NCT01812252 · REGISTRY CONDITION SEARCH

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

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE2
Status at capture: COMPLETED
Registry last update: 2024-02-15

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

→

baseline

Baseline characteristics

→

outcome · POSTED

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

→

outcome · POSTED

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.

→

outcome · POSTED

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.

→

outcome · POSTED

Overall Survival

→

outcome · POSTED

Number of Patients Who Relapse Post-transplant

→

outcome · POSTED

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

→

adverse events

Adverse events

→

more info

Limitations, agreements, and source contact

→

Who could take part

eligibility Criteria: Inclusion Criteria: * 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 Exclusion Criteria: * 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)
healthy Volunteers: false
minimum Age: 18 Years
sex: ALL
std Ages: ADULT
OLDER_ADULT

Treatment arms and interventions

arm Groups

description
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.
intervention Names
Drug: Azacitidine (AZC)
Drug: Decitabine
Other: Quality-of-Life Assessment
label
Arm A (decitabine or azacitidine)
type
OTHER
description
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.
intervention Names
Other: Quality-of-Life Assessment
label
Arm B (induction-like chemotherapy regimen)
type
OTHER

interventions

arm Group Labels
Arm A (decitabine or azacitidine)
description
Given IV or SC
name
Azacitidine (AZC)
other Names
5 AZC
5-AC
5-Azacytidine
5-AZC
AZACITIDINE
Azacytidine
Azacytidine, 5-
Ladakamycin
Mylosar
U-18496
Vidaza
type
DRUG
arm Group Labels
Arm A (decitabine or azacitidine)
description
Given IV or SC
name
Decitabine
other Names
5-Aza-2'-deoxycytidine
Dacogen
Decitabine for Injection
Deoxyazacytidine
Dezocitidine
type
DRUG
arm Group Labels
Arm A (decitabine or azacitidine)
Arm B (induction-like chemotherapy regimen)
description
Ancillary studies
name
Quality-of-Life Assessment
other Names
Quality of Life Assessment
type
OTHER

Study design

allocation

RANDOMIZED

intervention Model

PARALLEL

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 50
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
18-month failure-free survival (failure defined as death or relapse).
measure
Failure-free Survival (Failure Defined as Death or Relapse)
time Frame
18 months

secondary Outcomes

description
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.
measure
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.
time Frame
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).
description
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.
measure
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.
time Frame
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).
description
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.
measure
Overall Survival
time Frame
Up to 18 months
description
To compare which of the two, intensive chemotherapy versus hypomethylating agent-based therapy, have a factor of relapse post hematopoietic cell transplantation (HCT).
measure
Number of Patients Who Relapse Post-transplant
time Frame
Up to 18 months
description
Frequency at which the participants received a hematopoietic cell transplantation (HCT)
measure
Number of Participants Who Received a Hematopoietic Cell Transplantation (HCT).
time Frame
Up to 18 months

Full study description

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.
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.

Source references

references

citation
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.
pmid
9058730
type
BACKGROUND
citation
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.
pmid
21105791
type
BACKGROUND
citation
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.
pmid
12149198
type
BACKGROUND
citation
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.
pmid
20008642
type
BACKGROUND
citation
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.
pmid
19135940
type
BACKGROUND
citation
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.
pmid
15674354
type
BACKGROUND
citation
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.
pmid
15625546
type
BACKGROUND
citation
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.
pmid
10694545
type
BACKGROUND
citation
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.
pmid
17038533
type
BACKGROUND
citation
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.
pmid
19151791
type
BACKGROUND
citation
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.
pmid
19543327
type
BACKGROUND
citation
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.
pmid
19363531
type
BACKGROUND

Source notices and limitations

Discovery and provenance

release id
ctgov-registry-1336d216cbd1bbff780dac70
edge id
nct-edge-eefdd579c544dd6d299d
requested nct id
NCT01812252
primary nct id
NCT01812252
source kind
ctgov_condition_query
source url
https://clinicaltrials.gov/api/v2/studies?query.cond=Juvenile+Myelomonocytic+Leukemia&filter.advanced=AREA%5BHasResults%5Dtrue&pageSize=1000&format=json&countTotal=true
source record id
Source null
source file sha256
cddcd4e3a59550bc78924062a298c359d8f811413f73f4a4461963aecf3790d6
payload sha256
3a802599cd1a8ed051a43805e4c79e4c6d32cfb56fad03d3086aaf1972aa6c7a
payload
clinical indication matching status
condition_index_match_asserted_by_registry_not_reviewed
end exclusive
true
id
nct-edge-eefdd579c544dd6d299d
link basis
returned_by_explicit_condition_and_posted_results_filter_query
nct id
NCT01812252
occurrences
context exact
NCT01812252
end
11
exact text
NCT01812252
start
0
offset unit
unicode_code_points
query cond
Juvenile Myelomonocytic Leukemia
query disease state id
ds-juvenile-myelomonocytic-leukemia
source file
/Users/arunverma/Documents/ChatGPT/Cancer Statistics Website/research-data/2026-09-10/clinicaltrials-condition/round-0000/raw/cond-juvenile-myelomonocytic-leukemia-000.json
source file sha256
cddcd4e3a59550bc78924062a298c359d8f811413f73f4a4461963aecf3790d6
source json pointer
/studies/135/protocolSection/identificationModule/nctId
source kind
ctgov_condition_query
source record id
Source null
source string basis
parsed_JSON_string_unmodified
source string sha256
8cf101eee4b17091b8e962f1e3f05fc8a146b910538d1710d5c7bea5a819a0e6
source url
https://clinicaltrials.gov/api/v2/studies?query.cond=Juvenile+Myelomonocytic+Leukemia&filter.advanced=AREA%5BHasResults%5Dtrue&pageSize=1000&format=json&countTotal=true

Preserved source evidence · Not medical advice