Chemotherapy in Treating Patients With Myelodysplastic Syndrome Before Donor Stem Cell Transplant

NCT01812252 | Completed Phase 2 Results FDA Drug

• 4 other identifiers: 2661.00NCI-2013-005382661RG9215001
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 4

Brief Summary

This randomized clinical trial studies different chemotherapies in treating patients with myelodysplastic syndrome before donor stem cell transplant. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells, and may prevent the myelodysplastic syndrome from coming back after the transplant. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.

Conditions

Chronic Myelomonocytic Leukemia; de Novo Myelodysplastic Syndrome; Myelodysplastic Syndrome; Secondary Myelodysplastic Syndrome

Keywords

myelodysplastic syndrome; chronic myelomonocytic leukemia; bone marrow transplant; hypomethylating agent; induction chemotherapy

Outcome Measures

Primary Outcomes (1)

• Failure-free Survival (Failure Defined as Death or Relapse)

  18-month failure-free survival (failure defined as death or relapse).

  18 months

Secondary Outcomes (5)

• Quality of Life Will be Assessed Using the European Organization for Research and Treatment of Cancer Quality of Life (QoL) Questionnaire (EORTC QLQ-C30) Questionnaire.

  EORTC QLQ-C30 questionnaire will be collected at screening, after completion of therapy (HMA 4-6 month, and up to 6 months for induction-like chemotherapy) just prior to stem cell infusion and 100 (± 14) days after stem cell infusion (HSCT).

• Quality of Life Will be Assessed Using the EORTC QLQ-HDC29 a Supplementary Module Assessing the Quality of Life During and After High-dose Chemotherapy and Stem Cell Transplantation.

  EORTC QLQ-HDC29 questionnaire will be collected after completion of therapy (HMA 4-6 month, and up to 6 months for induction-like chemotherapy) pre stem cell infusion (HSCT), and 100 (± 14) days post stem cell infusion (HSCT).

• Overall Survival

  Up to 18 months

• Number of Patients Who Relapse Post-transplant

  Up to 18 months

• Number of Participants Who Received a Hematopoietic Cell Transplantation (HCT).

  Up to 18 months

Study Arms (2)

Arm A (decitabine or azacitidine)

OTHER

Patients receive decitabine or azacitidine IV or SC per standard of care. Treatment repeats per standard of care, every 28 days for 4 cycles of decitabine or 6 cycles of azacitidine in the absence of disease progression or unacceptable toxicity.

Drug: Azacitidine (AZC); Drug: Decitabine; Other: Quality-of-Life Assessment

Arm B (induction-like chemotherapy regimen)

OTHER

Patients receive physician choice of standard of care or other experimental protocol using induction-like chemotherapy regimen. No one specific regimen is required. Several regimens are listed in the protocol for example only.

Other: Quality-of-Life Assessment

Interventions

Azacitidine (AZC) DRUG

Given IV or SC

Also known as: 5 AZC, 5-AC, 5-Azacytidine, 5-AZC, AZACITIDINE, Azacytidine, Azacytidine, 5-, Ladakamycin, Mylosar, U-18496, Vidaza

Arm A (decitabine or azacitidine)

Decitabine DRUG

Given IV or SC

Also known as: 5-Aza-2'-deoxycytidine, Dacogen, Decitabine for Injection, Deoxyazacytidine, Dezocitidine

Arm A (decitabine or azacitidine)

Quality-of-Life Assessment OTHER

Ancillary studies

Also known as: Quality of Life Assessment

Arm A (decitabine or azacitidine); Arm B (induction-like chemotherapy regimen)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of de novo or secondary myelodysplastic syndrome (MDS), including chronic myelomonocytic leukemia, as defined by the 2008 World Health Organization classification system
• Patients must have measurable disease requiring cytoreduction, defined as a bone marrow myeloblast count \>= 5% and \< 20% on morphologic examination or by flow cytometry in cases in which adequate morphologic examination is not possible
• Considered a potential transplant candidate; the attending/treating physician will determine transplant candidacy at the time of consent
• Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent

You may not qualify if:

• A diagnosis of acute promyelocytic leukemia as defined by the 2008 World Health Organization classification system
• Previous treatment for MDS or acute myeloblastic leukemia (AML) with intensive chemotherapy regimen (induction chemotherapy) or hypomethylating agent
• Have any other severe concurrent disease, or have a history of serious organ dysfunction or disease involving the heart, kidney, liver, or other organ system that may place the patient at undue risk to undergo treatment
• Patients with a systemic fungal, bacterial, viral, or other infection not controlled (defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment)
• Females who are pregnant or breastfeeding
• Fertile men and women unwilling to use contraceptive techniques during and for 12 months following treatment
• Any uncontrolled or significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results
• Clinical evidence suggestive of central nervous system (CNS) involvement with MDS unless a lumbar puncture confirms the absence of leukemic blasts in the cerebrospinal fluid (CSF)

Sponsors & Collaborators

• Fred Hutchinson Cancer Center: lead

Study Sites (4)

Mayo Clinic in Arizona

Scottsdale, Arizona, 85259, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States

Location

Kaiser Permanente Washington

Seattle, Washington, 98112, United States

Location

Related Publications (16)

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• Field T, Perkins J, Huang Y, Kharfan-Dabaja MA, Alsina M, Ayala E, Fernandez HF, Janssen W, Lancet J, Perez L, Sullivan D, List A, Anasetti C. 5-Azacitidine for myelodysplasia before allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 2010 Feb;45(2):255-60. doi: 10.1038/bmt.2009.134. Epub 2009 Jun 22.

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• Gerds AT, Gooley TA, Estey EH, Appelbaum FR, Deeg HJ, Scott BL. Pretransplantation therapy with azacitidine vs induction chemotherapy and posttransplantation outcome in patients with MDS. Biol Blood Marrow Transplant. 2012 Aug;18(8):1211-8. doi: 10.1016/j.bbmt.2012.01.009. Epub 2012 Jan 16.

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MeSH Terms

Conditions

Leukemia, Myelomonocytic, Chronic; Myelodysplastic Syndromes

Interventions

Azacitidine; Decitabine; Injections

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Drug Administration Routes; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Bart Lee Scott M.D.
• Organization: Fred Hutch Cancer Center

bscott@fredhutch.org

(206)909-0977

Study Officials

• Bart L. Scott

  Fred Hutch/University of Washington Cancer Consortium

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: March 14, 2013
• First Posted: March 18, 2013
• Study Start: August 19, 2013
• Primary Completion: October 26, 2022
• Study Completion: October 26, 2022
• Last Updated: February 15, 2024
• Results First Posted: February 15, 2024

Record last verified: 2024-01

Locations

US (4)