Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

January 15, 2015 updated by: Charlotte Niemeyer, MD, University Hospital Freiburg

TCR Vbeta Repertoire and PNH Clones in Children With Refractory Cytopenia (RC). An Open Nonrandomised Multi-Center Prospective Study

RATIONALE: Studying biopsy, bone marrow, and blood samples from patients with cytopenia that did not respond to treatment may help doctors learn more about the disease and plan the best treatment.

PURPOSE: This laboratory study is assessing immune function in young patients with cytopenia that did not respond to treatment.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

OBJECTIVES:

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.

Study Type

Observational

Enrollment (Actual)

119

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Vienna, Austria, A-1090
        • St. Anna Children's Hospital
  • Belgium
      • Ghent, Belgium, B-9000
        • Ghent University
  • Czech Republic
      • Prague, Czech Republic, 150 06
        • University Hospital Motol
  • Denmark
      • Aarhus, Denmark, 8200
        • Arhus Universitetshospital - Skejby
  • Germany
      • Freiburg, Germany, D-79106
        • Universitaetskinderklinik - Universitaetsklinikum Freiburg
  • Ireland
      • Dublin, Ireland, 12
        • Our Lady´s Hospital for Sick Children
  • Italy
      • Pavia, Italy, 27100
        • Fondazione I.R.C.C.S. Policlinico San Matteo
  • Netherlands
      • Rotterdam, Netherlands, 3015 GJ
        • Erasmus MC - Sophia Children's Hospital
  • Spain
      • Barcelona, Spain, 08950
        • Hospital Sant Joan de Déu
  • Switzerland
      • Zurich, Switzerland, CH-8032
        • University Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 17 years (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

All patients with MDS

Description

DISEASE CHARACTERISTICS:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of patients with TCR V beta oligoclonality at diagnosis
Time Frame: 96 months
96 months
Immunophenotype of patients with oligoclonal T-cell expansion
Time Frame: 96 months
96 months
Number of patients with glycophosphatidylinositol (GPI) deficient clones
Time Frame: 96 months
96 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Number of patients with molecular response as compared to hematological response after IST
Time Frame: 96 months
96 months
Number of patients with HLA-DR15 antigen expression and molecular response as compared to number of patients with other HLA-DR antigens and molecular response
Time Frame: 96 months
96 months
Overall survival
Time Frame: 96 months
96 months
Failure-free survival
Time Frame: 96 months
96 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital Freiburg

Investigators

  • Study Chair: Marry M. Van Den Heuvel-Eibrink, MD, PhD, Erasmus MC - Sophia Children's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2007

Primary Completion (ACTUAL)

August 1, 2012

Study Completion (ACTUAL)

August 1, 2012

Study Registration Dates

First Submitted

July 10, 2007

First Submitted That Met QC Criteria

July 10, 2007

First Posted (ESTIMATE)

July 11, 2007

Study Record Updates

Last Update Posted (ESTIMATE)

January 16, 2015

Last Update Submitted That Met QC Criteria

January 15, 2015

Last Verified

January 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CDR0000553058
  • EWOG-MDS-RC-06 (OTHER: University Hospital Freiburg)

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.

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