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| Name | Class |
|---|---|
| Quintiles, Inc. | INDUSTRY |
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Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with Type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression.
The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa.
Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain.
Gaucher disease has been designated in the list of Specified Rare and Intractable Diseases by Specified Disease Treatment Research Program of Ministry of Health, Labor and Welfare (MHLW) as one of "lysosomal storage diseases" since 2001. Gaucher disease is also designated in the Medical Aid Program for Specified Categories of Chronic Pediatric Diseases.
The prevalence of mutations and the phenotype of patients with Gaucher disease in Japan differs from that in non-Japanese populations. Some patients with type 1 Gaucher disease in Japan have more severe and progressive disease compared to non-Japanese patients and the disease is characterized by an earlier onset of symptoms.
Velaglucerase alfa, a highly-purified form of the naturally occurring enzyme glucocerebrosidase, has been developed as an enzyme replacement therapy for Gaucher disease for the symptoms (anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone manifestation).
The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| velaglucerase alfa | Experimental | 15 to 60 U/kg, EOW via intravenous infusion |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| velaglucerase alfa | Drug | 15-60 U/kg, EOW |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) | An AE was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered related to investigational product. A SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. An infusion-related AE was defined as an AE that started either during or within 12 hours after the start of the infusion and that was judged as possibly or probably related to investigational product. | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) |
| Number of Participants Using Concomitant Medication | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) | |
| Number of Participants With Abnormal and Clinically Significant Laboratory Test Results | Laboratory test results were considered abnormal and clinically significant at the discretion of the investigator. | From Week 65 until the end of study (Week 155) |
| Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies | Serum samples were collected for all participants for determination of anti-velaglucerase alfa antibodies every 12 weeks. | From Week 65 until the end of study (Week 155) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Hemoglobin Concentration at Week 101 | Baseline was the modified baseline hemoglobin concentration, the average of the values from screening, baseline, and Week 1 Day 1 from Study HGT-GCB-087 (NCT01614574). | Baseline, Week 101 |
| Change From Baseline in Platelet Count at Week 101 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hamamatsu University School of Medicine | Hamamatsu | Shizuoka | 431-3192 | Japan | ||
| Iwata City Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27241455 | Derived | Ida H, Tanaka A, Matsubayashi T, Murayama K, Hongo T, Lee HM, Mellgard B. A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24months. Blood Cells Mol Dis. 2016 Jul;59:140-7. doi: 10.1016/j.bcmd.2015.10.002. Epub 2015 Oct 16. |
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Participants from study HGT-GCB-087 (NCT01614574) were enrolled in this study except one participant who was not enrolled due to personal reasons.
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| ID | Title | Description |
|---|---|---|
| FG000 | Velaglucerase Alfa (VPRIV®) | Velaglucerase alfa (VPRIV®) 15 to 60 units per kilogram (U/kg), every other week (EOW) administered as an intravenous (IV) infusion over 60 minutes from Week 53 (2 weeks after the last infusion [Week 51] in Study HGT-GCB-087 [NCT01614574]) to Week 155. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Safety population included all enrolled participants who received at least 1 investigational product infusion (full or partial).
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| ID | Title | Description |
|---|---|---|
| BG000 | Velaglucerase Alfa (VPRIV®) | Velaglucerase alfa (VPRIV®) 15 to 60 U/kg, EOW, administered as an IV infusion over 60 minutes from Week 53 (2 weeks after the last infusion [Week 51] in Study HGT-GCB-087 [NCT01614574]) to Week 155. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Age at the time of first dose in the current study (Week 53) |
| 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 Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) | An AE was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered related to investigational product. A SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. An infusion-related AE was defined as an AE that started either during or within 12 hours after the start of the infusion and that was judged as possibly or probably related to investigational product. | Safety population. | Posted | Number | participants | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) |
|
From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks)
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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 | Velaglucerase Alfa (VPRIV®) | Velaglucerase alfa (VPRIV®) 15 to 60 U/kg, EOW, administered as an IV infusion over 60 minutes from Week 53 (2 weeks after the last infusion [Week 51] in Study HGT-GCB-087 [NCT01614574]) to Week 155. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypoacusis | Ear and labyrinth disorders | MedDRA (9.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypoacusis | Ear and labyrinth disorders | MedDRA (9.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
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| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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| ID | Term |
|---|---|
| D005962 | Glucosylceramidase |
| ID | Term |
|---|---|
| D005959 | Glucosidases |
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
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Baseline was the modified baseline platelet count, the average of the values from screening, baseline, and Week 1 Day 1 from Study HGT-GCB-087 (NCT01614574). |
| Baseline, Week 101 |
| Change From Baseline in Liver Volume Normalized to Body Weight at Week 103 | Liver volume was measured using magnetic resonance imaging (MRI). Liver volume measurements were normalized to the percentage of body weight. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 103 |
| Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103 | Spleen volume was measured using MRI. Spleen volume measurements were normalized to the percentage of body weight. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 103 |
| Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score | BMD was measured by dual energy x-ray absorptiometry (DXA) for lumbar spine and femurs. To ensure standardization and allow for comparisons of BMD, results were converted to standardized Z-scores (matched for age and gender). Z-scores express the BMD as the number of standard deviations (SDs) above or below the average BMD of a healthy participant of the same age and gender. Statistical analysis plan only required summarization if greater than (>) 50 percent (%) of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 103 |
| Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score | BMD was measured by DXA for lumbar spine and femurs. To ensure standardization and allow for comparisons of BMD, results were converted to standardized T-scores; normal values were used from databases from Hologic based on standard criteria. T-scores are the number of SDs above or below the average for a young adult at peak BMD. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 103 |
| Change From Baseline in Bone Marrow Burden (BMB) Score at Week 103 | BMB Score was measured using MRI, range from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0-16 points. A higher BMB score signified more severe bone marrow involvement. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 103 |
| Change From Baseline in Growth Velocity at Week 101 : Height Z-Score | The Z-score, or Standard Deviation Score, is a measure of number of SDs above or below the average BMD of a healthy participant of the same age and gender. World Health Organization 2007 growth reference data were used for Z-score calculation. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 101 |
| Change From Baseline in Skeletal Age at Week 103: Z-Score | Skeletal age was measured via radiography (X-ray) of the left hand and wrist by the method of Greulich and Pyle. The Z-score, or Standard Deviation Score, is a measure of number of SDs above or below the average BMD of a healthy participant of the same age and gender. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 103 |
| Change From Baseline in Plasma Chitotriosidase Levels at Week 101 | Plasma chitotriosidase activity levels were measured using an enzymatic assay with 4-methylumbelliferyl-deoxychitobiose as a substrate. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 101 |
| Number of Participants With Change From Baseline in Neurological Status at Week 103 | Neurological status was considered normal or abnormal based on investigator's discretion. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 103 |
| Change From Baseline in Chemokine [C-C Motif] Ligand 18 (CCL18) Levels at Week 101 | Plasma CCL18 concentrations were measured using a time-resolved fluorescence assay. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Baseline, Week 101 |
| Ōkubo |
| Shizuoka |
| 438-8550 |
| Japan |
| The Jikei University School of Medicine | Minato-ku | Tokyo | 105-8471 | Japan |
| Chiba Children's Hospital | Chiba | 266-0007 | Japan |
| Osaka City University Hospital | Osaka | 545-8586 | Japan |
| Mean |
| Standard Deviation |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Velaglucerase Alfa (VPRIV®) |
Velaglucerase alfa (VPRIV®) 15 to 60 U/kg, EOW, administered as an IV infusion over 60 minutes from Week 53 (2 weeks after the last infusion [Week 51] in Study HGT-GCB-087 [NCT01614574]) to Week 155. |
|
|
| Primary | Number of Participants Using Concomitant Medication | Safety population. | Posted | Number | participants | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) |
|
|
|
| Primary | Number of Participants With Abnormal and Clinically Significant Laboratory Test Results | Laboratory test results were considered abnormal and clinically significant at the discretion of the investigator. | Safety population. | Posted | Number | participants | From Week 65 until the end of study (Week 155) |
|
|
|
| Primary | Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies | Serum samples were collected for all participants for determination of anti-velaglucerase alfa antibodies every 12 weeks. | Safety population. | Posted | Number | participants | From Week 65 until the end of study (Week 155) |
|
|
|
| Secondary | Change From Baseline in Hemoglobin Concentration at Week 101 | Baseline was the modified baseline hemoglobin concentration, the average of the values from screening, baseline, and Week 1 Day 1 from Study HGT-GCB-087 (NCT01614574). | Safety population | Posted | Mean | Standard Deviation | gram per deciliter (g/dL) | Baseline, Week 101 |
|
|
|
| Secondary | Change From Baseline in Platelet Count at Week 101 | Baseline was the modified baseline platelet count, the average of the values from screening, baseline, and Week 1 Day 1 from Study HGT-GCB-087 (NCT01614574). | Safety population | Posted | Mean | Standard Deviation | *10^9 platelets per liter | Baseline, Week 101 |
|
|
|
| Secondary | Change From Baseline in Liver Volume Normalized to Body Weight at Week 103 | Liver volume was measured using magnetic resonance imaging (MRI). Liver volume measurements were normalized to the percentage of body weight. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Safety population | Posted | Mean | Standard Deviation | Percentage of body weight | Baseline, Week 103 |
|
|
|
| Secondary | Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103 | Spleen volume was measured using MRI. Spleen volume measurements were normalized to the percentage of body weight. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Safety population | Posted | Mean | Standard Deviation | Percentage of body weight | Baseline, Week 103 |
|
|
|
| Secondary | Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score | BMD was measured by dual energy x-ray absorptiometry (DXA) for lumbar spine and femurs. To ensure standardization and allow for comparisons of BMD, results were converted to standardized Z-scores (matched for age and gender). Z-scores express the BMD as the number of standard deviations (SDs) above or below the average BMD of a healthy participant of the same age and gender. Statistical analysis plan only required summarization if greater than (>) 50 percent (%) of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Data was not reported as there were lesser than (<) 50% of participants with evaluable data. | Posted | Baseline, Week 103 |
|
|
| Secondary | Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score | BMD was measured by DXA for lumbar spine and femurs. To ensure standardization and allow for comparisons of BMD, results were converted to standardized T-scores; normal values were used from databases from Hologic based on standard criteria. T-scores are the number of SDs above or below the average for a young adult at peak BMD. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Data was not reported as there were <50% of participants with evaluable data. | Posted | Baseline, Week 103 |
|
|
| Secondary | Change From Baseline in Bone Marrow Burden (BMB) Score at Week 103 | BMB Score was measured using MRI, range from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0-16 points. A higher BMB score signified more severe bone marrow involvement. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Safety population. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 103 |
|
|
|
| Secondary | Change From Baseline in Growth Velocity at Week 101 : Height Z-Score | The Z-score, or Standard Deviation Score, is a measure of number of SDs above or below the average BMD of a healthy participant of the same age and gender. World Health Organization 2007 growth reference data were used for Z-score calculation. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Data was not reported as there was <50% of participants had evaluable data. | Posted | Baseline, Week 101 |
|
|
| Secondary | Change From Baseline in Skeletal Age at Week 103: Z-Score | Skeletal age was measured via radiography (X-ray) of the left hand and wrist by the method of Greulich and Pyle. The Z-score, or Standard Deviation Score, is a measure of number of SDs above or below the average BMD of a healthy participant of the same age and gender. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Data was not reported as there was <50% of participants had evaluable data. | Posted | Baseline, Week 103 |
|
|
| Secondary | Change From Baseline in Plasma Chitotriosidase Levels at Week 101 | Plasma chitotriosidase activity levels were measured using an enzymatic assay with 4-methylumbelliferyl-deoxychitobiose as a substrate. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Safety population. Number of participants analyzed signifies participants who were not deficient at baseline in chitotriosidase activity or who did not have a 24 base pair duplication in either copy of the chitotriosidase gene. | Posted | Mean | Standard Deviation | nanomole/milliliter/hour (nmol/mL/h) | Baseline, Week 101 |
|
|
|
| Secondary | Number of Participants With Change From Baseline in Neurological Status at Week 103 | Neurological status was considered normal or abnormal based on investigator's discretion. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Safety population. Number of participants analyzed signifies participants evaluable for this outcome. | Posted | Number | participants | Baseline, Week 103 |
|
|
|
| Secondary | Change From Baseline in Chemokine [C-C Motif] Ligand 18 (CCL18) Levels at Week 101 | Plasma CCL18 concentrations were measured using a time-resolved fluorescence assay. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint. | Safety population | Posted | Mean | Standard Deviation | nanogram per milliliter (ng/mL) | Baseline, Week 101 |
|
|
|
| 2 |
| 5 |
| 5 |
| 5 |
| Retinal detachment | Eye disorders | MedDRA (9.0) | Systematic Assessment |
|
| Vitreous opacities | Eye disorders | MedDRA (9.0) | Systematic Assessment |
|
| Retinal detachment | Eye disorders | MedDRA (9.0) | Systematic Assessment |
|
| Vitreous opacities | Eye disorders | MedDRA (9.0) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Enteritis | Gastrointestinal disorders | MedDRA (9.0) | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA (9.0) | Systematic Assessment |
|
| Adenoiditis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Hordeolum | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Otitis media acute | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Subcutaneous abscess | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (9.0) | Systematic Assessment |
|
| Concussion | Injury, poisoning and procedural complications | MedDRA (9.0) | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA (9.0) | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA (9.0) | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA (9.0) | Systematic Assessment |
|
| Heat stroke | Injury, poisoning and procedural complications | MedDRA (9.0) | Systematic Assessment |
|
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA (9.0) | Systematic Assessment |
|
| Wound | Injury, poisoning and procedural complications | MedDRA (9.0) | Systematic Assessment |
|
| Blood creatine phosphokinase increased | Investigations | MedDRA (9.0) | Systematic Assessment |
|
| C-Reactive protein increased | Investigations | MedDRA (9.0) | Systematic Assessment |
|
| Intraocular pressure increased | Investigations | MedDRA (9.0) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
| Muscle fatigue | Musculoskeletal and connective tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
| Trigger finger | Musculoskeletal and connective tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (9.0) | Systematic Assessment |
|
| Sleep disorder | Psychiatric disorders | MedDRA (9.0) | Systematic Assessment |
|
| Somatoform disorder | Psychiatric disorders | MedDRA (9.0) | Systematic Assessment |
|
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (9.0) | Systematic Assessment |
|
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
| D045762 |
| Enzymes and Coenzymes |
| Title | Measurements |
|---|---|
|