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Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment (SPINART)
This study has been completed.
Sponsor:
Bayer
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT00623480
First received: February 4, 2008
Last updated: November 5, 2014
Last verified: November 2014
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Purpose
To evaluate the effect of secondary prophylaxis as compared to episodic treatment on bleeding frequency (number of bleeds per year) and on joint damage.
| Condition | Intervention | Phase |
|---|---|---|
| Hemophilia A | Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222) | Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment |
| Official Title: | Randomized, Controlled, Parallel, Prospective Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects, as Applicable, Compared to That of Episodic Treatment |
Resource links provided by NLM:
Genetics Home Reference related topics:
hemophilia
MedlinePlus related topics:
Hemophilia
U.S. FDA Resources
Further study details as provided by Bayer:
Primary Outcome Measures:
- Bleeding Frequency (Number of Total Bleeds) [ Time Frame: After the last enrolled patient has been in the study for 1 year. At the cut-off, the median follow-up duration was 616 days (minimum was 111 days and maximum was 1109 days) ]
Secondary Outcome Measures:
- Change From Baseline to 3 Years in the MRI (Magnetic Resonance Imaging) Scale. [ Time Frame: Baseline and 3 years ]The Extended MRI Scale total score has a range between 0 (normal unaffected joint) to 45 (maximal joint damage) points. It is composed of 2 domains, the soft tissue domain with a maximum of 9 points and the osteochondral domain with a maximum of 36 points. A single score for each subject was to be calculated from the sum of both domains and the average over all joints for the Extended MRI endpoint. Higher MRI score denotes greater joint structure damage thus a positive change from baseline means worsening.
- Change From Baseline to 3 Years in the Colorado Adult Joint Assessment Scale [ Time Frame: Baseline and 3 years ]The total joint score is derived for each of six joints: left and right sides for knees (score: 0-25), ankles (score: 0-25), and elbows (score: 0-21). Higher CAJAS (Colorado Adult Joint Assessment Scale) score denotes greater joint structure damage thus a positive change from baseline means worsening. CAJAS total score is the sum of all 6 joints, ranging from 0 (best possible outcome) to 142 (worst possible outcome).
Other Outcome Measures:
- Change From Baseline to 3 Years in the Physical Functioning Domain of the Haemo-QoL-A [ Time Frame: Baseline and 3 years ]The Haemo-QoL-A total score as well as each of its domains have a range between 0 (worst Quality of Life) and 100 (best Quality of Life) points. Therefore, a higher Haemo-QoL-A score denotes greater Quality of Life.
| Enrollment: | 84 |
| Study Start Date: | March 2008 |
| Study Completion Date: | November 2013 |
| Primary Completion Date: | September 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Recombinant Factor VIII prophylaxis treatment
Participants received 25 IU/kg of Recombinant Factor VIII (Kogenate FS, BAY14-2222) intravenously (IV), 3 times per week. Dose escalation steps by 5 IU/kg (to 30 IU/kg or 35 IU/kg maximum) for patients exhibiting a bleeding frequency of 12 bleeding episodes per year or greater.
|
Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Prophylaxis treatment includes three times per week administration of 25 IU/kg of Kogenate FS. Dose escalation steps by 5 IU/kg (to 30 IU/kg or 35 IU/kg maximum) exhibiting a bleeding frequency of 12 bleeding episodes per year or greater.
|
|
Experimental: Recombinant Factor VIII on-demand treatment
Participants received Recombinant Factor VIII (Kogenate FS, BAY14-2222) IV for bleeds in accordance with package insert instructions and study physician recommendations.
|
Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Treated according to the Kogenate FS package insert indications and study physician recommendations
|
Eligibility| Ages Eligible for Study: | 12 Years to 50 Years (Child, Adult) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Males aged 12 to 50 years (US and Argentina)
- Males aged 18 to 50 years (other countries)
- Subjects with severe hemophilia A (<1% FVIII:C) as confirmed by the central lab from a sample obtained at least 96 hours after FVIII administration wash-out. Allow for the inclusion of a maximum of 10% (n=8) of patients with 1-2% FVIII:C baseline levels as long as they exhibit clinical severity and comply with all other inclusion criteria.
- Subjects with at least 150 prior exposure days with any FVIII
- Subjects who have been on episodic treatment and no known regular prophylaxis treatment for more than 12 consecutive months in the previous 5 years
- Subjects with 6 to 24 bleeding events and/or treatments in the previous 6 months prior to study entry which are documented and available in the subjects medical records. Documentation can include records from previous physicians, specific home treatment records, emergency room or hospital records, x-ray reports, etc. The investigator can also document with a detailed note the number of bleeds reported by the subject in the last 6 months.
-
Subjects with inhibitor formation surveillance (inhibitor or recovery testing) over the ten years prior to enrollment documented by the investigator and who do not have a history of any of the following:
- A positive inhibitor titer of 5.0 Bethesda Unit (BU) or greater by either BU assay system at any time since first exposure to exogenous factor VIII
- A positive inhibitor test result of 1.0 or greater performed by the original BU assay at any time in the past 10 years (A subject can have more than one positive inhibitor test of 0.6 or greater by the original BU assay test but all must be less than 1.0 BU using the original BU assay.)
- A positive inhibitor test result of 0.6 or greater performed by the Nijmegen method at any time in the past 10 years
- Subjects with no inhibitor activity by Nijmegen-modified Bethesda assay, either positive (> 0.6 BU is considered positive) or borderline (> 0.3 and < 0.6 BU is considered borderline) as measured in the current study reference laboratory
Exclusion Criteria:
- Subjects with any other bleeding disease besides hemophilia A (i.e. von Willebrand disease)
- Subjects with thrombocytopenia (platelets < 100,000/mm3)
- Subjects with abnormal renal function (Cockcroft-Gault Creatinine Clearance value of 60 mL/min or lower)
- Subjects with active hepatic disease (Aspartate aminotransferase [AST] or Alanine aminotransferase [ALT] > 5xUpper Limit of Normal (ULN))
- Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study (the following drugs are however allowed: interferon-a treatment for Hepatitis C virus (HCV), Highly active anti-retroviral therapy (HAART) therapy for human immunodeficiency virus (HIV) and/or a total of two courses of pulse treatment with steroids for a maximum of 7 days at 1mg/kg or less)
- Subjects with an absolute CD4 lymphocyte cell count < 200 cells/mm3 (due to HIV, HCV or another suspected medical condition)
- Subjects with known hypersensitivity to rFVIII, mouse or hamster proteins
- Subjects who are receiving or had received other experimental drugs within 1 month prior to study entry
- Subjects who require any pre-medication to tolerate FVIII injections (e.g. anti-histamines)
- Subjects who are unwilling to comply with study visits or either of the possible treatment regimens
- Subjects who have a planned orthopedic intervention to be performed during the study that may substantially affect bleeding (e.g. surgical or chemical or radiological synovectomy)
- Subjects who are not suitable for participation in this study for any reason, according to the Investigator
- Subjects who have poor joint status as defined by routine need for a wheelchair or unable to ambulate without the assistance of a brace, cane or crutches
- Three or more joints that are already fused or "frozen" also called ankylosis
Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00623480
Show 38 Study Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00623480
Show 38 Study Locations
Sponsors and Collaborators
Bayer
Investigators
| Study Director: | Bayer Study Director | Bayer |
More Information
Additional Information:
Publications:
| Responsible Party: | Bayer |
| ClinicalTrials.gov Identifier: | NCT00623480 History of Changes |
| Other Study ID Numbers: |
12800 2008-000985-21 ( EudraCT Number ) |
| Study First Received: | February 4, 2008 |
| Results First Received: | May 2, 2013 |
| Last Updated: | November 5, 2014 |
Keywords provided by Bayer:
|
Hemophilia A |
Additional relevant MeSH terms:
|
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Coagulation Protein Disorders |
Hemorrhagic Disorders Genetic Diseases, Inborn Factor VIII Coagulants |
ClinicalTrials.gov processed this record on August 18, 2017