People's involvement in clinical trials helps researchers uncover better ways to treat, prevent, diagnose, and understand different diseases and medical conditions. DOI: 10.1200/PO.21.00228 JCO Precision Oncology no. -Treatment with the selective small-molecule Gamma-secretase inhibitor Nirogacestat caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early phase clinical trial. Nirogacestat is currently in Phase 2 clinical trials . For more information, contact our Clinical Trials Office at (404) 778-1868. People take part in clinical trials for many reasons. The DeFi (Desmoid/Fibromatosis) trial is a Phase 3 clinical trial designed to evaluate the safety, efficacy and tolerability of an investigational drug called nirogacestat in people over the age of 18 with progressing desmoid tumors. Trial Description: This phase 2 clinical trial will study the effectiveness of nirogacestat in ovarian granulosa cell tumors (OvGCTs). Nirogacestat has been used in trials studying the treatment of Breast Cancer, HIV Infection, Desmoid Tumors, Advanced Solid Tumors, and Aggressive Fibromatosis, among others. LLC Clinical Trial: Janssen Research and Development LLC: More Information. About Nirogacestat. Background. Once participants are eligible to roll into the open-label phase, they will receive nirogacestat. . Nirogacestat (PF-03084014) is a potent, small molecule, selective, reversible, noncompetitive inhibitor of -secretase (GS) with a potential antitumor activity. CheckRare | 2,564 followers on LinkedIn. Leading Publisher and Learning Platform Focused on Rare Diseases. It was granted FDA breakthrough drug designation in September 2019 for adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.. Medical uses []. Know the risks and potential benefits of clinical studies and talk to your health care provider . Nirogacestat. The PBCAR269A clinical trial is being conducted at multiple U.S. sites. Serum Concentrations of Nirogacestat [ Time Frame: Up to 2 year and 5 months ] Serum concentration of nirogacestat will be reported. View Full Description. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Exclusion Criteria. For example, you could advance understanding of a disease and help others like yourself. The FDA has granted nirogacestat (PF-03084014), an investigational gamma-secretase inhibitor, with a breakthrough therapy designation for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. Study subjects in Cohort A will receive PBCAR269A and study subjects in Cohort B will receive PBCAR269A and nirogacestat. This phase 2 clinical trial will study the effectiveness of nirogacestat in ovarian granulosa cell tumors (OvGCTs). A Phase 1b clinical trial evaluating GlaxoSmithKline (GSK)'s belantamab mafodotin, in combination with SpringWorks Therapeutics ' nirogacestat, in people with relapsed or refractory multiple myeloma, has dosed its first patient, SpringWorks announced. Apply to this Phase 2 clinical trial treating Unresectable Desmoid Fibromatosis, Recurrent Desmoid Fibromatosis, Fibroma, Fibromatosis, Aggressive, Fibromatosis Desmoid. Nirogacestat is a gamma secretase inhibitor (GSI) which is hypothesized to decrease the growth and activity of ovarian granulosa tumors. 201 Considered by the investigator to have rapid clinical progression due to melanoma. The main objective of the intervention(s) being evaluated by the clinical trial. Nirogacestat is a gamma secretase inhibitor (GSI) which is hypothesized to decrease the growth and activity of ovarian granulosa tumors. Visit our clinical trials page to find information on all desmoid tumor clinical trials. that the company has entered into a clinical trial collaboration agreement with Pfizer Inc. (NYSE: PFE) to evaluate SpringWorks Therapeutics' investigational gamma secretase . Nirogacestat | C27H41F2N5O | CID 46224413 - structure, chemical names, physical and chemical properties, classification, patents, literature, biological activities . Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the . Summary. DrugBank Accession Number. The collaboration with Pfizer aims to test nirogacestat plus PF06863135 in a Phase 1b/2 . In a recent news release, biopharmaceutical companies AbbVie, Inc. and SpringWorks Therapeutics, Inc. ("SpringWorks") shared that the two had entered into a joint clinical trial collaboration agreement.Through this collaboration, AbbVie and SpringWorks will evaluate a combination treatment of nirogacestat and ABBV-383 for people with relapsed or refractory (R/R) multiple myeloma (MM). Nirogacestat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Nirogacestat has been used in trials studying the treatment of Breast Cancer, HIV Infection, Desmoid Tumors, Advanced Solid Tumors, and Aggressive Fibromatosis, among others. Nirogacestat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. PMID: 34994644 The safety and preliminary effectiveness of this combination are being assessed as a sub . Condition or disease Intervention/treatment . Show: NCT ID. Let's share, debate and celebrate all the new developments that we can bring to our practice. At Winship Cancer Institute, our patients have access to clinical trials for virtually every cancer type. This phase II trial studies the side effects and how well nirogacestat works in treating patients patients less than 18 years of age with desmoid tumors that has grown after at least one form of treatment by mouth or in the vein that cannot be removed by surgery. The main objectives of this study will be:. . Protocol ID. Bernd Kasper, MD, PhD, University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany and Principal Investigator of the phase 3 DeFi clinical trial, discusses the impressive results of the trial, which evaluated nirogacestat in the treatment of desmoid tumors. -The Notch pathway is a key regulator of cell differentiation, proliferation and apoptosis; aberrant signaling via the Notch pathway is associated . Nirogacestat may stop the growth of tumor cells by blocking some of the . The FDA has granted nirogacestat (PF-03084014), an investigational gamma-secretase inhibitor, with a breakthrough therapy designation for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. Nirogacestat showed improved progression-free survival, pain scores, symptoms, and quality of life in patients with progressive desmoid tumors in a phase 3 trial hailed as 'practice changing.' Trade Name: Nirogacestat: Generic: Nirogacestat: Nirogacestat Other Names: Nirogacestat: Type: Formula: C 27 H 41 F 2 N 5 O: Nirogacestat (PF-03084014) is a selective gamma secretase inhibitor developed by SpringWorks Therapeutics that has potential anti-tumor activity. In the double-blind phase, half of the participants will receive nirogacestat while the other half will receive placebo. In the DREAMM-5 Phase I/II platform trial BLENREP (belantamab mafodotin), a BCMA-targeting antibody-drug conjugate, is being evaluated in combination with nirogacestat to determine if the combination can result in similar efficacy and an improved ocular safety profile compared to the currently approved belamaf schedule (single agent dose 2.5 mg . Rejestr bada klinicznych. Purpose. Nirogacestat is a gamma secretase inhibitor (GSI) which is hypothesized to decrease the growth and activity of ovarian granulosa tumors. A.3.1: Title of the trial for lay people, in . Treatment. Find a Clinical Trial 21-147 - ARST1921 - A Study for Treating Desmoid Tumors That . Nirogacestat is an oral, small molecule inhibitor of gamma secretase - in other words, a pill that is designed . Placebo-Controlled, Phase 3 Trial of Nirogacestat Versus Placebo in Adult Patients with Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF). Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the . Physician. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has eight collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities . Get access to cutting edge treatment via Nirogacestat, Questionnaire Administration, Quality-of-Life Assessment. This study evaluates nirogacestat (PF-03084014) in the treatment of desmoid tumor/aggressive fibromatosis (DT/AF). Nirogacestat (PF-03084014) is a potent, small molecule, selective, reversible, noncompetitive inhibitor of -secretase (GS) with a potential antitumor activity. Learn more. Nirogacestat is being investigated for the treatment of desmoid tumors due to its ability to bind to GS, blocking proteolytic activation of Notch receptors. Ricardo Dolmetsch, PhD, President of Research and Development at uniQure, discusses positive data from the low-dose cohort of the ongoing phase 1/2 clinical trial of AMT-130, the first-ever AAV . SpringWorks is conducting a global, randomized, double-blind, placebo-controlled Phase 3 clinical trial (the DeFi trial) to evaluate the efficacy, safety, and tolerability of nirogacestat in adult patients with progressing desmoid tumors (NCT03785964). Generic Name. Participant has known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of nirogacestat (e.g., gastric bypass, lap band, or other gastric procedures that would alter absorption); delivery . ARST1921: A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014) in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors. Nirogacestat (PF-03084014) is a selective gamma secretase inhibitor developed by SpringWorks Therapeutics that has potential anti-tumor activity. Published online October 6, 2021. SpringWorks will now take the gamma-secretase inhibitor The ESMO Congress 2022 presented the very latest advances in the treatment of cancer and offered an excellent educational programme, with opportunities for an exchange of ideas among delegates through an enhanced onsite and virtual experience. In September 2022, positive data from the Phase 3 DeFi trial were presented at the ESMO . About Nirogacestat. Individual Patient Compassionate Use of Nirogacestat. ICH GCP. Use the filters below to find active studies currently open for enrollment. 1 The breakthrough designation was granted as a result of positive findings seen in phase I and II trials of nirogacestat monotherapy . This is a Phase 1/2a, nonrandomized, open-label, parallel assignment, single-dose, dose-escalation, and dose-expansion study to evaluate the safety and clinical activity of PBCAR269A, with or without nirogacestat, in adults with r/r MM. Nirogacestat. Apply to this Phase 3 clinical trial treating Fibroma, Fibromatosis, Aggressive, Desmoid Tumors, Aggression. The Real Story of Good Health ~ in 120 Seconds or Less. 202 Stage IVM1d with greater than 3 cerebral melanoma metastases, or clinically active cerebral melanoma metastases requiring therapy, and/or carcinomatous meningitis regardless of clinical stability. . 1 The breakthrough designation was granted as a result of positive findings seen in phase I and II trials of nirogacestat monotherapy . Approximately 4 sites will be participating, each one contributing 35 patients to the study dataset. Phase III Trial for Nirogacestat A new company named SpringWorks Therapeutics has been formed as a spinoff of Pfizer to focus on investigational therapies that hold significant promise for underserved populations. Our peer-to-peer content is a trusted source . Interventional study (clinical trial) studies new tests, treatments, drugs, surgical procedures or devices. Free dental implants clinical trials near me 2022. accident on hwy 200 yesterday Fiction Writing. Our platform allows users to gain insight and learn about rare diseases, current and emerging therapies, clinical trials, and the BioPharma companies behind them. Get access to cutting edge treatment via Nirogacestat oral tablet, Placebo Oral Tablet. View duration, location, compensation, and staffing details. Study Type The nature of the investigation or investigational use for which clinical study information is being submitted. Nirogacestat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Your dentist may go for the tooth extraction and they firstly numb the area to ease the moth and other processes followed by the dentists. 5 (2021) 1579-1583. Nirogacestat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. The tooth extraction may come between $75 to $200 per tooth. . View duration, location, compensation, and staffing details. Celem tego badania jest finansowane, czy te leczenie skojarzone z badanymi lekami belantamab mafodotyna i nirogacestat do bezpieczne leczenie dla . ARST1921 - A Safety, Pharmacokinetic and Efficacy Study of a -Secretase Inhibitor, Nirogacestat (PF-03084014; IND# 146375), in Children and Adolescents with Progressive, Surgically Unresectable Desmoid Tumors Desmoid Tumors that cannot be removed by surgery . You may also receive a new research . This study aims to enroll a total of 140 patients (140 implants ) who are in need of treatment with dental implants in one or more edentulous areas of the maxilla and/or mandible. SpringWorks Therapeutics announced new collaborations with two companies, Pfizer and Janssen, to investigate its oral therapy candidate nirogacestat in combination with BCMA-targeting agents to treat people with relapsed or refractory multiple myeloma. Number of Participants with Presence of Anti-Drug Antibodies to Teclistamab [ Time Frame: Up to 2 year and 5 months ] . In addition to its ongoing clinical collaborations with BCMA-directed therapies, SpringWorks is conducting a global Phase 3, double-blind, randomized, placebo-controlled clinical trial (the DeFi . In addition, a Phase 3 clinical trial, DeFi, is currently in progress for nirogacestat for adults with desmoid tumors and aggressive fibromatosis. Trial Name. DB12005. This drug entry is a stub and has not been fully annotated. Full Title A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014, IND 146375), in Children and Adolescents with Progressive, Surgically Unresectable Desmoid Tumors (ARST1921) (CIRB) Purpose The purpose of this study is to assess the safety and effectiveness of the investigational drug nirogacestat in children and adolescents with desmoid tumors that . The first patient was dosed in a phase 2 trial (NCT05348356) evaluating nirogacestat, an investigational gamma secretase inhibitor, as a monotherapy in patients with recurrent ovarian granulosa cell tumors, according to SpringWorks Therapeutics, Inc. 1 "The science behind this study is exciting because Notch signaling, a key target of gamma secretase inhibitors, is involved in the expression . This phase II trial studies the side effects and how well nirogacestat works in treating patients patients less than 18 years of age with desmoid tumors that has grown after at least one form of treatment by mouth or in the vein that cannot be removed by surgery. Nirogacestat is being investigated for the treatment of desmoid tumors due to its ability to bind to GS, blocking proteolytic activation of Notch receptors. 2145- 10.17.2022). The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. ARST1921 NCI-2019-07498 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) ) ARST1921 ( Other Identifier: Children's Oncology Group ) ARST1921 ( Other Identifier: CTEP ) U10CA180886 . SpringWorks is evaluating nirogacestat as a BCMA potentiator and has eight collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities . | CheckRare is a learning platform for health care professionals and patients. This phase 2 clinical trial will study the effectiveness of nirogacestat in ovarian granulosa cell tumors (OvGCTs). Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 . Desmoid tumors can grow anywhere on the body into surrounding healthy tissues, including joints, muscle, and viscera. It is scheduled to be annotated soon. AFLDS.org with Dr. Peterson Pierre presents Daily Dose: 'Title IX: What Parents Need to Know?' (Ep. , in been evaluated by the clinical Trial - Emory University < /a > About nirogacestat and. A.3.1: Title of the study sponsor and investigators signaling via the Notch is! 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