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Chronic Myelomonocytic LeukemiaPhase 2ICT-HCTNCT01812252

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

A Phase 2 study testing Azacitidine (AZC), Decitabine and Quality-of-Life Assessment in people with Chronic Myelomonocytic Leukemia and de Novo Myelodysplastic Syndrome, run by Fred Hutchinson Cancer Center at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Azacitidine (AZC), Decitabine and Quality-of-Life Assessment
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
4 sitesUnited States
Study dates
19 Aug 2013to 26 Oct 2022
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
  • Patients must be considered to have an acceptable risk of early mortality with intensive chemotherapy as determined by the attending physician at the time of the initial visit; since the specific therapy within each arm will be determined after randomization, there is no threshold of organ dysfunction or performance status for inclusion
  • 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

Who cannot join

  • 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)

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • Mayo Clinic in Arizona

    Scottsdale, Arizona, 85259

  • Fred Hutch/University of Washington Cancer Consortium

    Seattle, Washington, 98109

  • Kaiser Permanente Washington

    Seattle, Washington, 98112

Other trials in United States

About this study

Official title: Initial Cytoreductive Therapy for Myelodysplastic Syndrome Prior to Allogeneic Hematopoietic Cell Transplantation (the ICT-HCT Study)

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.

Read the detailed description

OUTLINE: Patients are randomized to 1 of 2 treatment arms.

Arm A: Patients receive decitabine or azacitidine intravenously (IV) or subcutaneously (SC) for 7 days. Treatment repeats every 28 days for 4 cycles of decitabine or 6 cycles of azacitidine in the absence of disease progression or unacceptable toxicity.

Arm B: Patients receive induction-like chemotherapy per standard of care or per experimental protocol. This study does not require a specific chemotherapy regimen for Arm B.

After completion of study treatment, patients are followed up for 18 months.

Being tested
Azacitidine (AZC), Decitabine, Quality-of-Life Assessment
Keywords
myelodysplastic syndrome, chronic myelomonocytic leukemia, bone marrow transplant, hypomethylating agent, induction chemotherapy
Medical subject headings
Leukemia, Myelomonocytic, Chronic, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Azacitidine (AZC)Drug

    Also called 5 AZC, 5-AC, 5-Azacytidine, 5-AZC

    Given IV or SC

  • DecitabineDrug

    Also called 5-Aza-2'-deoxycytidine, Dacogen, Decitabine for Injection, Deoxyazacytidine

    Given IV or SC

  • Quality-of-Life AssessmentOther

    Also called Quality of Life Assessment

    Ancillary studies

Study groups in NCT01812252
Study groupRoleWhat they receive
Arm A (decitabine or azacitidine)OtherPatients 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.
Receives: Drug: Azacitidine (AZC); Drug: Decitabine; Other: Quality-of-Life Assessment
Arm B (induction-like chemotherapy regimen)OtherPatients 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.
Receives: Other: Quality-of-Life Assessment

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Failure-free Survival (Failure Defined as Death or Relapse)

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

    Measured: 18 months

Secondary outcomes (5)

  1. 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.

    The European Organization for Research and Treatment of Cancer Quality of Life (EORTC QLQ-C30) (version 3) is a 30-item cancer-specific questionnaire for measuring the health-related quality of life (QOL) in cancer patients. It includes five functioning scales (physical, PF; role, RF; cognitive, CF; emotional, EF; and social, SF), three symptom scales (fatigue, FA; pain, PA; and nausea and vomiting, NV), a global health status/QOL scale (GL), and six single items (dyspnea, appetite loss, sleep disturbance, constipation, diarrhea, and financial impact of the disease and treatment). All items employ a 4-point Likert scale, ranging from 1 (not at all) to 4 (very much) with lower scores representing a better outcome and higher scores a worse outcome; with the exception of two items in the GL scale, which use 7-point scales (1=very poor to 7=excellent) lower scores representing a worse outcome and higher score representing a better outcome.

    Measured: 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).

  2. 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.

    European Organization for Research and Treatment of Cancer Quality of Life Questionnaire High Dose Chemotherapy (EORTC QLQ-HDC29) is a treatment-specific quality of life questionnaire that addresses treatment specific side effects as well as emotional, social, and family issues for patients treated with high dose regimens and HCT. The QLQ-HDC29 module includes 29 items, consisting of 6 multi-item scales and 8 single-items; all items employ a 4-point Likert scale, ranging from 1 (not at all) to 4 (very much) with lower scores representing a better outcome and higher scores a worse outcome with the exception of question 47 and 52 being the a higher score resulting in a better outcome and lower score a worse outcome.

    Measured: 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).

  3. Overall Survival

    The total length of follow-up will be 18 months from the start of treatment (day 1). Four categories were added for participants alive 18 months from start of treatment (day 1) 1) Participants alive after 18 months from start of treatment who received hematopoietic cell transplantation (HCT); 2) Participants alive after 18months from start of treatment who did not receive HCT.; 3) Participants deceased after 18 months from start of treatment who received HCT; 4) Participants deceased after 18 months from start of treatment who did not receive HCT.

    Measured: Up to 18 months

  4. Number of Patients Who Relapse Post-transplant

    To compare which of the two, intensive chemotherapy versus hypomethylating agent-based therapy, have a factor of relapse post hematopoietic cell transplantation (HCT).

    Measured: Up to 18 months

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

    Frequency at which the participants received a hematopoietic cell transplantation (HCT)

    Measured: Up to 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Mar 2013 (estimated)
  2. Study start19 Aug 2013
  3. Primary completion26 Oct 2022
  4. Study completion26 Oct 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01812252
Sponsor's protocol ID
2661.00
Other IDs
NCI-2013-00538, 2661, 2661.00, RG9215001
Acronym
ICT-HCT
Status last verified
Jan 2024
First submitted
14 Mar 2013
Last updated
15 Feb 2024
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, Sanz M, Vallespi T, Hamblin T, Oscier D, Ohyashiki K, Toyama K, Aul C, Mufti G, Bennett J. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997 Mar 15;89(6):2079-88.

    PubMed 9058730

  • Gooley TA, Chien JW, Pergam SA, Hingorani S, Sorror ML, Boeckh M, Martin PJ, Sandmaier BM, Marr KA, Appelbaum FR, Storb R, McDonald GB. Reduced mortality after allogeneic hematopoietic-cell transplantation. N Engl J Med. 2010 Nov 25;363(22):2091-101. doi: 10.1056/NEJMoa1004383.

    PubMed 21105791

  • Deeg HJ, Storer B, Slattery JT, Anasetti C, Doney KC, Hansen JA, Kiem HP, Martin PJ, Petersdorf E, Radich JP, Sanders JE, Shulman HM, Warren EH, Witherspoon RP, Bryant EM, Chauncey TR, Getzendaner L, Storb R, Appelbaum FR. Conditioning with targeted busulfan and cyclophosphamide for hemopoietic stem cell transplantation from related and unrelated donors in patients with myelodysplastic syndrome. Blood. 2002 Aug 15;100(4):1201-7. doi: 10.1182/blood-2002-02-0527.

    PubMed 12149198

  • Lim Z, Brand R, Martino R, van Biezen A, Finke J, Bacigalupo A, Beelen D, Devergie A, Alessandrino E, Willemze R, Ruutu T, Boogaerts M, Falda M, Jouet JP, Niederwieser D, Kroger N, Mufti GJ, De Witte TM. Allogeneic hematopoietic stem-cell transplantation for patients 50 years or older with myelodysplastic syndromes or secondary acute myeloid leukemia. J Clin Oncol. 2010 Jan 20;28(3):405-11. doi: 10.1200/JCO.2009.21.8073. Epub 2009 Dec 14.

    PubMed 20008642

  • Warlick ED, Cioc A, Defor T, Dolan M, Weisdorf D. Allogeneic stem cell transplantation for adults with myelodysplastic syndromes: importance of pretransplant disease burden. Biol Blood Marrow Transplant. 2009 Jan;15(1):30-8. doi: 10.1016/j.bbmt.2008.10.012.

    PubMed 19135940

  • Nakai K, Kanda Y, Fukuhara S, Sakamaki H, Okamoto S, Kodera Y, Tanosaki R, Takahashi S, Matsushima T, Atsuta Y, Hamajima N, Kasai M, Kato S. Value of chemotherapy before allogeneic hematopoietic stem cell transplantation from an HLA-identical sibling donor for myelodysplastic syndrome. Leukemia. 2005 Mar;19(3):396-401. doi: 10.1038/sj.leu.2403640.

    PubMed 15674354

  • Scott BL, Storer B, Loken MR, Storb R, Appelbaum FR, Deeg HJ. Pretransplantation induction chemotherapy and posttransplantation relapse in patients with advanced myelodysplastic syndrome. Biol Blood Marrow Transplant. 2005 Jan;11(1):65-73. doi: 10.1016/j.bbmt.2004.10.001.

    PubMed 15625546

  • Yakoub-Agha I, de La Salmoniere P, Ribaud P, Sutton L, Wattel E, Kuentz M, Jouet JP, Marit G, Milpied N, Deconinck E, Gratecos N, Leporrier M, Chabbert I, Caillot D, Damaj G, Dauriac C, Dreyfus F, Francois S, Molina L, Tanguy ML, Chevret S, Gluckman E. Allogeneic bone marrow transplantation for therapy-related myelodysplastic syndrome and acute myeloid leukemia: a long-term study of 70 patients-report of the French society of bone marrow transplantation. J Clin Oncol. 2000 Mar;18(5):963-71. doi: 10.1200/JCO.2000.18.5.963.

    PubMed 10694545

  • Estey E, de Lima M, Tibes R, Pierce S, Kantarjian H, Champlin R, Giralt S. Prospective feasibility analysis of reduced-intensity conditioning (RIC) regimens for hematopoietic stem cell transplantation (HSCT) in elderly patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). Blood. 2007 Feb 15;109(4):1395-400. doi: 10.1182/blood-2006-05-021907. Epub 2006 Oct 12.

    PubMed 17038533

  • De Padua Silva L, de Lima M, Kantarjian H, Faderl S, Kebriaei P, Giralt S, Davisson J, Garcia-Manero G, Champlin R, Issa JP, Ravandi F. Feasibility of allo-SCT after hypomethylating therapy with decitabine for myelodysplastic syndrome. Bone Marrow Transplant. 2009 Jun;43(11):839-43. doi: 10.1038/bmt.2008.400. Epub 2009 Jan 19.

    PubMed 19151791

  • 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.

    PubMed 19543327

  • Lubbert M, Bertz H, Ruter B, Marks R, Claus R, Wasch R, Finke J. Non-intensive treatment with low-dose 5-aza-2'-deoxycytidine (DAC) prior to allogeneic blood SCT of older MDS/AML patients. Bone Marrow Transplant. 2009 Nov;44(9):585-8. doi: 10.1038/bmt.2009.64. Epub 2009 Apr 13.

    PubMed 19363531

  • 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.

    PubMed 22252125

  • Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.

    PubMed 19230772

  • Lowenberg B, Ossenkoppele GJ, van Putten W, Schouten HC, Graux C, Ferrant A, Sonneveld P, Maertens J, Jongen-Lavrencic M, von Lilienfeld-Toal M, Biemond BJ, Vellenga E, van Marwijk Kooy M, Verdonck LF, Beck J, Dohner H, Gratwohl A, Pabst T, Verhoef G; Dutch-Belgian Cooperative Trial Group for Hemato-Oncology (HOVON); German AML Study Group (AMLSG); Swiss Group for Clinical Cancer Research (SAKK) Collaborative Group. High-dose daunorubicin in older patients with acute myeloid leukemia. N Engl J Med. 2009 Sep 24;361(13):1235-48. doi: 10.1056/NEJMoa0901409.

    PubMed 19776405

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Azacitidine (AZC), Decitabine and Quality-of-Life Assessment in people with Chronic Myelomonocytic Leukemia and de Novo Myelodysplastic Syndrome, run by Fred Hutchinson Cancer Center at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Failure-free Survival (Failure Defined as Death or Relapse) (time frame: 18 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01812252, which also lists 16 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Feb 2024.

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