Myelodysplastic SyndromesPhase 2NCT00551291

A Study of NeoRecormon (Epoetin Beta), CellCept (Mycophenolate Mofetil) and Prednisone in Patients With Low or Intermediate Myelodysplastic Syndromes.

A Phase 2 study testing Mycophenolate mofetil, Prednisone and Erythropoietin Beta in people with Myelodysplastic Syndromes, run by Hoffmann-La Roche at 8 sites in Spain.

Completed

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

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Myelodysplastic Syndromes
What is being tested?: Mycophenolate mofetil, Prednisone and Erythropoietin Beta
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 10 participants
Where: 8 sitesSpain
Study dates: Aug 2007to Jun 2009
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

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.

Who cannot join

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.

Trial locations and contacts

8 sites in 1 country.

City, postcode or hospital

8 sites in 1 country

Spain8 sites

Study site
Barakaldo, 48903
Study site
Barcelona, 08003
Study site
Barcelona, 08025
Study site
Barcelona, 08035
Study site
Barcelona, 08036
Study site
Cadiz, 11009
Study site
Madrid, 28040
Study site
Palma de Mallorca, 07198

Other trials in Spain

About this study

Official title: An Open Label Study of the Effects of a Combination of NeoRecormon, CellCept and Prednisone on Hematological Parameters and Cytogenesis in Patients With Low or Intermediate Risk Myelodysplastic Syndromes.¿

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.

Conditions: Myelodysplastic Syndromes
Being tested: Mycophenolate mofetil, Prednisone, Erythropoietin Beta
Medical subject headings: Myelodysplastic Syndromes

What participants receive

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

Mycophenolate mofetilDrug
Also called CellCept, MMF
1 gm twice daily orally until end of study.
PrednisoneDrug
10 mg/day orally until end of study.
Erythropoietin BetaDrug
Also called NeoRecormon
Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.

Study groups in NCT00551291
Study group	Role	What they receive
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta	Experimental	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. Receives: Drug: Mycophenolate mofetil; Drug: Prednisone; Drug: Erythropoietin Beta

What is being measured

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

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.
Measured: Up to approximately 2 years
Mean Number of Blood Transfusions Per Visit
Measured: Up to approximately 2 years

Secondary outcomes (1)

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.
Measured: Up to approximately 2 years

Timeline

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

Aug 2007Jun 2009

First posted30 Oct 2007 (estimated)
Study startAug 2007
Primary completionJun 2009
Study completionJun 2009

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 10 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00551291
Sponsor's protocol ID: ML20559
Status last verified: Jun 2016
First submitted: 29 Oct 2007
Last updated: 25 Jul 2016
Results posted: Yes, on ClinicalTrials.gov

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome

All Myelodysplastic Syndromes trials

Study at a glance

From the registry record, last updated by the sponsor on 25 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open Label Study of the Effects of a Combination of NeoRecormon, CellCept and Prednisone on Hematological Parameters and Cytogenesis in Patients With Low or Intermediate Risk Myelodysplastic Syndromes.¿
ClinicalTrials.gov ID: NCT00551291
Acronym / sponsor ID: ML20559
Conditions: Myelodysplastic Syndromes
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 10 participants (actual)
Lead sponsor: Hoffmann-La RocheIndustry (pharma, biotech, devices)
Study start: Aug 2007
Primary completion: Jun 2009 (actual)
Study completion: Jun 2009 (actual)
First posted: 30 Oct 2007 (estimated)
Results: Posted 23 Jun 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta	Experimental	Drug: Mycophenolate mofetil, Drug: Prednisone, Drug: Erythropoietin BetaMycophenolate 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.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

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.

Key exclusion criteria (5 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Mycophenolate mofetilAlso: CellCept, MMF1 gm twice daily orally until end of study.	Drug	Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
Prednisone10 mg/day orally until end of study.	Drug	Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
Erythropoietin BetaAlso: NeoRecormonRecombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.	Drug	Mycophenolate Mofetil + Prednisone + Erythropoietin Beta

Primary outcome

Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement
Time frame: 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
Time frame: Up to approximately 2 years

1 secondary outcome

Percentage of Participants With at Least One Adverse Event (AE)
Time frame: Up to approximately 2 years

Results and safety

Results posted to the registry on 23 Jun 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta	10	9

Primary outcome: Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement

Time frame: Up to approximately 2 years. Number. Unit: percentage of participants

Measure	Mycophenolate Mofetil + Prednisone + Erythropoietin Beta n = 10
Week 12 (n=4)	50.00
Week 18 (n=7)	71.43
End of study (n=3)	100.00

Primary outcome: Mean Number of Blood Transfusions Per Visit

Time frame: Up to approximately 2 years. Mean (Standard Deviation). Unit: transfusions/visit

Measure	Mycophenolate Mofetil + Prednisone + Erythropoietin Beta n = 10
Baseline (n=8)	4.13 (2.30)
Week 12 (n=6)	5.83 (2.86)
Week 18 (n=5)	2.80 (1.92)
End of Study (n=3)	2.33 (1.53)

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Up to approximately 2 years.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta	4 of 10 (40%)	9 of 10 (90%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Myelodysplastic Syndromes

RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Clinical Trials study director

Hoffmann-La Roche

8 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Mycophenolate mofetil, Prednisone and Erythropoietin Beta in people with Myelodysplastic Syndromes, run by Hoffmann-La Roche at 8 sites in Spain.

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 3 inclusion and 5 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 25 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in Spain. 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 23 Jun 2016). 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?

Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement (time frame: Up to approximately 2 years). The study lists 2 primary outcomes in all. 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 NCT00551291.

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 25 Jul 2016.