4 Solidarnosti Pr., St. Petersburg, Russian Federation
Real-World Outcomes of Patients With Metastatic Breast Cancer After Treatment With First-Line ET+CDK4/6i
This analysis will assess real-world outcomes in HR+/HER2- mBC patients who initiated a subsequent line of therapy (LOT) (i.e., 2L) after progressing on 1L treatment with ET+CDK4/6i. This study will also characterize the treatment patterns, clinical characteristics, and outcomes in this patient population.
Phase
N/ASpan
7 weeksSponsor
Daiichi SankyoNew York, New York
Recruiting
Primary Connections for Youth and Families
This randomized effectiveness trial compares a standard adolescent-only approach (SBIRT-A-Standard) versus a family-based approach (SBIRT-A-Family) in which caregivers are systematically included in screening, intervention, and referral activities. The study includes N = 2,300 adolescents (age 12 - 17) and their caregivers attending one of three hospital-affiliated pediatric settings serving diverse patients in major urban areas. Study recruitment, initial screening, randomization, and all SBIRT-A activities occur during a single pediatric visit. SBIRT-A procedures are delivered primarily in digital format on hand-held tablets using both patient-facing and provider-facing programming. Primary outcomes (AOD use, co-occurring behavior problems, parent-youth communication about AOD use) and secondary outcomes (adolescent quality of life, therapy attendance) are assessed at screen/initial and 3, 6, 9, and 12 months follow-up. The study is well powered to conduct all planned main and moderator (age, sex, race/ethnicity, youth AOD risk status) analyses.
Phase
N/ASpan
196 weeksSponsor
The National Center on Addiction and Substance Abuse at Columbia UniversityNew York, New York
Recruiting
Healthy Volunteers
A Tissue Collection Study in Patients Who Respond to Immune Checkpoint Inhibitors to Identify Targets of Tumor-Reactive T Cells.
Phase
N/ASpan
200 weeksSponsor
TScan Therapeutics, Inc.New York, New York
Recruiting
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible.1-3 Prior to formation of the Undiagnosed Diseases Network (UDN), the UDP had evaluated 3300 medical records, admitted 750 individuals with rare and undiagnosed conditions to the NIH, and identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the UDN, for fiscal years 2013-2022. The clinical sites perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN is furthering the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases.
Phase
N/ASpan
694 weeksSponsor
National Human Genome Research Institute (NHGRI)New York, New York
Recruiting
Healthy Volunteers
Efficacy and Safety of Extended TARPEYO® Treatment Beyond 9 Months in Adult Patients With Primary IgA Nephropathy
This clinical trial will investigate the efficacy and safety of TARPEYO® treatment extended for an additional 15 months in adult IgAN participants who have completed their 9-month TARPEYO® 16 mg QD commercial treatment regimen. Participants with residual proteinuria will be eligible for enrollment. The Treatment Period will consist of a 6-month Treatment Period with TARPEYO® 16 mg QD, followed by a 9-month Treatment Period with TARPEYO® 8 mg QD. This will be followed by a 3-month Follow-up Period, which includes the first 2 weeks of Tapering Period with TARPEYO® 4 mg QD. The overall aim of the extended treatment is to improve and maintain the treatment effect with reduced proteinuria and protection of kidney function over a total of 2 years of TARPEYO® treatment.
Phase
4Span
154 weeksSponsor
Calliditas Therapeutics ABNew York City, New York
Recruiting
Prospective Randomized Study of Accelerated Radiation Therapy (PRART)
Phase
3Span
536 weeksSponsor
Weill Medical College of Cornell UniversityNew York, New York
Recruiting
The ENCIRCLE Trial
This is a prospective single-arm, multicenter study.
Phase
N/ASpan
538 weeksSponsor
Edwards LifesciencesNew York, New York
Recruiting
Ocrelizumab Discontinuation in Relapsing Multiple Sclerosis
This study is a prospective, multi-center, randomized, double blinded, placebo-controlled study of OCR treatment-discontinuation in patients with early RMS. All eligible participants will be initiated on OCR using the standard approved administration schedule of two 300 mg infusions separated by 14 days (i.e., Days 0 and 14) for a total of 600 mg, followed by 600 mg infusions at Month 6, 12, 18, and 24. At Month 24, participants will be randomized (2:1) to one of two Arms with randomized treatment beginning at Month 30: Arm 1: placebo infusions every 6 months; Arm 2: OCR infusions every 6 months. The treatment period will be for a total of 48 months. The study will consist of the following periods, with participants undergoing study assessments, including physical exams, neurological exams, EDSS, LCLA, SDMT, MFIS, MSQOL-54, PHQ9, EQ-5D-5L, and frequent co-registered research-quality MRI to sensitively assess for new inflammatory disease activity (new or enlarging T2 lesions), under schedules described in detail below. Biological sampling includes blood samples for functional immune profiling and stool samples for microbiome studies. Biomarker-based assessments are to include but not be limited to NfL and B cell levels. Screening Period: After obtaining informed consent, all screening assessments, and procedures to establish eligibility will be performed. These may be completed during one or more study visits within the 30-day screening window. Biological sampling will include 2 samples obtained prior to initiation of OCR treatment. Open-label Treatment Period: Participants that meet all eligibility criteria for enrollment will be initiated on OCR using the standard approved administration and will be followed for 24 months under the standard of care clinical efficacy and safety monitoring described below. Biological sampling occurs at 6, 12, 18, and 24 months as well as prior to the 12 and 24 months visits (14 days +/- 7 days of the visit). Participants who discontinue study therapy during the open-label treatment period are asked to return for an early withdrawal visit and a safety follow-up phone call approximately 6 months after their last dose. Blinded Treatment Period: At Month 24, participants will be randomized (2:1) to either: (Arm 1) placebo infusions every 6 months; or (Arm 2) continue with OCR infusions every 6 months. The blinded treatment period will extend to Month 48 or until new disease activity is observed. All participants in the blinded treatment period will be closely monitored clinically and with frequent research MRIs using a standard protocol that assesses for the development of any new inflammatory disease activity observed following randomization. The development of such new inflammatory disease activity will represent the primary study endpoint. It will be defined as one or more of the following: (1) one or more new clinical relapse(s) (see relapse definition) or (2) MRI evidence on frequent (every 3 months) serial scans of new disease activity. The central imaging analysis will identify incident new or enlarging MRI T2 lesions as compared to prevalent brain lesions documented in each participant from time of randomization (Month 24) and thereafter. Frequent biological sampling will include blood samples collected every 3 months and stool samples collected every 6 months through the 48-month end of study. Unblinded Follow-up period: For participants who discontinue blinded study therapy before Month 48, including those meeting the primary endpoint by manifesting new disease activity following randomization as defined above, blinded treatment allocation will be revealed and discussed, as described in section 3.5.1. Participants may choose to receive OCR or other treatment and are encouraged to continue with study assessments per the schedule of events. For participants that meet the primary endpoint, OCR will be provided by the study for those who choose to receive OCR after unblinding and continue with study assessments per the schedule of events. Functional immune response profiles of reconstituting B cells as well as non-B cells (T cells and myeloid cells) will be compared between participants who do, versus those do not, benefit from durable remission of relapsing biology, to identify cellular immune response profiles that may underlie the state of durable tolerance versus lack of durable tolerance. Safety Follow-up: Participants who are treated with OCR through Month 48 will have a phone visit 6 months after their last dose. If there are safety concerns, the participant can be brought in for an unscheduled visit.
Phase
4Span
290 weeksSponsor
National Institute of Allergy and Infectious Diseases (NIAID)New York, New York
Recruiting
Precision-Based Genomics in Prostate Cancer
Background: - Prostate cancer is the most common cancer and the second leading cause of death in males in the United States with an estimated 191,930 new cases and 33,330 deaths in 2020. - There has been progress in identifying established risk factors for the development of prostate cancer, including genetic predisposition. The study of the molecular genetics of prostate cancer has identified pathogenic variants, such as BRCA1 and BRCA2 (associated with hereditary breast and ovarian cancer syndrome), HOXB13 (associated with hereditary prostate cancer), and DNA mismatch repair (MMR) gene variants (MLH1, MSH2, MSH6, PMS2, and EPCAM) associated with Lynch syndrome. - While our understanding of molecular genetics continues to grow, there remains a need to identify additional germline and somatic mutations and alterations that may increase an individual s risk to develop prostate cancer and potentially the aggressiveness of the disease. In studying the following alterations in prostate cancer, in both localized and advanced stages, potential expanded molecular findings may lead to actionable therapeutic targets and biomarker development. A better understanding of molecular genetics in a longitudinal study of subjects with prostate cancer may be helpful for the design of future treatment studies, and to develop a better understanding of the natural history of the disease Objectives: - To longitudinally evaluate subjects with prostate cancer with known germline and/or somatic variants in PIK3 and/or AKT, PALB2, BRIP1, RAD50, RAD51, RAD54, RB1, SPOP, Wnt/B-catenin pathway, CDK12, and MMR genes: MLH1, MSH2, MSH6, PMS2, and EPCAM to better understand the natural history of the disease. - To longitudinally evaluate subjects with tumor mutational burden-high (TMB-H) prostate cancer (greater than or equal to 10 mutations/megabase [mut/Mb] or blood TMB (bTMB) [greater than or equal to16 mut/Mb]). Eligibility: - Subjects with histologically confirmed prostate cancer - Must have known germline and/or somatic variants in PIK3 and/or AKT, PALB2, BRIP1, RAD50, RAD51, RAD54, RB1, SPOP, Wnt/B-catenin pathway, CDK12, and MMR genes: MLH1, MSH2, MSH6, PMS2, and EPCAM and/or TMB-high or be deemed an exceptional responder. NOTE: any platform for genomics testing is acceptable (research or CLIA-certified) - Age greater than or equal to 18 years old Design: - This will be a long-term multi-center study to comprehensively study participants with prostate cancer. - Participants will provide clinical information (including medical history, clinical tests, imaging studies and reports, surgical pathology reports, genetic test results). - Since long-term follow-up of individuals with prostate cancer is a major feature of the study, local sites intend to maintain active contact with study subjects for as long as possible. Participants will be followed throughout the course of their illnesses, with particular attention to patterns of disease recurrence and progression, response to therapies and duration of responses.
Phase
N/ASpan
303 weeksSponsor
National Cancer Institute (NCI)New York, New York
Recruiting
MILD® Percutaneous Image-Guided Lumbar Decompression: A Medicare Claims Study
In this study the treatment group will include all patients receiving MILD, and the control group will include all patients receiving IPD for the treatment of LSS during the enrollment period. Reoperation and harms data will be studied for the MILD and IPD procedures for a 24-month follow-up period after the index procedure using Medicare claims data. This study is exempt from IRB oversight (Department of Health and Human Services regulations 45 CFR 46) and does not require prior enrollment nor patient consent. The inclusion of the study's NCT number on MILD Medicare claims is required and results in enrollment.
Phase
N/ASpan
512 weeksSponsor
Vertos Medical, Inc.Brooklyn, New York
Recruiting