An observational study in Hemophilia A, sponsored by Octapharma. Completed at 39 sites in 13 countries. Open to male participants. Per ClinicalTrials.gov, last updated 2021-10-21.
Sponsored by Octapharma · Observational
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice. Besides aspects such as general product safety and efficacy, there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 80 is close to the median of 80 across 314 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →Octapharma is the lead sponsor of 69 studies on the registry; 8 are open to participants now.
Of its 16 completed or terminated interventional studies of FDA-regulated products, 14 (88%) have results posted.
Counted across the registry records on this site, refreshed daily.
The goal is to collect data on 200 previously treated male patients of any age with haemophilia (FVIII:C ≤ 2%). Patients from pre-authorisation studies can be followed up to at least 100 EDs. Newly enrolled patients have to be treated and followed for at least 100 EDs.
Exclusion Criteria:
Number of Patients With FVIII Inhibitors
FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Time frame: Screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Number of Patients With Adverse Drug Reactions
Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded by patients in treatment diaries which will be reviewed at each Follow-up Visit.
Time frame: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment
Total number of bleeding episodes under prophylaxis treatment divided by the duration of prophylactic phase (in years)
Time frame: Monitored throughout the study from screening through to study completion (minimum 3.7 months; maximum 21.2 months)
Assessment of the Efficacy of On-demand Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
At the end of a BE, treatment efficacy was to be assessed either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4-point scale including the four items 'excellent,' 'good,' moderate,' and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
Time frame: Monitored throughout the study from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians
At the end of the postoperative period, an overall assessment of the efficacy of treatment in the pre-, peri-, and postoperative periods using the 'excellent,' 'good,' moderate,' and 'none' scale will be done jointly by the surgeon and the hematologist. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
Time frame: From start of surgery until end of post-operative period
Assessment of the Efficacy of Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
At the end of a BE, treatment efficacy was to be assessed either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4-point scale including the four items 'excellent', 'good', 'moderate', and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
Time frame: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
| Milestone | Nuwiq® (Human-cl rhFVIII) |
|---|---|
| Started | 78 |
| Fas population | 78 |
| Saf population | 78 |
| Prophylactic treatment group | 77 |
| On demand treatment group | 2 |
| Surg population | 4 |
| Completed | 61 |
| Not completed | 17 |
| Withdrew: Protocol violation | 9 |
| Withdrew: Withdrawal by subject | 3 |
| Withdrew: Lost to follow-up | 1 |
| Withdrew: Death | 1 |
| Withdrew: Limited access to study medication | 3 |
FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
| Participants | FVIII Inhibitors Detected at Screening in Patients Treated With Nuwiq® | FVIII Inhibitors Detected Between Screening and Completion in Patients Treated With Nuwiq® | FVIII Inhibitors Detected at Completion in Patients Treated With Nuwiq® (Human-cl rhFVIII) |
|---|---|---|---|
| Number of Patients With FVIII Inhibitors | 0 | 0 | 0 |
Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded by patients in treatment diaries which will be reviewed at each Follow-up Visit.
| Participants | Nuwiq® (Human-cl rhFVIII) FAS Population |
|---|---|
| Number of Patients With Adverse Drug Reactions | 0 |
Total number of bleeding episodes under prophylaxis treatment divided by the duration of prophylactic phase (in years)
| Number of bleeding episodes per year | Nuwiq® in Prophylactic Treatment |
|---|---|
| All bleeding events | 2.39 (0 to 4.87) |
| Spontaneous bleeding events | 0.00 (0 to 2.47) |
| Traumatic bleeding events | 0.00 (0 to 1.93) |
At the end of a BE, treatment efficacy was to be assessed either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4-point scale including the four items 'excellent,' 'good,' moderate,' and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
| Number of Bleeding episodes | Nuwiq® (Human-cl rhFVIII) in On Demand Population |
|---|---|
| Excellent | 50 |
| Good | 4 |
| Moderate | 1 |
| None | 0 |
At the end of the postoperative period, an overall assessment of the efficacy of treatment in the pre-, peri-, and postoperative periods using the 'excellent,' 'good,' moderate,' and 'none' scale will be done jointly by the surgeon and the hematologist. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
| Surgeries | Nuwiq® (Human-cl rhFVIII) Surgical Prophylaxis |
|---|---|
| Excellent | 5 |
| Good | 0 |
| Moderate | 0 |
| None | 0 |
At the end of a BE, treatment efficacy was to be assessed either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4-point scale including the four items 'excellent', 'good', 'moderate', and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
| Bleeding episodes | Nuwiq in Prophylactic Population |
|---|---|
| Excellent | 167 |
| Good | 50 |
| Moderate | 26 |
| None | 3 |
Collected over Adverse drug reactions were monitored throughout the study from first treatment through to study completion (minimum 1.7 months; maximum 31.6 months). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Nuwiq® (Human-cl rhFVIII) SAF Population | 1/78 (1.3%) | 0/78 (0%) | 0/78 (0%) |
| Age, Customized(Participants) | Nuwiq® (Human-cl rhFVIII) SAF Population |
|---|---|
| Age — <12 yrs | 40 |
| Age — 12-<18 yrs | 12 |
| Age — >18 yrs | 26 |
| Sex: Female, Male(Participants) | Nuwiq® (Human-cl rhFVIII) SAF Population |
|---|---|
| Female | 0 |
| Male | 78 |
| Ethnicity (NIH/OMB)(Participants) | Nuwiq® (Human-cl rhFVIII) SAF Population |
|---|---|
| Hispanic or Latino | 25 |
| Not Hispanic or Latino | 53 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | Nuwiq® (Human-cl rhFVIII) SAF Population |
|---|---|
| Patient race — American Indian or Alaska Native | 7 |
| Patient race — Asian | 0 |
| Patient race — Native Hawaiian or Other Pacific Islander | 0 |
| Patient race — Black or African American | 1 |
| Patient race — White | 61 |
| Patient race — More than one race | 0 |
| Patient race — Other | 9 |
| Height(centimeters) | Nuwiq® (Human-cl rhFVIII) SAF Population |
|---|---|
| <12 yrs | 112 ± 17.59 |
| 12-<18 yrs | 168.8 ± 9.73 |
| >18 yrs | 172.9 ± 7.88 |
| Weight(kilograms) | Nuwiq® (Human-cl rhFVIII) SAF Population |
|---|---|
| <12 yrs | 21.7 ± 8.30 |
| 12-<18 yrs | 67.1 ± 15.55 |
| >18 yrs | 76.6 ± 17.21 |
| BMI(kg/m^2) | Nuwiq® (Human-cl rhFVIII) SAF Population |
|---|---|
| <12 yrs | 17.0 ± 3.34 |
| 12-<18 yrs | 23.6 ± 5.46 |
| >18 yrs | 25.5 ± 4.65 |
| Severity of Haemophilia A(Participants) | Nuwiq® (Human-cl rhFVIII) SAF Population |
|---|---|
| Moderate | 10 |
| Severe | 68 |
3 further baseline measures are reported on the registry.
Documents are hosted by the registry — open the source record to download them.
This study is completed, as verified in Sep 2021. You cannot join it, but the record below documents what was studied.
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