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protocol_id stringclasses 263 values	section_number stringlengths 1 12	title stringlengths 1 1.88k	content stringlengths 0 866k	merged_titles listlengths 0 491
NCT04183790	11.4.1	FE-1 and IRT	11.4.1 FE-1 and IRT Assessments of FE-1 and IRT will be conducted to assess exocrine pancreatic function. Fecal samples for assessment of FE-1 will be collected at the study center during the study visit; alternatively samples may be collected by the subject's caregiver up to 24 hours before the study visit (e.g., at h...	[]
NCT04183790	11.4.2	Blood Biomarkers	11.4.2 Blood Biomarkers Blood samples for blood biomarker analysis will be collected and banked for potential exploratory evaluation of correlations between blood biomarkers (e.g., proteins, peptides, lipids, vitamins, endogenous metabolites, and RNA) with PD, treatment benefit, and AEs. Specific instructions for the c...	[]
NCT04183790	11.5	Safety	11.5 Safety Safety evaluations will include reporting of AEs, clinical laboratory assessments, PEs, clinical evaluation of vital signs, pulse oximetry, standard 12-lead ECGs, and ophthalmologic examinations.	[]
NCT04183790	11.5.1	Adverse Events	11.5.1 Adverse Events All AEs will be assessed, documented, and reported in accordance with ICH GCP Guidelines. Section [13.1](#page-46-7) outlines the definitions, collection periods, criteria, and procedures for documenting, grading, and reporting AEs. Electronic AE case report form (CRF) completion guidelines for in...	[]
NCT04183790	11.5.2	Clinical Laboratory Assessments	11.5.2 Clinical Laboratory Assessments Blood and urine samples will be analyzed at a central laboratory with the exception of urine pregnancy tests, which will be analyzed locally. All blood samples will be collected while subjects are in a seated or supine position. Specific instructions for the collection, processing...	[]
NCT04183790	11.5.3	Physical Examinations and Vital Signs	11.5.3 Physical Examinations and Vital Signs A PE of all body systems and vital signs assessment will be performed at select study visits. At other visits, symptom-directed PEs and symptom-directed vital signs assessments can be performed at the discretion of the investigator or healthcare provider. A PE includes a rev...	[]
NCT04183790	11.5.4	Electrocardiograms	11.5.4 Electrocardiograms Standard 12-lead ECGs will be performed using a machine with printout. Additional standard 12-lead ECGs will be performed at any other time if clinically indicated. The performance of all ECGs will adhere to the following guidelines: - The ECG will be done before any other procedures that may ...	[]
NCT04183790	11.5.5	Ophthalmologic Examination	11.5.5 Ophthalmologic Examination Ophthalmologic examinations do not need to be completed if there is documentation of bilateral lens removal for the subject. All examinations will be conducted by a licensed ophthalmologist or optometrist and will include: - measurement of best-corrected distance visual acuity of each ...	[]
NCT04183790	11.5.6	Contraception and Pregnancy	11.5.6 Contraception and Pregnancy The effects of VX-445 monotherapy or in TC with TEZ and IVA on conception, pregnancy, and lactation in humans are not known. VX-445, TEZ, and IVA did not show genotoxic potential in a standard battery of in vitro (Ames test, chromosomal aberration, or micronucleus in cultured mammalia...	[]
NCT04183790	11.5.6.1	Contraception	11.5.6.1 Contraception Contraception requirement for a couple is waived for the following: - True abstinence for the subject, when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptab...	[ "Contraception requirement for a couple is waived for the following:", "Additional notes:" ]
NCT04183790	11.5.6.2	Pregnancy	11.5.6.2 Pregnancy Subjects will be counseled to inform the investigator of any pregnancy that occurs during study treatment and for 90 days after the last dose of study drug. If a subject, or the female partner of a male subject, becomes pregnant during study participation, study drug will be permanently discontinued ...	[]
NCT04183790	12	STATISTICAL AND ANALYTICAL PLANS	12 STATISTICAL AND ANALYTICAL PLANS This section presents a summary of the planned analyses for this protocol. Statistical analysis details will be provided in the statistical analysis plan (SAP), which will be finalized before the clinical data lock for the study.	[]
NCT04183790	12.1	Sample Size	12.1 Sample Size The primary and secondary objectives of the study are the evaluation of the long-term safety, tolerability, and efficacy of VX-445/TEZ/IVA. This is an open-label study that will enroll subjects who complete study treatment in the parent study and meet eligibility criteria. Approximately 56 subjects are...	[]
NCT04183790	12.2	Analysis Sets	12.2 Analysis Sets The following analysis sets will be defined for Part A and Part B separately: The Open-label All Subjects Set for Part A or B is defined as all subjects who were enrolled (defined as subject having data in the corresponding clinical database) in the corresponding Part. This analysis set will be used ...	[]
NCT04183790	12.3	Statistical Analysis	12.3 Statistical Analysis	[]
NCT04183790	12.3.1	General Considerations	12.3.1 General Considerations Continuous variables will be summarized using the following descriptive summary statistics: the number of subjects (n), mean, SD, median, minimum value (min), and maximum value (max). The precision of the measurement for each continuous variable will be specified in the SAP. Unless otherwi...	[]
NCT04183790	12.3.2	Background Characteristics	12.3.2 Background Characteristics	[]
NCT04183790	12.3.2.1	Subject Disposition	12.3.2.1 Subject Disposition Subject disposition will be summarized separately for each Part for the Open-label All Subjects Set. The number and percentage of subjects in the following categories will be summarized as appropriate: - Open-label All Subjects Set - Dosed (OL-SS) - Enrolled and dosed (OL-FAS) - Completed t...	[]
NCT04183790	12.3.2.2	Demographics and Baseline Characteristics	12.3.2.2 Demographics and Baseline Characteristics Demographics and baseline characteristics will be summarized separately for each Part by descriptive summary statistics. Baseline characteristics will be the same as the parent study baseline characteristics. The following demographics and baseline characteristics will...	[]
NCT04183790	12.3.2.3	Prior and Concomitant Medications	12.3.2.3 Prior and Concomitant Medications Medications will be coded using WHODrug and categorized as follows: - Prior medication: any medication that was administered within the 56 days before the first dose of study drug in each Part - Concomitant medication: medication continued or newly received during the TE Perio...	[]
NCT04183790	12.3.2.4	Study Drug Exposure and Compliance	12.3.2.4 Study Drug Exposure and Compliance Study drug exposure will be summarized separately for each Part based on the OL-SS, defined as the last day of study drug in each Part minus the first day of study drug in each Part plus 1, regardless of study drug interruption. Study drug compliance will be summarized based ...	[]
NCT04183790	12.3.2.5	Important Protocol Deviations	12.3.2.5 Important Protocol Deviations An important protocol deviation (IPD) is a deviation that may significantly affect the completeness, accuracy, or reliability of the study data or that may significantly affect a subject's rights, safety, or wellbeing. The rules for identifying an IPD will be described in the SAP....	[]
NCT04183790	12.3.3	Efficacy and Pharmacodynamic Analyses	12.3.3 Efficacy and Pharmacodynamic Analyses The secondary objective of the study is the evaluation of the long-term efficacy and PD of VX-445/TEZ/IVA.	[]
NCT04183790	12.3.3.1	Analysis of Primary Endpoint	12.3.3.1 Analysis of Primary Endpoint Not applicable. Efficacy and PD are not primary objectives.	[]
NCT04183790	12.3.3.2	Analysis of Secondary Efficacy and Pharmacodynamic Endpoints	12.3.3.2 Analysis of Secondary Efficacy and Pharmacodynamic Endpoints The secondary endpoints will be analyzed separately for each Part. • Absolute change in ppFEV1 One of the secondary efficacy endpoints is the absolute change from baseline in ppFEV1at visits in this open-label study, for subjects who receive at leas...	[ "• Absolute change in ppFEV1", "• Absolute change in SwCl", "• Absolute change in CFQ-R RD score", "• Absolute change in BMI and BMI-for-age z-score", "• Number of PEx and CF-related hospitalizations", "• Absolute change in LCI2.5", "• Absolute change in weight and weight-for-age z-score", "• Absolute...
NCT04183790	12.3.3.3	Multiplicity Adjustment	12.3.3.3 Multiplicity Adjustment Not applicable.	[]
NCT04183790	12.3.4	Safety Analysis	12.3.4 Safety Analysis The primary objective of the study is the evaluation of the long-term safety and tolerability of VX-445/TEZ/IVA. Data from Part A and Part B will be analyzed separately. For each Part, all safety analyses will be based on the TE Period for subjects in the OL-SS, unless otherwise specified. The ov...	[]
NCT04183790	12.3.4.1	Adverse Events	12.3.4.1 Adverse Events For analysis purposes, AEs will be classified as pretreatment AEs, TEAEs, or post-treatment AEs, defined as follows: - Pretreatment AE: any AE that occurred before the first dose of study drug in the TE Period in each Part - TEAE: any AE that worsened (either in severity or seriousness) or newly...	[]
NCT04183790	12.3.4.2	Clinical Laboratory Assessments	12.3.4.2 Clinical Laboratory Assessments For the treatment-emergent laboratory measurements, the observed values and change from baseline values of the continuous laboratory parameters will be summarized in SI units at each time point during the TE Period in each Part. The number and percentage of subjects with at leas...	[]
NCT04183790	12.3.4.3	Electrocardiogram	12.3.4.3 Electrocardiogram For the treatment-emergent ECG measurements, a summary of observed values and change from baseline values will be provided at each time point during the TE Period in each Part, for the following standard 12-lead ECG interval measurements (in msec): RR, PR, QT, QTc for heart rate (HR) interval...	[]
NCT04183790	12.3.4.4	Vital Signs	12.3.4.4 Vital Signs For the treatment-emergent vital signs measurements, the observed values and change from baseline values will be summarized at each time point during the TE Period in each Part. The following vital signs parameters will be summarized: systolic and diastolic blood pressure (mm Hg), body temperature ...	[]
NCT04183790	12.3.4.5	Pulse Oximetry	12.3.4.5 Pulse Oximetry For the treatment-emergent pulse oximetry measurements, a summary of observed values and change from baseline values will be provided at each time point during the TE Period in each Part, for the percent of oxygen saturation by pulse oximetry. The number and percentage of subjects with shift cha...	[]
NCT04183790	12.3.4.6	Physical Examination	12.3.4.6 Physical Examination No tables/figures/listings will be provided for PE data.	[]
NCT04183790	12.3.4.7	Other Safety Analyses	12.3.4.7 Other Safety Analyses Details of other safety analyses may be included in the SAP.	[]
NCT04183790	12.3.5	Other Analyses	12.3.5 Other Analyses Details of other analyses, including FE-1 and IRT, will be provided in a separate document.	[]
NCT04183790	12.3.6	Interim and IDMC Analyses	12.3.6 Interim and IDMC Analyses	[]
NCT04183790	12.3.6.1	Interim Analysis	12.3.6.1 Interim Analysis Interim analyses may take place at any time at the discretion of the sponsor.	[]
NCT04183790	12.3.6.2	IDMC Analysis	12.3.6.2 IDMC Analysis The IDMC (Section [9.1.6](#page-25-0)) will conduct regular safety reviews of study data as outlined in the IDMC charter. The IDMC's objectives, responsibilities, and operational details will be defined in a separate document (IDMC charter), which will be finalized before the first subject is enr...	[]
NCT04183790	13	PROCEDURAL, ETHICAL, REGULATORY, AND ADMINISTRATIVE CONSIDERATIONS	13 PROCEDURAL, ETHICAL, REGULATORY, AND ADMINISTRATIVE CONSIDERATIONS	[]
NCT04183790	13.1	Adverse Event and Serious Adverse Event Documentation, Severity Grading, and Reporting	13.1 Adverse Event and Serious Adverse Event Documentation, Severity Grading, and Reporting	[]
NCT04183790	13.1.1	Adverse Events	13.1.1 Adverse Events	[]
NCT04183790	13.1.1.1	Definition of an Adverse Event	13.1.1.1 Definition of an Adverse Event An AE is defined as any untoward medical occurrence in a subject during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or worsening of a pre-existing condition (e.g., increase in its severity or fre...	[]
NCT04183790	13.1.1.2	Clinically Significant Assessments	13.1.1.2 Clinically Significant Assessments Study assessments including laboratory tests, ECGs, PEs, and vital signs will be assessed and those deemed to have clinically significant worsening from baseline will be documented as an AE. When possible, a clinical diagnosis for the study assessment will be provided, rather...	[]
NCT04183790	13.1.1.3	Documentation of Adverse Events	13.1.1.3 Documentation of Adverse Events All AEs will be collected from the time the ICF is signed until the completion of study participation (Section [9.1.5\)](#page-24-6). All subjects' parents or legal guardians will be queried, using nonleading questions, about the occurrence of AEs at each study visit. When possi...	[]
NCT04183790	13.1.1.4	Adverse Event Severity	13.1.1.4 Adverse Event Severity The investigator will determine and record the severity of all serious and nonserious AEs. The guidance available at the following website will be consulted: Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0, Cancer Therapy Evaluation Program, [http://ctep.cancer.gov/pr...	[]
NCT04183790	13.1.1.5	Adverse Event Causality	13.1.1.5 Adverse Event Causality Every effort will be made by the investigator to assess the relationship of the AE, if any, to the study drug(s). Causality will be classified using the categories in [Table](#page-48-3) 13-2. Table 13-2 Classifications for AE Causality \| Classification \| Definition \| \|-----------------...	[]
NCT04183790	13.1.1.6	Study Drug Action Taken	13.1.1.6 Study Drug Action Taken The investigator will classify the study drug action taken with regard to the AE. The action taken will be classified according to the categories in [Table](#page-48-4) 13-3. Table 13-3 Classifications for Study Drug Action Taken With Regard to an AE \| Classificationa \| Definition \| \| \|...	[]
NCT04183790	13.1.1.7	Adverse Event Outcome	13.1.1.7 Adverse Event Outcome An AE will be followed until the investigator has determined and provided the final outcome. The outcome will be classified according to the categories in [Table](#page-49-4) 13-4. Table 13-4 Classifications for Outcome of an AE \| Classification \| Definition \| \|---------------------------...	[]
NCT04183790	13.1.1.8	Treatment Given	13.1.1.8 Treatment Given The investigator ensures adequate medical care is provided to subjects for any AEs, including clinically significant laboratory values related to study drug. In addition, the investigator will describe whether any treatment was given for the AE. "Yes" is used if any treatment was given in respo...	[]
NCT04183790	13.1.2	Serious Adverse Events	13.1.2 Serious Adverse Events	[]
NCT04183790	13.1.2.1	Definition of a Serious Adverse Event	13.1.2.1 Definition of a Serious Adverse Event An SAE is any AE that meets any of the following outcomes: - Fatal (death, regardless of cause, that occurs during participation in the study or occurs after participation and is suspected of being a delayed toxicity due to administration of the study drug) - Life-threaten...	[]
NCT04183790	13.1.2.2	Reporting and Documentation of Serious Adverse Events	13.1.2.2 Reporting and Documentation of Serious Adverse Events All SAEs that occur after obtaining informed consent and assent (where applicable) through the Safety Follow-up Visit, regardless of causality, will be reported by the investigator to Vertex GPS within 24 hours of identification. In addition, all SAEs that ...	[]
NCT04183790	13.1.2.3	Expedited Reporting and Investigator Safety Letters	13.1.2.3 Expedited Reporting and Investigator Safety Letters Vertex, as study sponsor, is responsible for reporting suspected, unexpected, serious adverse reactions (SUSARs) involving the study drug(s) to all regulatory authorities, IECs, and participating investigators in accordance with ICH Guidelines and/or local re...	[]
NCT04183790	13.2	Administrative Requirements	13.2 Administrative Requirements	[]
NCT04183790	13.2.1	Ethical Considerations	13.2.1 Ethical Considerations The study will be conducted in accordance with the current ICH GCP Guidelines, which are consistent with the ethical principles founded in the Declaration of Helsinki, and in accordance with local applicable laws and regulations. The IRB/IEC will review all appropriate study documentation ...	[]
NCT04183790	13.2.2	Subject Information and Informed Consent	13.2.2 Subject Information and Informed Consent After the study has been fully explained, written informed consent will be obtained from the subject or legal representative or guardian (if applicable), and assent will be obtained from the subject (if applicable), before study participation. The method of obtaining and ...	[]
NCT04183790	13.2.3	Investigator Compliance	13.2.3 Investigator Compliance No modifications to the protocol will be made without the approval of both the investigator and Vertex. Changes that significantly affect the safety of the subjects, the scope of the investigation, or the scientific quality of the study (i.e., efficacy assessments) will require IRB/IEC no...	[]
NCT04183790	13.2.4	Access to Records	13.2.4 Access to Records The investigator will make the office and/or hospital records of subjects enrolled in this study available for inspection by Vertex or its representative at the time of each monitoring visit and for audits. The records will also be available for direct inspection, verification, and copying, as ...	[]
NCT04183790	13.2.5	Subject Privacy	13.2.5 Subject Privacy To maintain subject confidentiality and to comply with applicable data protection and privacy laws and regulations, all data provided to Vertex, study reports, and communications relating to the study will identify subjects by assigned subject numbers, and access to subject names linked to such n...	[]
NCT04183790	13.2.6	Record Retention	13.2.6 Record Retention The investigator will maintain all study records according to ICH GCP Guidelines and/or applicable local regulatory requirement(s), whichever is longest, as described in the Clinical Trial Agreement. If the investigator withdraws from the responsibility of keeping the study records, custody will...	[]
NCT04183790	13.2.7	Study Termination	13.2.7 Study Termination At any time, Vertex may terminate this study in its entirety or may terminate this study at any particular site. In addition, for reasonable cause, either the investigators or their IRBs/IECs may terminate the study at their center. Conditions that may lead to reasonable cause and warrant termi...	[]
NCT04183790	13.2.8	End of Study	13.2.8 End of Study The end of study is defined as the last scheduled visit (or scheduled contact) of the last subject.	[]
NCT04183790	13.3	Data Quality Assurance	13.3 Data Quality Assurance Vertex or its designated representative will conduct a study site visit to verify the qualifications of each investigator, inspect clinical study site facilities, and inform the investigator of responsibilities and procedures for ensuring adequate and correct study documentation. Vertex will...	[]
NCT04183790	13.4	Monitoring	13.4 Monitoring The study will be monitored by Vertex or its designee in accordance with written procedures. Monitoring and auditing procedures developed or approved by Vertex for these activities comply with GCP regulatory requirements and guidelines. The monitoring strategy may include onsite, remote, and central mon...	[]
NCT04183790	13.5	Electronic Data Capture	13.5 Electronic Data Capture Sites will use an EDC tool to record data for each enrolled subject. It is the investigator's responsibility to ensure the accuracy, completeness, clarity, and timeliness of the data reported. The investigator is required to prepare and maintain adequate and accurate case histories designed...	[]
NCT04183790	13.6	Confidentiality and Disclosure	13.6 Confidentiality and Disclosure Any and all scientific, commercial, and technical information disclosed by Vertex in this protocol or elsewhere will be considered the confidential and proprietary property of Vertex. The investigator shall hold such information in confidence and shall not disclose the information to...	[]
NCT04183790	13.7	Publications and Clinical Study Report	13.7 Publications and Clinical Study Report	[]
NCT04183790	13.7.1	Publication of Study Results	13.7.1 Publication of Study Results Vertex is committed to reporting the design and results of all clinical studies in a complete, accurate, balanced, transparent, and timely manner, consistent with Good Publication Practices (GPP3).[23](#page-56-6) Publication Planning: Vertex staff along with the lead principal inves...	[]
NCT04183790	13.7.2	Clinical Study Report	13.7.2 Clinical Study Report A CSR, written in accordance with the ICH E3 Guideline, will be submitted in accordance with local regulations.	[]
NCT04183790	14	REFERENCES	14 REFERENCES - 1 Cystic Fibrosis Foundation. What is cystic fibrosis? Available at: [https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/.](https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/) Accessed 22 March 2018. - 2 Cystic Fibrosis Foundation. Patient Registry: 2017 Annual Data Report. Bethesda, MD: Cystic Fib...	[]
NCT04183790	15	PROTOCOL SIGNATURE PAGES	15 PROTOCOL SIGNATURE PAGES	[]
NCT04183790	15.1	Sponsor Signature Page	15.1 Sponsor Signature Page \| Protocol#: \| VX19-445-107 \| Version \| 2.0 \| VersionDate: \| 10 June2021 \| \|----------------------------------------------------------\|-----------------------------------------------\|--------------------------------------\|-------------------------\|-------------------------------------------\|...	[]
NCT04183790	15.2	Investigator Signature Page	15.2 Investigator Signature Page \| Protocol #: \| VX19-445-107 \| Version #: \| 2.0 \| Version Date: \| 10 June2021 \| \| \| \| \|----------------------\|---------------------------------------------------------------------------------------------------------------------------------------------------------------------------------...	[]
NCT04188379	1	INTRODUCTION	1. INTRODUCTION Background Information Efgartigimod (ARGX-113) is a modified human immunoglobulin (Ig) G1-derived crystallized fragment (Fc) of the za allotype that binds with nanomolar affinity to the human neonatal crystallized fragment receptor (FcRn). Efgartigimod encompasses IgG1 residues D221-K447 (EU numbering ...	[ "Background Information", "Benefit-Risk Assessment", "Trial Rationale" ]
NCT04188379	2	TRIAL OBJECTIVES	2. TRIAL OBJECTIVES Primary Objective x To evaluate the efficacy of efgartigimod compared to placebo in achieving a sustained platelet count response in patients with primary chronic ITP, with a sustained platelet count response defined as platelet counts of at least 50×109 /L for at least 4 of the 6 visits between we...	[ "Primary Objective", "Secondary Objectives", "Exploratory Objective" ]
NCT04188379	3	TRIAL ENDPOINTS	3. TRIAL ENDPOINTS Primary Endpoint x Proportion of patients with chronic ITP with a sustained platelet count response defined as achieving platelet counts of at least 50×109 /L for at least 4 of the 6 visits between week 19 and 24 of the trial. Patients who discontinue treatment prior to visit 24 due to lack of effic...	[ "Primary Endpoint", "Secondary Endpoints", "Key Secondary Efficacy Endpoints Subject to Alpha Control:", "Other Secondary Endpoints Not Subject to Alpha Control:", "Safety Evaluation", "Exploratory Endpoint" ]
NCT04188379	4	INVESTIGATIONAL PLAN	4. INVESTIGATIONAL PLAN Summary of Trial Design DESCRIPTION This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallelgroup, up to 31-week trial to evaluate the efficacy, safety, and impact on QoL of efgartigimod 10 mg/kg IV treatment in adult patients with primary ITP. The total maximum...	[ "Summary of Trial Design", "DESCRIPTION", "SAMPLE SIZE AND STRATIFICATION", "SCREENING AND TREATMENT", "ROLLOVER TO THE OPEN-LABEL EXTENSION TRIAL (ARGX-113-1803)", "CONCURRENT ITP THERAPY", "RESCUE THERAPY", "EARLY DISCONTINUATION FROM THE TRIAL", "TRIAL END", "Discussion of Trial Design", "Sel...
NCT04188379	4.3.1	Inclusion Criteria	4.3.1. Inclusion Criteria Patients will be randomized in this trial only if they meet all of the following criteria: - 1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the...	[]
NCT04188379	4.3.2	Exclusion Criteria	4.3.2. Exclusion Criteria Patients will not be enrolled in this trial if they meet any of the following criteria: argenx BVBA Confidential Page 49 of 162 - 1. ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocyto...	[ "Early Discontinuation" ]
NCT04188379	4.4.1	Early Discontinuation From Trial	4.4.1. Early Discontinuation From Trial Early discontinuation from the trial is defined as the permanent cessation of further participation in the trial prior to its planned completion and without the possibility to roll over to the open-label extension trial (ARGX-113-1803). The reason for early discontinuation from t...	[]
NCT04188379	4.4.2	Early Discontinuation From Treatment	4.4.2. Early Discontinuation From Treatment Early discontinuation from treatment means that the patient stops receiving the ongoing IMP treatment and does not restart IMP treatment, however, informed consent is not withdrawn. These patients will continue the weekly trial visits as specified in the SoA (Table 1) without...	[ "Temporary Withholding Treatment", "Protocol Deviations", "Screen Failures, Rescreening, and Retesting", "Early Termination of Trial or Site", "End-of-Trial Definition" ]
NCT04188379	5	TRIAL PROCEDURES	5. TRIAL PROCEDURES Every effort should be made to ensure that the protocol-required tests and procedures are completed as described. When a protocol-required procedure cannot be performed, the investigator will document the reason, and any corrective and preventive actions that he/she has taken to ensure that the norm...	[ "Informed Consent", "Screening", "Randomization", "Treatment Period" ]
NCT04188379	5.4.1	Screening and Treatment	5.4.1. Screening and Treatment During the screening period (up to 2 weeks) the patient's eligibility for trial participation will be evaluated. All eligible patients will be randomized to receive IV infusions of either efgartigimod 10 mg/kg body weight or matching placebo throughout the trial. All patients will initial...	[]
NCT04188379	5.4.2	Rollover to the Open-Label Extension Trial (ARGX-113-1803)	5.4.2. Rollover to the Open-Label Extension Trial (ARGX-113-1803) Patients who complete the 24-week trial period have the possibility to enter the open-label extension trial (ARGX-113-1803) to receive efgartigimod 10 mg/kg IV. The platelet counts from the ARGX-113-1801 trial will be taken into account to assess the dos...	[ "Follow-up Period", "Unscheduled Visit" ]
NCT04188379	6	TRIAL TREATMENT	6. TRIAL TREATMENT Treatment Administered The IMP infusions (efgartigimod or placebo) will be administered weekly from visits 1 to 4, either weekly or q2w from visits 5 to 16, and fixed on the dosing schedule of visit 16 (or the last visit at which IMP was administered) from visits 17 to 24 (ie, either weekly or q2w)....	[ "Treatment Administered", "Identity of Investigational Medicinal Product", "Packaging and Labeling", "Storage of Investigational Medicinal Products", "Method of Assigning Patients to Treatment Group", "Dosing Administration for Each Patient", "Blinding" ]
NCT04188379	6.7.1	Emergency Unblinding	6.7.1. Emergency Unblinding The process of breaking the blind will be handled through the IRT. Investigators are strongly discouraged from breaking the blind for an individual patient, unless there is a patient safety issue that via knowledge of the IMP treatment assignment would change patient management. The investig...	[ "Prior Treatments and Concomitant Medications" ]
NCT04188379	6.8.1	Concurrent ITP Therapy	6.8.1. Concurrent ITP Therapy Patients receiving at least 1 permitted concurrent ITP therapy are eligible for the trial, if the dose and schedule have remained unchanged in the last 4 weeks before randomization (ie, visit 1). Permitted concurrent ITP medications include oral corticosteroids, oral immunosuppressants, da...	[]
NCT04188379	6.8.2	Prohibited Medications and Therapy Prior to Randomization	6.8.2. Prohibited Medications and Therapy Prior to Randomization The following medications or treatments are not permitted within the specified time window prior to randomization: - x Use of anticoagulants (eg, vitamin K antagonists, direct oral anticoagulants) within 4 weeks prior to randomization. - x Use of any tran...	[]
NCT04188379	6.8.3	Prohibited Medications and Therapy During the Trial	6.8.3. Prohibited Medications and Therapy During the Trial The following medications or treatments are not permitted during the trial: - x Anti-CD20 therapy (eg, rituximab) - x Romiplostim - x Any monoclonal antibodies or Fc fusion proteins or investigational drug - x Live/live-attenuated vaccines	[]
NCT04188379	6.8.4	Rescue Therapy	6.8.4. Rescue Therapy "Rescue therapy" is defined as an occurrence where the patient needs treatment with 1 or more rescue treatments. An "occurrence" is defined as a period of maximum 5 days where 1 or more rescue treatments are administered simultaneously or consecutively to the trial patient. The start date/time of ...	[ "Medical Care of Patients After End-of-Treatment or Early Discontinuation", "Treatment Compliance", "Handling Missed Doses of the Investigational Medicinal Product", "Accountability of Investigational Medicinal Product", "Storage of Blood Samples in the Trial" ]
NCT04188379	7	TRIAL ASSESSMENTS	7. TRIAL ASSESSMENTS Demography Demographic characteristics comprise age, year of birth, gender, race, and ethnicity (per local regulations). Efficacy Procedures assessing the efficacy of efgartigimod will mainly focus on measures of response derived from platelet counts. These assessments will be performed at the we...	[ "Demography", "Efficacy" ]
NCT04188379	7.2.1	Platelet Count	7.2.1. Platelet Count The assessment of platelet count can be performed within 1 day prior to any other trialspecific assessment (except at screening, where the informed consent should be obtained and weight assessed first, and at randomization) at each visit as specified in the SoA (Table 1). The samples will be analy...	[]
NCT04188379	7.2.2	General Bleeding Assessment	7.2.2. General Bleeding Assessment Signs and symptoms of bleeding are the predominant clinical manifestation of ITP and are typically related to low platelet counts. The WHO bleeding scale is a widely recognized tool to assess bleeding and has been used in many previous ITP trials. The occurrence and severity of any bl...	[ "Safety" ]
NCT04188379	7.3.1	Adverse Events	7.3.1. Adverse Events Definition of AE An AE is any untoward medical occurrence in a clinical trial patient, temporally associated with the use of IMP, whether or not considered related to the medicinal product or IMP. Note: An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory f...	[ "Definition of AE", "Definition of SAE", "Overdose or Medication Error", "Severity", "Relationship" ]
NCT04188379	7.3.1.1	Adverse Events of Special Interest	7.3.1.1. Adverse Events of Special Interest An AESI (serious or non-serious, related or not related) is an event of scientific and medical concern specific to the sponsor's product or program (eg, an underlying condition being investigated, a mechanism of action like potential immunosuppression, etc). Further character...	[]
NCT04188379	7.3.1.2	Reporting of Adverse Events and Serious Adverse Events	7.3.1.2. Reporting of Adverse Events and Serious Adverse Events All AEs that occur during the trial, from signature of the ICF until the last trial-related activity are to be recorded on the appropriate AE pages (either "serious" or "non-serious") of the eCRF. The investigator should complete all the details requested,...	[]
NCT04188379	7.3.1.3	Reporting of Serious Adverse Events to Regulatory Authorities and Investigators	7.3.1.3. Reporting of Serious Adverse Events to Regulatory Authorities and Investigators The sponsor's designee will be responsible for reporting all suspected ADRs that are both serious and unexpected, and any other applicable reports to regulatory authorities, IRB/IEC, and investigators, in accordance with national r...	[]
NCT04188379	7.3.1.4	Follow-up of Adverse Events and Serious Adverse Events	7.3.1.4. Follow-up of Adverse Events and Serious Adverse Events Any AEs observed from signing the ICF to the last trial-related activity will be followed-up until resolution, until the patient is lost to follow-up, or until the patient withdraws consent. Resolution means that the patient has returned to a baseline stat...	[]
NCT04188379	7.3.1.5	Reporting and Follow-up Requirements for Pregnancies	7.3.1.5. Reporting and Follow-up Requirements for Pregnancies	[]
NCT04188379	7.3.1.5.1	Pregnancies in Female Patients	7.3.1.5.1. Pregnancies in Female Patients A serum pregnancy test will be performed centrally at screening. A urine pregnancy test will be conducted and analyzed locally at the visits detailed in the SoA (Table 1). argenx BVBA Confidential Page 73 of 162 If a patient becomes pregnant during the administration of IMP and...	[]

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