cyrilzakka/clinical-trials huggingface dataset:
Lithium in Inclusion Body Myositis (IBM)

IBM is the most common acquired muscle disease occurring over the age of 50. The underlying cause remains unknown and there is currently no effective treatment. Pathological studies have revealed abnormal collections of proteins in the muscle cells from patients with IBM. These include proteins called phosphorylated tau (p-tau). A similar process appears to occur in Alzheimer disease, with accumulations of p-tau developing in brain cells. Lithium decreases the activity of the GSK, an enzyme that has a key role in the development of p-tau. Lithium and other GSK inhibitors have been shown to decrease the accumulation of p-tau in nerve cells in animal models of Alzheimer disease. The proposed research is a pilot study to see if lithium might be an effective treatment for IBM

Trial Status Open for Enrollment

What is the Purpose of this Study? There is currently no effective treatment for IBM and its pathogenesis remains uncertain. This study is designed to test the hypothesis that treatment of patients with IBM with Lithium can improve muscle strength and reduce the markers of disease activity believed to be important in the disease pathogenesis.

Who is Eligible to Participate? Patients diagnosed with Inclusion Body Myositis above the age of 30 who have adequate muscle function for quantitative muscle testing and who have a FVC \>50%. Patients who have uncontrolled diabetes, renal insufficiency, congestive heart failure, cancer, hypothyroidism, current use of immunosuppressive medication, currently on warfarin or any known bleeding disorder are excluded.

What is involved in this Study? This is an open label trial looking at the effects of Li on muscle strength. Procedures consist of a baseline muscle biopsy, blood work, and other screening procedures. After the muscle biopsy patients are started on Li at 300mg/d. Lithium doses are dependent on tolerability and target lithium levels. Monthly visits consist of EKGs, Li level labs, questionnaires, and muscle strength testing. At month 6, patients will receive another muscle biopsy. The muscle biopsies are performed to analyze p-tau levels in the muscle.

How long is the Study? It is approximately 6 months long; however patients have the option to stay on the study for an additional 6 months.

Eligibility Criteria:
Inclusion Criteria:

* Age \>30
* Meet diagnostic criteria for definite IBM
* Muscle function adequate for quantitative muscle testing
* Able to give informed consent
* Women of child bearing potential must have a negative pregnancy test

Exclusion Criteria:

* Presence of uncontrolled diabetes, hypothyroidism, chronic infection, chronic renal insufficiency, congestive heart failure, cancer, or other chronic serious medical conditions
* Significant arrhythmias or conduction defect abnormalities on ECG
* Pregnant or lactating
* Coexistence of other neuromuscular or neurological diseases that would interfere with assessment
* Known bleeding disorder
* On Warfarin
* Contraindications to muscle biopsy: allergy to local anesthetic, skin infection, known bleeding disorder
Paracetamol Toxicity in Septic Patients

The investigators will examine the toxicity of therapeutic doses of paracetamol in patients in severe sepsis. Patients with fever and severe sepsis will be randomized to receive paracetamol or dypirone. The investigators will monitor blood glutathione and liver enzymes to look for potential toxicity.

Paracetamol is metabolized in liver using the glutathione system. This detoxification system is depressed during severe illness such as sepsis, trauma etc. The study will examine the toxicity of therapeutic doses of paracetamol in patients in severe sepsis. We believe that during sepsis, paracetamol metabolites are not fully detoxified and therefore are toxic to the patient.

Patients with fever and severe sepsis will be randomized to receive paracetamol or dypirone. The investigators will monitor blood glutathione (as a surrogate marker for liver glutathione), liver enzymes and various clinical data (such as length of hospitalization) to look for a potential toxicity.

Eligibility Criteria:
Inclusion Criteria:

* age\>18y
* hospitalized to int. med. dept. or ICU
* body temp\>38 and one or more from the following:

  1. pulse\>90 bpm
  2. resp. rate\>20/min or PaCO2\<32 mmHg
  3. WBC\>12000 or \<4000
* one of the following:

  1. hypotension
  2. disturbed end-organ perfusion (confusion, oliguria, lactic acidosis etc.)

Exclusion Criteria:

* acute / chronic liver disease
* acute CNS disorder
* current isoniazid treatment
* alcoholism
* active malignancy
* pregnancy
* TPN or PPN
Aerobic Exercise for the Improvement of Cognition and Enhancement of Recovery in Post-acute Schizophrenia

The study investigates the efficacy of aerobic exercise on cognitive performance and brain plasticity in schizophrenia.

This multi-center, two-arm, parallel-group, randomized placebo-controlled add-on clinical trial investigates the efficacy of aerobic exercise on cognitive performance and brain plasticity in schizophrenia. The aim is the enhancement of recovery with the use of 26 weeks of continuos endurance training (aerobic exercise) with stationary bicycles or a balance and tone program consisting of exercises for flexibility, core strength, balance and relaxation. Followed by a follow-up period of 26 weeks.

Eligibility Criteria:
Inclusion Criteria:

* Informed consent given by the subject; DSM-IV-TR diagnosis of schizophrenic psychosis (295.10-30, 295.90); age 18 to 65 years, male or female; PANSS at baseline: total score ≤ 75, proper contraception in female patients of childbearing potential; treatment with one or two antipsychotics in a stable dose of at least two weeks, body mass index between 18 and 40.

Exclusion Criteria:

* Lack of accountability; positive urine drug-screening for illicit drugs at screening (except benzodiazepines); serious suicidal risk at screening visit; other relevant interferences of axis 1 according to diagnostic evaluation (MINI); other relevant neurological or other medical disorders; pregnancy or lactation.
Study to Assess Safety, Tolerability and Phamacokinetics of KAE609 Administered Intravenously in Healthy Subjects

This was a randomized, subject and investigator-blinded, placebo-controlled, single and multiple ascending intravenous (iv) dose study in healthy subjects to assess the safety and tolerability of KAE609 given in the vein.

The study consisted of 2 parts: single-ascending dose (SAD) part and multiple ascending dose (MAD) part.

In Part A (Single-ascending dose (SAD) part), it was planned to recruit 6 active, 2 placebo subjects in each cohort:

* Cohort A1: 10.5 mg/placebo
* Cohort A2: 30 mg/placebo
* Cohort A3: 75 mg/placebo
* Cohort A4: 120 mg/placebo
* Cohort A5: 210 mg/placebo

In Part B (Multiple-ascending dose (MAD) part), Subjects were assigned to one of the following treatment arms in a ratio of 2:1 (6 active, 3 placebo):

* Cohort B1: 60 mg/placebo, every 24 hours (q24h) × 5 days
* Cohort B2: 120 mg/placebo, every 24 hours (q24h) × 5 days

Eligible subjects were randomized to receive a single or q24h x 5 doses of either KAE609 or placebo. Safety, tolerability and pharmacokinetics were assessed over the period of 8 days for single dose and 12 days for multiple dose up to end of study visit for each subject.

Eligibility Criteria:
Key Inclusion Criteria:

* Healthy male and female subjects 18 to 55 years of age inclusive, and in good health as determined by past medical history, physical examination, vital signs, electrocardiogram, and laboratory tests.
* Subjects must weigh at least 50 kg to participate in the study, and must have a body mass index (BMI) within the range of 18.0 - 30.0 kg/m2.

Key Exclusion Criteria:

* Use of other investigational drugs within 5 half-lives of Screening, or within 30 days of dosing, whichever is longer; or longer if required by local regulations.
* Significant illness which has not resolved within two (2) weeks prior to initial dosing.
* Pregnant or nursing (lactating) women.
* Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant.
* Sexually active males unwilling to use a condom during intercourse while taking investigational drug and for at least 2 weeks after last dose of investigational drug.
Safety and Potential Effect of Innovative Cell-based Therapy Using Adipose-derived Stromal Vascular Fraction in Patients With Autoimmune Xerostomia

The main objective of this study is to evaluate the tolerance and safety of autologous adipose-derived stromal vascular fraction injected in accessory salivary glands for treatment of autoimmune xerostomia in terms of adverse reactions through day 14 (D14).

Autoimmune xerostomia is a disabling condition affecting mostly patients suffering from Sjögren's disease, systemic lupus erythematosus, rheumatoid arthritis and systemic xerostomia.

Local therapies and systemic drug treatments (picarpine) remain the gold standards but have limited effects upon salivary flow action and many adverse effects.

Stem cell therapies and notably adipose tissue-derived stromal cells have shown promising potential for tissue repair. Autologous uncultured adipose-derived stromal vascular fraction (AD-SVF) is recognized as an easily accessible (by a standard lipoaspiration to obtain adipose tissue, from which AD-SVF are isolated by centrifugation), safe and well tolerated source of cells with angiogenic, anti-inflammatory, immunomodulatory and regenerative properties.

The purpose of our AD-SVF phase I trial is to evaluate, first the tolerance of autologous AD-SVF cells locally injected in the oral cavity and second their capability to improve the salivary function.

Eligibility Criteria:
Inclusion Criteria:

* Patients from 18 to 65 years.
* Patients suffering from xerostomia and autoimmune disease including : Gougerot-Sjögren disease according to AC/EULAR criteria and Secondary Gougerot-Sjögren syndrome related to systemic diseases (sclerodermia, rheumatoid arthritis, systemic lupus erythematosus...)
* Xerostomia visual scale (6-item visual analogue scale questionnaire) for assessment of salivary dysfunction : score ≥ 30/60
* Informed consent to participate (with signature)
* Negative β -HCG test and effective contraception for women being able to get pregnant
* Affiliation to the social security system

Exclusion Criteria:

* Medical history of head and neck neoplasia
* Recent (\<3 months) medication inducing and aggravating xerostomia : Standard treatment with tricyclic antidepressant and/or antipsychotics
* Body Mass Index \< 18
* Active smoking (\> 5 cigarettes a day)
* Active infectious disease and/or active viral serologies (HIV, HCV, HBV, HTLV I/II, TPHA/VDRL)
* Coagulation disorders including anticoagulant and antiplatelet treatment
* Any temporary or definitive contraindication due to any medical or surgical unstable condition
* Allergy to local anesthesia and/or albumin
* Pregnant or breastfeeding women
* Adult protected by the law (tutorship and curatorship)
* Patients already enrolled in another study
* Patients under 18 years of age
* Person deprived of liberty
* Patient non-affiliated to the social security
Role of microRNAs in T Cell-Driven Inflammation in Asthma

This will be a single center study of asthmatic subjects and healthy controls which will investigate mechanisms of asthma through detailed molecular analysis of airway tissues and fluids. The primary goal will be investigate the role of microRNAs in Th2-driven inflammation in asthma. The investigators hypothesize that asthma is associated with abnormal expression of miRNAs in T cells which favors differentiation into Th2-cells. The investigators further hypothesize that asthma is heterogeneous based on the presence and absence of Th2-driven inflammation and that abnormalities in T cell miRNA expression will be most prominent in a subgroup with high levels of Th2-driven inflammation (as assessed using molecular markers that the investigators have previously established). Finally, the investigators hypothesize that inhaled corticosteroids will normalize the T-cell miRNA abnormalities observed in asthma, as corticosteroids treat Th2-driven inflammation. The samples collected will also facilitate the pursuit of secondary analyses designed to investigate mechanisms of inflammation and remodeling in asthma as well as molecular phenotypes of asthma.


Eligibility Criteria:
Group A:

Inclusion Criteria:

* Male and female subjects between the ages of 18 and 70 years

Group B:

Inclusion Criteria:

* Male and female subjects between the ages of 18 and 70 years
* History of asthma
* No use of oral or inhaled corticosteroids for the treatment of asthma in the past 6 weeks
* Hyperreactivity to methacholine (PC20FEV1 Methacholine ≤ 8.0 mg/mL)
* At least one of the following symptoms, beta agonist use, or FEV1 criteria:

  * Asthma symptoms on at least two days per week; OR
  * Beta agonist use on at least two days per week; OR
  * FEV1 \< 85% predicted

Groups A \& B:

Exclusion Criteria:

* Current smokers (smoking within the last 12 months) or former smokers who have a total pack-year smoking history greater than 10
* Pregnant women
* Subjects with a history of lung disease other than asthma
* Subjects with a history of a medical disease, which in the opinion of the investigator may put the subject at extra risk from study-related procedures or because the disease may influence the results of the study
Evaluating Chronic Obstructive Pulmonary Disease (COPD) Patients' Treatment Compliance and Quality of Life Via Telemedicine

The Covid-19 pandemic has disrupted the follow-up and treatment processes for patients, and telemedicine has emerged as an important alternative method. Our aim was to test compare COPD Assessment Test (CAT) scores, inhaler treatment techniques and adherence between telemedicine-mediated healthcare and healthcare provided face-to-face.


Eligibility Criteria:
Inclusion Criteria:

* Patients who were diagnosed with COPD according to Global Initiative for Obstructive Lung Disease (GOLD)
* GOLD spirometric grade 3-4
* At least one hospital admission in the past year
* Acceptable score on the Mini-Mental State Examination
* Residing in Ankara
* Patient or caregiver must understand the process and be able to provide clear information
* Medical records and laboratory test results must be accessible to healthcare professionals
* Patient must have access to prescribed medications
* Comorbidities outside of the lungs are included

Exclusion Criteria:

* Patients diagnosed with asthma
* Patients with lung cancer
* Patients with combined pulmonary fibrosis and emphysema
* Patients with active infections such as pneumonia or COVID-19
* Patients being followed for COPD without spirometry
* Patients with low cognitive function as determined by the Mini-Mental State Examination
* Patients who do not consent to participate in the study
* Patients lacking adequate social support
* Patients with COPD grade 1-2
* Patients with no hospital admissions in the past year
* Patients diagnosed with psychotic disorders
* Patients residing outside of Ankara
* Patients enrolled in inpatient or supervised outpatient rehabilitation programs
* Patients unable to use telemedicine devices or without a caregiver to assist
* Patients and caregivers having difficulty providing information
* Patients without internet access or video call capabilities at home
Efficacy of CC-5013 (Revlimid or Lenalidomide) in Patients With Primary Systemic Amyloidosis

Patients with primary systemic amyloidosis will be treated with CC-5013 (lenalidomide; Revlimid) as a single agent for 3 months. If their disease worsens or does not improve during that time frame dexamethasone will be added to the treatment program.


Eligibility Criteria:
1. Histochemical diagnosis of amyloidosis as based on detection by polarizing microscopy of green birefringent material in Congo red-stained tissue specimens and immunohistochemical proof of AL
2. Measurable disease of AL amyloidosis as defined by one of the following:

   * Serum monoclonal protein \>=1.0 g by protein electrophoresis
   * \>200 mg of monoclonal protein in the urine on 24 hour electrophoresis
   * Serum immunoglobulin free light chain \& \>=10 mg/dL AND abnormal serum immunoglobulin kappa to lambda free light chain ratio
3. ECOG performance status (PS) 0, 1, 2, or 3
4. \>=18 years of age
5. The following laboratory values obtained \<=14 days prior to registration:

   * Creatinine \< = 3 mg/dL
   * Absolute neutrophil count \>=1000/microliter
   * Platelet \>=75000/microliter
   * Hemoglobin \> = 8.0 g/dL
6. Symptomatic organ involvement with amyloid to justify therapy. This could include liver involvement, cardiac involvement, renal involvement, peripheral neuropathy grade 1, or soft tissue involvement. Must have more than purpura or carpal tunnel syndrome
7. Previously treated or untreated. No limit to prior therapy provided there is adequate residual organ function
8. Ability to provide informed consent
9. Anticipated life expectancy of at least 3 months
10. None of the following:

    * Pregnant women or women of reproductive ability who are unwilling to use effective contraception
    * Nursing women
    * Men who are unwilling to use a condom (even if they have undergone a prior vasectomy) while having intercourse with any woman, while taking the drug and for 4 weeks after stopping treatment
    * Myelosuppressive chemotherapy \< 4 weeks prior to registration
    * Concomitant high dose corticosteroids
    * Grade 2 (or higher) peripheral neuropathy
    * Uncontrolled infection
    * Clinically overt multiple myeloma
    * Active malignancy
    * Prior hypersensitivity reaction to Thalidomide
    * Syncope within the past 30 days
Diagnostic Classifier for Cutaneous T-cell Lymphomas

Primary cutaneous T-cell lymphomas (CTCL) are a form of skin cancer that is derived from immune cells. The most common form of CTCL is mycosis fungoides (MF). While initially confined to the skin, MF may spread to lymph nodes, blood or inner organs, resulting in an overall poor prognosis for the patient. Thus, being a potentially lethal disease, an early and correct diagnosis of MF has very important implications for the patient. However, diagnosis of early MF is often difficult, as it usually shows a close resemblance to benign inflammatory conditions such as eczema and psoriasis. Strikingly, it takes an average of 3-6 (!) years from the appearance of the first skin lesions until a diagnosis of MF can be made. For this reason, a test to distinguishing early MF from benign inflammatory conditions is urgently mandated. By using skin suction blister fluid as well as skin biopsies from patients with MF, eczema and psoriasis, the investigators want to develop a classifier system that can distinguish early MF from benign inflammatory skin diseases.


Eligibility Criteria:
Inclusion Criteria:

* Clinical and/or histopathological diagnosis of MF, eczema or psoriasis
* Healthy control subjects without personal history of MF, eczema or psoriasis

Exclusion Criteria:

* Ongoing skin-targeted treatment (Wash out times: 2 weeks for topical, and 4 weeks for systemic treatments)
* Ongoing other treatment that might, in the opinion of the investigator, influence proteomic features of the samples to be acquired
The Impact of Melanoma and Drug Treatment in the Real World

An observational, non-interventional registry study to collect real-world data from people living with melanoma and its treatment, which will be available to researchers to further the knowledge of melanoma and improve patient care.

The registry uses Vitaccess' digital real-world evidence platform, and has been developed in collaboration with the patient advocacy group Melanoma UK.

Eligible participants install a study app on their smartphone or tablet. Researchers access aggregated, anonymised data via a cloud-based research portal. The platform provides benefits to participants, which it is hoped will encourage persistence with data submission; these include options to upload electronic documents such as scans, and access an online melanoma community.

The aggregated data are available in close to real time via "dashboards" and can be analysed according to a number of pre-set criteria (e.g. disease stage, age, geographic location).

State-of-the-art technologies and security policies are used in the platform to ensure industry-standard data storage and privacy for all users. Participants' personally identifiable information will remain confidential at all times, and researchers will not be able to identify individuals.

Eligibility Criteria:
Inclusion Criteria:

* adults with melanoma of any stage, resident in the UK with National Health Service (NHS) or Community Health Index (CHI) number, aged 18 and over, current or previous diagnosis of melanoma, willing to use their own smartphone or tablet.

Exclusion Criteria:

* no specific exclusion criteria
Impact of Changing the Dosing Regimen on the PK Profile of ODM-203

This Phase I study in healthy male volunteers will evaluate the impact of the effect of food on the pharmacokinetic profile of ODM-203.

This study will be a single dose part randomised cross over study with up to 6 study periods. ODM-203 may be dosed using a variety of different conditions with respect to food depending on emerging data. The impact of changing the presentation of ODM-203 may also be assessed to determine the PK of alternative formulations and identify appropriate drug formulations for further development.

Eligibility Criteria:
Inclusion Criteria:

* Written informed consent
* Able to speak, write and understand English
* Body mass index of 18.0-32.0
* Weight 55-95 kg
* Adequate method of contraception
* Good state of health

Exclusion Criteria:

* Receipt of IMP in a clinical research study or donation/loss of \>400ml blood within previous 3 months or previously enrolled in this study
* History of drug or alcohol abuse or positive drugs of abuse test or regular alcohol consumption or current smoker/user of nicotine replacement products
* Positive drugs of abuse test
* Positive hepatitis B, hepatitis C or HIV results
* Donation/loss of \>400ml blood within previous 3 months
* Poor compliance or inability to follow protocol requirements/instructions/restrictions.
* Vulnerable subjects
* Evidence of clinically relevant disease of any body system including relevant psychiatric disorders or conditions requiring regular concomitant medication
* History of significant hypersensitivity, anaphylaxis, intolerance to drugs/food
* Propensity to get headaches when refraining from caffeine containing beverages
* Any abnormal laboratory value, vital signs, ECG parameter or physical examination interfering with the test results or causing a health risk for the subject or failure to satisfy the investigator of fitness to participate for any other reason.