Survey on the success of individualized treatment for haemophilia A
A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled (High Dose) Immune Tolerance Induction (ITI)
This study is testing whether a personalized high-dose treatment can help people with haemophilia A who have developed inhibitors against factor VIII and if certain lab tests can predict how well this treatment will work.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 300 (estimated) |
| Sex | Male |
| Sponsor | Haemophilia Centre Rhine Main Academic / other |
| Drugs / interventions | emicizumab, Rituximab |
| Locations | 1 site (Frankfurt am Main, Hesse) |
| Trial ID | NCT02207894 on ClinicalTrials.gov |
What this trial studies
This observational program evaluates the success of immune tolerance induction (ITI) in 300 patients with haemophilia A who have developed inhibitors against factor VIII. The study focuses on high-dose treatment based on individualized concentrate selection to improve management of this complication. It also investigates the role of various in vitro tests in predicting ITI success rates. Data will be systematically documented and analyzed to enhance understanding of treatment efficacy.
Who should consider this trial
Good fit: Ideal candidates include male patients of any age with severe, moderate, or mild haemophilia A who have relevant inhibitor levels detected.
Not a fit: Patients who are female will not benefit from this study as they are excluded from participation.
Why it matters
Potential benefit: If successful, this study could significantly improve treatment outcomes for patients with haemophilia A who develop inhibitors.
How similar studies have performed: Previous studies have shown that high-dose ITI can be effective in inducing immune tolerance in haemophilia A patients, suggesting a foundation for this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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. Exclusion Criteria: * Female
Where this trial is running
Frankfurt am Main, Hesse
- Haemophilia Centre Rhine Main — Frankfurt am Main, Hesse, Germany (Recruiting)
Study contacts
- Principal investigator: Carmen Escuriola Ettingshausen, MD — Director HZRM
- Study coordinator: Carmen Escuriola Ettingshausen, MD
- Email: carmen.escuriola@hzrm.de
- Phone: +49 (0) 15115511159
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.