Grants Awarded

Histiocyte Society Clinical Trials Database System LCH-IV Treatment Protocol Study

Project Administrators
Histiocyte Society Executive Board
LCH Study Reference Center – Vienna, Austria

Date of Award
December 2012

Amount of Award

Layperson Summary
The Histiocyte Society Clinical Trials Database System (HSCTDS)—an online, remote data entry system—will be built under the supervision of the Histiocyte Society. The company creating and hosting the system, Austrian Research Centers, was chosen by the Histiocyte Society Executive Board following a review of proposals from a variety of database companies specializing in online clinical data management.

While the HSCTDS will initially be used to collect information for the LCH-IV Treatment Protocol Study (LCH-IVTPS), it will eventually serve as the database for all current and future Histiocyte Society Clinical Trials.  The system will allow participants (physicians placing a patient or patients in the study) from around the world to conduct direct entry of patient information, whereas previous studies have required that data to be sent to a central location responsible for data entry.  This new entry method decreases the time necessary to complete the lengthy paperwork associated with clinical trial questionnaires.

In order to facilitate effective data collection and patient enrollment, the HSCTDS will incorporate cutting edge technology that includes mandatory data field completion and an automatic, patient follow-up notification system that alerts participants of the need to provide a subsequent data report.  The increased efficiency of the HSCTDS will expedite the completion of the LCH-IVTPS, as well as other future studies, and result in the prompt publication and distribution of its findings, and those of subsequent projects.

LCH-IV Treatment Protocol Study Update

In designing the study, the Histiocyte Society Executive Board and study co-chairs, Drs. Carlos Rodriguez-Galindo and Milen Minkov, took a more ambitious approach than those used in previous histiocytosis clinical trials. Whereas past trials have been focused on evaluating the effectiveness of standard front-line treatment methods, the LCH-IV Study will incorporate multiple salvage alternatives in addition to the front-line treatment. This approach of combining multiple treatment modalities and research objectives in one protocol adds significant complexity to the study design. Few studies in medical research have used this approach, making its introduction an important milestone in the field of histiocytosis research.

In this highly sophisticated study, patients with newly diagnosed LCH will initially be enrolled on the front-line treatment protocol. Patients who experience reactivations of their disease will then be enrolled on the appropriate salvage treatment protocols included within the study. These salvage options, designed as independent treatment protocols, include low-risk, high-risk, and bone marrow transplant. As well, the study’s investigators have integrated an analysis of the long-term effects of LCH and its treatment as experienced by patients into the entire trial. The primary focus of this research will be to understand the risk factors for the development of neurodegeneration and neuroendocrine deficits, devise early diagnostic tools, and provide effective treatments.

Because of its comprehensive design, this study will allow researchers to evaluate the effectiveness of five protocols for the treatment of LCH and examine a variety of important questions related to the study’s findings. As the field of medical research is currently subject to increasingly stringent regulatory requirements, the study’s investigators intend for its comprehensive structure to result in a greater number of participating institutions, an increase in the amount and quality of data generated, and the study’s overall success. While its launch has been delayed slightly due to the complexity and originality of its design, the study’s primary investigators intend for it to be finalized and opened in advance of the Histiocyte Society Annual Meeting, which will take place in Boston, Massachusetts in October 2010.

The study’s investigators commend the staff of the Children’s Cancer Research Institute in Austria, Vienna, led by Dr. Milen Minkov, for their extraordinary efforts in finalizing the study’s document and database for its impending launch. The final version of the protocol will be circulated for review to participating researchers within the next two months.

The study’s authors express their appreciation to the Histiocytosis Association for supporting this important initiative.

With kind regards,
Drs. Carlos Rodriguez-Galindo and Milen Minkov
LCH-IV Study Co-Chairpersons

Twelve Month Report