cyrilzakka/clinical-trials huggingface dataset:
Rural Intervention for Caregivers' Heart Health

The investigators are testing a two-group, randomized, controlled trial with 280 primary caregivers of adult patients with a chronic illness to test the efficacy of the Rural Intervention for Caregivers' Heart Health (RICHH). Immediate (4-month) and long-term (12-month) effects of the RICHH intervention on CVD risk factors, self-management behaviors, and depressive symptoms will be compared to usual care. The investigators also will test the moderating effect of gender on intervention outcomes, given the many differences between male and female caregivers.

Specific Aim 1: To determine effects of the RICHH intervention on the primary outcomes of CVD risk factors (i.e., lipid profile, body mass index, and blood pressure) at 4 and 12 months.

Hypotheses 1: The intervention group will have better lipid profile, body mass index, and blood pressure outcomes than the usual care group at 4 and 12 months.

Specific Aim 2: To determine effects of the RICHH intervention on self-management behaviors (i.e., diet quality, physical activity level, and self-report adherence to specific CVD health behaviors).

Hypotheses 2: The intervention group will have better diet quality, higher physical activity levels, and better adherence to specific CVD health behaviors than the usual care group at 4 and 12 months.

Specific Aim 3: To determine effects of RICHH on depressive symptoms at 4 and 12 months.

Hypotheses 3: Caregivers receiving the intervention will have lower levels of depressive symptoms than caregivers receiving usual care at 4 and 12 months.

Specific Aim 4: To evaluate whether intervention effects on outcomes will differ by caregivers' gender.

Hypotheses 4: Effects will be stronger for male compared to female caregivers at 4 and 12 months.

Impact: The proposed study will have a substantial impact on caregivers' cardiovascular health and quality of life because it will provide needed CVD risk prevention, and health promotion to rural caregivers living in distressed environments where CVD risk reduction and self-management is difficult. The intervention holds the potential to produce major improvements in health among caregivers living in rural environments in the US.


Eligibility Criteria:
Inclusion Criteria:

* primary family rural caregiver of patients with chronic illnesses
* provided care for the patient for \> 6 months
* no cognitive impairment that would preclude understanding the consent process

Exclusion Criteria:

* chronic drug abuse
* current active cancer
* any physical or emotional impairment that limits participants' abilities to engage in self-management or that is likely to result in needing a caregiver in the next 12 months.
Real-Life Outcomes of Multiple Sclerosis Treatment With Rebif

The purpose of this study is to determine whether Rebif has an impact on employment status, quality of life and cognition.

With this study the investigator plans to evaluate the impact of Rebif on the Real-Life Outcomes (RLO) of Multiple Sclerosis (MS) patients followed at the Clinic within the last two years, and with a follow-up of up to 18 years.

The investigator will evaluate the employment outcomes with a questionnaire designed to document eventual changes in the employment status and other variables in the work conditions of the study participants.

Furthermore, to evaluate the quality of life (QoL),eligible patients will be asked to complete the Multiple Sclerosis Quality of Life (MSQOL)-54 Instrument, contains 52 items distributed into 12 scales, and two single items. This MS-specific QoL assessment tool uses the Short Form Health Survey (SF-36) as its generic core measure and includes 18 additional items under the following categories: health distress, sexual function, satisfaction with sexual function (one item), overall quality of life, cognitive function, energy, pain, and social function.

A sub-group of patients will be selected to come to the clinic to undergo the cognitive portion of the study, using the well-known and validated battery of tests named Minimal Assessment of Cognitive Function in MS (MACFIMS battery).

Socio-demographic data on education level, marital and family life will also be collected. All questionnaires (including the MSQoL-54) will be available by means of an online survey.

Eligibility Criteria:
Inclusion Criteria:

* Patients with CIS or definite MS (RRMS or SPMS);
* Patients seen at our Clinic within the last 2 years;
* Patients untreated, or treated with either low dose or high dose Rebif for at least two years;
* Patients between 18 to 60 years old at time of treatment initiation;
* EDSS ≤ 5.5 at treatment initiation;
* Patients able to read and write in French.

Exclusion Criteria:

* Patients diagnosed with primary progressive MS;
* Patients treated with other DMD, other than Rebif;
* Co-existence of other diseases that could influence outcomes.
Prospective Huntington At Risk Observational Study

The purpose of this study is to define the natural history and experiences of people who are at risk for developing Huntington's disease but who do not know their genetic status.

Huntington's disease (HD) is a genetic disease characterized by changes in movement and behavior. To date, little research has been done on individuals who are at risk for developing the disease. PHAROS is an observational study designed to monitor people who are at risk for developing HD, and to survey their attitudes and beliefs about their "at-risk" status. Investigators hope this study will help answer some important questions about HD, such as: 1.) What are the earliest signs of HD and when do they start? 2.) How accurate are the measures that physicians use in detecting the onset of HD? 3.) What factors influence the age at which a person carrying the HD gene develops the illness? and 4.) In a group of people at risk for HD, how many will develop signs of the illness over a minimum three-year period of observation?

Participants in the study will be evaluated every 9 months (for 5 years) using the Unified Huntington's Disease Rating Scale (UHDRS)--a clinical tool which looks at movement, psychological and behavioral function. The participants will also be asked to complete surveys about their mood, life events, and attitudes or beliefs about being at risk for HD. A blood sample, taken at the beginning of the study, will be confidentially tested to determine if the participant has the abnormal gene for HD. Neither the participant nor any of the PHAROS physicians or coordinators will learn the results of individual gene tests.

The scientists hope that this study will provide essential information for future trials of experimental drugs for HD.

Eligibility Criteria:
Inclusion:

* Men and women between 26-55 years of age;
* Who are at risk for HD by virtue of having (or had) a parent or sibling with the illness;
* Who have never been tested for the HD gene, and who do not desire to be tested for the HD gene during the duration of the study;
* Agree to blinded (sample will be coded) genetic testing of the HD gene; and
* Who have never been diagnosed with HD.

Exclusion:

Individuals Who:

* have been diagnosed with HD;
* are taking antipsychotic medications;
* use antiemetic (anti-nausea) medications with on a regular basis (greater than 3 times per month); or
* exhibit clinical signs/symptoms of psychosis at the screening visit
The Potential of a Low Glutamate Diet as a Treatment for Pediatric Epilepsy

The study is investigating if following the low glutamate diet for 1 month, as compared to care as usual, can improve seizure frequency, severity, and duration; cognitive functioning; and/or quality of life in children with epilepsy.

Epilepsy is estimated to effect 470,000 children within the U.S. About a third of epilepsy patients are refractory. For these patients, dietary therapy is an alternative option. However, current dietary therapies present issues with compliance, palatability, adverse events, and providing necessary nutrients for brain growth and development during childhood. A dietary option that can improve tolerability, increase compliance, and reduce or eliminate adverse effects, while also optimizing nutrient intake, is critical for the advancement of dietary therapy options in epilepsy.

Glutamate is found in the diet as a flavor enhancer; and is also an important neurotransmitter in the body which mediates seizure activity. Dietary sources of free glutamate are common and include numerous food additives (including many hidden sources) and some items which naturally contain higher amounts of glutamate. The low glutamate diet reduces the consumption of free glutamate, while optimizing dietary micronutrient and antioxidant intake, which can protect against the negative effects caused by high glutamate concentrations in the brain. Additionally, the low glutamate diet is balanced, nutritious and palatable, with no side effects.

The study is investigating if following the low glutamate diet for 1 month can improve seizure frequency, severity, and duration; cognitive functioning; and/or quality of life, in children with epilepsy. Participation in the study will be done virtually and over a 2- or 3- month study period, depending on group randomization. All participants will have the opportunity to undergo dietary training and follow the diet.

Eligibility Criteria:
Inclusion Criteria:

* 4 or more seizures per month
* Willing to keep all medications constant during the study.
* All medications have remained constant for 30 days prior to study enrollment
* Any previously attempted dietary therapies stopped before study enrollment

Exclusion Criteria:

* Known or suspected inborn errors of metabolism
* Non-English speaking households
Endometrial Safety Study of a 0.5 mg Estradiol and 2.5 mg Dydrogesterone Combination

The purpose of this study is to demonstrate endometrial safety of continuous combined 0.5 mg estradiol and 2.5 mg dydrogesterone.


Eligibility Criteria:
Inclusion Criteria:

* Amenorrhoea for \>= 12 months.
* Serum estradiol and FSH level within the postmenopausal range
* Baseline endometrial biopsy assessed by light microscopic histological evaluation revealing: insufficient endometrial tissue for diagnosis because of insufficient available (atrophic) endometrial tissue (not because of an inaccessible cervix) and endometrial thickness \< 5 mm (double layer) by transvaginal ultrasound, atrophic endometrium, secretory endometrium, menstrual type endometrium, proliferative endometrium

Exclusion Criteria:

* Previous systemic unopposed estrogen replacement therapy over 6 months or more.
* Any estrogen, progestogen, and/or androgen therapy in the last four weeks before Screening Visit (Visit 1). The baseline endometrial biopsy should in all cases be taken after cessation of the withdrawal bleeding due to previous hormone replacement therapy.
* History or presence of an estrogen dependent neoplasia (including breast- cancer).
* History or presence of malignant neoplasms other than basal or spinal cell carcinoma of the skin.
Inspiratory Muscle Training and Low Back Pain

Proprioceptive weighting changes may explain differences in postural control performance. In addition, the respiratory movement has a disturbing effect on postural balance. Postural balance seems to be impaired in individuals with respiratory disorders. Besides the essential role of respiration, the diaphragm may also play an important role in the control of the trunk and postural balance. Deficits in proprioception are found in a subgroup of patients with low back pain. In addition, disorders of respiration have been identified as strongly related to low back pain.

The aim of the study is to clarify whether inspiratory muscle training has a positive effect on proprioceptive postural control in individuals with recurrent low back pain.


Eligibility Criteria:
Inclusion Criteria individuals with low back pain:

* Age: 18-45 years old
* At least 1 year of low back pain with/without referred pain in buttock/thigh
* At least 3 episodes of disabling low back pain
* At least a score of 20% on the Oswestry Disability Index
* Willingness to sign the informed consent

Exclusion Criteria:

* History of major trauma and/or major orthopedic surgery of the spine, the pelvis or the lower quadrant
* One of the following conditions: Parkinson, multiple sclerosis, stroke, history of vestibular disorder, respiratory disease, pregnancy
* Radicular symptoms
* Not Dutch-speaking
* Strong opioids
* Neck pain
* Smoking history
GNT0006 Gene Therapy Trial in Patients With LGMDR9

Phase 1-2 study including a dose escalation safety and proof of concept phase (Stage 1, open label), followed by a double-blind, randomized, placebo-controlled confirmatory phase (Stage 2)

Multicenter, Phase 1-2 study evaluating safety, pharmacodynamic, efficacy, and immunogenicity of GNT0006, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.

This study will consist of 2 phases: an open-label dose escalation phase (Stage 1) and a double-blind placebo controlled, randomized phase (Stage 2), both with long-term follow-up (LTFU) period.

Stage 1 Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.

Stage 2 After selection of the effective dose in Stage 1, thirty-three (33) ambulant patients will be randomized at the optimal selected dose and followed up to the primary efficacy timepoint, i.e., one year after investigational medicinal product (IMP) (or placebo) administration.

At one-year post-IMP administration (timepoint of primary interest for efficacy), patients enrolled in placebo group will receive active IMP while patients randomized in the active IMP group will receive a placebo infusion.

All subjects will be followed for up to 5 years after active IMP (GNT0006) administration.

Eligibility Criteria:
Inclusion Criteria:

* 1. Female and male ambulant patients
* 2. Patients ≥ 16 years old
* 3. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
* 4. Moderate diaphragmatic muscle impairment

Exclusion Criteria:

* 1. Detectable serum neutralizing antibodies against AAV9
* 2. Cardiomyopathy
Precision Antiplatelet Therapy Guided by Platelet Aggregation Function in Patients with Acute Ischemic STROKE

The objective of this clinical trial is to evaluate the efficacy and safety of platelet aggregation function - guided precision anti - platelet therapy in patients with acute cerebral infarction. The main question it aims to answer is: among the cerebral infarction patients with possible clopidogrel resistance detected by platelet aggregation function tests, what is the efficacy and safety of using ticagrelor to replace the clopidogrel treatment regimen.


Eligibility Criteria:
Inclusion Criteria:

* 1.Aged between 18 and 80 years old; 2.Diagnosed with non-cardiogenic ischemic stroke or TIA according to WHO criteria, confirmed by cranial CT or MRI to exclude hemorrhagic stroke.; 3.This stroke onset is ≤ 48 hours; (The duration of onset is defined as the time from the last time the patient was seen normal to the time of taking 300 mg of clopidogrel and 100 - 300 mg of aspirin in combination) 4.The NIHSS score is ≤ 5 points before randomization, or for TIA patients, the ABCD2 score is ≥ 4 points; 5.Within 48 hours after the patient's onset, 5 - 20 hours after receiving anti - platelet therapy with a combination of 300 mg of clopidogrel and 100 - 300 mg of aspirin, the measured MARADP is ≥ 35%; 6.The patient or their family member signs a written informed consent form.

Exclusion Criteria:

* 1.Imaging examination indicates hemorrhagic stroke or hemorrhagic transformation.

  2.There is a history of diseases such as atrial fibrillation, left ventricular thrombosis, cardiomyopathy or valvular disease.

  3.There are the following congenital heart diseases: patent foramen ovale, ventricular septal defect, atrial septal defect and tetralogy of Fallot.

  4.Other P2Y12 receptor inhibitors (e.g., ticagrelor, prasugrel) except clopidogrel have been taken within 7 days.

  5.There is a history of gastrointestinal bleeding, intracranial bleeding, recent massive bleeding or blood transfusion (excluding a small amount of hemoptysis and a small amount of abnormal vaginal bleeding), as well as a history of other hemorrhagic diseases caused by abnormal blood coagulation function (such as purpura, etc.).

  6.There are contraindications or intolerance to anti - platelet drugs. 7.Patients with severe cardiac, pulmonary, hepatic and renal insufficiency, and those with serious comorbidities (e.g., cancer, chronic obstructive pulmonary disease, severe dementia, severe heart failure).

  8.Those with poor compliance who cannot cooperate to meet the requirements of the study.
Choice of Anesthesia in Microelectrode Recording Guided Deep Brain Stimulation for Parkinson's Disease

Subthalamic nucleus (STN)-deep brain stimulation (DBS) under general anesthesia has been applied to PD patients who cannot tolerate awake surgery, but general anesthesia will affect the electrical signal in microelectrode recording (MER) to some degree. This study is a prospective randomized controlled, noninferiority study, open label, endpoint outcome evaluator blinded, two-arm study. Parkinson's disease patients undergoing STN-DBS are randomly divided into a conscious sedation group (dexmedetomidine) and a general anesthesia group (desflurane). Normalized root mean square (NRMS) is used to compare the difference of neuronal activity between the two groups. The primary outcome is the percentage of high NRMS recorded by the MER signal (with the average NRMS recorded by MER after entering the STN greater than 2.0). The secondary outcomes are the NRMS, length of the STN, number of MER tracks, and differences in clinical outcomes 6 months after the operation.

STN-DBS under general anesthesia has been applied to PD patients who cannot tolerate awake surgery, but general anesthesia will affect the electrical signal in microelectrode recording (MER) to some degree. At present, there are some studies on the effects of desflurane on neuronal signal amplitude and discharge characteristics during STN-DBS in PD patients but there is no definite conclusion.

This study compares the influence of MER mapping during STN-DBS and the differences in postoperative clinical outcomes between desflurane general anesthesia and conscious sedation anesthesia to explore alternative anesthesia for DBS in PD patients who cannot tolerate local anesthesia or conscious sedation and to provide feasible anesthesia techniques for the application of MER during DBS under general anesthesia.

This study is a prospective randomized controlled, noninferiority study, open label, endpoint outcome evaluator blinded, two-arm study. Parkinson's disease patients undergoing STN-DBS are randomly divided into a conscious sedation group (dexmedetomidine) and a general anesthesia group (desflurane). The primary outcome is the percentage of high NRMS recorded by the MER signal (with the average NRMS recorded by MER after entering the STN greater than 2.0), which is used to compare the differences in neuronal electrical activities between conscious sedation and general anesthesia via desflurane groups. The secondary outcomes are the NRMS, length of the subthalamic nucleus, number of MER tracks, and differences in clinical outcomes 6 months after the operation.

Eligibility Criteria:
Inclusion Criteria:

1.50-80 years old, ASA grade II-III; 2.Bilateral STN-DBS of patients with Parkinson's disease; 3.Signed informed consent.

Exclusion Criteria:

1. Obstructive sleep apnea;
2. BMI \> 30kg/m2;
3. Estimated difficult airway;
4. Severe preoperative anxiety;
5. Serious dysfunction of important organs (i.e. heart failure, renal or liver dysfunction)
6. A history of allergy to the anaesthetics.
Sargramostim in Treating Patients With Kidney Cancer That Has Spread to the Lung

RATIONALE: Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood and may be an effective treatment for patients with kidney cancer that has spread to the lung.

PURPOSE: Phase II trial to study the effectiveness of sargramostim in treating patients who have kidney cancer that has spread to the lung.

OBJECTIVES:

* Determine the 4-month progression-free survival rate and overall survival rate in patients with metastatic renal cell carcinoma to the lung treated with aerosolized sargramostim (GM-CSF).
* Determine the toxicity of this regimen in these patients.
* Determine the immunomodulatory effects of this regimen in terms of natural killer cells cytotoxicity, and T-cell, B-cell, and dendritic cell activation markers.

OUTLINE: This is a multicenter study.

Patients receive aerosolized sargramostim (GM-CSF) by nebulizer over 10-15 minutes twice daily on days 1-7 and 14-21. Treatment repeats every 28 days in the absence of disease progression or unaceptable toxicity.

Patients are followed for disease progression and then every 3 months thereafter.

PROJECTED ACCRUAL: A total of 21-48 patients will be accrued for this study within 7-20 months.

Eligibility Criteria:
DISEASE CHARACTERISTICS:

* Histologically confirmed metastatic renal cell carcinoma for which no known standard therapy that is potentially curative or capable of extending life expectancy exists (e.g., surgery)
* Measurable metastatic disease in the lung

  * At least one unidimensionally measurable lesion at least 20 mm by conventional techniques
* No CNS metastases that require treatment

PATIENT CHARACTERISTICS:

Age

* 18 and over

Performance status

* ECOG 0-2

Life expectancy

* At least 12 weeks

Hematopoietic

* Absolute neutrophil count at least 1,000/mm\^3
* Platelet count at least 75,000/mm\^3
* Hemoglobin greater than 8.0 g/dL

Hepatic

* Bilirubin no greater than 2 times upper limit of normal (ULN)
* AST no greater than 3 times ULN

Renal

* Creatinine no greater than 2.5 times ULN

Pulmonary

* No hemoptysis of grade 3 or greater
* No reactive airway disease on active therapy

Other

* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
* No uncontrolled infection
* No other metastatic malignancy within the past 3 years except basal cell skin cancer

PRIOR CONCURRENT THERAPY:

Biologic therapy

* More than 2 weeks since prior immunotherapy
* More than 2 weeks since other prior biologic therapy

Chemotherapy

* More than 3 weeks since prior chemotherapy (6 weeks for mitomycin or nitrosoureas)

Endocrine therapy

* More than 2 weeks since prior corticosteroids
* No concurrent systemic glucocorticoids

Radiotherapy

* More than 2 weeks since prior radiotherapy
* No prior radiotherapy to more than 10% of total lung volume in the radiation field

Other

* At least 4 weeks since prior bronchodialators
* No concurrent immunosuppressive agents