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
A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate theEfficacy and Safety of ISIS 721744 in Patients with HereditaryAngioedema (HAE)
This study involves the use of a new medicine known as ISIS 721744 as a new treatment for Hereditary Angioedema, which is designed to lower the amount of a protein, known as prekallikrein (PKK), which is made by your liver. When PKK is made by the liver and released into your blood stream, it can lead to Hereditary Angioedema attacks. Therefore, the purpose of this study is to assess if reducing the amount of PKK can reduce your HAE attacks and to see how safe ISIS 721744 is for patients with HAE.
There will be 11 in-person visits over 11 months that you will need to come to where there will be blood drawn, as well as other assesments. You will receive and injection of the study drug once a month.
Must have a documented diagnosis of Hereditary Angioedema Type 1 or 2
Experience a minimum of 2 attacks during the screening period
Females must refrain from becoming pregnant for the duration of the study
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 Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels 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
This research study will test an investigational medication called KVD900, a new oral drug being developed to treat hereditary angioedema, the same condition you have. This study is being done to understand how well one or two doses of KVD900 300mg or KVD900 600mg works against placebo to bring relief from an attack of HAE.
If you decide to take part in the study, you will come to the site for 2 visits, and you will have 4 televisits, which may be done by phone or video. Your participation in the study will be approximately 25 weeks.You will need to complete a study e-diary that will use an app that will be downloaded onto your personal mobile device, or on a mobile device provided to you for the purpose of this study.We will perform vital signs, a physical exam, an electrocardiogram (ECG), pregnancy test, and blood collection.In this study every patient will treat one attack with 300mg of study drug, one attack with 600mg of study drug, and one attack with placebo. If after 3 hours following your first dose of study drug you do not experience the needed relief, your assigned kit includes a second dose of study drug that you may take for the attack. The second dose is the same treatment as the first dose. If after 1 hour of your second dose of study drug you do not experience the needed relief, you may dose with your conventional medication.
Up to $1,050
Confirmed diagnosis of HAE type I or II at any time in the medical history.
Patient has access to and ability to use conventional on-demand treatment for HAE attacks.
Patient provides signed informed consent or assent (when applicable). A parent or legally authorized representative must also provide signed informed consent when required.
Patients must be able to swallow trial tablets whole.
Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1-inhibitor deficiency, HAE with normal C1-INH (previously known as HAE type III), idiopathic angioedema, or angioedema associated with urticaria.
Use of angiotensin-converting enzyme (ACE) inhibitors after the Screening Visit or within 7 days prior to randomization.
Any estrogen containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Screening Visit.
History of substance abuse or dependence that would interfere with the completion of the trial, as determined by the Investigator.
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 will 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
Ongoing use of steroids (inhaled or oral)