A registry for hemophagocytic lymphohistiocytosis (HLH)
Organizational Data
- DRKS-ID:
- DRKS00010148
- Recruitment Status:
- Recruiting ongoing
- Date of registration in DRKS:
- 2016-03-15
- Last update in DRKS:
- 2024-01-22
- Registration type:
- Prospective
Acronym/abbreviation of the study
HLH registry
URL of the study
No Entry
Brief summary in lay language
HLH registry is a database initiated as collaborative project by two international research societies: Histiocyte Society (HS) and European Society of Immunodeficiencies (ESID). Hemophagocytic lymphohistiocytosis (HLH) is a rare life-threatening condition of severe hyperinflammation. Manifestation of the disease is promoted by a variety of underlying genetic conditions. HLH is a highly aggressive disease and therapy continues to be a challenge. The current established therapy protocol still results in a mortality of 40%, other protocols are currently investigated in pilot studies. After finalisation of these pilot studies both involved research societies intend to initiate a controled, randomized therapy trial. To prepare this trial an international registry for HLH shall be established. The main purpose of the HLH registry is a worldwide data collection as feasibility for the therapy trial. Apart from detailed analysis of the current standard of care the registry aims on enhancement of the international network of potential study centres and thus improving the infrastructure for the future trial.
Brief summary in scientific language
To improve therapy for HLH, a prospective randomized treatment study is highly desirable. The key open issue is the feasibility of such a trial design in a rare disease. For a feasibility assessment, the following questions need to be addressed in particular: 1. How many patients can potentially be recruited into an international HLH treatment trial? 2. How many patients are transplanted and within which time after initiation of treatment? 3. What are the outcomes for patients treated according to current standard of care? These questions shall be addressed in the international HLH Registry which is a database initiated as a collaborative project by the Histiocyte Society (HS) and the European Society for Immunodeficiencies (ESID) in collaboration with the Inborn Errors Working Party (IEWP) of the European Bone Marrow Transplantation Group (EBMT). The primary goal of the HLH Registry is to collect data relevant for assessment of feasibility and design of future interventional studies on the treatment of HLH. This requires a detailed analysis of the target patient population treated by centers potentially participating in such studies. Another important goal of implementing a HLH Registry is to improve the network of physicians collaborating on HLH and to prepare the infrastructure for a future trial. Implementing routines for patient identification (and central registration), diagnostic algorithms and training the use of a GCP database that is fully adapted to running interventional clinical trials are important milestones in facilitating the execution of a clinical trial
Health condition or problem studied
- Free text:
- hemophagocytic lymphohistiocytosis (HLH)
- ICD10:
- D76.1 - Haemophagocytic lymphohistiocytosis
- Healthy volunteers:
- No Entry
Interventions, Observational Groups
- Arm 1:
- Data collection: Demographic characteristics, Form of HLH, HLH clinical criteria, Diagnostic assays performed, CNS disease, HLH directed treatment and outcome, HSCT and and outcome, Chimerism, Disease-causing mutation
Endpoints
- Primary outcome:
- Collect data to judge feasibility of a future HLH trial: Number of eligible patients/year Outcome associated with standard of care Frequency of CNS disease Time to first HLH directed drug Time to Transplant
- Secondary outcome:
- Improve the infrastructure for a future HLH study : Standardization of patient registration Standardization of patient documentation Standardization of diagnostic evaluation Implementation of national and international coordination
Study Design
- Purpose:
- Other
- Retrospective/prospective:
- No Entry
- Study type:
- Non-interventional
- Longitudinal/cross-sectional:
- No Entry
- Study type non-interventional:
- No Entry
Recruitment
- Recruitment Status:
- Recruiting ongoing
- Reason if recruiting stopped or withdrawn:
- No Entry
Recruitment Locations
- Recruitment countries:
-
- France
- Germany
- Spain
- United Kingdom
- Number of study centers:
- Multicenter study
- Recruitment location(s):
-
- University medical center CCI Freiburg im Breisgau
Recruitment period and number of participants
- Planned study start date:
- 2016-05-02
- Actual study start date:
- 2016-05-02
- Planned study completion date:
- No Entry
- Actual Study Completion Date:
- No Entry
- Target Sample Size:
- 300
- Final Sample Size:
- No Entry
Inclusion Criteria
- Sex:
- All
- Minimum Age:
- no minimum age
- Maximum Age:
- no maximum age
- Additional Inclusion Criteria:
- • Patients with primary immunodeficiencies predisposing to HLH (all ages) • Patients fulfilling >/= 4/8 diagnostic criteria for HLH as defined by the Histiocyte Society and receiving HLH directed therapy (steroids are sufficient) • Patients presenting with a high suspicion of isolated CNS-HLH and receiving HLH directed therapy • Patients with active HLH that is not secondary to underlying conditions such as rheumatological disease, malignancy, metabolic disease or Leishmania infection (mostly “infection-only” HLH) (all ages) • Patients (or their legal representatives) having provided informed consent
Exclusion Criteria
• Patients with malignancy • Patients with rheumatological/autoinflammatory/autoimmune disease • Patients with metabolic diseases • Patients with Leishmania infection • No written consent available
Addresses
Primary Sponsor
- Address:
- Universitätsklinikum FreiburgCentrum für Chronische Immundefizienz (CCI)Prof. Dr. med. Stephan EhlBreisacher Str. 11579106 FreiburgGermany
- Telephone:
- +49 761 270 77550
- Fax:
- +49 761 270 77744
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- http://www.uniklinik-freiburg.de/cci/live/index.html
- Investigator Sponsored/Initiated Trial (IST/IIT):
- Yes
Contact for Scientific Queries
- Address:
- UKE HamburgKlinik und Poliklinik für Pädiatrische Hämatologie und OnkologiePD Dr. med. Kai LehmbergMartinistr. 5220246 HamburgGermany
- Telephone:
- +49 (0) 40 7410 - 52580
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- http://www.uke.de/hlh
Contact for Public Queries
- Address:
- UKE HamburgKlinik und Poliklinik für Pädiatrische Hämatologie und OnkologiePD Dr. med. Kai LehmbergMartinistr. 5220246 HamburgGermany
- Telephone:
- +49 (0) 40 7410 - 52580
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- http://www.uke.de/hlh
Principal Investigator
- Address:
- UKE HamburgKlinik und Poliklinik für Pädiatrische Hämatologie und OnkologiePD Dr. med. Kai LehmbergMartinistr. 5220246 HamburgGermany
- Telephone:
- +49 (0) 40 7410 - 52580
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- http://www.uke.de/hlh
Sources of Monetary or Material Support
Private sponsorship (foundations, study societies, etc.)
- Address:
- Deutsche Kinderkrebsstiftung53113 BonnGermany
- Telephone:
- +49(0)2 28 / 68 84 6 - 0
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- http://www.kinderkrebsstiftung.de
Private sponsorship (foundations, study societies, etc.)
- Address:
- Histio UKPL14 9DQ LISKEARDUnited Kingdom
- Telephone:
- +4407850 740241
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- http://www.histiouk.org
Ethics Committee
Address Ethics Committee
- Address:
- Ethik-Kommission der Albert-Ludwigs-Universität FreiburgEngelberger Str. 2179106 FreiburgGermany
- Telephone:
- +49-761-27072600
- Fax:
- +49-761-27072630
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Vote of leading Ethics Committee
- Vote of leading Ethics Committee
- Date of ethics committee application:
- 2015-12-22
- Ethics committee number:
- 610/15
- Vote of the Ethics Committee:
- Approved
- Date of the vote:
- 2016-01-26
Further identification numbers
- Other primary registry ID:
- No Entry
- EudraCT Number:
- No Entry
IPD - Individual Participant Data
- Do you plan to make participant-related data (IPD) available to other researchers in an anonymized form?:
- No
- IPD Sharing Plan:
- No Entry
Study protocol and other study documents
- Study protocols:
- No Entry
- Study abstract:
- No Entry
- Other study documents:
- No Entry
- Background literature:
- No Entry
- Related DRKS studies:
- No Entry
Publication of study results
- Planned publication:
- No Entry
- Publikationen/Studienergebnisse:
- No Entry
- Date of first publication of study results:
- No Entry
- DRKS entry published for the first time with results:
- No Entry
Basic reporting
- Basic Reporting / Results tables:
- No Entry
- Brief summary of results:
- No Entry