Myelodysplastic SyndromesNCT04484363
Expanded Access Program With Pevonedistat (Given With Azacitidine) for Adults With Higher-risk Myelodysplastic Syndromes
A study testing Pevonedistat and Azacitidine in people with Myelodysplastic Syndromes, run by Takeda.
Trial summary
Participants in the expanded access program are adults with higher-risk myelodysplastic syndromes who have no other treatment options available. The main aim of this program is to allow participants to have access to pevonedistat before FDA approval. This program will take place in the United States. 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?
- Pevonedistat and Azacitidine
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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
- Adult, Older adult
Who may be able to join
- Has morphologically confirmed diagnosis of MDS.
- With MDS have one of the following Prognostic Risk Categories, based on the Revised International Prognostic Scoring System (IPSS-R)
- Very high (greater than [>] 6 points).
- High (>4.5-6 points).
- Intermediate (>3-4.5 points): a participant determined to be in the Intermediate Prognostic Risk Category is only allowable in the setting of greater than or equal to (>=) 5 percent (%) bone marrow myeloblasts.
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2.
- Has never been treated with Azacitidine, or, has been treated with Azacitidine, two or fewer cycles of Azacitidine have been administered, and has not relapsed while being treated with Azacitidine or failed Azacitidine treatment.
Who cannot join
- Has previous treatment for HR MDS with chemotherapy or other antineoplastic agents including hypomethylating agent (HMAs) such as decitabine or more than two cycles of azacitidine.
- Previous treatment with lenalidomide is permitted, except that lenalidomide may not be given within 8 weeks of first dose of drug.
- Has acute promyelocytic leukemia as diagnosed by morphologic examination of bone marrow, by fluorescent in situ hybridization or cytogenetics of peripheral blood or bone marrow, or by other accepted analysis.
- Has either clinical evidence of or history of central nervous system involvement by acute myelogenous leukemia (AML).
- Active uncontrolled infection or severe infectious disease, such as severe pneumonia, meningitis, or septicemia.
- Has prothrombin time (PT) or activated partial thromboplastin time (aPTT) >1.5*upper limit of normal (ULN) or active uncontrolled coagulopathy or bleeding disorder. Participants therapeutically anticoagulated with warfarin, direct thrombin inhibitors, direct factor Xa inhibitors, or heparin are excluded from enrollment.
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- Has known hepatitis B surface antigen seropositivity, or known or suspected active hepatitis C infection. Note: Participants who have isolated positive hepatitis B core antibody (that is, in the setting of negative hepatitis B surface antigen and negative hepatitis B surface antibody) must have an undetectable hepatitis B viral load. Participants who have positive hepatitis C antibody may be included if they have an undetectable hepatitis C viral load.
- Has known hepatic cirrhosis or severe preexisting hepatic impairment.
- Has known cardiopulmonary disease defined as unstable angina, clinically significant arrhythmia, congestive heart failure (New York Heart Association Class III or IV), and/or myocardial infarction within 6 months before first dose, or severe pulmonary hypertension.
- Has treatment with strong cytochrome P 3A (CYP3A) inducers within 14 days before the first dose of pevonedistat.
- .
About this study
Official title: Expanded Access Program: Pevonedistat (in Combination With Azacitidine) for the First-line Treatment of Higher Risk Myelodysplastic Syndromes
Participants in the expanded access program are adults with higher-risk myelodysplastic syndromes who have no other treatment options available.
The main aim of this program is to allow participants to have access to pevonedistat before FDA approval.
This program will take place in the United States.
Read the detailed description
This is an expanded access program in which the drug being tested is called pevonedistat, which is used in combination with azcitidine. This study will provide expanded access of pevonedistat (in combination with azacitidine) for the first-line treatment to participants with HR-MDS and option of real world data (RWD) collection for the benefit of future participants.
All participants will receive azacitidine via intravenous or subcutaneous route in combination with pevonedistat intravenous infusion.
This multi-center trial will be conducted in the United States. Participant will continue treatment until benefit is no longer derived from the treatment (that is, until disease progression, or treatment is no longer tolerable), the benefit-risk no longer favors the individual, an appropriate alternative therapy becomes available, the participant chooses to discontinue the treatment, or pevonedistat becomes commercially available.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Pevonedistat, Azacitidine
- Keywords
- Drug Therapy
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PevonedistatDrug
Pevonedistat 20 milligram per square meter (mg/m^2), intravenous infusion, on Days 1, 3, and 5 of repeated 28-day cycles.
AzacitidineDrug
Azacitidine 75 mg/m^2, intravenous or subcutaneous infusion, on Days 1 to 5, Days 8 and 9 of repeated 28-day cycles or Days 1 through 7 of repeated 28-day cycles.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by TakedaThe 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04484363
- Sponsor's protocol ID
- Pevonedistat-5005
- Status last verified
- Sep 2022
- First submitted
- 22 Jul 2020
- Last updated
- 30 Sep 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Program: Pevonedistat (in Combination With Azacitidine) for the First-line Treatment of Higher Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT04484363
- Acronym / sponsor ID
- Pevonedistat-5005
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Drug Therapy
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- First posted
- 23 Jul 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Participants in the expanded access program are adults with higher-risk myelodysplastic syndromes who have no other treatment options available.
The main aim of this program is to allow participants to have access to pevonedistat before FDA approval.
This program will take place in the United States.
Detailed description
This is an expanded access program in which the drug being tested is called pevonedistat, which is used in combination with azcitidine. This study will provide expanded access of pevonedistat (in combination with azacitidine) for the first-line treatment to participants with HR-MDS and option of real world data (RWD) collection for the benefit of future participants.
All participants will receive azacitidine via intravenous or subcutaneous route in combination with pevonedistat intravenous infusion.
This multi-center trial will be conducted in the United States. Participant will continue treatment until benefit is no longer derived from the treatment (that is, until disease progression, or treatment is no longer tolerable), the benefit-risk no longer favors the individual, an appropriate alternative therapy becomes available, the participant chooses to discontinue the treatment, or pevonedistat becomes commercially available.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Has morphologically confirmed diagnosis of MDS.
- With MDS have one of the following Prognostic Risk Categories, based on the Revised International Prognostic Scoring System (IPSS-R)
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2.
- Has never been treated with Azacitidine, or, has been treated with Azacitidine, two or fewer cycles of Azacitidine have been administered, and has not relapsed while being treated with Azacitidine or failed Azacitidine treatment.
Key exclusion criteria (11 listed)
- Has previous treatment for HR MDS with chemotherapy or other antineoplastic agents including hypomethylating agent (HMAs) such as decitabine or more than two cycles of azacitidine.
- Has acute promyelocytic leukemia as diagnosed by morphologic examination of bone marrow, by fluorescent in situ hybridization or cytogenetics of peripheral blood or bone marrow, or by other accepted analysis.
- Has either clinical evidence of or history of central nervous system involvement by acute myelogenous leukemia (AML).
- Active uncontrolled infection or severe infectious disease, such as severe pneumonia, meningitis, or septicemia.
- Has prothrombin time (PT) or activated partial thromboplastin time (aPTT) >1.5*upper limit of normal (ULN) or active uncontrolled coagulopathy or bleeding disorder. Participants therapeutically anticoagulated with warfarin, direct thrombin inhibitors, direct factor Xa inhibitors, or heparin are excluded from enrollment.
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 |
|---|---|---|
| PevonedistatPevonedistat 20 milligram per square meter (mg/m^2), intravenous infusion, on Days 1, 3, and 5 of repeated 28-day cycles. | Drug | – |
| AzacitidineAzacitidine 75 mg/m^2, intravenous or subcutaneous infusion, on Days 1 to 5, Days 8 and 9 of repeated 28-day cycles or Days 1 through 7 of repeated 28-day cycles. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2022.
- Related InfoLink provided by the sponsor.
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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.
Study Director study director
Takeda
Questions about this trial
What is this clinical trial studying?
A study testing Pevonedistat and Azacitidine in people with Myelodysplastic Syndromes, run by Takeda.
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 7 inclusion and 11 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: status not confirmed, as of the sponsor's last update on 30 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
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.
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 30 Sep 2022.