NCT01159587

Brief Summary

The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII \< 1%). The observational period will cover at least 5 years per patient. The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
58

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jul 2004

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2004

Completed
6 years until next milestone

First Submitted

Initial submission to the registry

July 8, 2010

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 9, 2010

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2010

Completed
Last Updated

April 13, 2011

Status Verified

April 1, 2011

Enrollment Period

6.4 years

First QC Date

July 8, 2010

Last Update Submit

April 12, 2011

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

  • Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year

    Every 6 months

Secondary Outcomes (5)

  • Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes

    Every 6 months

  • Muscolo skeletal evaluation by Orthopedic Joint Score

    Every 12 months

  • Radiological Evaluation by Pettersson Score

    Baseline and after 3-5 years

  • Health related quality of life

    Every 12 months

  • cost-effectiveness and utility, patient compliance, adverse events

    Every 6 months

Study Arms (2)

Group 1

Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Group 2

Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Interventions

Recombinant Factor VIII (Kogenate FS, BAY14-2222)DRUG

Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly

Group 1

Eligibility Criteria

Age12 Years - 55 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

Haemophilics

You may qualify if:

  • age ≥ 12 years and ≤ 55 years
  • severe haemophilia A (FVIII \< 1%)
  • absence of inhibitors (Bethesda titre \< 0.6 BU/ml)
  • Previous Treated Patients (prior exposure days \> 200)
  • Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group
  • ≥ 6 joint bleeds requiring treatment with FVIII concentrates in the previous 6 months before enrollment, for on-demand group
  • written informed consent

You may not qualify if:

  • concomitant severe and chronic diseases or congenital skeletal malformation
  • unreliability of patient or likelihood of follow-up failure
  • presence of inhibitors or history of inhibitors (in the previous 2 years)
  • currently on immune tolerance treatment
  • hepatic cirrhosis or liver disease in rapid progression
  • AIDS
  • platelet count \< 75,000/mm3
  • presence of conditions that influence negatively patient´s compliance
  • participation in another study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Unknown Facility

Many Locations, Italy

Location

Related Publications (1)

  • Tagliaferri A, Feola G, Molinari AC, Santoro C, Rivolta GF, Cultrera DB, Gagliano F, Zanon E, Mancuso ME, Valdre L, Mameli L, Amoresano S, Mathew P, Coppola A; POTTER Study Group. Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thromb Haemost. 2015 Jul;114(1):35-45. doi: 10.1160/TH14-05-0407. Epub 2015 Apr 9.

    PMID: 25855376DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIIIF8 protein, humanBAY 14-2222

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Study Officials

  • Bayer Study Director

    Bayer

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

July 8, 2010

First Posted

July 9, 2010

Study Start

July 1, 2004

Primary Completion

December 1, 2010

Study Completion

December 1, 2010

Last Updated

April 13, 2011

Record last verified: 2011-04

Locations

IT(1)