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| Name | Class |
|---|---|
| Regeneron Pharmaceuticals | INDUSTRY |
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This was a Phase 4, open-label, single-arm, multicenter study to evaluate the efficacy and safety of dupilumab subcutaneous (SC) injection monotherapy in Japanese participants aged 18 or older with CRSwNP that is not adequately controlled with existing therapies.
Duration of study period (per participant):
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dupilumab | Experimental | Dupilumab every 2 weeks (q2w). Dosing interval may be changed from q2w to q4w at week 24 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dupilumab SAR231893 | Drug | Pharmaceutical form: solution for injection Route of administration: subcutaneous (SC) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With NPS Improvement From Baseline >=1 at Week 24 | The NPS was the sum of right and left nostril scores as assessed by central video recordings of nasal endoscopy (NE). For each nostril, NPS was graded based on polyp size: Grade 0:No polyps; Grade 1:Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate; Grade 2:Polyps reaching below the lower border of the middle turbinate; Grade 3:Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate; Grade 4:Large polyps causing complete obstruction of the inferior nasal cavity. Score ranged from 0-8; higher scores indicated more severity. The baseline value was defined as the last available value before first study treatment administration. | Baseline (Day 1) and Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Bilateral NPS at Week 24 | The NPS was the sum of right and left nostril scores as assessed by central video recordings of NE. For each nostril, NPS was graded based on polyp size: Grade 0:No polyps; Grade 1:Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate; Grade 2:Polyps reaching below the lower border of the middle turbinate; Grade 3:Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate; Grade 4:Large polyps causing complete obstruction of the inferior nasal cavity. Score ranged from 0-8; higher scores indicated more severity. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. |
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Inclusion Criteria:
Participants ≧18 years of age.
Participants with bilateral sinonasal polyposis that despite prior treatment with systemic corticosteroids (SCS) anytime within the past 2 years; and/or had a medical contraindication / intolerance to SCS; and/or had prior surgery for NP at the screening visit, had:
Participant's body weight > 30 kg at V1.
Signed written informed consent.
Exclusion Criteria:
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigational site JAPAN | Japan | Japan |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40884290 | Derived | Fujieda S, Takabayashi T, Ohta N, Yoshikawa M, Kibe Y, Ishida M, Suzuki K, Takahashi Y, Amin N, Wright L, Robinson L, Yahata K. Effectiveness and Safety of Dupilumab Monotherapy in Japanese Patients With Chronic Rhinosinusitis With Nasal Polyps. Laryngoscope. 2025 Dec;135(12):4625-4632. doi: 10.1002/lary.70037. Epub 2025 Aug 30. |
| Label | URL |
|---|---|
| LPS16872 Plain Language Results Summary | View source |
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Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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The study consisted of a screening period (up to 4 weeks) and a treatment period (up to 52 weeks). A total of 25 participants were enrolled in this study. The study duration for each participant was up to 56 weeks.
The study was conducted at 9 centers in Japan. A total of 30 participants were screened from 22 Oct 2021 to 22 Jun 2022 of which 5 were screen failures due to not meeting eligibility criteria.
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| ID | Title | Description |
|---|---|---|
| FG000 | Dupilumab 300 Milligrams (mg) | Participants received dupilumab 300 mg every 2 weeks (q2w) up to Week 24. Participants with stable disease (nasal polyp score [NPS] improvement from baseline to both Weeks 16 and 24 of greater than or equal to [>=]2 points) at Week 24 received dupilumab 300 mg every 4 weeks (q4w) after Week 24 up to Week 48 and those who could not achieve stable disease at Week 24 continued to receive dupilumab 300 mg q2w after Week 24 up to Week 50. All participants were followed up to Week 52. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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The Enrolled analysis set consisted of all participants with a treatment kit number allocated and recorded in the interactive response technology (IRT) database, regardless of whether the treatment kit was used or not.
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| ID | Title | Description |
|---|---|---|
| BG000 | Dupilumab 300 mg | Participants received dupilumab 300 mg q2w up to Week 24. Participants with stable disease (NPS improvement from baseline to both Weeks 16 and 24 of >=2 points) at Week 24 received dupilumab 300 mg q4w after Week 24 up to Week 48 and those who could not achieve stable disease at Week 24 continued to receive dupilumab 300 mg q2w after Week 24 up to Week 50. All participants were followed up to Week 52. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Percentage of Participants With NPS Improvement From Baseline >=1 at Week 24 | The NPS was the sum of right and left nostril scores as assessed by central video recordings of nasal endoscopy (NE). For each nostril, NPS was graded based on polyp size: Grade 0:No polyps; Grade 1:Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate; Grade 2:Polyps reaching below the lower border of the middle turbinate; Grade 3:Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate; Grade 4:Large polyps causing complete obstruction of the inferior nasal cavity. Score ranged from 0-8; higher scores indicated more severity. The baseline value was defined as the last available value before first study treatment administration. | The Per protocol (PP) analysis set consisted of all participants in modified intent-to-treat (mITT) set (Enrolled population with chronic rhinosinusitis with nasal polyp [CRSwNP] allocated to study treatment administered at least 1 dose of study treatment) and who did not receive intranasal corticosteroids (INCS) up to Week 24 | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline (Day 1) and Week 24 |
From first treatment administration up to Week 52
The Safety analysis set consisted of all participants allocated to study treatment administered at least 1 dose of study treatment during the intervention period.
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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 | Dupilumab 300 mg | Participants received dupilumab 300 mg q2w up to Week 24. Participants with stable disease (NPS improvement from baseline to both Weeks 16 and 24 of >=2 points) at Week 24 received dupilumab 300 mg q4w after Week 24 up to Week 48 and those who could not achieve stable disease at Week 24 continued to receive dupilumab 300 mg q2w after Week 24 up to Week 50. All participants were followed up to Week 52. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Suicide Attempt | Psychiatric disorders | MedDra 26.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Covid-19 | Infections and infestations | MedDra 26.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi aventis recherche & développement | 800-633-1610 | 6# | Contact-US@sanofi.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 11, 2021 | Dec 5, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 16, 2023 | Dec 5, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| C582203 | dupilumab |
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| Baseline (Day 1) and Week 24 |
| Change From Baseline in Nasal Congestion/Obstruction (NC) Symptom Severity Score Using the CRSwNP Nasal Symptom Diary at Week 24 | The NC was assessed by the participant using a diary on a daily basis from Visit 1 and throughout the study on a 0 to 3 categorical scale (where 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms and 3 = severe symptoms). It was scored as a reflective score (evaluation of symptom severity over the past 24 hours) by the participants. The score ranged from 0-3; higher scores indicated more severity. The baseline value was defined as the last available value before first study treatment administration. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | Baseline (Day 1) and Week 24 |
| Change From Baseline in Opacification of Sinuses Assessed by Computerized Tomography (CT) Scan Using the Lund Mackay (LMK) Score at Week 24 | The LMK system is based on localization with points given for degree of opacification: 0 = normal, 1 = partial opacification, 2 = total opacification. These points were applied to the maxillary, anterior ethmoid, posterior ethmoid, sphenoid, and frontal sinus on each side. The osteomeatal complex (OC) was graded as 0 = not occluded or 2 = occluded. The total score was the sum of scores from each side and ranged from 0 (normal) to 24 (more opacified); higher score indicated more severe disease. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | Baseline (Day 1) and Week 24 |
| Change From Baseline in Total Symptom Score (TSS) at Week 24 | The TSS was the sum of participant-assessed nasal symptom scores for nasal congestion and/or obstruction, decreased/loss of sense of smell and rhinorrhea (average of the non-missing anterior/posterior nasal discharge). On a daily basis from Visit 1 and throughout the study, participants used a diary to respond to the morning individual rhinosinusitis symptom questions using a 0 to 3 categorical scale (where 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms and 3 = severe symptoms). Total score ranged from 0 (no symptoms) to 9 (severe symptoms). Higher score indicated more severe symptoms. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | Baseline (Day 1) and Week 24 |
| Change From Baseline in Loss of Smell Symptom Severity Score Using the Nasal Symptom Diary at Week 24 | The severity of loss of smell was reported by the participants using the nasal symptom diary with a 0 to 3 categorical scale (where 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms and 3 = severe symptoms), higher score indicated more severe symptoms. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | Baseline (Day 1) and Week 24 |
| Change From Baseline in Visual Analogue Scale (VAS) for Rhinosinusitis at Week 24 | The VAS for rhinosinusitis was used to evaluate the total disease severity. Rhinosinusitis was divided into mild, moderate and severe based on total severity VAS score (0 to 10 centimeters [cm] where mild: VAS 0 to 3; moderate: VAS >3 to 7 and severe: VAS >7 to 10). The participants were asked to indicate on a VAS the answer to the question: "How troublesome are your symptoms of your rhinosinusitis?" The range of the VAS was from 0 (not troublesome) to 10 (worst thinkable troublesome), where higher score indicated worse disease. For participants who discontinued for other reasons, missing data were imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | Baseline (Day 1) and Week 24 |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs) and TEAEs Leading to Treatment Discontinuation at the End of 24-Week Treatment Period | An Adverse Event (AE) was defined as any untoward medical occurrence in a participant temporally associated with the use of study treatment, whether or not considered related to the study treatment. TEAEs were defined as AEs that developed or worsened in grade or became serious during TE period which was defined as the period from the time of first dose of study treatment until the last visit in the study. Serious adverse events (SAE) were defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. Data reported is till the end of 24-week treatment period. | From Baseline (Day 1) up to Week 24 |
| years |
|
| Sex: Female, Male | Count of Participants | Participants | No |
|
| Race (NIH/OMB) | Count of Participants | Participants | No |
|
| ID | Title | Description |
|---|
| OG000 | Dupilumab 300 mg | Participants received dupilumab 300 mg q2w up to Week 24. Participants with stable disease (NPS improvement from baseline to both Weeks 16 and 24 of >=2 points) at Week 24 received dupilumab 300 mg q4w after Week 24 up to Week 48 and those who could not achieve stable disease at Week 24 continued to receive dupilumab 300 mg q2w after Week 24 up to Week 50. All participants were followed up to Week 52. |
|
|
| Secondary | Change From Baseline in Bilateral NPS at Week 24 | The NPS was the sum of right and left nostril scores as assessed by central video recordings of NE. For each nostril, NPS was graded based on polyp size: Grade 0:No polyps; Grade 1:Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate; Grade 2:Polyps reaching below the lower border of the middle turbinate; Grade 3:Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate; Grade 4:Large polyps causing complete obstruction of the inferior nasal cavity. Score ranged from 0-8; higher scores indicated more severity. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | The PP analysis set consisted of all participants in mITT allocated to study treatment administered at least 1 dose of study treatment, and who did not receive INCS up to Week 24. | Posted | Mean | Standard Error | score on a scale | Baseline (Day 1) and Week 24 |
|
|
|
| Secondary | Change From Baseline in Nasal Congestion/Obstruction (NC) Symptom Severity Score Using the CRSwNP Nasal Symptom Diary at Week 24 | The NC was assessed by the participant using a diary on a daily basis from Visit 1 and throughout the study on a 0 to 3 categorical scale (where 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms and 3 = severe symptoms). It was scored as a reflective score (evaluation of symptom severity over the past 24 hours) by the participants. The score ranged from 0-3; higher scores indicated more severity. The baseline value was defined as the last available value before first study treatment administration. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | The PP analysis set consisted of all participants in mITT allocated to study treatment administered at least 1 dose of study treatment, and who did not receive INCS up to Week 24. | Posted | Mean | Standard Error | score on a scale | Baseline (Day 1) and Week 24 |
|
|
|
| Secondary | Change From Baseline in Opacification of Sinuses Assessed by Computerized Tomography (CT) Scan Using the Lund Mackay (LMK) Score at Week 24 | The LMK system is based on localization with points given for degree of opacification: 0 = normal, 1 = partial opacification, 2 = total opacification. These points were applied to the maxillary, anterior ethmoid, posterior ethmoid, sphenoid, and frontal sinus on each side. The osteomeatal complex (OC) was graded as 0 = not occluded or 2 = occluded. The total score was the sum of scores from each side and ranged from 0 (normal) to 24 (more opacified); higher score indicated more severe disease. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | The PP analysis set consisted of all participants in mITT allocated to study treatment administered at least 1 dose of study treatment, and who did not receive INCS up to Week 24. | Posted | Mean | Standard Error | score on a scale | Baseline (Day 1) and Week 24 |
|
|
|
| Secondary | Change From Baseline in Total Symptom Score (TSS) at Week 24 | The TSS was the sum of participant-assessed nasal symptom scores for nasal congestion and/or obstruction, decreased/loss of sense of smell and rhinorrhea (average of the non-missing anterior/posterior nasal discharge). On a daily basis from Visit 1 and throughout the study, participants used a diary to respond to the morning individual rhinosinusitis symptom questions using a 0 to 3 categorical scale (where 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms and 3 = severe symptoms). Total score ranged from 0 (no symptoms) to 9 (severe symptoms). Higher score indicated more severe symptoms. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | The PP analysis set consisted of all participants in mITT allocated to study treatment administered at least 1 dose of study treatment, and who did not receive INCS up to Week 24. | Posted | Mean | Standard Error | score on a scale | Baseline (Day 1) and Week 24 |
|
|
|
| Secondary | Change From Baseline in Loss of Smell Symptom Severity Score Using the Nasal Symptom Diary at Week 24 | The severity of loss of smell was reported by the participants using the nasal symptom diary with a 0 to 3 categorical scale (where 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms and 3 = severe symptoms), higher score indicated more severe symptoms. For participants who discontinued for other reasons, missing data are imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | The PP analysis set consisted of all participants in mITT allocated to study treatment administered at least 1 dose of study treatment, and who did not receive INCS up to Week 24. | Posted | Mean | Standard Error | score on a scale | Baseline (Day 1) and Week 24 |
|
|
|
| Secondary | Change From Baseline in Visual Analogue Scale (VAS) for Rhinosinusitis at Week 24 | The VAS for rhinosinusitis was used to evaluate the total disease severity. Rhinosinusitis was divided into mild, moderate and severe based on total severity VAS score (0 to 10 centimeters [cm] where mild: VAS 0 to 3; moderate: VAS >3 to 7 and severe: VAS >7 to 10). The participants were asked to indicate on a VAS the answer to the question: "How troublesome are your symptoms of your rhinosinusitis?" The range of the VAS was from 0 (not troublesome) to 10 (worst thinkable troublesome), where higher score indicated worse disease. For participants who discontinued for other reasons, missing data were imputed using multiple imputation techniques assuming missing at random, and non-missing data collected after treatment discontinuation were used in the analysis. The baseline value was defined as the last available value before first study treatment administration. | The PP analysis set consisted of all participants in mITT allocated to study treatment administered at least 1 dose of study treatment, and who did not receive INCS up to Week 24. | Posted | Mean | Standard Error | cm | Baseline (Day 1) and Week 24 |
|
|
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| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs) and TEAEs Leading to Treatment Discontinuation at the End of 24-Week Treatment Period | An Adverse Event (AE) was defined as any untoward medical occurrence in a participant temporally associated with the use of study treatment, whether or not considered related to the study treatment. TEAEs were defined as AEs that developed or worsened in grade or became serious during TE period which was defined as the period from the time of first dose of study treatment until the last visit in the study. Serious adverse events (SAE) were defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. Data reported is till the end of 24-week treatment period. | The Safety analysis set consisted of all participants allocated to study treatment and administered at least 1 dose of study treatment before Week 24. | Posted | Count of Participants | Participants | No | From Baseline (Day 1) up to Week 24 |
|
|
|
| 0 |
| 25 |
| 2 |
| 25 |
| 10 |
| 25 |
| Vestibular neuronitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDra 26.0 | Systematic Assessment |
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| Seasonal Allergy | Immune system disorders | MedDra 26.0 | Systematic Assessment |
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| Injection Site Erythema | General disorders | MedDra 26.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDra 26.0 | Systematic Assessment |
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| Accidental Overdose | Injury, poisoning and procedural complications | MedDra 26.0 | Systematic Assessment |
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The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
| Title | Measurements |
|---|---|
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