Pediatric Rheumatology Trials
Pediatric Rheumatology Current Trials
Pediatric Rheumatology Past Trials
Understanding and Diagnosing Allergic Disease in Twins
The purpose of this study is to gain better understanding of how the immune system works in twins with and without allergic disease. Healthy volunteers are not specifically targeted. Healthy non-allergic study participants may be found through the course of evaluation for the presence of allergies.Now accepting new patients
Efficacy and Safety of Several Doses of Viaskin Peanut in Adults and Children With Peanut Allergy
The objectives of this dose-finding study for the treatment of peanut allergy are:
- To determine the efficacy of 3 doses of Viaskin Peanut (50 mcg ,100 mcg and 250 mcg peanut protein per patch) to significantly desensitize peanut-allergic subjects to peanut after 12 months of treatment.
- To evaluate the safety of a long-term treatment with Viaskin Peanut.
InvestigatorNow accepting new patients
Safety Study of Viaskin Peanut to Treat Peanut Allergy
This study evaluates the safety of Viaskin Peanut 250 mcg in the treatment of peanut allergy in children from 4 to 11 years of age. Subjects will receive either Viaskin Peanut 250 mcg or a placebo for a period of 6 months, after which all subjects will be receiving the active treatment up to a period of 3 years under active treatment.Now accepting new patients
Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
Epidermolysis bullosa (EB) simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin. Patients with EB simplex develop lifelong painful thick soles on their feet, and current standard of care is supportive. This pilot study will target the dominant mutant keratin proteins in the skin to ameliorate the severity of EB simplex. The purpose is to improve the function of EB simplex feet with an application of topical sirolimus, 2%. The investigators plan on inhibiting the mTOR pathway to down regulate the translation of defective keratin proteins and work through anti proliferative pathways.Now accepting new patients
Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL)
This study is a 2-part (parts A and B) phase 2/3 study to evaluate the safety, pharmacokinetics (PK) and efficacy of dupilumab in participants 6 months to less than 6 years of age with moderate-to-severe atopic dermatitis (AD).Now accepting new patients
Peanut Reactivity Reduced by Oral Tolerance in an Anti-IgE Clinical Trial
The investigators will perform a double blind, placebo controlled clinical trial with Xolair (omalizumab) at four centers to safely and rapidly desensitize patients with severe peanut allergy. The investigators will determine if pretreatment with anti-IgE mAb (Xolair/omalizumab) can greatly reduce allergic reactions and allow for faster and safer desensitization.
InvestigatorNow accepting new patients
Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS)
PANS is an illness that comes on suddenly in children. The full name is Pediatric Acute-Onset Neuropsychiatric Syndrome. It can cause sudden obsessive-compulsive behaviors. It can also cause children to suddenly restricte their food intake. Researchers want to learn more about children with PANS. They also want to learn more about the illness.
To study some disorders of behavior and emotion that start in childhood.
Children 3 14 years old who have had severe obsessive-compulsive symptoms or food restriction start quickly
Parents will answer questions. The topics include:
Their child s medical history
Their child s physical and mental health
Their family history. The focus will be on neurodevelopmental and psychiatric conditions. A family tree will be drawn.
Participants will have a physical exam.
Participants may take tests on paper or computer. These will focus on thinking, memory, and behavior.
Participants and parents will give a blood sample.
Participants will have magnetic resonance imaging (MRI). A strong magnetic field and radio waves take pictures of the brain. Participants will lie on a table that slides in and out of a metal scanner.
Participants may have photos or videos taken.
Participants may have other tests. These may include heart tests, sleep tests, and lumbar puncture.
Sponsoring Institute: National Institute of Mental HealthNow accepting new patients
Immunogenicity of Co-administered Oral Polio Vaccine and Oral Cholera Vaccine
Concomitant administration of multiple vaccines, including live attenuated immunizations, is safe and effective. Some restrictions apply for live vaccines; administering a live-virus vaccine within 4 weeks after administration of another live-virus vaccine can decrease immunogenicity to the second administered vaccine. Thus, it is recommended that live-virus vaccines should be administered the same day or ≥4 weeks apart. Data on co-administration of the currently available whole-cell killed Oral Cholera Vaccine (OCVs) with other oral vaccines, specifically, oral polio vaccines (OPV) is lacking. Although the risk of immunological interference due to co-administration of live vaccines with non-live vaccines is considered small if at all, a theoretical concern of interference has been raised. Given the substantial geographic correlation between polio- and cholera-affected and at-risk areas, which include some of the world's most impoverished and hard-to-reach populations, a strategy of co-administration of OCV with OPV to children targeted to receive OPVs has the potential to optimize the use of limited resources and improve coverage for both vaccines. The manufacturer recommendation for a two-week interval between administration of OPV and OCV precludes an integrated campaign or routine use in which OCV could be co-administered with OPV.Now accepting new patients
Clinical Study Using Biologics to Improve Multi OIT Outcomes
Food allergy (FA) is a serious public health concern that causes potentially-life threatening reactions in affected patients. The prevalence of food allergy in the United States (U.S.) has increased substantially and now affects 15 million patients:4-8% of children (6 million children, 30% with multiple food allergies) and about 9% of adults. This is a prospective Phase 2, single-center, multi-allergen OIT study in participants with proven allergies to 2 or 3 different foods in which one must be a peanut. The total of participants in the clinical study will be 110, ages 6 to 25 years with a history of multiple food allergies of 2 to 3 different foods including peanut. Allergy will be confirmed by FA-specific IgE levels and positive skin prick test (SPT). Enrolled participants must be positive during the Double-blind Placebo-controlled Food challenge (DBPCFC) at or before the 300 mg (444 mg cumulative) dosing level of FA proteins.Now accepting new patients
Genetic and Environmental Factors in the Response to Influenza Vaccination
The purpose of the study is to investigate and compare the immune responses to influenza vaccination in monozygotic (identical) and dizygotic (fraternal) twins to determine the roles of genetics and environment in the response to flu vaccination.Now accepting new patients
A Study of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee
The objective of this study is to compare the efficacy and safety of MACI® vs arthroscopic microfracture in the treatment of patients aged 10 to 17 years with symptomatic articular chondral or osteochondral defects of the knee.
InvestigatorNow accepting new patients
Efficacy and Safety Study of Apremilast (CC-10004) in Pediatric Subjects From 6 Through 17 Years of Age With Moderate to Severe Plaque Psoriasis
This is a Phase 3, multicenter, randomized, placebo-controlled, double-blind study of the efficacy and safety of apremilast (CC-10004) in pediatric subjects with moderate to severe plaque psoriasis.
At least 230 pediatric subjects (ages 6 through 17 years) will be randomized 2:1 to receive either apremilast or placebo for the first 16 weeks and then all subjects will receive apremilast during the 36 week Extension Phase for a total of 52 weeks. Randomization to apremilast arm or placebo arm will be stratified by age group (6 to 11 years or 12 to 17 years). Subjects will receive apremilast treatment of either 20 mg twice daily (BID) or 30 mg BID, depending on weight. This Phase 3 study is being conducted to evaluate the safety and efficacy of apremilast in the treatment of pediatric subjects.Now accepting new patients
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneously Administered Guselkumab for the Treatment of Chronic Plaque Psoriasis in Pediatric Participants
The purpose of this study is to evaluate the efficacy and safety of guselkumab in pediatric participants aged greater than or equal to 6 through less than 18 years with chronic plaque psoriasis.Now accepting new patients