cyrilzakka/clinical-trials huggingface dataset:
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).


Eligibility Criteria:
Key Inclusion Criteria:

* Diagnosis of severe SCD as defined by:

  * Documented SCD genotypes
  * History of at least two severe VOCs events per year for the previous two years prior to enrollment
* Hydroxyurea (HU) failure unless HU intolerant
* Eligible for autologous stem cell transplant as per investigators judgment

Key Exclusion Criteria:

* A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor
* Prior hematopoietic stem cell transplant (HSCT).
* Clinically significant and active bacterial, viral, fungal, or parasitic infection

Other protocol defined Inclusion/Exclusion criteria may apply.
A Study of the Safety and Tolerance of Regadenoson in Subjects With Asthma or Chronic Obstructive Pulmonary Disease

This study is intended to determine the safety and tolerance of regadenoson in subjects with asthma or chronic obstructive pulmonary disease.


Eligibility Criteria:
Inclusion Criteria:

* Subject has asthma or stable chronic obstructive pulmonary disease (COPD).
* Subject has a diagnosis of coronary artery disease (CAD) or risk factors for CAD as determined by a current medical diagnosis of at least 2 of the following conditions: Type 2 diabetes, hypertension, hypercholesterolemia, current or history of cigarette smoking (minimum 10 pack-years exposure) or obesity Body Mass Index (BMI \> 30).
* Subject must abstain from smoking 3 hours prior and 8 hours post study drug administration.
* Subject must abstain from any intake of foods and beverages containing a methylated xanthine derivative (i.e. caffeine, theobromine, or methylxanthine) within 12 hours prior to study drug administration through the Follow-Up visit, as these foods may reduce the effects of regadenoson.
* Subject is able to safely abstain from theophylline for 12 hours prior to the Day 1 visit, as determined by the Investigator
* Asthma subject's frequency and severity of symptoms have remained unchanged within 30 days prior to study drug administration
* Asthma subject has FEV1 ≥60% predicted
* COPD subject has FEV1/FVC \< 0.70

Exclusion Criteria:

* Female subject who is pregnant, lactating or of childbearing potential who refuses to use a medically acceptable form of contraception until the Follow-Up visit is complete.
* Subject started on a course of corticosteroids, steroid combination with long-acting Beta2-agonist (LABA) (oral or inhaled) or anticholinergic, or has undergone a change in dose of such medications ≤ 30 days prior to study drug administration (subject on a stable dose of such medications for \> 30 days prior to study drug administration is allowed).
* Subject started leukotriene antagonists (e.g., montelukast), cromones (e.g., cromolyn sodium) or 5-lipoxygenase antagonists (e.g. zileuton or zyflo) or has undergone a change in dose of medications in these drug classes ≤ 7 days prior to study drug administration (subject on a stable dose of these medications for \> 7 days prior to study drug administration is allowed).
* Subject has a history of second or third degree heart block or sinus node dysfunction unless the subject has a functioning pacemaker.
* Subject has symptomatic hypotension (temporary and reversible conditions that no longer exist are allowed).
* Subject is allergic or intolerant to aminophylline.
* Subject has had a respiratory infection within 2 weeks prior to randomization.
* Subject has had surgery within 3 months prior to randomization.
Real-world Treatment Patterns of Endocrine Based Therapy Among Patients With Hormone Receptor-positive/Human Epidermal Growth Factor Receptor-2-negative (HR+/HER2-) Advanced Breast Cancer: An Analysis of Administrative Claims Data in Japan

This is a retrospective observational study focusing on patients diagnosed with advanced breast cancer(ABC) in Japan using de-identified claim data from Medical Data Vision (MDV) database.

The primary objective of this study is to describe patient demographics, treatment patterns and treatment duration of palbociclib, and subsequent treatment patterns and treatment duration after palbociclib-based therapy among ABC patients in Japan The secondary objective of the study is to describe patient demographics, treatment patterns of ABC patients and treatment duration of endocrine therapy, and subsequent treatment patterns and treatment duration after endocrine therapy among ABC patients in Japan.


Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of breast cancer based on International statistical classification of diseases and related health problems 10th revision (International Statistical Classification of Diseases and Related Health Problems \[ICD-10\]) (C50.xx)
* Received at least one prescription of endocrine therapy drugs
* Diagnosis of secondary malignant neoplasm based on ICD-10 (C77.x, C78.x, C79.x )

Exclusion Criteria:

-Received a prescription of anti-HER2 therapy (Trastuzumab, Trastuzumab emtansine, Pertuzumab, and Lapatinib tosilate hydrate)
A Study of HRS-7535 Compared With Placebo in Adult Participants With Type 2 Diabetes and Inadequately Controlled With Diet and Exercise

The aim of this trial is to evaluate the efficacy and safety of HRS-7535 compared with placebo in subjects with type 2 diabetes mellitus with inadequate glycemic control with diet and exercise


Eligibility Criteria:
Inclusion Criteria:

1. Male or female, 18-75 age years, both inclusive;
2. Type 2 diabetes mellitus diagnosed for at least 2 months before the screening visit;
3. HbA1c 7.0-10.0% (both inclusive) at screening;
4. Treated with diet and exercise, and without any antidiabetic drugs at least 8 weeks prior to screening.

Exclusion Criteria:

1. Known or suspected allergy to the investigational drug or its components or excipients.
2. Diagnosed or suspected with type 1 diabetes mellitus, special types of diabetes or secondary diabetes.
3. Have a history of acute complications of diabetes (diabetic ketoacidosis, lactic acidosis, hyperglycaemic hyperosmolar state, etc.) within 6 months prior to screening.
4. Proliferative retinopathy, maculopathy, painful diabetic neuropathy, diabetic foot ulcer or intermittent claudication requiring acute treatment;
5. Pregnancy, breast-feeding, intention of becoming pregnant during the trial; or women of childbearing potential (WOCBP) or male subject not using adequate contraceptive measures.
SPL026 (DMT Fumarate) in Healthy Subjects and MDD Patients

SPL026 (N,N-dimethyltryptamine \[DMT\] fumarate) is a psychedelic tryptamine being developed as a therapy for patients with major depressive disorder (MDD).

2-part study. Part A in psychedelic-naïve healthy volunteers. Part B in patients with MDD who score moderate-severe on Ham-D.

Healthy volunteers will receive a single dose of SPL026 in a dose-escalation parallel group study.

Patients will receive up to 2 single doses of SPL026, 2 weeks apart. Dose 1 will be randomised double-blind with placebo. Dose 2 will be open label, active SPL026.

SPL026 will be administered by IV injection. Safety and tolerability, PK, PD and efficacy will be measured.

Eligibility Criteria:
Inclusion Criteria:

Normotensive male or female, deemed healthy on the basis of a clinical history, physical examination, ECG, vital signs, laboratory tests of blood and urine, Mini-International Neuropsychiatric Interview (MINI) and Beck Scale for Suicidal Ideation (BSS); willing to follow the contraception requirements of the trial; willing to refrain from psychedelic drug use (excluding the study drug) during the trial and ≥ 3 months afterwards; willing to be contacted by email and video call, and have online access; able to give fully informed written consent. Part A only: psychedelic-naïve, ie have never taken a serotonergic psychedelic drug, in any form. Must be 25 years or older. Part B only: MDD diagnosis (as per DSM-V); not on antidepressant medication or willing to discontinue antidepressant medication (eg selective serotonin reuptake inhibitor \[SSRI\] treatment) for a sufficient time before and during the study; no psychedelic drug use in the 6 months before dosing.

Exclusion Criteria:

Pre-menopausal females who are pregnant or lactating, or who are sexually active and not using a reliable method of contraception; clinically relevant abnormal findings at the screening assessment; acute or chronic illness (other than MDD \[Part B only\]) or infection; clinically relevant abnormal medical history or concurrent medical condition (other than MDD \[Part B only\]); positive tests for hepatitis B \& C, or HIV; severe adverse reaction to any drug; use of over-the-counter or prescribed medication (excluding oral contraceptives) within previous 28 days (paracetamol \[acetaminophen\] permitted up to 7 days, and antidepressant medication must have ceased for at least 14 days; 28 days for MOAIs) before first dose of trial medication; drug or alcohol abuse; use of cannabis in the 24 h before each study visit; heavy smokers (\> 10 \[Part A\] or \> 20 cigarettes \[Part B\] daily); supine blood pressure, heart rate, or QTcF outside the acceptable ranges; participation in other clinical trials of unlicensed medicines, or loss of more than 400 mL blood, within the previous 3 months; phobia of needles or blood; possibility that volunteer will not cooperate with the study.
Impact of Sarpogrelate in the Function of Endothelial Progenitor Cells

The purpose of this study is to evaluate the effects of Sarpogrelate in endothelial progenitor cells of patients with atherosclerosis.

Endothelial progenitor cells play an important role in vasculogenesis and vascular repair. Sarpogrelate, a inhibitor of HT2A, is used an an anti-platelet drug, however, a direct effect in vascular cells is supposed.

Eligibility Criteria:
Inclusion Criteria:

* Patients with atherosclerosis

Exclusion Criteria:

* Patients with high risk of bleeding
* Patients with pregnancy
* Patients with allergic reaction of Salpogrelate
Pain Management in Corneal Collagen Crosslinking for Keratoconus

This is a prospective, randomized, single blind study. Patients enrolled in the study were randomly assigned to a ketorolac or a gabapentin group for pain control. Different pain scores and variables will be evaluated, along with others side effects variables.

This is a prospective, randomized, single blind study. Patients enrolled in the study were randomly assigned to the ketorolac or gabapentin group. The gabapentin (Neurontin, Pfizer, Mexico) group was instructed to take one 300-mg capsule the night before surgery and 1 capsule every 8 hours on the day of surgery and for the first 3 postoperative days. The ketorolac group (Dolac, Siegfried Rhein, Mexico) was instructed to take their 10-mg tablets every 8 hours on the day of surgery and for the first 3 postoperative days. Different pain scores and variables will be evaluated, along with others side effects variables.

Eligibility Criteria:
Inclusion Criteria:

* Patients with keratoconus older than 18 years with documented bilateral keratometric progression in the previous 6 to 12 months.

Exclusion Criteria:

* Previous ocular or systemic diseases that could affect epithelial healing; use of topical or systemic non-steroidal anti-inflammatory drugs, or chronic use of any other pain medication, pregnancy and allergy to the drugs of the study.
Side Effects of Anti-PD-(L)-1 and Anti CTLA-A4 in the Non Small Cells Lung Cancer

The immune-related adverse events (irAEs) linked to the important activation of the immune system by new immunotherapy treatments in patients affected by Non-small-cell lung carcinoma (NSCLC) have not received a lot of systematic study or been monitored over time outside of clinical trials.

This study aims to verify, on the basis of the data collected in a prospective and retrospective manner, that the side-effects due of anti-PD-(L)-1 or anti-CTLA-4 treatments observed in the target population of the controlled clinical trials are the same as in the general clinical population.


Eligibility Criteria:
Inclusion Criteria:

* Any patient with NSCLC treated in Cliniques Universitaires Saint-Luc with immunotherapy in first or second line.

Exclusion Criteria:

* The refusal to sign the consent.
Pilot and Feasibility Study of the Imaging Potential of EC17: Intraoperative Folate-fluorescein Conjugate (EC17) Lung Cancer (CA)

The goal of this study is to perform imaging of patient's tumors while the chest is open and the tumor is being removed.

According to the World Health Organization, lung cancer is the most common cause of cancer-related death in men and women, and is responsible for 1.3 million deaths worldwide annually as of 2004. Surgery remains the best option for patients presenting with operable Stage I or II cancers, however the five year survival rate for these candidates remains at a dismal 53% for Stage I and 32% for Stage II. The high rates of recurrence suggest that surgeons are unable to completely detect and remove primary tumor nodules in a satisfactory manner as well as lingering metastases in sentinel lymph nodes. By ensuring a negative margin through imaging during surgery it would be possible for us to improve the rates of recurrence free patients and thus overall survival.

Thoracic malignancies are the ideal disease to investigate intra-operative imaging. Over 85% of lung and pleural malignancies express folate receptor alpha (FRA). It is important to note that FRA is expressed only in the proximal tubules of the kidneys, activated macrophages, and in the choroidal plexus. Thus, the false positive detection rate is expected to be extremely low. A group well known to us in the Netherlands has completed a pilot study utilizing a folate-fluorescein isothiocyanate (folate-FITC) conjugate in 12 patients with ovarian cancer. Another group of investigators in Mayo have subsequently performed this study on 20 more patients without any serious adverse events (personal communication). They report excellent sensitivity and specificity with this technique with only grade 1 side effects (allergic reaction). All side effects reversed when the injection was halted. Patients with a history of allergic reactions to insect bites should not participate (fluorescein is derived from the firefly insect, folate is an essential vitamin).

This will identify the margins and the lymph nodes that may have cancer cells during cancer surgery

Eligibility Criteria:
Inclusion Criteria:

* Adult patients over 18 years of age
* Patients presenting with a lung or pleural nodule or mass presumed to be resectable on pre-operative assessment
* Good operative candidate
* Subject capable of giving informed consent and participating in the process of consent

Exclusion Criteria:

* Pregnant women as determined by urinary or serum beta human chorionic gonadotropin (hCG) within 72 hours of surgery
* Patients with a history of anaphylactic reactions to Folate-FITC or insects
* At-risk patient populations:

  * Homeless patients
  * Patients with drug or alcohol dependence
  * Children and neonates
  * Patients unable to participate in the consent process
SMS Reminders to Strengthen Demand for HPV Vaccination in Georgia

It is possible that achieving adequate HPV vaccination coverage will allow cervical cancer to be virtually eliminated. This is a priority for Georgia, where the burden of mortality and morbidity from cervical cancer is high and the yearly prevalence of cervical cancer is 14.5 per 100,000 population.

The objective of the intervention evaluated is to increase the uptake of the 1st dose of the HPV vaccination in Georgia. The intervention aims to achieve this by providing a SMS reminder informed by behavioural insights (BI) to remind caregivers of girls between 10-12 years that their daughter is due the first dose of her HPV vaccination, share information about the vaccine, and encourage them to book an appointment to receive the vaccination. Four different SMS messages will be compared to no SMS message.

The evaluation will be a 5-arm randomised controlled trial (RCT) which aims to answer the research question: Does each of the designed BI-informed SMS reminders increase the uptake of the first dose of the HPV vaccination among eligible girls in Georgia, compared to no reminder? Our hypothesis is that SMS reminders will increase the uptake of HPV vaccination.


Eligibility Criteria:
Inclusion Criteria:

* Girls
* Aged 10-12 years
* Not yet received any doses of the HPV vaccine
* With a caregiver's mobile phone number in the e-health system.

Exclusion Criteria:

* Boys
* Girls aged less than 10 or more than 12 years
* Girls with no caregiver phone number in the e-health system
* Girls who have received 1 or 2 doses of the HPV vaccine
The Efficacy of Biplane Versus Single Plane Ultrasound in Facilitating Caudal Epidural Anesthesia in Pediatric Patients.

Ultrasound guidance plays a pivotal role in caudal epidural block in pediatric patients. In comparison to conventional single-plane ultrasound, biplane ultrasound offers the advantage of providing simultaneous transverse and longitudinal imaging. Better visualization under biplane ultrasound is postulated to enhance the efficacy of caudal epidural blocks. This improvement is expected to manifest through higher first puncture success rate, shorter procedure time, and longer time to first postoperative analgesics use.

Our study aims to recruit pediatric patients receiving caudal epidural blocks as a complement to general anesthesia and for postoperative analgesia. Through a randomized allocation, we will administer the caudal injection with either a biplane ultrasound-guided approach or the single-plane ultrasound-guided technique. The assessment will encompass accuracy metrics, such as the first puncture success rate and number of needle redirections, and the efficiency indicators, including the time from initial probe placement on the skin to successful injection, the time to first postoperative analgesics use, and PACU pain score. Safety evaluation will be conducted, encompassing adverse events from the commencement of the caudal block procedure until discharge. We will also conduct post-discharge follow-up phone calls to evaluate patients' experience after discharge.

Eligibility Criteria:
Inclusion Criteria:

* ASA I/II
* Male patients aged 4 months to 10 years
* To undergo elective circumcision with caudal epidural anesthesia as an adjuvant to general anesthesia and for postoperative analgesia

Exclusion Criteria:

* Anatomical anomalies (e.g., tethered cord, sacral malformations, etc.).
* Potential coagulopathy.
* Preoperative analgesics use.
* Allergy to local anesthetics.
* Rash or infection at the injection site.
* Female patients
* Parents refuse to participate.
The Clinical and Subjective Performance of Three Different MPDS With Silicon Hydrogel Lenses

The purpose of the clinical investigation is to assess the clinical and subjective performance of three novel multipurpose disinfecting solutions (MPDS) in daily wear with two silicone hydrogel contact lenses (Lotrafilcon B and Comfilcon A).


Eligibility Criteria:
Inclusion Criteria:

* age above 18
* experienced contact lens user
* good health (self-reported)

Exclusion Criteria:

* eye disease or eye operations
* pregnancy
* medication that can affect sight
Diabetic Foot Ulcer Research Study

The goal of this clinical trial is to compare safety and performance of the study device to the current typical NHS standards of care device. The main outcomes it aims to achieve are:

Complete wound healing at 12 weeks (100% epithelialised)

% Reduction in wound area at 12 weeks

Participants will attend their NHS podiatry clinic appointment as normal and will be assigned either the study device or the current typical NHS standard of care device. Researchers will compare the study device group to the standard group to assess the outcomes mentioned previously.

The purpose of this clinical trial is to evaluate the safety and performance of the RTD Wound Dressing in the treatment of non-infected neuropathic, ischaemic, and neuro-ischaemic diabetic foot ulcers versus standard of care.

This clinical evaluation study will be performed under an open-label, comparative, design in the single health board of NHS Lanarkshire within 3 diabetic foot clinics.

Eligibility Criteria:
Inclusion Criteria:

* Male or female, aged ≥18 years old with a diagnosis of type 1 or type 2 diabetes.
* New presentation for treatment of a neuropathic, ischaemic or neuro- ischaemic non- infected DFU
* Subjects must be able to follow verbal and written instructions in English
* Subjects must have full mental capacity and able to give written informed consent to participation in the trial including medical photography for the purposes of the clinical trial or clinical care.
* DFU is \>10mm2 and \<100mm2

Exclusion Criteria:

* Known allergy/hypersensitivity to any ingredient contained in the Keneric healthcare RTD Wound Dressing
* Participants who will have problems following the protocol, including the offloading system, provided as standard of care, to wear on a daily basis
* Suspected malignancy in the wound
* Critical limb ischaemia
* Pregnant or breastfeeding females
* Women of childbearing potential and not using a medically accepted form of contraception when sexually active.