Trial document





This study has been imported from ClinicalTrials.gov without additional data checks.
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  DRKS00001397

Trial Description

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Title

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

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Trial Acronym

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URL of the Trial

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Brief Summary in Lay Language

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.

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Brief Summary in Scientific Language

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.

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Do you plan to share individual participant data with other researchers?

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Description IPD sharing plan:

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Organizational Data

  •   DRKS00001397
  •   2012/11/19
  •   2007/07/10
  •   yes
  •   Approved
  •   178/06, Ethik-Kommission der Albert-Ludwigs-Universität Freiburg
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Secondary IDs

  •   NCT00499070  (ClinicalTrials.gov)
  •   UKF000857  (Register klinischer Studien des Universitätsklinikums Freiburg)
  •   CDR0000553058  (University Hospital Freiburg)
  •   EWOG-MDS-RC-06 
  •   EU-20733 
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Health Condition or Problem studied

  •   Dyskeratosis Congenita
  •   Fanconi Anemia
  •   Myelodysplastic Syndromes
  •   Nonmalignant Neoplasm
  •   Pearson Marrow-pancreas Syndrome
  •   Shwachman-diamond Syndrome
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Interventions/Observational Groups

  •   Genetic: polymerase chain reaction
  •   Other: flow cytometry
  •   Other: immunologic technique
  •   Procedure: biopsy
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Characteristics

  •   Non-interventional
  •   Observational study
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  •   N/A
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Primary Outcome

- Number of patients with TCR V beta oligoclonality at diagnosis; time frame: 96 months
- Immunophenotype of patients with oligoclonal T-cell expansion; time frame: 96 months
- Number of patients with glycophosphatidylinositol (GPI) deficient clones; time frame: 96 months

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Secondary Outcome

- Number of patients with molecular response as compared to hematological response after IST; time frame: 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
- Overall survival; time frame: 96 months
- Failure-free survival; time frame: 96 months

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Countries of Recruitment

  •   Austria
  •   Belgium
  •   Czech Republic
  •   Denmark
  •   Germany
  •   Ireland
  •   Italy
  •   Netherlands
  •   Spain
  •   Switzerland
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Locations of Recruitment

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Recruitment

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  •   2007/01/31
  •   125
  •   Multicenter trial
  •   International
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Inclusion Criteria

  •   Both, male and female
  •   no minimum age
  •   17   Years
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Additional Inclusion Criteria

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

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Exclusion Criteria

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Addresses

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    • University Hospital Freiburg
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    • Erasmus MC - Sophia Children's Hospital
    • Marry M. Van Den Heuvel-Eibrink, MD, PhD 
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    • Erasmus MC - Sophia Children's Hospital
    • Marry M. Van Den Heuvel-Eibrink, MD, PhD 
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Sources of Monetary or Material Support

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Status

  •   Recruiting ongoing
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Trial Publications, Results and other Documents

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The parameters in ClinicalTrials.gov and DRKS are not identical. Therefore the data import from ClinicalTrials.gov required adjustments. For full details please see the DRKS FAQs .
  •   7
  •   2013/10/30
* This entry means the parameter is not applicable or has not been set.