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Trial record 6 of 158 for:    bleeding episodes | ( Map: United States )

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

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ClinicalTrials.gov Identifier: NCT01341912
Recruitment Status : Completed
First Posted : April 26, 2011
Results First Posted : February 3, 2014
Last Update Posted : February 3, 2014
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Severe Hemophilia A
Intervention Biological: Human-cl rhFVIII
Enrollment 3
Recruitment Details 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.
Pre-assignment Details  
Arm/Group Title Human-cl rhFVIII
Hide Arm/Group Description

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.

Period Title: Overall Study
Started 3
Completed 3
Not Completed 0
Arm/Group Title Human-cl rhFVIII
Hide Arm/Group Description

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 Number of Baseline Participants 3
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
<=18 years
0
   0.0%
Between 18 and 65 years
2
  66.7%
>=65 years
1
  33.3%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants
52  (12.29)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
Female
0
   0.0%
Male
3
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 3 participants
United States 0
Bulgaria 3
1.Primary Outcome
Title Long-term Immunogenicity
Hide Description 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.
Time Frame up to 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Human-cl rhFVIII
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: occurrence of inhibitors
0
2.Secondary Outcome
Title To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis
Hide Description

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.

Time Frame up to 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Human-cl rhFVIII
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding episodes
64
Measure Type: Number
Unit of Measure: percentage of bleeding episodes
Excellent 71.88
Good 26.56
Moderate 1.56
None 0
Time Frame Patient 1 = 91 days Patient 2 = 57 days Patient 3 = 93 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Human-cl rhFVIII
Hide Arm/Group Description

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.

All-Cause Mortality
Human-cl rhFVIII
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Human-cl rhFVIII
Affected / at Risk (%)
Total   0/3 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Human-cl rhFVIII
Affected / at Risk (%)
Total   0/3 (0.00%) 
Only 3 patients participated in the study and all 3 patients withdrew consent prematurely. Therefore the results are summarized descriptively only.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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
Results Point of Contact
Name/Title: VP of Clinical Research and Development
Organization: Octapharma
Phone: +41 554512141
Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT01341912     History of Changes
Other Study ID Numbers: GENA-11
First Submitted: April 22, 2011
First Posted: April 26, 2011
Results First Submitted: October 11, 2013
Results First Posted: February 3, 2014
Last Update Posted: February 3, 2014