Hemophilia B
Conditions
Keywords
Hemophilia B, gene therapy
Brief summary
This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.
Interventions
GENETICAAV5-hFIX
AAV5hFIX gene therapy
Sponsors
CSL Behring
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male 2. Age ≥ 18 years 3. Patients with congenital hemophilia B classified as one of the following: * Known severe FIX deficiency with plasma FIX activity level \< 1% and a severe bleeding phenotype defined by one of the following: * Currently on prophylactic FIX replacement therapy for a history of bleeding * Currently on on-demand therapy with a current or past history of frequent bleeding defined as four or more bleeding episodes in the last 12 months or chronic hemophilic arthropathy (pain, joint destruction, and loss of range of motion) in one or more joints * Known moderately severe FIX deficiency with plasma FIX activity level between ≥ 1% and ≤ 2% and a severe bleeding phenotype defined by one of the following: * Currently on prophylactic FIX replacement therapy for a history of bleeding * Currently on on-demand therapy with a current or past history of frequent bleeding defined as four or more bleeding episodes in the last 12 months or chronic hemophilic arthropathy (pain, joint destruction, and loss of range of motion) in one or more joints 4. More than 150 previous exposure days of treatment with FIX protein. 5. Acceptance to use a condom during sexual intercourse in the period from Investigational Medicinal Product (IMP) administration until AAV5 has been cleared from semen, as evidenced by the central laboratory from negative analysis results for at least 3 consecutively collected semen samples (this criterion is applicable also for subjects who are surgically sterilized) 6. Following receipt of verbal and written information about the trial, the subject has provided signed informed consent before any trial related activity is carried out.
Exclusion criteria
1. History of FIX inhibitors measured to be ≥ 0.6 Bethesda Units (BU)/mL 2. FIX inhibitors ≥ 0.6 BU/mL at Visit 1 (measured by the local laboratory) 3. Neutralizing antibodies against AAV5 at Visit 1 (measured by the central laboratory) 4. Visit 1 laboratory values (measured by the central laboratory): * alanine aminotransferase \> 2 times upper normal limit * aspartate aminotransferase \> 2 times upper normal limit * total bilirubin \> 2 times upper normal limit * alkaline phosphatase \> 2 times upper normal limit * creatinine \> 1.5 times upper normal limit 5. Positive HIV serological test at Visit 1, not controlled with anti-viral therapy as shown by cluster of differentiation 4+ counts ≤ 200 per μL or by a viral load of \>200 copies per mL (measured by the central laboratory) 6. Active infection with Hepatitis B or C virus as reflected by Hepatitis B Surface Antigen (HBsAg), Hepatitis B extracellular Antigen (HBeAg), Hepatitis B Virus DeoxyriboNucleic Acid (HBV DNA) or Hepatitis C Virus RiboNucleic Acid (HCV RNA) positivity, respectively, at Visit 1 (measured by the central laboratory). 7. History of Hepatitis B or C exposure, currently controlled by antiviral therapy 8. Any coagulation disorder other than hemophilia B 9. Thrombocytopenia, defined as a platelet count below 50 × 10E9 / L, at Visit 1 (measured by the central laboratory) 10. Body mass index \< 16 or ≥ 35 kg/m2 11. Planned surgery for the initial 6 months after IMP administration in this trial 12. Previous arterial or venous thrombotic event (e.g. acute myocardial infarction, cerebrovascular disease and venous thrombosis) 13. Active severe infection or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, haematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, alcoholism, drug dependency or any other psychological disorder evaluated by the investigator to interfere with adherence to the protocol procedures or with the degree of tolerance to the IMP 14. Known significant medical condition including disseminated intravascular coagulation, fibrinolysis and liver fibrosis which, in the opinion of the investigator, may confound, contraindicate or limit the interpretation of either safety or efficacy data 15. Known history of an allergic reaction or anaphylaxis to FIX products 16. Known uncontrolled allergic conditions or allergy/hypersensitivity to any component of the IMP excipients 17. Previous gene therapy treatment and/or previous participation in a gene therapy clinical trial 18. Receipt of an experimental agent within 60 days prior to Visit 1 19. Current participation or anticipated participation within one year after IMP administration in this trial in any other interventional clinical trial involving drugs or devices.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Adverse Events | From AMT-060 infusion through end of study (5 years post-dose) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Total Annualized Bleeding Rate (ABR) | From AMT-060 infusion through end of study (5 years post-dose) | Annualized: Sum of post-treatment bleeding episodes divided by subjects' average number of years (365.25 days) from treatment start to until the data cutoff date. |
| Total Consumption of FIX Replacement Therapy | From AMT-060 infusion through end of study (5 years post dose). | — |
| Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | From AMT-060 infusion through the end of study (5 years post dose) | Scores range from 0 to 100, with a higher score defining a more favorable health state. |
| Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | From AMT-060 infusion through end of study (5 years post dose). | — |
| Number of Subjects Developing Neutralizing Antibodies to AAV5 | From AMT-060 infusion through end of study (5 years post dose) | — |
| FIX-replacement-therapy-free FIX Activity | From AMT-060 infusion through end of study (5 years post-dose) | FIX activity measured any time from 72 hours after latest FIX replacement therapy administration and until next administration of FIX replacement therapy. Only assessments performed more than 10 days after most recent FIX-replacement therapy administration included. |
| Number of Subjects With a Positive AAV5 Capsid-specific T Cell Response | From AMT-060 infusion through 26 weeks post-dose | Specific AAV5 response (results \>17 SFC/million PBMCs) were regarded as positive. |
| Number of Subjects With Antibodies to FIX | From AMT-060 infusion through the end of study (5 years post dose) | — |
| Number of Subjects With FIX Inhibitors | From AMT-060 infusion through the end of study (5 years post dose) | — |
| Number of Subjects With Clinically Significant Inflammatory Markers: IL-1β, IL-2, IL-6, INFγ, MCP-1 | From AMT-060 infusion through 18 weeks post dose | — |
| Total IgG and IgM Antibody Titers to AAV5 | AMT-060 infusion through end of study (5 years post dose) | For subjects with a titer of 109350 and 50, the actual titer is \>109350 and \<50. |
Countries
Denmark, Germany, Netherlands
Participant flow
Pre-assignment details
Patients with congenital haemophilia B. Known severe FIX deficiency with plasma FIX activity level \< 1% and a severe bleeding phenotype. Known moderately severe FIX deficiency with plasma FIX activity level between ≥ 1% and ≤ 2% and a severe bleeding phenotype. More than 150 previous exposure days of treatment with FIX protein
Participants by arm
| Arm | Count |
|---|---|
| AAV5-hFIX Low Dose (Cohort 1) AAV5-hFIX 5 × 10E12 gc/kg intravenous single infusion
AAV5-hFIX: AAV5hFIX gene therapy | 5 |
| AAV5-hFIX High Dose (Cohort 2) AAV5-hFIX 2 × 10E13 gc/kg intravenous single infusion
AAV5-hFIX: AAV5hFIX gene therapy | 5 |
| Total | 10 |
Baseline characteristics
| Characteristic | AAV5-hFIX Low Dose (Cohort 1) | AAV5-hFIX High Dose (Cohort 2) | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 3 Participants | 0 Participants | 3 Participants |
| Age, Categorical Between 18 and 65 years | 2 Participants | 5 Participants | 7 Participants |
| Age, Continuous | 60.2 years STANDARD_DEVIATION 15.9 | 38.2 years STANDARD_DEVIATION 5.9 | 49.2 years STANDARD_DEVIATION 16.2 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 5 Participants | 5 Participants | 10 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 5 Participants | 4 Participants | 9 Participants |
| Region of Enrollment Denmark | 1 participants | 0 participants | 1 participants |
| Region of Enrollment Germany | 1 participants | 2 participants | 3 participants |
| Region of Enrollment Netherlands | 3 participants | 3 participants | 6 participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 5 Participants | 5 Participants | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 5 | 0 / 5 |
| other Total, other adverse events | 5 / 5 | 5 / 5 |
| serious Total, serious adverse events | 3 / 5 | 2 / 5 |
Outcome results
Primary
Number of Participants With Adverse Events
Time frame: From AMT-060 infusion through end of study (5 years post-dose)
Population: Full Analysis Set (FAS) which was comprised of all dosed subjects
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Number of Participants With Adverse Events | 5 participants |
| AAV5-hFIX High Dose (Cohort 2) | Number of Participants With Adverse Events | 5 participants |
Secondary
Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores
Scores range from 0 to 100, with a higher score defining a more favorable health state.
Time frame: From AMT-060 infusion through the end of study (5 years post dose)
Population: FAS
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Physical Functioning | 0.00 score on a scale | Standard Deviation 10 |
| AAV5-hFIX Low Dose (Cohort 1) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Role-Physical | -15.00 score on a scale | Standard Deviation 8.39 |
| AAV5-hFIX Low Dose (Cohort 1) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Bodily Pain | -9.00 score on a scale | Standard Deviation 9 |
| AAV5-hFIX Low Dose (Cohort 1) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | General Health | -0.80 score on a scale | Standard Deviation 20.22 |
| AAV5-hFIX Low Dose (Cohort 1) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Vitality | -11.25 score on a scale | Standard Deviation 19.96 |
| AAV5-hFIX Low Dose (Cohort 1) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Social Functioning | -20.00 score on a scale | Standard Deviation 25.92 |
| AAV5-hFIX Low Dose (Cohort 1) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Role-Emotional | -13.33 score on a scale | Standard Deviation 27.39 |
| AAV5-hFIX Low Dose (Cohort 1) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Mental Health | -13.00 score on a scale | Standard Deviation 22.8 |
| AAV5-hFIX High Dose (Cohort 2) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Mental Health | -9.00 score on a scale | Standard Deviation 12.94 |
| AAV5-hFIX High Dose (Cohort 2) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Physical Functioning | -7.00 score on a scale | Standard Deviation 9.75 |
| AAV5-hFIX High Dose (Cohort 2) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Vitality | -6.25 score on a scale | Standard Deviation 12.5 |
| AAV5-hFIX High Dose (Cohort 2) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Role-Physical | -10.00 score on a scale | Standard Deviation 22.79 |
| AAV5-hFIX High Dose (Cohort 2) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Role-Emotional | -10.00 score on a scale | Standard Deviation 13.69 |
| AAV5-hFIX High Dose (Cohort 2) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Bodily Pain | 1.20 score on a scale | Standard Deviation 14.81 |
| AAV5-hFIX High Dose (Cohort 2) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | Social Functioning | -5.00 score on a scale | Standard Deviation 14.25 |
| AAV5-hFIX High Dose (Cohort 2) | Change From Baseline in Short Form-36 (SF-36) Quality of Life (QoL) Scores | General Health | -2.40 score on a scale | Standard Deviation 8.99 |
Secondary
FIX-replacement-therapy-free FIX Activity
FIX activity measured any time from 72 hours after latest FIX replacement therapy administration and until next administration of FIX replacement therapy. Only assessments performed more than 10 days after most recent FIX-replacement therapy administration included.
Time frame: From AMT-060 infusion through end of study (5 years post-dose)
Population: FAS. Only assessments performed more than 10 days after most recent FIX-replacement therapy administration included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | FIX-replacement-therapy-free FIX Activity | one-stage aPTT assay | 7.43 Percent FIX activity | Standard Deviation 1.28 |
| AAV5-hFIX Low Dose (Cohort 1) | FIX-replacement-therapy-free FIX Activity | amidolytic/chromogenic assay | 4.58 Percent FIX activity | Standard Deviation 2.88 |
| AAV5-hFIX High Dose (Cohort 2) | FIX-replacement-therapy-free FIX Activity | one-stage aPTT assay | 6.60 Percent FIX activity | Standard Deviation 1.96 |
| AAV5-hFIX High Dose (Cohort 2) | FIX-replacement-therapy-free FIX Activity | amidolytic/chromogenic assay | 4.74 Percent FIX activity | Standard Deviation 1.43 |
Secondary
Number of Subjects Developing Neutralizing Antibodies to AAV5
Time frame: From AMT-060 infusion through end of study (5 years post dose)
Population: FAS
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Number of Subjects Developing Neutralizing Antibodies to AAV5 | 5 participants |
| AAV5-hFIX High Dose (Cohort 2) | Number of Subjects Developing Neutralizing Antibodies to AAV5 | 5 participants |
Secondary
Number of Subjects With Antibodies to FIX
Time frame: From AMT-060 infusion through the end of study (5 years post dose)
Population: FAS
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Number of Subjects With Antibodies to FIX | 1 participants |
| AAV5-hFIX High Dose (Cohort 2) | Number of Subjects With Antibodies to FIX | 0 participants |
Secondary
Number of Subjects With a Positive AAV5 Capsid-specific T Cell Response
Specific AAV5 response (results \>17 SFC/million PBMCs) were regarded as positive.
Time frame: From AMT-060 infusion through 26 weeks post-dose
Population: FAS
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Number of Subjects With a Positive AAV5 Capsid-specific T Cell Response | 1 participants |
| AAV5-hFIX High Dose (Cohort 2) | Number of Subjects With a Positive AAV5 Capsid-specific T Cell Response | 0 participants |
Secondary
Number of Subjects With Clinically Significant Inflammatory Markers: IL-1β, IL-2, IL-6, INFγ, MCP-1
Time frame: From AMT-060 infusion through 18 weeks post dose
Population: FAS
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Number of Subjects With Clinically Significant Inflammatory Markers: IL-1β, IL-2, IL-6, INFγ, MCP-1 | 0 participants |
| AAV5-hFIX High Dose (Cohort 2) | Number of Subjects With Clinically Significant Inflammatory Markers: IL-1β, IL-2, IL-6, INFγ, MCP-1 | 0 participants |
Secondary
Number of Subjects With FIX Inhibitors
Time frame: From AMT-060 infusion through the end of study (5 years post dose)
Population: FAS
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Number of Subjects With FIX Inhibitors | 0 participants |
| AAV5-hFIX High Dose (Cohort 2) | Number of Subjects With FIX Inhibitors | 0 participants |
Secondary
Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen
Time frame: From AMT-060 infusion through end of study (5 years post dose).
Population: FAS. Subject in Cohort 1 was unable to produce semen due to a historical medical condition.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Blood | 508.8 Days | Standard Deviation 261.7 |
| AAV5-hFIX Low Dose (Cohort 1) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Nasal secretions | 83.4 Days | Standard Deviation 41.7 |
| AAV5-hFIX Low Dose (Cohort 1) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Saliva | 75.8 Days | Standard Deviation 38.4 |
| AAV5-hFIX Low Dose (Cohort 1) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Urine | 46.4 Days | Standard Deviation 20.9 |
| AAV5-hFIX Low Dose (Cohort 1) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Feces | 74.0 Days | Standard Deviation 25.7 |
| AAV5-hFIX Low Dose (Cohort 1) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Semen | 227.8 Days | Standard Deviation 147.7 |
| AAV5-hFIX High Dose (Cohort 2) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Feces | 165.0 Days | Standard Deviation 68.9 |
| AAV5-hFIX High Dose (Cohort 2) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Blood | 705.4 Days | Standard Deviation 245.1 |
| AAV5-hFIX High Dose (Cohort 2) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Urine | 82.0 Days | Standard Deviation 41.1 |
| AAV5-hFIX High Dose (Cohort 2) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Nasal secretions | 108.4 Days | Standard Deviation 66 |
| AAV5-hFIX High Dose (Cohort 2) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Semen | 157.2 Days | Standard Deviation 78.4 |
| AAV5-hFIX High Dose (Cohort 2) | Time to Vector DNA Stopped Shedding From Blood, Nasal Secretions, Saliva, Urine, Feces, and Semen | Saliva | 129.2 Days | Standard Deviation 48.9 |
Secondary
Total Annualized Bleeding Rate (ABR)
Annualized: Sum of post-treatment bleeding episodes divided by subjects' average number of years (365.25 days) from treatment start to until the data cutoff date.
Time frame: From AMT-060 infusion through end of study (5 years post-dose)
Population: FAS. One subject in Cohort 2 did not report any bleeding information for the period 1 year prior to screening. Post-tapering period = from discontinuation of FIX prophylaxis until data cutoff.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Total Annualized Bleeding Rate (ABR) | One Year Prior to Screening | 14.40 bleeds/year/subject | Standard Deviation 5.73 |
| AAV5-hFIX Low Dose (Cohort 1) | Total Annualized Bleeding Rate (ABR) | Post-tapering Period | 5.39 bleeds/year/subject | Standard Deviation 5.94 |
| AAV5-hFIX High Dose (Cohort 2) | Total Annualized Bleeding Rate (ABR) | One Year Prior to Screening | 4.00 bleeds/year/subject | Standard Deviation 3.16 |
| AAV5-hFIX High Dose (Cohort 2) | Total Annualized Bleeding Rate (ABR) | Post-tapering Period | 0.71 bleeds/year/subject | Standard Deviation 0.58 |
Secondary
Total Consumption of FIX Replacement Therapy
Time frame: From AMT-060 infusion through end of study (5 years post dose).
Population: FAS. Post-tapering period = from discontinuation of FIX prophylaxis until data cutoff.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Total Consumption of FIX Replacement Therapy | One year prior to screening | 326532 IU | Standard Deviation 234900 |
| AAV5-hFIX Low Dose (Cohort 1) | Total Consumption of FIX Replacement Therapy | Post-tapering period | 252950 IU | Standard Deviation 222790 |
| AAV5-hFIX High Dose (Cohort 2) | Total Consumption of FIX Replacement Therapy | One year prior to screening | 233778 IU | Standard Deviation 156873 |
| AAV5-hFIX High Dose (Cohort 2) | Total Consumption of FIX Replacement Therapy | Post-tapering period | 85800 IU | Standard Deviation 84482 |
Secondary
Total IgG and IgM Antibody Titers to AAV5
For subjects with a titer of 109350 and 50, the actual titer is \>109350 and \<50.
Time frame: AMT-060 infusion through end of study (5 years post dose)
Population: FAS
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 1) | 79499 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 2) | 109350 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 3) | 109350 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 4) | 109350 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 5) | 109350 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 1) | 56 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 2) | 1321 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 3) | 557 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 4) | 11568 Titer |
| AAV5-hFIX Low Dose (Cohort 1) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 5) | 809 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 3) | 6649 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 1) | 109350 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 1) | 30071 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 2) | 109350 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 5) | 50 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 3) | 109350 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 2) | 20000 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 4) | 107344 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgM (subject 4) | 50 Titer |
| AAV5-hFIX High Dose (Cohort 2) | Total IgG and IgM Antibody Titers to AAV5 | IgG (subject 5) | 109350 Titer |