A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)
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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: NCT03861273 |
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Recruitment Status :
Active, not recruiting
First Posted : March 4, 2019
Results First Posted : March 27, 2024
Last Update Posted : May 3, 2024
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Sponsor:
Pfizer
Information provided by (Responsible Party):
Pfizer
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| Study Type | Interventional |
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| Study Design | Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
| Condition |
Hemophilia B |
| Intervention |
Biological: PF-06838435/ fidanacogene elaparvovec |
| Enrollment | 51 |
Participant Flow
| Recruitment Details | Participants with moderately severe to severe hemophilia B, who completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead-in study C0371004 (NCT03587116), were enrolled in this study. |
| Pre-assignment Details | A total of 51 participants were screened in this study and 45 participants received a single dose of PF-06838435 (fidanacogene elaparvovec). Results presented are for primary outcome measures and only those secondary measures whose analysis was complete on a data cutoff date of 16 November 2022 (primary completion date [PCD]). |
| Arm/Group Title | PF-06838435 |
|---|---|
 Arm/Group Description |
Participants received PF-06838435 infusion on Day 1 at a dose of 5*10^11 vector genomes per kilogram (kg) of body weight. Short term follow-up: Participants were followed up for 52 weeks/Year 1 post infusion. Long term follow-up: Participants were followed up from Year 2 to 6. |
| Period Title: Screening Phase (3 Weeks) | |
| Started | 51 |
| Completed | 45 |
| Not Completed | 6 |
| Reason Not Completed | |
| Other | 1 |
| Screen failure | 5 |
| Period Title: Treatment Phase (Day 1) | |
| Started | 45 |
| Completed | 45 |
| Not Completed | 0 |
| Period Title: Follow-up Phase (Year 1) | |
| Started | 45 |
| Completed | 43 |
| Not Completed | 2 |
| Reason Not Completed | |
| Ongoing | 2 |
| Period Title: Long-Term Follow-Up (Year 2 to Year 6) | |
| Started | 43 |
| Completed | 0 |
| Not Completed | 43 |
| Reason Not Completed | |
| Ongoing | 42 |
| Withdrawal by Subject | 1 |
Baseline Characteristics
| Arm/Group Title | PF-06838435 | |
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Arm/Group Description |
All participants enrolled in the study and who received PF-06838435 infusion. | |
| Overall Number of Baseline Participants | 45 | |
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Baseline Analysis Population Description |
Safety analysis set included all participants enrolled in the study and who received PF-06838435 infusion.
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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| Number Analyzed | 45 participants | |
| 33.18 (10.947) | ||
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 45 participants | |
| Female |
0 0.0%
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| Male |
45 100.0%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 45 participants | |
| Hispanic or Latino |
2 4.4%
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| Not Hispanic or Latino |
35 77.8%
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| Unknown or Not Reported |
8 17.8%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 45 participants | |
| American Indian or Alaska Native |
0 0.0%
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| Asian |
7 15.6%
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| Native Hawaiian or Other Pacific Islander |
0 0.0%
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| Black or African American |
1 2.2%
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| White |
33 73.3%
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| More than one race |
0 0.0%
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| Unknown or Not Reported |
4 8.9%
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Outcome Measures
Adverse Events
Limitations and Caveats
C0371004 lead-in ongoing study: did not involve any investigational intervention; designed to collect bleed, infusion data while participants were on their prophylaxis FIX therapy for a minimum of 6 months; data served as within participant comparison group for C0371002; participant flow and AEs are missing as the study has not concluded yet (some participants in the C0371004 are ongoing and supporting a different study NCT04370054) and data will be provided at conclusion of the lead-in study.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Results Point of Contact
| Name/Title: | Pfizer ClinicalTrials.gov Call Center |
| Organization: | Pfizer Inc. |
| Phone: | 1-800-718-1021 |
| EMail: | ClinicalTrials.gov_Inquiries@pfizer.com |
| Responsible Party: | Pfizer |
| ClinicalTrials.gov Identifier: | NCT03861273 |
| Other Study ID Numbers: |
C0371002 2018-003086-33 ( EudraCT Number ) 2022-502844-11-00 ( Other Identifier: EU CT ) |
| First Submitted: | March 1, 2019 |
| First Posted: | March 4, 2019 |
| Results First Submitted: | November 1, 2023 |
| Results First Posted: | March 27, 2024 |
| Last Update Posted: | May 3, 2024 |