Myelodysplastic SyndromesPhase 2NCT03151304
A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes
A Phase 2 study testing Pracinostat and Azacitidine in people with Myelodysplastic Syndromes, run by Helsinn Healthcare SA at 25 sites in United States.
Trial summary
This is a Phase 2, two-stage study of the safety and efficacy of pracinostat in combination with azacitidine in patients with IPSS-R high and very high risk myelodysplastic syndrome (MDS) who are previously untreated with hypomethylating agents (HMAs). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Pracinostat and Azacitidine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 25 sitesUnited States
- Study dates
- 1 Jun 2017to 1 Dec 2020
- 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
- Female or male subjects ≥18 years-of-age.
- Histologically or cytologically documented diagnosis of MDS according to the World Health Organization (WHO) classification (Vardiman 2009, Arber 2016) with >5% and <20% bone marrow blasts by morphology and a peripheral white blood cell (WBC) count of <20,000/μL
- If WBC ≥20,000/μL, cytoreduction with hydroxyurea is permitted prior to enrollment.
- chronic myelomonocytic leukemia CMML-1 and CMML-2 subtypes
- Classified as high or very high risk according to the Revised International Prognostic Scoring System (IPSS-R) risk category. CMML-1 and CMML-2 subtypes will be considered high-risk MDS and will not require IPSS-r scoring
- Bone marrow biopsy (BMBx) and/or aspirate within 28 days prior to first study treatment.
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- Clinical indication for treatment with azacitidine.
- Previously untreated with HMAs (prior therapy with transfusions, hematopoietic growth factors, or immunosuppressive therapy is allowed).
- subjects who require the start of an HMA (e.g., azacitidine) due to progressing MDS may receive up to 1 cycle of azacitidine within 30 days prior to planned first dose (Cycle 1 Day 1)
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
- Adequate organ function as evidenced by:
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 × the upper limit of normal (ULN).
- Total bilirubin ≤1.5 × ULN or total bilirubin of ≤2 mg/dL, whichever is higher. Total bilirubin < 3 x ULN for patients with Gilbert-Meulengracht Syndrome
- Serum creatinine <1.5 mg/dL, or creatinine clearance>40 mL/min.
- QTcF interval ≤450 msec using the mean of triplicate electrocardiograms (ECGs).
- Female subjects of childbearing potential and male subjects with female partners of childbearing potential are required to use two forms of acceptable contraception, including one barrier method, during their participation in the study and for 30 days following last dose. Female subjects of childbearing potential must not be breastfeeding, or planning to breastfeed, and must have a negative pregnancy test ≤7 days before first study drug administration.
- Male subjects must also refrain from donating sperm during their participation in the study.
- Voluntary written informed consent before performance of any study-related procedure not part of normal medical care.
- Have the willingness and ability to understand the nature of this study and to comply with the study and follow-up procedures.
Who cannot join
- Bone marrow blasts ≥20%, indicating a diagnosis of acute myeloid leukemia (AML).
- 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 enrollment, whichever is longer.
- Hydroxyurea within 48 hours prior to first day of 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 28 days prior to first study treatment.
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- Subjects who have not recovered from side effects of previous therapy.
- Cardiopulmonary function criteria:
- Current unstable arrhythmia requiring treatment.
- History of symptomatic congestive heart failure (New York Heart Association [NYHA] Class III or IV).
- History of myocardial infarction, pulmonary embolism or cerebrovascular accident within 6 months of enrollment.
- Current unstable angina.
- Prior treatment for MDS with histone deacetylase (HDAC) inhibitors Zolinza (vorinostat), Belenodaq (belinostat), Farydak (panobinostat), Istodax (romidepsin/depsipetide), or investigational agent with significant action as an HDAC inhibitor.
- Clinical evidence of central nervous system involvement.
- Subjects 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).
- Uncontrolled infection with human immunodeficiency virus (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, non-melanomatous skin cancer, or malignancies treated with curative intent and no evidence of active disease in prior 12 months and felt to be low risk for recurrence. Other malignancies may be considered after consultation with the Medical Monitor
- An unwillingness or inability (including breastfeeding women, prohibited concomitant medications, uncontrolled infections, psychological, familial, sociological, or geographical conditions) to comply with study and/or follow-up procedures as outlined in the protocol
- Known hypersensitivity to any components of pracinostat, azacitidine or mannitol
- Current smoking or vaporizing of tobacco or cannabis-related products (use of patches, chewing tobacco, or nicotine gum is permitted). Subjects who stopped smoking at least 8 days prior to first pracinostat dosing can be, provided they refrain from smoking during the whole study.
Trial locations and contacts
25 sites in 1 country.
25 sites in 1 country
United States25 sites
New Mexico Cancer care Alliance
Albuquerque, New Mexico, 87131
Georgia Cancer Center at Augusta University
Augusta, Georgia, 30912
georgia cancer Center
Augusta, Georgia, 30912
RCCA MD LLC (The Center for Cancer and Blood Disorders)
Bethesda, Maryland, 20817
UVA Health System Division of Hematology & Oncology
Charlottesville, Virginia, 22908
Oncology Hematology Care
Cincinnati, Ohio, 45242
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Pontchartrain cancer Center
Covington, Louisiana, 70433
UT Southwestern Medical Center
Dallas, Texas, 75390
City of Hope
Duarte, California, 91010
Duke University Medical Center
Durham, North Carolina, 27710
Michigan Center of Medical Research
Farmington Hills, Michigan, 48334
Southeastern Medical Oncology Center
Goldsboro, North Carolina, 27534
Michigan State University, Breslin Cancer Center
Lansing, Michigan, 48910
Cancer Centers of Southwest Oklahoma
Lawton, Oklahoma, 73501
Universityof Wisconsin Clinical Science Center
Madison, Wisconsin, 53792
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
university of minnesota medical Center, Fairview
Minneapolis, Minnesota, 55455
Providence Portland Medical center
Portland, Oregon, 97213
University of Rochester Medical Center
Rochester, New York, 14642
Scripps Cancer Center-Mercy
San Diego, California, 92103
Swedish Cancer Institute
Seattle, Washington, 98104
Mercy Medical Research Institute
Springfield, Missouri, 65807
Stony Brook University
Stony Brook, New York, 11794
Oklahoma Cancer Specialists and Research Institute
Tulsa, Oklahoma, 74146
About this study
Official title: A Two-Stage, Open-Label Phase 2 Study of Pracinostat and Azacitidine in Patients With IPSS-R High and Very High Risk Myelodysplastic Syndromes Previously Untreated With Hypomethylating Agents
This is a Phase 2, two-stage study of the safety and efficacy of pracinostat in combination with azacitidine in patients with IPSS-R high and very high risk myelodysplastic syndrome (MDS) who are previously untreated with hypomethylating agents (HMAs). Enrollment in this study will be limited to high/very high risk MDS because this group represents the highest unmet need, with median survival of less than 18 months.
Stage 1a will be conducted as an open-label single arm in up to 40 subjects to assess if this regimen with a lower pracinostat dose results in a discontinuation rate that meets a predefined threshold and in efficacy that justifies expansion of enrollment into Stage 1b.
Read more
A discontinuation rate of approximately ≤10% in Stage 1a, a rate comparable to that observed with azacitidine alone in study MEI-003 (NCT01873703), supports expansion into Stage 1b.
Stage 1b will be conducted as expansion of stage 1a. Approximately 20 additional subjects will be enrolled. Study drugs should be continued until disease progression or intolerable toxicity. It is important to note that the median time to achieving a response with azacitidine alone is 4 to 5 months. Furthermore, the median time to achieving a Complete Response (CR) in study MEI-003 (NCT01873703) was 4 cycles. Therefore, early (<6 months) discontinuation of trial therapy for 'no response' should be avoided. The Medical Monitor should be contacted prior to discontinuing a subject from the study to discuss the rationale for discontinuation.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Pracinostat, Azacitidine
- Keywords
- MDS
- 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
45 mg capsule
AzacitidineDrug
SC or IV injection
| Study group | Role | What they receive |
|---|---|---|
| Stage 1a and 1b open-label pracinostat plus azacitidine | Experimental | open-label single arm pracinostat plus azacitidine. Pracinostat: 45 mg administered orally 3 days each week for 3 consecutive weeks, followed by 1 week of rest, in 28-day cycles.
In later cycles (i.e., after Cycle 4), pracinostat dose reduction to 45 mg orally 3 days each week × 2 weeks (instead of 3 weeks) or dose interruption is allowed to manage toxicity such as fatigue, gastrointestinal toxicity, or myelosuppression.
Azacitidine: 75 mg/m2 for 7 days of each 28-day cycle. Administration will occur by subcutaneous (SC) injection, or IV infusion if SC injections are not tolerated, on one of two schedules:
* Schedule 1 - daily therapy on Days 1 through 7
* Schedule 2 - 5-2-2 schedule in which subjects receive azacitidine for 5 consecutive days (Days 1 through 5) with rest on Days 6 and 7, and resume azacitidine dosing the first two days of the next week (Days 8 and 9) of each 28-day cycle Receives: Drug: Pracinostat; Drug: Azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
Percentage of subjects with confirmed complete remission (CR), partial remission (PR) and marrow CR, as per modified International Working Group (IWG) criteria:
CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hemoglobin (Hb) ≥11 g/dL; Platelets ≥100 × 10^9/L; Neutrophils ≥1.0 × 10^9/L; Blasts 0%.
PR: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by ≥ 50% over pre-treatment but still >5%; Cellularity and morphology not relevant Marrow CR: Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pre-treatment
Measured: 36 months
Secondary outcomes (12)
Complete Response (CR) Rate
Percentage of subjects with confirmed CR (i.e., 2 CRs at least 28 days apart) as per modified IWG criteria:
CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hb ≥11 g/dL; Platelets ≥100 × 109/L; Neutrophils ≥1.0 × 109/L; Blasts 0%.
Measured: 36 months
Overall Hematologic Improvement (HI) Response Rate
Percentage of subjects demonstrating major hematologic improvement according to modified IWG:
Erythroid response (pre-treatment, <11 g/dL): Hb increase by ≥1.5 g/dL; Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks compared with the pre-treatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of ≤9.0 g/dL pre-treatment will count in the RBC transfusion response evaluation.
Platelet response (pre-treatment, <100 × 10^9/L): Absolute increase of ≥30 × 10^9/L for patients starting with >20 × 10^9/L platelets; Increase from <20 × 10^9/L to >20 × 10^9/L and by at least 100%.
Neutrophil response (pre-treatment, <1.0 × 10^9/L): At least 100% increase and an absolute increase >0.5 × 10^9/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 Hb by≥1.5 g/dL
- Transfusion dependence
Measured: 36 months
Clinical Benefit Rate
Percentage of subjects with confirmed CR, PR, Marrow CR, and HI with clinical benefit rate, defines as rate of CR + PR + HI + Marrow CR. All subjects who achieve hematologic CR, PR, marrow CR, or hematologic improvement on the erythrocytic lineage per modified IWG response criteria will be considered responders
Measured: 36 months
Rate of Cytogenetic CR
Percentage of subjects with confirmed CR by cytogenetic assessment. Complete cytogenetic response is defined per modified IWG response criteria:
Complete: Disappearance of the chromosomal abnormality without appearance of new ones Partial: At least 50% reduction of the chromosomal abnormality
Measured: 36 months
Duration of Response (DoR)
For subjects who have achieved CR, PR, Marrow CR, or HI, DoR is defined as the time from the initial determination of response to the time of disease progression or death on study, whichever occurs first.
Measured: 36 months
Rate of Leukemic Transformation
Percentage of subjects with leukemic transformation at landmark time point of 6 months
Measured: 6 months
Event-free Survival (EFS)
time from the first day of study drug administration (Day 1) to failure or death from any cause
Measured: 36 months
Progression-free Survival (PFS)
time from the first day of study drug administration (Day 1) to disease recurrence or progression as defined by the IWG criteria, or death on study:
Disease progression for subjects with:
Less than 5% blasts: ≥50% increase in blasts to >5% blasts 5%-10% blasts: ≥50% increase to >10% blasts 10%-20% blasts: ≥50% increase to >20% blasts 20%-30% blasts: ≥50% increase to >30% blasts
Any of the following:
At least 50% decrement from maximum remission/response in granulocytes or platelets Reduction in Hb by ≥2 g/dL Transfusion dependence
Measured: 36 months
Overall Survival (OS)
time from the first day of study drug administration (Day 1) to death on study
Measured: form day 1 to death on study, assessed up to 36 months
Rate of Leukemic Transformation
Percentage of subjects with leukemic transformation at landmark time point of 12 months
Measured: 12 months
Rate of Leukemic Transformation
Percentage of subjects with leukemic transformation at landmark time point of 18 months
Measured: 18 months
Rate of Leukemic Transformation
Percentage of subjects with leukemic transformation at landmark time point of 24 months
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2017
- Study start1 Jun 2017
- Primary completion1 Dec 2020
- Study completion1 Dec 2020
Why it stopped: Sponsor's decision as at the completion of the primary analysis, the data were considered mature
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.
- 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
- Model description
- Two-Stage, Open-Label
- Masking
- None (open label)
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Helsinn Healthcare SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Richard Ghalie, MD, Study director, MEI PharmaEhab Atallah, MD, Study chair, Medical College of Wisconsin adn Froedtert Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03151304
- Sponsor's protocol ID
- MEI-011
- Status last verified
- Feb 2022
- First submitted
- 5 May 2017
- Last updated
- 2 Mar 2022
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two-Stage, Open-Label Phase 2 Study of Pracinostat and Azacitidine in Patients With IPSS-R High and Very High Risk Myelodysplastic Syndromes Previously Untreated With Hypomethylating Agents
- ClinicalTrials.gov ID
- NCT03151304
- Acronym / sponsor ID
- MEI-011
- Conditions
- Myelodysplastic Syndromes
- Keywords
- MDS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor's decision as at the completion of the primary analysis, the data were considered mature
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Helsinn Healthcare SAIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2017 (actual)
- Primary completion
- 1 Dec 2020 (actual)
- Study completion
- 1 Dec 2020 (actual)
- First posted
- 12 May 2017 (actual)
- Results
- Posted 2 Mar 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 2, two-stage study of the safety and efficacy of pracinostat in combination with azacitidine in patients with IPSS-R high and very high risk myelodysplastic syndrome (MDS) who are previously untreated with hypomethylating agents (HMAs). Enrollment in this study will be limited to high/very high risk MDS because this group represents the highest unmet need, with median survival of less than 18 months.
Stage 1a will be conducted as an open-label single arm in up to 40 subjects to assess if this regimen with a lower pracinostat dose results in a discontinuation rate that meets a predefined threshold and in efficacy that justifies expansion of enrollment into Stage 1b.
A discontinuation rate of approximately ≤10% in Stage 1a, a rate comparable to that observed with azacitidine alone in study MEI-003 (NCT01873703), supports expansion into Stage 1b.
Stage 1b will be conducted as expansion of stage 1a. Approximately 20 additional subjects will be enrolled. Study drugs should be continued until disease progression or intolerable toxicity. It is important to note that the median time to achieving a response with azacitidine alone is 4 to 5 months. Furthermore, the median time to achieving a Complete Response (CR) in study MEI-003 (NCT01873703) was 4 cycles. Therefore, early (<6 months) discontinuation of trial therapy for 'no response' should be avoided. The Medical Monitor should be contacted prior to discontinuing a subject from the study to discuss the rationale for discontinuation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Two-Stage, Open-Label
| Arm | Type | Receives |
|---|---|---|
| Stage 1a and 1b open-label pracinostat plus azacitidine | Experimental | Drug: Pracinostat, Drug: Azacitidineopen-label single arm pracinostat plus azacitidine. Pracinostat: 45 mg administered orally 3 days each week for 3 consecutive weeks, followed by 1 week of rest, in 28-day cycles. In later cycles (i.e., after Cycle 4), pracinostat dose reduction to 45 mg orally 3 days each week × 2 weeks (instead of 3 weeks) or dose interruption is allowed to manage toxicity such as fatigue, gastrointestinal toxicity, or myelosuppression. Azacitidine: 75 mg/m2 for 7 days of each 28-day cycle. Administration will occur by subcutaneous (SC) injection, or IV infusion if SC injections are not tolerated, on one of two schedules: * Schedule 1 - daily therapy on Days 1 through 7 * Schedule 2 - 5-2-2 schedule in which subjects receive azacitidine for 5 consecutive days (Days 1 through 5) with rest on Days 6 and 7, and resume azacitidine dosing the first two days of the next week (Days 8 and 9) of each 28-day cycle |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Female or male subjects ≥18 years-of-age.
- Histologically or cytologically documented diagnosis of MDS according to the World Health Organization (WHO) classification (Vardiman 2009, Arber 2016) with >5% and <20% bone marrow blasts by morphology and a peripheral white blood cell (WBC) count of <20,000/μL
- Classified as high or very high risk according to the Revised International Prognostic Scoring System (IPSS-R) risk category. CMML-1 and CMML-2 subtypes will be considered high-risk MDS and will not require IPSS-r scoring
- Bone marrow biopsy (BMBx) and/or aspirate within 28 days prior to first study treatment.
- Clinical indication for treatment with azacitidine.
Key exclusion criteria (21 listed)
- Bone marrow blasts ≥20%, indicating a diagnosis of acute myeloid leukemia (AML).
- Received any of the following within the specified time frame prior to administration of study medication:
- Subjects who have not recovered from side effects of previous therapy.
- Cardiopulmonary function criteria:
- Prior treatment for MDS with histone deacetylase (HDAC) inhibitors Zolinza (vorinostat), Belenodaq (belinostat), Farydak (panobinostat), Istodax (romidepsin/depsipetide), or investigational agent with significant action as an HDAC inhibitor.
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 |
|---|---|---|
| PracinostatAlso: SB93945 mg capsule | Drug | Stage 1a and 1b open-label pracinostat plus azacitidine |
| AzacitidineSC or IV injection | Drug | Stage 1a and 1b open-label pracinostat plus azacitidine |
Primary outcome
Overall Response Rate (ORR)
Time frame: 36 months
Percentage of subjects with confirmed complete remission (CR), partial remission (PR) and marrow CR, as per modified International Working Group (IWG) criteria: CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hemoglobin (Hb) ≥11 g/dL; Platelets ≥100 × 10^9/L; Neutrophils ≥1.0 × 10^9/L; Blasts 0%. PR: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by ≥ 50% over pre-treatment but still >5%; Cellularity and morphology not relevant Marrow CR: Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pre-treatment
12 secondary outcomes
Complete Response (CR) Rate
Time frame: 36 months
Overall Hematologic Improvement (HI) Response Rate
Time frame: 36 months
Clinical Benefit Rate
Time frame: 36 months
Rate of Cytogenetic CR
Time frame: 36 months
Duration of Response (DoR)
Time frame: 36 months
Rate of Leukemic Transformation
Time frame: 6 months
Event-free Survival (EFS)
Time frame: 36 months
Progression-free Survival (PFS)
Time frame: 36 months
Overall Survival (OS)
Time frame: form day 1 to death on study, assessed up to 36 months
Rate of Leukemic Transformation
Time frame: 12 months
Rate of Leukemic Transformation
Time frame: 18 months
Rate of Leukemic Transformation
Time frame: 24 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2022.
- 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.
Richard Ghalie, MD study director
MEI Pharma
Ehab Atallah, MD study chair
Medical College of Wisconsin adn Froedtert Hospital
25 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 Pracinostat and Azacitidine in people with Myelodysplastic Syndromes, run by Helsinn Healthcare SA at 25 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 19 inclusion and 21 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 2 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 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 2 Mar 2022). 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 (ORR) (time frame: 36 months). 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 2 Mar 2022; AzTrials last refreshed its copy on 7 Nov 2026.