Trial Outcomes & Findings for A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects (NCT NCT04394286)
NCT ID: NCT04394286
Last Updated: 2022-05-19
Results Overview
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this investigational product (IP) or medicinal product. A Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly.
TERMINATED
PHASE1/PHASE2
2 participants
From study start date to Month 12
2022-05-19
Participant Flow
The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
Participant milestones
| Measure |
Cohort 1: SHP648
Cohort 1 participants were planned to receive a single intravenous (IV) infusion of SHP648 at dose 4.0\*10\^11 vector genome (vg) per kilogram (kg) body weight (BW) on the day of dosing (Day 0).
|
Cohort 2: SHP648
Cohort 2 participants were planned to receive a single IV infusion of SHP648 at dose 8.0\*10\^11 vg/kg BW or 1.2\*10\^12 vg/kg BW on the day of dosing (Day 0). The dosing of Cohort 2 was to be decided upon evaluation of Cohort 1 data and recommendation from an external Data Monitoring Committee.
|
Cohort 3: SHP648
Cohort 3 participants were planned to receive a single IV infusion of SHP648 at dose 1.6\*10\^12 vg/kg BW or 2.4\*10\^12 vg/kg BW or 3.6\*10\^12 vg/kg BW on the day of dosing (Day 0). The dosing of Cohort 3 was to be decided upon evaluation of Cohort 2 data and recommendation from an external Data Monitoring Committee.
|
|---|---|---|---|
|
Overall Study
STARTED
|
0
|
0
|
0
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects
Baseline characteristics by cohort
Baseline data not reported
PRIMARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this investigational product (IP) or medicinal product. A Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality
Plasma FIX levels before and after SHP648 infusion were planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
ABR was to be assessed based upon each individual bleeding episode. A bleed episode was defined as subjective (example, pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FIX. ABR before and after SHP648 administration was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
Number of participants with positive binding antibodies titers to AAV8 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
Number of participants with positive neutralizing antibodies to AAV8 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
Number of participants with T-cell response to AAV8 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
Number of participants with T-cell response to FIX transgene products was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
Duration of SHP648 genomes present in bodily fluids such as serum, blood, saliva, urine, stool, and semen was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From study start date to Month 12Population: The study was terminated by the Sponsor. Only 2 participants were screened and signed informed consent but none received any treatment during this study. No participants were evaluated, and no data was collected to be reported here, in order to protect and maintain participant's privacy/confidentiality.
Percent change in consumption of exogenous FIX before and after gene transfer was planned to be reported.
Outcome measures
Outcome data not reported
Adverse Events
Cohort 1: SHP648
Cohort 2: SHP648
Cohort 3: SHP648
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER