Trial Outcomes & Findings for Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy (NCT NCT03333590)
NCT ID: NCT03333590
Last Updated: 2025-03-13
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
2 participants
Primary outcome timeframe
2 years
Results posted on
2025-03-13
Participant Flow
Participant milestones
| Measure |
Cohort 1 (Minimal Efficacious Dose) rAAVrh74.MCK.GALGT2
N = 3 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2 (Minimal Efficacious Dose) rAAVrh74.MCK.GALGT2
N = 3 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
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|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
|
Overall Study
COMPLETED
|
1
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
Cohort 1 (Minimal Efficacious Dose) rAAVrh74.MCK.GALGT2
n=1 Participants
N = 3 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2 (Dose Escalation) rAAVrh74.MCK.GALGT2
n=1 Participants
N=3 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
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Total
n=2 Participants
Total of all reporting groups
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|---|---|---|---|
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Age, Continuous
|
9 years
n=99 Participants
|
7 years
n=107 Participants
|
8 years
n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=99 Participants
|
1 participants
n=107 Participants
|
2 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Cohort 1 (Minimal Efficacious Dose)
n=1 Participants
N = 1 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2
n=1 Participants
N = 1 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
|---|---|---|
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Number of Unanticipated Grade III or Higher Treatment-Related Toxicities
|
0 events
|
0 events
|
SECONDARY outcome
Timeframe: Day 90 (Cohort 2) and Day 120 (Cohort 1)Percentage of fibers expressing GALGT2 in each biopsy sample.
Outcome measures
| Measure |
Cohort 1 (Minimal Efficacious Dose)
n=1 Biopsies
N = 1 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2
n=1 Biopsies
N = 1 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
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|---|---|---|
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Expression of GALGT2 as Demonstrated by Immunofluorescent Staining With Anti-CT Epitope Antibodies or WFA Lectin in Muscle Biopsy Sections at 120 Days Post Injection (Cohort 1) and 90 Days Post-injection (Cohort 2).
|
1.95 Percentage of Positive Fibers
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1.72 Percentage of Positive Fibers
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SECONDARY outcome
Timeframe: Day 90 (Cohort 2) and Day 120 (Cohort 1)Outcome measures
| Measure |
Cohort 1 (Minimal Efficacious Dose)
n=1 Biopsies
N = 1 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2
n=1 Biopsies
N = 1 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
|---|---|---|
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GALGT2 Protein Expression Quantified by Western Blot and Assessed by Densitometry in Muscle Biopsy Tissue at 120 Days Post-injection (Cohort 1) and 90 Days Post-injection (Cohort 2)
|
12 ng/mg total protein
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14.6 ng/mg total protein
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OTHER_PRE_SPECIFIED outcome
Timeframe: Day 90 (Cohort 2) and Day 120 (Cohort 1) and Day 180 for both cohortsOutcome measures
| Measure |
Cohort 1 (Minimal Efficacious Dose)
n=1 Participants
N = 1 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2
n=1 Participants
N = 1 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
|---|---|---|
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Number of Meters Walked During the 6 Minute Walk Test
Day 90 (Cohort 2) /Day 120 (Cohort 1)
|
320 meters
|
405 meters
|
|
Number of Meters Walked During the 6 Minute Walk Test
Day 180
|
324 meters
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416 meters
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OTHER_PRE_SPECIFIED outcome
Timeframe: Days 90 (Cohort 2), 120 (Cohort 1) and both Cohorts at Day 180, Months 12, 18 and 24Outcome measures
| Measure |
Cohort 1 (Minimal Efficacious Dose)
n=1 Participants
N = 1 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2
n=1 Participants
N = 1 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
|---|---|---|
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Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Day 180-Right Knee Flexion
|
6.1 kg
|
4.24 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Day 90/Day 120-Right Knee Extension
|
7.42 kg
|
7.04 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Day 90/Day 120-Right Knee Flexion
|
6.06 kg
|
8.12 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Day 90/Day 120- Left Knee Extension
|
8.78 kg
|
5.9 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Day 90/Day 120-Left Knee Flexion
|
6.12 kg
|
8.4 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Day 180-Right Knee Extension
|
7.13 kg
|
9.73 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Day 180-Left Knee Extension
|
8.66 kg
|
8.19 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Day 180-Left Knee Flexion
|
6.69 kg
|
5.25 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 12-Right Knee Extension
|
7.49 kg
|
9.85 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 12-Right Knee Flexion
|
5.67 kg
|
5.85 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 12-Left Knee Extension
|
7.5 kg
|
8.02 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 12-Left Knee Flexion
|
5.32 kg
|
5.12 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 18-Right Knee Extension
|
4.55 kg
|
7.67 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 18-Right Knee Flexion
|
6.11 kg
|
6.89 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 18-Left Knee Extension
|
4.96 kg
|
7.34 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 18-Left Knee Flexion
|
6.26 kg
|
6.08 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 24-Right Knee Extension
|
5.06 kg
|
9.81 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 24-Right Knee Flexion
|
4.41 kg
|
5.04 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 24-Left Knee Extension
|
6.93 kg
|
5.21 kg
|
|
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
Month 24-Left Knee Flexion
|
4.17 kg
|
4.87 kg
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Days 90 (Cohort 2), 120 (Cohort 1); both Cohorts at Day 180, Months 12, 18 and Cohort 2 at Month 24Population: There is no data for Cohort 1, Month 24 because single subject was unable to perform the test.
Outcome measures
| Measure |
Cohort 1 (Minimal Efficacious Dose)
n=1 Participants
N = 1 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2
n=1 Participants
N = 1 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
|---|---|---|
|
Time Taken to Walk 100 Meters During the 100 Meter Walk Test.
Day 90/Day 120
|
98.2 seconds
|
56.1 seconds
|
|
Time Taken to Walk 100 Meters During the 100 Meter Walk Test.
Day 180
|
110.9 seconds
|
44.9 seconds
|
|
Time Taken to Walk 100 Meters During the 100 Meter Walk Test.
Month 12
|
144.5 seconds
|
44.7 seconds
|
|
Time Taken to Walk 100 Meters During the 100 Meter Walk Test.
Month 18
|
167.8 seconds
|
65.6 seconds
|
|
Time Taken to Walk 100 Meters During the 100 Meter Walk Test.
Month 24
|
—
|
48.4 seconds
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Days 90 (Cohort 2), 120 (Cohort 1) and both Cohorts at Day 180, Months 12, 18 and 24The NSAA provides a score between 0 and 34 where higher numbers represent greater muscle function.
Outcome measures
| Measure |
Cohort 1 (Minimal Efficacious Dose)
n=1 Participants
N = 1 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 2
n=1 Participants
N = 1 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
|---|---|---|
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Score of Muscle Function Using the The North Star Ambulatory Assessment (NSAA).
Day 90/Day 120
|
16 score on a scale
|
21 score on a scale
|
|
Score of Muscle Function Using the The North Star Ambulatory Assessment (NSAA).
Day 180
|
14 score on a scale
|
23 score on a scale
|
|
Score of Muscle Function Using the The North Star Ambulatory Assessment (NSAA).
Month 12
|
10 score on a scale
|
23 score on a scale
|
|
Score of Muscle Function Using the The North Star Ambulatory Assessment (NSAA).
Month 18
|
6 score on a scale
|
23 score on a scale
|
|
Score of Muscle Function Using the The North Star Ambulatory Assessment (NSAA).
Month 24
|
2 score on a scale
|
23 score on a scale
|
Adverse Events
Cohort 1 (Minimal Efficacious Dose) rAAVrh74.MCK.GALGT2
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Cohort 1 rAAVrh74.MCK.GALGT2
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort 1 (Minimal Efficacious Dose) rAAVrh74.MCK.GALGT2
n=1 participants at risk
N = 1 \[2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
Cohort 1 rAAVrh74.MCK.GALGT2
n=1 participants at risk
N = 1 \[5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)\]
rAAVrh74.MCK.GALGT2: Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)
|
|---|---|---|
|
Vascular disorders
Bleeding at the femoral catheterization site
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
0.00%
0/1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Vascular disorders
Bruising
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Cardiac disorders
Decrease in ejection fraction
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
0.00%
0/1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Blood and lymphatic system disorders
Low Hemoglobin
|
0.00%
0/1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Blood and lymphatic system disorders
Decreased Lymphocyte
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
100.0%
1/1 • Number of events 2 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Endocrine disorders
Cushingoid Face
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
0.00%
0/1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Blood and lymphatic system disorders
Pharyngitis and Adenitis
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
0.00%
0/1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Endocrine disorders
Weight Gain
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
0.00%
0/1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
|
Cardiac disorders
Tachycardia
|
100.0%
1/1 • Number of events 1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
0.00%
0/1 • Adverse events started to be collected on Day 0 as defined by the day of Gene Transfer and continued through during the active 2-year period following gene transfer as described in the protocol.
|
Additional Information
Dr. Kevin Flanigan
Abigail Wexner Research Institute at Nationwide Children's Hospital
Phone: 614-355-2947
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place