2011 Study Group Reports
LCH Study Group Report
Submitted by Milen Minkov and Carlos Rodriguez-Galindo, Co-Chairs
LCH-III Study (April 2001 – October 2007)
This prospective clinical trial chaired by Prof. H. Gadner, Vienna, closed for patient enrollment on October 1st 2007. The major preliminary findings are summarized below:
- Group 1 (Risk MS-LCH): A randomized trial including a standard two-drug arm A (PRED/VBL) and a experimental three-drug arm B (PRED/VBL/MTX) was performed in patients with MS-LCH. In both arms initial 6-week course was obligatory for all patients. A second initial course was given to patients with insufficient response. The initial therapy was followed by a continuation treatment to total treatment duration of 1 year. Totally 234 patients were included in the randomized trial (115 in Arm A and 119 in Arm B). There was no statistically significant difference between the standard arm A and the experimental arm B with respect to early response, survival and reactivations.
- Group 2 (Low-risk MS-LCH): The randomized trial for patients without risk organ involvement at diagnosis used a two-drug regiment (PRED/VBL) for total treatment duration of 6 (LR6) and 12 months (LR12), to study the influence of treatment duration on reactivation rate. One hundred eighty-seven patients were included (98 patients in the LR6 Arm and 89 in the LR12 Arm). The experimental arm (LR12) showed statistically significant reduction of the reactivation arm as compared to the standard arm (LR6). Therefore, the conclusion has been made that prolongation of treatment duration can reduced reactivation rate. It is still too early to address the secondary endpoint, namely the rate of permanent consequences in the two randomization arms.- Group 3 (Multifocal Bone LCH and special SS-LCH): The rate of reactivation in this group is in the range of 30-40% at 3 years, and therefore in the range observed in MS-LCH and MFB with the same treatment duration.
The analysis in April 2011 showed results identical to the previous analysis done in August 2010. The final evaluation has been completed and the manuscript approved by the writing committee is nearly to be submitted for publication.
LCH-IV Study Preparation
Similarly to 2010 there was no interim meeting this year and the communication took place primarily by email. The LCH Study Group Meeting took place in Vienna on October 15, 2011. The main accomplishments during the last year are as follows:
- The study protocol document has been finalized by April 2011.
- Submission to the Ethic Committee of the Medical University of Vienna was performed in May 2011. The provisional positive decision was met in June with the request for some minor changes and adaptations concerning the patient information and consent forms.
- A final approval by the above mentioned Ethic Committee was issued by December 2011 and has been translated in to English by an authorized translator.
- The submission to the Austrian Regulatory Authorities was done by August 2011. Their position and requirements for changes of the drug labeling was announced by September 2011. Since then there are ongoing consultations and negotiations for a facilitated approval (with respect to labeling of the drugs as “IMP” or “NIMP”, which has important implications on pharmacovigilance, drug accountability and financing the study). A last round of negotiations is scheduled for January 16th, 2012. In the worst case scenario, meaning no agreement with the authorities on a less strict drug labelling, we will have to perform the required changes and re-apply. In any case the final approval will be expected by the end of February 2012.
- We are proud to announce that the clinical trial remote database has been finalized and we now enter the test phase. We expect to have accomplished the test phase by the end of January 2011 and to be able to launch the database for regular use by the end of February 2012.
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