects information from participants (or their authorized respondents, heretofore referred to collectively as "participants") who are affected by metachromatic leukodystrophy.

Data are collected at pre-baseline, baseline, 3, 6, 9, and 12 months through online surveys, telephone Interviews, web-based virtual assessments with a clinical study coordinator, and a (optional - only for U.S. residents) mobile application. Data entered into this study includes name, date of birth, diagnosis, treatments, medical history, family history, quality of life, disease progression, treatment - past and proposed, general medical information, genetic test results and mutations, blood level results, upload of medical records.

Eligibility Criteria:
Inclusion Criteria:

The study is open to English speaking individuals of all ages who have a diagnosis consistent with metachromatic leukodystrophy (MLD). MLD is defined as:

* Mutations in the ASA and PSAP genes identified by genetic testing;
* A diagnosis of MLD by MRI of the brain; or
* Sulfatase enzyme activity and urinary sulfatide excretion identified by biochemical testing.

Exclusion Criteria:

Patients will be excluded from the study if they do not meet inclusion criteria.

* Non-English speaking individuals
* No confirmed diagnosis of metachromatic leukodystrophy.
Extracorporeal Shock Wave Therapy on Wrist and Hand Functions in Spastic Chronic Stroke Patients

40 chronic stroke patients with upper limb spasticity will randomly divided into two groups. First group will receive 3 sessions of radial extracorporeal shock wave therapy (rESWT) with one week apart without cessation of current physical therapy, while the second group will continue to receive conventional rehabilitative program.

Assessment will be done at baseline, 2 weeks after rESWT and 3 months after rESWT using Modified Ashworth Scale, Fugl Meyer Assessment for hand function and wrist control, motricity index for pinch grip and Hmax/ Mmax amplitude ratio of flexor carpi radialis muscle.


Eligibility Criteria:
Inclusion Criteria:

* Patients with chronic stroke with disease duration more than one year will be included in the study with stable modified Ashworth scale for upper limb spasticity ranged from 1+ to 4.

Exclusion Criteria:

* Patients more than 65 years,Patients with double stroke and patients with fixed contractures of wrist \& hand ,Patients received anti-spastic measures (botulinum toxins, nerve block) within 6 months,Also patients with contraindication to extra-corporeal shock wave therapy i.e. malignancy at treatment area, coagulopathies, active infection (viral or TB), o bleeding wounds, and patients with pacemakers will be excluded
The (Cost-)Effectiveness of Nurse Practitioners Working at the Primary Out of Hours Emergency Service

The aim of this study is to explore whether the implementation of Nurse Practitioners can lead to a more accessible and efficient patient care at the primary out of hours service.

The primary objectives of the proposed study are:

1. What are the effects of the implementation of NPs on the primary out of hours service in comparison with the current out of hours service? Effects in terms of accessibility, objective and subjective workload of general practitioners, quality of care and patient satisfaction.
2. How efficient is the implementation of NPs in the primary out of hours services?
3. What is the feasibility of the implementation of NPs in the out of hours services? And under which conditions?
4. What are the barriers and facilitating factors considering the implementation of NPs?

The emergency care and primary out of hours care in the Netherlands is under pressure. There is a rising demand from patients for acute care at the primary out of hours service (run by General Practitioners) as well as for the emergency departments (EDs) at the hospitals. The workload for healthcare professionals in these acute care setting is high. Without changes in the organization of primary out of hours care and emergency care, the quality, accessibility and efficiency of the acute care can't be guaranteed in the future.

The substitution of care from General Practitioners (GPs) to Nurse Practitioners (NPs) is seen as one possible solution to decrease the GPs' workload and improve accessibility and efficiency of care without reducing the quality of care.

It turned out that about 80% of the acute complaints is U3 and U4 (low complex and not urgent) and does not necessarily to be seen by a physician.

Based on previous research we expect that the NPs are competent to diagnose and treat almost all low complex and not urgent complaints. During surgery hours (day time) the NPs act in about 90% of the consultations independently.

Hypothetical substitution of care should contribute to enhancing quality, improving accessibility and reducing the workload of doctors. It can also benefit the efficiency of the acute (primary out of hours) care.

However, specific scientific evidence for this is lacking.

In this study we examine whether substitution of care from GPs to NPs in a primary out of hours care setting can contribute to a more accessible and efficient patient care. Also the feasibility of implementing NPs in a primary our of hours setting is examined.

Comparison: Care provided by the Nurse Practitioner will be compared to care provided by a General Practitioner.

Eligibility Criteria:
Inclusion Criteria:

* Patients (with urgency U2, U3, or U4) requesting an appointment at the primary out of hours emergency service during the weekend between 10.00 and 17.00 hours.

Exclusion Criteria (patients seen by a NP):

* Patients under the age of 1 year
* Patients with psychiatric complaints
* Patients with abdominal pain, abdominal infections, chest pain or neck complaints (angina pectoris), headache and dizziness.
Multicenter Comparative Study of the Activity of the Medial Prefrontal Cortex in Vulnerability to Depression

To examine whether the medial prefrontal cortex displays an increased activity during self-referential processing among remitted depressed patients compared to healthy controls


Eligibility Criteria:
Inclusion criteria (patients)

* History of at least one major depressive episode
* In clinical remission for at least 6 months (BDI-21 ≤ 10 \& Montgomery and Asberg Depression Rating Scale ≤ 7)
* Aged 20-65

Exclusion criteria :

* first depressive episode after the age of 59
* last episode treated by cognitive behavior therapy
* currently receiving a antipsychotic or mood-stabilizing drug
* personal history of: major depressive episode with psychotic features, other axis-I disorder (except generalized anxiety disorder), substance-related disorder (except nicotine dependence), bipolar disorder
* first-degree familial history of bipolar disorder
* cognitive impairment due to another central nervous system disease
* contra-indication to MRI
* pregnancy
* refusal to be informed of abnormal findings during the MRI
Oral Verapamil in Acute Paroxysmal Supra Ventricular Tachycardia(PSVT) Recurrence Control

PSVT is a common rhythm disorder in emergency department which can be recurrent in some cases.

Intravenous Adenosine is the drug of choice in PSVT control in acute settings but is a very short acting agent.

Providing a complementary medication to decrease the rate of recurrence of PSVT after its initial control will be promising.

This study evaluates the role of the oral Verapamil in recurrence control of PSVT in emergency situations.


Eligibility Criteria:
Inclusion Criteria:

* Age\>18 years old
* PSVT on emergency department presentation

Exclusion Criteria:

* Instability in hemodynamic status
* Allergy to Verapamil
FOCUS on US: Adapting the FOCUS Program for Sexual and Gender Minority (SGM) Cancer Patients and Caregivers

Our aim for this study is to assess the feasibility and acceptability and explore potential outcomes of the adapted FOCUS On Us intervention in a two-arm pilot trial. The investigators will randomize 80 sexual and/or gender minority (SGM; e.g., lesbian, gay, bisexual, transgender, queer, etc.) cancer patients and their ≥80 informal caregivers (total N≥160) to either FOCUS On Us (adapted from the evidence-based FOCUS program) or a waitlist control.


Eligibility Criteria:
Inclusion Criteria:

Patients must:

1. have been diagnosed or had a biochemical recurrence or progression of any invasive (e.g., Stage ≥1) cancer within the past 24 months;
2. have at least one informal (unpaid) caregiver willing to participate in the program;
3. identify as a sexual and/or gender minority (SGM) OR have a caregiver who identifies as SGM (e.g., either the patient or caregiver(s) must be SGM);
4. be age 18 or older;
5. be able to complete all written and oral components of the study in English;
6. provide informed consent;
7. live in the United States.

Caregivers must:

1. self-report having provided support for a cancer patient as an informal (unpaid) caregiver;
2. have a cancer patient for whom they provided support willing to participate in the program;
3. identify as a sexual and/or gender minority (SGM) OR have provided support to a patient who identifies as SGM (e.g., either the patient or caregiver(s) must be SGM);
4. be age 18 or older;
5. be able to complete all written and oral components of the study in English;
6. provide informed consent;
7. live in the United States
An Investigation Into The Impact Of Enteric Coated Of Aspirin In Patients With Newly Diagnosed Ischemic Stroke.

Uncertainty remains regarding the impact of enteric-coated (EC) aspirin as it relates to the reduction of CV risk. We hypothesize that EC formulation based on the previous report may blunt aspirin response as evidenced by reduced Thromboxane A2 (TXA 2) levels in diabetic patients.


Eligibility Criteria:
Inclusion Criteria:

1. Newly diagnosed ischemic stroke who are just about to start aspirin.
2. No prior history of cardiovascular morbidity (including ischemic heart disease, chronic kidney disease, peripheral vascular disease) 3-18- 75 years.

4-Patients with ischemic stroke who underwent reperfusion intervention (catheter-directed thrombolysis and/or thrombectomy) regardless of the time of presentation (i.e within or outside the therapeutic window as the managing stroke deem fit.

Exclusion Criteria:

1. Concomitant antiplatelet therapy (irrespective of the duration of the treatment).
2. Patients on any prostaglandins related medications (non-steroidal anti-inflammatory drugs, misoprostol, and other ant secretory drugs among others).
3. Any salicylate-containing supplements.
4. Patients on the NG tube will be excluded from the study
Treatment of Postoperative Delirium With Continuous Theta Burst Stimulation

To determine whether continuous theta burst stimulation can cure postoperative delirium in senior patients.

Postoperative delirium(POD), a syndrome characterized by an acute change in attention, awareness and cognition, is one of the most common postoperative complications among elderly patients. Impaired neuronal network connectivity is likely one of the several neurobiological processes that contribute to POD pathogenesis. Recently, continuous theta burst stimulation (cTBS) was demonstrated to improve cognitive function in patients with mild cognitive impairment.Recent research suggests that cTBS has positive effect on improving the connectivity and reorganization of the brain network. Therefore, we plan to conduct an exploratory study in participants undergoing elective surgeries to determine whether cTBS can cure POD in this senior patient population.

Eligibility Criteria:
Inclusion Criteria:

* Aged between 60 to 80 years;
* Having delirium after elective surgery;
* Being able to complete cognitive assessment and confusion assessment methods (CAM);
* Willing to participate before surgery and being competent to provide informed consent.

Exclusion Criteria:

* Having brain tumor, stroke, or mental disorders (e.g., depression or dementia);
* Having contraindications of cTBS (e.g., head trauma, history of epilepsy or metal implants in the head or heart);
* Participating in other clinical studies.
Fibre Specific Signalling in the Locomotor Myopathy of Chronic Obstructive Pulmonary Disease

Skeletal muscle is composed of two fibre types which are intertwined. Skeletal muscle weakness, particularly of the walking muscles, is an important complication of Chronic Obstructive Pulmonary Disease (COPD) but so far the investigators do not know what mechanisms drive the process.

All existing studies have investigated signalling pathways in the whole muscle so they have been forced to consider type I and type II fibres together. It is possible that disease selectively affects one fibre type, most likely type I fibres which are in fact lost in COPD patients. For this reason mechanisms of disease may have been overlooked by current studies.

The applicants have acquired the technology which allows type I and type II fibres in a muscle specimen to be split (by laser capture microdissection) and so signalling pathways can be assessed separately in type II and type I fibres which is what this proposal sets out to do.

The proposal therefore aims to capture well characterised clinical data from 60 COPD patients and 20 age matched controls, from whom a biopsy of the main walking muscle, the quadriceps, will be taken. In the samples the investigators will assess at a fibre specific level inflammatory signalling. Surplus material will be retained for subsequent fibre specific analysis.


Eligibility Criteria:
Inclusion Criteria:

* Male or female aged 40 - 90 years inclusive.
* A clinical diagnosis of COPD based on spirometry (FEV1/FVC ratio \< 70% on entry to the study with FEV1 \< 80% predicted)

Exclusion Criteria:

* History or current diagnosis of alpha 1-anti-trypsin deficiency, asthma, lung cancer, bronchiectasis or other significant respiratory disease (including treated TB in the past 10 years)
* Contraindication to biopsy for research purpose (e.g. use of anticoagulant in the 1 week prior to biopsy)
* Joint or neurological disease which on its own would preclude exercise
Laser In-situ Keratomileusis With Crosslinking Compared to Conventional LASIK in Patients With High Myopia

The objectives of this study are to evaluate the safety and efficacy of a treatment regimen for high myopia and myopic astigmatism: LASIK followed by crosslinking performed with the KXL Crosslinking-System and VibeX Xtra (Riboflavin Ophthalmic Solution, Avedro, USA), as compared to LASIK alone, with regards to regression of refractive outcome, as measured by manifest refraction spherical equivalent (MRSE) and keratometry.

This is a controlled study of the safety and efficacy of the KXL System and VibeX Xtra (Riboflavin Ophthalmic Solution, Avedro, USA) for performing crosslinking in eyes undergoing LASIK for high myopia or myopic astigmatism. Each subject will have bilateral LASIK performed and one eye will be randomized to undergo LASIK followed by crosslinking.

Subjects will undergo bilateral LASIK. Following randomisation one eye of each subject will be treated with VibeX Xtra (Riboflavin Ophthalmic Solution) following LASIK and will be irradiated with the KXL System at 30 mW/cm2 intensity for 90 seconds continuous UV-A light treatment for a total radiant exposure of 2.7 J /cm2.

All eyes will be assessed at 1 day, 1, 3, 6, and 12 months after treatment.

Eligibility Criteria:
Inclusion Criteria:

* eligible for bilateral myopic fs-LASIK with -6.00 to -12.00 D (diopters) with a maximum of 5.00 D astigmatism
* age \> 18 years
* provided written informed consent
* difference between the MRSE and cycloplegic SE less than 0.75 D
* MRSE needed to be stable for the last 12 months (\<0.5 D).

Exclusion Criteria:

* prior corneal surgery,
* forme fruste or manifest keratoconus,
* history of corneal scarring, melting, ulceration
* repeating inflammations of the eye
* taking vitamin C 1 week prior to the treatment
Immune Profiling During Ocrelizumab Treatment in Multiple Sclerosis

The purpose of this study is to assess effects of B cell depletion on the immune system in patients with relapsing forms of multiple sclerosis (MS) treated with ocrelizumab. This will be done by collecting blood from patients starting treatment with ocrelizumab before the first infusion and before the infusions at 6 and 12 months. The effects on the immune system will be assessed by performing transcriptome profiling of immune cells, measuring serum levels of microRNAs, and analyzing serum autoantibody immune signatures using antigen arrays. In addition, blood will be collected once from a group of healthy individuals to serve as controls.


Eligibility Criteria:
Inclusion Criteria:

* Patients with relapsing form of MS starting treatment with ocrelizumab at Partners MS Center
* Healthy controls
* Ability to understand and sign informed consent

Exclusion Criteria - All subjects:

* Other concomitant autoimmune or inflammatory diseases
* Ongoing treatment with other immunomodulatory medications
* Previous treatment with rituximab, methotrexate, cyclophosphamide, mitoxantrone, mycophenolate mofetil, or alemtuzumab
* Pregnancy or lactation
* Hypersensitivity to ocrelizumab

Exclusion Criteria - Healthy Controls:

* MS
* First-degree relatives with MS
Effect of Dexmedetomidine and Remimazolam on Body Temperature in Patients Undergoing Spinal Anesthesia

This study aims to evaluate the effect of dexmedetomidine and remimazolam on body temperature in patients undergoing spinal anesthesia.


Eligibility Criteria:
Inclusion Criteria:

* Patients scheduled for elective surgery under spinal anesthesia

Exclusion Criteria:

* Severe vascular disease
* Thyroid dysfunction
Effect of Pulmonary Rehabilitation in Lung Cancer Survivors

Patients who have surgery to cure lung cancer often have multiple problems at hospital discharge and later on. This includes poor exercise performance and quality of life, breathlessness, pain and tiredness. Currently there is little formal physical or psychological support for such patients. This pilot study aims to investigate whether outpatient pulmonary rehabilitation (an exercise training and education programme), started within 2 weeks of hospital discharge, can lead to improvements in exercise performance and quality of life in patients who have undergone lung cancer surgery. The study will also assess whether pulmonary rehabilitation is acceptable for patients and will analyze the safety profile. The hypothesis is that patients; discharged from hospital after undergoing lung cancer surgery, have improved exercise performance and quality of life following early outpatient pulmonary rehabilitation compared with usual care.

The research question is to investigate

* whether patients who have undergone curative surgical resection for lung cancer benefit from 8 weeks of early outpatient pulmonary rehabilitation (started within 2 weeks of hospital discharge).
* to identify potential barriers to this approach

Pulmonary rehabilitation (PR) is the most effective non-pharmacological treatment for patients with chronic respiratory diseases, especially COPD. Many patients with lung cancer have co-existing smoking-related lung disorders such as COPD. To date there have been no randomised controlled trials of PR in post-resection lung cancer survivors. However there is indirect evidence that PR may be of benefit in this patient cohort. Cesario et al (2007)reported a 32% improvement in exercise capacity with in-patient PR, and Spruit et al (2009) demonstrated a 43% improvement in six minute walk distance following an 8-week in-patient PR programme. However, both studies had small numbers, and no randomised control group. Furthermore, inpatient PR is not an economically justifiable intervention in the NHS where emphasis is on self-management and ambulatory care.

Eligibility Criteria:
Inclusion Criteria:

* All patients undergoing lung cancer surgery with curative intent.

Exclusion Criteria:

* Unstable cardiovascular disease Severe musculo-skeletal problems that would limit exercise training(neuromuscular disorders, severe joint arthritis of lower limbs) Severe peripheral vascular disease Unable to walk 10 metres unaided Patients unable to give informed consent Recent (within 3 months of completing treatment) or untreated pulmonary TB, Untreated/uncontrolled diabetes or epilepsy Recent or recurrent untreated spontaneous pneumothorax