cyrilzakka/clinical-trials huggingface dataset:
A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)

This study is the first study in the RestorAATion clinical program.

The purpose of this first-in human (FIH), double-blind, randomized, placebo-controlled, single ascending dose (SAD) and multiple-dose Phase 1 study is to assess the safety, tolerability, and PK of WVE-006 compared to placebo in healthy participants following a single dose (Period 1) and multiple doses (Period 2) of WVE-006.

This information will be used to determine doses and regimes that have the potential to be pharmacologically active in patients with Alpha-1 antitrypsin deficiency in the RestorAATion 2 study, and the maximum safe and tolerable dose that may be given to these patients.


Eligibility Criteria:
Inclusion Criteria:

* Healthy as determined by the Investigator, based on a medical evaluation.
* Genetic testing confirming PI\*MM.
* Participant has been a non-smoker for at least 1 year prior to screening.

Exclusion Criteria:

* Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc).
* Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections.
* Any ongoing or recent infections.
* Any recent or planned vaccinations during the study.
* Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week.
* Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits.
* Participant has a history of caffeine consumption exceeding 8 cups of coffee/day.
* Use of prescription or non-prescription medications, including vitamin, dietary, and herbal supplements (including St John's Wort) within 7 days prior to the first dose of study treatment unless, in the opinion of the Investigator and Sponsor, the medication will not interfere with interpretation of study assessments. Contraception and hormone replacement therapy (HRT) are permitted. If needed, over-the-counter (OTC) medications such as paracetamol/acetaminophen may be used acutely.
* Any recent or planned major surgery during the study.
* Donation of blood or blood products in excess of 500 mL within 12 weeks prior to Screening Visit and/or unwilling to refrain from blood donation for the duration of the study.
* Participant has received an investigational agent within 3 months of the Screening Visit.
Technology-enabled Task-sharing for Depression in Primary Care

This study will explore and test the feasibility, acceptability, usability, and preliminary effectiveness of a technology-enabled intervention for depression using task-sharing in primary care. We will a) discover barriers and facilitators to task-sharing by frontline primary care staff; b) design an implementation strategy to support task-sharing to deliver a technology-enabled intervention for depression; and c) conduct a small open-label usability trial of the technology-enabled intervention for depression.

Older adults with depression typically present to primary care rather than specialty mental health treatment and are often un- or undertreated, as the demand for mental health services is greater than the supply of trained providers. Technology is one method to improve access to care by making evidence-based psychosocial interventions (EBPIs) readily accessible. A second method comes from global mental health research, demonstrating that task-sharing can equip non-specialists to provide effective mental health care. This study combines these two approaches, exploring how technology-enhanced EBPI could be used by frontline primary care staff (e.g., nurses, medical assistants) to expand workforce capacity to deliver acceptable, sustainable, and effective treatment for depression. Specifically, we will use task-sharing to deliver a mobile Motivational Physical Activity Targeted Intervention (MPATI), which is based on behavioral activation for depression and uses wearable accelerometer technology to trigger personalized activity goal monitoring. This proposal uses the Discover, Design/Build, Test (DDBT) framework, which leverages user-centered design and implementation science to discover implementation barriers to using task-sharing to deliver MPATI in primary care, to design an implementation strategy to support MPATI delivery, and to conduct a pilot usability trial to test the implementation strategy with the most suitable frontline staff.

Eligibility Criteria:
Inclusion Criteria:

Clinics:

1. have at least 1 full-time registered nurse (RN) and/or medical assistant (MA) on staff
2. include older adults on their patient panels.

Clinic administrators

1. have an administrative or leadership role in the clinic
2. have been employed in their current role for at least 6 months.

Frontline staff

1. provide care as RN, MA, case manager, behavioral health consultant, or similar role identified by Practice Champion
2. be employed at the participating clinic for at least 6 months.

Patients

1. be ≥65 years of age
2. report moderate to moderately severe depressive symptoms based on a PHQ-9 score of 10-20
3. own or have access to a smartphone
4. have internet or cellular data plan
5. receive medical clearance from their primary care provider to participate in unstructured physical activity.

Patient exclusion criteria will be based on medical chart review by Practice Champion and include:

1. current suicidality
2. severe vision or hearing impairment
3. pronounced cognitive impairment
4. use of assistive devices that would impede physical activity.
Evaluate the Safety and Efficacy of CLL1+CD33 CAR-T in Patients With R/R AML

This is an open label, phase I study to assess the safety and efficacy of CLL1+CD33 CAR-T in patients with relapsed and refractory acute myeloid leukemia


Eligibility Criteria:
Inclusion Criteria:

1. All subjects must sign and date the Informed Consent before initiating any study specific procedures or activities;
2. Diagnosed as relapse/refractory (r/r) de novo or secondary acute myeloid leukemia (AML);
3. The expression of CLL1 and CD33 in AML blast is positive ;
4. The patient has recovered from the toxicity of previous treatment;
5. ECOG score ≤ 2 and expected survival period is not less than 3 months;
6. Adequate organ function defined as:

   AST ≤3×ULN; ALT ≤3×ULN; Total bilirubin ≤1.5×ULN; Serum creatinine ≤1.5×ULN, or CCR≥60 mL/min; Hemoglobin ≥60g/L ; Indoor oxygen saturation ≥92%; LVEF≥45%;
7. Pregnancy testing: females of childbearing potential must have a negative serum or urine pregnancy test;
8. From the use of study drug to 2 years after treatment, males and female of childbearing potential must agree to use an effective method of contraception

Exclusion Criteria:

1. Diagnosis of acute promyelocytic leukemia;
2. History or presence of a CNS disorder;
3. HBsAg or HBcAb are positive; HCV 、HIV and Syphilis antibody are positive, CMV DNA in peripheral blood is more than≥500 copies /mL;
4. History of severe hypersensitivity reaction;
5. History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, New York Heart Association Class II or greater congestive heart failure, atrial fibrillation, or other clinically significant cardiac disease within 12 months before enrollment;
6. History of organ transplant surgery;
7. Required systemic application of immunosuppressive or other drugs;
8. Auto-SCT within the 3 months before enrollment;
9. Active autoimmune or inflammatory diseases of the nervous system (e.g., Guillain-Barre syndrome (GBS), amyotrophic lateral sclerosis (ALS)) and clinically active cerebrovascular diseases (e.g., cerebral edema, posterior reversible encephalopathy syndrome (PRES));
10. Requirement for urgent therapy due to ongoing or impending oncologic emergency (eg, leukostasis or tumor lysis syndrome (TLS)) ;
11. Presence or suspicion of a fungal, bacterial, viral, or other infection that is uncontrolled or requiring antimicrobials for management;
12. Live vaccine received within the ≤ 4 weeks before enrollment;
13. Persons with serious mental illness;
14. History of major surgical operations four weeks before enrollment;
15. History of alcoholism or substance abuse;
16. Was identified by the investigators as unsuitable to participate in the study.
Comparison of MiSight 1-Day and Acuvue Oasys for Presbyopia in Myopic Children

This study will compare the effects on myopia progression over the course of one year for eyes wearing MiSight 1 Day contact lenses compared to ACUVUE OASYS for PRESBYOPIA contact lenses.


Eligibility Criteria:
Inclusion Criteria:

1. Aged 6-12 years (inclusive)
2. Is willing to wear soft contact lenses daily
3. Spherical equivalent myopic refractive error between -0.50 to -5.00 D in each eye and astigmatism \< 1.00 D
4. Best corrected logMAR visual acuity of 0.1 or better in both eyes
5. Good ocular and general health
6. No previous use of myopia control interventions for more than 1 month or within the last 30 days
7. Competent enough in English to be able to fully understand the participant information and consent form

Exclusion Criteria:

1. Strabismus at distance or near, amblyopia or significant anisometropia (\>1D)
2. Systemic or ocular conditions that may affect contact lens use (e.g., allergy) or that may affect refractive development (e.g., ptosis)
3. Previous history of ocular surgery, trauma, or chronic ocular disease
4. Contraindications to contact lens use
5. Ocular or systemic medication use which may interfere or interact with contact lens wear or ocular development
6. Plans to migrate or move during the study
7. Child, parents or guardians not willing to comply with treatment and/or follow-up schedule
PK/PD of Vitamin D3 in Adults With CF

Despite the extensive literature on adverse clinical outcomes associated with vitamin D deficiency, there are currently no proven treatment strategy that effectively achieves and maintains optimal serum vitamin D status in cystic fibrosis (CF) patients. For the treatment of vitamin D deficiency, CF Foundation currently recommends 2,000 IU daily. However, because achieving adequate serum 25(OH)D levels is a challenge in CF, higher doses of vitamin D may be necessary to reach and maintain vitamin D sufficiency. Poor oral bioavailability of ergocalciferol has been demonstrated in CF patients, which may potentially also be an issue with cholecalciferol. In order to optimize the treatment of vitamin D deficiency in CF, the kinetic disposition must be well understood. However, there are very few data currently available describing the kinetics of both vitamin D and 25-hydroxyvitamin D, and to the investigator's knowledge, no studies have yet characterized the pharmacokinetic disposition of vitamin D and its metabolites in cystic fibrosis. Addressing this issue is crucial in effectively and safely correcting vitamin D deficiency in CF.

Clinically stable CF patients with a history of pancreatic insufficiency (n=6) and matching non-CF subjects (n=6) will be recruited in this study. All subjects will be pre-screened for 25(OH)D status to include those with 25(OH)D levels below 30 ng/mL. The subjects will receive a single oral dose (300,000 - 600,000 IU) of vitamin D3, and the dose will be based on study participant's baseline 25-hydroxyvitamin D3 level. For CF patients, the dose will be administered with food and pancreatic enzyme supplement. This dose was chosen as previous studies in pediatric CF patients demonstrated that a large single dose of up to 600,000 IU vitamin D3 raised and maintained sufficient 25(OH)D concentrations without any signs of adverse events.

Eligibility Criteria:
Inclusion Criteria:

* For CF, diagnosis of CF based on positive sweat chloride or known CF mutation
* For CF, Patients with pancreatic insufficiency
* Age ≥ 18 years
* Serum 25(OH)D concentrations below 30 ng/mL (75 nmol/L)

Exclusion Criteria:

* Pregnancy
* History of lung transplant,
* Severe anemia (hemoglobin concentration \< 7 g/dL),
* Liver disease (AST/ALT \> 3x ULN), kidney disease (GFR ≤ 40 mL/min), or granulomatous conditions
* Patients taking steroids, cholesterol-lowering drug (cholestyramine), weight-loss drugs (orlistat) , statins, anti-tuberculosis drugs (rifampin and isoniazid), phenobarbital, phenytoin, carbamazepine, immunosuppressants (cyclosporine, tacrolimus)
Prospective Study to Observe and Manage Progression of Pulmonary Nodules

Based on the previous review and research, we would like to further observe the nodule changes in patients with pulmonary nodules. These changes include factors such as the reason for the further enlargement or deterioration of the nodule, time, lifestyle, the patient's region, and hereditary medical history. At the same time, we will further look at the corresponding characteristics of a population that does not change the size and nature of the nodule, including age, gender, and all the factors discussed above. On this basis, we can propose more scientific methods for the treatment and management of pulmonary nodules, and provide a more reliable clinical reference for the clinical treatment of pulmonary nodules.


Eligibility Criteria:
Inclusion Criteria:

LNs patients aged 18 years and older who were previously treated in Guang'anmen hospital.

Exclusion Criteria:

(1) had the LNs removed; (2) LNs less than or equal to 3 mm in size; (3) had not undergone thorax CT; (4) had failed to complete treatment; (5) had incomplete medical records or was lost to follow-up.
INDEPENDENCE Spacer for Lumbar Fusion

The primary objective of this study is to evaluate radiographic outcomes, intra-operative parameters, clinical outcomes, and patient satisfaction following circumferential fusion using the INDEPENDENCE® integrated plate and spacer with REVOLVE® Stabilization System for the treatment of severe low back pain due to lumbar disc degeneration unresponsive to 6 months of non-operative treatment.

A total of thirty subjects will be enrolled at Scott \& White Healthcare. The INDEPENDENCE spacer will be filled with autogenous bone graft material. The devices subject to this Agreement have received 510(k) clearance from the FDA for the indications specified in this Agreement.

Patients will be followed for 2 years with evaluations at the following time intervals: preoperatively (within 2 months of surgery) and postoperatively at 6 weeks, 3 months, 6 months, 12 months, and 24 months.

Eligibility Criteria:
Inclusion Criteria:

* • Objective evidence of degenerative disc disease at 1 or 2 level(s) between L2 and S1 with degeneration confirmed by patient history and radiographic examination

  * Experience pain, functional deficit and/or neurological deficit for a minimum of six months
  * Unresponsiveness to documented non-surgical treatment modalities for a minimum of six months and/or presentation with progressive symptoms of nerve root or spinal cord compression prior to enrollment
  * At least 18 years of age and maximum 65 years of age
  * Ability to provide Informed Consent for study participation and to return for all follow-up visits

Exclusion Criteria:

* • Presence of systemic or localized infection at the site of surgery

  * More than 2 levels to be instrumented
  * Previous fusion attempt at the involved level(s)
  * More than three previous open, posterior, lumbar spinal surgical procedures at the involved level(s)
  * Trauma at the level(s) to be fused
  * More than grade 1 spondylolisthesis
  * Previous documentation of osteopenia, osteoporosis, or osteomalacia to a degree that spinal instrumentation would be contraindicated
  * Diagnosis of a condition or requires postoperative medication(s), which may interfere with bony/soft tissue healing
  * Presence of a disease entity or condition which totally precludes possibility of bony fusion (e.g. metastatic cancer, HIV, long term use of steroids, etc.)
  * Immunosuppressive disorder
  * Pregnant or interested in becoming pregnant during the duration of the study
  * History of substance abuse (drugs or alcohol)
  * Any known allergy to a metal alloy
  * Mentally incompetent or prisoner
  * Currently a participant in another study
InterStim Basic Evaluation Lead Post-Market Clinical Follow-up Study

Post-market clinical follow-up for continued assessment of safety and performance of the InterStim basic evaluation lead and foramen needle(s) used during a therapy evaluation.


Eligibility Criteria:
Inclusion Criteria:

1. Subjects 18 years of age or older
2. Candidate for sacral neuromodulation in accordance with the InterStim System labeling
3. Have a diagnosis of overactive bladder (OAB) as demonstrated by either urinary urge incontinence and/or urinary frequency on a 3-day voiding diary
4. Willing and able to accurately complete study diaries, questionnaire, attend visits, and comply with the study protocol
5. Willing and able to provide signed and dated informed consent

Exclusion Criteria:

1. Have neurological conditions such as multiple sclerosis, clinically significant peripheral neuropathy or spinal cord injury (e.g., paraplegia)
2. Have implantable pacemakers, or defibrillators
3. Have primary stress incontinence or mixed incontinence where the stress component overrides the urge component
4. Have knowledge of planned MRIs, diathermy, microwave exposure, high output ultrasonic exposure, or radio frequency (RF) energy exposure not included within the scanning conditions provided with the InterStim System labeling
5. Women who are pregnant or planning to become pregnant during participation in the study
6. Characteristics indicating a poor understanding of the study or characteristics that indicate the subject may have poor compliance with the study protocol requirements
7. Concurrent participation in another clinical study that may add additional safety risks and/or confound study results.
Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)

Long-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab (Soliris®) and ravulizumab (Ultomiris®).

At the time of enrollment in the Registry, participant records will be queried for retrospective information about the participants' medical history and gMG disease treatment history. Following enrollment, prospective data collection will be performed using data obtained as part of the routine clinical care and through patient-reported outcome methods in use. Data will be collected using an electronic data capture system. The duration of data collection for the Registry will be up to 5 years from the day of enrollment.

Eligibility Criteria:
Inclusion Criteria:

1. Participants with a diagnosis of gMG who are treated with Alexion C5IT at the time of enrollment, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.
2. Capable of giving signed informed consent, which includes compliance with the protocol requirements and restrictions.
3. Participants must have myasthenia gravis historical data, such as MG-ADL and MGFA class, available to be enrolled in the Registry.

Exclusion Criteria:

1. Participants currently enrolled in an Alexion-sponsored interventional clinical study for treatment of gMG cannot be enrolled in the Alexion gMG Registry while enrolled/participating in the clinical study for gMG therapy.
The Effect of Oily Fish and Poultry on Children's Cardiometabolic Health and Cognitive Function

The purpose of FiSK Junior is to investigate whether intake of oily fish compared to poultry affects cardiovascular risk markers, cognitive function, and behaviour in healthy children.

The FiSK Junior study compares the effects of consuming oily fish versus poultry (control) in healthy 8-9 year-old Danish children. The children will be randomized to consume approximately 300 g/week of either oily fish or poultry for 12 weeks. Measurements and biological sampling will be performed at baseline and at the end of the intervention.

The main objective of the study is to investigate the effect on cardiovascular risk markers, primarily blood pressure and plasma triacylglycerol. Moreover, the effects on cognitive function, behaviour, emotions and physiological stress response will be assessed. In addition, change in n-3 long chain polyunsaturated fatty acid status (compliance), anthropometry, body composition, vitamin D status, inflammatory markers, growth markers, physical activity and diet will be measured. Outcomes will be analysed with focus on potential gender- and genotype-specific effects.

Eligibility Criteria:
Inclusion Criteria:

Children must:

* Be 8 - 9 years of age at the start of the intervention
* Be healthy
* Like oily fish and chicken
* Be willing to be randomized to eat fish or chicken
* Not consume oily fish more than once per week 3 months prior to intervention start
* Not consume fish oil 3 months prior to intervention start
* Speak Danish in order to understand the study procedures

Moreover:

* Parents must read and speak Danish, in order to be properly informed about the study procedures
* The child must live in a household with ≤5 persons

Exclusion Criteria:

* Serious chronic illnesses and diseases that may interfere with study outcomes
* Diagnosed Attention Deficit Hyperactivity Disorder (ADHD) or other psychiatric illness
* Intake of medication that may affect study outcomes
* Concomitant participation in other studies involving dietary supplements or blood sampling
* Living in a household with another participating child