This study has been transitioned to CTIS with ID 2023-507153-16-00 check the CTIS register for the current data. To assess the efficacy of venetoclax in combination with AZA compared to placebo with AZA in treatment-naive higher-risk MDS.
ID
Source
Brief title
Condition
- Leukaemias
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Overall Survival (OS)
Secondary outcome
- Complete remission (CR)
- Overall hematological improvement (HI) (HI-platelet, HI-neutrophil, or
HI-erythroid)
- Red blood cell (RBC) and platelet transfusion independence for subjects who
are transfusion dependent on RBC and/or platelet at baseline
- Change from baseline in fatigue, as measured by the Patient-Reported Outcomes
Measurement Information System (PROMIS)-Fatigue SF 7a
- Time to deterioration in physical functioning, as measured by the physical
functioning domain of EORTC QLQ-C30
- Overall response (OR) defined as CR + partial response (PR)
- Modified overall response (mOR) defined as CR + PR + marrow CR (mCR).
Background summary
Myelodysplastic Syndrome (MDS) is a group of disorders that gradually affect
the ability of a person's bone marrow (semi-liquid tissue present in many bones
like backbones) to produce normal blood cells. Some people with MDS have a risk
of the disease progressing to acute myeloid leukemia (AML), and a risk of death
from the disease itself.
Symptoms of MDS include fatigue, shortness of breath, unusual paleness due to
anemia (low red blood cell count), easy or unusual bruising, and red spots just
beneath the skin caused by bleeding. The purpose of this study is to see how
safe and effective venetoclax and azacitidine (AZA) combination are when
compared to AZA and a placebo (contains no medicine), in participants with
newly diagnosed higher-risk MDS.
Study objective
This study has been transitioned to CTIS with ID 2023-507153-16-00 check the CTIS register for the current data.
To assess the efficacy of venetoclax in combination with AZA compared to
placebo with AZA in treatment-naive higher-risk MDS.
Study design
This is a randomized, double-blind, placebo-controlled study.
Intervention
Participants in one arm will receive oral doses of venetoclax tablet and
intravenous (infusion in the vein) or subcutaneous (given under the skin) AZA
solution.
Participants in another arm will receive oral doses of placebo tablet and
intravenous or subcutaneous AZA solution.
Study burden and risks
There may be higher treatment burden for participants in this trial compared to
their standard of care. Participants will attend regular visits during the
course of the study at a hospital or clinic. The effect of the treatment will
be checked by medical assessments, blood and bone marrow tests, checking for
side effects, and completing questionnaires.
Knollstrasse 50
Ludwigshafen 67061
DE
Knollstrasse 50
Ludwigshafen 67061
DE
Listed location countries
Age
Inclusion criteria
Adult male or female, at least 18 years old. Diagnosis of MDS according to the
2016 WHO classification with presence of < 20% bone marrow blasts per bone
marrow biopsy/aspirate at screening. Subject meets the following disease
activity criteria:
* Overall IPSS-R score > 3 (intermediate, high, or very high; Appendix E);
* Eastern Cooperative Oncology Group (ECOG) performance status <= 2;
* HSCT eligible with no pre-arranged HSCT at the time of Study Day 1, or HSCT
ineligible without plan for HSCT at the time of Study Day 1. No prior therapy
for MDS with any hypomethylating agent (for example, azacitidine, decitabine),
chemotherapy or allogeneic stem cell transplantation.
Exclusion criteria
No previous diagnosis of:
* Therapy-related MDS (t-MDS)
* MDS evolving from a pre-existing myeloproliferative neoplasm (MPN)
* MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic
myeloid leukemia (aCML), juvenile myelomonocytic leukemia (JMML) and
unclassifiable MDS/MPN
* No prior therapy for MDS with any hypomethylating agent (for example,
azacitidine, decitabine), chemotherapy or allogeneic stem cell transplantation.
Lenalidomide, anti-thymocyte globulin (ATG), and cyclosporin are also excluded
as these are considered disease-modifying agents.
* No known active SARS-CoV-2 infection. If a subject has signs/symptoms of
SARS-CoV-2 infection, they should undergo molecular (e.g., PCR) testing to rule
out SARS-CoV-2 infection.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EU-CTR | CTIS2023-507153-16-00 |
| EudraCT | EUCTR2020-000744-55-NL |
| ClinicalTrials.gov | NCT04401748 |
| CCMO | NL74107.056.20 |