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NCT05118386
10.1.8
Source Documents
Source documentation consists of existing medical records and/or study records developed and maintained by the investigator. Source documents provide evidence for the existence of the participant and substantiate the integrity of the data collected. Data recorded on source documents will be transcribed onto electronic...
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NCT05118386
10.1.9
Record Retention
Records and documents pertaining to the conduct of this study must be retained by the investigator for a minimum of 10 years after study completion unless local regulations or institutional policies require a longer retention period. No records may be destroyed during the retention period without the written approval ...
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NCT05118386
10.1.10
Study and Site Closure
The sponsor reserves the right to close the study site(s) or terminate the study at any time for any reason at their sole discretion. Study sites will be closed upon study completion. A study site is considered closed when all required documents and study supplies have been collected and a study site closure visit has ...
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NCT05118386
10.1.11
Publication Policy
The results of this study may be published or presented at scientific meetings. If this is foreseen, the investigator agrees to submit all manuscripts or abstracts to the sponsor before submission. This allows the sponsor to protect proprietary information and to provide comments. The sponsor will comply with the requ...
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NCT05118386
10.2
Appendix 2: Adverse Events: Definitions and Procedures for Recording, Evaluating, Follow-up, and Reporting
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NCT05118386
10.2.1
Definition of AE
An AE is any untoward medical occmTence in a patient or clinical study pa1ticipant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. NOTE: An AE can therefore be any unfavorable and unintended sign (including an abno1mal laborato1y finding), sympt...
[ "AE Definition", "Events Meeting the AE Definition", "Events Meeting the AE Definition" ]
NCT05118386
10.2.2
Definition of SAE
If an event is not an AE per definition above, then it cannot be an SAE even if serious conditions are met (e.g., hospitalization for signs/symptoms of the disease under study, death due to progression of disease). Results in death Is life-threatening The te1m 'life-threatening' in the definition of 'serious' refers...
[ "A SAE is defined as any untoward medical occurrence that, at any dose:" ]
NCT05118386
10.2.3
Definition of Serious Adverse Drug Reaction (Serious ADR)
When an AE is judged to be serious and related to an investigational product, it is a Serious Adverse Drng Reaction and is subject to expedited repo1iing based on the parameters of this study.
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NCT05118386
10.2.4
Definition of AESI
AESis are AEs that the sponsor will carefully monitor. The following AEs will be collected and repo1ied as AESis: anaphylaxis or hypersensitivity reactions, and/or infusion reactions resulting in pennanent discontinuation of study inte1vention infusion during IV administration.
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NCT05118386
10.2.5
Recording and Follow-Up of AE, SAE, AESI
Care will be taken not to introduce bias when detecting AEs, SAEs and AESis. Open-ended and non-leading verbal questioning of the pa1iicipant is the prefe1Ted method to inquire about AE occurrences. When an AE/SAE/ AES! occurs, it is the responsibility of the investigator to review all available documentation ( e.g....
[ "Recording", "Follow-up and Resolution" ]
NCT05118386
10.2.5.1
Assessment of Intensity
The investigator will assess the intensity of each AE reported during the study. In the case of local reactions, vital signs, systemic events, and laboratory abnormalities, the Grades delineated in Section 10.4, Appendix 4 will be employed. In the case of an AE or abnormal clinical laboratory result not included in Ap...
[ "Grade 4 Life-threatening consequences; urgent intervention indicated", "Notes:" ]
NCT05118386
10.2.5.2
Assessment of Causality
The investigator is obligated to assess the relationship between study intervention and each occurrence of each AE/SAE/AESI. Only AEs that are local reactions to an IM injection are automatically assumed to be related to the study intervention, and therefore are not required to have causality assessed by the investigat...
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NCT05118386
10.2.5.3
Assessment of Expectedness
Not applicable
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NCT05118386
10.2.5.4
Assessment of Outcome
The outcome of each SAE/AESI must be reported. For analysis purposes, the outcome for serious adverse events will be determined on the final study visit. Outcome of all AEs will be classified as one of the following: •Resolved - Resolved with sequelae - •Ongoing - •Death .
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NCT05118386
10.2.6
SAEs, AESis, and Serious ADR Reporting
The primary mechanism for reporting an SAE or AESI by the investigator to the sponsor or delegate will be the electronic data collection tool. The site will enter the SAE or AESI data into the electronic system as soon as it is identified. All SAEs (related and unrelated) and AESis are reported to the sponsor or de...
[ "Reporting to Sponsor or Delegate Via the Electronic Data Collection Tool", "Reporting via Paper CRF" ]
NCT05118386
10.3
Appendix 3: Contraceptive Guidance and Collection of Pregnancy Information
Male paiiicipants with pa1iners of childbearing potential must agree to use condoms during the study and for 90 days after the study, and they must refrain from spe1m donation for at least 90 days after the end of study. Female pa1iicipants of childbeai·ing potential must not be pregnant, breastfeeding, or attemptin...
[ "Contraceptive Guidance:", "Collection of Pregnancy Information:" ]
NCT05118386
10.4
Appendix 4: Toxicity Tables
The following toxicity tables are extracted from the FDA Guidance for Industry [FDA 2007]. Table 8: FDA Toxicity Grading Scale- Clinical Abnormalities | Local Reaction toInjectable Product | Mild(Grade 1) | Moderate(Grade 2) | S...
[ "Local Reactions", "Vital Signs" ]
NCT05118386
11
References
AstraZeneca. Nirsevimab MELODY Phase III trial met primary endpoint of reducing RSV lower respiratory tract infections in healthy infants. 26 April 2021. https://www.astrazeneca.com/media-centre/press-releases/2021/nirsevimab-phase-iii-trial-metprimary-endpoint.html Aliprantis A, Wolford D, Caro L, Maas BM, et al. A ...
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NCT05118386
12
Protocol Version History
The Protocol Version updates and amendment summary of changes is provided below. - Version 1, dated 26 May 2021 - Version 1.1, dated 09 June 2021 - Version 2.0, dated 20 July 2021 - Version 3.0, dated 10 December 2021 - Version 4.0, dated 21 January 2022 Rationale for changes to V1: The update from Version 1 to Versi...
[ "Version 1.1, dated 09 June 2021", "Diary Card", "Memory aid", "Version 2.0, dated 20 July 2021", "Complete List of Changes:", "Document History", "Synopsis:", "Dose Expansion Phase:", "Synopsis", "Pausing Rules: Pausing rules include:", "new vers10n", "Schedule of Activities (SoA)", "Table ...
NCT04230876
A1
Study Abstract
A1 Study Abstract Auditory training as the potential to dramatically affect older persons' adjustment to a new hearing aid and to maximize the benefits they receive from wearing one. In turn, by wearing hearing aids, they experience easier and more successful communication patterns. They enhance their ability to engage...
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NCT04230876
A2
Primary Hypothesis
A2 Primary Hypothesis Use of the web-based clEAR auditory brain training system with concomitant support from a clEAR in-house audiologist improves satisfaction with new hearing aids and increases daily use time.
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NCT04230876
A3
Purpose of the Study Protocol
A3 Purpose of the Study Protocol Determine the extent to which web-based clEAR auditory brain training with concomitant support from a clEAR in-house audiologist affects satisfaction with new hearing aids and increases daily use time.
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NCT04230876
B
Background
B Background B1 Prior Literature and Studies Barcroft, J., Sommers, M., Tye-Murray, N., Mauzé, E., Schroy, C., & Spehar, B. (2011). Tailoring auditory training to patient needs with single and multiple talkers: Transfer-appropriate gains on a four-choice discrimination test. International Journal of Audiology, 50(11), ...
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NCT04230876
B2
Rationale for this Study
B2 Rationale for this Study clEAR auditory brain training has been shown to be effective and that patients like the contact with an audiologist during training. Barcroft et al. (2011; see also Tye-Murray et al., 2017) showed that computerized, game-like auditory training is beneficial for older adults (mean age=66 year...
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NCT04230876
C
Study Objectives
C Study Objectives
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NCT04230876
C1
Primary Aim
C1 Primary Aim It is the objective of this study to determine if the use of clEAR following a first-time hearing aid fitting will increase satisfaction and daily hearing aid use.
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NCT04230876
C2
Secondary Aim
C2 Secondary Aim N'A
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NCT04230876
C3
Rationale for the Selection of Outcome Measures
C3 Rationale for the Selection of Outcome Measures - 1. Primary outcome measures are questionnaires used in hearing clinics to assess hearing aid satisfaction. - 2. Hearing aids log use time. This information will also be collected to determine the average daily use time for each participant.
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NCT04230876
D
Investigational Agent
D Investigational Agent D1 Preclinical Data N/A D2 Clinical Data to Date N/A D3 Dose Rationale and Risk/Benefits N/A
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NCT04230876
E
Study Design
E Study Design
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NCT04230876
E1
Overview or Design Summary
E1 Overview or Design Summary All qualified participants will first be fitted with one hearing aid using manufacturer recommended settings. The settings will be verified using real-ear measurements and any initial adjustments to the hearing aid will be made at the time of fitting. The participants will return again in ...
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NCT04230876
E2
Subject Selection and Withdrawal
E2 Subject Selection and Withdrawal 2.a Inclusion Criteria All participants have the same inclusion/exclusion criteria but be assigned to one of two groups that differ only in the schedule, this will allow participants will act as their own control group. All participants will: - 1. Be over 60 years old - 2. Have a bi...
[ "2.a Inclusion Criteria", "2.a Exclusion Criteria", "2.b Ethical Considerations", "2.c Subject Recruitment Plans and Consent Process", "2.d Randomization Method and Blinding", "2.e Risks and Benefits", "2.f Early Withdrawal of Subjects", "2.g When and How to Withdraw Subjects", "2.h Data Collection ...
NCT04230876
F
Study Procedures
F Study Procedures
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NCT04230876
F1
Screening for Eligibility
F1 Screening for Eligibility A telephone survey will screen all potential participants that would not be eligible for reasons other than the hearing and vision screening. Hearing and vision testing will be conducted after the informed consent process.
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NCT04230876
F2
Schedule of Measurements
F2 Schedule of Measurements | F3 | Visit 1 | |-----|------------------------------------------------| | F4 | Visit 2 etc. | | F5 | Safety and Adverse Events | | 5.a | Safety and Compliance Monitoring | | 5.b | Medical Monitoring | | i | Investigator only | | ii | Independent expert to monitor | | iii | Institutional Da...
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NCT04230876
G
Statistical Plan
G Statistical Plan
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NCT04230876
G1
Sample Size Determination and Power
G1 Sample Size Determination and Power We determined the power to detect an effect size for differences as large as or larger than in Tye-Murray et al. (mean = .733, SD = .783) with an alpha level of .05 will exceed .95 using a one-tailed test for the within-subjects comparisons in each group.
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NCT04230876
G2
Interim Monitoring and Early Stopping
G2 Interim Monitoring and Early Stopping We will monitor progress in the software to be sure participants are following protocol.
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NCT04230876
G3
Analysis Plan
G3 Analysis Plan We will assess the effect of training on hearing aid use time, changes in speech perception, and subjective changes indicated by the questionnaire rating-scale responses. Three post-fitting times will be assessed to look at the effects of clEAR testing (Baseline/post-fitting, post-training, post contro...
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NCT04230876
G4
Statistical Methods
G4 Statistical Methods Data will be analyzed with t-tests and repeated measures ANOVA.
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NCT04230876
G5
Missing Outcome Data
G5 Missing Outcome Data Participants with missing data will not be included in the analyses for that outcome measure.
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NCT04230876
G6
Unblinding Procedures
G6 Unblinding Procedures N/A
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NCT04230876
H
Data Handling and Record Keeping
H Data Handling and Record Keeping | | Confidentiality and Security | |----|-----------------------------------------------------------------------------------| | H2 | Training | | H3 | Case Report Forms and Source Documents | | H4 | Records Retention | | H5 | Performance Monitoring | | | | | I | Study Monitoring, Audi...
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NCT04230876
J5
Study Timetable
J5 Study Timetable
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NCT04230876
K
Publication Plan
K Publication Plan
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NCT04230876
L
Attachments
L Attachments
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NCT04230876
L1
Tables
L1 Tables
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NCT04230876
L2
Informed consent documents
L2 Informed consent documents | Adult SBIR HA and AT Informed | Consent & Assent | 1 | 407 k | E | 08/20/19 | |-------------------------------|------------------|---|-------|---|----------| | | | | | | | | consent.rtf | Forms | | | | |
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NCT04230876
L3
Patient education brochures
L3 Patient education brochures
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NCT04230876
L4
Special procedures protocols
L4 Special procedures protocols
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NCT04230876
L5
Questionnaires or surveys
L5 Questionnaires or surveys | Attachment Name | Category | Ver | Size | | Attached | |-----------------|-----------------------------------------------|-----|------|---|----------| | JIT Letter.doc | Notice of Just in Time (JIT)Documentation | 1 | 25 k | E | 08/15/19 | | Telephone | Recruitment Script: Phone | 1 | 59 ...
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NCT04230876
M
References
M References Barcroft, J., Sommers, M., Tye-Murray, N., Mauzé, E., Schroy, C., & Spehar, B. (2011). Tailoring auditory training to patient needs with single and multiple talkers: Transfer-appropriate gains on a four-choice discrimination test. International Journal of Audiology, 50(11), 1802–1808. Barcroft, J., Spehar,...
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NCT01283542
7.5
Safety
7.5 Safety - Added new section 7.5.2.3 Liver safety monitoring. This section describes how to treat patients who experience liver function increases especifically defined by the protocol - Added Figure 7-3 graphically describing liver safety monitoring procedures outlined on section 7.5.2.3
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NCT01283542
7.5.5.2
Coagulation
7.5.5.2 Coagulation Added further timepoints for the evaluation of PT coagulation parameter for patients treated with pasireotide LAR
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NCT01283542
7.5.5.3
Chemistry
7.5.5.3 Chemistry - Definition of liver function test panel and addition of further timepoints for liver function evaluations for patients treated with pasireotide LAR - 7.5.7 Radiology tests - Clarification that an abdominal ultrasound is required in case of abnormal liver function criteria - Table 7.2 Blood collectio...
[ "Rationale for the Amendment", "Changes to the protocol", "Rationale for the amendment", "Changes to the protocol", "REC Approval", "Synopsis of the oncology clinical trial protocol", "Endpoints (efficacy, safety)", "Primary Objective Endpoint:", "Secondary Objectives Outcomes:", "Extension Phase:...
NCT01283542
7
Visits and evaluations schedule
7 Visits and evaluations schedule Tables 7-1 and 7-2 show a list of all evaluations for the Main Phase and the Extension Phase, respectively, and indicate with an "X" the visits to be performed. All data obtained from these evaluations must be confirmed in the patient's source-documentation. The table indicates which d...
[ "The pasireotide dose in this visit will be administered to selected patients, at the physician's discretion, if the patient is included in the Extension Phase, based on the response evaluated by the investigator to the benefit from the drug (Section 6.4)." ]
NCT01283542
7.1
Information to be collected in case of screening failure
7.1 Information to be collected in case of screening failure Patients who do not meet eligibility requirements on Visit 1 are considered as screening failures. If a patient cannot start treatment for any reason, this reason must be entered in the Screening Record eCRF, and the patient's demographics will be entered in ...
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NCT01283542
7.2
Demographics / other baseline patient characteristics
7.2 Demographics / other baseline patient characteristics Patients' demographics and baseline characteristics will include the following: demographics, diagnosis and disease extent, relevant medical history / current medical conditions, previous / concurrent medication administered, physical examination, including vita...
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NCT01283542
7.3
Treatments
7.3 Treatments The study medication administered during the study must be documented in the Dose Administration eCRF. Date and time of dose administration will be recorded in the Dose Administration Record. Compliance will be evaluated by the investigator and/or study staff on all visits. Records of study drug used, tr...
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NCT01283542
7.4
Efficacy
7.4 Efficacy Tumor volume, hormone response, and relevant disease-related symptoms will be evaluated, as previously described in this protocol.
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NCT01283542
7.4.1
Primary efficacy evaluation
7.4.1 Primary efficacy evaluation Tumor volume will be evaluated by MRI with gadolinium contrast in the Screening Phase (and will be considered as baseline imaging), on Weeks 4, 12, and 24 in the Main Phase, and on Weeks 48, 72, and 96 in the Extension Phase (see Tables 7-1 and 7-2).
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NCT01283542
7.4.2
Secondary efficacy evaluations
7.4.2 Secondary efficacy evaluations Tumor volume will also be evaluated as described on Section 7.4.1. Time to response will also be obtained (i.e., time to reach tumor volume decrease ≥ 20%). Disease-related symptoms will be reported by patients and recorded by the medical staff, on all Main Phase visits and on visit...
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NCT01283542
7.5
Safety
7.5 Safety
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NCT01283542
7.5.1
Adverse events
7.5.1 Adverse events An AE for the purposes of this protocol is the onset of (or worsening of) untoward (preexisting) sign(s), symptom(s), or clinical condition(s) occurring after signature of the Informed Consent Form, even if the event is clearly considered not related to the study drug(s). Refer to Section 6 for spe...
[ "Unlike routine safety evaluations, SAEs are continuously monitored and have special reporting requirements; See Section 8.1." ]
NCT01283542
7.5.2
Special Safety
7.5.2 Special Safety
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NCT01283542
7.5.2.1
Changes in blood glucose
7.5.2.1 Changes in blood glucose The principal investigator must instruct patients about signs and symptoms of hyperglycemia, and evaluate the risks of hyperglycemia in all patients. It is recommended to follow the guidelines established by international associations specialized in diabetes, such as the American Diabet...
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NCT01283542
7.5.2.2
Changes in QT interval
7.5.2.2 Changes in QT interval If, at any visit, a QTcF > 480 msec is seen in patients receiving pasireotide LAR, the following actions will be taken: - A visit to a cardiologist must be scheduled as soon as possible, but within no more than 7 days of the initial abnormal ECG, and the cardiologist must re-evaluate the ...
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NCT01283542
7.5.2.3
Liver safety monitoring
7.5.2.3 Liver safety monitoring If any of the criteria below is observed on any scheduled or unscheduled visit, the sponsor must be informed as soon as the occurrence is acknowledged. - ALT or AST > 3 x ULN and Total Bilirubin ≥ 2 x ULN - ALT or AST > 5 x ULN and 8 x ULN The following tests will be performed immediatel...
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NCT01283542
7.5.2.4
Gadolinium
7.5.2.4 Gadolinium There are known adverse events after the use of Gadolinium as contrast material in MRI scans. Caution must be taken with patients with current and previous renal failure.
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NCT01283542
7.5.3
Physical examination, weight, height
7.5.3 Physical examination, weight, height Physical examinations will be performed and weight will be measured on all visits. This may be performed by the Investigator or any other qualified healthcare professional. The information on physical examinations must be present in the source-documentation at the study site. ...
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NCT01283542
7.5.4
Vital signs
7.5.4 Vital signs Body temperature, blood pressure in supine position, and heart rate in supine position will be evaluated on all main phase visits and on Visits 13 (week 36), 16 (week 48), 19 (week 60), 22 (week 72), 25 (week 84), 28 (week 96), and 29 (week 100) in the extension phase.
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NCT01283542
7.5.5
Performance status
7.5.5 Performance status Eastern Cooperative Oncology Group (ECOG) performance status will be evaluated on each visit in the main phases and on Visits 13 (week 36), 16 (week 48), 19 (week 60), 22 (week 72), 25 (week 84), 28 (week 96), and 29 (week 100) in the extension phase, using the criteria on Table 7-3. Table 7-3 ...
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NCT01283542
7.5.6
Laboratory evaluations
7.5.6 Laboratory evaluations Laboratory samples will be submitted to a local laboratory for analysis, except alpha subunit , which will be submitted to a central laboratory. Patients will fast overnight for 12 hours before all blood samples are collected. Blood samples must be performed in the morning. Drinking water i...
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NCT01283542
7.5.6.1
Hematology
7.5.6.1 Hematology Hematology parameters will be evaluated in the Screening Period, before administration of the study drug, and on every Main Phase visit (except Visit 2, Visit 3 [Week 3], Visit 6 [Week 11], and Visit 10 [Week 24]), and will include: WBC count with differential, hemoglobin, HCT, RBC count and platelet...
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NCT01283542
7.5.6.2
Coagulation
7.5.6.2 Coagulation Coagulation parameters APTT and PT will be evaluated on visit 1 (screening), visit 2 (baseline), visit 6, and visit 10 (completion of the study) for all patients. Coagulation parameter PT will also be evaluated on visit 3, visit 4, visit 401 (after 48 days), and visit 5 for patients randomized to th...
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NCT01283542
7.5.6.3
Chemistry
7.5.6.3 Chemistry A complete chemistry profile will be evaluated in the screening period and before administration of the study drug on each visit (except on Visit 2, Visit 3, Visit 6, and Visit 11, the Follow-up Visit) and will include: total cholesterol, low density lipoprotein (LDL) cholesterol, high density lipopro...
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NCT01283542
7.5.6.5
Serology
7.5.6.5 Serology Hepatitis B surface antigen (HBs-Ag) and Hepatitis C antibody (anti-HCV) tests will be performed on visit 1 (screening).
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NCT01283542
7.5.6.6
Urinalysis
7.5.6.6 Urinalysis Specific gravity, pH, glucose, protein, bilirubin, ketones, leukocytes, and blood will be evaluated. Urine must be collected in the Screening Phase and before administration of the study drug on each visit (except on Visit 2, Visit 3, Visit 6, and Visit 11 or Follow-up Visit) in the Main Phase and on...
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NCT01283542
7.5.6.7
Pregnancy test
7.5.6.7 Pregnancy test Blood pregnancy test is performed in the screening period, on Weeks 12 and 24 in the Main Phase, and on Weeks 48, 72, and 96 in the Extension Phase. It may be performed on the Follow-up Visit (56 ± 3 days after the last dose of the study medication) if the patient reports late menstruation or at ...
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NCT01283542
7.5.7
Radiology tests
7.5.7 Radiology tests A gallbladder ultrasound will be locally performed in the Screening Phase (this will be considered as baseline data), on Week 24 in the Main Phase, and Weeks 48 and 96 in the Extension Phase. Patients with history of symptomatic colelithiasis are excluded from participation in the study (refer to ...
[ "MRI Evaluation / Tumor volume measurement:" ]
NCT01283542
7.5.8
Electrocardiogram (ECG)
7.5.8 Electrocardiogram (ECG) All ECGs will be locally performed. An ECG will be performed in the Screening Period or Visit 1 (to evaluate eligibility) and then on all visits before injection of the study drug in the Main Phase, except for Visits 3 and 6, on which they will be performed independently of drug administra...
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NCT01283542
7.5.9
Visual evaluations
7.5.9 Visual evaluations Visual field evaluations will be performed at screening, on Visit 2 (if the visual field evaluation at Screening is performed no more than 14 days before Visit 2, it may be considered as baseline data and there is no need to repeat it for Visit 2), on all visits in the Main Phase, except for Vi...
[ "Visual Field Test" ]
NCT01283542
7.6
Tolerability
7.6 Tolerability In addition to overall safety data, information on dose decreases will be collected. Furthermore, any AEs related to the study drug, e.g., gastrointestinal disorders, glucose metabolic disorders, cardiac dysfunctions, laboratory abnormalities, infections, and injection site reactions, will be summarize...
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NCT01283542
7.7
Patient reported outcomes
7.7 Patient reported outcomes Not applicable.
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NCT01283542
8
Safety monitoring
8 Safety monitoring
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NCT01283542
8.1
Serious adverse event reporting
8.1 Serious adverse event reporting In order to ensure patient safety, each SAE, regardless of the suspected causality, occurring after the beginning of any period in which the study protocol interferes with the standard medical care given to the patient (for example, withdrawal of treatment during washout period, chan...
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NCT01283542
8.2
Pregnancy
8.2 Pregnancy In order to ensure patient safety, every pregnancy in a patient under administration of the study drug must be reported to Novartis within 24 hours from acknowledgement of its occurrence. Pregnancy must be followed up in order to determine the outcome, including miscarriage or abortion, details about the ...
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NCT01283542
9
Data review and management
9 Data review and management
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NCT01283542
9.1
Site monitoring
9.1 Site monitoring Before the study is started, in a site initiation visit or in a meeting with the investigator, the Novartis team (or an appointed CRO) will review the protocol and eCRFs with the investigators and their staff. During the study, the field monitor will visit the site regularly to check for correct com...
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NCT01283542
9.2
Data collection (eCRF)
9.2 Data collection (eCRF) The investigator's designated staff must enter the information required by the protocol in the Novartis eCRFs. Field monitors will review the eCRFs for completion and accuracy and will instruct the site personnel to perform any corrections or inclusions required.
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NCT01283542
9.3
Database management and quality control
9.3 Database management and quality control The data of eCRFs are entered in the study database by the CRO Data Management team after its own internal standard operating procedures are reviewed and approved by Novartis. Then, the data entered are systematically verified by the CRO Data Management team with the use of e...
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NCT01283542
10
Statistic methods and data analysis
10 Statistic methods and data analysis The following sections describe the analyses to be performed. For the Main Phase of the study, efficacy and safety analyses will be performed with the data collected when all patients enrolled complete their EOS visit. All Follow-up Visit safety data from patients who do not enter...
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NCT01283542
10.1
Analysis populations
10.1 Analysis populations Full analysis set (FAS): comprises all patients successfully completing Screening and receiving at least one dose of the study medication. The FAS will be the primary analysis set for efficacy analyses. Safety analysis set: comprises all patients receiving at least one dose of the study drug a...
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NCT01283542
10.2
Demographics / other baseline patient characteristics
10.2 Demographics / other baseline patient characteristics Demographics and other baseline data, including disease characteristics (e.g., age, gender, and race), will be descriptively summarized based on the FAS. Categorical data will be presented as frequencies and percentages. For continuous data, the mean, standard ...
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NCT01283542
10.3
Treatments (study drug, concurrent therapies, compliance)
10.3 Treatments (study drug, concurrent therapies, compliance) Duration of exposure to the study drug, number of patients with dose adjustment (dose interruptions, changes), and dose intensity will be summarized for the main period and the study extension period. Significant concurrent medication and non-drug therapies...
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NCT01283542
10.4
Primary objective
10.4 Primary objective The primary objective is to evaluate the efficacy of pasireotide LAR on NFPA, based on tumor volume decrease.
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NCT01283542
10.4.1
Variable
10.4.1 Variable The primary efficacy variable is the proportion of patients with NFPA reaching a tumor volume decrease of at least 20% after treatment Week 24 with pasireotide LAR.
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NCT01283542
10.4.2
Statistical analysis
10.4.2 Statistical analysis The proportion of patients with a decrease of at least 20% in tumor volume will be summarized in terms of incidence rates and the exact 90% confidence interval at the end of the main study for the FAS population (week 24).
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NCT01283542
10.4.3
Control of missing values / censoring / discontinuations
10.4.3 Control of missing values / censoring / discontinuations A patient whose tumor volume evaluation at the beginning of the study (Screening Period, baseline data) is missing will not be considered for efficacy evaluations. Only patients with baseline evaluation and at least one later evaluation will be considered ...
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NCT01283542
10.4.4
Support Analysis
10.4.4 Support Analysis A sensitivity analysis will be performed in the primary efficacy endpoint after the imputation of missing values on week 24 from the last observation carried forward (LOCF). This will be possible for patients with baseline data and at least one later value.
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