cyrilzakka/clinical-trials huggingface dataset:
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (192 weeks).


Eligibility Criteria:
Inclusion Criteria:

* Age 4 to 16 years inclusive, at the time of informed consent/assent.
* Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
* Upper extremity muscle group that is amenable to muscle biopsy.
* Brooke Upper Extremity Scale score of 1 or 2.
* Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for \<2 years before enrollment.
* Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration, with the expectation of maintaining a stable dose during the Placebo-Controlled and Open-Label Periods of the study (unless dose adjustment is required by weight change).
* Left ventricular ejection fraction of ≥50% by echocardiogram or ≥55% by cardiac magnetic resonance imaging (MRI).

Exclusion Criteria:

* Uncontrolled clinical symptoms and signs of congestive heart failure (CHF).
* Any change in prophylaxis/treatment for CHF within 3 months prior to the start of study treatment.
* History of major surgical procedure within 12 weeks prior to the start of study drug administration or an expectation of a major surgical procedure during the study.
* Requirement of daytime ventilator assistance.
* Percent predicted FVC \<40 % (applies only for participants who are age ≥7 years).
* Receipt of eteplirsen, or alternative exon-skipping/dystrophin-modifying therapy, within 12 weeks of randomization.
* Receipt of non-exon skipping investigational drug within 4 months before the start of study drug administration.
* Receipt of gene therapy at any time.

Other inclusion and exclusion criteria may apply.
Can Drospirenone be Used to Prevent LH Surge in Controlled Ovarian Stimulation in PCOS?!

The goal of this clinical trial is to learn if drospirenone can work to prevent LH surge in controlled ovarian stimulation in PCOS cases undergoing ART cycles. The main questions it aims to answer are:

Can drospirenone prevent LH surge in controlled ovarian stimulation in PCOS cases ? Can it prevent the occurance of ovarian hyperstimulation?

Researchers will compare drospirenone to cetrorelix (A well known drug for such cases) to see if drospirenone works the same way.

Participants will:

Take drospirenone or cetrorelix from stimulation day 5 ( 5 days of ovarian stimulation) till day of hCG trigger

- monitoring will be done before the cycle and through the cycles with vaginal ultrasound assessment and lab testing

The goal of this novel clinical trial is to study if drospirenone can work to prevent LH surge in controlled ovarian stimulation in PCOS cases undergoing ART cycles in comparison to a gold standard drug cetrorelix:

All PCOS cases: 50 case in this novel trial. 25 in each group will be randomized after taking 5 days of ovarian stimulation to either drospirenone tablet/d or cetrorelix injection/d till the day of hCG.

At the day of hCG, lab testing will be done in addition to ultrasound assessment by transvaginal probe. the tests are: progesterone, E2, LH.

Assesment will be done for the number quality of follicles and the number quality of embryos developed, development of OHSS, vitrification/thawing of embryos after 1 month then the clinical and chemical pregnancy rate.

Eligibility Criteria:
Inclusion Criteria:

* PCOS was diagnosed according to the Rotterdam consensus criteria (two out of three of the following criteria):

  1. oligo- or anovulation
  2. clinical and/or biochemical signs of hyperandrogenism
  3. polycystic ovaries).
* A diagnosis of congenital adrenal hyperplasia, Cushing's syndrome, androgen-producing tumours, hyperprolactinemia and thyroid dysfunction were all rulled out.

Exclusion Criteria:

* age 38 years
* basal FSH level 12 mIU/mL
* previous ovarian surgery
* congenital uterine anomaly anomaly, intrauterine adhesion and male partner with non-obstructive azoospermia.
Efficacy of Prophylactic Treatment of Oral Prochlorperazine for Acute Mountain Sickness

A field-based trial was conducted to determine if oral prochlorperazine demonstrates efficacy in the prophylactic treatment of AMS, and/or decreases the incidence of the symptoms of acute mountain sickness including headache, GI symptoms, fatigue and dizziness based on data collected in the Lake Louise AMS score.

This was a prospective, single-blinded, field-based interventional trial involving consenting, healthy subjects aged \>17 years. The study was conducted on the big Island of Hawaii, at sea level and at the summit of Mauna Kea volcano. Following randomization, subjects received either placebo or 10mg prochlorperazine, then immediately drove for 2 hours from sea level to the volcano summit (4205m). Participants spent 240 min at the summit, and they recorded their symptoms. AMS was defined by the previously validated Lake Louise Acute Mountain Sickness Score (LLAMS). Categorical variables were analyzed by chi-square; continuous variables analyzed by t-tests.

Eligibility Criteria:
Inclusion Criteria - Healthy subjects aged \>17 years

Exclusion criteria-subject meets any of the following:

* Known allergy or contraindication to the study drug.
* Known or suspected pregnancy.
* Use of another analgesic or antiemetic within 8 hours of enrollment.
* History of chronic headaches.
* Inability to provide informed consent.
* Use of central nervous system depressants, including alcohol, opiates, and/or barbiturates, within 24 hours of the study start time.
* History of severe depression.
* History of dementia.
* Prior ascent and stay at 2500m for duration over 6 weeks within 30 days prior.
Observational Study on Patients With Hepatobiliary Tumors

Hepatobiliary tumors have a poor prognosis and high individual heterogeneity, the patient with hepatobiliary tumors even accepted radical surgery, the postoperative recurrence rate is still high. Therefore, it is of great significance to find important prognostic markers to improve patient prognosis and formulate new treatment plans. In recent years, targeted therapy and immunotherapy make cancer treatment enter a new field, However, tumor heterogeneity is the greatest challenge in cancer therapeutics and biomarkers discovery. In this study, we collected a wide rang of patients' information, including photos of patients' face, physical strength and nutrition indicators, blood ,stool and pathological tissue specimens from tumor patients, then Multi-omics testing were applied to Looking for novel therapeutic targets and prognostic markers to predict patient response to treatment. Clinicians choose the best treatment plan for the patient based on the test results to improve the patient's survival time and quality of life.

This is an ongoing, longitudinal, observational clinical cohort study of patients with tumors of the alimentary tract, mainly hepatobiliary tumors. All the patients are diagnosed, treated and followed up. In the future, some other patient information will be gradually updated to this database

Eligibility Criteria:
Inclusion Criteria:

1. Be older than 18 years old;
2. Clinical or pathological diagnosis of malignant hepatobiliary tumors;
3. Patients have received or are about to receive surgery, chemotherapy radiotherapy, targeted therapy, local therapy, immunotherapy, etc;
4. Patients understand and are willing to sign written informed consent.

Exclusion Criteria:

1. Patients considered by the doctor to be unsuitable for entry into this study (mental disorder or poor compliance, etc.);
2. The patient or guardian is unwilling to participate in this study;
3. Pregnant women;
4. Combined with other serious systemic diseases (heart failure, renal failure,hematopoietic system disease or Uncontrollable acute infection);
5. Estimated survival time is less than 1 month.
Study of a Bupivacaine Patch (Eladur™) to Treat Post- Herpetic Neuralgia

Neuropathic pain is caused by a virus commonly associated with chicken pox. This virus may become dormant in the nervous system and later reactivate causing herpes zoster, also known as "shingles". Post-herpetic neuralgia (PHN) is a persistent pain in the area of healed skin lesions. This study will test the safety and efficacy of treating PHN patients with the analgesic patch, Bupivacaine TTS (Eladur™).


Eligibility Criteria:
Inclusion Criteria:

* Male or females age 21 years or older.
* Pain in thoracic or lumbar regions for a minimum of 90 days after crusting of Herpes Zoster (HZ) lesions.
* Stable prescribed medications regimen (including opioids, anticonvulsants, and tricyclic antidepressants).
* Intact, unbroken skin over the painful area to be treated.
* Body Mass Index (BMI) no more than 35 kg/m2.

Exclusion Criteria:

* Active HZ lesions, dermatitis, Central Nervous System (CNS) injury.
* Any immunosuppressed condition, including but not limited to AIDS/HIV and Hodgkin's lymphoma.
* Pain control by nerve block or neurosurgical intervention.
* Evidence of clinically significant hepatic, gastrointestinal, renal, hematologic, urologic, neurologic, respiratory, endocrine or cardiovascular system abnormalities, psychiatric disorders, or acute infection.
* Connective tissue disorders (systemic lupus erythematosus, scleroderma, mixed connective tissue disease).
* Recent use (within 30 days preceding the first treatment visit) of any topically applied pain medication, such as nonsteroidal anti-inflammatory drugs, menthol, methyl salicylate, local anesthetics (including Lidoderm®), steroids or capsaicin products on the painful areas.
* Significant pain of an etiology other than PHN, for example, compression-related neuropathies (e.g., spinal stenosis), fibromyalgia or arthritis. Patients must not have significant ongoing pain from other cause(s) that may interfere with judging PHN related pain.
* Participation in a clinical trial of an investigational product or device within 30 days of the screening visit or concurrently during the conduct of this study.
Dose-finding Study Comparing Efficacy and Safety of a PARP Inhibitor Against Doxil in BRCA+ve Advanced Ovarian Cancer

The purpose of the study is to compare the efficacy and safety of 2 doses of drug AZD2281 against liposomal doxorubicin to see which is effective and well tolerated in treating patients with measurable BRCA1- or BRCA2-positive advanced ovarian cancer and who have failed previous platinum therapy.


Eligibility Criteria:
Inclusion Criteria:

* Advanced ovarian cancer with positive BRCA1 or BRCA2 status
* Progressive or recurrent disease after platinum-based chemotherapy
* Measurable disease by RECIST

Exclusion Criteria:

* Previous anthracycline treatment
* Brain metastases
* Less than 28 days since last treatment used to treat the disease
* Considered a poor medical risk due to a serious uncontrolled disorder
Physical Status and Upper Airway Function in Neuromuscular Patients

Inside of studies that report an upper airway function impaired, the upper limb strength has been demonstrated to be determinant in breath, speech and swallow performance. Nevertheless, no previous studies in neuromuscular disease have explored the relation between general disability and upper airway function.

There is a suspicion of upper airway which is associated with physical deterioration, however the extent to which if feeds back has not been explored.


Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of Neuromuscular disease
* Between 6 and 65 years
* Impairment of the upper airway
* Accepted to sign the informed consent

Exclusion Criteria:

* Cognitive impairment.
* Diagnose of autism spectrum disorder or severe intellectual disability
* Physical or functional impairment that limits the performance of evaluation
Color Doppler Analysıs Of Uterin And Intraovarian Blood Flow Before And After Treatment Wıth Cabergoline In Hyperprolactinemic Patients

the investigators examined the hypothesis that hyperprolactinemia may alter uterin, endometrial and intraovarian blood flow and may contribute to increased infertility by another way.


Eligibility Criteria:
Inclusion Criteria:

* Women who had blood prolactin level higher than 25ng/dl

Exclusion Criteria:

* Pregnancy
* Use of drugs effecting vasculature such as hormonal therapy, oral contraceptives, antihypertensives
* Diseases like pelvic inflammatory disease and systemic hypertension
* Women with clinically overt or treated cardiovascular disease
* Concurrent illness other than hyperprolactinemia
Transcutaneous Mechanical Nerve Stimulation (TMNS) by Vibration in the Treatment of Incontinence

During transcutaneous mechanical nerve stimulation in spinal cord injured men an increase in pressure was observed in the external urethral sphincter along with an increase in bladder capacity. In a subsequent study it was demonstrated that TMNS in women could induce pressure increment of the external urethral sphincter. A pilot study have since shown that after 6 weeks of stimulation 24 out of 33 women suffering from urinary stress incontinence were able to contract their pelvic floor muscles and had become free of symptoms. Another pilot study has shown promising effect on the overactive bladder syndrome.

The present study aims to treat urinary incontinence and includes 3 groups of patients with 40 patients in each group: Women suffering from urinary stress incontinence, women suffering from urge incontinence and men suffering from urinary incontinence after a prostatectomy. A medical vibrator is used and in each group the subjects will be randomized to active treatment or placebo treatment.

In women the stimulation will be performed at the perineum every day for 6 weeks with an amplitude of 2 mm and a frequency of 100 Hz. In men the vibration parameter/location will be determined by the results of a pressure profile study also included. Results will be evaluated on the basis of questionnaires, micturition diaries and diaper tests.

If we are able to demonstrate a significant reduction in the incontinence symptoms in the subjects we asses that vibration can be a way of reestablishing a normal function of the pelvic floor muscles and bladder function in incontinent patients.


Eligibility Criteria:
Inclusion Criteria:

* Women suffering from stress incontinence
* Women suffering from overactive bladder syndrome
* Men who are still incontinent at a minimum of one year after a radical prostatectomy

Exclusion Criteria:

* Pregnant and nursing women will not be included in the study
Study Investigating Tailored Treatment With Infliximab for Active Crohn's Disease

To investigate whether sustained trough levels of IFX can be achieved using IFX (Infliximab) trough level measurements and adjustment of dosing based upon these levels by means of two different standardized algorithms in comparison with 'standard of care' IFX treatment and its effects on clinical and endoscopic outcomes.


Eligibility Criteria:
Inclusion Criteria:

* Age \> 18 years
* Active CD (CDAI\>220) and signs of active inflammation as evidenced by elevated serum hsCRP levels (\>5 mg/L) and/or elevated fecal calprotectin levels (\>250 µg/g) and endoscopically visible ulcers.
* Patients must be naïve to biologics with indication for starting anti-TNF therapy in accordance with national reimbursement criteria.
* Patients must be naïve to thiopurines or have failed therapy with thiopurines (in which case AZA will be continued).
* Ongoing steroids are allowed if at stable dose for at least 2 weeks and at a maximum of prednisone 40 mg/d or budesonide 9 mg/day.
* Patients who consent to receiving Infliximab 5 mg/kg at week 0, 2 and 6 and further on every 8 weeks in conjunction with azathioprine (2,5 mg/kg/day). Patients who develop AZA intolerance during the trial are continued in the trial without AZA (ie IFX monotherapy).

Exclusion Criteria:

* Absence of endoscopically visible ulcers
* Prior exposure to infliximab (other biologics allowed)
* Ongoing steroid therapy at doses \> 40 mg/d prednisolone equivalent
* Previous intolerance to azathioprine leading to drug discontinuation
* Ongoing infections
* Positive tuberculosis screen per local guidelines
* Serious other diseases including cancer in the 5 years prior to inclusion excluding non-melanoma skin cancer
* Indication for immediate surgery
* Pregnant or breast-feeding woman.
* Positive fecal culture for Salmonella, Shigella, Yersinia and Campylobacter and/or presence of Clostridium difficile B toxin in the stools
* Active tuberculosis
* Untreated latent tuberculosis (see national recommendations. Appendix 2).
* Non-compliant subjects.
* Participation in another therapeutic study.
Immune Response to COVID-19 Vaccine in Immunotherapy (IO) and Non-IO Treated Cancer Patients

Evaluation of antibody response against SARS-CoV-2 vaccination in patients treated with IO-treatment, in comparison with antibody titers in patients treated with non-IO treatment.

Blood samples will be analyzed to determine the magnitude of SARS-CoV-2 immunity in cancer patients treated with immunotherapy by quantifying antibodies and other tumor or blood-based biomarkers against SARS-CoV-2 epitopes following vaccination. We will also explore for potential development of autoantibodies and other blood-based biomarkers, following SARS-CoV-2 vaccination, and their correlation with clinical manifestation of IO related immune-related adverse events.

Eligibility Criteria:
Inclusion Criteria:

1. Patients with solid malignancies treated with either IO or non-IO treatment.
2. All categories of IO treatment allowed (immune-checkpoint inhibitors, bi-specific T cell engagers, cytokine agonists and others), as well as all categories of non-IO treatment (such as chemotherapy and targeted therapies). These can be given as part of standard of care or within participation in a clinical trial.
3. Patients are allowed to have any of the approved vaccines in Canada, such as Pfizer, Moderna, AstraZeneca or their approved combinations.
4. Patients must receive all appropriate doses of SARS-CoV-2 vaccination, as dictated by the respective vaccine schedules.

Exclusion Criteria:

* None
Post-marketing Surveillance of GSK Biologicals' Cervarix™ When Administered to Healthy Females in Sri Lanka

This PMS study aims to collect safety and reactogenicity data of Cervarix in the local population as per the licensing requirement of the Sri Lankan regulatory authority.

Subjects who received one or two doses of Cervarix prior to the start of this PMS study can also be enrolled in the study. These subjects would receive either 2 doses or 1 dose respectively after being enrolled in the study.

Since there is lack of clarity regarding the PMS study requirement (both from Regulatory agency and Ethics Committee), it was decided that GSK will submit Local PSURs on a regular basis as desired by Local regulatory agency. The same is communicated to Regulatory agency and if they want something more they are requested to revert back with specific requirements.

Eligibility Criteria:
Inclusion Criteria:

* Subjects who the investigator believes that they and/or their parent(s)/ LAR(s) can and will comply with the requirements of the protocol should be enrolled in the PMS study.
* A female aged 10 years and above at the time of the first vaccination.
* Written informed consent obtained from the subject. For subjects below the legal age of consent, written informed consent must be obtained from the parent(s)/LAR(s) of the subject and informed assent must be obtained from the subject. Where subject/subject's parent(s)/LAR(s) are illiterate, the consent form will be countersigned by an impartial witness.
* Subjects of childbearing potential must not be pregnant. Absence of pregnancy should be verified as per the investigator's clinical judgement.

Exclusion Criteria:

* Subjects with contraindication according to the locally approved PI.
* Child in care.
* Previous administration of more than two doses of Cervarix.
* Previous vaccination with a HPV vaccine other than Cervarix.
* Planned administration of another HPV vaccine other than Cervarix during the PMS study.
Prednisone Withdrawal Versus Prednisone Maintenance After Kidney Transplant

The purpose of the study was to determine if rapid discontinuation of corticosteroids (also known as prednisone withdrawal) and maintenance immunosuppression with Prograf (tacrolimus) and CellCept (mycophenolate mofetil) while using Thymoglobulin (Rabbit antithymocyte globulin) will give similar safety and efficacy results compared to continuation of corticosteroids (also known as prednisone maintenance) and standard maintenance immunosuppression with Prograf (tacrolimus), CellCept (mycophenolate mofetil) while using Thymoglobulin (Rabbit antithymocyte globulin).

Corticosteroids (one specific type is prednisone) have been used in clinical transplantation for more than 30 years. There are many side effects of corticosteroids including significant bone disease, diabetes (elevated blood sugar levels), fluid retention and hypertension (high blood pressure), psychosis, peptic ulcer disease, hyperlipidemia (elevated lipid levels such as cholesterol and triglycerides), obesity (overweight), acne, and susceptibility to infections. It is hoped that the new generation of potent immunosuppressive medications (such as Prograf and CellCept) will permit avoidance or withdrawal of corticosteroids for the majority of patients to avoid both short- and long-term complications of corticosteroid use in kidney transplant recipients.

Eligibility Criteria:
Inclusion Criteria:

* First time kidney transplant recipients who receives a kidney from a cadaveric, living related or living unrelated donor
* Age greater than 18 years and less than 75 years
* Caucasian recipients
* Patients with current low panel reactive antibody (PRA) levels (\<10%)
* Patients with signed and dated informed consent
* Women of childbearing potential must have a negative pregnancy test at baseline and agree to use a medically acceptable method of contraception throughout the treatment period.

Exclusion Criteria:

* Other than Caucasian ethnicity
* Patients with HIV+ or
* Patients with HbsAg+ or Hepatitis C positive
* Patients with a history of malignancy in the past 5 years
* Patients with active systemic or localized major infection
* Patients with a history of chronic steroid use for other diseases