Search Results Within Category "Allergies"
Prevalence of Avoidant Restrictive Food Intake Disorder (ARFID) traits among children and adolescents with Food Allergy
This study looks at understanding eating behaviors and attitudes toward food in children/adolescents. More specifically the study's goal is to compare those with and without food allergies to gain a better understanding of a possible underlying factor towards certain behaviors. Children and their caregivers will complete a survey composed of a short section on the child's medical history regarding allergies followed with questions in commonly used clinical assessments.
Age 18 years or older
Intellectual disability preventing comprehension of questions
Child and/or caregiver unable to respond to English-language questionnaire
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with Hereditary Angioedema Type I or II
KONFIDENT-S is a trial to evaluate the long-term safety of KVD900 in patients who are 12 years of age or older with HAE type I or II. This trial will be conducted on an outpatient basis and includes in-clinic visits and televisits. The study will have multiple centers that will be participating in the study. Subjects will receive the study drug as this study does not include a placebo drug.
There will be 10 in-person visits and 14 televisits. Blood will be drawn at all of the in-person visits. The patient will need to complete a daily diary, and complete electronic questionnaires.
Have had at least 2 documented HAE attacks within 3 months prior to the enrollment visit.
Must have been on a stable dose and regimen with one of the protocol-allowed therapies for long-term prophylactic treatment for at least 6 months prior to the enrollment visit.
Male or Female
Must be 12 years of age and older.
Use of attenuated androgens (e.g. stanozolol, danazol, oxandrolone, methyltestosterone, testosterone), or anti-fibrinolytics (e.g. tranexamic acid) within 28 days prior to the Enrollment Visit.
Any clinically significant comorbidity or systemic dysfunction, which in the opinion of the Investigator, would jeopardize the safety of the patient by participating in the trial.
History of substance abuse or dependence that would interfere with the completion of the trial, as determined by the Investigator.
Any pregnant or breastfeeding patient.
A Phase 1/2, Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of BMN 331, an Adeno-Associated Virus (AAV) Vector-Mediated Gene Transfer of Human SERPING1, in Subjects with Hereditary Angioedema (HAE) due to Human C1 Esterase Inhibitor (C1-INH) Deficiency
BMN 331 is an experimental drug that is being tested for the treatment of patients with HAE. This study is the first time BMN 331, which is a gene therapy, is being used in humans. BMN 331 is given only once during this research study and it is given as an infusion. The purpose of this study is to find out what effects, good and/or bad, the Study Drug has on you and your HAE.
You will have a total of 68 in person visits over 68 months where there will be blood drawn and other lab tests. You will have to travel to an off site location to receive the drug once and travel will be paid for by the sponsor of the trial.
Have a diagnosis of Hereditary Angioedema type I or type II
Willing to abstain from alcohol consumption for 52 weeks after the infusion
Females of childbearing potential must be willing to use contraception
Active cancer, autoimmune, hematologic, cardiac, or renal diseases which require regular treatment
Long term use of attenuated androgens
using immunosuppressants including corticosteroids
A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency–Associated Liver Disease With METAVIR Stage F2 to F4 Fibrosis
This research study of fazirsiran (FAZ-i-sir-an; also called TAK-999 or the “study drug”) because you have alpha-1 antitrypsin deficiency-associated liver disease (AATD LD) with METAVIR stage F2 to F4 fibrosis (METAVIR is a system used to score the amount of inflammation and fibrosis seen in a liver biopsy).In AATD, abnormal (Z-AAT) proteins build up in liver cells, leading to varying amounts of liver problems. The goal of treatment with fazirsiran is to prevent and improve the build up of these abnormal proteins that cause liver injury and fibrosis
-The total amount of time you may be involved in the study is about 4 ½ years (230 weeks).-Screening Period of up to 70 days.-Treatment Period of 196 weeks or about 4 years. During the study, you will get the study drug or placebo at the study site.-Follow-up Period of 6 months. You will have visits 6, 12, and 24 weeks after your last injection of the study drug or placebo.-You will have lung function tests (PFT and DLCO) to check how your lungs are workingAt every visit during the treatment period you will have:-Your vital signs will be measured, this includes your heart rate, blood pressure, breathing rate, temperature, and amount of oxygen in your blood.-You will have a brief physical exam.-Your weight will be measured.-You will have an ECG.-Collect lab samplesDuring specific study visits during the treatment period the following tests will be performed:-An abdominal ultrasound-FibroScan-CT scansYou will complete questionnaires.
The participant must use highly effective contraception
The participant must have suitable vein access for blood sampling
Participant agrees not to smoke at any time during the study.
The participant must have a diagnosis of the PiZZ genotype AATD
The participant has a recent lower respiratory tract infection, such as pneumonia, within the last 6 months before screening.
The participant is expected to have severe and unavoidable high-level exposure to inhaled pulmonary toxins during the study such as may occur with occupational exposure to mineral dusts or metals.
The participant has a history of malignancy within the last 5 years
The participant has evidence of other forms of chronic liver diseases
Saliva microRNA signatures in infants with wheezing associated respiratory illness
Wheezing is a common symptom of respiratory distress in infants and children. Infants who wheeze are at increased risk of being diagnosed with asthma, the most common chronic disease of childhood. This study aims to yield an objective measure of asthma risk using molecular markers obtained from saliva. Saliva miRNA (markers used in this study) levels will be measured using HiSeq technology. Refinement and validation of this measure in future large-scale studies could allow clinicians to accurately predict for families an infant’s risk of asthma and optimize medical management to prevent future hospitalizations.
If your child has a respiratory illness you will be asked to provide a saliva sample at one clinic visit. Six months after the initial encounter, you will be asked to do complete surveys designed to be done remotely at home.
$20.00
Presence of respiratory illness symptoms (cough, congestion, shortness of breath, runny nose)
Concurrent pneumonia (bacterial lung infection) at the time of enrollment
Bronchopulmonary dysplasia
Concurrent bacterial infection requiring antibiotics (e.g. otitis media)
A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of the Efficacy and Safety of Brensocatib in Participants with Chronic Rhinosinusitis Without Nasal Polyps – The BiRCh Study
The purpose of this study is to find out if the experimental drug (brensocatib) improves symptoms of Chronic Rhinosinusitis Without Nasal Polyps and is safe. Study treatments include the study drug and mometasone furoate nasal spray (referred to as mometasone). The study drug will either be brensocatib (“active” drug that contains “real” drug) or placebo (looks like brensocatib but does not have any “real” drug in it). Mometasone will be taken by all participants throughout the study as routine care.
If you choose to participate in the study, the following main activities and procedures are required.•Complete questionnaires asking about your chronic rhinosinusitis symptoms and quality of life•Measure the amount of air you can breathe in through both sides of your nose (nasal airflow)•Electrocardiogram (ECG) and vital signs•Physical examination, including examinations of your mouth and skin•Provide blood and urine samples•Sinus computed tomography (CT) scan•Attend 9 study visits•Nasal endoscopy
Unknown at this time
Participants who have at least a 12-week history before Screening Visit of Chronic Rhinosinusitis without nasal polyps
Received a course of antibiotics or steroids to treat symptoms of Chronic Rhinosinusitis within 1 year or previous surgery for Chronic Rhinosinusitis
Diagnosis of Cystic Fibrosis or Primary Ciliary Dyskinesia
Scheduled sinus surgery at any time during the study or have had nasal surgery within 4 weeks of the first study visit
Participants with seasonal allergic rhinitis whose symptoms occur during the treatment period of the study
Current smoker
A Phase 3 Open-label Study to Evaluate the Safety,Pharmacokinetics, Pharmacodynamics, and Efficacy ofCSL312 (Garadacimab) in the Prophylactic Treatment ofHereditary Angioedema in Pediatric Subjects 2 to 11 Years ofAge
This is a voluntary research study that will help us see if the study medication, CSL312, is safe to use in children 2-11 years of age. The research study will also help to see if the medication works to prevent HAE attacks in this age group.
Attend 8 to 9 visits to the study center. Provide personal information about the child participating in the study and complete electronic diaries (eDiaries). The child participating in the study will have physical exams, vital signs, and blood/urine tests completed. The study medication will be given via injection.
Diagnosis of Hereditary Angioedema Type 1 or Type 2
Two or more Hereditary Angioedema attacks within the last 6 months or the 6 months before starting a preventative medication
Receiving any other experimental drug
Diagnosis of any other form of angioedema