Therapie des tripel--negativen Mammakarzinoms in Sicht?

2010 ◽  
Vol 01 (01) ◽  
pp. 16-16
Author(s):  
Alexander Kretzschmar
Keyword(s):  
Drug Administration ◽  
Phase Ii ◽  
Food And Drug Administration ◽  
Phase Iii

Nach positiven Phase-II-Daten ist jetzt für den PARP-Hemmer BSI-201 eine Phase-III-Studie angelaufen, in der die Wirksamkeit und Sicherheit in der Therapie des tripelnegativen Mammakarzinoms untersucht wird. Die US-amerikanische Food and Drug Administration (FDA) hat ein beschleunigtes Prüfverfahren bewilligt.

Novel agents for myelodysplastic syndromes

2021 ◽  
pp. 107815522110379
Author(s):  
Katie Xu ◽  
Elizabeth Hansen
Keyword(s):  
Drug Administration ◽  
Myelodysplastic Syndromes ◽  
Primary Outcome ◽  
Food And Drug Administration ◽  
Review Article ◽  
Phase Iii ◽  
Phase Iii Trial ◽  
Safety And Efficacy ◽  
Transfusion Independence ◽  
The Us

Review objective There have been several advances in the field of myelodysplastic syndromes over the past year, yielding two new US Food and Drug Administration drug approvals. The pharmacology, pharmacokinetics, clinical trials, therapeutic use, adverse effects, clinical use controversies, product description, and upcoming trials for myelodysplastic syndromes novel agents luspatercept-aamt and decitabine/cedazuridine are reviewed. Data sources This review article utilized primary information obtained from both the published studies involved in the approval of luspatercept-aamt and decitabine/cedazuridine and package inserts for the respective medications. This review article utilized secondary information obtained from National Comprehensive Cancer Network guidelines using filters and keywords to sustain information relevancy as well as key studies using the keywords, “luspatercept-aamt, myelodysplastic syndromes, decitabine, cedazuridine, hypomethylating agent, ASTX727” from scholarly journal database PubMed. Data summary Myelodysplastic syndromes consist of myeloid clonal hemopathies with a diverse range of presentation. Until recently, there have been relatively few new therapies in the myelodysplastic syndromes treatment landscape. On April 3, 2020 the US Food and Drug Administration approved Reblozyl®(luspatercept-aamt), then on July 7, 2020, the US Food and Drug Administration approved INQOVI® (decitabine and cedazuridine). Luspatercept-aamt acts as a erythroid maturation agent through differentiation of late-stage erythroid precursors. The safety and efficacy of luspatercept-aamt was demonstrated in the MEDALIST trial, a phase III trial in patients with very low-intermediate risk refractory myelodysplastic syndromes and ring sideroblasts. Luspatercept-aamt met both primary and secondary endpoints of transfusion independence of 8 weeks or longer and transfusion independence of 12 weeks or longer, respectively. Decitabine/cedazuridine has a unique mechanism of action in which decitabine acts as a nucleoside metabolic inhibitor promoting DNA hypomethylation and cedazuridine then prevents degradation of decitabine. The safety and efficacy of decitabine/cedazuridine was shown in the ASCERTAIN study, a phase III trial in patients with intermediate or high risk myelodysplastic syndromes or chronic myelomonocytic leukemia. The primary outcome evaluated was 5-day cumulative area under the curve between decitabine/cedazuridine and IV decitabine as well as additional outcomes including safety. Decitabine/cedazuridine met primary outcome and had a similar safety profile to IV decitabine. Conclusion The novel myelodysplastic syndromes agents luspatercept-aamt and decitabine/cedazuridine provide a clinical benefit in the studied populations.

scholarly journals Anwendung von Apremilast bei moderater bis schwerer atopischer Dermatitis bei Kindern

2017 ◽  
Vol 5 (2) ◽  
pp. 103-106
Author(s):  
Rachael C Saporito ◽  
David Cohen
Keyword(s):  
Drug Administration ◽  
Phase Ii ◽  
Food And Drug Administration ◽  
Atopische Dermatitis ◽  
Immunglobulin E ◽  
Symptomatische Behandlung

Die atopische Dermatitis (AD) ist eine chronische, pruritische Hauterkrankung, die häufig durch eine bakterielle Superinfektion erschwert wird und von der 10,7% der US-amerikanischen Kinder betroffen sind. Zur Pathogenese gehört ein Zusammenbruch der Hautbarriere neben einer Dysfunktionalität sowohl der angeborenen als auch der adaptiven Immunantwort, einschließlich einer unausgewogenen Zunahme von T-Helferzellen vom Typ 2 (Th2-Zellen) und einer Hyperimmunglobulinämie E. Die erhöhte Anzahl an Th2-Zellen führt durch die Freisetzung von Interleukin (IL)-4, IL-5 und IL-13 zur Produktion von Immunglobulin E und zur Eosinophilie und durch die Freisetzung von IL-10 zu einer Verminderung des Schutzes vor einer bakteriellen Superinfektion. Die gegenwärtig von der Food and Drug Administration (FDA) zugelassene symptomatische Behandlung der AD umfasst topische Salben, topische und systemische Kortikosteroide, topische immunmodulierende Therapien, Antibiotika sowie Phototherapie. Es gibt jedoch keine zugelassenen gezielten Therapien oder Heilmethoden. Der hier vorgestellte Fallbericht eines 8-jährigen afroamerikanischen Jungen unterstützt eine neuartige Therapie der moderaten bis schweren AD mit Apremilast, einem Phosphodiesterase-Typ-4-Inhibitor. Kürzlich wurde eine klinische Phase-II-Studie (NCT02087943) zu Apremilast für die Behandlung von AD bei Erwachsenen abgeschlossen. Dieser Fallbericht veranschaulicht das Potenzial von Apremilast als AD-Therapie für Kinder, bei denen ein großer Bedarf an sicheren und wirksamen Medikationen besteht. Übersetzung aus Case Rep Dermatol 2016;8:179-184 (DOI: 10.1159/000446836)

Strategies to overcome barriers to accrual (BtA) to NCI-sponsored clinical trials: A project of the NCI-Myeloma Steering Committee Accrual Working Group (NCI-MYSC AWG).

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 8592-8592
Author(s):  
Matthias Weiss ◽  
Morie A. Gertz ◽  
Richard F. Little ◽  
Vincent Rajkumar ◽  
Susanna J. Jacobus ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Phase Ii ◽  
Working Group ◽  
Treatment Options ◽  
Insurance Coverage ◽  
Scientific Progress ◽  
Phase Iii ◽  
Eligibility Criteria ◽  
Ct Protocol

8592 Background: Accrual to NCI clinical trials(CT)is often slower than planned at times mandating premature closure resulting in loss of valuable resources and delay of scientific progress. Methods: The NCI-MYSC AWG identified 10 potential BtA. SWOG, ECOG and Alliance investigators were queried and agreed that these barriers impede accrual (results stratified for academic and community sites). The MYSC AWG developed in collaboration with NCI and FDA strategies to overcome these barriers. Results: Strategies listed for the 3 most often cited BtA: 1. Reimbursement for CT related expenses:increase awareness of improved reimbursement for phase II CT; tailor reimbursement according to CT complexity; request funds from industry and other sources ( http://biqsfp.cancer.gov ) for qualifying ancillary CT components. 2. Spectrum of available treatment options influences CT participation: educate patients and providers about the significance of a new CT using social media, presentations at national meetings and by adding educational material to CT protocol; encourage opinion leaders and advocacy groups not to promote a new therapy as “standard” in the absence of phase III data. 3. Requirement of CT specific therapy at NCI designated sites only: “MYSC AWG Drug Administration Table” describes NCI/FDA approved rules for CT specific drug administration; CT protocol will outline which standard treatment components of a CT can be administered at any site as long as protocol specific guidelines are followed and conduct is supervised by enrolling investigator. Examples of additional strategies to overcome identified BtA: determine feasibility, indication and insurance coverage of CT specific tests during protocol development; discourage narrow eligibility criteria; avoid competing CT; allow up to 1 cycle of commercially available therapy prior to enrollment; CIRB support for phase II CT. Conclusions: The MYSC Accrual Working Group developed in collaboration with NCI and FDA strategies to overcome barriers to myeloma clinical trial accrual. These strategies may be applicable to NCI-sponsored clinical trials evaluating interventions in other diseases.

scholarly journals Predicting Successful Phase Advancement and Regulatory Approval in Multiple Myeloma From Phase I Overall Response Rates

2017 ◽  
pp. 1-14 ◽  
Author(s):  
Ehsan Malek ◽  
Caner Saygin ◽  
Rebecca Ye ◽  
Fahrettin Covut ◽  
Byung-Gyu Kim ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Drug Development ◽  
Phase I ◽  
Drug Administration ◽  
Phase Ii ◽  
Food And Drug Administration ◽  
Regulatory Approval ◽  
Phase I Trials ◽  
Overall Response ◽  
Investigational Agents

Purpose Drug development in oncology is resource intensive, time consuming, and frequently unsuccessful. Here, we hypothesized that therapeutic benefit of published phase I studies of antimyeloma investigational agents was associated with advancement to phase II and future regulatory approval. Patients and Methods Seventy four phase I trials that treated patients with multiple myeloma (n = 2,408) conducted from 2004 to 2015 were analyzed to assess drug safety, efficacy, phase advancement, and regulatory approval. Results The median overall response rate (ORR) for all single-agent trials evaluated was 13.2%. However, the ORR in trials that advanced to phase II was 19%, whereas it was only 4% in trials that failed to advance. The median ORR was 23% for trials testing agents that were ultimately approved by the US Food and Drug Administration compared with only 8% for trials testing agents that were not approved (hazard ratio, 2.21; 95% CI, 2.01 to 2.61; P = .012). Importantly, the absolute number of phase I trials in multiple myeloma, but not the success rate, significantly increased over the period studied. The proportion of industry-sponsored trials also steadily increased over that same period. The ratio of initial dose to maximum tolerated dose was 0.29, suggesting that many patients were undertreated. Conclusion Investigational agents with higher ORRs in phase I trials were more likely to advance to phase II trials and achieve US Food and Drug Administration approval. Our results suggest that designing phase I trials to maximize the antimyeloma efficacy of a given compound may lead to more successful and cost-effective drug development.

INSIDE INDUSTRY

2015 ◽  
Vol 19 (06) ◽  
pp. 35-39
Keyword(s):  
Bladder Cancer ◽  
Drug Administration ◽  
Rare Diseases ◽  
Oncolytic Virus ◽  
Food And Drug Administration ◽  
Phase Iii ◽  
Cancer Drugs ◽  
Research Risks ◽  
Lost Decade

Two Promising Phase III Cancer Drugs: SIRT & Afatinib CAVATAK™ Presentations at Oncolytic Virus Therapeutics Conference Reinforce Potential in Bladder Cancer and Melanoma Receptos subjected to M&A Speculation Antibiotics Research Risks another Lost Decade while Rare Diseases Research Steams Ahead, Global Patent Study Reveals Intertek gains Official Accreditation with Philippines Food and Drug Administration

Changing the Paradigm in the Treatment of Platinum-Sensitive Recurrent Ovarian Cancer: From Platinum Doublets to Nonplatinum Doublets and Adding Antiangiogenesis Compounds

2009 ◽  
Vol 19 (Suppl 2) ◽  
pp. S63-S67 ◽  
Author(s):  
Bradley J. Monk ◽  
Robert L. Coleman
Keyword(s):  
Ovarian Cancer ◽  
Drug Administration ◽  
Liposomal Doxorubicin ◽  
Food And Drug Administration ◽  
Pegylated Liposomal Doxorubicin ◽  
Recurrent Ovarian Cancer ◽  
Phase Iii ◽  
Clinical Activity ◽  
Platinum Sensitive ◽  
The Us

Objectives:The optimal treatment for women with recurrent epithelial ovarian cancer is evolving. The objective of this review is to outline the transition away from platinum doublets toward nonplatinum combinations and review emerging data on antiangiogenesis therapy in this setting.Materials and Methods:Recently published and presented data as well as ongoing clinical trials are discussed.Results:Current clinical practice largely harmonizes with a paradigm that outlines a treatment algorithm for recurrent ovarian cancer based on the duration of platinum-free exposure. In this model, patients whose penultimate platinum compound exposure (platinum-free interval [PFI]) is longer than 6 months are generally offered a platinum agent or a platinum-containing doublet; those with a shorter interval are usually treated with a single nonplatinum agent. This is based on the simple contention that better clinical outcomes will be realized with platinum in those deemed platinum sensitive (PFI >6 months). However, it is becoming clear from various phase II and phase III clinical studies that the performance of many nonplatinum chemotherapeutic agents is also influenced by this parameter (PFI). Indeed, although definitive comparisons of nonplatinum drugs to novel cytotoxic agents are lacking, the clinical activity of these compounds might approach or exceed that of platinum agents. Although recognized by clinicians, the dichotomy that determines therapy based on PFI has not been formally accepted by the US Food and Drug Administration in all cases of drug labeling. For instance, whereas the combination of gemcitabine and carboplatin is now approved for patients with platinum-sensitive recurrent ovarian cancer, traditionally used platinum-resistant (PFI <6 months) agents such as topotecan and pegylated liposomal doxorubicin are also approved by the US Food and Drug Administration as single agents in platinum-sensitive patients. Furthermore, the nonplatinum doublet pegylated liposomal doxorubicin and trabectedin has recently documented comparable activity to platinum combinations among patients with a PFI of longer than 6 months. To that end, the most prolific developmental therapeutics arena in ovarian cancer is biologically targeted therapy, particularly angiogenesis inhibitors. Although it is unknown if the clinical activity from these new agents will respect the chemotherapy-sensitive dichotomy, it is clear that they have the potential to augment efficacy, possibly in both traditionally chemosensitive and chemoresistant phenotypes.Conclusions:The term platinum sensitive should probably be replaced by chemotherapy sensitive, particularly as new nonplatinum agents and combinations are identified as active in this setting. Nonplatinum doublets are effective in treating platinum-sensitive recurrent disease, and adding antiangiogenesis agents to these combinations is a research priority.

scholarly journals THE STATE OF THE NATION: A 50-STATE COVID-19 SURVEY REPORT #9: WILL AMERICANS VACCINATE THEMSELVES AND THEIR CHILDREN AGAINST COVID-19?

2020 ◽  
Author(s):  
Roy H. Perlis ◽  
David Lazer ◽  
Katherine Ognyanova ◽  
Matthew Baum ◽  
Mauricio Santillana ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
District Of Columbia ◽  
The State ◽  
Phase Iii ◽  
Phase Iii Clinical Trials ◽  
Survey Report ◽  
The U.S

At least 5 companies have launched Phase III clinical trials of COVID-19 vaccines, the final step before seeking approval from the U.S. Food and Drug Administration (FDA). According to NIAID director Anthony Fauci, vaccines may be widely available in the U.S. by spring 2021 if these trials are successful.But should these vaccines become available, will Americans accept them? Between July 10 and July 26, we surveyed 19,058 adults in all 50 U.S. states and the District of Columbia. We asked about the likelihood that they would seek vaccination for themselves, and for their children. We also asked about the factors that would influence their decision making.We find that, overall, 66% of adults would be somewhat or extremely likely to vaccinate themselves; 66% would be somewhat or extremely likely to vaccinate their children. These rates vary markedly between states, as shown on the figure below.

Bewegungstherapie und körperliche Aktivität bei Patienten mit Herzinsuffizienz

Praxis ◽  
2018 ◽  
Vol 107 (17-18) ◽  
pp. 951-958 ◽  
Author(s):  
Matthias Wilhelm
Keyword(s):  
Phase I ◽  
Phase Ii ◽  
Körperliche Aktivität ◽  
Phase Iii

Zusammenfassung. Herzinsuffizienz ist ein klinisches Syndrom mit unterschiedlichen Ätiologien und Phänotypen. Die überwachte Bewegungstherapie und individuelle körperliche Aktivität ist bei allen Formen eine Klasse-IA-Empfehlung in aktuellen Leitlinien. Eine Bewegungstherapie kann unmittelbar nach Stabilisierung einer akuten Herzinsuffizienz im Spital begonnen werden (Phase I). Sie kann nach Entlassung in einem stationären oder ambulanten Präventions- und Rehabilitationsprogramm fortgesetzt werden (Phase II). Typische Elemente sind Ausdauer-, Kraft- und Atemtraining. Die Kosten werden von der Krankenversicherung für drei bis sechs Monate übernommen. In erfahrenen Zentren können auch Patienten mit implantierten Defibrillatoren oder linksventrikulären Unterstützungssystemen trainieren. Wichtiges Ziel der Phase II ist neben muskulärer Rekonditionierung auch die Steigerung der Gesundheitskompetenz, um die Langzeit-Adhärenz bezüglich körperlicher Aktivität zu verbessern. In Phase III bieten Herzgruppen Unterstützung.

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