Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A."

NCT02581969 | Completed

• 1 other identifier: 18251
• Study Type: observational
• Target: 43
• Locations: 1 country
• Sites: 1

Brief Summary

Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study. The present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.

Conditions

Hemophilia A

Keywords

Hemophilia A, Congenital

Outcome Measures

Primary Outcomes (1)

• Number of joint bleeding episodes per year

  5 years period

Secondary Outcomes (13)

• Total number of bleeding episodes per year

  5 years period

• Total amount of Recombinant Factor VIII consumption

  5 years period

• Changes of prophylaxis dose

  5 years period

• Changes of frequency of infusions

  5 years period

• Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)

  5 years period

Study Arms (2)

Prophylaxis

Prophylaxis group: treatment is based on regularly repeated infusions of clotting factor, 20-30 IU/kg -3 times a week

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

On-demand

On-demand group: treatment administered when bleeding episode occur

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

Interventions

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

Administered 20-30 IU/kg 3 times a week

Prophylaxis

Eligibility Criteria

• Age: 17 Years - 60 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

A total of approximately 50 patients with a diagnosis of severe haemophilia A (FVIII \< 1%) , who are already on Kogenate Bayer treatment ongoing for long-term secondary prophylaxis or on-demand therapy and have properly completed the follow-up in the original Potter study are expected to be enrolled.

You may qualify if:

• Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint;
• Written informed consent specifically issued for the 5-year extension.

You may not qualify if:

• Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.

Sponsors & Collaborators

• Bayer: lead

Study Sites (1)

Unknown Facility

Multiple Locations, Italy

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII; F8 protein, human; BAY 14-2222

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Bayer Study Director

  Bayer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2015
• First Posted: October 21, 2015
• Study Start: April 14, 2016
• Primary Completion: March 16, 2017
• Study Completion: June 21, 2018
• Last Updated: November 18, 2020

Record last verified: 2020-11

Locations

IT (1)