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Hemophilia APhase 3SPINARTNCT00623480

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

A Phase 3 study testing Recombinant Factor VIII (Kogenate FS, BAY14-2222) in people with Hemophilia A, run by Bayer at 38 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

To evaluate the effect of secondary prophylaxis as compared to episodic treatment on bleeding frequency (number of bleeds per year) and on joint damage. Read more about this study

Who is it for?
Ages 12 to 50 years, men only
Conditions the study is for
Hemophilia A
What is being tested?
Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
84 participants
Where
38 sitesin 4 countries
Study dates
Mar 2008to Nov 2013
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 50 years
  • Men only
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.
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  • 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

Who cannot join

  • 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)
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  • 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

Trial locations and contacts

38 sites in 4 countries.

38 sites in 4 countries

United States28 sites

  • Study site

    Atlanta, Georgia, 30322

  • Study site

    Aurora, Colorado, 80045

  • Study site

    Boston, Massachusetts, 02115

  • Study site

    Chicago, Illinois, 60611

  • Study site

    Chicago, Illinois, 60612

  • Study site

    Cleveland, Ohio, 44106

  • Study site

    Detroit, Michigan, 48201-2196

  • Study site

    Hershey, Pennsylvania, 17033-0850

  • Study site

    Houston, Texas, 77030

  • Study site

    Indianapolis, Indiana, 46260

  • Study site

    Iowa City, Iowa, 52242-1089

  • Study site

    Kansas City, Missouri, 64108

  • Study site

    Knoxville, Tennessee, 37920

  • Study site

    Las Vegas, Nevada, 89109-2803

  • Study site

    Little Rock, Arkansas, 72202

  • Study site

    Louisville, Kentucky, 40202

  • Study site

    Milwaukee, Wisconsin, 53226

  • Study site

    Minneapolis, Minnesota, 55455

  • Study site

    New York, New York, 10029

  • Study site

    New York, New York, 10065

  • Study site

    Newark, New Jersey, 07112

  • Study site

    Orange, California, 92868

  • Study site

    Orlando, Florida, 32801

  • Study site

    Pittsburgh, Pennsylvania, 15213

  • Study site

    Sacramento, California, 95817

  • Study site

    Salt Lake City, Utah, 84132

  • Study site

    Tucson, Arizona, 85724

  • Study site

    Washington D.C., District of Columbia, 20007-2197

Other trials in United States

Romania5 sites

  • Study site

    Brasov, 50035

  • Study site

    Bucharest, 022328

  • Study site

    Bucharest, 11026

  • Study site

    Constanța, 900591

  • Study site

    Timișoara, Timiș County, 300011

Other trials in Romania

Bulgaria3 sites

  • Study site

    Plovdiv, 4002

  • Study site

    Sofia, 1756

  • Study site

    Varna, 9010

Other trials in Bulgaria

Argentina2 sites

  • Study site

    Buenos Aires, Ciudad Auton. de Buenos Aires, C1221 ADC

  • Study site

    Rosario, Santa Fe Province, S2000CKF

Other trials in Argentina

About this study

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

To evaluate the effect of secondary prophylaxis as compared to episodic treatment on bleeding frequency (number of bleeds per year) and on joint damage.

Conditions
Hemophilia A
Being tested
Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Keywords
Hemophilia A
Medical subject headings
Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Recombinant Factor VIII (Kogenate FS, BAY14-2222)Biological

    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.

  • Recombinant Factor VIII (Kogenate FS, BAY14-2222)Biological

    Treated according to the Kogenate FS package insert indications and study physician recommendations

Study groups in NCT00623480
Study groupRoleWhat they receive
Recombinant Factor VIII prophylaxis treatmentExperimentalParticipants 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.
Receives: Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Recombinant Factor VIII on-demand treatmentExperimentalParticipants received Recombinant Factor VIII (Kogenate FS, BAY14-2222) IV for bleeds in accordance with package insert instructions and study physician recommendations.
Receives: Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Bleeding Frequency (Number of Total Bleeds)

    Measured: 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 outcomes (2)

  1. Change From Baseline to 3 Years in the MRI (Magnetic Resonance Imaging) Scale.

    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.

    Measured: Baseline and 3 years

  2. Change From Baseline to 3 Years in the Colorado Adult Joint Assessment Scale

    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).

    Measured: Baseline and 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Feb 2008 (estimated)
  2. Study startMar 2008
  3. Primary completionSep 2011
  4. Study completionNov 2013

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
84 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00623480
Sponsor's protocol ID
12800
Other IDs
2008-000985-21, 2008
Acronym
SPINART
Status last verified
Nov 2014
First submitted
4 Feb 2008
Last updated
17 Nov 2014
Data monitoring committee
Yes

Publications

  • Manco-Johnson MJ, Kempton CL, Reding MT, Lissitchkov T, Goranov S, Gercheva L, Rusen L, Ghinea M, Uscatescu V, Rescia V, Hong W. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). J Thromb Haemost. 2013 Jun;11(6):1119-27. doi: 10.1111/jth.12202.

    PubMed 23528101

  • Manco-Johnson MJ, Lundin B, Funk S, Peterfy C, Raunig D, Werk M, Kempton CL, Reding MT, Goranov S, Gercheva L, Rusen L, Uscatescu V, Pierdominici M, Engelen S, Pocoski J, Walker D, Hong W. Effect of late prophylaxis in hemophilia on joint status: a randomized trial. J Thromb Haemost. 2017 Nov;15(11):2115-2124. doi: 10.1111/jth.13811. Epub 2017 Oct 10.

    PubMed 28836341

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Recombinant Factor VIII (Kogenate FS, BAY14-2222) in people with Hemophilia A, run by Bayer at 38 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 38 sites in United States, Romania, Bulgaria, Argentina. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Aug 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00623480, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Nov 2014.

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