protocol_id stringclasses 263
values | section_number stringlengths 1 12 | title stringlengths 1 1.88k | content stringlengths 0 866k | merged_titles listlengths 0 491 |
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NCT04153929 | 8.3.2 | Direct access to source data and documents | 8.3.2 Direct access to source data and documents The sponsor will monitor the conduct of the trial by regular on-site monitoring visits and in-house data quality review. The frequency of site monitoring will be determined by assessing all characteristics of the trial, including its nature, objective, methodology and th... | [] |
NCT04153929 | 8.3.3 | Storage period of records | 8.3.3 Storage period of records
Trial sites: The trial sites must retain the source and essential documents (including ISF) according to contract or the local requirements valid at the time of the end of the trial (whatever is longer). Sponsor: The sponsor must retain the essential documents according to the sponsor's... | [
"Trial sites:"
] |
NCT04153929 | 8.4 | EXPEDITED REPORTING OF ADVERSE EVENTS | 8.4 EXPEDITED REPORTING OF ADVERSE EVENTS BI is responsible to fulfil their legal and regulatory reporting obligation in accordance with regulatory requirements. | [] |
NCT04153929 | 8.5 | STATEMENT OF CONFIDENTIALITY AND PATIENT PRIVACY | 8.5 STATEMENT OF CONFIDENTIALITY AND PATIENT PRIVACY Data protection and data security measures are implemented for the collection, storage and processing of patient data in accordance with the principles 7 and 12 of the WHO GCP handbook. Individual patient data obtained as a result of this trial is considered confiden... | [] |
NCT04153929 | 8.5.1 | Collection, storage and future use of biological samples and corresponding data | 8.5.1 Collection, storage and future use of biological samples and corresponding data Measures are in place to comply with the applicable rules for the collection, biobanking and future use of biological samples and clinical data, in particular - Sample and data usage has to be in accordance with the separate biobankin... | [] |
NCT04153929 | 8.6 | TRIAL MILESTONES | 8.6 TRIAL MILESTONES The start of the trial is defined as the date when the first patient in the whole trial signs informed consent. The end of the trial is defined as the date of the last visit of the last patient in the whole trial ("Last Patient Completed"). The "Last Patient Last Treatment" (LPLT) date is defined a... | [
"c26686857-02 Clinical Trial Protocol Page 77 of 87"
] |
NCT04153929 | 8.7 | ADMINISTRATIVE STRUCTURE OF THE TRIAL | 8.7 ADMINISTRATIVE STRUCTURE OF THE TRIAL The trial is sponsored by Boehringer Ingelheim (BI). A Coordinating Investigator is responsible to coordinate investigators at the different sites participating in this trial. Tasks and responsibilities are defined in a contract. A safety monitoring committee (SMC) composed of ... | [] |
NCT04153929 | 9 | REFERENCES | 9. REFERENCES | [] |
NCT04153929 | 9.1 | PUBLISHED REFERENCES | 9.1 PUBLISHED REFERENCES | P19-01871 | American Diabetes Association StandardsofMedicalCareinDiabetes– 2019abridgedforprimarycareproviders. Clin Diabetes 37 (1), 11 -34 (2019) | |-----------|---------------------------------------------------------------------------------------------------------------------------------... | [] |
NCT04153929 | 9.2 | UNPUBLISHED REFERENCES | 9.2 UNPUBLISHED REFERENCES | c14085752-06 | Investigator's Brochure: BI 456906, Version 6.0; Indication 1404.P01,1404.P02, 1404.P03 | |--------------|--------------------------------------------------------------------------------------------------------------------------------------------------------------------------... | [] |
NCT04153929 | 10 | APPENDICES | 10. APPENDICES Not applicable c26686857-02 Clinical Trial Protocol Page 82 of 87 Proprietary confidential information © 2020 Boehringer Ingelheim International GmbH or one or more of its affiliated companies | [] |
NCT04153929 | 11 | DESCRIPTION OF GLOBAL AMENDMENT(S) | 11. DESCRIPTION OF GLOBAL AMENDMENT(S) | [] |
NCT04153929 | 11.1 | GLOBAL AMENDMENT 1 | 11.1 GLOBAL AMENDMENT 1 | Date of amendment | 28September2020 | | | |---------------------------------------|----------------------------------------------------------|--|--| | EudraCT number | 2019-002390-60 | | | | EU number | | | | | BI Trial number | 1404-0002 | | | | BI Investigational Medicinal | BI 456906 | | | ... | [
"Boehringer Ingelheim 28 Sep 2020 BI Trial No.: 1404-0002",
"APPROVAL / SIGNATURE PAGE",
"Signatures (obtained electronically)"
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NCT04161495 | 1.1 | SYNOPSIS | 1.1 SYNOPSIS Protocol title: A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fe-von Willebrand Factor-XTEN Fusion Protein (tFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients >12 Years of Age With Severe Hemophilia A Short t... | [
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NCT04161495 | 1.2 | SCHEMA | 1.2 SCHEMA Figure 1 - Graphical study design  | [] |
NCT04161495 | 1.3 | SCHEDULE OF ACTIVITIES (SOA) | 1.3 SCHEDULE OF ACTIVITIES (SOA) Table 1 - Overall schedule of activities from screening to safety follow-up call or visit | Tests and assessments | Screeninga(Week -8 toDay 1) | BaselinebBIVV001Day 1 | Week 4c±7 days | Week 13c±7 days | Week26c,d±7 days | Week 39c±7days | Week 52/EOS/ETc±7 days | Unscheduledvisite | S... | [
"Amended clinical Trial Protocol 05 EFC16293 - efanesoctocog alfa"
] |
NCT04161495 | 2 | INTRODUCTION | 2 INTRODUCTION Recombinant coagulation factor VIII Fc – von Willebrand factor – XTEN fusion protein (rFVIIIFc-VWF-XTEN, efanesoctocog alfa) (BIVV001; BIIB073) is designed to be a nextgeneration extended half-life (EHL) blood clotting factor VIII (FVIII) product. As with existing FVIII products, BIVV001 temporarily repl... | [] |
NCT04161495 | 2.1 | STUDY RATIONALE | 2.1 STUDY RATIONALE BIVV001 is designed to be a next-generation extended half-life (EHL) blood clotting factor VIII. Preclinical and clinical experience indicate that BIVV001 has an extended half-life, which can potentially achieve and maintain substantially higher factor activity levels than currently available treatm... | [
"Current therapies for Hemophilia A"
] |
NCT04161495 | 2.2 | BACKGROUND | 2.2 BACKGROUND | [] |
NCT04161495 | 2.2.1 | BIVV001 | 2.2.1 BIVV001 BIVV001 is a recombinant fusion protein consisting of single-chain FVIII, the Fc domain of human immunoglobulin G1 (IgG1), the FVIII-binding D′D3 domain of von Willebrand factor (VWF), and 2 XTEN linkers. It is believed that the plasma t½ of FVIII is prolonged by its interaction with VWF. All current EHL ... | [
"Brief summary of clinical data of BIVV001",
"Study 242HA101",
"Safety results",
"Pharmacokinetic results",
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"Safety results",
"Pharmacokinetic results"
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NCT04161495 | 2.3 | BENEFIT/RISK ASSESSMENT | 2.3 BENEFIT/RISK ASSESSMENT
Benefits BIVV001 is designed to be a new class of blood clotting FVIII engineered to be independent of VWF. A new class of FVIII products that prevents and controls bleeding episodes for longer periods of time potentially reduces the burden of frequent IV administration and in turn, may imp... | [
"Benefits",
"Risks",
"Conclusion"
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NCT04161495 | 3 | OBJECTIVES AND ENDPOINTS | 3 OBJECTIVES AND ENDPOINTS Table 5 - Objectives and endpoints | | Objectives | Endpoint | | | |-------------------------------|------------------------------------------------------------------------------------------|------------------------------------------------------------------------------------------------------... | [] |
NCT04161495 | 3.1 | APPROPRIATENESS OF MEASUREMENTS | 3.1 APPROPRIATENESS OF MEASUREMENTS Each of the safety and efficacy assessments chosen for use in this study are considered well established and relevant in hemophilia. In addition, suitable steps have been built into each of these assessments to ensure their reliability and accuracy to minimize any risks to participan... | [] |
NCT04161495 | 4 | STUDY DESIGN | 4 STUDY DESIGN | [] |
NCT04161495 | 4.1 | OVERALL DESIGN | 4.1 OVERALL DESIGN This is a Phase 3, open-label, multinational, multicenter study of the safety, efficacy and PK of IV BIVV001 in approximately 150 previously treated patients PTPs ≥12 years of age with severe hemophilia A (defined as <1 IU/dL [<1%] endogenous FVIII). Approximately 124 participants currently on a prop... | [
"Screening",
"Enrollment and baseline (Day 1)",
"Study clinic visits"
] |
NCT04161495 | 4.2 | SCIENTIFIC RATIONALE FOR STUDY DESIGN | 4.2 SCIENTIFIC RATIONALE FOR STUDY DESIGN This multicenter, open-label, Phase 3 clinical study is being conducted to determine the safety, efficacy, and PK of BIVV001, administered as QW prophylaxis or on-demand at a dose of 50 IU/kg IV. The study will enroll previously treated patients (PTPs) with severe hemophilia A ... | [] |
NCT04161495 | 4.3 | JUSTIFICATION FOR DOSE | 4.3 JUSTIFICATION FOR DOSE The dose of 50 IU/kg IV QW was selected as the prophylactic and on-demand dose in adults and adolescents ≥12 years. The once-weekly dosing schedule was chosen to optimize participant protection and to reduce the treatment burden on participants and their families. The dose was selected and co... | [] |
NCT04161495 | 4.4 | END OF STUDY DEFINITION | 4.4 END OF STUDY DEFINITION End of Study will occur when all of the following criteria have been met: - 104 participants have reached 50 exposure days (EDs) and have completed a valid inhibitor test after the 50th ED. - At least 63 participants in Arm A have at least 6 months of participation in this study and have at ... | [] |
NCT04161495 | 4.5 | STUDY STOPPING RULES | 4.5 STUDY STOPPING RULES The Sponsor may terminate the study at any time, after informing Investigators, Institutional Review Boards/Ethics Committees, and applicable regulatory agencies. Investigators will be notified by the Sponsor (or designee) if enrollment and dosing are suspended, completed, or closed. For France... | [] |
NCT04161495 | 4.5.1 | Criteria for dose and/or enrollment suspension | 4.5.1 Criteria for dose and/or enrollment suspension The occurrence of specific study events will require that further enrollment in the study and further dosing be suspended. In this situation, the event(s) will be investigated prior to enrollment and dosing of any additional participants. In this study, events requir... | [] |
NCT04161495 | 5 | STUDY POPULATION | 5 STUDY POPULATION Prospective approval of protocol deviations to recruitment and enrollment criteria, also known as protocol waivers or exemptions, are not permitted. | [] |
NCT04161495 | 5.1 | INCLUSION CRITERIA | 5.1 INCLUSION CRITERIA Participants are eligible to be included in the study only if all of the following criteria apply:
Age I 01. Participant must be equal to or greater than 12 years of age inclusive, at the time of signing the informed consent.
Type of participant and disease characteristics - I 02. Severe hemoph... | [
"Age",
"Type of participant and disease characteristics",
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NCT04161495 | I | 08. Male or Female | I 08. Male or Female Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. - a) Male participants - No contraceptive measures required for this study. - b) Female participants - A female participant is eligible t... | [
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NCT04169750 | A | multicenter, randomized, single-blind non-inferiority trial | A multicenter, randomized, single-blind non-inferiority trial Version 1.0, 02 Novembre 2018 Supported by FISM (Fondazione Italiana Sclerosi Multipla) cod. 2017/R/22 and financed or co-financed with the '5 per mille' public funding EudraCT number: NOT APPLICABLE | Principal Investigator | Luca Prosperini, MD, PhD | | |-... | [
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"INTRODUCTION",
"Background and rationale for conducting... |
NCT04183790 | 1 | TITLE PAGE | 1 TITLE PAGE  VERTEX PHARMACEUTICALS INCORPORATED
Clinical Study Protocol A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older Vertex Study Number: VX19-445-107 VX-445 IND ... | [
"Clinical Study Protocol",
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NCT04183790 | 2 | PROTOCOL SYNOPSIS | 2 PROTOCOL SYNOPSIS Title A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older Brief Title Evaluation of Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosi... | [
"Objectives Primary Objective",
"Secondary Objective",
"Endpoints Primary Endpoint",
"Secondary Endpoints",
"Other Endpoints",
"Strength:",
"Other Assessments:"
] |
NCT04183790 | 3 | SCHEDULE OF ASSESSMENTS | 3 SCHEDULE OF ASSESSMENTS [Table 3-1](#page-6-0) and [Table](#page-9-0) 3-2 provide the schedules of assessments. All visits are to be scheduled relative to the Day 1 Visit in each Part. The Cystic Fibrosis Questionnaire-Revised (CFQ-R) must be completed before any other assessment (except informed consent form [ICF] s... | [
"List of Abbreviations"
] |
NCT04183790 | 5 | INTRODUCTION | 5 INTRODUCTION | [] |
NCT04183790 | 5.1 | Background | 5.1 Background Cystic fibrosis (CF) is an autosomal recessive chronic disease with serious morbidities and frequent premature mortality. CF affects more than 70,000 individuals worldwide[1](#page-55-1) (approximately 30,000 in the US[2](#page-55-2) and 45,000 in the EU[3](#page-55-3) ). Based on its prevalence, CF qual... | [] |
NCT04183790 | 5.2 | Study Rationale | 5.2 Study Rationale This study will provide data on the long-term safety, efficacy, and durability of VX-445/TEZ/IVA in subjects with CF who are 6 years of age and older and who are homozygous or heterozygous for the F508del mutation. | [] |
NCT04183790 | 6 | STUDY OBJECTIVES | 6 STUDY OBJECTIVES | [] |
NCT04183790 | 6.1 | Primary Objective | 6.1 Primary Objective To evaluate the long-term safety and tolerability of VX-445/TEZ/IVA in subjects with CF who are 6 years of age and older | [] |
NCT04183790 | 6.2 | Secondary Objective | 6.2 Secondary Objective To evaluate the long-term efficacy and pharmacodynamics (PD) of VX-445/TEZ/IVA | [] |
NCT04183790 | 7 | STUDY ENDPOINTS | 7 STUDY ENDPOINTS | [] |
NCT04183790 | 7.1 | Primary Endpoint | 7.1 Primary Endpoint Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, pulse oximetry, and ophthalmologic examinations | [] |
NCT04183790 | 7.2 | Secondary Endpoints | 7.2 Secondary Endpoints Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) Absolute change in sweat chloride (SwCl) Absolute change in CFQ-R respiratory domain (RD) score Absolute change in body mass index (BMI) and BMI-for-age z-score Number of pulmonary exacerbations (PEx) and CF-relat... | [] |
NCT04183790 | 7.3 | Other Endpoints | 7.3 Other Endpoints Absolute change in fecal elastase-1 (FE-1) levels Absolute change in serum levels of immunoreactive trypsinogen (IRT) | [] |
NCT04183790 | 8 | STUDY POPULATION | 8 STUDY POPULATION Eligibility will be reviewed and documented by an appropriately qualified member of the investigator's team before subjects are enrolled in Part A and Part B. Subjects who meet all of the inclusion criteria and none of the exclusion criteria will be eligible. In the criteria below, "parent study" is ... | [] |
NCT04183790 | 8.1 | Part A | 8.1 Part A | [] |
NCT04183790 | 8.1.1 | Inclusion Criteria | 8.1.1 Inclusion Criteria - 1. Subject (or his or her legally appointed and authorized representative) will sign and date an ICF, and, when appropriate, an assent form. - 2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study p... | [] |
NCT04183790 | 8.1.2 | Exclusion Criteria | 8.1.2 Exclusion Criteria - 1. History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. - 2. Pregnant or breast-feeding females. Female subjects must have a negative pregnancy test at the Part A Day... | [] |
NCT04183790 | 8.2 | Part B | 8.2 Part B | [] |
NCT04183790 | 8.2.1 | Inclusion Criteria | 8.2.1 Inclusion Criteria - 1. Subject (or his or her legally appointed and authorized representative) will sign and date an ICF, and, when appropriate, an assent form. - 2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study p... | [] |
NCT04183790 | 8.2.2 | Exclusion Criteria | 8.2.2 Exclusion Criteria - 5. History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. - 6. Pregnant or breast-feeding females. Female subjects must have a negative pregnancy test at the Part B Day... | [] |
NCT04183790 | 9 | STUDY IMPLEMENTATION | 9 STUDY IMPLEMENTATION | [] |
NCT04183790 | 9.1 | Study Design | 9.1 Study Design This is a Phase 3, 2-part, multicenter, open-label extension study for subjects who completed the Treatment Period in the parent study (VX18-445-106 Part B) and meet eligibility criteria. The study design is shown in [Figure 9-1.](#page-22-1) Subjects who complete Part A will have the opportunity to en... | [
"Part A:",
"Part B:"
] |
NCT04183790 | 9.1.1 | Treatment Period | 9.1.1 Treatment Period Treatment Period assessments are listed in [Table 3-1](#page-6-0) and [Table](#page-9-0) 3-2.
Part A: Subjects will receive the first dose of study drug on Part A Day 1 after obtaining informed consent (and assent, when applicable) and confirming eligibility. Subjects who enroll in this study on... | [
"Part A:",
"Part B:"
] |
NCT04183790 | 9.1.2 | Safety Follow-up | 9.1.2 Safety Follow-up In Parts A and B, the Safety Follow-up Visit is scheduled to occur 28 (± 7) days after the last dose of study drug. The Safety Follow-up Visit assessments are listed in [Table 3-1](#page-6-0) and [Table](#page-9-0) 3-2. The Safety Follow-up Visit is required for all subjects. However, subjects wi... | [] |
NCT04183790 | 9.1.3 | Early Termination of Treatment | 9.1.3 Early Termination of Treatment If a subject prematurely discontinues study drug treatment, an ETT Visit should be scheduled as soon as possible after the decision to discontinue treatment. If the ETT Visit occurs 3 weeks or later following the last dose of study drug, then the ETT Visit will replace the Safety Fo... | [] |
NCT04183790 | 9.1.4 | Lost to Follow-up | 9.1.4 Lost to Follow-up A subject will be considered lost to follow-up if both of the following occur: - The subject misses 2 consecutive study visits (telephone contact and/or clinic visit) and is subsequently unable to be contacted by telephone (3 documented attempts by telephone within 2 weeks following the second m... | [] |
NCT04183790 | 9.1.5 | Completion of Study Participation | 9.1.5 Completion of Study Participation Completion of study participation for each individual subject is defined as: the Safety Follow-up Visit; or, in situations in which the ETT Visit or the Part A or B Week 96 Visit replaces the Safety Follow-up Visit (Section [9.1.2\)](#page-24-0), the ETT Visit or Part A or B Week... | [] |
NCT04183790 | 9.1.6 | Independent Data Monitoring Committee | 9.1.6 Independent Data Monitoring Committee This study will be monitored by an independent data monitoring committee (IDMC), which will conduct periodic reviews of safety data from the study (Section [12.3.6.2](#page-46-5)). Procedural details of the IDMC structure and function, frequency of meetings, and data planned ... | [] |
NCT04183790 | 9.2 | Method of Assigning Subjects to Treatment Groups | 9.2 Method of Assigning Subjects to Treatment Groups This is an open-label study. Randomization is not required because all subjects will be treated identically in a single cohort. An interactive web response system (IWRS) will be used to dispense dosage based on subject weight and age. | [] |
NCT04183790 | 9.3 | Rationale for Study Elements | 9.3 Rationale for Study Elements | [] |
NCT04183790 | 9.3.1 | Study Design | 9.3.1 Study Design This Phase 3 study will enroll subjects who completed the last treatment period visit in the parent study of VX-445/TEZ/IVA and meet eligibility criteria. Results from this study will provide information on the long-term safety and efficacy of TC treatment with VX-445/TEZ/IVA in subjects with CF who ... | [] |
NCT04183790 | 9.3.2 | Study Drug Dose and Duration | 9.3.2 Study Drug Dose and Duration To evaluate long-term safety, tolerability, and efficacy, the study drug doses evaluated are the same as the doses evaluated in the parent study. Subjects will receive a study drug dose appropriate for their weight, as described in Section [9.1.](#page-21-0) The overall treatment dura... | [] |
NCT04183790 | 9.3.3 | Rationale for Study Assessments | 9.3.3 Rationale for Study Assessments The safety, efficacy, and PD assessments are standard parameters for clinical studies in drug development and are generally recognized as reliable, accurate, and relevant to the study of subjects with CF. Baseline and follow-up ophthalmologic examinations are recommended for monito... | [] |
NCT04183790 | 9.4 | Study Restrictions | 9.4 Study Restrictions | [] |
NCT04183790 | 9.4.1 | Prohibited Medications | 9.4.1 Prohibited Medications [Table](#page-26-3) 9-2 lists prohibited medications. A non-exhaustive list of study prohibitions and cautions for medication will be provided in the Study Reference Manual. Guidance for concomitant medications is provided in Section [9.5.](#page-26-2) Table 9-2 Prohibited Medications | | T... | [] |
NCT04183790 | 9.5 | Prior and Concomitant Medications | 9.5 Prior and Concomitant Medications Information regarding prior and concomitant medications, including CF medications, other medications, and herbal and naturopathic remedies, will be collected in each subject's source documentation for medications taken within the 56 days before the first dose of study drug in this ... | [] |
NCT04183790 | 9.6 | Administration | 9.6 Administration | [] |
NCT04183790 | 9.6.1 | Dosing | 9.6.1 Dosing Study drug will be administered orally. Additional information is provided in the Pharmacy Manual. Study drug should be administered with a fat-containing meal or snack, such as a standard "CF" meal or snack or a standard meal. - 1. It is recommended that the dose be taken within 30 minutes of the start of... | [] |
NCT04183790 | 9.6.2 | Missed Doses | 9.6.2 Missed Doses If 6 hours or less have passed since the missed morning or evening dose, the subject should take the missed dose as soon as possible and continue on the original schedule. Morning dose: If more than 6 hours have passed since the missed morning dose, the subject should take the missed dose as soon as ... | [] |
NCT04183790 | 9.7 | Dose Modification for Toxicity | 9.7 Dose Modification for Toxicity Modifications of the study drug dose are prohibited. Should any unacceptable toxicity arise, individual subjects will be withdrawn from the study and dosing will cease. | [] |
NCT04183790 | 9.8 | Study Drug Interruption and Stopping Rules | 9.8 Study Drug Interruption and Stopping Rules In subjects who have interrupted study drug for >72 hours for any reason, the investigator should resume study drug only after a thorough investigation of the cause for interruption. The investigator will evaluate the subject's clinical stability and should consider resump... | [] |
NCT04183790 | 9.8.1 | Liver Function Tests | 9.8.1 Liver Function Tests The central laboratory will notify the medical monitor of alanine transaminase (ALT) or aspartate transaminase (AST) >3 × upper limit of normal (ULN) and total bilirubin >2 × ULN that are derived from centrally submitted samples. Subjects with new treatment-emergent ALT or AST elevations of >... | [] |
NCT04183790 | 9.8.2 | Rash | 9.8.2 Rash Individuals who develop a generalized rash will be monitored closely. Study drug dosing should be interrupted if a subject develops a generalized rash of Grade 3 or higher (Section [13.1.1.4\)](#page-47-1), or a rash that is considered a serious adverse event (SAE). The investigator will notify the medical m... | [] |
NCT04183790 | 9.9 | Removal of Subjects | 9.9 Removal of Subjects Subjects may withdraw from the study at any time at their own request. Subjects may be withdrawn from study drug treatment at any time at the discretion of the investigator or Vertex for safety, behavior, noncompliance with study procedures, or administrative reasons. In addition, a subject must... | [] |
NCT04183790 | 9.10 | Replacement of Subjects | 9.10 Replacement of Subjects Subjects who withdraw or are withdrawn during the study drug Treatment Period will not be replaced. | [] |
NCT04183790 | 10 | STUDY DRUG INFORMATION AND MANAGEMENT | 10 STUDY DRUG INFORMATION AND MANAGEMENT Study drug refers to VX-445/TEZ/IVA and IVA. | [] |
NCT04183790 | 10.1 | Preparation and Dispensing | 10.1 Preparation and Dispensing Study drug may be dispensed only under the supervision of the investigator or an authorized designee and only for administration to the study subjects. | [] |
NCT04183790 | 10.2 | Packaging and Labeling | 10.2 Packaging and Labeling Study drug tablets will be supplied in blister cards by Vertex. Study drug labeling will be in compliance with applicable local and national regulations. Additional details regarding packaging, labeling, and dispensing for study drug will be in the Pharmacy Manual. | [] |
NCT04183790 | 10.3 | Study Drug Supply, Storage, and Handling | 10.3 Study Drug Supply, Storage, and Handling Study drug will be supplied as film-coated tablets [\(Table](#page-31-4) 10-1). The investigator, or an authorized designee (e.g., a licensed pharmacist), will ensure that all investigational product is stored in a secured area, under recommended storage conditions, and in ... | [] |
NCT04183790 | 10.4 | Drug Accountability | 10.4 Drug Accountability The pharmacist or designated study site staff will maintain information about the dates and amounts of (1) study drug received; (2) study drug dispensed to the subjects; and (3) study drug returned by the subjects. Subjects will be instructed to return all used and unused materials associated w... | [] |
NCT04183790 | 10.5 | Disposal, Return, or Retention of Unused Drug | 10.5 Disposal, Return, or Retention of Unused Drug The study site staff or pharmacy personnel will retain all materials returned by the subjects until the study monitor has performed drug accountability. The investigator will ensure that the materials are destroyed in compliance with applicable environmental regulation... | [] |
NCT04183790 | 10.6 | Compliance | 10.6 Compliance To ensure treatment compliance, the investigator or designee will supervise all study drug dosing that occurs at the site. At each visit, site personnel will review that the subject is compliant with study drug dosing and remind the subject of study drug dosing requirements. Compliance will also be asse... | [] |
NCT04183790 | 10.7 | Blinding and Unblinding | 10.7 Blinding and Unblinding This will be an open-label study; however, subjects and their legally appointed and authorized representative (e.g., parent or legal guardian) should not be informed of their study-related spirometry and LCI, SwCl, FE-1, and IRT results until the study has been completed, regardless if the ... | [] |
NCT04183790 | 11 | ASSESSMENTS | 11 ASSESSMENTS The schedule of assessments is shown in [Table 3-1](#page-6-0) and [Table](#page-9-0) 3-2. | [] |
NCT04183790 | 11.1 | Subject and Disease Characteristics | 11.1 Subject and Disease Characteristics Subject and disease characteristics include the following: demographics, medical history, height, and weight. Select demographic and medical history will be derived from the parent study. | [] |
NCT04183790 | 11.2 | Pharmacokinetics | 11.2 Pharmacokinetics Not applicable. | [] |
NCT04183790 | 11.3 | Efficacy and Pharmacodynamics | 11.3 Efficacy and Pharmacodynamics | [] |
NCT04183790 | 11.3.1 | Spirometry | 11.3.1 Spirometry Spirometry will be performed according to the American Thoracic Society Guidelines[15](#page-55-15) and according to the additional guidelines that follow. Pre-bronchodilator spirometry is defined as spirometry testing performed for subjects who have - withheld their short-acting bronchodilators (e.g.... | [] |
NCT04183790 | 11.3.2 | Sweat Chloride | 11.3.2 Sweat Chloride The SwCl test is a standard diagnostic tool for CF, serving as a biomarker of CFTR activity. Sweat samples will be sent to a central laboratory for testing and interpretation of results. Individual SwCl test results will not be disclosed to the study sites. Specific instructions for collection, ha... | [] |
NCT04183790 | 11.3.3 | Multiple Breath Washout | 11.3.3 Multiple Breath Washout The N2-MBW testing will be performed in multiple replicates for each visit and the final LCI value will be calculated from the technically acceptable washout replicates by a central reader. The final LCI value at each visit will be the value provided by the LCI vendor based on the replica... | [] |
NCT04183790 | 11.3.4 | Height and Weight | 11.3.4 Height and Weight Height and weight will be measured with shoes off and before the dose of the study drug at each clinic visit during the Treatment Periods. | [] |
NCT04183790 | 11.3.5 | Cystic Fibrosis Questionnaire-Revised | 11.3.5 Cystic Fibrosis Questionnaire-Revised The questionnaires provide information about demographics; general quality of life, school, work, or daily activities; and symptom difficulties (pertaining to CF). Subjects/caregivers will be asked to complete the CFQ-R in their native language, if validated translations are... | [] |
NCT04183790 | 11.3.6 | Other Events Related to Outcome | 11.3.6 Other Events Related to Outcome | [] |
NCT04183790 | 11.3.6.1 | Antibiotic Therapy for Sinopulmonary Sign/Symptoms | 11.3.6.1 Antibiotic Therapy for Sinopulmonary Sign/Symptoms New or changed antibiotic therapy (intravenous [IV], inhaled, or oral) for the following sinopulmonary signs/symptoms will be determined and documented at visits as indicated in [Table 3-1](#page-6-0) and [Table](#page-9-0) 3-2: - Change in sputum - New or inc... | [] |
NCT04183790 | 11.3.6.2 | Hospitalization for CF | 11.3.6.2 Hospitalization for CF Subjects will be queried about planned and unplanned hospitalizations lasting ≥24 hours that occurred during the study. The dates of hospitalizations and the reasons for hospitalizations will be documented. For any hospitalization (planned and unplanned), the procedures for safety report... | [] |
NCT04183790 | 11.4 | Other Assessments | 11.4 Other Assessments These data will be used for internal exploratory purposes and may or may not be included in the clinical study report (CSR). Detailed procedures for the collection of blood samples will be provided in a separate document. | [] |
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