Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With AML or High Risk MDS

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 Identifier: NCT03066648

Novartis Reference Number: CPDR001X2105

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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 in AML and high risk MDS patients, and to identify recommended doses for future studies.

Condition 
Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Preleukemia
Bone Marrow Diseases
Hematologic Diseases
Phase 
Phase 1
Overall status 
Recruiting
Enrollment count 
235 participants
Start date 
Jul 06, 2017
Completion date 
Nov 01, 2021
Gender 
All
Age(s)
18 Years and older (Adult, Older Adult)

Interventions

Drug
Decitabine
Decitabine is a cytidine deoxynucleoside analogue that selectively inhibits DNA methyltransferases at low doses, resulting in gene promoter hypomethylation.
Drug
PDR001
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.
Drug
MBG453
MBG453 is a high-affinity, humanized anti-TIM-3 IgG4 monoclonal antibody which blocks the binding of TIM-3 to phosphatidylserine (PtdSer).

Eligibility Criteria

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:

Refractory/relapsed AML following ≥1 prior therapies 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)
De novo 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)
High risk MDS (patients who are suitable for standard re-induction chemotherapy or hematopoietic stem cell transplantation and willing to receive it are excluded)

Arms 4-5:

Refractory / relapsed AML following ≥1 prior therapies (Arms 4a & 5a)
High risk MDS 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.)
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.

Study Locations

United States
Massachusetts General Hospital
Recruiting
Boston, 02114
Massachusetts
United States
Oregon Health and Science University
Recruiting
Portland, 97239
Oregon
United States
MD Anderson Cancer Center
Recruiting
Houston, 77030
Texas
United States
Australia
Novartis Investigative Site
Recruiting
Melbourne, 3004
Victoria
Australia
Finland
Novartis Investigative Site
Recruiting
Helsinki, FIN 00290
-
Finland
France
Novartis Investigative Site
Recruiting
Marseille, 13273
-
France
Germany
Novartis Investigative Site
Recruiting
Dresden, 01307
-
Germany
Novartis Investigative Site
Recruiting
Jena, 07740
-
Germany
Netherlands
Novartis Investigative Site
Recruiting
Amsterdam, 1081 HV
-
Netherlands
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08036
Catalunya
Spain
United Kingdom
Novartis Investigative Site
Recruiting
Cardiff, CF4 4XN
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
1-888-669-6682
Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Email: 

Have a question?

Call 1-999-669-6682 or email [email protected]