cyrilzakka/clinical-trials huggingface dataset:
Efficacy and Safety of Stannsoporfin in Neonates

This is a multi-center, randomized, sham injection-controlled (placebo) masked trial of a single intramuscular injection of Stannsoporfin compared to "sham" (placebo) in healthy term and near-term newborns admitted to the well-baby nursery and enrolled with "intention to treat".

For purposes of analysis, 2 patient populations are defined. One population consists of those babies who did not develop severe hyperbilirubinemia (TSB \<9 mg/dL) during the first 36 hours of age. This population is referred to as the untreated population of screened but not randomized patients. The second population of babies was defined as those infants who develop severe hyperbilirubinemia. These infants were randomized to treatment with either stannsoporfin or the sham injection and will be the focus of the efficacy and safety analysis. This population was referred to as the treated population.

The treated population was used for the efficacy and safety analysis. These infants were randomized to either stannsoporfin or the sham injection treatment group.

Eligibility Criteria:
Patients were enrolled who meet the following criteria:

1. The infant's family may be approached at anytime from 6 hours after birth onward to discuss the possibility of enrollment. However, Informed Consent may not be signed until the baby is between 12 and 36 hours of age.
2. Consent may be obtained as soon as possible after 12 hours of age, when the baby enters the well-baby nursery.
3. An infant may be included if the mother has received, or the baby is receiving, antibiotics and the infant is asymptomatic. For example, the baby has no signs of cardiorespiratory distress and is feeding well. Symptomatic infants are excluded (see Exclusions, below).
4. Born at the study hospital site and admitted to the well-baby nursery or admitted to the intensive-care nursery for \<12 hours for post delivery observation (delayed transition, temperature or dextrose instability, meconium staining and/or parental concern).
5. All babies on oral feeding with stable cardiorespiratory status and deemed "healthy" upon clinical examination, with normal perfusion as defined by capillary filling of the fingernail.
6. Term neonate (≥38 weeks of completed gestation), OR
7. Near-term neonate (\>36\<38 weeks of completed gestation; \>2,000g birth weight), OR
8. Near-term neonate (\>35\<38 weeks of completed gestation; \>2,500g birth weight)
9. Care provided (primarily) in the well-baby nursery population;
10. Absence of concurrent cardiorespiratory distress, sepsis, major congenital anomalies or need for care in an intensive care nursery;
11. Not participating in another concurrent unrelated study.

Exclusion Criteria

The study enrolled patients who did not have any of the following exclusion criteria:

1. Any condition that in the opinion of the investigator would make the subject unsuitable for the study.
2. Infants receiving antibiotics who are also symptomatic are excluded. For example, a baby who is feeding poorly or is in cardiorespiratory distress is excluded.
3. Mother has received phenobarbital in past 30 days.
A Study to Evaluate the Safety, Tolerability and Drug Levels of BMS-986454 in Healthy Participants

The purpose of this study is to assess the safety and tolerability of BMS-986454 in healthy adult participants.


Eligibility Criteria:
Inclusion Criteria:

* Male and female participants who are healthy as determined by the investigator based on review of medical history, physical examinations, 12-lead ECGs, and clinical laboratory tests obtained during the screening period.
* Participant is willing and able to adhere to the study visit schedule and other protocol requirements.
* A negative test for COVID-19, which will be performed in the manner mandated by the clinical site where this study is being conducted, at screening and admission.

Exclusion Criteria:

* Evidence of organ dysfunction or any clinically significant deviation from normal in physical examination, vital signs, 12-lead ECG, or clinical laboratory tests beyond what is consistent with a healthy population in the region in which the study is conducted.
* Participant was exposed to an investigational drug (new chemical entity) within 30 days preceding the first dose administration, or 5 half-lives of that investigational drug, if known (whichever is longer).
* Inability to be venipunctured or tolerate venous access.

Note: Other protocol-defined inclusion/exclusion criteria apply
RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease

The objective of the study is to determine the safety and tolerability of RG2133 in patients with Mitochondrial Disease.


Eligibility Criteria:
Mitochondrial Disease
Perioperative Pregabalin and Lidocaine to Reduce Chronic Breast Cancer Pain

Breast tissue and the areas surrounding the breast contain many small to medium-sized nerves. During surgery on the breast, these nerves can be inadvertently cut or damaged. Chemotherapy and radiotherapy after surgery worsen the injury experienced by these nerves. These nerves eventually become abnormal and provide pain signals to the brain well beyond the healing period after surgery. Patients with these abnormal nerves suffer from chronic pain in the breast area that persists for several years after surgery. Chronic pain is associated with a reduced quality of life, daily functioning, psychological distress, and contributes to excessive health care expenditures. There is encouraging data suggesting that an infusion of Lidocaine during surgery and Pregabalin given around the time of surgery can prevent the development of chronic pain after breast cancer surgery. A large randomized controlled trial is needed to determine the efficacy of these two interventions on reducing chronic pain after breast cancer surgery (e.g. within 3 months of surgery). A pilot trial is first needed to determine the feasibility of undertaking such a trial.


Eligibility Criteria:
Inclusion Criteria:

* female patients 18-75 years of age
* undergoing a unilateral or bilateral mastectomy or partial-mastectomy (breast conserving surgery), for prophylactic (e.g. family history or BRCA gene mutation) or belief of isolated (non-metastatic) cancerous lesions
* receiving a general anesthetic

Exclusion Criteria:

* previous breast surgery within six months of index surgery
* undergoing a DIEP (Deep Inferior Epigastric Perforator) flap procedure
* patient has chronic pain or a chronic pain syndrome for which they have taken 4 or more daily medications (i.e. opioids, anti-convulsants, anti-spasmodic, anti-depressants, anti-inflammatories) or routine pain intervention (i.e. nerve blocks) during the past 3 months
* documented hypersensitivity or allergy to pregabalin, gabapentin, or lidocaine
* history of ventricular tachycardia, ventricular fibrillation, or atrioventricular block ≥ type II
* history of congestive heart failure
* renal insufficiency with creatinine \> 120 µmol/L
* known or previously documented cirrhosis
* pregnant
* unable to swallow study medications
* patient's surgeon believes patient is inappropriate for inclusion in trial
* unlikely to comply with follow-up (e.g. no fixed address, plans to move out of town)
* language difficulties that would impede valid completion of questionnaires
* patient requires gabapentin or pregabalin for a medical condition or has been taking gabapentin or pregabalin daily during the past 1 week
Onchocerciasis-Associated Epilepsy, an Explorative Case-control Study

This is an exploratory case control study with the aim to compare the Onchocerca volvulus virome between persons with onchocerciasis-associated epilepsy (OAE) and persons with onchocerciasis but without epilepsy.

The main question we want to address is: Is there a virus contained in the O. volulus worm that may have a pathogenic role in causing OAE.

In Maridi County, South Sudan, 20 persons with OAE with onchocerciasis nodules, and 20 age- and village-matched controls without OAE will be enrolled in a nodulectomy study.The adult O. volvulus worms will be extracted from the nodules and a viral metagenomic study of the worms. The O. volvulus virome of persons with and without OAE will be compared.

A high prevalence of onchocerciasis-associated epilepsy (OAE) has been observed in onchocerciasis-endemic areas with high ongoing Onchocerca volvulus transmission. However, the pathogenesis of OAE remains to be elucidated. We hypothesise that the O. volvulus virome could be involved in inducing epilepsy. With this study, we aim to describe the O. volvulus virome and identify possible viruses linked to OAE.

In Maridi County, South Sudan, 20 persons with OAE with onchocerciasis nodules, and 20 age- and village-matched controls without OAE will be enrolled in the study. For each study participant, two skin snips at the iliac crest will be obtained to collect O. volvulus microfilaria, and one nodulectomy will be performed to obtain adult worms. A viral metagenomic study will be conducted on microfilariae and adult worms, and the O. volvulus virome of persons with and without OAE will be compared. Moreover, the prevalence, numbers and localisation of onchocerciasis nodules in persons with and without OAE in villages with a high prevalence of epilepsy in Maridi will be described.

Eligibility Criteria:
Inclusion Criteria:

* Onchocerciasis nodules
* Age 12-35 years old
* informed consent signed
* Living in Maridi County, Western Equatorial State, South Sudan

Exclusion Criteria:

* below the age of 12
Endoscopic Findings in Patients With Upper GIT Bleeding

This study aims to identify the causes of upper gastrointestinal bleeding through comprehensive evaluation via upper endoscopy in Assiut University Hospital and contribute valuable insights into effective management strategies.

Upper gastrointestinal bleeding (UGIB) is a critical medical situation that arises when there is bleeding proximal to the ligament of Treitz \[1\]. This condition is a leading cause of morbidity and mortality, with death rates ranging from 5-14% and increasing to over 40% in patients with liver disease\[2\] Symptoms of UGIB may include anemia, hematemesis, melena, or hematochezia \[3\].

Neoplasms, arteriovenous malformations, esophageal varices, gastric ulcers, gastric erosions, duodenal ulcers, reflux esophagitis, Mallory-Weiss tears, and fundal varices are causes of UGIB \[4\].

Current strategies for managing UGIB emphasize early recognition and intervention. Endoscopy is the primary diagnostic and therapeutic modality, allowing for direct visualization of the gastrointestinal tract and enabling interventions such as cauterization, clipping, or band ligation to control bleeding \[5\]. Recent advancements in endoscopic techniques have improved outcomes by facilitating earlier detection and treatment of UGIB episodes. For instance, the use of hemostatic powders and over-the-scope clips has shown promise in managing refractory cases\[6,7\].

Endoscopic therapies may have resulted in earlier detection and treatment of UGIB, which could have led to a reduction in the incidence and severity of the condition. Upper GI endoscopy is considered the primary modality for identifying the underlying etiology and administering appropriate therapeutic measures to the patient present with upper GIT bleeding by ligation ,clips, electrocoagulation or by taking biopsies that help in diagnosis. The rationale for this research stems from the need to enhance understanding of UGIB's underlying causes and improve patient outcomes through timely endoscopic intervention. By identifying specific etiologies through upper endoscopy\[8,9\].

Eligibility Criteria:
Inclusion Criteria:

* Adult patients present with upper GIT bleeding of both gender older than 18 years old

Exclusion Criteria:

* 1- Patients younger than 18 2- Pregnant women 3- patients with end stage disease that not fit for endoscopic intervention
Chemotherapy for Patients With Non-Small Cell Lung Cancer Who Are Non-Smokers

The purpose of this study is to compare the efficacy and safety of chemotherapy followed sequentially by gefitinib versus chemotherapy alone in the first line treatment of non-small cell lung cancer (NSCLC). This study will be conducted in Asian patients who are classified as 'never smoker' since it is suggested that these patients are more likely to respond favorably to treatment with gefitinib.


Eligibility Criteria:
Inclusion Criteria:

* Histologic or cytologic diagnosis non-small cell lung cancer (NSCLC) (Stage IIIB or IV)
* Have not received any prior chemotherapy, molecular therapy, immunotherapy, biological therapy, or radiotherapy. Exception: palliative radiotherapy that is completed at least 4 weeks prior to study enrolment.
* Have 'never smoked' (defined as having smoked \<100 cigarettes during his/her lifetime)
* Eastern Cooperative Oncology Group (ECOG) performance status 0 to 1

Exclusion Criteria:

* Concurrent administration of any other tumor therapy
* Other co-existing malignancies
* Pregnancy or breast feeding
* Serious concomitant disorders
* Inability or unwillingness to take folic acid or vitamin B12 supplementation
Defining an Obesity QTL on Chromosome 3q

To investigate the genetic basis of obesity by fine mapping an obesity quantitative trail linkage (QTL) linked to chromosome 3q.

BACKGROUND:

In the last several decades obesity has emerged as a major public health threat. While prevention through lifestyle change is the only long-term solution, better understanding of the physiologic mechanisms would greatly assist development of drugs and targeted prevention. Obesity is a highly heritable condition and while genes must account for a substantial proportion of individual susceptibility they have eluded detection. Powerful new genetic and genomic tools now permit comprehensive evaluation of candidate genes, including all genes under linkage peaks. These tools include new genomic resources (the human genome sequence, databases of common SNPs, and the haplotype map), rapid and inexpensive discovery and genotyping and new analytic methods (haplotype-based association and admixture mapping).

DESIGN NARRATIVE:

In a large African American family set Dr. Zhu and colleagues have obtained strong linkage evidence for obesity on chromosome 3q (combined LOD score = 3.7). A prime candidate (adiponectin) lies near this peak. They propose to follow up that finding by combining the epidemiologic data with high-throughput genotyping and move from linkage to association analysis. The results for this QTL will be evaluated within the available environmental factors to assess potential gene-environment and gene-gene interactions. The available phenotypes include body composition, resting metabolic rate, physical activity, plasma insulin, glucose, and leptin. In a family-based design they will examine the linkage peak centered on position 188 cM on chromosome 3q (20 Cm 1-LOD support interval), with the following step-wise strategy: (a) Genotype 200 single nucleotide polymorphisms (SNPs) in this region on 300 families (1,000 individuals); (b) Conduct linkage, linkage disequilibrium and admixture mapping to potentially further narrow the region; and (c) Conduct resequencing and haplotype-based association studies for all candidate genes under the peak. Statistical analysis incorporating intermediate phenotypes and environmental covariates will be used to characterize potential gene x gene or gene x environment interactions. Replication will be tested in additional populations of African and European origin.

Eligibility Criteria:
The survey enrolled a representative random sample of the population the age of 18 and 74, regardless of obesity phenotype.
Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

This study is a post approval commitment to evaluate the accuracy and precision of retroviral insertion site (RIS) analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects treated with Strimvelis.

This non-interventional, retrospective, methodology study will use peripheral blood and bone marrow samples previously taken from subjects treated with Strimvelis. The study does not require subjects to undergo any further treatment, intervention or blood withdrawal.

The objective of this methodology study is to evaluate the accuracy and precision of shearing extension primer tag selection ligation-mediated polymerase chain reaction (S-EPTS/LM-PCR) for RIS analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects previously treated with Strimvelis for severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.

Eligibility Criteria:
Inclusion Criteria

Subjects must have previously received treatment with gRV-GT, either during clinical development (clinical trials and early access programs) or in the post-marketing setting as the approved product (Strimvelis) or under hospital exemption, and for whom at least one biological sample is available that meets the following eligibility criteria::

1. Peripheral blood, bone marrow, or DNA extracted from either source.
2. Taken at least 6 months after gRV-GT.
3. Stored at -20oC or below since the time of sampling.
4. Likely to provide at least 1.5 μg of DNA (following extraction by the central laboratory).

Exclusion Criteria N/A
A Safety Study to Evaluate 12 Weeks of Treatment with Clevudine in Patients Infected with Hepatitis B Virus.

The purpose of the study is to evaluate the safety and effectiveness of 12 weeks of treatment with clevudine, at one of three doses, in patients chronically infected with hepatitis B virus.


Eligibility Criteria:
Inclusion Criteria:

* HBV DNA positive with DNA levels at screening greater than or equal to 3,000,000 copies/mL.
* Documented to be HBsAg positive for \> 6 months OR HBsAg positive and IgM anti-HBc negative and anti-HBs negative. Patients may be HBeAg positive, and anti-HBe negative OR HBeAg negative (pre-core mutant) and anti-HBe positive.
* HBeAg positive and anti-HBe negative OR HBeAg negative (pre-core mutant) and anti-HBe positive with positive HBsAg for the previous 6 months
* AST and ALT levels which are less than or equal to 10 times the upper limit of normal.
* Bilirubin levels less than or equal to 1.5 x ULN or bilirubin levels \> 1.5 x ULN with diagnosis of Gilbert's disease and conjugated bilirubin within normal limits.

Exclusion Criteria:

* Currently receiving antiviral, immunomodulatory or corticosteroid therapy
* Previous treatment with lamivudine, lobucavir, adefovir, famciclovir, or any other investigational nucleoside for HBV infection
* Previous treatment with interferon must have ended at least 6 months prior to screening visit
* History of ascites, variceal hemorrhage or hepatic encephalopathy
* Co-infection with HCV or HIV
* Evidence of cirrhosis or hepatocellular carcinoma (alpha fetoprotein)
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to \< 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to \< 15 years old, with Sickle Cell Disease. The study will be conducted at approximately 50 international clinical sites, and will enroll approximately 224 participants. Participants will be randomized in a 1:1 ratio to receive voxelotor or placebo. All participants younger than 12 years of age and randomized to voxelotor will receive a dose based on their body weight, to provide exposure corresponding to the adult dose of 1500 mg/day.

Eligibility Criteria:
Inclusion Criteria:

1. Male or female participants with Sickle Cell Anemia (SCA) HbSS, HbSβ0 thalassemia genotype
2. TCD time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow ≥ 170 to 200cm/sec during the Screening Period
3. Hb ≥ 5.5 and ≤ 10.5 g/dL during screening
4. For participants taking HU, the dose of HU (mg/kg) must be stable for at least 90 days prior to signing the informed consent form (ICF) and/or assent form, and with no anticipated need for dose adjustments (other than weight based) or for initiation of HU for non-chronic use during the study, in the opinion of the Investigator
5. Written informed parental/guardian consent and participant assent (where applicable) has been obtained per IRB/EC policy and requirements, consistent with ICH guidelines.

Exclusion Criteria:

1. Body weight \< 10kg at the screening visit
2. Hospitalization for VOC or acute chest syndrome (ACS) within the 14 days prior to execution of informed consent/assent
3. More than 10 VOCs within the past 12 months that required hospitalization, emergency room, or clinic visit
4. Stroke resulting in focal neurological deficit; previous silent infarcts are permitted.
5. Known history or findings suggestive of significant cerebral vasculopathy
6. History of seizure disorder
7. Has been treated with erythropoietin or other hematopoietic growth factors within 28 days of signing informed consent/assent or if, in the opinion of the Investigator, there is an anticipated need for such agents during the study
8. RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion) or has received an RBC transfusion or exchange transfusion for any reason within 90 days of signing the informed consent/assent
A Study To Examine The Safety, Tolerability And Pharmacokinetics Of PF-04958242 In Psychiatrically Stable Subjects With Schizophrenia

To evaluate the safety and tolerability of multiple, ascending doses of PF-04958242 administered orally to psychiatrically stable subjects with schizophrenia receiving antipsychotic and adjunctive medication.

This study was previously posted by Pfizer, Inc. Sponsorship of the trial was transferred to Biogen.

Eligibility Criteria:
Inclusion Criteria:

* Psychiatrically stable subjects with schizophrenia
* Evidence of stable schizophrenia symptomatology ≥ 3 months
* Score on MCCB Letter-number span + Spatial span subtest \< 40.

Exclusion Criteria:

* History of seizures or of a condition with risk of seizures
* History of abnormal EEG.
* Pregnant or nursing females, and women of child bearing potential
EMR Versus ESD for Resection of Large Distal Non-pedunculated Colorectal Adenomas

Endoscopic resection of adenomas in the colon is the cornerstone of effective colorectal cancer prevention. Endoscopic mucosal resection (EMR) is currently the most used technique for resection of large distal colorectal adenomas, however, maintains some important limitations. In large lesions, EMR can often only be performed in a piecemeal fashion resulting in relatively low R0-resection rates and high recurrence rates. Endoscopic submucosal dissection (ESD) is a newer procedure that is more difficult resulting in a longer procedural time, but is promising due to the high en-bloc resection rates and the very low recurrence rates. The aim of this multicenter randomized study is to compare EMR and ESD with regard to recurrence rates and radical (R0) resection rates, and to put this into perspective against the costs and complication rates of both strategies and the burden perceived by patients on the long term-term.


Eligibility Criteria:
Inclusion Criteria:

* non-pedunculated polyp larger than 20 mm in the rectum, sigmoid or descending colon found during colonoscopy
* indication for endoscopic treatment
* ≥18 years old
* Written informed consent

Exclusion Criteria:

* suspicion of malignancy, as determined by endoscopic findings (invasive Kudo pit pattern, Hiroshima type C) or proven malignancy at histology
* prior endoscopic resection attempt
* presence of synchronous distal advanced carcinoma that requires surgical resection
* the risk exceeds the benefit of endoscopic treatment, such as patient's with an extremely poor general condition or a very short life expectancy
* the inability to provide informed consent