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Myelodysplastic Syndromes (MDS)Phase 2NCT00594230

LBH589 in Refractory Myelodysplastic Syndromes (MDS)

A Phase 2 study testing Panobinostat in people with Myelodysplastic Syndromes (MDS), run by SCRI Development Innovations, LLC at 7 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This will be a single arm Phase II study. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes (MDS)
What is being tested?
Panobinostat
Could I receive a placebo?
No, everyone receives an active treatment
How many people
26 participants
Where
7 sitesUnited States
Study dates
Jan 2008to Mar 2011
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

  • Histologically or cytological documented diagnosis of myelodysplastic syndrome (MDS).
  • Male or female patients aged >= 18 years old.
  • MDS patients who have failed hypomethylating (azacitidine or decitabine) therapy.
  • Patients with 5q-cytogenic abnormalities must also have progressed on or been intolerant to lenalidomide.
  • Patients with up to and including 30% blasts (FAB RAEB-T) will be eligible to enroll.
  • CMML with >= 5% blasts will be eligible to enroll.
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  • ECOG PS 0, 1 or 2.
  • Laboratory values must be as follows:
  • Bilirubin <= 1.5 mg/dL AST/SGOT <= 2.5 x ULN ALT/SGPT Creatinine <= 2.0 mg/dL or 24-hour Creatinine Clearance >= 50 ml/min Albumin >= 3 g/dL Potassium >= lower limit normal (LLN) Phosphorous >= LLN Calcium >= LLN Magnesium >= LLN
  • Women of childbearing potential must have a negative serum pregnancy test performed within 7 days prior to start of treatment.
  • Life expectancy >= 12 weeks.

Who cannot join

  • Prior treatment with an HDAC inhibitor.
  • Prior intensive chemotherapy or high dose ara-C (>= 1 gm/m2)
  • More than one prior single agent chemotherapy regimen. Prior hydroxyurea for cytoreduction will be permitted however.
  • Impaired cardiac function
  • Active CNS disease, including leptomeningeal metastases.
  • Unresolved diarrhea > CTCAE grade 1.
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  • Chemotherapy, investigational drug therapy, major surgery < 4 weeks prior to starting study drug or patients that have not recovered from side effects of previous therapy.
  • Patient is < 5 years free of another primary malignancy except if the other primary malignancy is not currently clinically significant or requiring active intervention, or if other primary malignancy is a basal cell skin cancer or a cervical carcinoma in situ. Existence of any other malignant disease is not allowed.
  • Women who are pregnant or breast feeding or women of childbearing potential (WOCBP) not willing to use a double barrier method of contraception during the study and 3 months after the end of treatment. One of these methods of contraception must be a barrier method.
  • Male patients whose sexual partners are WOCBP not using a double method of contraception during the study and 3 months after the end of treatment. One of these methods must be a condom.
  • Patients with gastrointestinal (GI) tract disease, causing the inability to take oral medication, malabsorption syndrome, a requirement for intravenous (IV) alimentation, prior surgical procedures affecting absorption, uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis).
  • Other concurrent severe, uncontrolled systemic fungal, bacterial, viral or other infection or intercurrent illness, including but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Patients with uncontrolled coagulopathy.
  • Abnormal thyroid function (TSH or free T4) detected at screening. Patients with known hypothyroidism who are stable on thyroid replacement are eligible.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites

  • Center for Cancer and Blood Disorders

    Bethesda, Maryland, 20817

  • Chattanooga Oncology Hematology Associates

    Chattanooga, Tennessee, 37404

  • Oncology Hematology Care

    Cincinnati, Ohio, 45242

  • Florida Cancer Specialists

    Fort Myers, Florida, 33901

  • Northeast Georgia Medical Center

    Gainesville, Georgia, 30501

  • Consultants in Blood Disorders and Cancer

    Louisville, Kentucky, 40207

  • Tennessee Oncology, PLLC

    Nashville, Tennessee, 37023

Other trials in United States

About this study

Official title: A Phase II Trial of LBH589 in Refractory Myelodysplastic Syndromes (MDS) Patients

This will be a single arm Phase II study.

Read the detailed description

LBH589 (20 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator.

Being tested
Panobinostat
Keywords
Myelodysplastic Syndromes (MDS), Refractory, LBH589
Medical subject headings
Myelodysplastic Syndromes

What participants receive

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

  • PanobinostatDrug

    Also called LBH589

    Panobinostat(20 mg or 30 mg PO) will be administered three times a week on Monday, Wednesday and Friday. Treatment will be given over 21 days followed by a 7 day rest period and repeated every 28 days. Patients will be assessed for toxicity on an ongoing basis and disease assessment will be determined every 2 treatment cycles (8 weeks). Patients will be allowed to continue on treatment for a maximum of eight four week treatment cycles. Treatment will be discontinued if there is evidence of disease progression, unacceptable toxicity and/or at the discretion of the investigator.

Study groups in NCT00594230
Study groupRoleWhat they receive
Panobinostat 20 mgExperimentalTreatment with LBH589 (Panobinostat) 20 mg
Receives: Drug: Panobinostat
Panobinostat 30 mgExperimentalTreatment with LBH589 (Panobinostat) 30 mg
Receives: Drug: Panobinostat

What is being measured

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

  1. Overall Response Rate (CR, Marrow CR + PR) of LBH in Patients With Relapsed or Refractory MDS.

    Overall response rate (ORR) is defined by the modified International Working Group (IWG) Response Criteria for MDS. In the marrow, Complete Response (CR) is <= 5% blasts present with normal maturation of all cell lines. In peripheral blood, CR is defined as hemoglobin >= 11 g/dL, ANC >= 1000/mL, and platelets >= 100,000 with 0% blasts present. Partial Response (PR) is defined the same as CR with blasts decreased by >= 50% and >= 5% blasts in the marrow.

    Measured: Every 8 weeks up to 24 months on-study.

Secondary outcomes (6)

  1. Time to Disease Progression

    Time to disease progression is defined as the time between day 1 cycle 1 and time to first documented disease progression. Disease progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.

    Measured: Every 8 weeks up to 24 months on-study, every 3 months in follow-up until progression of disease

  2. Hematologic Improvement, Including Transfusion Independence

    Hematologic measures will include total WBC and platelets

    Measured: Every 8 weeks up to 24 months on-study

  3. Duration of Response

    Duration of response is defined as the time from when objective response is realized until time to first documented disease progression. Disease progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Objective Response = CR + PR. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI or CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions.

    Measured: Every 8 weeks up to 24 months on-study

  4. Median Time to Treatment Failure

    Time to treatment failure is defined as measuring the time between cycle 1 day 1 to discontinuation for any reason.

    Measured: 24 months

  5. Median Overall Survival

    The Length of Time, in Months, That Patients Were Alive From Their First Date of Protocol Treatment Until Death

    Measured: 24 months on-study, patients followed every 3 months in follow-up

  6. Safety and Tolerability of LBH589 in Patients With Relapsed/Refractory MDS by Measuring the Number of Participants With Adverse Events

    The reported incidence of AEs and SAEs with an onset on or after the initiation of therapy was graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0

    Measured: 24 months

Timeline

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

  1. First posted15 Jan 2008 (estimated)
  2. Study startJan 2008
  3. Primary completionMar 2011
  4. Study completionMar 2011

Why it stopped: Per protocol, the results of a planned interim analysis demonstrated insufficient efficacy and led to early termination of the study.

Resources

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
Parallel groups
Masking
None (open label)
Enrolment
26 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00594230
Sponsor's protocol ID
SCRI MDS 07
Status last verified
Nov 2021
First submitted
19 Dec 2007
Last updated
22 Nov 2021
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Panobinostat in people with Myelodysplastic Syndromes (MDS), run by SCRI Development Innovations, LLC at 7 sites in United States.

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 11 inclusion and 14 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 22 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States. 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 19 Dec 2012). 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?

Overall Response Rate (CR, Marrow CR + PR) of LBH in Patients With Relapsed or Refractory MDS. (time frame: Every 8 weeks up to 24 months on-study.). 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 NCT00594230.

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 22 Nov 2021.

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