ObsITI research program - Summary
The Observational Immune Tolerance Induction (ObsITI) research program is an international open-label, uncontrolled, non-interventional, multi-centre observational program (ClinicalTrials.gov NCT 02207894) initiated by the the Paediatric Centre of the Johann Wolfgang-Goethe University Frankfurt, and continued by the Haemophilia Centre Rhine-Main (HZRM), Frankfurt-Mörfelden, Germany. The study started in 2005 and has a retrospective and a prospective arm. Data from patients, who currently undergo ITI or have undergone ITI will be collected in this study. The ITI outcome will be correlated to patient therapy variables and immunological related variables. In the prospective part, the patients are also invited to take part in several sub-studies.
All information regarding the study and the study protocol can be obtained from firstname.lastname@example.org.
Primary ObjectiveThe primary objective of the ObsITI study is to evaluate and document data on the success rate of ITI in prospective and retrospective haemophilia A patients with newly developed or already existing FVIIIinhibitors, including patients with risk factors associated with a poor ITI prognosis, and those with prior ITI failure. It is recommended to treat patients according to the Bonn protocol.
Secondary ObjectiveThe secondary objective of the ObsITI research program is to investigate the possible inﬂuence of the following variables on the duration and success of ITI:
- Patient age at the start of ITI
- Inhibitor titres (at start of ITI and peak titres)
- Surgical procedures
- Inflammatory status during ITI
- Dose and frequency of FVIII administration
- Type of FVIII concentrate used
- Epitope mapping and IgG subclass analysis
- The impact of in-vitro tests (modifed Oxford inhibitor assay, IBA, TGA)
- The analysis and dynamics of lymphocytes and further immunological parameters during an ITI course
- Immunogenotype and HLA-type
- TGT to monitor bleeding tendency during ITI using in vitro and ex vivo tests
- Frequency and severity of bleeding episodes before and during ITI
- Intensity and modality of previous treatment
The observational study protocol, the patient information and informed consent form, as well as all other essential documents (e.g. diaries provided to the patients) have been approved by the Ethics Committee of the University Hospital in Frankfurt (Main), Germany and the Landesärztekammer Hessen, Germany.