DOCSLIB.ORG

A Phase 2A Study to Evaluate the Safety, Tolerability, and Immunogenicity of One Dose of NDV-3A Vaccine in Patients with STAT3-Mutated Hyper-Ige Syndrome

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
A Phase 2A Study to Evaluate the Safety, Tolerability, and Immunogenicity of One Dose of NDV-3A Vaccine in Patients with STAT3-Mutated Hyper-Ige Syndrome A Phase 2a Study to Evaluate the Safety, Tolerability, and Immunogenicity of One Dose of NDV-3A Vaccine in Patients With STAT3-Mutated Hyper-IgE Syndrome NIAID Protocol Number: 17-I-0017 Version Number: 3.0 Date: August 13, 2018 Investigational New Drug (IND) Number: 17230 IND Sponsor: Office of Clinical Research Policy and Regulatory Operations (OCRPRO) Division of Clinical Research (DCR) National Institute of Allergy and Infectious Diseases (NIAID) National Institutes of Health (NIH) Sponsor Medical Monitor: H. Preston Holley, MD, FIDSA Medical Monitor Leidos Biomedical Research 5705 Industry Lane Frederick, MD 21701 Phone: 301 228-4005 Email: [email protected] Pharmaceutical Support Provided by: NovaDigm Therapeutics, Inc. 4201 JaMes Ray Drive Technology Transfer Building Grand Forks, ND 58202 OHRP Assurance: FWA00011288 Principal Investigator: Alexandra FreeMan, MD Laboratory of Clinical IMMunology and Microbiology (LCIM) Building 10 Room 11N234 10 Center Drive Bethesda, MD 20814 Phone: 301-594-9045 Email: [email protected] Conducted by: LCIM, NIAID, NIH NDV-3A Vaccine Study Protocol Version 3.0 August 13, 2018 Study Staff Roster Associate Investigators: Lisa Barnhart, RN, MSN PaMela Welch, RN, BSN LCIM/NIAID Leidos Biomedical Research, Inc Phone: (301) 496-5270 LCIM/NIAID Email: [email protected] Phone: (301) 402-0449 Email: [email protected] Angelique Biancotto, PhD Center for Human ImMunology AutoiMMunity, and InflaMMation External Collaborators: (CHI)/National Heart, Lung and Blood John P. Hennessey, Jr, PhD Institute (NHLBI) NovaDigm Therapeutics, Inc. Phone: (301) 594-0060 4201 JaMes Ray Drive Email: [email protected] Technology Transfer Building Grand Forks, ND 58202 Jinguo Chen, MD Phone: (267) 640-5189 CHI/NHLBI Email: [email protected] Phone: (301) 594-4636 Dr. Hennessey will supply the vaccine from Email: [email protected] NovaDigm. He will also perform the serologic assays, including anti-rAls3 IgG Elise Ferré, PA-C, MPH and IgA1 responses, at baseline and to the LCIM/NIAID vaccine on the de-identified saMples. He Phone: (301) 496-8985 will also assist in the data analysis of the Email: [email protected] cytokine responses to the vaccination. Steven Holland, MD LCIM/NIAID Phone: (301) 402-7684 Email: [email protected] External Collaborators (Cont.): Jack E. Edwards, Jr, MD Sally Hunsberger, PhD Los Angeles Biomedical Research Institute Biostatistics Research Branch/NIAID Harbor-UCLA Medical Center Phone: (240) 669-5257 Division of Infectious Diseases (RB2) Email: [email protected] 1124 W. Carson Street Torrance, CA 90502 Michail Lionakis, MD Phone: (310) 222-3813 LCIM/NIAID Email: [email protected] Phone: (301) 443-5089 Dr. Edwards will assist in the interpretation Email: [email protected] of the serologic and cytokine responses to the vaccines on de-identified saMples. AManda Urban, CRNP Leidos Biomedical Research, Inc LCIM/NIAID Phone: (301) 451-3191 Email: [email protected] Page 2 of 47 NDV-3A Vaccine Study Protocol Version 3.0 August 13, 2018 Study Site: Institutional Review Board (IRB): NIH Clinical Center (CC) NIAID IRB 10 Center Drive 5601 Fishers Lane Bethesda, MD 20814 Bethesda, MD 20892 Page 3 of 47 NDV-3A Vaccine Study Protocol Version 3.0 August 13, 2018 Table of Contents Study Staff Roster ............................................................................................................................ 2 Table of Contents ............................................................................................................................ 4 List of Abbreviations ....................................................................................................................... 7 Protocol SumMary ........................................................................................................................... 8 Précis ............................................................................................................................................. 10 1 Background Information and Scientific Rationale ................................................................ 11 Background Information ............................................................................................... 11 Description of the Study Agent ............................................................................. 11 SumMary of Preclinical Studies of NDV-3 and NDV-3A .................................... 11 SumMary of Relevant Clinical Studies of NDV-3 and NDV-3A .......................... 13 SumMary of Relevant Clinical Studies of Other Vaccines ................................... 14 Rationale ........................................................................................................................ 15 2 Study Objectives .................................................................................................................... 16 PriMary Objective .......................................................................................................... 16 Secondary Objectives .................................................................................................... 16 Exploratory Objectives .................................................................................................. 16 3 Study Design .......................................................................................................................... 17 Description of the Study Design .................................................................................... 17 Study Endpoints ............................................................................................................ 17 PriMary Endpoint ................................................................................................... 17 Secondary Endpoints ............................................................................................. 17 Exploratory Endpoints ........................................................................................... 17 4 Study Population .................................................................................................................... 18 RecruitMent Plan ........................................................................................................... 18 Subject Inclusion Criteria .............................................................................................. 19 Subject Exclusion Criteria ............................................................................................. 19 Justification for Exclusion of Special Populations ........................................................ 21 Exclusion of women .............................................................................................. 21 Exclusion of minors ............................................................................................... 21 5 Study Agent/Interventions ..................................................................................................... 21 Disposition and Dispensation ........................................................................................ 21 Formulation, Packaging, and Labeling .................................................................. 21 Study Agent Storage and Stability ................................................................................ 22 Preparation, Administration, and Dosage of Study Agent ............................................ 22 Study Product Accountability Procedures ..................................................................... 22 AssessMent of Subject Compliance with Study Agent ................................................. 22 Concomitant Medications and Procedures .................................................................... 22 Prohibited Medications and Procedures ........................................................................ 23 6 Study Schedule and Procedures ............................................................................................. 23 Screening (Day −28 to baseline) ................................................................................... 23 Baseline Visits (Days –3 to 0) ....................................................................................... 24 Visit for Vaccination (Days 0 to 2) ............................................................................... 24 Follow-up Visits ............................................................................................................ 25 Early Termination Visit ................................................................................................. 26 Pregnancy and Follow-up Visit ..................................................................................... 26 Page 4 of 47 NDV-3A Vaccine Study Protocol Version 3.0 August 13, 2018 7 Potential Risks and Benefits .................................................................................................. 26 Potential Risks ............................................................................................................... 26 Potential Benefits ........................................................................................................... 28 8 Research Use of Stored Human SaMples, SpeciMens, or Data ............................................. 28 9 Data Sharing Plan .................................................................................................................. 29 10 ReMuneration Plan for Participants ......................................................................................
Load more

Recommended publications
  • A Comparison of the Tolerability of the Direct Renin Inhibitor Aliskiren and Lisinopril in Patients with Severe Hypertension
    Journal of Human Hypertension (2007) 21, 780–787 & 2007 Nature Publishing Group All rights reserved 0950-9240/07 $30.00 www.nature.com/jhh ORIGINAL ARTICLE A comparison of the tolerability of the direct renin inhibitor aliskiren and lisinopril in patients with severe hypertension RH Strasser1, JG Puig2, C Farsang3, M Croket4,JLi5 and H van Ingen4 1Technical University Dresden, Heart Center, University Hospital, Dresden, Germany; 2Department of Internal Medicine, La Paz Hospital, Madrid, Spain; 31st Department of Internal Medicine, Semmelweis University, Budapest, Hungary; 4Novartis Pharma AG, Basel, Switzerland and 5Novartis Institutes for Biomedical Research, Cambridge, MA, USA Patients with severe hypertension (4180/110 mm Hg) LIS 3.4%). The most frequently reported AEs in both require large blood pressure (BP) reductions to reach groups were headache, nasopharyngitis and dizziness. recommended treatment goals (o140/90 mm Hg) and At end point, ALI showed similar mean reductions from usually require combination therapy to do so. This baseline to LIS in msDBP (ALI À18.5 mm Hg vs LIS 8-week, multicenter, randomized, double-blind, parallel- À20.1 mm Hg; mean treatment difference 1.7 mm Hg group study compared the tolerability and antihyperten- (95% confidence interval (CI) À1.0, 4.4)) and mean sitting sive efficacy of the novel direct renin inhibitor aliskiren systolic blood pressure (ALI À20.0 mm Hg vs LIS with the angiotensin converting enzyme inhibitor À22.3 mm Hg; mean treatment difference 2.8 mm Hg lisinopril in patients with severe hypertension (mean (95% CI À1.7, 7.4)). Responder rates (msDBPo90 mm Hg sitting diastolic blood pressure (msDBP)X105 mm Hg and/or reduction from baselineX10 mm Hg) were 81.5% and o120 mm Hg).
    [Show full text]
  • Clinical Pharmacology 1: Phase 1 Studies and Early Drug Development
    Clinical Pharmacology 1: Phase 1 Studies and Early Drug Development Gerlie Gieser, Ph.D. Office of Clinical Pharmacology, Div. IV Objectives • Outline the Phase 1 studies conducted to characterize the Clinical Pharmacology of a drug; describe important design elements of and the information gained from these studies. • List the Clinical Pharmacology characteristics of an Ideal Drug • Describe how the Clinical Pharmacology information from Phase 1 can help design Phase 2/3 trials • Discuss the timing of Clinical Pharmacology studies during drug development, and provide examples of how the information generated could impact the overall clinical development plan and product labeling. Phase 1 of Drug Development CLINICAL DEVELOPMENT RESEARCH PRE POST AND CLINICAL APPROVAL 1 DISCOVERY DEVELOPMENT 2 3 PHASE e e e s s s a a a h h h P P P Clinical Pharmacology Studies Initial IND (first in human) NDA/BLA SUBMISSION Phase 1 – studies designed mainly to investigate the safety/tolerability (if possible, identify MTD), pharmacokinetics and pharmacodynamics of an investigational drug in humans Clinical Pharmacology • Study of the Pharmacokinetics (PK) and Pharmacodynamics (PD) of the drug in humans – PK: what the body does to the drug (Absorption, Distribution, Metabolism, Excretion) – PD: what the drug does to the body • PK and PD profiles of the drug are influenced by physicochemical properties of the drug, product/formulation, administration route, patient’s intrinsic and extrinsic factors (e.g., organ dysfunction, diseases, concomitant medications,
    [Show full text]
  • Exploring the Concept of Safety and Tolerability
    SECOND ANNUAL WORKSHOP ON CLINICAL OUTCOME ASSESSMENTS IN CANCER CLINICAL TRIALS April 25, 2017 Bethesda, MD Co-sponsored by Session 1 Exploring the Concepts of Safety and Tolerability: Incorporating the Patient Voice SECOND ANNUAL WORKSHOP ON CLINICAL OUTCOME ASSESSMENTS IN CANCER CLINICAL TRIALS April 25, 2017 Bethesda, MD Co-sponsored by Disclaimer • The views and opinions expressed in the following slides are those of the individual presenters and should not be attributed to their respective organizations/companies, the U.S. Food and Drug Administration or the Critical Path Institute. • These slides are the intellectual property of the individual presenters and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. All trademarks are the property of their respective owners. 3 Session Participants Chair • Bindu Kanapuru, MD – Medical Officer, Division of Hematology Products, OHOP, FDA Presenters • James (Randy) Hillard, MD – Professor of Psychiatry, Michigan State University • Crystal Denlinger, MD, FACP – Associate Professor, Department of Hematology/Oncology; Chief, Gastrointestinal Medical Oncology; Director, Survivorship Program; Deputy Director, Phase 1 Program, Fox Chase Cancer Center • Katherine Soltys, MD – Acting Director, Bureau of Medical Sciences, Therapeutic Products Directorate, Health Products and Food Branch, Health Canada • Karen E. Arscott, DO, MSc – Associate Professor of Medicine-Patient Advocate and Survivor, Geisinger Commonwealth
    [Show full text]
  • Glaucoma Medical Therapy Ocular Tolerability, Adherence, and Patient Outcomes
    CME Monograph November 30, 2017 September 23, 2016 Expiration: Original Release: November 1, 2016 Last Review: GLAUCOMA MEDICAL THERAPY OCULAR TOLERABILITY, ADHERENCE, AND PATIENT OUTCOMES ACTIVITY DESCRIPTION commercial interests in the form of Consultant/Advisory Therapy to lower intraocular pressure effectively reduces the Board: Alcon; Bausch & Lomb Incorporated; and Inotek risk of glaucoma progression. The rate of nonadherence with Pharmaceuticals Corporation; Contracted Research: Alcon; PROGRAM CHAIR therapy, however, remains high. Therapeutic nonadherence and F. Hoffmann-La Roche Ltd. is a complex and multifactorial issue that is influenced by Richard K. Parrish II, MD James C. Tsai, MD, MBA, had a financial agreement factors attributable to the physician, patient, and medication. or affiliation during the past year with the following Edward W. D. Norton Chair in Ophthalmology All medications have side effects, and tolerability issues can commercial interests in the form of Consultant/ Professor contribute to nonadherence if patients perceive that the Advisory Board: Aerie Pharmaceuticals, Inc; and Inotek disadvantages of therapy outweigh the benefits of controlling Director, Glaucoma Service Pharmaceuticals Corporation. a disease that is often asymptomatic. Therefore, the selection Bascom Palmer Eye Institute of initial and adjunctive therapy should consider the NEW YORK EYE AND EAR INFIRMARY OF MOUNT SINAI University of Miami Miller School of Medicine potential for tolerability issues that may adversely affect PEER REVIEW DISCLOSURE Miami, Florida adherence. Novel therapies in late-stage clinical development Joseph F. Panarelli, MD, had a financial agreement or affiliation will offer innovative mechanisms of action and unique risk/ during the past year with the following commercial interest FACULTY benefit profiles.
    [Show full text]
  • Efficacy, Tolerability and Serum Phenytoin Levels After Intravenous Fosphenytoin Loading Dose in Children with Status Epilepticus
    R E S E A R C H P A P E R Efficacy, Tolerability and Serum Phenytoin Levels after Intravenous Fosphenytoin Loading Dose in Children with Status Epilepticus KAVITA SRIVASTAVA, SHIRISH BHARTIYA, VRUSHABH GAVLI, RAHUL PATIL AND SUREKHA RAJADHYAKSHA From Pediatric Neurology Unit, Department of Pediatrics, Bharati Vidyapeeth Deemed University Medical College, Pune, India. Correspondence to: Dr Kavita Srivastava, Professor in Pediatrics, 3rd floor, Bharati Hospital, Katraj, Pune 411 043, India. [email protected] Received: July 05, 2018; Initial review: December 03, 2018; Accepted: December 04, 2019. Objective: To evaluate the efficacy and tolerability of intravenous children showed any local or systemic adverse effects. Serum fosphenytoin in children with status epilepticus, and resulting total phenytoin levels were in the therapeutic range (10-20 µg/mL) serum total phenytoin levels. in 12 (23.5%), sub-therapeutic in 16 (31.3%) and supra- Methods: In this prospective study, 51 children aged less than 18 therapeutic in 25 (49%) children. There was weak correlation of years received intravenous loading dose of fosphenytoin (18-20 the phenytoin levels with dose of fosphenytoin received, seizure mg/kg). Serum total phenytoin levels were estimated at 90 -100 control, or adverse effects. minutes. Outcomes studied were (i) seizure control and local and/ Conclusion: Intravenous fosphenytoin loading dose of 20 mg/kg or systemic adverse effects in next 24 hours and (ii) phenytoin is effective in controlling seizures in 88% of children with status levels and its correlation with dose received, seizure control and epilepticus, with a good safety profile. Seizure control and adverse adverse effects. effects appear to be independent of serum total phenytoin levels Results: The actual dose of fosphenytoin received varied from achieved.
    [Show full text]
  • Draft Outline for Manuscript on Effects of Nintedanib in Patients with IPF
    ERJ Express. Published on May 24, 2018 as doi: 10.1183/13993003.02106-2017 Early View Research letter Safety and tolerability of nintedanib in patients with IPF in the United States Imre Noth, David Oelberg, Manika Kaul, Craig S Conoscenti, Ganesh Raghu Please cite this article as: Noth I, Oelberg D, Kaul M, et al. Safety and tolerability of nintedanib in patients with IPF in the United States. Eur Respir J 2018; in press (https://doi.org/10.1183/13993003.02106-2017). This manuscript has recently been accepted for publication in the European Respiratory Journal. It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJ online. Copyright ©ERS 2018 Copyright 2018 by the European Respiratory Society. Safety and tolerability of nintedanib in patients with IPF in the United States Imre Noth,1 David Oelberg,2 Manika Kaul,3 Craig S Conoscenti,3 Ganesh Raghu.4 1University of Virginia School of Medicine, Charlottesville, Virginia, USA; 2Western Connecticut Health Network, Danbury Hospital, Danbury, Connecticut, USA; 3Boehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, Connecticut, USA; 4Department of Medicine, University of Washington, Seattle, Washington, USA Corresponding author: Imre Noth University of Chicago Pulmonary and Critical Care Medicine Section of Pulmonary and Critical Care Medicine 5841 South Maryland Ave MC 6076 Chicago Illinois 60637 USA Email address: [email protected] Take-home message: The safety and tolerability profile of nintedanib in the clinical setting is consistent with the product label.
    [Show full text]
  • A) General Scientific Data
    Contents A) General Scientific data ....................................................................................................... 1 Histopathology ................................................................................................. 1 Epidemiology .................................................................................................... 2 Clinical Data ..................................................................................................... 2 Diagnostics ........................................................................................................ 3 Molecular Biology ............................................................................................ 3 Vaccines ............................................................................................................ 4 B) Pathology Practice Data including contribution from ESP Affiliated National Societies ..................................................................................................................................... 4 General data ..................................................................................................... 4 Autopsy Pathology ........................................................................................... 4 C) Other useful links ................................................................................................................ 5 A) General Scientific data Histopathology 1. Histopathologic Changes and SARS–CoV-2 Immunostaining in the Lung of a Patient With
    [Show full text]
  • Comparative Effectiveness Research and Personalized Medicine: from Contradiction to Synergy
    Comparative Effectiveness Research and Personalized Medicine: From Contradiction to Synergy Prepared for: Personalized Medicine Coalition Clifford Goodman, PhD The Lewin Group Center for Comparative Effectiveness Research October 28, 2009 Comparative Effectiveness Research and Personalized Medicine 10-28-09 ABOUT THE LEWIN GROUP CENTER FOR COMPARATIVE EFFECTIVENESS RESEARCH The Lewin Group Center for Comparative Effectiveness Research manages, conducts, interprets and supports the use of comparative effectiveness research to make informed decisions regarding patient outcomes, health care policy and the optimal use of health care resources. The Lewin Group is a premier national health care and human services consulting firm that has delivered objective analyses and strategic counsel to prominent public agencies, nonprofit organizations, industry associations and private companies across the United States for nearly 40 years. In keeping with our tradition of objectivity, The Lewin Group is not an advocate for or against any legislation. The Lewin Group is part of Ingenix, Inc., which is a wholly owned subsidiary of UnitedHealth Group. To assure the independence of its work, The Lewin Group has editorial control over all of its work products. Go to www.lewin.com/integrity for more information. The Lewin Group is widely recognized for its expertise in the U.S. health care system, its record in health technology assessment and evidence-based medicine; health care policy and other issues, and its independence and objectivity. In the Lewin Center, we are combining these strengths with the unique capabilities of our sister company, i3, in clinical trials and study design, health economics and outcomes research; and through Ingenix, access to one of the world’s largest and most robust patient data sets including de-identified, integrated diagnosis, medical, and pharmacy claims data for millions of individuals.
    [Show full text]
  • Safety and Tolerability of High Doses of Intrathecal Fentanyl for the Treatment of Chronic Pain
    case study Safety and tolerability of high doses of intrathecal fentanyl for the treatment of chronic pain Sulane Do Ouro, MD Santiago Esteban, BS Una Sibirceva, MD Beverly Whittenberg, MD Russell Portenoy, MD Ricardo A. Cruciani, MD, PhD abstract up to 300 mg/d have been reported. 3 Although higher doses are used clinically and the potential outcomes Fentanyl is commonly used systemically or neuraxially associated with this approach are assumed to be compa - for the management of chronic pain. It can be adminis - rable to those observed during neuraxial infusion with tered intrathecally via implanted pump, but it is generally other opioids, there are no published observations. We considered only after trials of intrathecal (IT) morphine describe four patients with chronic pain whose doses of and hydromorphone have proven ineffective. Published IT fentanyl were gradually titrated to levels as high as 20 experience with IT fentanyl is limited, and long-term ther - times those previously reported. These cases are relevant apy at relatively high doses has not been described previ - to an understanding of both safety and tolerability of IT ously. We describe four patients who were treated with IT fentanyl during long-term therapy. fentanyl after other analgesic approaches had failed and who gradually underwent dose escalation to levels as high case reports as 20 times those previously reported. Safety and tolerabil - ity were maintained during dose titration. Our experi - case 1 ence highlights an expanding scope of practice in the use of IT opioids in general and fentanyl specifically and sug - A 58-year-old diabetic man was experiencing severe gests that high-dose fentanyl can be used safely in highly chronic pain from multiple sources.
    [Show full text]
  • Free PDF Download
    European Review for Medical and Pharmacological Sciences 2019; 23 (4 Suppl): 21-26 Efficacy and tolerability of tapentadol prolonged release during rehabilitation: a prospective, observational study M. ORFEI1, P. MILIA1, M. CASERIO1, T.C. BAGAPHOU2 1Istituto Prosperius Tiberino, Intensive Rehabilitation Centre, Umbertide, Perugia, Italy 2Intensive Care and Pain Medicine, Section of Anesthesia, Città di Castello Hospital, Perugia, Italy Abstract. – OBJECTIVE: The treatment of Analgesic treatment and interventional pro- chronic pain frequently combines pharmacologic cedures are usually associated in order to obtain and non-pharmacological options, and analgesia better pain control. Moreover, analgesia after after surgery is of major importance. Tapentadol surgery is of major importance. is both µ-opioid receptor agonist (with a 40% ag- Tapentadol is indicated for the treatment onism on these receptors) and noradrenaline re- uptake inhibitor, with similar analgesic efficacy of chronic severe pain. Its dual mechanism to strong opioids, but fewer adverse effects. For of action consists of a 40% µ-opioid receptor these reasons, tapentadol may represent a valu- agonism (MOR) associated to noradrenaline able first choice option in the treatment of chronic, reuptake inhibition (NRI)4. Tapentadol pro- neuropathic, and mixed pain. longed release (PR) demonstrated a similar or PATIENTS AND METHODS: The primary end- superior efficacy for pain control compared point of the present study was the proportion of responder patients, with ≥30% reduction in pain with strong opioids, but the reduced µ-load intensity during loading on the NRS; several ad- carries out a lower incidence of adverse effects ditional endpoints were also evaluated. and an overall better tolerability profile with RESULTS: Twenty-five adult patients were en- a lower risk of treatment discontinuation and rolled, with a rate of response to treatment of improved patients’ quality of life5.
    [Show full text]
  • Ashp Accreditation Standard for Specialty Pharmacy Practice
    ASHP ACCREDITATION STANDARD FOR SPECIALTY PHARMACY PRACTICE Introduction The ASHP Accreditation Standard for Specialty Pharmacy Practice reflects contemporary best practice for specialty pharmacy. The accreditation process is designed to be easy to understand, flexible, and consultative in nature and can be tailored to different models of care. The accreditation focuses on optimal care delivery through evaluation of: Effective patient care plans to achieve desired medication therapy outcomes Patient-specific assessments and optimal collection, use, and documentation of information Inclusion of specialty drug-specific assessment and disease state-specific assessment requirements Comprehensive review of the patient’s medication history and medication list prior to each fill Documentation of all pharmacy case management activities Patient consultation and education Quality metrics and quality improvement plans This standard combines the Center for Pharmacy Practice Accreditation (CPPA) standards1 with the support of ASHP’s pharmacy expertise and accreditation history. Successful accreditation signifies to payers, patients, and other healthcare providers that the pharmacy provides an advanced level of high- quality, safe, and efficient patient care in a predictable and measurable way. Purpose of the Standard for Specialty Pharmacy Practice Specialty pharmacy generally revolves around the provision of 1) high cost medications with 2) special handling procedures and 3) requiring complex patient care.2 Specialty pharmaceuticals have at least four of the following characteristics: 3 Typically high in cost ($600 or more per month) 1 Version III, February 27, 2019; Standards used with permission of Center for Pharmacy Practice Accreditation (CPPA) 2 Blaser DA, Lewtas AJ, et al. How to define specialty pharmaceuticals—a systematic review. Am J Pharm Benefits.
    [Show full text]
  • A5343 a Trial of the Safety, Tolerability, and Pharmacokinetics of Bedaquiline and Delamanid, Alone and in Combination, Among Pa
    A5343 A Trial of the Safety, Tolerability, and Pharmacokinetics of Bedaquiline and Delamanid, Alone and in Combination, among Participants Taking Multidrug Treatment for Drug-Resistant Pulmonary Tuberculosis A Limited-Center Trial of the AIDS Clinical Trials Group (ACTG) Sponsored by: National Institute of Allergy and Infectious Diseases Industry Support Provided by: Janssen Pharmaceuticals, Inc. Otsuka Pharmaceutical Company, Ltd. ViiV Healthcare, Ltd. IND# 127,382 The ACTG Tuberculosis Transformative Science Group Gavin Churchyard, MBBCh, MMED, FCP, PhD, Chair Protocol Co-Chairs: Kelly Dooley, MD, PhD Gary Maartens, MBChB, MMed Protocol Vice Chair: Francesca Conradie, MD, DTM&H DAIDS Clinical Representatives: Richard Hafner, MD Roxana Rustomjee MbChB, MMed, FCPhM, FRCP, PhD Clinical Trials Specialists: Laura E. Moran, MPH Chanelle Houston, BS FINAL Version 4.0 June 26, 2018 A5343 FINAL Version 4.0 06/26/18 A Trial of the Safety, Tolerability, and Pharmacokinetics of Bedaquiline and Delamanid, Alone and in Combination, among Participants Taking Multidrug Treatment for Drug-Resistant Pulmonary Tuberculosis SIGNATURE PAGE I will conduct the study in accordance with the provisions of this protocol and all applicable protocol-related documents. I agree to conduct this study in compliance with United States (US) Health and Human Service regulations (45 CFR 46); applicable U.S. Food and Drug Administration regulations; standards of the International Conference on Harmonization Guideline for Good Clinical Practice (E6); Institutional Review
    [Show full text]