Trial Outcomes & Findings for Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs) (NCT NCT01341912)

Results Overview

Patients will be monitored for inhibitors against FVIII every 3 months. Blood samples were drawn and inhibitor activity was determined by the modified Bethesda assay (Nijmegen modification) in the central lab.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

3 participants

Primary outcome timeframe

up to 3 years

Results posted on

2020-03-17

Participant Flow

This study was open to all patients who had completed the GENA-01 study with at least 50 exposure days after at least 6 months of study participation (EU patients) or at least 15 months of study participation (US patients). Overall 21 patients were eligible for this study but only 3 patients decided to participate in this extension study.

Participant milestones

Participant milestones
Measure	Human-cl rhFVIII Recombinant FVIII derived from a human cell line. Human-cl rhFVIII : Human-cl rhFVIII is administered intravenously on demand for bleeding episodes and prophylactically in case of surgery. The dosage depends on the severity of the bleeding episodes and the surgery.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Human-cl rhFVIII n=3 Participants Recombinant FVIII derived from a human cell line. Human-cl rhFVIII : Human-cl rhFVIII is administered intravenously on demand for bleeding episodes and prophylactically in case of surgery. The dosage depends on the severity of the bleeding episodes and the surgery.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=93 Participants
Age, Categorical >=65 years	1 Participants n=93 Participants
Age, Continuous	52 years STANDARD_DEVIATION 12.29 • n=93 Participants
Sex: Female, Male Female	0 Participants n=93 Participants
Sex: Female, Male Male	3 Participants n=93 Participants
Region of Enrollment Bulgaria	3 participants n=93 Participants

PRIMARY outcome

Timeframe: up to 3 years

Patients will be monitored for inhibitors against FVIII every 3 months. Blood samples were drawn and inhibitor activity was determined by the modified Bethesda assay (Nijmegen modification) in the central lab.

Outcome measures

Outcome measures
Measure	Human-cl rhFVIII n=3 Participants Recombinant FVIII derived from a human cell line. Human-cl rhFVIII : Human-cl rhFVIII is administered intravenously on demand for bleeding episodes and prophylactically in case of surgery. The dosage depends on the severity of the bleeding episodes and the surgery.
Long-term Immunogenicity	0 occurrence of inhibitors

SECONDARY outcome

Timeframe: up to 3 years

The efficacy of human-cl rhFVIII will be determined using a 4 point efficacy assessment scale. After each infusion of IMP and at the end of a BE, the following efficacy assessment is made by the subject (together with the Investigator in case of on-site treatment): Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion. Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 - 12 hours after an infusion requiring up to 2 infusions for complete resolution. Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution. None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution. The assessment was made at the end of a BE in case more than one infusion was needed.

Outcome measures

Outcome measures
Measure	Human-cl rhFVIII n=64 Bleeding episodes Recombinant FVIII derived from a human cell line. Human-cl rhFVIII : Human-cl rhFVIII is administered intravenously on demand for bleeding episodes and prophylactically in case of surgery. The dosage depends on the severity of the bleeding episodes and the surgery.
To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis Excellent	71.88 percentage of bleeding episodes
To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis Good	26.56 percentage of bleeding episodes
To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis Moderate	1.56 percentage of bleeding episodes
To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis None	0 percentage of bleeding episodes

Adverse Events

Human-cl rhFVIII

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

VP of Clinical Research and Development

Octapharma

Phone: +41 554512141

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Octapharma agreements may vary with individual investigators, but will not prohibit any investigator from publishing. Octapharma supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial. Octapharma also reserves the right to review data prior to publishing and provide comments/changes within a certain time period
Publication restrictions are in place

Restriction type: OTHER