A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled ITI

NCT02207894 | Recruiting

• 1 other identifier: HZRM_ObsITI_4th Am.Version
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 1

Brief Summary

This research program is initiated to evaluate and document data on the success of ITI in 300 haemophilia A patients with newly developed or already existing FVIII-inhibitors (also patients who might potentially have failed in earlier ITIs), which will be treated with ITI - preferably high-dose based on individualized product selection, in order to improve management of this potentially devastating complication of haemophilia treatment. In order to investigate the role of in vitro tests on individual ITI success rate in patients undergoing ITI, the inhibitor plasma samples can be assayed against different FVIII concentrates using the following in vitro tests: Batch selection, Thrombin generation assay (TGA), Thrombin Generation Test (TGT) to monitor FVIII efficacy, Epitope mapping,IgG Subclasses specific for FVIII, Immunogenotyping.

Conditions

Haemophilia A

Keywords

Haemophilia A, factor VIII, development of inhibitor

Outcome Measures

Primary Outcomes (1)

• The efficacy of ITI, the primary endpoint of this observation, is defined according to the following criteria (The measure is a composite).: Inhibitor titre <0.6 BU, Incremental recovery of FVIII in the normal range,Half-life of FVIII > 7 hours.

  The efficacy of ITI, the primary endpoint of this observation, is defined according to the following criteria (The measure is a composite): * Inhibitor titre \<0.6 BU (at least 2 consecutive determinations) * Incremental recovery of FVIII in the normal range (\> 80% of normal) with samples taken prior to and 15 or 30 minutes after FVIII treatment. * Half-life of FVIII \> 7 hours (blood samples for FVIII determination should be taken prior to and 15 or 30 minutes, 1, 2, 4, 8 and either 12 or 24 hours after FVIII treatment. Complete Success: All three criteria above met. Partial Success: Two of the three criteria above met. Partial Response: One of the three criteria above met. Partial Failure of ITI-treatment: Inhibitor still present, but titre has decreased to \<5 BU. Complete Failure of ITI-treatment: None of the above mentioned criteria met, and the inhibitor titre is still ≥5 BU.

  one year

Secondary Outcomes (1)

• The following secondary endpoints will also be evaluated.The measure is a composite.

  one year

Eligibility Criteria

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

Study Population

all male patients with haemophilia A and inhibitor

You may qualify if:

• Based on the decision of the treating physicians in the participating centres, male patients at any age suffering from severe (FVIII activity \< 1%), moderate (FVIII activity \>1% - 5%), or mild (FVIII activity \> 5%) haemophilia A will be included into this post marketing observation if relevant inhibitor levels (\> 0.6 BU) have been detected, or - in case of an inhibitor level \<0.6 BU - with reduced recovery or half-life of FVIII.
• The observation is also open for patients who failed an earlier ITI attempt.

You may not qualify if:

• Female

Sponsors & Collaborators

• Haemophilia Centre Rhine Main: lead
• University of Bonn: collaborator
• Skane University Hospital: collaborator
• Hopital Cardiologique Louis Pradel, Unité d'Hemostase Clinique Bron: collaborator

Study Sites (1)

Haemophilia Centre Rhine Main

Frankfurt am Main, Hesse, 60596, Germany

RECRUITING

Related Links

• Click here for more information about this study:The Observational Immune Tolerance Induction (ObsITI) research program

Biospecimen

Retention: SAMPLES WITH DNA

whole blood, serum, white cells, plasma.

MeSH Terms

Conditions

Hemophilia A

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

Study Officials

• Carmen Escuriola Ettingshausen, MD

  Director HZRM

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Carmen Escuriola Ettingshausen, MD

CONTACT

+49 (0) 15115511159; carmen.escuriola@hzrm.de

Study Design

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

Study Record Dates

• First Submitted: May 6, 2014
• First Posted: August 4, 2014
• Study Start: August 1, 2006
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: April 21, 2026

Record last verified: 2026-04

Locations

DE (1)