Myelodysplastic Syndrome (MDS)Phase 2GEPARDNCT01034657
LBH589 Alone or in Combination With Erythropoietin Stimulating Agents (ESA) in Patients With Low or Int-1 Risk Myelodysplastic Syndromes (MDS)
A Phase 2 study testing LBH589 and Epoetin Alfa in people with Myelodysplastic Syndrome (MDS), run by Novartis Pharmaceuticals at 12 sites in Germany.
Trial summary
This study assessed the efficacy and safety of LBH589 as single agent and in combination with ESA in red blood cell transfusion-dependent Low and Int-1 MDS patients being either refractory to ESA or with a low probability of response. The study had a non-randomized core phase followed by a randomized phase. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome (MDS)
- What is being tested?
- LBH589 and Epoetin Alfa
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 12 sitesGermany
- Study dates
- Nov 2009to Aug 2012
- 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
- Patients with a lower risk MDS (LOW or INT-1 according to IPSS)
- Red blood cell transfusion dependency of at least 4 Units/8 weeks.
- Not responding to Erythropoietin stimulating agents (ESA) or having a low chance to do so
- Age-adjusted normal cardiac, kidney, liver function
Who cannot join
- Concomitant use of ESA
- Concomitant use of any other investigational drug
- Other malignancy that is not in remission for at least 1 year
- Platelet Count < 75 x 109/L
- Impaired cardiac function or clinically significant cardiac diseases
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
Germany12 sites
Novartis Investigative Site
Berlin, 12203
Novartis Investigative Site
Bonn, 53105
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Duisburg, 47166
Novartis Investigative Site
Düsseldorf, 40225
Novartis Investigative Site
Frankfurt, 60590
Novartis Investigative Site
Göttingen, 37075
Novartis Investigative Site
Hanover, 30625
Novartis Investigative Site
Leipzig, 04103
Novartis Investigative Site
Mannheim, Baden-Wurttemberg, 68305
Novartis Investigative Site
München, 81675
Novartis Investigative Site
Ulm, 89081
About this study
Official title: A One Year, Open Label, Multicenter Trial of LBH589 Alone or in Combination With ESA in Red Blood Cell Transfusion-dependent LOW and INT-1 MDS Patients Being Either Refractory to ESA or With a Low Probability of Response - the GErman PAnobinostat Low Risk MDS Trial - GEPARD Study
This study assessed the efficacy and safety of LBH589 as single agent and in combination with ESA in red blood cell transfusion-dependent Low and Int-1 MDS patients being either refractory to ESA or with a low probability of response. The study had a non-randomized core phase followed by a randomized phase.
- Conditions
- Myelodysplastic Syndrome (MDS)
- Being tested
- LBH589, Epoetin Alfa
- Keywords
- MDS, bone marrow, anemia, cytopenia, transfusion dependance, EPO, ESA, erythropoietin, LBH589, Myelodysplastic Syndromes, hematopoietic improvement, IPSS Low, IPSS Int-1, HI-E, HDAC Inhibitor, HDAC-I, DAC-I, Deacetylase-Inhibitor, Histone Deacetylase-Inhibitor, red blood cell transfusions
- Medical subject headings
- Myelodysplastic Syndromes, Anemia, Cytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LBH589Drug
Also called Panobinostat
LBH589 was supplied at dose strengths of 5 mg or 20 mg hard gelatin capsules.
Epoetin AlfaDrug
Also called ESA, HEXAL®
Epoetin alfa was supplied as 10000 IU/1 mL in a ready-to-use syringe.
| Study group | Role | What they receive |
|---|---|---|
| LBH589 | Experimental | During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months. Receives: Drug: LBH589 |
| LBH589 + Epoetin Alfa | Experimental | During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months. Receives: Drug: LBH589; Drug: Epoetin Alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Hematological Response of the Erythropoetic System (HI-E) - Core Phase
HI-E was assessed according to the modified international working group (IWG) criteria for HI. Erythroid response (pretreatment, <11 g/dL): Hgb increase by ≥ 1.5 g/dL, relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk, and only RBC transfusions given for a Hgb of ≤ 9.0 g/dL pretreatment were counted in the RBC transfusion response evaluation; Platelet response (pretreatment, < 100 x 109/L): absolute increase of ≥ 30 x 109/L for participants starting with > 20 x 109/L and platelets Increase from < 20 x 109/L to > 20 x 109/L and by at least 100%; Neutrophil response (pretreatment, < 1.0 x 109/L): at least 100% increase and an absolute increase > 0.5 x 109/L; Progression or relapse after HI: At least 1 of the following: At least 50% decrement from maximum response levels in granulocytes or platelets, reduction in Hgb by ≥1.5 g/dL, or transfusion dependence.
Measured: 16 weeks
Secondary outcomes (13)
Percentage of Participants With HI-E - Randomized Phase
HI-E was assessed according to the modified international working group (IWG) criteria for HI. Erythroid response (pretreatment, <11 g/dL): Hgb increase by ≥ 1.5 g/dL, relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk, and only RBC transfusions given for a Hgb of ≤ 9.0 g/dL pretreatment were counted in the RBC transfusion response evaluation; Platelet response (pretreatment, < 100 x 109/L): absolute increase of ≥ 30 x 109/L for participants starting with > 20 x 109/L and platelets Increase from < 20 x 109/L to > 20 x 109/L and by at least 100%; Neutrophil response (pretreatment, < 1.0 x 109/L): at least 100% increase and an absolute increase > 0.5 x 109/L; Progression or relapse after HI: At least 1 of the following: At least 50% decrement from maximum response levels in granulocytes or platelets, reduction in Hgb by ≥1.5 g/dL, or transfusion dependence.
Measured: 32 weeks, 52 weeks
Percentage of Participants With Objective Response During Core Phase
Objective response (complete remission (CR) + partial remission (PR) and HI-platelet (HI-P) response + HI-neutrophil (HI-N) response) was assessed according to the modified IWG criteria: CR bone marrow with 5% myeloblasts with normal maturation of al cell lines (persistent dysplasia is noted) and peripheral blood with Hgb >= 11 g/dL platelets >=100 X 10^9/L, neutrophils >= 1.0 x 10^9/L and blasts 0%. PR = All CR if abnormal before treatment except bone marrow blasts decreased by>=50% over pretreatment but still >5% (ellularity and morphology not relevant). HI-P (pretreatment, < 100 x 109/L) = absolute increase of ≥ 30 x 109/L for participants starting with > 20 x 109/L and platelets Increase from < 20 x 109/L to > 20 x 109/L and by at least 100%; HI-N (pretreatment, < 1.0 x 109/L) = at least 100% increase and an absolute increase > 0.5 x 10^9/L.
Measured: 16 weeks
Percentage of Participants With Objective Response During the Randomized Phase
Objective response (complete remission (CR) + partial remission (PR) and HI-platelet (HI-P) response + HI-neutrophil (HI-N) response) was assessed according to the modified IWG criteria: CR bone marrow with 5% myeloblasts with normal maturation of al cell lines (persistent dysplasia is noted) and peripheral blood with Hgb >= 11 g/dL platelets >=100 X 10^9/L, neutrophils >= 1.0 x 10^9/L and blasts 0%. PR = All CR if abnormal before treatment except bone marrow blasts decreased by>=50% over pretreatment but still >5% (ellularity and morphology not relevant). HI-P (pretreatment, < 100 x 109/L) = absolute increase of ≥ 30 x 109/L for participants starting with > 20 x 109/L and platelets Increase from < 20 x 109/L to > 20 x 109/L and by at least 100%; HI-N (pretreatment, < 1.0 x 109/L) = at least 100% increase and an absolute increase > 0.5 x 10^9/L.
Measured: 32 weeks, 48 weeks
Frequency Distribution of IPSS Score Status - Core Phase
The IPSS score values were calculated based on the results of bone marrow analysis. A score value of 0 has bone marrow blast <5%, karyotype of normal, sole: -Y, del 5Q, del 20q and cytopenias (lineages affected) of 0 to 1. Score value of 0.5 has 5-10 bone marrow blasts, karyotype of Others and cytopenias of 2 to 3. A score value of 1.0 has complex >= 3 chromosomal abnormalities and/or chromosome 7 anomalies. A score of 1.5 has 11-20 bone marrow blasts and a score of 2.0 has 21-30 bone marrow blasts. The prognostic score is determined by the sum of the single scoring values. The risk groups are determined as follows: Low = 0 points (5.7 years of median survival); intermediate -1 (INT-1) = 0.5-1.0 points (3.5 years of median survival); INT-2 = 1.5-2.0 points (1.2 years of median survival); and high >=2.5 points (6 months of median survival).
Measured: baseline
Frequency Distribution of IPSS Score Status - Randomized Phase
The IPSS score values were calculated based on the results of bone marrow analysis. A score value of 0 has bone marrow blast <5%, karyotype of normal, sole: -Y, del 5Q, del 20q and cytopenias (lineages affected) of 0 to 1. Score value of 0.5 has 5-10 bone marrow blasts, karyotype of Others and cytopenias of 2 to 3. A score value of 1.0 has complex >= 3 chromosomal abnormalities and/or chromosome 7 anomalies. A score of 1.5 has 11-20 bone marrow blasts and a score of 2.0 has 21-30 bone marrow blasts. The prognostic score is determined by the sum of the single scoring values. The risk groups are determined as follows: Low = 0 points (5.7 years of median survival); intermediate -1 (INT-1) = 0.5-1.0 points (3.5 years of median survival); INT-2 = 1.5-2.0 points (1.2 years of median survival); and high >=2.5 points (6 months of median survival).
Measured: 52 weeks
Mean Single Scoring Values of the IPSS - Core Phase
The IPSS score values were calculated based on the results of bone marrow analysis. A score value of 0 has bone marrow blast <5%, karyotype of normal, sole: -Y, del 5Q, del 20q and cytopenias (lineages affected) of 0 to 1. Score value of 0.5 has 5-10 bone marrow blasts, karyotype of Others and cytopenias of 2 to 3. A score value of 1.0 has complex >= 3 chromosomal abnormalities and/or chromosome 7 anomalies. A score of 1.5 has 11-20 bone marrow blasts and a score of 2.0 has 21-30 bone marrow blasts. The prognostic score is determined by the sum of the single scoring values. The risk groups are determined as follows: Low = 0 points (5.7 years of median survival); intermediate -1 (INT-1) = 0.5-1.0 points (3.5 years of median survival); INT-2 = 1.5-2.0 points (1.2 years of median survival); and high >=2.5 points (6 months of median survival).
Measured: baseline
Mean Single Scoring Values of the IPSS - Randomized Phase
The IPSS score values were calculated based on the results of bone marrow analysis. A score value of 0 has bone marrow blast <5%, karyotype of normal, sole: -Y, del 5Q, del 20q and cytopenias (lineages affected) of 0 to 1. Score value of 0.5 has 5-10 bone marrow blasts, karyotype of Others and cytopenias of 2 to 3. A score value of 1.0 has complex >= 3 chromosomal abnormalities and/or chromosome 7 anomalies. A score of 1.5 has 11-20 bone marrow blasts and a score of 2.0 has 21-30 bone marrow blasts. The prognostic score is determined by the sum of the single scoring values. The risk groups are determined as follows: Low = 0 points (5.7 years of median survival); intermediate -1 (INT-1) = 0.5-1.0 points (3.5 years of median survival); INT-2 = 1.5-2.0 points (1.2 years of median survival); and high >=2.5 points (6 months of median survival).
Measured: 52 weeks
Overall Survival (OS) - Overall Period
OS was defined as the time from start of treatment to death from any cause.
Measured: 48 weeks
Time to Response - Overall Period
Time to response was defined as the time from start of treatment to the first documented response (complete [CR] or partial [PR]) according to modified IWG criteria for HI.
Measured: 52 weeks
Event-free Survival (EFS) - Overall Period
EFS was defined as the time from start of treatment to failure or death from any cause.
Measured: 52 weeks
Progression-free Survival (PFS) - Overall Period
PFS was defined as the time from start of treatment to disease progression or death from MDS.
Measured: 52 weeks
Disease-free Survival (DFS) - Overall Period
DFS was defined as the time from start of treatment to the time to relapse.
Measured: 52 weeks
Time to Cause-specific Death - Overall Period
Time to cause-specific death was defined as the time from start of treatment to death related to MDS.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2009 (estimated)
- Study startNov 2009
- Primary completionAug 2012
- Study completionAug 2012
Why it stopped: The study was terminated due to lack of efficacy of single agent LBH589 in the 4 month open label core phase and due to enrollment difficulties.
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 Syndrome (MDS)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01034657
- Sponsor's protocol ID
- CLBH589BDE04
- Other IDs
- EudraCT 2009-010403-84, 2009-010403-84, 2009
- Acronym
- GEPARD
- Status last verified
- Aug 2017
- First submitted
- 16 Dec 2009
- Last updated
- 11 Aug 2017
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A One Year, Open Label, Multicenter Trial of LBH589 Alone or in Combination With ESA in Red Blood Cell Transfusion-dependent LOW and INT-1 MDS Patients Being Either Refractory to ESA or With a Low Probability of Response - the GErman PAnobinostat Low Risk MDS Trial - GEPARD Study
- ClinicalTrials.gov ID
- NCT01034657
- Acronym / sponsor ID
- GEPARD / CLBH589BDE04
- Conditions
- Myelodysplastic Syndrome (MDS)
- Keywords
- MDS, bone marrow, anemia, cytopenia, transfusion dependance, EPO, ESA, erythropoietin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated due to lack of efficacy of single agent LBH589 in the 4 month open label core phase and due to enrollment difficulties.
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Nov 2009
- Primary completion
- Aug 2012 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 17 Dec 2009 (estimated)
- Results
- Posted 25 Jan 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study assessed the efficacy and safety of LBH589 as single agent and in combination with ESA in red blood cell transfusion-dependent Low and Int-1 MDS patients being either refractory to ESA or with a low probability of response. The study had a non-randomized core phase followed by a randomized phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LBH589 | Experimental | Drug: LBH589During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months. |
| LBH589 + Epoetin Alfa | Experimental | Drug: LBH589, Drug: Epoetin AlfaDuring the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients with a lower risk MDS (LOW or INT-1 according to IPSS)
- Red blood cell transfusion dependency of at least 4 Units/8 weeks.
- Not responding to Erythropoietin stimulating agents (ESA) or having a low chance to do so
- Age-adjusted normal cardiac, kidney, liver function
Key exclusion criteria (5 listed)
- Concomitant use of ESA
- Concomitant use of any other investigational drug
- Other malignancy that is not in remission for at least 1 year
- Platelet Count < 75 x 109/L
- Impaired cardiac function or clinically significant cardiac diseases
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 |
|---|---|---|
| LBH589Also: PanobinostatLBH589 was supplied at dose strengths of 5 mg or 20 mg hard gelatin capsules. | Drug | LBH589, LBH589 + Epoetin Alfa |
| Epoetin AlfaAlso: ESA, HEXAL®Epoetin alfa was supplied as 10000 IU/1 mL in a ready-to-use syringe. | Drug | LBH589 + Epoetin Alfa |
Primary outcome
Percentage of Participants With Hematological Response of the Erythropoetic System (HI-E) - Core Phase
Time frame: 16 weeks
HI-E was assessed according to the modified international working group (IWG) criteria for HI. Erythroid response (pretreatment, <11 g/dL): Hgb increase by ≥ 1.5 g/dL, relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk, and only RBC transfusions given for a Hgb of ≤ 9.0 g/dL pretreatment were counted in the RBC transfusion response evaluation; Platelet response (pretreatment, < 100 x 109/L): absolute increase of ≥ 30 x 109/L for participants starting with > 20 x 109/L and platelets Increase from < 20 x 109/L to > 20 x 109/L and by at least 100%; Neutrophil response (pretreatment, < 1.0 x 109/L): at least 100% increase and an absolute increase > 0.5 x 109/L; Progression or relapse after HI: At least 1 of the following: At least 50% decrement from maximum response levels in granulocytes or platelets, reduction in Hgb by ≥1.5 g/dL, or transfusion dependence.
13 secondary outcomes
Percentage of Participants With HI-E - Randomized Phase
Time frame: 32 weeks, 52 weeks
Percentage of Participants With Objective Response During Core Phase
Time frame: 16 weeks
Percentage of Participants With Objective Response During the Randomized Phase
Time frame: 32 weeks, 48 weeks
Frequency Distribution of IPSS Score Status - Core Phase
Time frame: baseline
Frequency Distribution of IPSS Score Status - Randomized Phase
Time frame: 52 weeks
Mean Single Scoring Values of the IPSS - Core Phase
Time frame: baseline
Mean Single Scoring Values of the IPSS - Randomized Phase
Time frame: 52 weeks
Overall Survival (OS) - Overall Period
Time frame: 48 weeks
Time to Response - Overall Period
Time frame: 52 weeks
Event-free Survival (EFS) - Overall Period
Time frame: 52 weeks
Progression-free Survival (PFS) - Overall Period
Time frame: 52 weeks
Disease-free Survival (DFS) - Overall Period
Time frame: 52 weeks
Time to Cause-specific Death - Overall Period
Time frame: 52 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
12 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 LBH589 and Epoetin Alfa in people with Myelodysplastic Syndrome (MDS), run by Novartis Pharmaceuticals at 12 sites in Germany.
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 4 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: stopped early, as of the sponsor's last update on 11 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Germany. 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 25 Jan 2017). 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 Hematological Response of the Erythropoetic System (HI-E) - Core Phase (time frame: 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 Aug 2017.