cyrilzakka/clinical-trials huggingface dataset:
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against factor VIII (FVIII).


Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of mild (FVIII level between \>5% and \<40%) or moderate (FVIII level between ≥1% and ≤5%) congenital Hemophilia A without FVIII inhibitors
* Weight ≥3 kilograms (kg)
* Need for prophylaxis based on investigator assessment
* A negative test for inhibitor (i.e., \<0.6 Bethesda Units per milliliter \[BU/mL\]) within 8 weeks prior to enrollment
* No documented inhibitor (i.e., \<0.6 BU/mL), FVIII half-life \<6 hours, or FVIII recovery \<66% in the last 5 years
* Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks prior to enrollment
* Adequate hematologic, hepatic, and renal function
* For women of childbearing potential: agreement to remain abstinent or use contraception (as defined in the protocol) during the treatment period and for at least 24 weeks after the final dose of study drug

Exclusion Criteria:

* Inherited or acquired bleeding disorder other than mild or moderate congenital hemophilia A
* History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator's judgment
* Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease
* Other conditions that may currently increase the risk of bleeding or thrombosis
* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
* Planned surgery during the emicizumab loading dose phase (surgeries in participants on emicizumab from Week 5 onwards are allowed)
* Known HIV infection with CD4 counts \<200 cells per microlitre (/μL)
* Concomitant disease, condition, significant abnormality on screening evaluation or laboratory tests, or treatment that could interfere with the conduct of the study, or that would in the opinion of the investigator, pose an additional unacceptable risk in administering study drug to the participant
* Receipt of any of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration with the exception of prior emicizumab prophylaxis; A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter; or Any other investigational drug currently being administered or planned to be administered
* Inability to comply with the study protocol in the opinion of the investigator
* Pregnant or breastfeeding, or intending to become pregnant during the study (women of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of study drug)
Dose Response Study of Inhaled Ciclesonide (Glucocorticosteroid) to Patients With Persistent Asthma

To investigate the efficacy and safety of ciclesonide inhaled into the lungs in a range of doses in a new dry powder inhaler compared to ciclesonide inhaled into the lungs using a metered dose inhaler


Eligibility Criteria:
Inclusion Criteria:

* Males or females 12 years or older; History of persistent bronchial asthma for at least 6 months; Documented use of an inhaled steroid for at least 1 month before screening; At screening, FEV1 40% or more of predicted normal and at randomization, FEV1 between 40 and 80% predicted normal; Reversibility of FEV1 by at least 12% and 200 ml post-bronchodilator; Be able to use oral inhalers; Non-smokers

Exclusion Criteria:

* History of life-threatening asthma; other pulmonary diseases; URI within 4 weeks before screening; Use of systemic steroids within 1 month before screening or more than 3 times in previous 6 months; beta-adrenergic blocking agent use; More than 2 in-patient hospitalizations or ER visits due to asthma exacerbations in the prior year before screening; Pregnant or breast-feeding females; Females of child-bearing potential not using adequate means of birth control; Clinically relevant cardiovascular, hepatic, neurologic, endocrine, or other major systemic disease; Abnormal clinical laboratory parameters that would limit participation in the study or interfere with interpretation of study results; History of drug or alcohol abuse; Treatment with any investigational product within 30 days prior to study entry
Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

This study is designed to help identify patients with HoFH due to mutations in the LDLR as confirmed by genotyping.

This is a non-interventional study; no investigational product is administered in this study. Information collected in this study may be used to identify potential participants for clinical gene therapy trials in HoFH.

The investigator will discuss the study with participants who have a clinical presentation consistent with HoFH and where possible, the treating physician, in order to assess their interest to participate. After informed consent has been obtained, participants will be asked to provide a blood sample (up to 40 mL) for genotyping to confirm genetic diagnosis of HoFH due to mutations in LDLR, a lipid panel and anti-AAV8 NAb titer. Participants and, whenever possible, their treating physician will complete a medical history questionnaire and provide supporting documentation. The informed consent form (ICF) and data collection methods may vary depending whether the informed consent is obtained remotely or at a participating study site.

Information collected will include the following:

* patient demographics (age, sex, weight)
* medical history
* previous genotype results (if available)
* results of most recent lipid panel(s)
* use of lipid lowering therapies, including failure to respond
* assessment of presence of liver disease, including history of hepatitis B and C, human immunodeficiency virus (HIV), cirrhosis, and alcohol use

Once this information is received by the investigator and Sponsor, a preliminary assessment of confirmation of a diagnosis of HoFH will be performed. Participants and/or their treating physician will be informed of the results of the genetic testing. All participants will be provided an opportunity to speak with a genetic counselor upon receiving the results of the genetic testing.

Data collected in this study may be used to identify potential candidates for separate clinical trial(s) using gene therapy.

Eligibility Criteria:
Inclusion Criteria:

1. Males and females ≥ 18 years of age
2. Clinical presentation consistent with HoFH

Exclusion Criteria:

1. History of cirrhosis based on documented histological evaluation or noninvasive imaging
2. Documented diagnosis of liver diseases
3. History of immunodeficiency diseases, including a positive HIV test result
4. Previous organ transplantation
A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation

This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, RLY-2608, in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations driven by PIK3CA mutation. Part 1 is a dose selection, Part 2 is a basket design with exploratory single-arm cohorts for various subpopulations of participants, and Part 3 is randomized, double-blinded study vs placebo.


Eligibility Criteria:
Key Inclusion Criteria:

* The participant must have a clinical diagnosis of PROS or a malformation within the ISSVA 2018 classification.
* One or more documented activating PIK3CA mutation(s) that are targeted by selective PI3Kα inhibitors in lesional tissue and/or cell-free DNA from the lesion or blood
* Lansky (\<16 yo) or Karnofsky (≥16 yo) performance status of ≥50.
* Agree to provide archived lesional fluid and/or tissue or be willing to undergo pretreatment lesional biopsy (if considered safe and medically feasible) to assess PIK3CA status.

Key Exclusion Criteria:

* History of hypersensitivity to PI3K inhibitors.
* Any factors that increase the risk of QTc prolongation or risk of arrhythmic events
* Clinically significant, uncontrolled cardiovascular disease
* Received disease-directed therapy prior to the first dose of study drug:

  1. Systemic therapy within 5 half-lives of the drug or 14 days, whichever is longer and received antibody therapy within 28 days.
  2. Local therapy including radiation, surgery, or other procedures within 28 days; lesion(s) must have demonstrated progression after the procedure.
Efficacy Study in Patients With Acne Vulgaris.

Safety and efficacy study in patients with acne vulgaris

randomized, double-blind, placebo controlled, parallel-group, multiple-center study to evaluate the efficacy and safety of ADPS in the treatment of acne vulgaris.

Eligibility Criteria:
Inclusion Criteria:

* Healthy male or non pregnant female aged ≥ 9 years with a clinical diagnosis of acne vulgaris

Exclusion Criteria:

* Female Subjects who are pregnant, nursing or planning to become pregnant during study participation.
Patient Buddy App for the Prevention of Avoidable Readmission in Cirrhosis

To use the PatientBuddy App to reduce 30-day avoidable readmissions in cirrhotic patients using a patient and caregiver perspective.

Specific aim 1: To perform a pilot and feasibility study of disseminating current knowledge of readmission prevention in cirrhosis by using the Patient Buddy App with patient and caregiver interfaces. Eighty subjects (40 cirrhotic inpatients and 40 caregivers) will be enrolled and trained on the Patient Buddy at discharge. The App focuses on daily communication between patient/caregivers and the study team based on previously identified risk factors associated with readmission. The study team will initiate measures to prevent readmission using these communications. A detailed analysis of 30-day readmissions and input of the patients,caregivers and nurse manager will be performed to improve the App. Avoidable readmissions will be defined by a committee of hepatologists who is not part of the study at VCU.

Specific aim 2: To incorporate the opinion of key stakeholders (patients, caregivers and case managers) towards improving the Patient Buddy App in the prevention of avoidable readmission in cirrhosis. The input of the key stakeholders and the analysis of readmissions prevented during the pilot study will be used to update the App for preventing avoidable readmissions in future studies

Eligibility Criteria:
Inclusion Criteria:

* Cirrhosis patients ≥21 years of age hospitalized for non-elective reasons
* Adult caregiver and the patient living in the same house
* Both (caregiver and patient) should be able to complete the Patient Buddy training and evaluation
* Discharged home from the hospital
* Patients who have VCU as a primary hospital base

Exclusion Criteria:

* Elective hospitalization
* Lack of an adult caregiver
* Active alcohol/substance abuse within 1 month of the hospitalization
* Unable to perform training or give consent
* Patients discharged to hospice, nursing home or extended care facilities
* Patients on Hemodialysis
INP105 Proof-of-concept Study for the Acute Treatment of Agitation in Adolescents and Young Adults With ASD

This is a Phase 2a, proof-of concept, 2-way, 2-period crossover, double-blind study to evaluate the safety and efficacy of INP105 as an acute treatment versus placebo in adolescents and young adults with autism spectrum disorder (ASD) experiencing agitation. Approximately 32 ASD patients who are currently being treated for agitation/aggression at several inpatient units specializing in behavioral treatment will be enrolled.

INP105 is a novel combination product that sprays a powder formulation of olanzapine to the upper nasal space. An earlier formulation showed a similar extent, but faster rate of absorption compared to the approved intramuscular product. In this study, 5 mg of olanzapine or placebo will be delivered nasally by this combination product to moderately or severely agitated participants.

Participants will undergo several screening assessments, including observation session(s) of episode(s) of agitation resulting from a frustration task (eg, a non-preferred activity). At least one observation session must result in a documented moderate to severe agitation episode prior to the participant being eligible to enroll in the study and be randomized to treatment.

The study will be conducted in 2 phases. A pilot phase will initially enroll at least 6 participants, who will receive both 5 mg INP105 (5 mg olanzapine) and placebo in random order, in the same crossover design as later participants. Participants will be dosed during a documented moderate to severe episode of agitation. Once 6 participants have completed both dosing periods and have at least 48 hours of post-dose safety data collected, a safety and preliminary efficacy analysis will be performed by an independent unblinded statistical group, and a summary report forwarded to a sponsor-led Data and Safety Review Committee (DSRC), who will remain blinded. Enrollment will be paused during the DSRC pilot phase safety and preliminary efficacy results review. Absent any concerning safety signals, the second phase will enroll all remaining participants. The DSRC may suggest revisions to the protocol, and the protocol amended and approved as necessary, prior to further participants being enrolled.


Eligibility Criteria:
Inclusion Criteria:

* Confirmed autism spectrum disorder diagnosis
* Admitted as an inpatient to a behavioral unit prior to informed consent
* Displays episodes of moderate to severe agitation

Exclusion Criteria:

* Hypersensitivity to olanzapine
* History of severe head trauma, stroke, endocrine disorder, or cardiovascular disease
* History of hypotension
* Currently on a chronic dose of olanzapine
* Currently taking ciprofloxacin, enoxacin, fluvoxamine, or carbamazepine
Candonilimab Plus Bevacizumab for Patients With Advanced HCC Who Progressed on A+T

To explore the efficacy and safety of candonilimab plus bevacizumab for patients with advanced hepatocellular carcinoma who progressed on atezolizumab plus bevacizumab.

Atezolizumab plus bevacizumab is the first-line treatent for patients with advanced hepatocellular carcinoma. However, the second-line treatment is absent for patients who progressed on atezolizumab plus bevacizumab. Candonilimab is a humanized bisspecific monoclonal antibody against PD-1/ CTLA-4 IgG1. Candonilimab plus lenvatinib showed strong anti-tumor effect, with objective response of 44%. This single-arm, prospective, phase 2 trial is to explore the efficacy and safety of candonilimab plus bevacizumab for patients with advanced hepatocellular carcinoma who progressed on atezolizumab plus bevacizumab.

Eligibility Criteria:
Inclusion Criteria:

* The diagnosis of HCC was based on the diagnostic criteria for HCC used by the European Association for the Study of the Liver (EASL)
* Patients must have at least one tumor lesion that can be accurately measured according to EASL criteria.
* Barcelona clinic liver cancer-stage C
* Eastern Cooperative Oncology Group performance status of 0 to 2
* Patients have received atezolizumab plus bevacizumab and the tumor have progressed. Patients have not received other treatmets except for the atezolizumab plus bevacizumab.
* No Cirrhosis or cirrhotic status of Child-Pugh class A only
* Not amendable to surgical resection ,local ablative therapy and any other cured treatment.
* The following laboratory parameters:

Hemoglobin ≥ 8.5 g/dL Total bilirubin ≤ 30mmol/L Serum albumin ≥ 32 g/L ASL and AST ≤ 5 x upper limit of normal Serum creatinine ≤ 1.5 x upper limit of normal INR ≤ 1.5 or PT/APTT within normal limits Absolute neutrophil count (ANC) \>1,500/mm3

• Ability to understand the protocol and to agree to and sign a written informed consent document

Exclusion Criteria:

* Evidence of hepatic decompensation including ascites, gastrointestinal bleeding or hepatic encephalopathy
* Known history of HIV
* History of organ allograft
* Known or suspected allergy to the investigational agents or any agent given in association with this trial.
* Cardiac ventricular arrhythmias requiring anti-arrhythmic therapy
* Evidence of bleeding diathesis.
* Patients with clinically significant gastrointestinal bleeding within 30 days prior to study entry.
* Known central nervous system tumors including metastatic brain disease
Pharmacokinetics, Pharmacodynamics and Safety Evaluation of SAR279356 in Intensive Care Unit Mechanically Ventilated Patients

Primary Objective:

- To determine the pharmacokinetics (PK) of a single intravenous (IV) dose of SAR279356 administered to intensive care unit (ICU) patients on mechanical ventilation

Secondary Objectives:

* To determine the safety and tolerability of SAR279356
* To evaluate the pharmacodynamics (PD) and immunogenicity of SAR279356
* Exploratory efficacy of SAR279356 on prevention of bacterial infections

Total study duration (from screening to last follow-up visit) is 91 days break down as follows:

* Screening: 1 day prior to dosing;
* Treatment period: one IV injection on Day 1;
* Follow-up period: 90 days

Eligibility Criteria:
Inclusion criteria :

* ICU patients on mechanical ventilation at the time of randomization
* Patients or legally authorized representative (LAR) giving written informed consent.

Exclusion criteria:

* Patients \<18 years of age;
* Documented Pseudomonas infection or colonization in the last 30 days;
* Immunocompromised patients
* Hypersensitivity to the study drug and/or to prior therapy with monoclonal antibodies;
* Patient who has taken other investigational drugs or prohibited therapy for this study within 1 month or 5 half-lives, whichever is longer;
* Patient or LAR who withdraws consent during the screening (starting from signed informed consent form);
* Acute liver injury related criteria at the time of study entry:
* Pregnant or breast-feeding women

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Community Based Management of Diabetes in Nepal

Type 2 diabetes is the largest growing concern across the globe, and this is particularly apparent in developing countries, including Nepal. Despite the growing burden and chronic nature of diabetes, prevention and control of this disease is far from adequate setting in Nepal. Moreover, there is lack of evidence on effectiveness and affordability of a population approach on reducing blood glucose level among diabetes persons. The main aim of this study is to determine the effect of Female Community Health Volunteers' (FCHVs)-led family-based health education and screening on blood sugar level in adults at community level in Nepal. This is a community-based cluster randomized controlled trial involving family based health education through FCHVs in Nepal. People ≥25 years of age listed in the Community-Based Management of Hypertension (COBIN) study and who were diabetics are eligible for eligible for participation in the study. FCHVs will conduct family based health education and fasting blood glucose measurements in the treatment arm vs not any interventions in the control arm. Independent assessors will conduct the baseline and end line assessment of the intervention. Intention to treat analysis and per protocol analysis will be used in analysis to detect significant differences between treatment and control group participants at baseline and follow up. For individual outcomes, proportions will be compared using Chi-square test and continuous measures will be compared using t-tests. Random effect mixed regression analysis will be adjusted for age and sex. Since randomisation will be at the cluster level in the study, a random-effect model will be used to account for clustering effect. Effectiveness of the intervention will be tested by analysis of covariance, which will allow us to adjust for baseline differences between groups. It is anticipated that the study can give valuable information regarding effectiveness, acceptability, and feasibility of an innovative way to improve diabetes management. Assessing the FCHVs' ability to address diabetes may contribute to develop a policy that can be scaled-up to a national level.


Eligibility Criteria:
Inclusion Criteria:

* Those ≥25 years of age and listed in the COBIN study
* Those who participated in our prevalence study
* Those who give consent to participate in our intervention study
* Those who do not have any plan to migrate from the study area for at least 1 year
* Those who are non-pregnant women
* Those who are not severely ill

Exclusion Criteria:

* Those who do not give consent
* Those who are pregnant women
* Those who are severely ill
* Those who have plans to migrate from the study area within 1 year
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).


Eligibility Criteria:
Inclusion Criteria:

* At least two pathogenic mutations of ATP-Binding Cassette (ABC)A4 gene confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory
* Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive

Exclusion Criteria:

* Macular atrophy secondary to any condition other than STGD1 in either eye
* Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye
* Participation in an interventional study of a vitamin A derivative \</= 3 months prior to screening
* Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia
* Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region
* Diabetes mellitus
* Hemoglobin A1c (HbA1c) value of \>/=6.5%
* Stroke within 12 months of trial entry
* Any major surgical procedure within one month of trial entry or anticipated during the trial
* Any treatment with an investigational agent in the past 60 days for any condition
* Women who are pregnant or nursing
* Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the avacincaptad pegol formulation