Biomarkers in Samples From Patients With Down Syndrome and Acute Megakaryoblastic Leukemia

This study is ongoing, but not recruiting participants.
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group Identifier:
First received: June 5, 2010
Last updated: May 4, 2015
Last verified: May 2015

RATIONALE: Studying samples of blood or tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.

PURPOSE: This research study is studying biomarkers in samples from patients with Down syndrome and acute megakaryoblastic leukemia.

Condition Intervention
Other: cell culture procedure
Other: laboratory biomarker analysis

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Development of Novel Therapeutics for AMKL

Resource links provided by NLM:

Further study details as provided by Children's Oncology Group:

Primary Outcome Measures:
  • Proliferation arrest [ Designated as safety issue: No ]
  • Polyploidization [ Designated as safety issue: No ]
  • Terminal differentiation [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: July 2009
Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Detailed Description:


  • Determine whether megakaryocyte differentiation agents comprising a Rho kinase inhibitor and a JAK3 inhibitor can lead to proliferation arrest, polyploidization, and terminal differentiation of blasts in specimens from patients with Down syndrome and acute megakaryoblastic leukemia.

OUTLINE: Cryopreserved specimens are cultured and treated in vivo (in mice) and in vitro with megakaryocytic differentiation agents comprising a Rho kinase inhibitor and a JAK3 inhibitor.


Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Diagnosis of acute megakaryoblastic leukemia or diagnosis of Down syndrome when cryopreserved specimens are available.


  • Diagnosis of acute megakaryoblastic leukemia
  • Diagnosis of Down syndrome
  • Cryopreserved specimens available


  • Not specified


  • Not specified
  Contacts and Locations
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Please refer to this study by its identifier: NCT01139307

Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
Principal Investigator: John Crispino, PhD Robert H. Lurie Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: Children's Oncology Group Identifier: NCT01139307     History of Changes
Other Study ID Numbers: AAML10B1, COG-AAML10B1, CDR0000671427, NCI-2011-02218
Study First Received: June 5, 2010
Last Updated: May 4, 2015
Health Authority: United States: Federal Government

Keywords provided by Children's Oncology Group:
adult acute megakaryoblastic leukemia (M7)
childhood acute megakaryocytic leukemia (M7)
recurrent adult acute myeloid leukemia
recurrent childhood acute myeloid leukemia
untreated adult acute myeloid leukemia
untreated childhood acute myeloid leukemia and other myeloid malignancies

Additional relevant MeSH terms:
Leukemia, Megakaryoblastic, Acute
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type processed this record on November 25, 2015