May 20th, 2022 - The hypothesis is that the addition of daratumumab administered SC to standard MM regimens will improve responses compared to response data observed in completed phase 3 studies without daratumumab. Disease evaluations will include measurements of myeloma proteins, bone marrow examinations, skeletal surveys, assessment of extramedullary plasmacytomas, and measurements of serum calcium corrected...

May 20th, 2022 - The Laboratory of Immune System Biology plays a major role in fostering the growth of systems biology efforts across the National Institutes of Health, in large measure through its development of new tools for high-throughput data generation and complex systems modeling. The lab s experimental component requires ongoing assay development and optimization, which depend on the availability of hum...

May 20th, 2022 - Background Children and young adults diagnosed with a high-grade or high-risk cancer (e.g., diffuse intrinsic pontine glioma, glioblastoma multiforme, relapsed-refractory leukemia, refractory metastatic sarcomas) face a poor prognosis given limited curative options. Recent research has indicated that this population of patients and their parents experience elevated stress and poorer health-rela...

May 20th, 2022 - 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.

May 20th, 2022 - Background: Acute lymphoblastic leukemia (ALL) accounts for approximately 25% of childhood cancer. Survival rates have improved, but outcomes for some subgroups, including infants and young adults remain poor, and survival for patients who relapse is < 50%, despite allogeneic stem cell transplant following second remission. CD19 immune escape has been observed by several groups following CD19-C...

May 20th, 2022 - See brief summary

May 20th, 2022 - Participants with AML that have gone into remission and come back (relapsed) or gone into remission with a number of leukemia cells still in their system (refractory) will be recruited for this study. They will also be positive for FLT3-ITD mutation. Participants will receive a combined dose of quizartinib and milademetan that have not been approved by the US Food and Drug Administration yet (m...

May 20th, 2022 - Description: This is a longitudinal study of the natural history of the collagen-related disorder osteogenesis imperfecta (OI), that includes enrolling new patients under the age of 12 years, along with an extended data collection from adult patients on whom previous childhood data was collected at the NIH. Objectives: Primary Objectives: 1) Identify and monitor longitudinal functional outcomes...

May 20th, 2022 - At present, there are no clear guidelines as to when antiretroviral therapy for human immunodeficiency virus (HIV) infection should be stopped in the setting of elevated liver enzymes. In large part, this is due to a limited understanding of the natural history of antiretroviral-related hepatotoxicity. Although several antiretrovirals have been reported to cause fatal acute hepatitis, more ofte...

May 20th, 2022 - Background Small cell lung cancer (SCLC) is an aggressive cancer with a poor prognosis. Although highly responsive to chemotherapy initially, SCLC relapses quickly and becomes refractory to treatment within a few months. Extrapulmonary small cell cancers are extremely rare and management of systemic disease with chemotherapy is patterned after the approach used in SCLC. The inability to destroy...

May 20th, 2022 - BMN 190 is a recombinant form of human tripeptidyl peptidase 1 (TPP1), the enzyme deficient in patients with CLN2 diseases (also known as classical late-infantile CLN2, cLINCL, or Jansky-Bielschowsky disease), a form of Batten Disease. As an enzyme replacement therapy (ERT), BMN 190 is designed to help restore TPP1 enzyme activity. BMN 190 is designed to reduce the progressive, pathologic accum...

May 20th, 2022 - This is a dose-escalation and expansion study of ASP1948 as a single agent and in combination with nivolumab or pembrolizumab. The study consists of 3 periods for monotherapy and combination therapy: screening, treatment and follow up, followed by an optional Re-treatment period for participants that qualify. The escalation cohorts will evaluate escalating dose levels of ASP1948 in participants...

May 20th, 2022 - As observed in other immunocompromised individuals with selective or combined T cell deficits, the prevalence and morbidity of human papillomavirus (HPV)-related disease is increased in patients with idiopathic CD4 T cell lymphocytopenia (ICL). The high burden and aggressive clinical course of HPV-associated disease in patients with ICL requires the development of effective preventive measures ...

May 20th, 2022 - Mosquito-borne viruses continue to cause significant global morbidity and mortality, particularly in Southeast Asia. When mosquitoes deliver the virus into the skin of humans while probing for a blood meal, they deposit also saliva, which contains a myriad of pharmacologically active compounds that modulate the host immune system. Most vaccines against vector-borne diseases under development ig...

May 20th, 2022 - Severe aplastic anemia (SAA),myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are life-threatening bone marrow disorders. For SAA patients, long term survival can be achieved with immunosuppressive treatment. However, of those patients treated with immunosuppressive therapy, one quarter to one third will not respond, and about 50% of responders will relapse. Althoug...

May 20th, 2022 - In persons with type 1 diabetes (T1DM), hypoglycemia is the major limiting factor in achieving optimal blood glycemic control. All persons with T1DM are at risk for hypoglycemia (blood glucose level < 70 mg/dl), which is life-threatening and accompanied by serious physical and psychological symptoms, resulting in a profound fear of hypoglycemia (FOH) and reduced quality of life. Young adults wi...

May 20th, 2022 - This study will evaluate the safety and efficacy of JNJ-68284528. The study will include two phases. In Phase1b the study will enroll adults with multiple myeloma with interval assessments for potential dose escalation or de-escalation in subsequent participants. The dose selected at the completion of phase 1b will be used in Phase 2. Following consent, enrolled participants will undergo an aph...

May 20th, 2022 - This is a prospective (the participants are identified and then followed forward in time for the outcome of the study), multicenter (when more than one hospital or medical school team work on a medical research study), post-marketing surveillance ([PMS], surveillance of drugs, devices, appliances, etc., for efficacy or adverse effects, after they have been released for general sale) study. The ...

May 20th, 2022 - We intend to enroll up to 350 subjects with chronic hepatitis C virus (HCV) infection. Subjects will be recruited from two sources: Phase I: treatment naive or experienced who have failed a prior treatment (including DAA-experienced) who are willing to undergo a pre-treatment liver biopsy. Subjects yet to achieve an SVR will receive 12 weeks of therapy with sofosbuvir/velapatasvir (Epclusa ) fi...

May 20th, 2022 - This open label (all participants know the identity of the study drug), multicenter (more than one study site), first-in-human study consists of 2 parts. Part 1 is a Amivantamab Monotherapy and Combination Dose Escalations and Part 2 Amivantamab Monotherapy and Combination Dose Expansions. In Part 1, participants with evaluable NSCLC will be enrolled into cohorts at increasing dose levels of Am...