LeukemiaPhase 1NCT03066648
Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With AML or High Risk MDS
A Phase 1 study testing Decitabine, PDR001 and MBG453 in people with Leukemia and Leukemia, Myeloid, run by Novartis Pharmaceuticals at 11 sites in 8 countries.
Trial summary
To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and/or MBG453 in combination with decitabine or azacitidine in AML and intermediate or high- risk MDS patients, and to identify recommended doses for future studies. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Leukemia, Myeloid, Leukemia, Myeloid, Acute
- What is being tested?
- Decitabine, PDR001 and MBG453
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 241 participants
- Where
- 11 sitesin 8 countries
- Study dates
- 6 Jul 2017to 8 Sep 2023
- Phase
- Phase 1
- 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
- Written informed consent must be obtained prior to any screening procedures
- Male or female patients ≥ 18 years of age who present with one of the following:
- Arms 1-3:
- Relapsed/refractory AML following ≥1 prior therapies who have relapsed or exhibited refractory disease (primary failure) and are deemed by the investigator not to be candidates for standard therapy, including re-induction with cytarabine or other established chemotherapy regimens for patients with AML (patients who are suitable for standard re-induction chemotherapy or hematopoietic stem cell transplantation and willing to receive it are excluded)
- Newly diagnosed AML patients who are suitable for treatment with decitabine (patients who are suitable for standard induction chemotherapy or hematopoietic stem cell transplantation and willing to receive it are excluded)
- Intermediate or high risk MDS or MDS/MPN including CMML (patients who are suitable for standard re-induction chemotherapy or hematopoietic stem cell transplantation and willing to receive it are excluded)
Show all 16Show fewer
- Arms 4-5:
- Refractory / relapsed AML following ≥1 prior therapies (Arms 4a \& 5a)
- Intermediate or high risk MDS or MDS/MPN including CMML who have failed hypomethylating agent therapy (Arms 4b \& 5b) (Note: hypomethylating agent failure is defined as progressive disease on hypomethylating agent therapy or lack of clinically meaningful response as deemed by investigator after at least 4 cycles of hypomethylating agent therapy.)
- Arm 6:
- Newly diagnosed AML patients who are suitable for treatment with azacitidine (patients who are suitable for standard induction chemotherapy or hematopoietic stem cell transplantation and willing to receive it are excluded) (Arm 6a)
- Intermediate or high-risk MDS or MDS/MPN including CMML (patients who are suitable for standard induction chemotherapy or hematopoietic stem cell transplantation and willing to receive it are excluded) (Arm 6b)
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Patient must be a candidate for serial bone marrow aspirate and/or biopsy according to the institutions guidelines and be willing to undergo a bone marrow aspirate and/biopsy at screening, during and at the end of therapy on this study. Exceptions may be considered after documented discussion with Novartis.
- Arms 1-3: Patients must be fit for standard treatment with decitabine as determined by the investigator and as per local decitabine package insert.
- Arm 6: Patients must be fit for standard treatment with azacitidine as determined by the investigator and as per the local azacitidine package insert.
Who cannot join
- Arms 1-3 or Arm 6: Patients who have received prior hypomethylating agent treatment for AML or MDS.
- Patients with active, known or suspected autoimmune disease. Patients with vitiligo, type I diabetes, residual hypothyroidism only requiring hormone replacement, psoriasis not requiring systemic treatment or conditions not expected to recur should not be excluded.
- History of, or current drug-induced interstitial lung disease or pneumonitis grade ≥ 2.
- Patients who discontinued prior PD-1 or PD-L1 directed therapy due to a treatment related toxicity should not be included in the PDR001 containing arms of the study. Patients previously exposed to anti-PD-1/PD-L1 treatment who are adequately treated for skin rash or with replacement therapy for endocrinopathies should not be excluded.
- Systemic antineoplastic therapy (including cytotoxic chemotherapy, alphainterferon, kinase inhibitors or other targeted small molecules, and toxinimmunoconjugates) or any experimental therapy within 14 days or 5 half-lives, whichever is shorter, before the first dose of study treatment.
- Systemic chronic corticosteroid therapy (>10 mg/day prednisone or equivalent) or any immunosuppressive therapy within 7 days of first dose of study treatment. Topical, inhaled, nasal and ophthalmic steroids are allowed.
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
11 sites in 8 countries.
11 sites in 8 countries
United States3 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
MD Anderson Cancer Center
Houston, Texas, 77030
Oregon Health Sciences University Main Center
Portland, Oregon, 97239
Germany2 sites
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Jena, 07740
About this study
Official title: Phase 1b, Multi-arm, Open-label Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome
To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and/or MBG453 in combination with decitabine or azacitidine in AML and intermediate or high- risk MDS patients, and to identify recommended doses for future studies.
Read the detailed description
This is a phase 1b, multi-arm, open-label study in patients with acute myeloid leukemia (AML) or intermediate or high risk myelodysplastic syndrome (MDS). Patients with myelodysplastic-myeloproliferative neoplasms (MDS/MPN), including chronic myelomonocytic leukemia (CMML) could also be enrolled.
The study was comprised of six arms as described below. Arms 1-3 enrolled patients with newly diagnosed AML who were planned for non-intensive chemotherapy, relapsed/refractory (R/R) AML, or intermediate or high-risk MDS.
- Arm 1: Evaluation of a fixed dose of the standard of care agent decitabine in combination with fixed dose PDR001.
- Arm 2: Evaluation of a fixed dose of the standard of care agent decitabine in combination with escalating dose MBG453.
- Arm 3: Evaluation of a fixed dose of the standard of care agent decitabine in combination with fixed dose PDR001 and escalating dose MBG453.
Arms 4-5 enrolled patients with R/R AML or intermediate or high-risk MDS who had failed hypomethylating agent therapy.
- Arm 4: Evaluation of an escalating dose of MBG453
- Arm 5: Evaluation of an escalating dose of MBG453 in combination with fixed dose PDR001 Arm 6 enrolled patients with newly diagnosed AML who were planned for non-intensive chemotherapy, or intermediate or high-risk MDS.
- Arm 6: Evaluation of a fixed dose of the standard of care agent azacitidine in combination with an escalating dose of MBG453.
Patients received the assigned treatment until disease progression, unacceptable toxicity, start of a new anti-neoplastic therapy, discontinuation at the discretion of the investigator or patient, lost to follow-up, death, or the study termination, whichever occurred first.
- Conditions
- Leukemia, Leukemia, Myeloid, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Preleukemia, Bone Marrow Diseases
- Being tested
- Decitabine, PDR001, MBG453, Azacitidine
- Keywords
- Acute Myeloid Leukemia, Myelodysplastic syndromes, Chronic Myelomonocytic Leukemia
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Preleukemia, Bone Marrow Diseases, Hematologic Diseases, Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DecitabineDrug
Also called 5-aza-2'-deoxycytidine
Decitabine is a cytidine deoxynucleoside analogue that selectively inhibits DNA methyltransferases at low doses, resulting in gene promoter hypomethylation.
PDR001Drug
Also called spartalizumab
PDR001 is a high-affinity, ligand-blocking, humanized IgG4 monoclonal antibody directed against PD-1 that blocks the binding of PD-L1 and PD-L2.
MBG453Drug
Also called sabatolimab
MBG453 is a high-affinity, humanized anti-TIM-3 IgG4 monoclonal antibody which blocks the binding of TIM-3 to phosphatidylserine (PtdSer).
AzacitidineDrug
Also called 5-azacytidine
Azacitidine (5-azacytidine) is a cytidine nucleoside analogue that selectively inhibits DNA methyltransferases at low doses, resulting in gene promoter hypomethylation
| Study group | Role | What they receive |
|---|---|---|
| Decitabine and PDR001 | Experimental | Decitabine in combination with PDR001 Receives: Drug: Decitabine; Drug: PDR001 |
| Decitabine and MBG453 | Experimental | Decitabine in combination with MBG453 Receives: Drug: Decitabine; Drug: MBG453 |
| Decitabine, PDR001 and MBG453 | Experimental | Decitabine in combination with PDR001 and MBG453 Receives: Drug: Decitabine; Drug: PDR001; Drug: MBG453 |
| MBG453 | Experimental | MBG453 alone Receives: Drug: MBG453 |
| MBG453 and PDR001 | Experimental | MBG453 in combination with PDR001 Receives: Drug: PDR001; Drug: MBG453 |
| Azacitidine and MBG453 | Experimental | Azacitidine in combination with MBG453 Receives: Drug: MBG453; Drug: Azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of MBG453 single agent treatment or MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine or azacitidine.
Incidence and severity of AEs and SAEs
Measured: 24 months
Incidence of Dose Limiting Toxicities (DLTs)
The incidence of DLTs during the first two cycles of treatment with MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine.
Measured: 2 months
Incidence of Dose Limiting Toxicities (DLTs)
The incidence of DLTs during the first cycle of MBG453 single agent treatment or during the first two cycles of treatment with MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine.
Measured: 1 month
Tolerability of MBG453 single agent treatment or MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine or azacitidine.
Incidence and severity of AEs and SAEs
Measured: 24 months
Secondary outcomes (10)
AUC of PDR001, MBG453, decitabine and azacitidine.
AUC
Measured: 24 months
Cmax of PDR001, MBG453, decitabine and azacitidine
Cmax
Measured: 24 months
Tmax of PDR001, MBG453, decitabine and azacitidine
Tmax
Measured: 24 months
Half-life of PDR001, MBG453, decitabine and azacitidine
Half-life
Measured: 24 months
Overall Response Rate (ORR)
Determine ORR in each arm of the study
Measured: 24 months
Best Overall Response (BOR)
Determine BOR in each arm of the study
Measured: 24 months
Progression Free Survival (PFS)
Determine PFS in each arm of the study
Measured: 24 months
Time to Progression (TTP)
Determine TTP in each arm of the study
Measured: 24 months
Duration of Response (DOR)
Determine DOR in each arm of the study
Measured: 24 months
Number of participants with anti-PDR001 and anti-MBG453 antibodies
Presence of anti-PDR001 and anti-MBG453 antibodies.
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 posted28 Feb 2017
- Study start6 Jul 2017
- Primary completion8 Sep 2023
- Study completion8 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 241 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03066648
- Sponsor's protocol ID
- CPDR001X2105
- Other IDs
- 2016-005060-33, 2016
- Status last verified
- May 2025
- First submitted
- 18 Feb 2017
- Last updated
- 18 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1b, Multi-arm, Open-label Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT03066648
- Acronym / sponsor ID
- CPDR001X2105
- Conditions
- Leukemia, Leukemia, Myeloid, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Preleukemia, Bone Marrow Diseases
- Keywords
- Acute Myeloid Leukemia, Myelodysplastic syndromes, Chronic Myelomonocytic Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 241 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 6 Jul 2017 (actual)
- Primary completion
- 8 Sep 2023 (actual)
- Study completion
- 8 Sep 2023 (actual)
- First posted
- 28 Feb 2017 (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.
To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and/or MBG453 in combination with decitabine or azacitidine in AML and intermediate or high- risk MDS patients, and to identify recommended doses for future studies.
Detailed description
This is a phase 1b, multi-arm, open-label study in patients with acute myeloid leukemia (AML) or intermediate or high risk myelodysplastic syndrome (MDS). Patients with myelodysplastic-myeloproliferative neoplasms (MDS/MPN), including chronic myelomonocytic leukemia (CMML) could also be enrolled.
The study was comprised of six arms as described below. Arms 1-3 enrolled patients with newly diagnosed AML who were planned for non-intensive chemotherapy, relapsed/refractory (R/R) AML, or intermediate or high-risk MDS.
- Arm 1: Evaluation of a fixed dose of the standard of care agent decitabine in combination with fixed dose PDR001.
- Arm 2: Evaluation of a fixed dose of the standard of care agent decitabine in combination with escalating dose MBG453.
- Arm 3: Evaluation of a fixed dose of the standard of care agent decitabine in combination with fixed dose PDR001 and escalating dose MBG453.
Arms 4-5 enrolled patients with R/R AML or intermediate or high-risk MDS who had failed hypomethylating agent therapy.
- Arm 4: Evaluation of an escalating dose of MBG453
- Arm 5: Evaluation of an escalating dose of MBG453 in combination with fixed dose PDR001 Arm 6 enrolled patients with newly diagnosed AML who were planned for non-intensive chemotherapy, or intermediate or high-risk MDS.
- Arm 6: Evaluation of a fixed dose of the standard of care agent azacitidine in combination with an escalating dose of MBG453.
Patients received the assigned treatment until disease progression, unacceptable toxicity, start of a new anti-neoplastic therapy, discontinuation at the discretion of the investigator or patient, lost to follow-up, death, or the study termination, whichever occurred first.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Decitabine and PDR001 | Experimental | Drug: Decitabine, Drug: PDR001Decitabine in combination with PDR001 |
| Decitabine and MBG453 | Experimental | Drug: Decitabine, Drug: MBG453Decitabine in combination with MBG453 |
| Decitabine, PDR001 and MBG453 | Experimental | Drug: Decitabine, Drug: PDR001, Drug: MBG453Decitabine in combination with PDR001 and MBG453 |
| MBG453 | Experimental | Drug: MBG453MBG453 alone |
| MBG453 and PDR001 | Experimental | Drug: PDR001, Drug: MBG453MBG453 in combination with PDR001 |
| Azacitidine and MBG453 | Experimental | Drug: MBG453, Drug: AzacitidineAzacitidine in combination with MBG453 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Written informed consent must be obtained prior to any screening procedures
- Male or female patients ≥ 18 years of age who present with one of the following:
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Patient must be a candidate for serial bone marrow aspirate and/or biopsy according to the institutions guidelines and be willing to undergo a bone marrow aspirate and/biopsy at screening, during and at the end of therapy on this study. Exceptions may be considered after documented discussion with Novartis.
- Arms 1-3: Patients must be fit for standard treatment with decitabine as determined by the investigator and as per local decitabine package insert.
Key exclusion criteria (7 listed)
- Arms 1-3 or Arm 6: Patients who have received prior hypomethylating agent treatment for AML or MDS.
- Patients with active, known or suspected autoimmune disease. Patients with vitiligo, type I diabetes, residual hypothyroidism only requiring hormone replacement, psoriasis not requiring systemic treatment or conditions not expected to recur should not be excluded.
- History of, or current drug-induced interstitial lung disease or pneumonitis grade ≥ 2.
- Patients who discontinued prior PD-1 or PD-L1 directed therapy due to a treatment related toxicity should not be included in the PDR001 containing arms of the study. Patients previously exposed to anti-PD-1/PD-L1 treatment who are adequately treated for skin rash or with replacement therapy for endocrinopathies should not be excluded.
- Systemic antineoplastic therapy (including cytotoxic chemotherapy, alphainterferon, kinase inhibitors or other targeted small molecules, and toxinimmunoconjugates) or any experimental therapy within 14 days or 5 half-lives, whichever is shorter, before the first dose of study treatment.
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 |
|---|---|---|
| DecitabineAlso: 5-aza-2'-deoxycytidineDecitabine is a cytidine deoxynucleoside analogue that selectively inhibits DNA methyltransferases at low doses, resulting in gene promoter hypomethylation. | Drug | Decitabine and MBG453, Decitabine and PDR001, Decitabine, PDR001 and MBG453 |
| PDR001Also: spartalizumabPDR001 is a high-affinity, ligand-blocking, humanized IgG4 monoclonal antibody directed against PD-1 that blocks the binding of PD-L1 and PD-L2. | Drug | Decitabine and PDR001, Decitabine, PDR001 and MBG453, MBG453 and PDR001 |
| MBG453Also: sabatolimabMBG453 is a high-affinity, humanized anti-TIM-3 IgG4 monoclonal antibody which blocks the binding of TIM-3 to phosphatidylserine (PtdSer). | Drug | Azacitidine and MBG453, Decitabine and MBG453, Decitabine, PDR001 and MBG453, MBG453, MBG453 and PDR001 |
| AzacitidineAlso: 5-azacytidineAzacitidine (5-azacytidine) is a cytidine nucleoside analogue that selectively inhibits DNA methyltransferases at low doses, resulting in gene promoter hypomethylation | Drug | Azacitidine and MBG453 |
Primary outcome
Safety of MBG453 single agent treatment or MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine or azacitidine.
Time frame: 24 months
Incidence and severity of AEs and SAEs
Incidence of Dose Limiting Toxicities (DLTs)
Time frame: 2 months
The incidence of DLTs during the first two cycles of treatment with MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine.
Incidence of Dose Limiting Toxicities (DLTs)
Time frame: 1 month
The incidence of DLTs during the first cycle of MBG453 single agent treatment or during the first two cycles of treatment with MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine.
Tolerability of MBG453 single agent treatment or MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine or azacitidine.
Time frame: 24 months
Incidence and severity of AEs and SAEs
10 secondary outcomes
AUC of PDR001, MBG453, decitabine and azacitidine.
Time frame: 24 months
Cmax of PDR001, MBG453, decitabine and azacitidine
Time frame: 24 months
Tmax of PDR001, MBG453, decitabine and azacitidine
Time frame: 24 months
Half-life of PDR001, MBG453, decitabine and azacitidine
Time frame: 24 months
Overall Response Rate (ORR)
Time frame: 24 months
Best Overall Response (BOR)
Time frame: 24 months
Progression Free Survival (PFS)
Time frame: 24 months
Time to Progression (TTP)
Time frame: 24 months
Duration of Response (DOR)
Time frame: 24 months
Number of participants with anti-PDR001 and anti-MBG453 antibodies
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2025.
- Study ResultsLink provided by the sponsor.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Leukemia
RecruitingPhase 2
Tafasitamab With Acalabrutinib and Venetoclax for the Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic Lymphocytic Leukemia, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Enrolling by invitationObservational study
Natural History Study of Early Life Exposures in Agriculture (ELEA)
Prostate Cancer, Leukemia
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 1
Vididencel in CML-CP Patients With MRD
CML (Chronic Myelogenous Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
11 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Decitabine, PDR001 and MBG453 in people with Leukemia and Leukemia, Myeloid, run by Novartis Pharmaceuticals at 11 sites in 8 countries.
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 16 inclusion and 7 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 18 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Germany, Australia, Finland, France and 3 more countries. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Safety of MBG453 single agent treatment or MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine or azacitidine. (time frame: 24 months). The study lists 4 primary outcomes in all. 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 18 May 2025.