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Research Studies

Division of Rheumatology Research Studies

Find research studies available to children cared for by the Division of Rheumatology team.

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Anifrolumab Trial for Systemic Lupus Erythematosus (SLE)

The purpose of this study is to find out if the study drug, Anifrolumab, is effective and safe for pediatric participants with moderate to severe active systemic lupus erythematosus (SLE) while on background standard of care therapy. Researchers will also be testing how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body (pharmacokinetics). Subjects will be asked to come into the Rheumatology clinic for blood, hepatitis/HIV, urine, and pregnancy testing, physical exam, an electrocardiogram (ECG), questionnaire completion, medical record review, corticosteroid use, and administration of study drug. Anifrolumab is administered as an infusion every 4 weeks over the course of the study. 

Phase: Phase III

Actively recruiting: Yes

Category: Children

Healthy Controls EndoPAT

The purpose of this research study is to learn more about the normal function of blood vessels in healthy children and adolescents. This will help us discover differences between healthy individuals and children with lupus. Healthy control participants will be asked to make 1 visit to CHOP to complete a brief survey, a fasting blood draw and a test to measure the blood pressure in your fingers.

Phase: N/A

Actively recruiting: No

Category: Adults, Children, Healthy Controls, One-Time Visit Studies

MAS825 Trial for NLRC4-GOF

The purpose of this study is to find out if the study drug, MAS825, iseffective in preventing flares and safe for children with NLRC4-Gain ofFunction (GOF). Researchers will also be testing how the body processes a drug by measuringhow quickly the drug is absorbed by the body, how quickly it is broken down bythe body, and how long it remains in the body (pharmacokinetics). Subjects willbe asked to come into the Rheumatology clinic for blood and stool testing,physical exam, an electrocardiogram (ECG), questionnaire completion, andadministration of study drug. Participation will last for up to 4 years. 

Phase: Phase II

Actively recruiting: No

Category: Adults, Children

Posterity Trial

The purpose of this study is to find out if the study drug, Obinutuzumab, is effective in achieving a complete renal response for adolescents with active class III or IV lupus nephritis. Researchers will also be testing how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body (pharmacokinetics). Subjects will be asked to come into the Rheumatology clinic for blood, hepatitis and pregnancy testing, physical exam, an electrocardiogram (ECG), questionnaire completion, chest x-ray, MMF/oral corticosteroid use, medical record review, optional genetic testing, and administration of study drug. Obinutuzumab is administered as an infusion 5 times over the course of the study. 

Phase: Phase II

Actively recruiting: Yes

Category: Children

Therapeutic use of Tadekinig alfa in NLRC4 mutation and XIAP deficiency

This is a clinical trial to study the use of Tadekinig alfa or placebo (medication) in the treatment of NLRC4 MAS mutation or XIAP deficiency. This study involves taking the medication every 48 hours for up to 26 weeks. Participants will also be required to attend up to 14 in-person study visits to review their disease state while they are taking the medication. Compensation for time and travel related to study visits may be provided to qualified participants.

Phase: Phase III

Actively recruiting: No

Category: Children

VOCAL Trial

This is a study to measure the effectiveness of the medication voclosporin compared to placebo in adolescent patients with active lupus nephritis. Volcosporin is an outpatient, oral medication.

Phase: Phase III

Actively recruiting: Yes

Category: Children

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