Prospective Phase II Study of Decitabine Induction Therapy to Reduce Pre-transplant Disease Burden Prior to Allogeneic Hematopoietic Cell Transplant in Patients With Newly Diagnosed Myelodysplastic Syndromes.
21 Years
65 Years
Both
Myelodysplastic Syndrome
Trial Information
Prospective Phase II Study of Decitabine Induction Therapy to Reduce Pre-transplant Disease Burden Prior to Allogeneic Hematopoietic Cell Transplant in Patients With Newly Diagnosed Myelodysplastic Syndromes.
Primary endpoint:
1. safety and tolerability of Decitabine prior to transplant (assessed by occurence of
non-hematologic toxicities of grade 3 or more as defined by CTC grading)
2. reduction in pre-transplant disease burden ability to achieve blast <5% in the bone
marrow and peripheral blood
Secondary endpoints:
1. Proportion of patients with suitable donor able to proceed to an allogeneic
hematopoietic cell transplant.
2. Non-relapse mortality
3. time to neutrophil engraftment
4. Overall survival and disease-free survival.
Patients will receive Decitabine until blast <5% is achieved, suitable HLA-matched donor or
umbilical cord blood is available up to a maximum of 6 cycles. Patient who progress on
therapy or are unable to find a donor by 6 cycles will be removed from protocol. The
method, conditioning regimen and choice of donor will be determined based on patient's age
and functional status, and transplant physician's discretion. The available regimens are
standardized within the center
Inclusion Criteria:
1. Newly diagnosed MDS patients aged 21 to 65 years belonging to any of the following
categories: refractory cytopenia with multilineage dysplasia (RCMD) with or without
ringed sideroblasts (i.e. RCMD and RCMD-RS), refractory anemia with excess blasts-1
(RAEB-1) or RAEB-2 if the prognostic scores are IPSS (international prognostic
scoring system) Int-2 or IPSS-high or with WPSS (WHO prognostic scoring system) 3 and
above
2. Therapy-related MDS with IPSS Int-2 and above or WPSS 3
3. Acceptable cardiac function MUGA or Echocardiography left ventricular ejection
fraction of 40% and above
4. Acceptable lung function: FEV1>70% predicted, DLCO>60% predicted
5. Acceptable renal function: CCT > 50ml/min
6. Acceptable liver function: abnormalities in bilirubin or transaminases not > 2times
upper limit of normal
7. Performance status of ECOG 2 or HCT-specific Comorbidity Index < 3
Exclusion Criteria:
1. Any co-morbidity other than MDS which limits life-expectancy to <3mth
2. Diagnosis of other active cancer other than squamous cell carcinoma, basal cell
carcinoma or carcinoma-in-situ 1 or 2 of the cervix
3. Presence of active infections not under control
4. Receipt of 5-azacytidine or other induction chemotherapy for MDS/AML
5. Patients not keen to explore allogeneic HCT as part of curative treatment plan
6. Pregnancy
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Reduction in pre-transplant disease burden
Outcome Time Frame:
2 years
Safety Issue:
No
Principal Investigator
Yvonne Loh
Investigator Role:
Principal Investigator
Investigator Affiliation:
Singapore General Hospital
Authority:
Singapore: Health Sciences Authority
Study ID:
Decitabine01
NCT ID:
NCT01333449
Start Date:
July 2010
Completion Date:
October 2012
Related Keywords:
- Myelodysplastic Syndrome
- Decitabine
- MDS, myelodysplastic syndrome
- prospective open label, phaseII
- Allogeneic Hematopoietic cell transplant
- Myelodysplastic Syndromes
- Preleukemia
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