Serial Analysis of Chimerism in Patients With Refractory Cytopenia (RC) Transplanted With Reduced Intensity Conditioning (RIC)

NCT00682799 | Completed

• 1 other identifier: EWOG MDS SCT RC RIC-06
• Study Type: observational
• Target: 112
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective, non-randomized multi-center multi-national study to evaluate the chimerism measured by STR and SNP in patients with hypoplastic RC and normal karyotype transplanted with a preparative regimen of reduced intensity. Primary objectives:

• To study hematopoietic chimerism in whole blood and different cell population (CD14, CD15, CD 56, CD3, CD19) as well as in dendritic cells and regulatory T-cells after SCT with RIC in patients with RC
• To compare the results of chimerism obtained with standard STR PCR (sensitivity 1%) with those obtained with SNP PCR (sensitivity 0.1- 0.01%) Secondary objectives:
• To evaluate the relationship between mixed chimerism and hematological engraftment, OS and EFS
• To study the impact of mixed chimerism in plasmacytoid dendritic and regulatory T-cells on the incidence of acute and chronic GVHD

Conditions

Refractory Cytopenia of Childhood; Reduced Intensity Conditioning (RIC); Myelodysplastic Syndrome (MDS); Hematopoietic Stem Cell Transplantation (SCT)

Keywords

RC; RIC; MDS; SCT

Outcome Measures

Primary Outcomes (2)

• To study hematopoietic chimerism in whole blood and different cell population (CD14, CD15, CD 56, CD3, CD19) as well as in dendritic cells and regulatory T-cells after SCT with RIC in patients with RC

  5 years

• To compare the results of chimerism obtained with standard STR PCR (sensitivity 1%) with those obtained with SNP PCR (sensitivity 0.1- 0.01%)

  5 years

Secondary Outcomes (2)

• To evaluate the relationship between mixed chimerism and hematological engraftment, OS and EFS

  5 years

• To study the impact of mixed chimerism in plasmacytoid dendritic and regulatory T-cells on the incidence of acute and chronic GVHD

  5 years

Eligibility Criteria

• Age: Up to 215 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Patients will only be allowed to enter the trial if they or their caretakers provide written informed consent about their participation (following full explanation of the trial) and if the physician has verified that the patient meets all of the Inclusion Criteria and none of the Exclusion Criteria.

You may qualify if:

• RC Patients with hypocellular BM normal karyotype included in the EWOG-MDS 2006 protocol who receive SCT from a MFD or a compatible (8/8) or one allelic mismatch UD
• Written informed consent by the caretakers and whenever possible the patient's assent.
• Age less than 18 years The caretakers will have given their written informed consent to participate in the study. Consent will be documented by the caretaker's dated signature which will be also signed and dated by the investigator in the participating center. If the patient is able to understand the meaning and consequences of the study and its procedures his/her written informed assent is also needed. Written informed consent has to be obtained prior to enrollment into the study.

You may not qualify if:

• Transplanted with a preparative regimen other than thiotepa, fludarabine

Sponsors & Collaborators

• Charlotte Niemeyer, MD: lead

Study Sites (1)

University Children´s Hospital

Frankfurt am Main, Hesse, 60590, Germany

Location

Related Links

• Homepage of the EWOG-MDS-Study Group

Biospecimen

Retention: SAMPLES WITH DNA

Hematopoietic chimerism will be investigated from PB samples. Prior to transplant 5 ml EDTA PB from patient and donor are required and will be sent to the laboratory of the Coordinating Investigator (P.B.). Post transplant PB samples (5 -10 ml EDTA blood) from day +30 (4 days), +60(4 days), +100 (7 days) and +180 (7 days) will also being sent to the laboratory of the Coordinating Investigator (P.B.) From EDTA PB cell subpopulations will be isolated and DNA will be extracted and stored for further investigations.

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Peter Bader, M.D.

  University Children´s Hospital Frankfurt am Main

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Prof. Dr. Charlotte Niemeyer, MD

Study Record Dates

• First Submitted: May 16, 2008
• First Posted: May 22, 2008
• Study Start: April 1, 2007
• Primary Completion: March 1, 2013
• Study Completion: March 1, 2013
• Last Updated: January 15, 2015

Record last verified: 2015-01

Locations

DE (1)