NCT00723112

Brief Summary

The purpose of the study is to elucidate the causative molecular events responsible for the abnormal erythropoiesis in MDS.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Feb 2007

Typical duration for all trials

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2007

Completed
1.5 years until next milestone

First Submitted

Initial submission to the registry

July 24, 2008

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 28, 2008

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2010

Completed
Last Updated

November 23, 2010

Status Verified

November 1, 2010

Enrollment Period

3.7 years

First QC Date

July 24, 2008

Last Update Submit

November 22, 2010

Conditions

Myelodysplastic Syndrome

Keywords

Myelodysplastic SyndromeErythropoiesisIneffective HematopoiesisErythroid ProgenitorsDyserythropoiesis

Outcome Measures

Primary Outcomes (1)

  • Evaluate the EpoR cDNA sequence and its level of expression in the clonal erythroid progenitors of MDS patients to determine whether mutations in the EpoR may be responsible for an aberrant Epo signal transduction in MDS.

    After Samples are obtained

Secondary Outcomes (3)

  • Analyze intrinsic erythroid Epo expression to determine whether it differs between normal controls and patients with MDS.

    After samples are obtained

  • Perform a microarray analysis of genes associated with Epo signal transduction to determine if MDS patients have abnormal expression of signal transduction proteins.

    After samples are obtained

  • Determine how often and what percent clonality occurs in MDS patients and try to predict who has early MDS by clonality testing.

    After samples from female patients have been obtained

Study Arms (2)

Affected Group

Adult subjects with the diagnosis of MDS based on the French-American-British classification system.

Healthy Controls

Control subjects will be selected using frequency matching on gender and age by decade. That is for each MDS patient a healthy volunteer of the same gender and decade (50-59, 60-69, 70-79, etc) will be selected

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Subjects diagnosed with myelodysplastic syndrome

You may qualify if:

  • Adult subjects greater than 18 years of age
  • Diagnosis of MDS based on the French-American-British classification system (including secondary causes of MDS)

You may not qualify if:

  • Subjects not meeting the criteria listed above

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

University of Utah

Salt Lake City, Utah, 84132, United States

Location

VA Salt Lake City Health Care System

Salt Lake City, Utah, 84148, United States

Location

Related Publications (12)

  • Aul C, Arning M, Runde V, Schneider W. Serum erythropoietin concentrations in patients with myelodysplastic syndromes. Leuk Res. 1991;15(7):571-5. doi: 10.1016/0145-2126(91)90025-o.

    PMID: 1861540BACKGROUND

  • Stasi R, Brunetti M, Terzoli E, Abruzzese E, Amadori S. Once-weekly dosing of recombinant human erythropoietin alpha in patients with myelodysplastic syndromes unresponsive to conventional dosing. Ann Oncol. 2004 Nov;15(11):1684-90. doi: 10.1093/annonc/mdh428.

    PMID: 15520072BACKGROUND

  • Casadevall N, Durieux P, Dubois S, Hemery F, Lepage E, Quarre MC, Damaj G, Giraudier S, Guerci A, Laurent G, Dombret H, Chomienne C, Ribrag V, Stamatoullas A, Marie JP, Vekhoff A, Maloisel F, Navarro R, Dreyfus F, Fenaux P. Health, economic, and quality-of-life effects of erythropoietin and granulocyte colony-stimulating factor for the treatment of myelodysplastic syndromes: a randomized, controlled trial. Blood. 2004 Jul 15;104(2):321-7. doi: 10.1182/blood-2003-07-2252. Epub 2004 Mar 30.

    PMID: 15054036BACKGROUND

  • Solignac M; European Hematology Association. [Epoetin beta, new strategies to optimise the management of anaemia in cancer patients]. Presse Med. 2003 Sep 13;32(29):1385-8. No abstract available. French.

    PMID: 14534506BACKGROUND

  • Fontenay-Roupie M, Bouscary D, Guesnu M, Picard F, Melle J, Lacombe C, Gisselbrecht S, Mayeux P, Dreyfus F. Ineffective erythropoiesis in myelodysplastic syndromes: correlation with Fas expression but not with lack of erythropoietin receptor signal transduction. Br J Haematol. 1999 Aug;106(2):464-73. doi: 10.1046/j.1365-2141.1999.01539.x.

    PMID: 10460607BACKGROUND

  • Takeshita A, Shinjo K, Naito K, Ohnishi K, Higuchi M, Ohno R. Erythropoietin receptor in myelodysplastic syndrome and leukemia. Leuk Lymphoma. 2002 Feb;43(2):261-4. doi: 10.1080/10428190290006026.

    PMID: 11999556BACKGROUND

  • Shinjo K, Takeshita A, Higuchi M, Ohnishi K, Ohno R. Erythropoietin receptor expression on human bone marrow erythroid precursor cells by a newly-devised quantitative flow-cytometric assay. Br J Haematol. 1997 Mar;96(3):551-8. doi: 10.1046/j.1365-2141.1997.d01-2071.x.

    PMID: 9054663BACKGROUND

  • McMullin MF, Percy MJ. Erythropoietin receptor and hematological disease. Am J Hematol. 1999 Jan;60(1):55-60. doi: 10.1002/(sici)1096-8652(199901)60:13.0.co;2-v.

    PMID: 9883806BACKGROUND

  • Kralovics R, Sokol L, Broxson EH Jr, Prchal JT. The erythropoietin receptor gene is not linked with the polycythemia phenotype in a family with autosomal dominant primary polycythemia. Proc Assoc Am Physicians. 1997 Nov;109(6):580-5.

    PMID: 9394420BACKGROUND

  • Kralovics R, Indrak K, Stopka T, Berman BW, Prchal JF, Prchal JT. Two new EPO receptor mutations: truncated EPO receptors are most frequently associated with primary familial and congenital polycythemias. Blood. 1997 Sep 1;90(5):2057-61.

    PMID: 9292543BACKGROUND

  • Stopka T, Zivny JH, Stopkova P, Prchal JF, Prchal JT. Human hematopoietic progenitors express erythropoietin. Blood. 1998 May 15;91(10):3766-72.

    PMID: 9573013BACKGROUND

  • Sato TN. A new role of lipid receptors in vascular and cardiac morphogenesis. J Clin Invest. 2000 Oct;106(8):939-40. doi: 10.1172/JCI11304. No abstract available.

    PMID: 11032853BACKGROUND

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • Josef T Prchal, MD

    University of Utah

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER

Study Record Dates

First Submitted

July 24, 2008

First Posted

July 28, 2008

Study Start

February 1, 2007

Primary Completion

October 1, 2010

Study Completion

October 1, 2010

Last Updated

November 23, 2010

Record last verified: 2010-11

Locations

US(2)