cyrilzakka/clinical-trials huggingface dataset:
Immune Function and the Progression to T1D

To elucidate the mechanisms by which type 1 diabetes-associated genes; IFIH1, TYK2, IKZF4, as well as total genetic risk, impart functional immunoregulatory abnormalities that result in expansion of self-reactive adaptive immune cells, defective regulatory/effector mechanisms in T cells, inflammatory antigen presenting cells, and abnormal immune function in T cells and B cells.

Newly proposed studies will identify the inflammatory cues that draw immune cells into islets for disease initiation (Project 1); probe the motility of immune cells through inflamed vasculature to the target organ and antigen priming sites within secondary lymphatics (Project 2); and characterize the T1D-associated adaptive immune signatures in blood and immune tissues (Project 3).

The overall hypothesis of the renewed P01 states: 1) the impact of T1D-risk variants will vary by tissue, cell subset, and activation state, and 2) risk variants, cellular stress, and defects in immunologic pathways are key to engender the autoimmune destruction of pancreatic B-cells that results in T1D.

Eligibility Criteria:
Inclusion Criteria: able to have blood drawn -

Exclusion Criteria: none

-
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).

This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.

Eligibility Criteria:
Inclusion Criteria:

1. Male or female children and adolescents aged ≥6 years and ≤16 years
2. Diagnosis of Congenital DM1 (also known as Steinert's disease)

   * Diagnosis must be genetically confirmed
   * One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth:

     * Hypotonia
     * Generalized weakness
     * Respiratory insufficiency
     * Feeding difficulties
     * Clubfoot or another musculoskeletal deformity
3. Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
4. Written, voluntary informed consent must be obtained before any study related procedures are conducted.

   * Where a parent or LAR provides consent, there must also be assent from the subject
5. Subject's caregiver must be willing and able to support participation for duration of study
6. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol

Exclusion Criteria:

1. Not able to walk; (full time wheel chair use)
2. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
3. New or change in medications/therapies within 4 weeks prior to Screening
4. Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
5. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
6. Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months
7. Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
8. Hypersensitivity to tideglusib and its excipients including allergy to strawberry
Internet CBT for Trichotillomania and Skin Picking Disorder

The primary aim of the study is to test the feasibility, safety and efficacy of a manual based, Internet-delivered, behavior therapy (I-BT) for trichotillomania and skin picking disorder.


Eligibility Criteria:
Inclusion Criteria:

* Primary diagnosis of trichotillomania and/or skin picking disorder according to DSM-5
* Living in Sweden with a Swedish personal identification number
* Able to participate in study assessment in Swedish and understand Swedish text
* Have regular access to a computer with Internet access and skills to use Internet
* Signed informed consent
* Outpatient

Exclusion Criteria:

* Psychotropic medication changes that can affect symptoms of trichotillomania or skin picking disorder within 2 months prior to treatment
* Other ongoing psychological treatments that could affect symptoms of trichotillomania or skin picking disorder
* Completed CBT for trichotillomania or skin picking disorder within the last 24 months
* Ongoing substance dependence or misuse
* Lifetime bipolar disorder or psychosis
* Suicidal ideation (a score of 5 points or higher on item 9 on the self-reported Montgomery-Åsberg depression rating scale
* Psychiatric comorbidity that could severely jeopardize treatment participation
Glucocorticoid Receptor Blockade With Mifepristone in Patients With Mild Adrenal Hypercortisolism

The purpose of this study is to determine whether mifepristone is an effective treatment for hyperglycemia due to mild hypercortisolism.

* To test the hypothesis that GR blockade with mifepristone will decrease the severity of metabolic syndrome features as measured by waist circumference, lipid profile, body mass index, blood pressure and insulin resistance, measured by HOMA-IR score.
* To test the hypothesis that GR blockade with mifepristone will improve QoL, depression and anxiety scores, measured by validated assessments, in patients with mild hypercortisolism.


Eligibility Criteria:
Inclusion Criteria:

* \>18 years of age
* Incidentally noted adrenal nodule \<4 cm with benign imaging characteristics
* Evidence of mild hypercortisolism
* Evidence of diabetes or abnormal glucose tolerance

Exclusion Criteria:

* contraindication to mifepristone
* Indication for unilateral adrenalectomy
* Evidence of other adrenal hormone hypersecretion
* lactating mothers
* women of childbearing age unwilling to use an effective, nonhormonal form of contraception
Efficacy and Safety Study of ZOMETA® in Treatment of High-level NTX Non Small Cell Lung Cancer With Bone Metastasis

A multicenter prospective study to assess the efficacy and safety of ZOMETA® in treatment of high-level NTX non small cell lung cancer with bone metastasis.


Eligibility Criteria:
Inclusion Criteria:

* Age \> 18
* Histologically confirmed non-small cell cancer
* One bone metastasis at least confirmed by image(X ray, CT or others)
* NTX \> 50nM BCE/mM creatinine
* Life expectancy \> 6 M
* ECOG \<= 2
* Signed ICF

Exclusion Criteria:

* Women who are pregnant or in lactation
* Patients with hyperostosis with brain metastasis(exception of those without symptom or with Metastasis Lesions under controlled
* Previous or current treatment with any other bisphosphonates, bone- protecting, cytotoxic or targeted therapy
* Severe co-morbidity of any type that may interfere with assessment of the patient for the study -
Safety and Effectiveness of Ticagrelor in Patients Undergoing Carotid Stenting

patients undergoing carotid stenting take dual anti platelet therapy to prevent thrombotic complications usually used drugs are aspirin plus clopidogrel but there are patients not respond so ticagrelor may be an effective and safe alternative


Eligibility Criteria:
Inclusion Criteria:

1. Ischemis stroke of both sex.
2. More than 18 years.
3. Accepting all study requirements.

Exclusion Criteria:

1. Refuse to comply with all study requirements.
2. bleeding tendency or liver diseases
A Feasibility Study of Wearable Device in Assessing Respiratory Condition Change of Patients With Exacerbation of COPD

The study will include daily SenseGuard measurement of patients hospitalized with exacerbations of COPD. from admission to discharge. In parallel clinical evaluation of patient's respiratory condition was done daily by routine tests and questionnaires.

A study for evaluating the feasibility of SenseGuard in assessing the respiratory condition change of hospitalized patients with exacerbation of COPD, by monitoring respiratory parameters. The study duration for each patient will start at the time of enrollment until the patient is released, or until the optional post-discharge follow-up visit.

The study will include at least one visit per day: The visit and measurement with SenseGuard will start prior to the first medical exacerbation treatment effect-onset given on each study day (by inhalation or IV). The measurement with SenseGuard will be done before and after treatment.

At the investigator discretion, additional two similar visits could be performed on the same study day, with or without the admission of the medical exacerbation treatment.

During the study, both the investigator and the patients will be asked to fill questionnaires to assess the patient's condition Additionally, measurements that are performed and collected as a standard practice or at the request of the medical personnel as part of patient's treatment course shall be documented and used as a reference for the study device assessment.

Eligibility Criteria:
Inclusion Criteria:

1. Primary or secondary diagnosis of AECOPD (Acute Exacerbation of Chronic Obstructive Pulmonary Disease) in patients with GOLD 1-4/A-D
2. WHO performance status ≤ 3
3. Provision of signed and dated informed consent form.
4. Male or female, aged ≥18.
5. Ability and stated willingness to adhere to the study regimen and availability for the duration of the study.

Exclusion Criteria:

3. Inability to provide informed consent including patients still impacted by the use of narcotics as part of emergency care.

4. Intubated patients or patients undergoing a BIPAP/CPAP/NHF treatment and are medically not able to suspend the treatment for the required duration of the visits or expected intolerance of the sensor in their face due to severe respiratory distress.

5. Presence of implanted electronic device intended to regulate cardiac activity (e.g., pacemaker or cardioverter/defibrillator).

6. Pregnancy or lactation. 7. Known allergic reactions to materials used in the components of the SenseGuard™ system, i.e. polyethylene or silicon, or gold, nickel, copper or alloys containing any of the above.

8. Participation in another study that is related to treatment of the respiratory condition.
Effects of Lumbosacral Mobility Program on Tight Hamstrings

The study will be randomized controlled trial to determine the effects of lumbosacral mobility program on muscle length in sedentary young adults with tight hamstrings. Only the eligible subjects who fulfill the inclusion criteria will be included in the study. A sample of 78 subjects will be taken and randomly allocated into two groups with 39 each. Group A will be given three treatments. Manual therapy, mobility exercise therapy and home based self stretching program. Manual therapy mobilization posterior-anterior glides at lumber facets joints, transverse glides at spinous process \& Posterior-anterior glides at SI joint. Glides will be performed 3 times with 5 repetitions. Mobility exercise therapy (Single knee to chest, double knee to chest, Lumbar rotation, Lumbar extension, Pelvic tilting). A total of 30 min sessions three times a week on alternate days for 1 week \& home-based self-stretches program twice a day for three times a week on alternate days for 1 week. Group B will be given home-based self-stretching program twice a day for three times a week on alternate days for 1 week .The baseline values of all the dependent variable will be recorded on day one and at the end of the 1-week treatment session, using goniometry, active and passive knee extension test, passive straight leg raising and lower extremity functional scale will be used

The hamstring muscle group consists of four muscles (semitendinosus, semimembranosus, biceps femoris long head, biceps femoris short head). It's a two-joint muscle working simultaneously to act as a hip extensor and knee flexor. It also functions to maintain the stability of the knee joint. The hamstring plays important role in daily activities like running \& walking. A lot of factors affecting hamstring function, one of them is flexibility, it is defined as the extent to which muscle length contributes to maximal joint range of motion. It is a well-known muscle of has a shortening tendency. There are many methods for treating hamstrings flexibility/tightness/shortening. Routinely, physiotherapists are using stretching \& strengthening exercises, instruments assisted in soft tissue mobilization. The current study focuses on two treatments manual therapy \& lumber mobility exercises

Eligibility Criteria:
Inclusion Criteria:

* Age 20-30 years
* Sedentary life style
* BMI \< 23 non-obese
* Hamstring shortness ≥ 20° using the active knee extension test (aket)
* No history of orthopedic/neurologic disorders in the lower extremity
* No history of back pain in last 2 weeks
* No contraindication for using manual therapy
* No joint limitation in the lower extremities

Exclusion Criteria:

* Any systemic soft tissue and bony disease
* Fracture
* Neuropathy
* Congenital deformities of spine
Interdisciplinary Value-based Cognitive Behavioral Treatment for People with Persistent Whiplash Associated Disorders.

The goal of this clinical trial is to address the critical need for effective interventions by developing and testing an interdisciplinary values-based cognitive behavioral therapy (V-CBT) program for individuals with persistent whiplash. The main question it aims to answer are:

* What is the treatment effect of the interdisciplinary values-based cognitive behavioral therapy (V-CBT) program on individuals with persistent whiplash?
* What are the temporal relationships between primary and secondary outcome measures in the context of the V-CBT program?

Participants will undergo a replicated multiple baselines single-case experimental design (SCED). Twelve participants who meet specific eligibility criteria will be block-randomized to a 1, 2, or 3-week baseline phase, serving as their own controls. The intervention involves an interdisciplinary team delivering a manualized adapted V-CBT program over ten sessions, focusing on psychological and physical rehabilitation. Participants will be required to attend these sessions as part of the treatment protocol. Data collection includes daily online self-reports and comprehensive measurements at pre-treatment, post-treatment, and a three-month follow-up.


Eligibility Criteria:
Inclusion Criteria:

* have persistent symptoms after a whiplash injury that happened more than 3 months ago
* have pain and disability defined as \> 35 % on Neck Disability Index (NDI)
* be 18 years or older
* speak and read Danish

Exclusion Criteria:

* denote headache as the primary problem
* show red flags in the neurological and physiotherapeutic examination such as known or suspected serious spinal pathology, confirmed fracture or dislocation in the neck at the time of injury or signs of nerve root compromise (weakness, reflex changes or sensory loss)
* have had spinal surgery within the past 12 months
* are socially and occupationally unresolved
* have daily significant functional limitations related to other pain conditions
* have severe psychiatric illness
* are pregnant
* are not willing or able to respond to items on a daily basis.
Tobacco Control Policy-related Survey 2021 (Online)

Aim: to collect further information on issues related to tobacco control which should be addressed and advocated in further tobacco control measures in Hong Kong.

The objectives of this policy-related survey by online panel are as follows:

1. to update information on prevalence of conventional cigarettes, e-cigarettes, heated tobacco products and waterpipe tobaccos;
2. to investigate the effects of new pictorial health warning policy in the public;
3. to investigate the level of public support on current and future tobacco control measures and taxation;
4. to address the timely impacts of smoke-free policies, public support for further tobacco control measures and assist COSH in shaping policy direction for government;

Current study is a repeated cross-sectional online survey based on questionnaires. A total of 2 waves of survey will be conducted 4 months apart starting from early 2021. The target sample size in each wave of survey is planned to be 900 Hong Kong residents. The online survey will be conducted by a survey agent under the monitor of applicants. All eligible registered members of the survey agent panel will be invited to respond to the questionnaire. Once a member responded to the first wave of this study, he/she would no longer receive invitation for the subsequent wave. All responses will be kept anonymously. Analysis of survey data will aim to identify current opinion on tobacco control policies and implications for future policies. All eligible subjects who complete the online survey will be included in the analysis. Comparison with past trends conducted by landline and mobile phone surveys and, where possible, extrapolation of future trends will be explored. Weightings will be applied to adjust for the sex and age of Hong Kong population, and to handle the over-sampling issues of ex- and current smokers. The data analysis will be conducted by experienced statisticians in the applicants' team.


Eligibility Criteria:
Inclusion Criteria:

* Hong Kong resident aged 15 and above
* Traditional Chinese readers

Exclusion Criteria:

* Unable to provide a consent
* Know languages other than Chinese
Expanded Access Use of Omegaven® in the Treatment of Parenteral Nutrition Induced Liver Injury in Children

This is an expanded access study to assess the safety profile and changes in serum direct bilirubin levels in infants with PN associated cholestasis. Eligible patients will receive therapy with Omegaven on an expanded access basis by method of continuous infusion. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition. The same standards of care provided to all patients receiving parenteral nutrition solution will be followed.

This expanded access protocol provides a mechanism for critically ill infants with parenteral nutrition-associated liver disease (PNALD) to receive Omegaven for compassionate use situations for which there are no satisfactory alternative treatments.

Eligibility Criteria:
Inclusion Criteria:

* Greater than 4 weeks (28 days) old and less then 5 years of age
* Diagnosis of PNALD as defined by serum direct bilirubin greater than 2 mg/dL on 2 consecutive occasions
* Expected to require intravenous nutrition for at least an additional 28 days
* Have been PN-dependent for at least four weeks prior to planned Omegaven initiation
* PN-dependent and unable to meet nutritional requirements by enteral means
* Have failed standard therapies to prevent progression of PNALD
* Hospitalized at time of Omegaven initiation

Exclusion Criteria:

* Have a congenitally lethal condition (e.g. Trisomy 13).
* Have evidence of a viral hepatitis or primary liver disease as the primary etiology of their cholestasis.
* Have other health problems such that survival is extremely unlikely even if the infant's cholestasis improves.
* Have been in another clinical trial within 30 days prior to enrollment or received an investigational drug within 30 days prior to enrollment or scheduled to receive an investigational drug other than Omegaven during the study period.
* Severe and/or unstable concomitant systemic disease such as complex congenital cardiac disease, renal failure, autoimmune disease, sepsis, inborn error of metabolism, genetic liver disease
* Bleeding disorder
* Biochemical disturbance with potential of worsening with proposed treatment, e.g. persistent hyperglycemia, hypertriglyceridemia, hypercalcemia.
Morphine Analgesia in Patients With Acute Appendicitis

The purpose of the study is to evaluate morphine effect on pain reduction and diagnostic accuracy in patients admitted to emergency ward with the diagnosis of acute appendicitis.

Analgesic administration for pain control in patients with acute abdominal pain, regarding acute appendicitis, is controversial. Despite the results previous studies indicating the safeness of analgesic use in ED for acute abdominal pain, physicians still prefer to postpone their use after performing surgical evaluations. Since many patients admitted to ED are unlikely to receive prompt surgical consultation, many of them need pain relief to prevent unnecessary suffering. On the other hand such pain relief must not interfere with the diagnosis and treatment of patients and do not cause any complications. Morphine is a common and effective analgesic which, if shown to have no effect on correct diagnosis of acute abdomen, can be used for this purpose.

Thus we evaluated morphine effect on pain reduction and diagnostic accuracy in patients admitted to ED with the diagnosis of acute appendicitis.

Eligibility Criteria:
Inclusion Criteria:

* clinical signs of acute appendicitis
* planned for appendectomy
* supposed to wait for at least 1 hour in the ED before operation

Exclusion Criteria:

* suspicion to perforated appendicitis
* being under the age of 13
* pregnancy (according to history and ß-HCG result)
* opium addiction
* systolic blood pressure of less than 90 mmHG
* known cases of chronic obstructive pulmonary disease (COPD)
* known sensitivity to morphine
* history of sickle cell disease
* self administration of analgesics before enrollment
* refusal to participate
Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis

Brief Summary: This study was designed to explore a safe dose and characterize the preliminary safety and efficacy of ICL670 in adult patients with previously documented history of homozygous C282Y.


Eligibility Criteria:
Inclusion Criteria:

* Age 18 years of age or older
* Male or female patients homozygous for the C282Y mutation.
* Iron overload as documented by serum ferritin and transferrin saturation
* No known allergy or contraindication to the administration of deferasirox
* Ability to comply with all study-related procedures, medications, and evaluations
* Effective use of birth control measures.

Exclusion Criteria:

* Iron overload not due to hereditary hemochromatosis
* Males with hemoglobin \<13 mg/dL, females with hemoglobin \<12 mg/dL
* Desferal treatment within 1 month of the screening visit
* Patients currently or previously treated with deferiprone or deferasirox
* Significant medical condition interfering with the ability to partake in this study
* Presence of a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any study drug
* Clinical evidence of Active Hepatitis B or C
* Positive HIV serology
* Pregnant or breast feeding patients
* Patients treated with systemic investigational drug within 4 weeks prior or with topical investigational drug within 7 days prior to the screening visit

Other protocol-defined inclusion/exclusion criteria may apply.