Trophy trial nejm

Open-label, dose-escalation clinical trial of AVXS injected intravenously through a peripheral limb vein. Short-term safety will be evaluated over a two year period. Patients will be tested at baseline and return for follow up visits on days 7, 14, 21, 30, followed by once every month through 12 months post dose, and then every three months through two 2 years post infusion.

Unscheduled visits may occur if the PI determines that they are necessary. The primary analysis for efficacy will be assessed when all patients reach A follow-up safety analysis will be completed at the time point at which the last patient reaches 24 months post-dose. Upon completion of the 2-year study period, patients will be monitored annually as per standard of care for up to 15 years. Other Outcome Measures: Number of Participants With Assessed Improvement in Motor Function [ Time Frame: 24 months post-dose ] Improvement in motor function was determined by achievement of developmental milestones, specifically achievement of ability to sit unassisted for at least 30 seconds, determined by physical therapist and confirmed by an independent central video reviewer.

Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies. Six or nine months of age and younger depending on cohort on day of vector infusion with Type 1 SMA as defined by the following features:. Try the modernized ClinicalTrials.

Learn more about the modernization effort. Hide glossary Glossary Study record managers: refer to the Data Element Definitions if submitting registration or results information.

Search for terms. Save this study. Warning You have reached the maximum number of saved studies Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U. Federal Government.

Read our disclaimer for details. Results First Posted : May 10, Last Update Posted : June 14, Study Description. The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS as a treatment of spinal muscular atrophy Type 1 SMN1. Detailed Description:. Resource links provided by the National Library of Medicine MedlinePlus Genetics related topics: Spinal muscular atrophy Spinal muscular atrophy with respiratory distress type 1.

The effect of reducing blood pressure in this population has not been established. An estimated 65 million Americans one in three adults have hypertension or high blood pressure. Lifestyle changes, such as weight loss, exercise, stopping smoking, and dietary adjustments can help reduce elevated blood pressure, and there are a number of classes of antihypertensive medications for those individuals who require drug treatment.

The recommended initial dose for treating heart failure is 4 mg once daily. The target dose is 32 mg once daily, which is achieved by doubling the dose at approximately 2-week intervals, as tolerated by the patient. These conditions should be corrected prior to administration of ATACAND, or the treatment should start under close medical supervision.

Caution should be observed for hypotension when initiating therapy. Evaluation of patients with heart failure should always include assessment of renal function and volume status. Monitoring of blood pressure, serum creatinine, and serum potassium is recommended during dose escalation and periodically thereafter.

The adverse-event profile of ATACAND in heart failure patients was consistent with the pharmacology of the drug and the health status of the patients. AstraZeneca is a major international healthcare business engaged in the research, development, manufacture and marketing of prescription pharmaceuticals and the supply of healthcare services.

Our aim is to replicate these practice-changing findings in other tumor types where patients are in need of effective treatment options.

Additional evaluation across multiple solid tumors is also underway. About Trodelvy Trodelvy sacituzumab govitecan-hziy is a first-in-class antibody and topoisomerase inhibitor conjugate directed to the Trop-2 receptor, a protein frequently expressed in multiple types of epithelial tumors, including metastatic triple-negative breast cancer TNBC and metastatic urothelial cancer UC , where high expression is associated with poor survival and relapse.

Important Safety Information for Trodelvy. Neutropenia: Severe, life-threatening, or fatal neutropenia can occur and may require dose modification.

One patient had intestinal perforation following diarrhea. Neutropenic colitis occurred in 0. At onset, evaluate for infectious causes and if negative, promptly initiate loperamide, 4 mg initially followed by 2 mg with every episode of diarrhea for a maximum of 16 mg daily.

Discontinue loperamide 12 hours after diarrhea resolves. Additional supportive measures e. Patients who exhibit an excessive cholinergic response to treatment can receive appropriate premedication e. Severe signs and symptoms included cardiac arrest, hypotension, wheezing, angioedema, swelling, pneumonitis, and skin reactions. The incidence of anaphylactic reactions was 0. Pre-infusion medication is recommended.

Observe patients closely for hypersensitivity and infusion-related reactions during each infusion and for at least 30 minutes after completion of each infusion.