A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B

NCT03055611 | Completed

• 1 other identifier: Sobi.HAEM89-002
• Study Type: observational
• Target: 201
• Locations: 1 country
• Sites: 22

Brief Summary

Elocta (rFVIIIFc) and Alprolix (rFIXFc) are recombinant extended half-life coagulation factor products. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Elocta and Alprolix in the prophylactic treatment of haemophilia A and B.

Conditions

Haemophilia A; Haemophilia B

Keywords

Blood Coagulation Disorder; Hematologic Diseases; Coagulation Protein Disorder; Hemorrhagic Disorder; Genetic Diseases, Inborn

Outcome Measures

Primary Outcomes (3)

• Annualised bleeding rate (ABR)

  Based on bleeding episodes assessed by local practice

  24 months

• Annualised injection frequency

  Assessed by prescription

  24 months

• Annualised factor consumption (IU)

  Assessed by dispensed factor product

  24 months

Study Arms (2)

Haemophilia A patients

Elocta will be prescribed according to local practice and administered by patients with haemophilia A for prophylactic treatment

Drug: ELOCTA

Haemophilia B patients

Alprolix will be prescribed according to local practice and administered by patients with haemophilia B for prophylactic treatment

Drug: ALPROLIX

Interventions

ELOCTA DRUG

Extended half-life factor VIII product

Also known as: Eloctate, efmoroctocog alfa, rFVIIIFc

Haemophilia A patients

ALPROLIX DRUG

Extended half-life factor IX product

Also known as: Eftrenonacog alfa, rFIXFc

Haemophilia B patients

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All eligible Elocta and Alprolix patients who present for a routine Clinical visit will be asked to participate in the study by the treating physician at participating haemophilia treatment centres in Germany.

You may qualify if:

• Have a diagnosis of haemophilia A or B and previously treated with factor Product
• Have started prophylactic Elocta/Alprolix treatment prior to enrollment visit, or at enrollment prescribed prophylactic treatment with Elocta or Alprolix regardless of participation in the study
• Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, Before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations.

You may not qualify if:

• Participation in an investigational medicinal product trial, from four weeks prior to first injection with Elocta or Alprolix to enrollment visit.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (22)

Swedish Orphan Biovitrum Reserach site

Berlin, Germany

Location

Swedish Orphan Biovitrum Reserach Site

Blaubeuren Abbey, Germany

Location

Swedish Orphan Biovitrum Research Site

Bonn, Germany

Location

Swedish Orphan Biovitrum Research Site

Delmenhorst, Germany

Location

Swedish Orphan Biovitrum Research Site

Duisburg, Germany

Location

Swedish Orphan Biovitrum Reserach site

Erlangen, Germany

Location

Swedish Orphan Biovitrum Research Site

Frankfurt, Germany

Location

Swedish Orphan Biovitrum Reserach site

Frankfurt, Germany

Location

Swedish Orphan Biovitrum Research Site

Fürth, Germany

Location

Swedish Orphan Biovitrum Research Site

Hamburg, Germany

Location

Swedish Orphan Biovitrum Research Site

Hanover, Germany

Location

Swedish Orphan Biovitrum Reserach site

Hanover, Germany

Location

Swedish Orphan Biovitrum Reserach site

Heidelberg, Germany

Location

Swedish Orphan Biovitrum Reserach site

Homburg, Germany

Location

Swedish Orphan Biovitrum Research Site

Jena, Germany

Location

Swedish Orphan Biovitrum Reserach Site

Jena, Germany

Location

Swedish Orphan Biovitrum Reserach site

Leipzig, Germany

Location

Swedish Orphan Biovitrum Reserach Site

Mörfelden-Walldorf, Germany

Location

Swedish Orphan Biovitrum Research Site

München, Germany

Location

Swedish Orphan Biovitrum Reserach site

München, Germany

Location

Swedish Orphan Biovitrum Reserach site

Münster, Germany

Location

Swedish Orphan Biovitrum Research Site

Würzburg, Germany

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn

Interventions

factor VIII-Fc fusion protein; factor IX Fc fusion protein

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Study Physician, MD

  Swedish Orphan Biovitrum

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: February 14, 2017
• First Posted: February 16, 2017
• Study Start: May 9, 2017
• Primary Completion: April 19, 2022
• Study Completion: April 19, 2022
• Last Updated: October 2, 2024

Record last verified: 2024-10

Locations

DE (22)