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| ID | Type | Description | Link |
|---|---|---|---|
| SPK-9001-101 | Other Identifier | Alias Study Number |
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A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
Hemophilia B, or Christmas disease, is a genetic bleeding disorder resulting in the lack of ability to produce blood-clotting factor IX (FIX). Individuals with hemophilia B suffer repeated bleeding events, which can cause chronic joint disease and sometimes leads to death due to the inability for blood to clot efficiently. This chronic joint disease can have significant physical, psychosocial, and quality-of-life effects, including financial burden. The current treatment is intravenous infusion of FIX protein products, either prophylactically or in response to bleeding.
The approach being tested in this study uses a novel recombinant adeno-associated virus (AAV), which in nature causes no disease, to deliver the human factor IX (hFIX) gene to the liver cells where FIX is normally made. Recent data of a gene therapy study showed preliminary encouraging results with the approach of using an AAV vector carrying the factor IX gene. This study will seek to determine the safety and kinetics of a single IV infusion of SPK-9001 (a novel AAV vector carrying a high specific activity factor IX variant).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SPK-9001 | Experimental | Single intravenous (i.v.) infusion of SPK-9001 [an adeno-associated viral (AAV) vector with human factor IX gene] Intervention: Gene Therapy / Gene Transfer |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SPK-9001 | Biological | A novel, bioengineered adeno-associated viral vector carrying human factor IX variant |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings | Physical examination included examination of the head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. The examination assessed the participants for any potential changes in general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms. Findings were considered to be clinically significant based on investigator's decision. | Baseline up to Week 52 |
| Number of Participants With Clinically Significant Change From Baseline in Vital Signs | Vital signs (temperature, respiratory rate, pulse rate, height, weight, systolic and diastolic blood pressure) were obtained with participant in the seated position, after having sat calmly for at least 5 minutes. Clinical significance of vital signs was determined at the investigator's discretion. | Baseline up to Week 52 |
| Number of Participants With Clinical Laboratory Abnormalities Reported as TEAE | Following parameters were analyzed for laboratory examination: hematology (neutrophils, lymphocytes, monocytes, eosinophils, basophils, red blood cell [RBC] count, hemoglobin, hematocrit, platelet count); liver function (albumin, total bilirubin, total protein, direct bilirubin, indirect bilirubin, aspartate aminotransferase, alanine aminotransferase, lactate dehydrogenase, alkaline phosphatase, GGT); Lipid panel (HDL, VLDL, triglycerides, total cholesterol); clinical chemistry (sodium, potassium, chloride, bicarbonate, glucose, phosphate, serum creatinine, BUN); urinalysis (specific gravity, pH, glucose, protein, blood, ketones; coagulation, immunology, etc. Investigators determined which laboratory abnormalities were reported as treatment-emergent adverse events (TEAEs). | Baseline up to Week 52 |
| Number of Participants With Drug -Related TEAEs and Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and AEs. An AE was regarded as TEAE if the start date was on or after the infusion of SPK-9001 but before participant's last visit on study (or the date of withdrawal/the date of being lost to follow-up). Severe TEAEs were TEAEs that interfered significantly with participants' usual function. Treatment-related TEAEs were determined by the investigator. |
| Measure | Description | Time Frame |
|---|---|---|
| FIX:C Activity | All samples collected from participants for plasma FIX activity levels were analyzed and used to determine peak and steady-state vector-derived circulating FIX activity levels. The vector-derived endogenous (not affected by intercurrent FIX product infusions) FIX:C activity levels were characterized by post-treatment population mean. Dose escalation and dose level expansion strategies were employed in the study based on vector-derived FIX activity levels as well as any immune responses against AAV capsid. Steady-state levels were based on 2 separate vector-derived FIX:C activity level measurements (at least 2 weeks apart) starting from Week 8-12 with adequate washout. |
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Inclusion Criteria:
Exclusion Criteria:
Genetic Males
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Davis Comprehensive Cancer Center | Sacramento | California | 95817 | United States | ||
| UC Davis CTSC Clinical Research Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40750723 | Derived | Wojciechowski J, Gaitonde P, Hughes JH, Ravva P. Population Modeling of Factor IX Activity Following Administration of Fidanacogene Elaparvovec Gene Therapy in Participants with Hemophilia B. Clin Pharmacokinet. 2025 Oct;64(10):1531-1548. doi: 10.1007/s40262-025-01535-y. Epub 2025 Aug 1. | |
| 38863155 | Derived |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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A total of 22 participants were screened, 15 participants were assigned to treatment and completed study. All 15 participants received the lowest dose in the study (5 x 10^11 vg/kg). No participants were assigned to the 2 higher dose arms. Results presented here are from the lowest dose level.
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| ID | Title | Description |
|---|---|---|
| FG000 | SPK-9001 (5 x 10^11 vg/kg) IV Infusion | Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10^11 vg/kg) for approximately 60 minutes via infusion pump. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Baseline analysis population included all participants who received at least 1 dose of study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | SPK-9001 (5 x 10^11 vg/kg) IV Infusion | Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10^11 vg/kg) for approximately 60 minutes via infusion pump. |
| 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 | Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings | Physical examination included examination of the head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. The examination assessed the participants for any potential changes in general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms. Findings were considered to be clinically significant based on investigator's decision. | All participants who received the infusion of SPK-9001. | Posted | Count of Participants | Participants | Baseline up to Week 52 |
|
Baseline up to Week 52
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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 | SPK-9001 (5 x 10^11 vg/kg) IV Infusion | Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10^11 vg/kg) for approximately 60 minutes via infusion pump. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Microcytic anaemia | Blood and lymphatic system disorders | MedDRA 21.1 | Non-systematic Assessment |
15 participants were treated at the first dose planned for this dose escalation study. Requirements for dose escalation were not met as per protocol and the study completed once all participants completed 52 weeks of follow-up at the initial dose.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 1, 2019 | Mar 31, 2020 | SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Jun 14, 2019 | Apr 1, 2020 | Prot_001.pdf |
| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| D001778 | Blood Coagulation Disorders |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
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| Baseline up to Week 52 |
| Number of Participants With Positive Immune Reponses Against Adeno-associated Virus Vector (AAV) Capsid | Peripheral blood mononuclear cells (PBMC) results by interferon gamma enzyme-linked immunospot assay (ELISPOT) to assess cellular immune responses to AAV capsid and to FIX were presented. The ELISPOT is a type of assay that focuses on quantitatively measuring the frequency of cytokine secretion for a single cell. The positive ELISPOT results suggested a T-cell reaction to capsid protein. | Baseline up to Week 52 |
| Number of Participants Who Reached > 150% Vector-derived FIX:C Activity Level After SPK-9001 Infusion | Based on non-clinical studies in non-human primates (NHPs), it was not predicted that vector-derived FIX:C activity levels >150% of normal would be achieved in this study. However, thrombin antithrombin (TAT) levels as thrombotic potential were to be measured if vector derived FIX:C activity levels >150% of normal were achieved in any participant during the study. Blood samples for TAT at Day 0 visit (prior to FIX protein product infusion) were used to establish baseline value. | Baseline up to Week 52 |
| Number of Participants With FIX Inhibitor | FIX inhibitors were measured using the Bethesda assay from the central and local laboratory. The Bethesda assay measures the amount of factor (FIX) inactivated when the plasma from the patient is incubated with an external source of factor for 2 hours at 37ºC. Inhibitor levels are quantified in Bethesda units (BU). An inhibitor titer of ≥ 0.6 BU/ml is to be taken as clinically significant. | Baseline up to Week 52 |
| Incremental Recovery of FIX Product | Incremental recovery was determined as the peak factor level recorded within the first 3 hours after infusion and was reported as (IU/ml)/(IU/kg), using the formula:([Activity IU/mL peak post infusion] - [Activity IU/mL pre-infusion]) / (IU/kg infused). | Day 0 and Week 52 |
| Baseline up to Week 52 |
| Change From Baseline in FIX:C Antigen Level at Steady State | The vector-derived endogenous (not affected by intercurrent FIX product infusions) FIX:C activity antigen levels were characterized by post-treatment population mean. | Week 12 up to Week 52 |
| Sacramento |
| California |
| 95817 |
| United States |
| UC Davis Ellison Ambulatory Care Clinic | Sacramento | California | 95817 | United States |
| UC Davis Investigational Pharmacy | Sacramento | California | 95817 | United States |
| UC Davis Medical Center | Sacramento | California | 95817 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | 39110 | United States |
| Weill Cornell Medicine - New York Presbyterian Hospital | New York | New York | 10065 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Royal Prince Alfred Hospital | Camperdown/Sydney | New South Wales | 2050 | Australia |
| Pittman DD, Carrieri C, Soares H, McKay J, Tan CY, Liang JZ, Rakhe S, Marshall JC, Murphy JE, Gaitonde P, Rupon J. Field Study and Correlative Studies of Factor IX Variant FIX-R338L in Participants Treated with Fidanacogene Elaparvovec. Thromb Haemost. 2024 Oct;124(10):912-921. doi: 10.1055/s-0044-1787734. Epub 2024 Jun 11. |
| 29211678 | Derived | George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | Participants |
|
|
|
| Primary | Number of Participants With Clinically Significant Change From Baseline in Vital Signs | Vital signs (temperature, respiratory rate, pulse rate, height, weight, systolic and diastolic blood pressure) were obtained with participant in the seated position, after having sat calmly for at least 5 minutes. Clinical significance of vital signs was determined at the investigator's discretion. | All participants who received the infusion of SPK-9001. | Posted | Count of Participants | Participants | Baseline up to Week 52 |
|
|
|
| Primary | Number of Participants With Clinical Laboratory Abnormalities Reported as TEAE | Following parameters were analyzed for laboratory examination: hematology (neutrophils, lymphocytes, monocytes, eosinophils, basophils, red blood cell [RBC] count, hemoglobin, hematocrit, platelet count); liver function (albumin, total bilirubin, total protein, direct bilirubin, indirect bilirubin, aspartate aminotransferase, alanine aminotransferase, lactate dehydrogenase, alkaline phosphatase, GGT); Lipid panel (HDL, VLDL, triglycerides, total cholesterol); clinical chemistry (sodium, potassium, chloride, bicarbonate, glucose, phosphate, serum creatinine, BUN); urinalysis (specific gravity, pH, glucose, protein, blood, ketones; coagulation, immunology, etc. Investigators determined which laboratory abnormalities were reported as treatment-emergent adverse events (TEAEs). | All participants who received the infusion of SPK-9001. | Posted | Count of Participants | Participants | Baseline up to Week 52 |
|
|
|
| Primary | Number of Participants With Drug -Related TEAEs and Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and AEs. An AE was regarded as TEAE if the start date was on or after the infusion of SPK-9001 but before participant's last visit on study (or the date of withdrawal/the date of being lost to follow-up). Severe TEAEs were TEAEs that interfered significantly with participants' usual function. Treatment-related TEAEs were determined by the investigator. | All participants who received the infusion of SPK-9001. | Posted | Count of Participants | Participants | Baseline up to Week 52 |
|
|
|
| Primary | Number of Participants With Positive Immune Reponses Against Adeno-associated Virus Vector (AAV) Capsid | Peripheral blood mononuclear cells (PBMC) results by interferon gamma enzyme-linked immunospot assay (ELISPOT) to assess cellular immune responses to AAV capsid and to FIX were presented. The ELISPOT is a type of assay that focuses on quantitatively measuring the frequency of cytokine secretion for a single cell. The positive ELISPOT results suggested a T-cell reaction to capsid protein. | All participants who received the infusion of SPK-9001. | Posted | Count of Participants | Participants | Baseline up to Week 52 |
|
|
|
| Primary | Number of Participants Who Reached > 150% Vector-derived FIX:C Activity Level After SPK-9001 Infusion | Based on non-clinical studies in non-human primates (NHPs), it was not predicted that vector-derived FIX:C activity levels >150% of normal would be achieved in this study. However, thrombin antithrombin (TAT) levels as thrombotic potential were to be measured if vector derived FIX:C activity levels >150% of normal were achieved in any participant during the study. Blood samples for TAT at Day 0 visit (prior to FIX protein product infusion) were used to establish baseline value. | All participants who received the infusion of SPK-9001. | Posted | Count of Participants | Participants | Baseline up to Week 52 |
|
|
|
| Primary | Number of Participants With FIX Inhibitor | FIX inhibitors were measured using the Bethesda assay from the central and local laboratory. The Bethesda assay measures the amount of factor (FIX) inactivated when the plasma from the patient is incubated with an external source of factor for 2 hours at 37ºC. Inhibitor levels are quantified in Bethesda units (BU). An inhibitor titer of ≥ 0.6 BU/ml is to be taken as clinically significant. | All participants who received the infusion of SPK-9001. | Posted | Count of Participants | Participants | Baseline up to Week 52 |
|
|
|
| Primary | Incremental Recovery of FIX Product | Incremental recovery was determined as the peak factor level recorded within the first 3 hours after infusion and was reported as (IU/ml)/(IU/kg), using the formula:([Activity IU/mL peak post infusion] - [Activity IU/mL pre-infusion]) / (IU/kg infused). | Participants who had received 100 IU/kg of FIX protein product infusion and completed the blood sample collection within the first 3 hours post infusion for FIX protein product enabling determination of FIX incremental recovery. | Posted | Mean | Standard Deviation | [IU/ml]/[IU/kg] | Day 0 and Week 52 |
|
|
|
| Secondary | FIX:C Activity | All samples collected from participants for plasma FIX activity levels were analyzed and used to determine peak and steady-state vector-derived circulating FIX activity levels. The vector-derived endogenous (not affected by intercurrent FIX product infusions) FIX:C activity levels were characterized by post-treatment population mean. Dose escalation and dose level expansion strategies were employed in the study based on vector-derived FIX activity levels as well as any immune responses against AAV capsid. Steady-state levels were based on 2 separate vector-derived FIX:C activity level measurements (at least 2 weeks apart) starting from Week 8-12 with adequate washout. | Participants who had received SPK-9001 and had collected vector-derived FIX:C activity levels enabling acceptable determination of the peak and steady-state derived activity level. | Posted | Mean | Standard Deviation | Percentage of Normal | Baseline up to Week 52 |
|
|
|
| Secondary | Change From Baseline in FIX:C Antigen Level at Steady State | The vector-derived endogenous (not affected by intercurrent FIX product infusions) FIX:C activity antigen levels were characterized by post-treatment population mean. | Participants who had received SPK-9001 and had collected vector-derived FIX:C activity levels enabling acceptable determination of the peak and steady-state derived activity level. | Posted | Mean | Standard Deviation | Percentage of Normal | Week 12 up to Week 52 |
|
|
|
| 0 |
| 15 |
| 0 |
| 15 |
| 14 |
| 15 |
| Normocytic anaemia | Blood and lymphatic system disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Palpitations | Cardiac disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Conjunctivitis allergic | Eye disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Aphthous ulcer | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Food poisoning | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Catheter site bruise | General disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Face oedema | General disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Seasonal allergy | Immune system disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
|
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
|
| Muscle injury | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
|
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
|
| Transaminases increased | Investigations | MedDRA 21.1 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Calcification of muscle | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Costochondritis | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Exostosis | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Joint range of motion decreased | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Dysgeusia | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Sinus headache | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Irritability | Psychiatric disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Haematuria | Renal and urinary disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
|
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| Week 16 |
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| Week 18 |
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| Week 22 |
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| Week 26 |
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| Week 32 |
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| Week 42 |
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| Week 52 |
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