Myelodysplastic Syndromes
Conditions
Brief summary
This single arm study will evaluate the efficacy and safety of a combination of NeoRecormon, CellCept and prednisone in patients with low or moderate risk myelodysplastic syndromes (MDS). In the first phase of the study, patients will receive CellCept (1g p.o. twice daily) plus prednisone. After 3 months, if patients have not responded to treatment, NeoRecormon (30000 IU/week, s.c.) will be added to the treatment regimen. If there is no response to NeoRecormon after 6 weeks, the dose will be increased to 60000 IU/week. The anticipated time on study treatment is 3-12 months, and the target sample size is \<100 individuals.
Interventions
DRUGPrednisone
10 mg/day orally until end of study.
DRUGMycophenolate mofetil
1 gm twice daily orally until end of study.
Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
Sponsors
Hoffmann-La Roche
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* adult patients, \>=18 years of age; * diagnosis of MDS, according to International Prognostic Scoring System (IPSS) criteria; * low or intermediate risk, who are not candidates for treatment with growth factors, or who have not responded to these treatments.
Exclusion criteria
* previous treatment with CellCept, or any erythropoietin-stimulating drug; * diagnosis of proliferative chronic myelomonocytic leukemia; * prior or concomitant malignancies other than MDS, with the exception of basocellular, spinocellular or adequately treated in situ cervical cancer, in the past 3 years; * biological antitumor and myelosuppressive treatment within 28 days before start of study; * bone marrow precursor cell transplantation previous to study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement | Up to approximately 2 years | International Working Group (IWG) criteria for hematological improvement was defined as having hemoglobin (Hgb) \<11 g/dL (pretreatment) and an increase in Hgb ≥1.5 g/dL after ≥8 weeks of treatment. |
| Mean Number of Blood Transfusions Per Visit | Up to approximately 2 years | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With at Least One Adverse Event (AE) | Up to approximately 2 years | An AE was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events. |
Countries
Spain
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks. | 10 |
| Total | 10 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Physician Decision | 1 |
Baseline characteristics
| Characteristic | Mycophenolate Mofetil + Prednisone + Erythropoietin Beta |
|---|---|
| Age, Continuous | 75.00 years STANDARD_DEVIATION 5.17 |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 9 / 10 |
| serious Total, serious adverse events | 4 / 10 |
Outcome results
Primary
Mean Number of Blood Transfusions Per Visit
Time frame: Up to approximately 2 years
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | Mean Number of Blood Transfusions Per Visit | Week 12 (n=6) | 5.83 transfusions/visit | Standard Deviation 2.86 |
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | Mean Number of Blood Transfusions Per Visit | Week 18 (n=5) | 2.80 transfusions/visit | Standard Deviation 1.92 |
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | Mean Number of Blood Transfusions Per Visit | Baseline (n=8) | 4.13 transfusions/visit | Standard Deviation 2.3 |
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | Mean Number of Blood Transfusions Per Visit | End of Study (n=3) | 2.33 transfusions/visit | Standard Deviation 1.53 |
Primary
Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement
International Working Group (IWG) criteria for hematological improvement was defined as having hemoglobin (Hgb) \<11 g/dL (pretreatment) and an increase in Hgb ≥1.5 g/dL after ≥8 weeks of treatment.
Time frame: Up to approximately 2 years
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement | Week 12 (n=4) | 50.00 percentage of participants |
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement | Week 18 (n=7) | 71.43 percentage of participants |
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement | End of study (n=3) | 100.00 percentage of participants |
Secondary
Percentage of Participants With at Least One Adverse Event (AE)
An AE was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events.
Time frame: Up to approximately 2 years
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | Percentage of Participants With at Least One Adverse Event (AE) | 90.00 percentage of participants |