Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
NCT ID: NCT00499070
Last Updated: 2015-01-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
119 participants
OBSERVATIONAL
2007-01-31
2012-08-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
PURPOSE: This laboratory study is assessing immune function in young patients with cytopenia that did not respond to treatment.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma
NCT00900055
Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants
NCT00896740
CHAPTER: Clonal Haematopoiesis Assessment: Prevention, Treatment and Research
NCT07313059
Collection of Blood and Bone Marrow From Patients With Aplastic Anemia for Analysis of Adhesion Molecules, Chemokines and Their Receptors
NCT01680055
Blood Cell Collection for Future Use in Individuals With Fanconi Anemia
NCT00271089
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Primary
* To evaluate the value of TCR V beta repertoire analysis for the determination of autoimmunity in refractory cytopenia (RC).
* To evaluate which immunophenotypic hematopoietic subclones are associated with oligoclonal T-cell expansion in RC.
* To evaluate the presence of paroxysmal nocturnal hemoglobinuria (PNH) clones in RC.
Secondary
* To compare the molecular response with the hematologic response in patients with RC after treatment with immunosuppressive therapy (IST).
* To compare the molecular response with human leukocyte histocompatability antigen (HLA) expression in patients with RC after treatment with IST.
OUTLINE: This is an open-label, multicenter, nonrandomized, prospective study.
Patients undergo biopsy, bone marrow, and blood sample collection periodically for immunological studies. Samples are analyzed for TCR V beta repertoire and paroxysmal nocturnal hemoglobinuria (PNH) clone analysis via PCR heteroduplex analysis and immunophenotyping of CD14, CD16 , CD55, CD59, and CD24 expression via flow cytometry.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
COHORT
PROSPECTIVE
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
polymerase chain reaction
flow cytometry
For analyzing GPI deficient clones full blood will be analyzed by phenotyping using flowcytometry. For that purpose CD14, CD16 and CD24 expression will be evaluated in CD45 positive cells. Erythroid cells will be evaluated for CD55 and CD59 expression searching for clear populations with a lack of GPI-linked molecules. In addition, immunophenotyping using flowcytometry will be performed to evaluate which differentiation stages of the major hematopoietic lineages in BM and PB are associated with TCRVβ repertoire skewing. Comparison between BM and PB will identify which is the optimal compartment to analyze the responsible hematopoietic clones.
immunologic technique
biopsy
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Diagnosis of refractory cytopenia (RC) including any of the following:
* Severe aplastic anemia (SAA)
* Fanconi's anemia
* Shwachman Diamond syndrome
* Dyskeratosis congenita
* Pearson syndrome
* All RC patients included in the EWOG MDS 2006 protocol irrespective of therapy
* Patients who have undergone hematopoietic stem cell transplantation (HSCT) may be enrolled on EWOG-MDS SCT RC RIC 06 or EWOG-MDS SCT MDS 06 protocol
PATIENT CHARACTERISTICS:
* Not specified
PRIOR CONCURRENT THERAPY:
* No prior immunosuppressive therapy for refractory cytopenia
17 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
University Hospital Freiburg
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Charlotte Niemeyer, MD
MD Prof. Dr. med. Niemeyer
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Marry M. Van Den Heuvel-Eibrink, MD, PhD
Role: STUDY_CHAIR
Erasmus MC-Sophia Children's Hospital
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
St. Anna Children's Hospital
Vienna, , Austria
Ghent University
Ghent, , Belgium
University Hospital Motol
Prague, , Czechia
Arhus Universitetshospital - Skejby
Aarhus, , Denmark
Universitaetskinderklinik - Universitaetsklinikum Freiburg
Freiburg im Breisgau, , Germany
Our Lady´s Hospital for Sick Children
Dublin, , Ireland
Fondazione I.R.C.C.S. Policlinico San Matteo
Pavia, , Italy
Erasmus MC - Sophia Children's Hospital
Rotterdam, , Netherlands
Hospital Sant Joan de Deu
Barcelona, , Spain
University Children's Hospital
Zurich, , Switzerland
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Aalbers AM, van den Heuvel-Eibrink MM, Baumann I, Dworzak M, Hasle H, Locatelli F, De Moerloose B, Schmugge M, Mejstrikova E, Novakova M, Zecca M, Zwaan CM, Te Marvelde JG, Langerak AW, van Dongen JJ, Pieters R, Niemeyer CM, van der Velden VH. Bone marrow immunophenotyping by flow cytometry in refractory cytopenia of childhood. Haematologica. 2015 Mar;100(3):315-23. doi: 10.3324/haematol.2014.107706. Epub 2014 Nov 25.
Aalbers AM, van den Heuvel-Eibrink MM, Baumann I, Beverloo HB, Driessen GJ, Dworzak M, Fischer A, Gohring G, Hasle H, Locatelli F, De Moerloose B, Noellke P, Schmugge M, Stary J, Yoshimi A, Zecca M, Zwaan CM, van Dongen JJ, Pieters R, Niemeyer CM, van der Velden VH, Langerak AW. T-cell receptor Vbeta skewing frequently occurs in refractory cytopenia of childhood and is associated with an expansion of effector cytotoxic T cells: a prospective study by EWOG-MDS. Blood Cancer J. 2014 May 2;4(5):e209. doi: 10.1038/bcj.2014.28.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
EWOG-MDS-RC-06
Identifier Type: OTHER
Identifier Source: secondary_id
CDR0000553058
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.