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All nine subjects dosed in the study have rolled over to the Long-Term Follow-up Study IVPRP-LT01 [NCT04628871]
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The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the IDS gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDS enzyme.
The objectives of the study are to provide long term expression of IDS and improve the current clinical outcome of enzyme replacement therapy (ERT) in subjects with MPS II, a recessive lysosomal storage disorder that results from mutations in the gene encoding IDS. SB-913 is a therapeutic for ZFN-mediated genome editing which will be delivered by adeno-associated virus (AAV)-derived vectors. SB-913 is intended to function by placement of the corrective copy of IDS transgene into the genome of the subject's own hepatocytes, under the control of the highly expressed endogenous albumin locus, and is expected to provide permanent, liver-specific expression of Iduronate 2-Sulfatase for the lifetime of an MPS II patient.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg | Experimental | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
|
| Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg | Experimental | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
|
| Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg | Experimental | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SB-913 | Biological | Single dose of each of the 3 components of SB-913: ZFN1, ZFN2 and hIDS Donor |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to 36 Months After the SB-913 Infusion | Number of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) in subjects who receive SB-913 as assessed by Common Terminology Criteria for Adverse Events (CTCAE) | Up to 36 months after the SB-913 infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Effect of SB-913 on IDS Activity | Change from baseline in clinical laboratory measurement of IDS activity measured in blood, at Month 33. | Baseline and Month 33 after the SB-913 infusion |
| Effect of SB-913 on Urine Glycosaminoglycans (GAG) Levels |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Sangamo Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | United States | ||
| Ann & Robert H. Lurie Children's Hospital of Chicago |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36299240 | Derived | Harmatz P, Prada CE, Burton BK, Lau H, Kessler CM, Cao L, Falaleeva M, Villegas AG, Zeitler J, Meyer K, Miller W, Wong Po Foo C, Vaidya S, Swenson W, Shiue LH, Rouy D, Muenzer J. First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B. Mol Ther. 2022 Dec 7;30(12):3587-3600. doi: 10.1016/j.ymthe.2022.10.010. Epub 2022 Oct 25. |
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The original enrollment goal was 32 subjects: Subjects with MPS II disease sequentially enrolled in age cohorts:
age >18 (adult cohorts 1 through 4), age 12-17 (pediatric cohorts 5 and 6), and age 5-11 (pediatric cohorts 7 and 8).
Due to the lack of observed clinical benefit, Sangamo Therapeutics, Inc. decided to stop enrollment in this study at 9 subjects, all adults in 3 cohorts. We continue to monitor the subjects in a 10-year, long-term follow-up study ST-IVPRP-LT01 (NCT04628871).
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| ID | Title | Description |
|---|---|---|
| FG000 | Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
| FG001 | Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Dec 12, 2019 | Mar 7, 2022 |
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Change from baseline in total GAG, Dermatan Sulfate GAG, and Heparan Sulfate GAG measured in urine at Month 36
| Baseline and 36 months after the SB-913 infusion |
| Annualized Frequency of Idursulfase (or Equivalent ERT) Administration. | Change from baseline in annualized frequency of idursulfase (or equivalent ERT) | Up to 36 months after the SB-913 infusion |
| AAV2/6 Clearance in Plasma, Saliva, Urine, Stool, and Semen | Subjects with AAV2/6 clearance in plasma, saliva, urine, stool, and semen by PCR by Week 24. All the subjects had AAV2/6 clearance in all the samples assessed (i.e., plasma, saliva, urine, stool, and semen) by week 24. Subjects were only tested until Week 24 because, by that time, they all had 3 consecutive negative tests in all body fluids. | Up to 36 months after the SB-913 infusion |
| Chicago |
| Illinois |
| 60611 |
| United States |
| NYU School of Medicine, Neurogenetics Division | New York | New York | 10016 | United States |
| University of North Carolina | Chapel Hill | North Carolina | 27514 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
| FG002 | Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
| BG001 | Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
| BG002 | Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. |
| BG003 | Total | Total of all reporting groups |
| 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 | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||
| Baseline Iduronate 2 Sulfatase (IDS) Results | Mean | Standard Deviation | nmol/mL/hr |
| |||||||||||||||
| Baseline Urine Glycosaminoglycans (GAG) Levels | Mean | Standard Deviation | (g/mol creatinine) |
|
| 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 Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to 36 Months After the SB-913 Infusion | Number of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) in subjects who receive SB-913 as assessed by Common Terminology Criteria for Adverse Events (CTCAE) | All subjects in this study who received any portion of the SB-913 infusion | Posted | Number | participants | Up to 36 months after the SB-913 infusion |
|
|
| ||||||||||||||||||||||||||||||||
| Secondary | Effect of SB-913 on IDS Activity | Change from baseline in clinical laboratory measurement of IDS activity measured in blood, at Month 33. | All subjects in this study who received any dose of the SB-913 infusion | Posted | Mean | Standard Deviation | nmol/mL/hr | Baseline and Month 33 after the SB-913 infusion |
| |||||||||||||||||||||||||||||||||
| Secondary | Effect of SB-913 on Urine Glycosaminoglycans (GAG) Levels | Change from baseline in total GAG, Dermatan Sulfate GAG, and Heparan Sulfate GAG measured in urine at Month 36 | All subjects in this study who received any dose of the SB-913 infusion | Posted | Mean | Standard Deviation | g/mol creatinine | Baseline and 36 months after the SB-913 infusion |
| |||||||||||||||||||||||||||||||||
| Secondary | Annualized Frequency of Idursulfase (or Equivalent ERT) Administration. | Change from baseline in annualized frequency of idursulfase (or equivalent ERT) | All subjects in this study who received any dose of the SB-913 infusion | Posted | Mean | Standard Deviation | Number of Infusions | Up to 36 months after the SB-913 infusion |
| |||||||||||||||||||||||||||||||||
| Secondary | AAV2/6 Clearance in Plasma, Saliva, Urine, Stool, and Semen | Subjects with AAV2/6 clearance in plasma, saliva, urine, stool, and semen by PCR by Week 24. All the subjects had AAV2/6 clearance in all the samples assessed (i.e., plasma, saliva, urine, stool, and semen) by week 24. Subjects were only tested until Week 24 because, by that time, they all had 3 consecutive negative tests in all body fluids. | All subjects in this study who received any portion of the SB-913 infusion | Posted | Count of Participants | Participants | Up to 36 months after the SB-913 infusion |
|
Adverse event data was collected from the subject's date of screening until their end of study visit which ranged from 24 to 36 months.
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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 | Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. | 0 | 2 | 1 | 2 | 2 | 2 |
| EG001 | Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. | 0 | 2 | 1 | 2 | 2 | 2 |
| EG002 | Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg | A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. | 0 | 5 | 3 | 5 | 5 | 5 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial Fibrillation | Cardiac disorders | Systematic Assessment |
| ||
| Incarcerated Umbilical Hernia | Gastrointestinal disorders | Systematic Assessment |
| ||
| Intestinal Obstruction | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment | fever |
| |
| Bronchitis | Infections and infestations | Systematic Assessment |
| ||
| Influenza | Infections and infestations | Systematic Assessment |
| ||
| Spinal Stenosis | Musculoskeletal and connective tissue disorders | Systematic Assessment | worsening |
| |
| Lacunar Stroke | Nervous system disorders | Systematic Assessment |
| ||
| Device Dislocation | Product Issues | Systematic Assessment |
| ||
| Respiratory Failure | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Leukopenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Splenomegaly | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Thrombocytopenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Aortic Stenosis | Vascular disorders | Systematic Assessment |
| ||
| Cardiomegaly | Cardiac disorders | Systematic Assessment |
| ||
| Left Atrial Dilatation | Cardiac disorders | Systematic Assessment |
| ||
| Lumbar Spinal Stenosis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Mitral Spinal Stenosis | Cardiac disorders | Systematic Assessment |
| ||
| Palpatations | Cardiac disorders | Systematic Assessment |
| ||
| Tachycardia | Cardiac disorders | Systematic Assessment |
| ||
| Tachypnea | Cardiac disorders | Systematic Assessment |
| ||
| Ear Congestion | Ear and labyrinth disorders | Systematic Assessment |
| ||
| Vertigo | Ear and labyrinth disorders | Systematic Assessment |
| ||
| Vision Blurred | Eye disorders | Systematic Assessment |
| ||
| Visual Impairment | Eye disorders | Systematic Assessment |
| ||
| Abdomen Pain, Upper | Gastrointestinal disorders | Systematic Assessment |
| ||
| Abdominal Discomfort | Gastrointestinal disorders | Systematic Assessment |
| ||
| Abdominal Pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| Abdominal Pain, Lower | Gastrointestinal disorders | Systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Dyspepsia | Gastrointestinal disorders | Systematic Assessment |
| ||
| Flatulence | Gastrointestinal disorders | Systematic Assessment |
| ||
| Food Poisoning | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastritis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastroesophageal Reflux Disease | Gastrointestinal disorders | Systematic Assessment |
| ||
| Jejunal Stenosis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Oesophageal Stenosis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pancreatic Cyst | Gastrointestinal disorders | Systematic Assessment |
| ||
| Chest Pain | General disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Impaired Healing | General disorders | Systematic Assessment |
| ||
| Influenza like Illness | General disorders | Systematic Assessment |
| ||
| Non-Cardiac Chest Pain | General disorders | Systematic Assessment |
| ||
| Prosthetic Cardiac Valve Stenosis | General disorders | Systematic Assessment |
| ||
| Thirst | General disorders | Systematic Assessment |
| ||
| Cholelithiasis | Hepatobiliary disorders | Systematic Assessment |
| ||
| Gastroenteritis Viral | Infections and infestations | Systematic Assessment |
| ||
| Hepatocellular Injury | Hepatobiliary disorders | Systematic Assessment |
| ||
| Hepatomegaly | Hepatobiliary disorders | Systematic Assessment |
| ||
| Mild Fibrosis in few portal tracts | Hepatobiliary disorders | Systematic Assessment |
| ||
| Portal Fibrosis | Hepatobiliary disorders | Systematic Assessment |
| ||
| Seasonal Allergy | Immune system disorders | Systematic Assessment |
| ||
| Bacterial Test Positive | Infections and infestations | Systematic Assessment |
| ||
| Blood Urine Present | Investigations | Systematic Assessment |
| ||
| Ear Infection | Infections and infestations | Systematic Assessment |
| ||
| Sinusitis | Infections and infestations | Systematic Assessment |
| ||
| Skin Candida | Infections and infestations | Systematic Assessment |
| ||
| Upper Respiratory Tract Congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Upper Respiratory Tract Infection | Infections and infestations | Systematic Assessment |
| ||
| Contusion | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Incision Site Pain | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Infusion Related Reaction | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Post Procedural Discharge | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Post Procedural Hematoma | Injury, poisoning and procedural complications | Systematic Assessment | following liver biosy |
| |
| Post Procedural Vomiting | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Procedural Pain | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Skin Abrasion | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Sunburn | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Alanine Aminotransferase Increased | Investigations | Systematic Assessment | elevated liver function test |
| |
| Aspartate Aminotransferase Increased | Investigations | Systematic Assessment | elevated liver function test |
| |
| Blood Phosphorus Decreased | Investigations | Systematic Assessment |
| ||
| Blood Pressure Systolic Increased | Investigations | Systematic Assessment |
| ||
| Blood Thyroid Stimulating Hormone Increased | Investigations | Systematic Assessment |
| ||
| CSF Glucose Decreased | Investigations | Systematic Assessment |
| ||
| Crystals Urine Present | Investigations | Systematic Assessment |
| ||
| Decreased Phosphorous | Investigations | Systematic Assessment |
| ||
| Lmphocyte Count Decreased | Investigations | Systematic Assessment |
| ||
| Neutrophils Count Increased | Investigations | Systematic Assessment |
| ||
| Prothrombin Time Prolonged | Investigations | Systematic Assessment |
| ||
| Red Cell Distribution Width Increased | Investigations | Systematic Assessment |
| ||
| Urinary Glycosaminoglycans Increased | Investigations | Systematic Assessment |
| ||
| White Blood Cell Count Increased | Investigations | Systematic Assessment |
| ||
| Obesity | Metabolism and nutrition disorders | Systematic Assessment | worsening |
| |
| Weight Decreased. | Investigations | Systematic Assessment |
| ||
| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Back Pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Bursitis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Cervical Spinal Stenosis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Cervical Vertebral Fracture | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Intervertebral Disc Degeneration | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Intervertebral Disc Protrusion | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Mobility Decreased | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Muscle Spasms Musculoskeletal Stiffness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Muscle Strain | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Muscular Weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Pain in Extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Cerebral Atrophy | Nervous system disorders | Systematic Assessment | thinning and atrophy (noted on ct) |
| |
| Dizziness | Nervous system disorders | Systematic Assessment |
| ||
| Dysarthria | Nervous system disorders | Systematic Assessment | transient slurred speech |
| |
| Dysgeusia | Nervous system disorders | Systematic Assessment |
| ||
| Hemiparesis | Nervous system disorders | Systematic Assessment |
| ||
| Hypaesthesia | Nervous system disorders | Systematic Assessment |
| ||
| White Matter Lesion | Nervous system disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Devise Dislocation | Product Issues | Systematic Assessment |
| ||
| Foreign Body Aspiration | Injury, poisoning and procedural complications | Systematic Assessment | aspiration from c-pap machine |
| |
| Agitation | Psychiatric disorders | Systematic Assessment |
| ||
| Anxiety | Psychiatric disorders | Systematic Assessment |
| ||
| Insomnia | Psychiatric disorders | Systematic Assessment |
| ||
| Irritability | Psychiatric disorders | Systematic Assessment |
| ||
| Suicidal Ideation | Psychiatric disorders | Systematic Assessment |
| ||
| Bacteriuria | Infections and infestations | Systematic Assessment |
| ||
| Hematuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Hepatic Lesion | Renal and urinary disorders | Systematic Assessment |
| ||
| Pollakiuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Proteinuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Renal Cyst | Renal and urinary disorders | Systematic Assessment |
| ||
| Urinary Incontinence | Renal and urinary disorders | Systematic Assessment |
| ||
| Urinary Tract Infection | Infections and infestations | Systematic Assessment |
| ||
| Scrotal Swelling | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Atelectasis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| COVID-19 | Infections and infestations | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | worsening |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | bloody nose |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
| ||
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pneumonia | Infections and infestations | Systematic Assessment |
| ||
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Sore Throat | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Tracheal Disorder | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Acne | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Application Site Rash | General disorders | Systematic Assessment |
| ||
| Basal Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment | left mid scalp (superficial and nodular patterns) |
| |
| Cold Sweat | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Erythema | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Pruritus | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Rash on wrists, bilaterally | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Skin Abrasion | Skin and subcutaneous tissue disorders | Systematic Assessment | arm |
| |
| Skin Irritation | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Loss Of Personal Independence In Daily Activities | Social circumstances | Systematic Assessment |
| ||
| Aortic Dilatation | Vascular disorders | Systematic Assessment |
| ||
| Deep Vein Thrombosis | Vascular disorders | Systematic Assessment | lower left extremity |
| |
| Flushing | Vascular disorders | Systematic Assessment | during and post ert infusion |
| |
| Hypertension | Vascular disorders | Systematic Assessment |
| ||
| Hypotension | Vascular disorders | Systematic Assessment |
| ||
| Lymphoedema | Vascular disorders | Systematic Assessment |
| ||
| Infusion Site Extravasation | General disorders | Systematic Assessment |
| ||
| Swelling | General disorders | Systematic Assessment |
| ||
| Lower Respiratory Infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Blood Lactate Dehydrogenase Increased | Investigations | Systematic Assessment |
| ||
| Hypoaesthesia | Nervous system disorders | Systematic Assessment |
| ||
| Urticaria | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Device Loosening | Product Issues | Systematic Assessment |
| ||
| Bronchitis | Infections and infestations | Systematic Assessment |
| ||
| Atrial Fibrillation | Cardiac disorders | Systematic Assessment |
| ||
| Incarcerated Umbilical Hernia | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Spinal Stenosis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
Due to the limited sample size of 9 subjects, the primary and secondary endpoints could not be analyzed, and this study could not report any conclusions.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Sangamo Therapeutics, Inc. | (510) 307-7266 | clinicaltrials@sangamo.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 30, 2020 | Mar 7, 2022 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D016532 | Mucopolysaccharidosis II |
| D013398 | Sudden Infant Death |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D003645 | Death, Sudden |
| D003643 | Death |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D066088 | Infant Death |
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Heparan Sulfate, Urine (g/mol creatinine) |
|
| Total GAG (g/mol creatinine) |
|
| Units | Counts |
|---|
| Participants |
|
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| Units | Counts |
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| Participants |
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| Units | Counts |
|---|---|
| Participants |
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A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion. SB-913: Single dose of each of the 3 components of SB-913: ZFN1, ZFN2 and hIDS Donor |
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