protocol_id stringclasses 263
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NCT03913130 | 2.2.2 | Clinical Experience | 2.2.2 Clinical Experience The clinical development program started in the second half of 2017 with the first-in-human (FIH) clinical study (PQ-110-001), a Phase 1b/2, open-label, multiple-dose, dose-escalation to evaluate the safety and tolerability of QR-110. Following individual reports of efficacy an interim analysi... | [] |
NCT03913130 | 2.3 | Study Rationale | 2.3 Study Rationale This extension study allows subjects completing study PQ-110-001, who derive benefit from QR-110 treatment, to continue to receive treatment and to observe the long-term safety, tolerability, PK, and efficacy of treatment with QR-110. Additionally, this extension study allows for the treatment of th... | [] |
NCT03913130 | 2.3.1 | Dose Selection | 2.3.1 Dose Selection Each subject will receive a maintenance dose of 80 µg at 6-monthly intervals in the first treated eye. Treatment of the contralateral eye will begin with a loading dose of 160 µg and is followed by a maintenance dose of 80 µg at 6-monthly intervals. The contralateral eye and the first treated eye w... | [] |
NCT03913130 | 2.4 | Study Population | 2.4 Study Population Subjects who completed study PQ-110-001, met eligibility requirements, and for whom the assessment of benefit/risk is positive may participate in the study. | [] |
NCT03913130 | 2.4.1 | Pediatric Considerations | 2.4.1 Pediatric Considerations In accordance with best practices, the parent or legal representative of a participating pediatric subject must be informed as to procedures that are part of usual care and those specific to participation in this clinical study. Age-appropriate explanations of procedures should be given t... | [] |
NCT03913130 | 3.0 | STUDY OBJECTIVES | 3.0 STUDY OBJECTIVES | [] |
NCT03913130 | 3.1 | Primary Objective | 3.1 Primary Objective The primary objective of the study is to evaluate long-term safety and tolerability of QR-110 administered via IVT injection. | [] |
NCT03913130 | 3.2 | Secondary Objectives | 3.2 Secondary Objectives The secondary objectives of the study are to: - Evaluate long-term and sustained efficacy of QR-110 administered by IVT injection, as assessed by functional and structural outcome measures - Evaluate changes in Patient Reported Outcome (PROs) measures in subjects treated with QR-110 - Evaluate ... | [] |
NCT03913130 | 3.3 | Exploratory Objectives | 3.3 Exploratory Objectives | [] |
NCT03913130 | 4.0 | STUDY OVERVIEW | 4.0 STUDY OVERVIEW | [] |
NCT03913130 | 4.1 | Criteria for Evaluation | 4.1 Criteria for Evaluation | [] |
NCT03913130 | 4.1.1 | Primary Endpoint | 4.1.1 Primary Endpoint - Frequency and severity of ocular adverse events (AEs) - Frequency and severity of non-ocular AEs | [] |
NCT03913130 | 4.1.2 | Secondary Endpoints | 4.1.2 Secondary Endpoints - Change in BCVA - Change in Mobility course score (unilateral and binocular) - Change in photoreceptor outer segment layer thickness by optical coherence tomography (OCT) (if applicable) - Change in OCI - Change in Full-FST (blue and red stimuli; white at Investigator discretion) (thresholds)... | [] |
NCT03913130 | 4.1.3 | Exploratory Endpoints (at Investigator Discretion) | 4.1.3 Exploratory Endpoints (at Investigator Discretion) | [] |
NCT03913130 | 4.2 | Study Design | 4.2 Study Design PQ-110-002 study is an open-label extension study to evaluate the safety, tolerability, efficacy, and pharmacokinetics of QR-110 in subjects with LCA due to c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. Subjects will be given the opportunity to enroll into the extension phase study PQ-110-002... | [] |
NCT03913130 | 4.2.1 | Study Plan | 4.2.1 Study Plan | [] |
NCT03913130 | 4.2.1.1 | Screening/ Day 1 | 4.2.1.1 Screening/ Day 1 The Screening/Day 1 visit for this study should be the last visit (EOS visit) of study PQ-110-001, ie, all Screening/Day 1 assessments should take place during the EOS visit of study PQ-110-001. If these visits do not occur at the same time, a separate Screening/Day 1 visit should be performed ... | [] |
NCT03913130 | 4.2.1.2 | Study Drug Administration | 4.2.1.2 Study Drug Administration Subjects will receive study drug via unilateral IVT injection in accordance with the procedures outlined by the current international guidelines [\(Avery 2014\)](#page-58-3) in their first treated eye and in the contralateral (previously untreated) eye and will be assessed for safety a... | [] |
NCT03913130 | 4.2.1.3 | Assessment and Follow-up | 4.2.1.3 Assessment and Follow-up Subjects will be monitored in clinic for increases in intraocular pressure (IOP) and signs of intraocular inflammation and endophthalmitis during the post-injection period, ie, the day of the injection, and on the day after injection (1-day post-dose). The Day 7 post-dose visit will be ... | [] |
NCT03913130 | 4.2.2 | Stopping Criteria | 4.2.2 Stopping Criteria The Investigator or the Medical Monitor may stop treatment for an individual subject due to an AE (Section [9.0\)](#page-37-0). The severity of the event(s), as well as the temporal relationship to study drug administration, potential for worsening of the event(s) with continued QR-110 treatment... | [] |
NCT03913130 | 4.2.3 | Subject Withdrawal | 4.2.3 Subject Withdrawal Subjects are free to withdraw from the study at any time (ie, discontinue study drug and assessments), without prejudice to further treatment. A subject who withdraws consent will always be asked about the reason(s) and the presence of any AEs. The Investigator should follow-up on AEs outside o... | [] |
NCT03913130 | 4.2.4 | Discontinuation of the Study | 4.2.4 Discontinuation of the Study The Medical Monitor will evaluate the safety and tolerability data as described in Section [4.2.2](#page-24-0) and thereafter on an ongoing basis to recommend if the study should continue or cease, or if any modifications should be made as to how subjects are treated or managed. | [] |
NCT03913130 | 5.0 | SELECTION OF STUDY POPULATION | 5.0 SELECTION OF STUDY POPULATION | [] |
NCT03913130 | 5.1 | Study Population | 5.1 Study Population A maximum of 11 subjects who have completed Study PQ-110-001, meet all eligibility requirements, and for whom the assessment of benefit/risk is positive are eligible to participate in this extension study. | [] |
NCT03913130 | 5.2 | Selection of Subjects | 5.2 Selection of Subjects Subjects will be evaluated against all assessments of eligibility criteria as presented in the SOE Results of assessments for all eligibility criteria must be available and reviewed prior to the subject's first dose of study drug in this extension study. | [] |
NCT03913130 | 5.3 | Eligibility Criteria | 5.3 Eligibility Criteria | [] |
NCT03913130 | 5.3.1 | Inclusion Criteria | 5.3.1 Inclusion Criteria The subject is eligible for the study if all the following inclusion criteria apply at Screening: - 1. Subjects who completed participation in study PQ-110-001 and who may derive benefit from continued treatment with QR-110, as assessed by the Investigator, in consultation with the Medical Moni... | [] |
NCT03913130 | 5.3.2 | Exclusion Criteria | 5.3.2 Exclusion Criteria The subject is ineligible for the study if any of the following criteria apply at Screening: - 1. Any contraindication to IVT injection according to the Investigator's clinical judgment and international guidelines [\(Avery 2014\)](#page-58-3) - 2. Safety issue during study PQ-110-001 that may ... | [] |
NCT03913130 | 6.0 | STUDY DRUG AND CONCOMITANTTHERAPIES | 6.0 STUDY DRUG AND CONCOMITANTTHERAPIES | [] |
NCT03913130 | 6.1 | Study Drug | 6.1 Study Drug | [] |
NCT03913130 | 6.1.1 | Study Drug Description and Supply | 6.1.1 Study Drug Description and Supply The QR-110 study drug is a solution for IVT injection. The QR-110 study drug is supplied in glass vials with rubber stoppers, aluminum seal, and white flip-off cap. Vials are individually packed in cartons. Vials and cartons are labelled in compliance with local regulatory requir... | [] |
NCT03913130 | 6.1.2 | Placebo | 6.1.2 Placebo No placebo is used. | [] |
NCT03913130 | 6.1.3 | Study Drug Shipment and Storage | 6.1.3 Study Drug Shipment and Storage Please refer to the Pharmacy Manual for details on shipment, storage, handling, and preparation. | [] |
NCT03913130 | 6.1.4 | Study Drug Accountability and Reconciliation | 6.1.4 Study Drug Accountability and Reconciliation The Investigator must designate a research pharmacist or other staff member to maintain an inventory record of drugs received and dispensed. Used vials should be retained for drug accountability by the Sponsor representative (monitor), unless prohibited by local proced... | [] |
NCT03913130 | 6.1.5 | Dosage and Administration | 6.1.5 Dosage and Administration Subjects will receive study drug with a 6-month dosing interval. Dose level or interval modifications may be considered on the basis of emerging safety and efficacy data from any QR-110 studies if this is anticipated to be in the best interest of the subject. QR 110 will be administered ... | [] |
NCT03913130 | 6.2 | Concomitant Medications and AncillaryTherapy | 6.2 Concomitant Medications and AncillaryTherapy | [] |
NCT03913130 | 6.2.1 | Permitted Concomitant Medications | 6.2.1 Permitted Concomitant Medications The medications usually used in ophthalmology care are permitted, including but not limited to: topical anesthetic agents, carbonic anhydrase inhibitors (intravenous, oral, topical), beta blocker ophthalmic solutions, prostaglandin analog ophthalmic solutions, ophthalmic solution... | [] |
NCT03913130 | 6.2.2 | Prohibited Concomitant Medications | 6.2.2 Prohibited Concomitant Medications The use of any investigational drug (other than QR-110) or device within 90 days or 5 half-lives of the drug at Day 1, whichever is longer, or plans to participate in another investigational study during the PQ-110-002 study period is prohibited. During the study, use of any new... | [] |
NCT03913130 | 6.2.3 | Adequate Forms of Birth Control | 6.2.3 Adequate Forms of Birth Control Female subjects who have reached menarche and male subjects must either practice true abstinence in accordance with their preferred and usual lifestyle, or agree to use acceptable, highly effective methods of contraception Women of non-childbearing potential may be included without... | [] |
NCT03913130 | 7.0 | STUDY VISITS | 7.0 STUDY VISITS All study visits, assessments and procedures should be completed as indicated per the SOE | [] |
NCT03913130 | 7.1 | Visit and Assessment Windows | 7.1 Visit and Assessment Windows The timing of assessments, procedures, and sample collections are indicated in the SOE Study center phone calls to the subjects will occur 7 days post-IVT injection, ±3 days. Study visits will occur every 3 months. If no dosing occurs, the phone call visits may be skipped at the discret... | [] |
NCT03913130 | 7.2 | Dosing and Follow-up Visits | 7.2 Dosing and Follow-up Visits Dosing visits occur on Day 1 for the contralateral eye and then every 6 months. Dosing visits for the previously (first) treated eye will occur 3 months after the dosing visit for the contralateral eye and then every 6 months. This between-eye interval could be adapted in agreement with ... | [] |
NCT03913130 | 7.3 | End of Study Visit | 7.3 End of Study Visit The End of Study (EOS) visit occurs 3 months after the last dose of study drug for a subject. Subjects who discontinue study treatment but do not withdraw consent will be encouraged to remain in the study for observation for at least 3 months post last dose. | [] |
NCT03913130 | 8.0 | STUDY ASSESSMENT PROCEDURES | 8.0 STUDY ASSESSMENT PROCEDURES Safety parameters will be assessed by monitoring AEs, vital signs, physical examinations, ophthalmic exams, OCTs, infrared imaging, electrocardiograms (ECGs), and laboratory data (serum chemistry, hematology, and inflammatory markers). Assessments will be conducted as indicated on the SO... | [] |
NCT03913130 | 8.1 | Adverse Events | 8.1 Adverse Events Information regarding occurrence of AEs will be captured throughout the study. Duration (start and stop dates), severity/grade, outcome, treatment and relationship to study drug will be recorded. Refer to Section [9.0,](#page-37-0) Assessment of Safety or Adverse Events and Serious Adverse Events. | [] |
NCT03913130 | 8.2 | Vital Signs | 8.2 Vital Signs Blood pressure, heart rate and oral temperature will be measured after the subject has been resting in a seated position for a minimum of 5 minutes. | [] |
NCT03913130 | 8.3 | Laboratory Evaluations | 8.3 Laboratory Evaluations All laboratory evaluations will be conducted at a central laboratory. Reference ranges for all laboratory parameters are provided in the Laboratory Manual. A list of all clinical laboratory evaluations is presented in [Appendix 2.](#page-65-0) Serum chemistries will include sodium, potassium,... | [] |
NCT03913130 | 8.4 | Immunogenicity | 8.4 Immunogenicity Blood samples will be obtained to monitor for possible development of antibodies to QR-110, as indicated per the SOE | [] |
NCT03913130 | 8.7 | Pharmacokinetic Evaluations | 8.7 Pharmacokinetic Evaluations Blood samples will be obtained from all subjects to assess QR-110 PK parameters (area under the curve from 0 hour to infinity [AUC0-∞], area under the curve from 0 hour to time of the last measurable concentration BCVA [AUC0-tlast], maximum concentration (Cmax), trough value [C0], time t... | [] |
NCT03913130 | 8.8 | Electrocardiogram | 8.8 Electrocardiogram Twelve-lead ECG will be obtained as indicated per the SOE Electrocardiograms should be done in triplicate after the subject has been resting comfortably in a supine position for a minimum of 8 minutes. The mean of triplicate results will be captured in the eCRF. A repeat of ECGs (in triplicate) is... | [] |
NCT03913130 | 8.9 | Physical Examination | 8.9 Physical Examination Complete and symptom-directed physical examinations (urogenital exams not required) will be performed as indicated per the SOE | [] |
NCT03913130 | 8.10 | Ophthalmic Exams | 8.10 Ophthalmic Exams Ophthalmic examinations will be performed per the SOE The ophthalmic exam is comprised of: anterior segment examination, including grading of anterior chamber inflammation according to the Standardization of Uveitis Nomenclature (SUN) Working Group Grading Scheme for Anterior Chamber Flare; OCI; F... | [] |
NCT03913130 | 8.11 | Optical Coherence Tomography | 8.11 Optical Coherence Tomography Changes in OCT findings are of relevance to the safety profile of QR-110. Optical Coherence Tomography images will be evaluated for changes in the remaining volume of ONL and foveal thickness as safety parameters. All OCT scans should be performed in accordance with the procedures outl... | [] |
NCT03913130 | 8.12 | Efficacy Assessments | 8.12 Efficacy Assessments Efficacy assessments include BCVA, mobility course, FST, OCI, PROs, PLR, and NIRAF. If possible, photoreceptor outer segment layer thickness will be calculated from OCT images as a secondary efficacy parameter. This assessment will be conducted for those subjects for whom high quality imaging ... | [] |
NCT03913130 | 9.0 | ASSESSMENT OF SAFETY OR ADVERSE EVENTS AND SERIOUSADVERSE EVENTS | 9.0 ASSESSMENT OF SAFETY OR ADVERSE EVENTS AND SERIOUSADVERSE EVENTS All subjects who receive study drug will be assessed for safety. Investigators are responsible for monitoring the safety of subjects who have entered this study and for alerting the Sponsor regarding any event that seems unusual, even if this event ma... | [] |
NCT03913130 | 9.1 | Review of Safety Data | 9.1 Review of Safety Data A program-specific review of safety data, which may include independent experts, will be in place to assess emerging data from clinical studies on an ongoing basis. | [] |
NCT03913130 | 9.2 | Definitions of Adverse Event, Serious Adverse Event, and Suspected Unexpected Serious Adverse Event | 9.2 Definitions of Adverse Event, Serious Adverse Event, and Suspected Unexpected Serious Adverse Event | [] |
NCT03913130 | 9.2.1 | Adverse Events | 9.2.1 Adverse Events An AE is any untoward medical occurrence associated with the use of a drug in humans, whether considered drug related or not. Adverse events can include any unfavorable, noxious, unintended sign, symptom, or disease temporally associated with use of a study drug or other protocolimposed interventio... | [
"Adverse events include:"
] |
NCT03913130 | 9.2.2 | Serious Adverse Events | 9.2.2 Serious Adverse Events A serious adverse event (SAE) is any AE that suggests a significant hazard, contraindication, side effect, or precaution regardless of the relationship to study drug. An SAE is any AE that results in any of the following outcomes: - Death - Life-threatening AE. This definition implies that ... | [] |
NCT03913130 | 9.2.3 | Suspected Unexpected Serious Adverse Reaction Definition | 9.2.3 Suspected Unexpected Serious Adverse Reaction Definition A suspected unexpected serious adverse reaction (SUSAR) is a suspected unexpected serious adverse reaction. In order to be qualified as a SUSAR, the AE must meet 3 criteria: the event is serious, there is a certain degree of probability that the event is a ... | [] |
NCT03913130 | 9.2.4 | Adverse Events of SpecialInterest | 9.2.4 Adverse Events of SpecialInterest The following are considered AEs of special interest, based on the route of administration and safety profile for QR-110. | [] |
NCT03913130 | 9.3 | Assessment of Adverse Events | 9.3 Assessment of Adverse Events The Investigator is responsible for assessing the severity and causality of AEs. | [] |
NCT03913130 | 9.3.1 | Assessment of Severity (Intensity) of AdverseEvents | 9.3.1 Assessment of Severity (Intensity) of AdverseEvents On the AE eCRF, the Investigator will use the adjectives MILD, MODERATE, or SEVEREto describe the maximum intensity of the AE. For purposes of consistency, these intensity grades are defined as follows: - MILD: Does not interfere with subject's usual function. -... | [] |
NCT03913130 | 9.3.2 | Assessment of the Relationship of Adverse Events to StudyDrug | 9.3.2 Assessment of the Relationship of Adverse Events to StudyDrug The Investigator will make a causality assessment about the relationship of each AE to study drug. To ensure consistency of AE and SAE causality assessments, Investigators should apply the following general guideline: Not Related: The AE has an etiolog... | [] |
NCT03913130 | 9.3.3 | Assessment of the Outcome of Adverse Events | 9.3.3 Assessment of the Outcome of Adverse Events The Investigator will record the outcome of AEs and SAEs using the following criteria: - Recovered/resolved: The subject has fully recovered from the event, with no residual effects observable. - Recovered/resolved with sequelae: The subject has recovered from the event... | [] |
NCT03913130 | 9.4 | Reporting of Adverse Events | 9.4 Reporting of Adverse Events The Investigator is responsible for ensuring that all AEs and SAEs are recorded in the subject's medical record, AE eCRF, and/or SAE form, and reported to the Sponsor in accordance with protocol instructions. | [] |
NCT03913130 | 9.4.1 | Adverse Event Reporting Period | 9.4.1 Adverse Event Reporting Period Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. In the first treated eye all events/conditions will be captured as an AE as treatment continues from study PQ-110-001. All AEs and SAEs regardless o... | [] |
NCT03913130 | 9.4.2 | Eliciting Adverse Events | 9.4.2 Eliciting Adverse Events A consistent methodology of non-directive questioning for eliciting AEs at all subject evaluation time points should be adopted. Examples of non-directive questions include: "How have you felt since your last clinic visit?" "Have you had any new or changed health problems since you were l... | [] |
NCT03913130 | 9.4.3 | Recording Adverse and Serious AdverseEvents | 9.4.3 Recording Adverse and Serious AdverseEvents Investigators should use correct medical terminology/concepts when recording AEs or SAEs on the eCRF and/or SAE form. Colloquialisms and abbreviations should be avoided. Serious AEs must also be recorded on the AE eCRF. Only one medical concept should be recorded in the... | [] |
NCT03913130 | 9.4.3.1 | Adverse Events Occurring Secondary to Other Events | 9.4.3.1 Adverse Events Occurring Secondary to Other Events In general, AEs occurring secondary to other events (eg, cascade events or clinical sequelae) should also be entered as separate AEs. For example, if severe diarrhea is known to have resulted in dehydration, both diarrhea and dehydration should be entered as AE... | [] |
NCT03913130 | 9.4.3.2 | Persistent or Recurrent Adverse Events | 9.4.3.2 Persistent or Recurrent Adverse Events A persistent AE is one that extends continuously, without resolution between subject evaluation time points. Such events should only be recorded once in the eCRF unless their severity increases. If a persistent AE becomes more severe or occurs more frequently, it should be... | [] |
NCT03913130 | 9.4.3.3 | Abnormal Laboratory Values | 9.4.3.3 Abnormal Laboratory Values Only clinically significant laboratory abnormalities will be recorded as AEs on the eCRF and SAE form (if applicable). If the laboratory abnormality can be characterized by a precise clinical term, the clinical term should be recorded as the AE or SAE. For example, an elevated serum p... | [] |
NCT03913130 | 9.4.3.4 | Deaths | 9.4.3.4 Deaths All deaths that occur during the protocol-specified AE reporting period (Section [9.4.1\)](#page-41-2), regardless of attribution, will be recorded on the AE eCRF and SAE form and reported to the Sponsor within 24 hours of event knowledge. When recording a death, the event or condition that caused or con... | [] |
NCT03913130 | 9.4.3.5 | Preexisting Medical Conditions | 9.4.3.5 Preexisting Medical Conditions A preexisting medical condition is one that is present at the start of the study. Such conditions should be recorded on the Medical and Surgical History eCRF. A preexisting medical condition should be recorded as an AE or SAE only if the frequency, severity, or character of the co... | [] |
NCT03913130 | 9.4.3.6 | Hospitalization, Prolonged Hospitalization or Surgery | 9.4.3.6 Hospitalization, Prolonged Hospitalization or Surgery Any AE that results in hospitalization or prolonged hospitalization should be documented and reported as an SAE unless specifically instructed otherwise in this protocol. | [] |
NCT03913130 | 9.4.3.7 | Pregnancy | 9.4.3.7 Pregnancy If a female subject or a female partner of a male subject becomes pregnant while the female subject or the male partner is receiving study drug a Pregnancy Report form should be completed and faxed to the Drug Safety designee within 24 hours of learning of the pregnancy, using the fax numbers listed i... | [] |
NCT03913130 | 9.4.3.8 | Overdose Reporting | 9.4.3.8 Overdose Reporting Overdoses must be reported to the Sponsor on an AE eCRF and an SAE form for tracking purposes and will be considered a protocol deviation. Overdose is defined as any study drug dose administered above the intended dose. Additional instructions for reporting overdose information will be provid... | [] |
NCT03913130 | 9.5 | Serious Adverse Events Notification | 9.5 Serious Adverse Events Notification For all SAEs, regardless of suspected causality, an SAE form must be completed (or faxed if using paper form) within 24 hours of discovery of the event to:
DRUG SAFETY
Fax toll-free: See Study Reference Manual for Fax Number Any fatal or life threatening (ie, imminent risk of d... | [
"DRUG SAFETY",
"Fax toll-free: See Study Reference Manual for Fax Number",
"DRUG SAFETY",
"Phone toll-free: See Study Reference Manual for Phone Number"
] |
NCT03913130 | 9.6 | Expedited Reporting of Suspected Unexpected Serious AdverseReactions | 9.6 Expedited Reporting of Suspected Unexpected Serious AdverseReactions - The Sponsor or its designee is responsible for notifying the study centers of all expedited SAEs (ie, 7/15 Day SUSARs) that occur during any clinical studies that are using the study drug. The Sponsor or its designee shall also notify Central EC... | [] |
NCT03913130 | 10.0 | STATISTICAL METHODOLOGY | 10.0 STATISTICAL METHODOLOGY | [] |
NCT03913130 | 10.1 | General Considerations | 10.1 General Considerations Analyses will generally be descriptive in nature and will focus on data collected during this study. Baseline functional and structural measurements for the first treated eye will be those from study PQ-110-001. Baseline functional and structural measurements for the contralateral eye will b... | [] |
NCT03913130 | 10.2 | Determination of Sample Size | 10.2 Determination of Sample Size The sample size is determined by the number of subjects completing study PQ-110-001 who meet the inclusion and exclusion criteria and provide informed consent. | [] |
NCT03913130 | 10.2.1 | Randomization andBlinding | 10.2.1 Randomization andBlinding This is an open-label study; randomization and blinding are not required. | [] |
NCT03913130 | 10.2.2 | Replacement of Subjects | 10.2.2 Replacement of Subjects Not applicable as this is an extension study. | [] |
NCT03913130 | 10.3 | Analysis of Populations | 10.3 Analysis of Populations Safety Population: the population for safety analysis will consist of all subjects who receive any QR-110 or pre-medications required for the study. Efficacy Evaluable Population: the population for efficacy (clinical activity) analysis will consist of all subjects who receive any QR-110 wi... | [] |
NCT03913130 | 10.4 | Subject Disposition, Demographics and Baseline Disease Characteristics | 10.4 Subject Disposition, Demographics and Baseline Disease Characteristics Subject disposition will be summarized for the safety population by dose group and age. The number and percentage of subjects enrolled in each study center will be presented by geographic region (North America and Europe). The number and percen... | [] |
NCT03913130 | 10.5 | Treatment Compliance | 10.5 Treatment Compliance All doses are observed and administered by study staff. Treatment compliance will be determined by source records documenting treatment observations and summarized. | [] |
NCT03913130 | 10.6 | Safety Analyses | 10.6 Safety Analyses | [] |
NCT03913130 | 10.6.1 | Treatment Emergent Adverse Events | 10.6.1 Treatment Emergent Adverse Events A treatment emergent adverse event (TEAE) is defined as an event that was not present prior to administration of the first dose of study drug and present after the first dose or if it represents the exacerbation of an event that was present prior to the first dose. Adverse event... | [] |
NCT03913130 | 10.6.2 | Vital Signs | 10.6.2 Vital Signs Vital signs measurements will consist of heart rate, blood pressure and temperature. Descriptive summaries (number of subjects, mean, standard deviation, median, minimum, and maximum) of actual values and changes from baseline will be presented for each time point. These summaries will be presented f... | [] |
NCT03913130 | 10.6.3 | Laboratory Assessments | 10.6.3 Laboratory Assessments Laboratory measurements (hematology, chemistry and urinalysis) obtained at baseline and each study visit will be summarized by dose group and age by: - Descriptive statistics of actual results (number of subjects, mean, standard deviation, median, minimum, and maximum) for the continuous d... | [] |
NCT03913130 | 10.6.4 | Other Safety Assessments | 10.6.4 Other Safety Assessments Other safety assessments, such as ophthalmic exams, OCT, infrared imaging, ECGs and physical examinations will be summarized and listed as specified in the SAP. | [] |
NCT03913130 | 10.7 | Pharmacokinetics Analyses | 10.7 Pharmacokinetics Analyses To determine the PK profile of IVT injections at the different dose levels of QR-110, the following PK parameters will be calculated if sufficient data are available for each dose: - AUC0-∞: Area under the curve to infinity will be calculated based on the last observed concentration (Clas... | [] |
NCT03913130 | 10.8 | Efficacy Analyses | 10.8 Efficacy Analyses The secondary efficacy evaluations will include changes from baseline in: - BCVA - Mobility course score - FST - OCI - OCT (Change in photoreceptor outer segment layer thickness) - PLR - NIRAF - VFQ-25 (adult subjects) - CVAQC (pediatric subjects) Descriptive statistics of clinical efficacy will ... | [] |
NCT03913130 | 10.9 | Exploratory Analyses | 10.9 Exploratory Analyses | [] |
NCT03913130 | 10.10 | Interim Analysis | 10.10 Interim Analysis Ongoing review of safety and efficacy data will be conducted to inform the program. Interim analyses may be performed, eg, yearly, or at key time points during clinical development. | [] |
NCT03913130 | 10.11 | Subgroup Analyses | 10.11 Subgroup Analyses Exploratory analyses may be carried out for subgroups based on age, baseline visual acuity, and other factors identified in the SAP. | [] |
NCT03913130 | 11.0 | QUALITY CONTROL AND QUALITYASSURANCE | 11.0 QUALITY CONTROL AND QUALITYASSURANCE | [] |
NCT03913130 | 11.1 | Data Collection and Study Monitoring | 11.1 Data Collection and Study Monitoring An eCRF will be used for this study. Study center personnel will be trained and authorized to use the system in compliance with the Code of Federal Regulations (CFR) 21CFR Part 11, International Council on Harmonization Good Clinical Practice (GCP) and local regulations, before... | [] |
NCT03913130 | 11.2 | Audits and Inspections | 11.2 Audits and Inspections Authorized representatives of the Sponsor, a regulatory authority, or an IRB/EC may perform audits or inspections at the study center, including source data verification. The purpose of an audit or inspection is to systematically and independently examine all study-related activities and doc... | [] |
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