We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03041324
Recruitment Status : Terminated (All nine subjects dosed in the study have rolled over to the Long-Term Follow-up Study IVPRP-LT01 [NCT04628871])
First Posted : February 2, 2017
Results First Posted : September 27, 2022
Last Update Posted : September 27, 2022
Sponsor:
Information provided by (Responsible Party):
Sangamo Therapeutics

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Mucopolysaccharidosis II
MPS II
Intervention Biological: SB-913
Enrollment 9
Recruitment Details

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).

Pre-assignment Details  
Arm/Group Title Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Hide Arm/Group Description 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. 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. 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.
Period Title: Overall Study
Started 2 2 5
Completed 2 2 5
Not Completed 0 0 0
Arm/Group Title Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg Total
Hide Arm/Group Description 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. 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. 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. Total of all reporting groups
Overall Number of Baseline Participants 2 2 5 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 2 participants 5 participants 9 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
2
 100.0%
2
 100.0%
5
 100.0%
9
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 2 participants 5 participants 9 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
2
 100.0%
2
 100.0%
5
 100.0%
9
 100.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 2 participants 5 participants 9 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
  50.0%
0
   0.0%
0
   0.0%
1
  11.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
1
  50.0%
2
 100.0%
5
 100.0%
8
  88.9%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 2 participants 2 participants 5 participants 9 participants
2 2 5 9
Baseline Iduronate 2 Sulfatase (IDS) Results  
Mean (Standard Deviation)
Unit of measure:  nmol/mL/hr
Number Analyzed 2 participants 2 participants 5 participants 9 participants
4.93  (1.79605) 1.21  (0.55154) 7.054  (3.74949) 5.2833  (3.68909)
Baseline Urine Glycosaminoglycans (GAG) Levels  
Mean (Standard Deviation)
Unit of measure:  (g/mol creatinine)
Number Analyzed 2 participants 2 participants 5 participants 9 participants
Dermatan Sulfate, Urine (g/mol creatinine) 4.255  (1.70413) 5.165  (1.83141) 4.094  (0.79563) 4.3678  (1.14354)
Heparan Sulfate, Urine (g/mol creatinine) 4.955  (0.24749) 9.795  (4.16486) 5.074  (0.76843) 6.0967  (2.62108)
Total GAG (g/mol creatinine) 4.785  (1.44957) 6.88  (0.9051) 4.816  (2.96001) 5.2678  (2.36252)
1.Primary Outcome
Title Number of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to 36 Months After the SB-913 Infusion
Hide Description 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)
Time Frame Up to 36 months after the SB-913 infusion
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects in this study who received any portion of the SB-913 infusion
Arm/Group Title Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Hide Arm/Group Description:

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

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

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

Overall Number of Participants Analyzed 2 2 5
Measure Type: Number
Unit of Measure: participants
2 2 5
2.Secondary Outcome
Title Effect of SB-913 on IDS Activity
Hide Description Change from baseline in clinical laboratory measurement of IDS activity measured in blood, at Month 33.
Time Frame Baseline and Month 33 after the SB-913 infusion
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects in this study who received any dose of the SB-913 infusion
Arm/Group Title Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 2 0 0
Mean (Standard Deviation)
Unit of Measure: nmol/mL/hr
-2.6600  (3.5213)
3.Secondary Outcome
Title Effect of SB-913 on Urine Glycosaminoglycans (GAG) Levels
Hide Description Change from baseline in total GAG, Dermatan Sulfate GAG, and Heparan Sulfate GAG measured in urine at Month 36
Time Frame Baseline and 36 months after the SB-913 infusion
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects in this study who received any dose of the SB-913 infusion
Arm/Group Title Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 2 1 5
Mean (Standard Deviation)
Unit of Measure: g/mol creatinine
Total GAG 1.035  (0.03536) -2.6 [1]   (NA) 1.734  (1.58357)
Dermatan Sulfate Urine -0.025  (1.18087) -3.39 [2]   (NA) 0.262  (0.6985)
Heparan Sulfate Urine 1.895  (2.39709) -7.6 [3]   (NA) 2.880  (2.91261)
[1]
For total GAG, at month 36, only one patient in cohort 2 had a sample tested for total GAG, so no standard deviation was available.
[2]
For DS GAG, at month 36, only one patient in cohort 2 had a sample tested for DS GAG, so no standard deviation was available.
[3]
For HS GAG, at month 36, only one patient in cohort 2 had a sample tested for HS GAG, so no standard deviation was available.
4.Secondary Outcome
Title Annualized Frequency of Idursulfase (or Equivalent ERT) Administration.
Hide Description Change from baseline in annualized frequency of idursulfase (or equivalent ERT)
Time Frame Up to 36 months after the SB-913 infusion
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects in this study who received any dose of the SB-913 infusion
Arm/Group Title Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 1 2 5
Mean (Standard Deviation)
Unit of Measure: Number of Infusions
-2.8 [1]   (NA) 9.9  (25.5) -0.5  (6.13)
[1]
Only one subject in this cohort had ERT withdrawal, so no standard deviation is available.
5.Secondary Outcome
Title AAV2/6 Clearance in Plasma, Saliva, Urine, Stool, and Semen
Hide Description Subjects with AAV2/6 clearance in plasma, saliva, urine, stool, and semen by PCR by Week 24.
Time Frame Up to 36 months after the SB-913 infusion
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects in this study who received any portion of the SB-913 infusion
Arm/Group Title Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Hide Arm/Group Description:

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

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

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

Overall Number of Participants Analyzed 2 2 5
Measure Type: Count of Participants
Unit of Measure: Participants
2
 100.0%
2
 100.0%
5
 100.0%
Time Frame 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.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Hide Arm/Group Description 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. 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. 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.
All-Cause Mortality
Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/2 (0.00%)      0/2 (0.00%)      0/5 (0.00%)    
Hide Serious Adverse Events
Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/2 (50.00%)      1/2 (50.00%)      3/5 (60.00%)    
Cardiac disorders       
Atrial Fibrillation   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Gastrointestinal disorders       
Incarcerated Umbilical Hernia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Intestinal Obstruction   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
General disorders       
Pyrexia  [1]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Infections and infestations       
Bronchitis   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Influenza   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Spinal Stenosis  [2]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Nervous system disorders       
Lacunar Stroke   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Product Issues       
Device Dislocation   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Respiratory, thoracic and mediastinal disorders       
Respiratory Failure   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Indicates events were collected by systematic assessment
[1]
fever
[2]
worsening
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/2 (100.00%)      2/2 (100.00%)      5/5 (100.00%)    
Blood and lymphatic system disorders       
Anemia   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Leukopenia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Splenomegaly   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Thrombocytopenia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Cardiac disorders       
Cardiomegaly   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Left Atrial Dilatation   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Mitral Spinal Stenosis   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Palpatations   0/2 (0.00%)  0 1/2 (50.00%)  4 0/5 (0.00%)  0
Tachycardia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Tachypnea   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Atrial Fibrillation   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Ear and labyrinth disorders       
Ear Congestion   0/2 (0.00%)  0 0/2 (0.00%)  0 2/5 (40.00%)  2
Vertigo   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Eye disorders       
Vision Blurred   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Visual Impairment   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Gastrointestinal disorders       
Abdomen Pain, Upper   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  1
Abdominal Discomfort   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Abdominal Pain   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  1
Abdominal Pain, Lower   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Constipation   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  1
Diarrhea   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Dyspepsia   1/2 (50.00%)  2 0/2 (0.00%)  0 1/5 (20.00%)  2
Flatulence   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Food Poisoning   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Gastritis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Gastroesophageal Reflux Disease   1/2 (50.00%)  4 0/2 (0.00%)  0 1/5 (20.00%)  1
Jejunal Stenosis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Nausea   0/2 (0.00%)  0 1/2 (50.00%)  1 2/5 (40.00%)  2
Oesophageal Stenosis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Pancreatic Cyst   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Incarcerated Umbilical Hernia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
General disorders       
Chest Pain   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Fatigue   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  2
Impaired Healing   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Influenza like Illness   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Non-Cardiac Chest Pain   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Prosthetic Cardiac Valve Stenosis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Thirst   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Application Site Rash   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Infusion Site Extravasation   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Swelling   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Pyrexia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Hepatobiliary disorders       
Cholelithiasis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Hepatocellular Injury   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Hepatomegaly   0/2 (0.00%)  0 0/2 (0.00%)  0 2/5 (40.00%)  2
Mild Fibrosis in few portal tracts   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Portal Fibrosis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Immune system disorders       
Seasonal Allergy   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Infections and infestations       
Gastroenteritis Viral   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Bacterial Test Positive   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Ear Infection   1/2 (50.00%)  1 0/2 (0.00%)  0 1/5 (20.00%)  1
Sinusitis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Skin Candida   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Upper Respiratory Tract Infection   2/2 (100.00%)  8 0/2 (0.00%)  0 0/5 (0.00%)  0
Bacteriuria   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Urinary Tract Infection   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  1
COVID-19   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Nasopharyngitis   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Pneumonia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Bronchitis   1/2 (50.00%)  1 0/2 (0.00%)  0 1/5 (20.00%)  1
Injury, poisoning and procedural complications       
Contusion   0/2 (0.00%)  0 1/2 (50.00%)  2 1/5 (20.00%)  2
Incision Site Pain   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Infusion Related Reaction   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  1
Post Procedural Discharge   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  2
Post Procedural Hematoma  [1]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Post Procedural Vomiting   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Procedural Pain   1/2 (50.00%)  2 2/2 (100.00%)  3 2/5 (40.00%)  3
Skin Abrasion   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Sunburn   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Cervical Vertebral Fracture   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Muscle Strain   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Foreign Body Aspiration  [2]  1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Skin Irritation   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Investigations       
Blood Urine Present   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Alanine Aminotransferase Increased  [3]  1/2 (50.00%)  1 1/2 (50.00%)  1 2/5 (40.00%)  2
Aspartate Aminotransferase Increased  [3]  1/2 (50.00%)  1 1/2 (50.00%)  2 2/5 (40.00%)  2
Blood Phosphorus Decreased   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Blood Pressure Systolic Increased   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  4
Blood Thyroid Stimulating Hormone Increased   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
CSF Glucose Decreased   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Crystals Urine Present   0/2 (0.00%)  0 0/2 (0.00%)  0 2/5 (40.00%)  2
Decreased Phosphorous   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Lmphocyte Count Decreased   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Neutrophils Count Increased   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Prothrombin Time Prolonged   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Red Cell Distribution Width Increased   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Urinary Glycosaminoglycans Increased   1/2 (50.00%)  1 0/2 (0.00%)  0 1/5 (20.00%)  1
White Blood Cell Count Increased   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Weight Decreased.   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Blood Lactate Dehydrogenase Increased   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Metabolism and nutrition disorders       
Obesity  [4]  1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Lumbar Spinal Stenosis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Arthralgia   0/2 (0.00%)  0 0/2 (0.00%)  0 2/5 (40.00%)  2
Back Pain   1/2 (50.00%)  1 1/2 (50.00%)  2 1/5 (20.00%)  1
Bursitis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Cervical Spinal Stenosis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Intervertebral Disc Degeneration   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Intervertebral Disc Protrusion   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Mobility Decreased   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Muscle Spasms Musculoskeletal Stiffness   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Muscular Weakness   1/2 (50.00%)  1 0/2 (0.00%)  0 1/5 (20.00%)  1
Pain in Extremity   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Spinal Stenosis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Basal Cell Carcinoma  [5]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Nervous system disorders       
Cerebral Atrophy  [6]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Dizziness   1/2 (50.00%)  1 0/2 (0.00%)  0 1/5 (20.00%)  1
Dysarthria  [7]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Dysgeusia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Hemiparesis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Hypaesthesia   0/2 (0.00%)  0 0/2 (0.00%)  0 2/5 (40.00%)  2
White Matter Lesion   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Headache   0/2 (0.00%)  0 1/2 (50.00%)  3 1/5 (20.00%)  2
Hypoaesthesia   0/2 (0.00%)  0 0/2 (0.00%)  0 2/5 (40.00%)  2
Product Issues       
Devise Dislocation   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Device Loosening   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Psychiatric disorders       
Agitation   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Anxiety   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Insomnia   0/2 (0.00%)  0 1/2 (50.00%)  2 1/5 (20.00%)  1
Irritability   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Suicidal Ideation   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Renal and urinary disorders       
Hematuria   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Hepatic Lesion   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Pollakiuria   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Proteinuria   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Renal Cyst   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Urinary Incontinence   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Reproductive system and breast disorders       
Scrotal Swelling   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Respiratory, thoracic and mediastinal disorders       
Upper Respiratory Tract Congestion   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  1
Atelectasis   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Cough  [4]  1/2 (50.00%)  1 0/2 (0.00%)  0 1/5 (20.00%)  1
Dyspnoea   0/2 (0.00%)  0 1/2 (50.00%)  2 1/5 (20.00%)  2
Epistaxis  [8]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Hypoxia   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Nasal Congestion   0/2 (0.00%)  0 0/2 (0.00%)  0 2/5 (40.00%)  2
Oropharyngeal Pain   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Pneumothorax   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Rhinorrhea   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  2
Sore Throat   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Tracheal Disorder   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Lower Respiratory Infection   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Skin and subcutaneous tissue disorders       
Acne   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Cold Sweat   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Erythema   0/2 (0.00%)  0 1/2 (50.00%)  1 0/5 (0.00%)  0
Pruritus   1/2 (50.00%)  1 0/2 (0.00%)  0 1/5 (20.00%)  1
Rash on wrists, bilaterally   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Skin Abrasion  [9]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Urticaria   1/2 (50.00%)  1 0/2 (0.00%)  0 1/5 (20.00%)  1
Social circumstances       
Loss Of Personal Independence In Daily Activities   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Vascular disorders       
Aortic Stenosis   1/2 (50.00%)  1 0/2 (0.00%)  0 0/5 (0.00%)  0
Aortic Dilatation   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Deep Vein Thrombosis  [10]  0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Flushing  [11]  0/2 (0.00%)  0 1/2 (50.00%)  2 2/5 (40.00%)  2
Hypertension   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  1
Hypotension   0/2 (0.00%)  0 1/2 (50.00%)  1 1/5 (20.00%)  1
Lymphoedema   0/2 (0.00%)  0 0/2 (0.00%)  0 1/5 (20.00%)  1
Indicates events were collected by systematic assessment
[1]
following liver biosy
[2]
aspiration from c-pap machine
[3]
elevated liver function test
[4]
worsening
[5]
left mid scalp (superficial and nodular patterns)
[6]
thinning and atrophy (noted on ct)
[7]
transient slurred speech
[8]
bloody nose
[9]
arm
[10]
lower left extremity
[11]
during and post ert infusion
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.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Monitor
Organization: Sangamo Therapeutics, Inc.
Phone: (510) 307-7266
EMail: clinicaltrials@sangamo.com
Layout table for additonal information
Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT03041324    
Other Study ID Numbers: SB-913-1602
First Submitted: January 13, 2017
First Posted: February 2, 2017
Results First Submitted: May 7, 2022
Results First Posted: September 27, 2022
Last Update Posted: September 27, 2022