Search results for "Extension study"

showing 6 items of 6 documents

Open-label extension study of the RNAi therapeutic ALN-VSP02 in cancer patients responding to therapy.

2012

3062 Background: ALN-VSP02 is an RNA interference (RNAi) therapeutic comprised of lipid nanoparticle-formulated small interfering RNAs targeting vascular endothelial growth factor (VEGF)-A and kinesin spindle protein (KSP). In a phase 1 trial, ALN-VSP02 administered as an iv infusion q2 wks was well-tolerated and showed evidence of anti-VEGF pharmacology and antitumor activity. Methods: Patients treated on the phase I trial with stable disease (SD) or better after 4 months (8 doses) were eligible to continue on an extension study until disease progression. Main objectives included continued evaluation of safety/tolerability and assessment of disease response. Results: Seven of 37 patients …

Cancer Researchbiologybusiness.industryExtension studyVEGF receptorsCancermedicine.diseaseVascular endothelial growth factorchemistry.chemical_compoundOncologychemistryRNA interferencebiology.proteinCancer researchMedicineOpen labelbusinessJournal of Clinical Oncology
researchProduct

Reveglucosidase alfa (BMN 701), a GILT-tagged recombinant human acid alpha glucosidase (rhGAA), evaluation in late-onset Pompe disease: Preliminary c…

2015

Neurologybusiness.industryExtension studyPediatrics Perinatology and Child HealthMedicineRecombinant human acid alpha-glucosidaseLate onsetNeurology (clinical)DiseaseClinical efficacybusinessVirologyGenetics (clinical)Neuromuscular Disorders
researchProduct

A Phase III Extension Study of Aldurazyme®(Laronidase) in Mucopolysaccharidosis I

2007

PharmacologyPediatricsmedicine.medical_specialtybusiness.industryExtension studyLARONIDASEPhase (matter)Mucopolysaccharidosis IMedicineAldurazymePharmacology (medical)businessClinical Therapeutics
researchProduct

Correction: Corrigendum: Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

2013

CORRIGENDUM: Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

Veterinary medicinePediatricsmedicine.medical_specialtybusiness.industryIdursulfaseExtension studyHunter syndromesocial sciencesmedicine.diseasehumanitiesbehavior and behavior mechanismsmedicinepopulation characteristicsbusinessgeographic locationsGenetics (clinical)medicine.drugGenetics in Medicine
researchProduct

Sustained safety and efficacy of ligelizumab in patients with chronic spontaneous urticaria: A one‐year extension study

2021

Background: Ligelizumab, a next-generation, humanized anti-immunoglobulin E (IgE) monoclonal antibody is in development as a treatment for patients with chronic spontaneous urticaria, whose symptoms are inadequately controlled with standard-of-care therapy. Objective: To evaluate the long-term safety and re-treatment efficacy of ligelizumab 240 mg in patients who completed the core study and extension study. Methods: This open-label, single-arm, long-term Phase 2b extension study was designed to assess patients who were previously administered various doses of ligelizumab, omalizumab or placebo in the Phase 2b, dose-finding core study and who presented with active disease after Week 32. In …

medicine.medical_specialtyUrticariaImmunologyLigelizumab610OmalizumabOmalizumabImmunoglobulin EPlaceboAntibodies Monoclonal HumanizedInternal medicineActive diseasemedicineImmunology and AllergyHumansIn patientChronic UrticariaAdverse effectbiologybusiness.industryExtension studyChronic spontaneous urticariaTreatment OutcomeLigelizumabbiology.proteinIgEbusiness600 Technik Medizin angewandte Wissenschaften::610 Medizin und Gesundheit::610 Medizin und Gesundheitmedicine.drug
researchProduct

Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks: An Open-Label Extension Study …

2018

medicine.medical_specialtybiologybusiness.industryC1 Esterase Inhibitor ProteinExtension studymedicine.diseaseC1-inhibitorInternal medicineHereditary angioedemabiology.proteinLong term outcomesMedicineOpen labelbusinessSSRN Electronic Journal
researchProduct