Trial Outcomes & Findings for HemLibra Prophylaxis in Patients With Hemophilic Pseudotumor (NCT NCT03921294)
NCT ID: NCT03921294
Last Updated: 2024-04-11
Results Overview
Maintenance or increase of hemoglobin (g/dl) from participants' baseline level based on serial blood tests.
TERMINATED
PHASE4
1 participants
Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
2024-04-11
Participant Flow
Participant milestones
| Measure |
Emicizumab (Single Arm)
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement. Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
Enrolled
|
1
|
|
Overall Study
Completed Treatment
|
1
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
HemLibra Prophylaxis in Patients With Hemophilic Pseudotumor
Baseline characteristics by cohort
| Measure |
Baseline Characteristics
n=1 Participants
Patients with factor VIII deficiency and a hemophilic pseudotumor.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
31 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 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
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.Maintenance or increase of hemoglobin (g/dl) from participants' baseline level based on serial blood tests.
Outcome measures
| Measure |
Single Arm
n=1 Participants
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.
Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Hemoglobin
|
9.5 gm/dL
Interval 8.0 to 11.0
|
PRIMARY outcome
Timeframe: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.Whether or not the patient requires blood transfusions (units of RBCs) due to blood loss secondary to lack of hemostatic efficacy during the duration of study treatment duration.
Outcome measures
| Measure |
Single Arm
n=1 Participants
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.
Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Participants' Need for Blood Transfusions or Lack of
|
0 Participants
|
SECONDARY outcome
Timeframe: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.Number of breakthrough bleeding events that require hemostatic therapy in addition to Hemlibra prophylaxis
Outcome measures
| Measure |
Single Arm
n=1 Participants
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.
Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
Breakthrough Bleeds
|
14 breakthrough bleeding events
|
SECONDARY outcome
Timeframe: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.Population: Patient was unable to return for repeat or final assessments of pseudotumor size by radiology scan after baseline assessment due to global COVID19 pandemic. Only baseline measurements were collected and no follow-up measurements, therefore any change in the pseudotumor status could not be evaluated/determined.
Radiologic evaluation (CT and/or MRI) to evaluate control of progression, stabilization or regression per change in size (measured in cm) of the pseudotumor
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.Population: Patient was unable to return for repeat or final QoL assessments after baseline assessment, due to the COVID19 global pandemic. Only baseline measurements were collected and no follow-up measurements, therefore any change in the QoL status could not be evaluated/determined.
The subjective change in quality of life and activity with Hemlibra (emicizumab) prophylaxis will be evaluated during the study. QoL and activity will be assessed using Haem-A-QoL and EQ-5D-5L. Changes in these measures will be determined by changes from the baseline scores compared to follow-up scores.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.Population: Patient was unable to return for repeat or final QoL assessments after baseline assessment, due to the COVID19 global pandemic. Only baseline measurements were collected and no follow-up measurements, therefore any change in the QoL status could not be evaluated/determined.
The subjective change in quality of life and activity with Hemlibra (emicizumab) prophylaxis will be evaluated during the study. QoL and activity will be assessed using Haem-A-QoL and EQ-5D-5L. Changes in these measures will be determined by changes from the baseline scores compared to follow-up scores.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.Population: AEs were collected during participant's study participation until study treatment / participation withdrawal.
Number of adverse events while on HemLibra (emicizumab) prophylaxis.
Outcome measures
| Measure |
Single Arm
n=1 Participants
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.
Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
Adverse Events
|
3 adverse events
|
SECONDARY outcome
Timeframe: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.Number of SAEs while on HemLibra (emicizumab) prophylaxis
Outcome measures
| Measure |
Single Arm
n=1 Participants
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.
Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
Serious Adverse Events
|
0 serious adverse events
|
SECONDARY outcome
Timeframe: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.Number of participants with adverse events while on HemLibra (emicizumab) prophylaxis
Outcome measures
| Measure |
Single Arm
n=1 Participants
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.
Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
Number of Participants With Adverse Events
|
1 participants
|
SECONDARY outcome
Timeframe: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.Development of emicizumab anti-drug antibodies using the ADA assay
Outcome measures
| Measure |
Single Arm
n=1 Participants
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.
Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
Anti-Drug Antibodies (ADA)
|
0 participants
|
SECONDARY outcome
Timeframe: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.Population: Patient did not develop ADA (anti-drug antibody) during study participation.
If the patient develops an ADA: the ADA's effect on the patient's APTT
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.Population: Patient did not develop ADA (anti-drug antibody) during study participation.
If the patient develops an ADA: the ADA's effecton the patient's FVIII assay
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.Population: Patient did not require surgery during study treatment.
If the patient requires surgery: Whether the procedure(s) was/were planned versus unplanned
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.Population: Patient did not require surgery during study participation
If the patient requires surgery: Whether hemostatic agents in addition to Hemlibra were required to achieve or maintain hemostasis
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.Population: Patient did not have surgery during study participation.
If the patient requires surgery: Whether blood loss exceeded the estimated/predicted blood loss relative to a patient without hemophilia
Outcome measures
Outcome data not reported
Adverse Events
Single Arm
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Single Arm
n=1 participants at risk
Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.
Emicizumab: bispecific monoclonal antibody binding to activated Factor IX and Factor X
|
|---|---|
|
General disorders
Upper respiratory infection
|
100.0%
1/1 • Number of events 1 • Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Standard definitions
|
|
Musculoskeletal and connective tissue disorders
Fall with injurty
|
100.0%
1/1 • Number of events 1 • Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Standard definitions
|
|
Infections and infestations
Malaria infection
|
100.0%
1/1 • Number of events 1 • Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Standard definitions
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Sponsor wishes to review final study report prior to submission for publication.
- Publication restrictions are in place
Restriction type: OTHER