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Myelodysplastic SyndromePhase 2NCT01873703

Phase 2 Study of Pracinostat With Azacitidine in Patients With Previously Untreated Myelodysplastic Syndrome

A Phase 2 study testing pracinostat and Azacitidine in people with Myelodysplastic Syndrome, run by Helsinn Healthcare SA at 24 sites in United States.

Completed

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

Official record
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Trial summary

The purpose of this randomized, double-blind, placebo-controlled study is to determine the safety and efficacy of pracinostat compared to placebo when combined with azacitidine, and FDA approved treatment for Myelodysplastic Syndrome (MDS). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndrome
What is being tested?
pracinostat and Azacitidine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
102 participants
Where
24 sitesUnited States
Study dates
Jun 2013to Nov 2016
Phase
Phase 2
Are there results?
None posted in the registry

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

  • Voluntary written informed consent
  • Histologically or cytologically documented diagnosis of MDS (any French-American-British [FAB] classification subtype; that is classified as intermediate 2 (1.5 to 2.0 points) or high risk (≥2.5 points) according to the International Prognostic Scoring System risk category, with >5% and <30% blasts, and a peripheral blast count of <20,000
  • Bone marrow aspirate smears and bone marrow biopsies within 28 days of first study treatment
  • There must be a clinical indication for treatment with azacitidine.
  • Previously untreated with hypomethylating agents (prior therapy with transfusions, hematopoietic growth factors, or immunosuppressive therapy is allowed)
  • Eastern Cooperative Oncology Group performance status of 0, 1, or 2
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  • Adequate organ function as evidenced by:
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 x the upper limit of normal (ULN) (≤5 x ULN for patients with hepatic metastases
  • Total bilirubin ≤1.5 x ULN or total bilirubin of 2, whichever is higher
  • Serum creatinine <2 mg/dL, or creatinine clearance ≤1.5 x ULN
  • QTcF interval ≤470 msec
  • Female or male patients ≥18 years-of-age
  • Male patients who are surgically sterile or willing to use adequate contraceptive measures or abstain from heterosexual intercourse during the entire study treatment period
  • Female patients who are surgically sterile or post menopausal or female patients who are not of child-bearing potential and female patients of child-bearing potential who agree to use adequate contraceptive measures or abstain from intercourse during the study treatment period, who are not breastfeeding, and who have had a negative serum pregnancy test ≤7 days prior to first study treatment.
  • Willingness and ability to comply with the trial and follow-up procedures

Who cannot join

  • Received any of the following within the specified time frame prior to administration of study medication:
  • Any investigational agent within 14 days or 5 half-lives prior to first study treatment, whichever is longer
  • Previous therapy for malignancy within 21 days prior to first study treatment, including any chemotherapy, immunotherapy, biological or hormonal therapy (6 weeks for nitrosoureas or mitomycin C)
  • Hydroxyurea within 48 hours prior to first study treatment
  • Hematopoietic growth factors: erythropoietin, granulocyte colony stimulating factor (G-CSF), granulocyte macrophage colony stimulating factor (GM-CSF), or thrombopoietin receptor agonists at least 7 days (14 days for Aranesp) prior to study enrollment
  • Major surgery within 4 weeks prior to first study treatment
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  • Patients that have not recovered from side effects of previous therapy
  • Cardiopulmonary function exclusion:
  • Current unstable arrhythmia requiring treatment
  • History of symptomatic congestive heart failure (New York Heart Association Classes III or IV)
  • History of myocardial infarction within 6 months of enrollment
  • Current unstable angina
  • Concomitant treatment with histone deacetylase (HDAC) inhibitors or drugs with significant action as HDAC inhibitors, such as valproic acid, is not permitted
  • Clinical evidence of central nervous system involvement
  • Patients with gastrointestinal (GI) tract disease, causing the inability to take oral medication, malabsorption syndrome, a requirement for IV alimentation, prior surgical procedures affecting absorption, uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis).
  • Active infection with HIV or chronic hepatitis B or C
  • Life-threatening illness unrelated to cancer, or any serious medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with participation in this study
  • Presence of a malignant disease within the last 12 months, with the exception of adequately treated in-situ carcinomas, basal or squamous cell carcinoma, or non-melanomatous skin cancer
  • Inability (including psychological, familial, sociological, or geographical conditions) to comply with trial and/or follow-up procedures

Trial locations and contacts

24 sites in 1 country.

24 sites in 1 country

United States24 sites

  • Sidney Kimmel Comprehensive Cancer at Johns Hopkins

    Baltimore, Maryland, 21287

  • Center for Cancer and Blood Disorders

    Bethesda, Maryland, 20817

  • Tennessee Oncology - Chattanooga

    Chattanooga, Tennessee, 37404

  • Oncology Hematology Care

    Cincinnati, Ohio, 45242

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • Colorado Blood Cancer Institute

    Denver, Colorado, 80218

  • Henry Ford Hospital

    Detroit, Michigan, 48202

  • Florida Cancer Specialists South

    Fort Myers, Florida, 33916

  • Fort Wayne Medical Oncology and Hematology

    Fort Wayne, Indiana, 46804

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • Indiana University Simon Cancer Ctr

    Indianapolis, Indiana, 46202

  • Scripps Cancer Center

    La Jolla, California, 92037

  • Michigan State University

    Lansing, Michigan, 48910

  • Comprehensive Cancer Centers of Nevada

    Las Vegas, Nevada, 89148

  • Southern Cancer Center

    Mobile, Alabama, 36608

  • Sarah Cannon Cancer Center, Tennessee Oncology

    Nashville, Tennessee, 37203

  • Weill Cornell Medical Center

    New York, New York, 10065

  • Nebraska Methodist

    Omaha, Nebraska, 68114

  • Woodlands Medical Specialists

    Pensacola, Florida, 32503

  • Cancer Care Centers of South Texas

    San Antonio, Texas, 78229

  • Swedish Cancer Institute

    Seattle, Washington, 98104

  • Florida Cancer Specialists North

    St. Petersburg, Florida, 33705

  • Florida Cancer Specialist and Research Institute

    Tallahassee, Florida, 32308

  • H. Lee Moffitt Cancer Center

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: A Phase 2 Randomized Double-Blind Placebo-Controlled Study of Pracinostat in Combination With Azacitidine in Patients With Previously Untreated International Prognostic Scoring System (IPSS) Intermediate Risk-2 or High-Risk Myelodysplastic Syndrome (MDS)

The purpose of this randomized, double-blind, placebo-controlled study is to determine the safety and efficacy of pracinostat compared to placebo when combined with azacitidine, and FDA approved treatment for Myelodysplastic Syndrome (MDS).

Being tested
pracinostat, Placebo, Azacitidine
Keywords
MDS, High risk myelodysplastic syndrome, Intermediate-2 Myelodysplastic syndrome, Untreated
Medical subject headings
Myelodysplastic Syndromes

What participants receive

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

  • pracinostatDrug

    Also called SB939

    Histone deacetylase inhibitor (HDACi)

  • PlaceboDrug

    Placebo

  • AzacitidineDrug

    Also called Vidaza

    Active comparator 75 mg/m2 Azacitidine for 7 days of each 28 day cycle, via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable

Study groups in NCT01873703
Study groupRoleWhat they receive
pracinostat plus azacitadineExperimental60 mg of pracinostat by mouth 3 times a week for 3 weeks followed by 1 week of rest repeated every 28 days. 75 mg/m2 Azacitidine for 7 days of each 28 day cycle, via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable
Receives: Drug: pracinostat; Drug: Azacitidine
Placebo with AzacitadinePlacebo comparatorPlacebo by mouth 3 times a week for 3 weeks followed by 1 week of rest repeated every 28 days. 75 mg/m2 Azacitidine for 7 days of each 28 day cycle, via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable
Receives: Drug: Placebo; Drug: Azacitidine

What is being measured

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

  1. Estimate efficacy

    Estimate the relative efficacy, measured by complete remission rate of treatment wiht pracinostat plus azacitidine versus placebo plus azacitidine

    Measured: 6 months

Secondary outcomes (7)

  1. Overall response rate

    Estimate the overall response rate [ORR = CR + complete remission + partial response (PR)]

    Measured: 6 months

  2. Hematologic Improvement

    Estimate the overall hematologic improvement (HI) response rate by review of hematologic lab values each cycle including bone marrow blast counts, platelets and erythrocytes.

    Measured: 6 months

  3. Duration of response

    Estimate the duration of response

    Measured: 6 months

  4. Progression free survival

    Estimate the progression-free survival (PFS) duration of pracinostat plus azacitidine and the relative benefit of that combination versus placebo plus azacitidine as assessed by the PFS hazard ratio

    Measured: 12 months

  5. Rate of leukemic transformation

    Estimate the rate of leukemic transformation at landmark time points (6 months, 12 months, 18 months, and 24 months) using clinical review of hematologic lab counts each cycle

    Measured: 6 - 24 months

  6. Overall survival

    Estimate the overall survival (OS) duration of pracinostat plus azacitidine and the relative benefit of that combination versus placebo plus azacitidine as assessed by the OS hazard ratio

    Measured: 6-24 months

  7. AE profile

    Assess the adverse event (AE) profile of pracinostat and placebo when combined with azacitidine by clinical review of safety events by grade, relationship and event outcomes.

    Measured: 12 months

Timeline

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

  1. First posted10 Jun 2013 (estimated)
  2. Study startJun 2013
  3. Primary completionNov 2015
  4. Study completionNov 2016

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
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
102 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01873703
Sponsor's protocol ID
MEI-003
Status last verified
Sep 2018
First submitted
22 May 2013
Last updated
13 Sep 2018
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing pracinostat and Azacitidine in people with Myelodysplastic Syndrome, run by Helsinn Healthcare SA at 24 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 15 inclusion and 19 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 13 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 24 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. Some participants in this study may receive a placebo. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Estimate efficacy (time frame: 6 months). 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 NCT01873703.

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 13 Sep 2018.

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