Trial Outcomes & Findings for Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults (NCT NCT04370054)

Results Overview

Bleeds for evaluation included both treated and untreated bleeds. Treated bleed: bleeding event necessitated administration of coagulation factor within 72 hours of signs or symptoms of bleeding. Untreated bleed: bleeding event did not necessitate administration of coagulation factor within 72 hours of signs and symptoms of bleeding. In this outcome measure total ABR following infusion of PF-07055480 in current study (post-infusion) and on prior FVIII prophylaxis regimen prior to infusion of PF-07055480 (pre-infusion, data collected from lead-in study C0371004) were reported. Total ABR for FVIII prophylaxis= \[(Number of total bleeding episodes during pre-infusion period)\*365.25\]/ \[(Number of days of follow-up in pre-infusion period)\]. Total ABR for PF-07055480: \[(Number of total bleeding episodes during post-infusion period)\*365.25\] / \[(last date of post-infusion period - date of PF-07055480 infusion) - 77 days + 1\].

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

77 participants

Primary outcome timeframe

FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Results posted on

2025-08-17

Participant Flow

Participants with moderately severe to severe hemophilia A, who completed routine Factor VIII (FVIII) prophylaxis follow-up during the lead-in study C0371004 (NCT03587116), were enrolled in this study. Participants in this study received single infusion of PF-07055480/giroctocogene fitelparovec.

Results are presented for primary outcome measures and only those secondary outcome measures whose analysis was complete on a data cutoff date of 17 June 2024 (primary completion date \[PCD\]). Remaining outcome measures would be reported upon completion of their analyses at study completion date.

Participant milestones

Participant milestones
Measure	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vector genomes per kilogram (vg/kg) intravenously on Day 1.
Treatment Phase STARTED	77
Treatment Phase Treated	75
Treatment Phase COMPLETED	75
Treatment Phase NOT COMPLETED	2
Follow-Up Phase STARTED	75
Follow-Up Phase COMPLETED	0
Follow-Up Phase NOT COMPLETED	75

Reasons for withdrawal

Reasons for withdrawal
Measure	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vector genomes per kilogram (vg/kg) intravenously on Day 1.
Treatment Phase Lost to Follow-up	1
Treatment Phase Withdrawal by Subject	1
Follow-Up Phase Ongoing	75

Baseline Characteristics

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	PF-07055480 n=75 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Age, Continuous	32.27 Years STANDARD_DEVIATION 10.441 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	75 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	59 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	13 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	14 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants
Race (NIH/OMB) White	56 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

Bleeds for evaluation included both treated and untreated bleeds. Treated bleed: bleeding event necessitated administration of coagulation factor within 72 hours of signs or symptoms of bleeding. Untreated bleed: bleeding event did not necessitate administration of coagulation factor within 72 hours of signs and symptoms of bleeding. In this outcome measure total ABR following infusion of PF-07055480 in current study (post-infusion) and on prior FVIII prophylaxis regimen prior to infusion of PF-07055480 (pre-infusion, data collected from lead-in study C0371004) were reported. Total ABR for FVIII prophylaxis= \[(Number of total bleeding episodes during pre-infusion period)\*365.25\]/ \[(Number of days of follow-up in pre-infusion period)\]. Total ABR for PF-07055480: \[(Number of total bleeding episodes during post-infusion period)\*365.25\] / \[(last date of post-infusion period - date of PF-07055480 infusion) - 77 days + 1\].

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Total Annualized Bleeding Rate (ABR)	4.73 Total bleeds per year Interval 2.86 to 6.6	1.24 Total bleeds per year Interval -0.66 to 3.14

SECONDARY outcome

Timeframe: At 15 months following infusion of PF-07055480

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting.

FVIII activity level based on central laboratory chromogenic assay at Month 15

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Percentage of Participants With Coagulation FVIII Activity Levels Greater Than (>)5 Percent (%) at 15 Months	84.00 Percentage of participants Interval 70.89 to 92.83	—

SECONDARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

Treated bleed was defined as an event which necessitated administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure treated ABR following infusion of PF-07055480 in current study (post-infusion) and on prior FVIII prophylaxis regimen prior to infusion of PF-07055480 (pre-infusion, data collected from lead-in study C0371004) were reported. Treated ABR for FVIII prophylaxis= \[(Number of treated bleeding episodes during pre-infusion period)\*365.25\]/ \[(Number of days of follow-up in pre-infusion period)\]. Treated ABR for PF-07055480: \[(Number of treated bleeding episodes during post-infusion period)\*365.25\] / \[(last date of post-infusion period - date of PF-07055480 infusion) - 77 days + 1\].

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Treated ABR	4.08 Treated bleeds per year Interval 2.52 to 5.64	0.07 Treated bleeds per year Interval 0.01 to 0.13

SECONDARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

AIR: \[(Number of exogenous FVIII product infusions for any purpose during the given time period) \* 365.25\]/ (Number of days of follow-up in the given time period). In this outcome measure AIR of exogenous FVIII activity in current study (post-infusion) and on prior FVIII prophylaxis regimen prior to infusion of PF-07055480 (pre-infusion, data collected from lead-in study C0371004) were reported.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Annualized Infusion Rate (AIR) of Exogenous FVIII Activity	124.39 Exogenous infusions per year Standard Deviation 53.882	0.21 Exogenous infusions per year Standard Deviation 0.601

SECONDARY outcome

Timeframe: Week 12 through Month 15 following PF-07055480 Infusion

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting.

FVIII activity levels were assessed using chromogenic assay and one-stage clotting assay analyzed in the central laboratory. As pre-specified, for each participant, a geometric mean laboratory reading for FVIII activity was used to derived one single FVIII level by including all assessments from Week 12 through 15 months following PF-07055480 infusion; then mean and standard deviation as summary statistics was calculated across all evaluable participants and reported as data for this outcome measure. This resulting Factor VIII activity is expressed as percent of normal (%); which is used interchangeable with international unit per deciliter (IU/dL). "Normal Range" is typically considered 50-150% (or 50-150 IU/dL).

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
FVIII Activity Levels From Week 12 Through 15 Months Following PF-07055480 Infusion Chromogenic Assay	96.84 Percentage of Normal Standard Deviation 90.521	—
FVIII Activity Levels From Week 12 Through 15 Months Following PF-07055480 Infusion One-Stage Assay	134.95 Percentage of Normal Standard Deviation 116.161	—

SECONDARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

Annualized FVIII consumption was reported in international units per kilogram (IU/kg) and total units (in IU). This outcome measure compared data collected from lead-in study C0371004 and from current study C3731003. Data is reported in this outcome measure as mean of annualized FVIII consumption from all participants in this analysis population.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Annualized FVIII Consumption	4082.7 IU/kg Standard Deviation 2291.90	6.6 IU/kg Standard Deviation 21.41

SECONDARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

Bleeds based on cause:spontaneous(bleeding for no apparent/known reason particularly into the joint,muscles and soft tissue)and traumatic(bleeding event occurring for an apparent/known reason).Bleeds related to a procedure/surgery such as hematomas/bruising which resulted from any surgeries or invasive procedure,or invasive diagnostic procedures were not counted as bleeds.Treated:necessitated administration of coagulation factor within 72hrs of sign or symptom of bleeding. Untreated:did not necessitate administration of coagulation factor within 72hrs of sign and symptom of bleeding.ABR for FVIII prophylaxis=\[(Number of total bleeding episodes during pre-infusion)\*365.25\]/(Number of days of follow-up in pre-infusion period).ABR for PF-07055480\[(Number of total bleeding episodes during post-infusion period)\*365.25\]/\[(last date of post-infusion period-date of PF-07055480 infusion)-77 days+1\].This outcome measure compared data collected from lead-inC0371004and from current studyC3731003.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Total (Treated and Untreated) ABR Based on Cause Spontaneous	3.27 Bleeds per year Standard Deviation 5.591	1.07 Bleeds per year Standard Deviation 6.540
Total (Treated and Untreated) ABR Based on Cause Traumatic	1.46 Bleeds per year Standard Deviation 3.416	0.14 Bleeds per year Standard Deviation 0.320

SECONDARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

Bleeds based on cause were spontaneous (bleeding for no apparent/known reason particularly into the joint, muscles and soft tissues) and traumatic (bleeding event occurring for an apparent/known reason). Bleeds related to a procedure/surgery such as hematomas/bruising which resulted from any surgeries or invasive procedures, or invasive diagnostic procedure were not counted as bleeds. Treated: necessitated administration of coagulation factor within 72 hours of signs or symptoms of bleeding. Treated ABR for FVIII prophylaxis= \[(Number of treated bleeding episodes during pre-infusion period)\*365.25\]/ (Number of days of follow-up in pre-infusion period). Treated ABR for PF-07055480: \[(Number of treated bleeding episodes during post-infusion period)\*365.25\] /\[(last date of post-infusion period - date of PF-07055480 infusion) - 77 days + 1\]. This outcome measure compared data collected from lead-in study C0371004 and from current study C3731003.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Treated ABR Based on Cause Spontaneous	2.81 Bleeds per year Standard Deviation 4.469	0.02 Bleeds per year Standard Deviation 0.119
Treated ABR Based on Cause Traumatic	1.27 Bleeds per year Standard Deviation 3.270	0.07 Bleeds per year Standard Deviation 0.213

SECONDARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

Target joint:major joint(hip,elbow,wrist,shoulder,knee\&ankle)with repeated bleed(3 or more spontaneous bleed into a single joint within6month).Joint bleed:bleeding episode by rapid loss of motion as compared with baseline,associated with pain or unusual sensation,swelling\&warmth over the joint.Muscle:bleeding episode into a muscle,determined by imaging study,associated with pain\&or swelling \&functional impairment.Treated:necessitated administration of coagulation factor within72hrs of sign or symptom of bleeding.Untreated:did not necessitate administration of coagulation factor within72hrs of sign\&symptom of bleeding.FVIII prophylaxis=\[(Number of total bleeding episodes during pre-infusion)\*365.25\]/(Number of days of follow-up in pre-infusion).PF-07055480:\[(Number of total bleeding episodes during post-infusion)\*365.25\]/\[(last date of post infusion-date of PF-07055480 infusion)-77days+1\].This outcome measure compared data collected from lead-inC0371004 and from current studyC3731003.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Total (Treated and Untreated) ABR Based on Location All Joints	3.28 Bleeds per year Standard Deviation 5.358	0.51 Bleeds per year Standard Deviation 2.819
Total (Treated and Untreated) ABR Based on Location Target Joints	1.13 Bleeds per year Standard Deviation 4.026	0.02 Bleeds per year Standard Deviation 0.130
Total (Treated and Untreated) ABR Based on Location Soft Tissue/Muscle/Other	1.55 Bleeds per year Standard Deviation 3.346	0.76 Bleeds per year Standard Deviation 4.303

SECONDARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

Target joint:major joint(hip,elbow,wrist,shoulder,knee,and ankle)with repeated bleeds(3 or more spontaneous bleeds into a single joint within 6month).Joint bleed:bleeding episode characterized by rapid loss of range of motion as compared with baseline,associated with pain or an unusual sensation in the joint,palpable swelling,and warmth of the skin over the joint.Muscle:bleeding episode into a muscle,determined clinically and/or by imaging study,associated with pain and/or swelling and functional impairment.Treated:necessitated administration of coagulation factor within72 hrs of sign or symptom of bleeding.FVIII prophylaxis=\[(Number of treated bleeding episodes during pre-infusion)\*365.25\]/(Number of days of follow-up in pre-infusion).PF-07055480:\[(Number of treated bleeding episodes during post-infusion)\*365.25\]/\[(last date of post-infusion-date of PF-07055480 infusion)-77days+1\].This outcome measure compared data collected from lead-in studyC0371004 and from current studyC3731003.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Treated ABR Based on Location All Joints	2.81 Bleeds per year Standard Deviation 4.449	0.06 Bleeds per year Standard Deviation 0.205
Treated ABR Based on Location Target Joints	0.88 Bleeds per year Standard Deviation 2.905	0.00 Bleeds per year Standard Deviation 0.000
Treated ABR Based on Location Soft Tissue/Muscle/Other	1.34 Bleeds per year Standard Deviation 2.985	0.03 Bleeds per year Standard Deviation 0.136

SECONDARY outcome

Timeframe: FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)

Population: Efficacy analysis population included all participants in the 'Dosed' population \[enrolled and received the study intervention\] who completed at least 6 months of follow up in the lead-in study and at least 15 months of follow-up or discontinued from the study C3731003 prior to the data cutoff for reporting. Data within the same participants were compared between Pre and Post infusion (2 groups described below), with different specified periods/durations.

Bleeds for evaluation included both treated and untreated bleeds. Treated bleed: necessitated administration of coagulation factor within 72 hours of signs or symptoms of bleeding. Untreated bleed: did not necessitate administration of coagulation factor within 72 hours of signs and symptoms of bleeding. In this outcome measure percentage of participants without bleeds following infusion of PF-07055480 in current study (post-infusion) and on prior FVIII prophylaxis regimen prior to infusion of PF-07055480 (pre-infusion) were reported. This outcome measure compared data collected from lead-in study C0371004 and from current study C3731003.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=50 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 n=50 Participants Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Percentage of Participants Without Bleeds Without treated bleeds	30 Percentage of participants	88 Percentage of participants
Percentage of Participants Without Bleeds Without treated and untreated bleeds	26 Percentage of participants	64 Percentage of participants

SECONDARY outcome

Timeframe: At Weeks 24, 52, 65 and 104

Population: Dosed analysis population included all participants enrolled in the study and who received the study intervention. Here "Number Analyzed" at each row represents the number of participants with valid FVIII assessments at the respective time points.

FVIII activity levels were assessed using chromogenic and one-stage clotting assay and which were analyzed in the central laboratory.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=75 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
FVIII Activity Level at Weeks 24, 52, 65, and 104 Week 24: Chromogenic Assay	101.59 Percentage of Normal Interval 78.68 to 124.51	—
FVIII Activity Level at Weeks 24, 52, 65, and 104 Week 24: One-Stage Assay	147.14 Percentage of Normal Interval 116.91 to 177.37	—
FVIII Activity Level at Weeks 24, 52, 65, and 104 Week 52: Chromogenic Assay	51.68 Percentage of Normal Interval 37.67 to 65.69	—
FVIII Activity Level at Weeks 24, 52, 65, and 104 Week 52: One-Stage Assay	80.14 Percentage of Normal Interval 60.53 to 99.76	—
FVIII Activity Level at Weeks 24, 52, 65, and 104 Week 65: Chromogenic Assay	51.08 Percentage of Normal Interval 35.51 to 66.65	—
FVIII Activity Level at Weeks 24, 52, 65, and 104 Week 65: One-Stage Assay	73.99 Percentage of Normal Interval 54.34 to 93.65	—
FVIII Activity Level at Weeks 24, 52, 65, and 104 Week 104: Chromogenic Assay	51.90 Percentage of Normal Interval 31.2 to 72.61	—
FVIII Activity Level at Weeks 24, 52, 65, and 104 Week 104: One-Stage Assay	72.91 Percentage of Normal Interval 47.22 to 98.61	—

SECONDARY outcome

Timeframe: Baseline (before infusion on Day 1); Weeks 24 and 52

Population: Dosed analysis population included all participants enrolled in the study and who received the study intervention. Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies number of participants evaluable for at specific timepoints.

A qualified healthcare professional assessed six joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) scored from 0 to 20 based on: swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain, and strength. Gait was scored (0 to 4) based on walking, stairs, running, hopping on one leg. Total HJHS score = sum of scores from all six joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) + gait score, ranged from 0 to 124, with the higher score indicated the number equating to more severe joint damage.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=69 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Change From Baseline in Joint Health as Measured by Hemophilia Joint Health Score (HJHS) Instrument at Weeks 24 and 52 Change at Week 24	-1.4 Units on a scale Interval -2.76 to -0.11	—
Change From Baseline in Joint Health as Measured by Hemophilia Joint Health Score (HJHS) Instrument at Weeks 24 and 52 Change at Week 52	-2.4 Units on a scale Interval -3.8 to -0.96	—

SECONDARY outcome

Timeframe: Baseline (before infusion on Day 1); Weeks 12, 24 and 52

Population: Dosed analysis population included all participants enrolled in the study and who received the study intervention. All participants reported under "Number of Participants Analyzed" contributed data to the table; however, may not have evaluable data for every row. Here, "Number Analyzed" signifies number of participants evaluable for specified timepoints.

Haem-A-QoL assessed health-related quality of life in adult participants with hemophilia. It contains 46 items with 10 domains that assesses health in the following areas: physical health; feelings; view of self; sports and leisure; work and school; dealing with Haemophilia; treatment; future; family planning; and partnership and sexuality. All items are based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring is performed by averaging non-missing item responses for each domain, then rescaled to be on 0 to 100, with lower scores representing higher quality of life. Total score= was averaged across the 46 items values and then rescaled on 0 to 100, with lower scores representing better quality of life and physical health status. Change from baseline in physical Health domain score was reported in this outcome measure.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=75 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Change From Baseline in Haemophilia Quality of Life Questionnaire (Haem-A-QoL) for Adults Physical Health Domain Score at Weeks 12, 24 and 52 Change at Week 12	-13.56 Units on a scale Interval -18.52 to -8.6	—
Change From Baseline in Haemophilia Quality of Life Questionnaire (Haem-A-QoL) for Adults Physical Health Domain Score at Weeks 12, 24 and 52 Change at Week 24	-14.10 Units on a scale Interval -19.6 to -8.6	—
Change From Baseline in Haemophilia Quality of Life Questionnaire (Haem-A-QoL) for Adults Physical Health Domain Score at Weeks 12, 24 and 52 Change at Week 52	-9.63 Units on a scale Interval -15.38 to -3.88	—

SECONDARY outcome

Timeframe: Baseline (before infusion on Day 1); Weeks 12, 24 and 52

Population: Dosed analysis population included all participants enrolled in the study and who receive the study intervention. All participants reported under "Number of Participants Analyzed" contributed data to the table; however, may not have evaluable data for every row. Here, "Number Analyzed" signifies number of participants evaluable for specific timepoints.

The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults. The 7 domains of this instrument contained 42 items in total, as follows:lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was calculated according to the Van Genderen scoring algorithm, had a transformed score range from 0 to 100; higher scores indicate better quality of life, that is, less functional limitations in performing tasks. Change from baseline in complex lower extremity activities component score was reported in this outcome measure.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=75 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Weeks 12, 24 and 52 Change at Week 12	3.1 Units on a scale Interval -1.5 to 7.6	—
Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Weeks 12, 24 and 52 Change at Week 24	3.0 Units on a scale Interval -1.5 to 7.5	—
Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Weeks 12, 24 and 52 Change at Week 52	2.5 Units on a scale Interval -2.9 to 8.0	—

SECONDARY outcome

Timeframe: From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months)

Population: Safety population included all participants enrolled in the study who received the study intervention.

An AEs was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. Severe AEs: An event that prevented normal everyday activities. Severe was a category utilized for rating the intensity of an event, and both AEs and serious adverse events (SAEs) could be assessed as severe.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=75 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Number of Participants With Adverse Events (AEs) and Severe AEs AEs	74 Participants	—
Number of Participants With Adverse Events (AEs) and Severe AEs Severe AEs	7 Participants	—

SECONDARY outcome

Timeframe: From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months)

Population: Safety population included all participants enrolled in the study who received the study intervention.

An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AESI included all medically important events that were reported as a serious adverse events (SAE), and any clinical reported thrombotic event or hypersensitivity/infusion-related reactions events assessed as non-serious AEs.

Outcome measures

Outcome measures
Measure	FVIII Prophylaxis n=75 Participants Participants who received FVIII prophylaxis replacement therapy during the lead-in study C0371004 and were enrolled to receive PF-07055480 infusion in current study C3731003. In this arm, data from participants for pre-infusion (of PF-07055480) from the lead-in study C0371004 period was included.	PF-07055480 Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Number of Participants With AEs of Special Interest	70 Participants	—

Adverse Events

PF-07055480

Serious events: 15 serious events

Other events: 70 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	PF-07055480 n=75 participants at risk Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Eye disorders Ocular hyperaemia	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Gastrointestinal disorders Pancreatitis acute	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Chills	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Facial pain	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Pyrexia	6.7% 5/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Swelling face	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Immune system disorders Immune-mediated adverse reaction	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Infections and infestations COVID-19	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Infections and infestations Gastroenteritis	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Infections and infestations Myelitis	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Infections and infestations Subcutaneous abscess	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Injury, poisoning and procedural complications Infusion related reaction	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Injury, poisoning and procedural complications Muscle rupture	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Anti factor VIII antibody positive	2.7% 2/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Transaminases increased	2.7% 2/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Metabolism and nutrition disorders Dehydration	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Musculoskeletal and connective tissue disorders Soft tissue mass	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Ductal adenocarcinoma of pancreas	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Skin and subcutaneous tissue disorders Erythema	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Vascular disorders Deep vein thrombosis	1.3% 1/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.

Other adverse events

Other adverse events
Measure	PF-07055480 n=75 participants at risk Participants received a single infusion of PF-07055480 3.0\*10\^13 vg/kg IV on Day 1.
Blood and lymphatic system disorders Iron deficiency anaemia	5.3% 4/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Cardiac disorders Tachycardia	8.0% 6/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Gastrointestinal disorders Abdominal pain upper	6.7% 5/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Gastrointestinal disorders Diarrhoea	13.3% 10/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Gastrointestinal disorders Dyspepsia	6.7% 5/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Gastrointestinal disorders Nausea	30.7% 23/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Gastrointestinal disorders Vomiting	10.7% 8/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Asthenia	6.7% 5/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Chills	21.3% 16/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Fatigue	14.7% 11/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Pain	5.3% 4/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
General disorders Pyrexia	60.0% 45/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Infections and infestations COVID-19	12.0% 9/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Infections and infestations Influenza	10.7% 8/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Infections and infestations Nasopharyngitis	8.0% 6/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Infections and infestations Upper respiratory tract infection	14.7% 11/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Alanine aminotransferase increased	49.3% 37/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Aspartate aminotransferase increased	17.3% 13/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Coagulation factor VIII level decreased	10.7% 8/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Coagulation factor VIII level increased	9.3% 7/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Factor VIII activity decreased	9.3% 7/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Factor VIII activity increased	24.0% 18/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Investigations Transaminases increased	5.3% 4/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Metabolism and nutrition disorders Iron deficiency	5.3% 4/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Musculoskeletal and connective tissue disorders Arthralgia	21.3% 16/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Musculoskeletal and connective tissue disorders Back pain	6.7% 5/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Musculoskeletal and connective tissue disorders Myalgia	8.0% 6/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Nervous system disorders Dizziness	8.0% 6/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Nervous system disorders Headache	50.7% 38/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Nervous system disorders Hypoaesthesia	5.3% 4/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Psychiatric disorders Insomnia	8.0% 6/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	6.7% 5/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Skin and subcutaneous tissue disorders Acne	6.7% 5/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Skin and subcutaneous tissue disorders Pruritus	8.0% 6/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Skin and subcutaneous tissue disorders Rash	5.3% 4/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.
Vascular disorders Hypertension	10.7% 8/75 • From Day 1 up to 104 weeks after last dose of PF-07055480 (maximum for 44.4 months) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both. SAS included all participants enrolled in the study and who received the study intervention.

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer Inc.

Phone: 8007181021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication 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.
Publication restrictions are in place

Restriction type: OTHER