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| Name | Class |
|---|---|
| European Commission | OTHER |
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Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).
This is an open-label, multi-center, exploratory study to estimate the effect of intranasal administration of QR-010 on the nasal mucosa in the restoration of CFTR function, as measured by nasal potential difference (NPD), in the nasal epithelium of adult subjects with CF who are homozygous or compound heterozygous for the ΔF508 CFTR mutation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ΔF508 Homozygous | Experimental | QR-010 administered intranasally as an atomized liquid 10 mg (5 mg per nostril), 3 times weekly for 4 weeks. |
|
| ΔF508 Compound Heterozygous | Experimental | QR-010 administered intranasally as an atomized liquid 10 mg (5 mg per nostril), 3 times weekly for 4 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| QR-010 | Drug | Single-stranded RNA antisense oligonucleotide in isoosmolar solution |
|
| Measure | Description | Time Frame |
|---|---|---|
| Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD). | The primary endpoint was the within-subject change from baseline in total chloride transport as measured by NPD, after the Chloride-free+isoproterenol solution (Cl-free+iso), and was based on the average measurements of both nostrils. To provide baseline stability, baseline was defined as the average of the two most recent pre-dose values, where each pre-dose value was the average of two nostrils. A negative change from baseline of Cl-free+iso shows an improvement. | Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Subjects With a -6.6 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study. | This analysis is the proportion (amount) of subjects with an average Cl-free+iso actual value of -6.6 mV or more negative after treatment (ie, responders), as measured by NPD and was based on the average measurements of both nostrils. The actual value of -6.6 mV is used to discriminate individuals with CF (>-6.6 mV) from normal individuals (≤ -6.6 mV). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| John P Clancy, MD | Cincinnati Childrens Hospital Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| National Jewish Health |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31215818 | Derived | Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27. |
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Subjects were screened and enrolled at 5 hospitals in the USA and Europe. Screenings for Cohort 1 and Cohort 2 occurred in parallel between between 19th October 2015 and 14th July 2016.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1: Homozygous for the F508del-CFTR Mutation | Subjects homozygous for the F508del-CFTR (Cystic Fibrosis Transmembrane conductance Regulator) mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses. |
| FG001 | Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation | Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1: Homozygous for the F508del-CFTR Mutation | Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses. |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD). | The primary endpoint was the within-subject change from baseline in total chloride transport as measured by NPD, after the Chloride-free+isoproterenol solution (Cl-free+iso), and was based on the average measurements of both nostrils. To provide baseline stability, baseline was defined as the average of the two most recent pre-dose values, where each pre-dose value was the average of two nostrils. A negative change from baseline of Cl-free+iso shows an improvement. | Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement. | Posted | Mean | Standard Deviation | mV | Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment. |
|
After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1: Homozygous for the F508del-CFTR Mutation | Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | ProQR Therapeutics | +31 6 20 183 437 | clinical@proqr.com |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| C000708084 | eluforsen |
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| 2 and 4 weeks, and at 3 weeks post-treatment. |
| Number of Subjects With a -4 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study. | This analysis is the proportion of subjects with an average Cl-free+iso actual value of -4 mV or more negative after treatment (ie, responders), as measured by NPD and was based on the average measurements of both nostrils. The value of -4 mV is considered a clinically relevant response to treatment based on data from studies of other CFTR therapies. | 2 and 4 weeks, and at 3 weeks post-treatment. |
| Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study. | This endpoint was the within-subject change in Sodium transport (average Potential Difference prior to perfusion of any solution); this is the average of Potential Difference measured at five sites in the inferior meatus of the nose. A positive change from baseline shows an improvement. | Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment. |
| The Mean Change in CFTR-mediated Total Chloride Transport. | The mean change in CFTR-mediated Total Chloride Transport compared to Baseline, per cohort; this is the mean of Potential Difference measured at five sites in the inferior meatus of the nose measured in millivolts (mV). A negative change from baseline shows an improvement. | 2 and 4 weeks, and at 3 weeks post-treatment. |
| Number of Subjects Experiencing Serious Adverse Events From Baseline Through End of Study. | Number of subjects experiencing serious adverse events from baseline through End of Study. | 3 weeks post-treatment. |
| Number of Subject Discontinuations Due to AEs From Baseline Through End of Study. | Number of subject discontinuations due to AEs from baseline through End of Study. No discontinuations occurred. | 3 weeks post-treatment. |
| Number of Subjects With Abnormalities of Laboratory Parameters From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality in laboratory parameters (chemistry, hematology and urinalysis) that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | 3 weeks post-treatment. |
| Number of Subjects With Abnormalities of Vital Signs & Oximetry From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality vital signs & oximetry that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | 3 weeks post-treatment. |
| Number of Subjects With Abnormalities of Physical Examinations From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality in physical examination that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | 3 weeks post-treatment. |
| Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study. | The NERS was performed by site staff prior to each dose and as part of the NPD procedure; it is a set of scales ranging from 0 (no symptoms) to 3 (most severe symptoms) for assessing the severity of each of the following nasal symptoms, separately for each nostril: mucosal disruption, edema, erythema, polyp, secretions. The range of the total sum is 0-30. | 3 weeks post-treatment. |
| Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study. | Subjects were asked to score a list of 22 symptoms on the SNOT-22 regarding social and emotional consequences. Outcomes were graded as 0 (no problem), to 5 (problem as bad as it could be). The list included: need to blow nose, sneezing, dripping nose, cough, postnasal drip, dense nasal drip, ear fullness, dizziness, ear pain, facial pain/pressure, difficulty falling asleep, waking at night, lack of a good night's sleep, waking up tired, fatigue, reduced productivity, reduced concentration, frustrated/restless/irritable, sad, embarrassed, decrease in smell and taste, and nasal obstruction. The range of the total sum is 0-110, with higher values indicating worse outcome. | 3 weeks post-treatment. |
| Denver |
| Colorado |
| 80206 |
| United States |
| Cincinnati Childrens Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| U.Z. Leuven | Leuven | 3000 | Belgium |
| Hopital Necker-Enfants Malades | Paris | 75743 | France |
| Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation |
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| FEV1% predicted | Lung function; Forced Expiratory Volume in 1 second; a percent of the "predicted values" of patients with similar characteristics. | Mean | Full Range | Percent of the predicted value |
|
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses. |
| OG001 | Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation | Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses. |
|
|
| Secondary | Number of Subjects With a -6.6 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study. | This analysis is the proportion (amount) of subjects with an average Cl-free+iso actual value of -6.6 mV or more negative after treatment (ie, responders), as measured by NPD and was based on the average measurements of both nostrils. The actual value of -6.6 mV is used to discriminate individuals with CF (>-6.6 mV) from normal individuals (≤ -6.6 mV). | Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement. | Posted | Count of Participants | Participants | 2 and 4 weeks, and at 3 weeks post-treatment. |
|
|
|
| Secondary | Number of Subjects With a -4 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study. | This analysis is the proportion of subjects with an average Cl-free+iso actual value of -4 mV or more negative after treatment (ie, responders), as measured by NPD and was based on the average measurements of both nostrils. The value of -4 mV is considered a clinically relevant response to treatment based on data from studies of other CFTR therapies. | Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement. | Posted | Count of Participants | Participants | 2 and 4 weeks, and at 3 weeks post-treatment. |
|
|
|
| Secondary | Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study. | This endpoint was the within-subject change in Sodium transport (average Potential Difference prior to perfusion of any solution); this is the average of Potential Difference measured at five sites in the inferior meatus of the nose. A positive change from baseline shows an improvement. | Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement. | Posted | Mean | Standard Deviation | mV | Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment. |
|
|
|
| Secondary | The Mean Change in CFTR-mediated Total Chloride Transport. | The mean change in CFTR-mediated Total Chloride Transport compared to Baseline, per cohort; this is the mean of Potential Difference measured at five sites in the inferior meatus of the nose measured in millivolts (mV). A negative change from baseline shows an improvement. | Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement. | Posted | Mean | Standard Error | mV | 2 and 4 weeks, and at 3 weeks post-treatment. |
|
|
|
| Secondary | Number of Subjects Experiencing Serious Adverse Events From Baseline Through End of Study. | Number of subjects experiencing serious adverse events from baseline through End of Study. | Safety population: all subjects enrolled in the study. | Posted | Count of Participants | Participants | 3 weeks post-treatment. |
|
|
|
| Secondary | Number of Subject Discontinuations Due to AEs From Baseline Through End of Study. | Number of subject discontinuations due to AEs from baseline through End of Study. No discontinuations occurred. | Safety population: all subjects enrolled in the study; incidence of discontinuations due to AEs from baseline through End of Study will be reported in the Adverse Events section. No discontinuations occurred. | Posted | Count of Participants | Participants | 3 weeks post-treatment. |
|
|
|
| Secondary | Number of Subjects With Abnormalities of Laboratory Parameters From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality in laboratory parameters (chemistry, hematology and urinalysis) that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | Safety population: all subjects enrolled in the study. | Posted | Count of Participants | Participants | 3 weeks post-treatment. |
|
|
|
| Secondary | Number of Subjects With Abnormalities of Vital Signs & Oximetry From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality vital signs & oximetry that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | Safety population: all subjects enrolled in the study. | Posted | Count of Participants | Participants | 3 weeks post-treatment. |
|
|
|
| Secondary | Number of Subjects With Abnormalities of Physical Examinations From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality in physical examination that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | Safety population: all subjects enrolled in the study. | Posted | Count of Participants | Participants | 3 weeks post-treatment. |
|
|
|
| Secondary | Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study. | The NERS was performed by site staff prior to each dose and as part of the NPD procedure; it is a set of scales ranging from 0 (no symptoms) to 3 (most severe symptoms) for assessing the severity of each of the following nasal symptoms, separately for each nostril: mucosal disruption, edema, erythema, polyp, secretions. The range of the total sum is 0-30. | Safety population: all subjects enrolled in the study. 1 measurement was missing from 1 subject in Cohort 2 for Week 4. | Posted | Mean | Standard Deviation | units on a scale | 3 weeks post-treatment. |
|
|
|
| Secondary | Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study. | Subjects were asked to score a list of 22 symptoms on the SNOT-22 regarding social and emotional consequences. Outcomes were graded as 0 (no problem), to 5 (problem as bad as it could be). The list included: need to blow nose, sneezing, dripping nose, cough, postnasal drip, dense nasal drip, ear fullness, dizziness, ear pain, facial pain/pressure, difficulty falling asleep, waking at night, lack of a good night's sleep, waking up tired, fatigue, reduced productivity, reduced concentration, frustrated/restless/irritable, sad, embarrassed, decrease in smell and taste, and nasal obstruction. The range of the total sum is 0-110, with higher values indicating worse outcome. | Safety population: all subjects enrolled in the study. | Posted | Mean | Standard Deviation | units on a scale | 3 weeks post-treatment. |
|
|
|
| 0 |
| 10 |
| 0 |
| 10 |
| 9 |
| 10 |
| EG001 | Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation | Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses. | 0 | 8 | 0 | 8 | 7 | 8 |
| Constipation | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Gastritis | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Glossitis | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 17.1 | Systematic Assessment |
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| Malaise | General disorders | MedDRA 17.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 17.1 | Systematic Assessment |
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| Galbladder pain | Hepatobiliary disorders | MedDRA 17.1 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
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| Staphylococcal infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
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| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
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| Wound | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
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| Dehydration | Metabolism and nutrition disorders | MedDRA 17.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
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| Migraine | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
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| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
|
| Increased viscosity of bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Intranasal hypoaesthesia | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Nasal mucosal erosion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Paranasal sinus discomfort | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
|
Investigators are restricted from disclosure or results until after multi-center publication or 12 months after the completion of the Study at all participating research centers. Sponsor can review results communication prior to public release and can embargo communications regarding trial results for a period that is more than 60 days (varying per site). Sponsor may request redaction of confidential information.
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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