- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00426101
Treatment Protocol for Hemophagocytic Lymphohistiocytosis 2004
HLH-2004 Treatment Protocol
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
-
-
-
Stockholm, Sweden, S-171 76
- Childhood Cancer Research Unit, Karolinska Hospital
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Patients who fulfil the diagnostic criteria of HLH.
Exclusion Criteria:
- Prior cytotoxic or cyclosporin treatment for HLH.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Etoposide, Dexamethasone, Cyclosporin A plus IT MTX & Steroids
As compared to the HLH-94 treatment, the main changes are that
Drugs, dosage, frequency and duration are described in the paragraph "Interventions" below. |
10 mg/m2 daily wk 1-2 5 mg/m2 daily wk 3-4 2.5 mg/m2 daily wk 5-6 1.25 mg/m2 daily wk 7 Steroids tapered wk 8 If continuation: Pulses every 2nd wk, 10 mg/m2 for 3 days 150 mg/m2 iv twice/wk (wk 1-2) 150 mg/m2 iv once/wk (wk 3-8) If continuation: 150 mg/m2 iv, every 2nd wk WK 1-8: - Aim at around 200 microgram/L (trough value). Start: 6 mg/kg daily (divided in 2 daily doses) wk 1, if kidney function is normal. If continuation: - Aim for around 200 microgram/L. Monitor GFR. If at 2 wks there are progressive neurological symptoms or if an abnormal CSF (cell count and protein) has not improved, then give 4 wkly intrathecal inj. Be aware that some pat may have increased intracranial pressure. Methotrexate: <1 yr 6 mg, 1-2 yrs 8 mg, 2-3 yrs 10 mg, >3 yrs 12 mg. Prednisolone: <1 yr 4 mg, 1-2 yrs 6 mg, 2-3 yrs 8 mg, >3 yrs 10 mg.
The SCT procedure is up to the treating physician.
However, a suggested regimen is provided.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Survival
Time Frame: 1-year after diagnosis
|
1-year after diagnosis
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Late effects
Time Frame: 5-years after diagnosis
|
5-years after diagnosis
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Jan-Inge Henter, MD, PhD, Karolinska Institutet
Publications and helpful links
General Publications
- Henter JI, Horne A, Arico M, Egeler RM, Filipovich AH, Imashuku S, Ladisch S, McClain K, Webb D, Winiarski J, Janka G. HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 2007 Feb;48(2):124-31. doi: 10.1002/pbc.21039.
- Bergsten E, Horne A, Arico M, Astigarraga I, Egeler RM, Filipovich AH, Ishii E, Janka G, Ladisch S, Lehmberg K, McClain KL, Minkov M, Montgomery S, Nanduri V, Rosso D, Henter JI. Confirmed efficacy of etoposide and dexamethasone in HLH treatment: long-term results of the cooperative HLH-2004 study. Blood. 2017 Dec 21;130(25):2728-2738. doi: 10.1182/blood-2017-06-788349. Epub 2017 Sep 21.
- Bergsten E, Horne A, Hed Myrberg I, Arico M, Astigarraga I, Ishii E, Janka G, Ladisch S, Lehmberg K, McClain KL, Minkov M, Nanduri V, Rosso DA, Sieni E, Winiarski J, Henter JI. Stem cell transplantation for children with hemophagocytic lymphohistiocytosis: results from the HLH-2004 study. Blood Adv. 2020 Aug 11;4(15):3754-3766. doi: 10.1182/bloodadvances.2020002101.
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Lymphatic Diseases
- Histiocytosis, Non-Langerhans-Cell
- Histiocytosis
- Lymphohistiocytosis, Hemophagocytic
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Autonomic Agents
- Peripheral Nervous System Agents
- Enzyme Inhibitors
- Anti-Inflammatory Agents
- Antirheumatic Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Antiemetics
- Gastrointestinal Agents
- Glucocorticoids
- Hormones
- Hormones, Hormone Substitutes, and Hormone Antagonists
- Antineoplastic Agents, Hormonal
- Antineoplastic Agents, Phytogenic
- Topoisomerase II Inhibitors
- Topoisomerase Inhibitors
- Dermatologic Agents
- Antifungal Agents
- Calcineurin Inhibitors
- Dexamethasone
- Etoposide
- Cyclosporine
- Cyclosporins
Other Study ID Numbers
- HLH-2004
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hemophagocytic Lymphohistiocytosis
-
The First Affiliated Hospital with Nanjing Medical...Unknown
-
Children's Hospital Medical Center, CincinnatiBaylor College of Medicine; Sobi, Inc.RecruitingHemophagocytic LymphohistiocytosesUnited States
-
Swedish Orphan BiovitrumLight Chain Bioscience - Novimmune SATerminatedHemophagocytic LymphohistiocytosesUnited States
-
Swedish Orphan BiovitrumHarvard Medical School (HMS and HSDM); Brigham and Women's HospitalCompletedHemophagocytic LymphohistiocytosesUnited States
-
University of California, San FranciscoIncyte CorporationNot yet recruitingRuxolitinib With De-Intensified HLH-94 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH)Hemophagocytic LymphohistiocytosesUnited States
-
Beijing Friendship HospitalNot yet recruitingEBV | Hemophagocytic Lymphohistiocytoses | MetabonomicsChina
-
Beijing Friendship HospitalRecruitingHemophagocytic LymphohistiocytosisChina
-
St. Jude Children's Research HospitalIncyte Corporation; North American Consortium for Histiocytosis; Cures Within...RecruitingHemophagocytic LymphohistiocytosisUnited States
-
Swedish Orphan BiovitrumSeventh Framework ProgrammeCompletedHemophagocytic LymphohistiocytosisUnited States, France, Italy, Spain, United Kingdom
-
Children's Hospital Medical Center, CincinnatiCompletedHemophagocytic LymphohistiocytosisUnited States, Canada
Clinical Trials on Dexamethasone
-
Ottawa Hospital Research InstituteCompletedPain Syndrome | Early-stage Breast CancerCanada
-
Centre hospitalier de l'Université de Montréal...CompletedPrevention of Hypersensitivity Reactions to PaclitaxelCanada
-
Vanderbilt University Medical CenterTerminatedAsthma | CroupUnited States
-
Dr. Stephen ChoiThe Physicians' Services Incorporated FoundationCompletedShoulder Surgery | Nerve BlockCanada
-
Shanghai Jiao Tong University Affiliated Sixth...CompletedAnalgesia | Time | Brachial Plexus Block | Shoulder Surgery | Dexamethasone | Intravenous Drug UsageChina
-
Universidade Federal de PernambucoCompletedDiabetic Macular EdemaBrazil
-
Universitätsklinikum Hamburg-EppendorfGemeinsamer Bundesausschuss (G-BA); Staburo GmbHRecruiting
-
Poznan University of Medical SciencesRecruitingWrist Injuries | Hand Injuries | Hand Injuries and Disorders | Hand Disease | Wrist DiseasePoland
-
University of California, San FranciscoCompletedOral Lichen Planus | Pemphigus Vulgaris | Mucous Membrane Pemphigoid | Chronic Graft-versus-host-diseaseUnited States
-
University of BelgradeCompleted