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A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines. DPPMs can cause dysfunctions throughout the body, especially in the brain, blood, kidneys, and immune system. People with DPPMs might have no symptoms, mild symptoms, or they may have severe, chronic symptoms, that can be fatal. DPPMs are not well understood, and researchers want to learn more about what causes them and how to treat them. Objective: To learn more about factors that affect DPPMs by comparing test results from affected, uaffected family members, and healthy people. Eligibility: Three types of participants are needed:...
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An Efficacy and Safety Study of Intravenous Anifrolumab to Treat Systemic Lupus Erythematosus in Pediatric Participants
A Study to evaluate the PK, PD, efficacy, and safety of Anifrolumab in children with moderate to severe active SLE
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An Evaluation of Hip Preservation Outcomes
The purpose of this study is to assess outcomes of hip preservation surgeries including open and arthroscopic treatment of femoroacetabular impingement (FAI).
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An Immediate Functional Progression Program for Adolescent Athletes With Spondylolysis
Spondylolysis, a stress fracture in the pars interarticularis of a lumbar vertebra, is the most common identifiable cause of low back pain (LBP) in adolescent athletes, occurring in 14-30% of athletes who experience LBP. Spondylolysis can cause significant pain and disability and months of exclusion from sports or an active lifestyle. Standard care of spondylolysis in adolescent athletes is primarily based on expert opinion, with dramatic variations in clinical practice, including restrictive bracing, extended rest periods before the intervention, long durations out of sport and activity, and suboptimal long-term clinical outcomes. As the next step towards our research goal, the...
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An Open-Label Study of Effectiveness of Immunomodulatory Medications for Patients With Relapsing Polychondritis
Open label pragmatic two-stage non-randomized trial comparing the effectiveness of five different standard of care treatment options for patients with relapsing polychondritis (RP).
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An Study to Evaluate Safety and Tolerability of OA-SYS in Subjects With Moderate to Severe Osteoarthritis of the Knee Joint
This study is being conducted to evaluate the safety and tolerability of OA-SYS in patients with moderate to severe OA of the knee joint.
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A Phase 1/2a to Evaluate the Safety and Efficacy of Adipose Tissue Allograft (BRC-OA) in Patients With Osteoarthritis of the Knee
The purpose of this study is to evaluate the safety profile of BRC-OA for pain treatment in patients with mild to severe OA of the knee, focusing particularly on two distinct doses. Alongside safety assessments, the study also aims to investigate the initial efficacy of BRC-OA in treating pain among these patients. The study product is a cryopreserved devitalized adipose tissue allograft (BRC-OA). BRC-OA is composed of a devitalized human adipose particulate that retains the inherent properties of adipose such as tissue architecture, extracellular matrix, and signaling molecules.
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A Phase 1/2 Study of NKX019 in Subjects With Autoimmune Disease (Ntrust-1)
This is an open-label, multi-center, non-randomized, Phase 1/2 study to determine the safety and tolerability of NKX019 (allogeneic CAR NK cells targeting CD19) in participants with active lupus nephritis (LN) or primary membranous nephropathy (pMN).
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A Phase 1/2 Study of NKX019 in Subjects With Immune-Mediated Diseases (Ntrust-2)
This is a Phase 1/2, open-label, multi-center, multi-cohort, non-randomized dose escalation and dose expansion basket study to determine the safety and tolerability of NKX019 (allogeneic CAR NK cells targeting CD19) in participants with autoimmune diseases.
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A Phase 1b Study of Budoprutug in Systemic Lupus Erythematosus (SLE)
The main objective is to assess the safety and tolerability of budoprutug in adults with SLE. Pharmacokinetics, pharmacodynamics, and preliminary clinical efficacy will also be assessed.
