Platform Study of Novel Ruxolitinib Combinations in Myelofibrosis Patients

A Randomized, Open-label, Phase I/II Open Platform Study Evaluating Safety and Efficacy of Novel Ruxolitinib Combinations in Myelofibrosis Patients

ClinicalTrials.gov Identifier: NCT04097821

Novartis Reference Number: CINC424H12201

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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

The purpose of this study is to investigate the safety, pharmacokinetics and preliminary efficacy of combinations treatment of ruxolitinib with 3 novel compounds: siremadlin, crizanlizumab and MBG453 in myelofibrosis (MF) subjects.

Condition 
Myelofibrosis
Phase 
Phase 1
Phase 2
Overall status 
Recruiting
Enrollment count 
130 participants
Start date 
Sep 26, 2019
Completion date 
Jun 10, 2025
Gender 
All
Age(s)
18 Years and older (Adult, Older Adult)

Interventions

Drug
Ruxolitinib
5 mg tablets for oral use
Drug
Siremadlin
10 mg, 20 mg, or 40 mg capsules for oral use
Drug
Crizanlizumab
100 mg/mL concentrate for infusion for intravenous use
Drug
MBG453
100 mg/mL concentrate for infusion for intravenous use

Eligibility Criteria

Inclusion Criteria:

Subjects have diagnosis of primary myelofibrosis (PMF) according to the 2016 World Health Organization (WHO) criteria, or diagnosis of post-essential thrombocythemia (ET) (PET-MF) or post-polycythemia vera (PV) myelofibrosis (PPV-MF) according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) 2007 criteria
Palpable spleen of at least 5 cm from the left costal margin (LCM) to the point of greatest splenic protrusion or enlarged spleen volume of at least 450 cm3 per MRI or CT scan at baseline (a MRI/CT scan up to 8 weeks prior to first dose of study treatment can be accepted).
Have been treated with ruxolitinib for at least 24 weeks prior to first dose of study treatment
Are stable (no dose adjustments) on the prescribed ruxolitinib dose (between 5 and 25 mg twice a day (BID)) for ≥ 8 weeks prior to first dose of study treatment

Exclusion Criteria:

Not able to understand and to comply with study instructions and requirements.
Received any investigational agent for the treatment of MF (except ruxolitinib) within 30 days of first dose of study treatment or within 5 half-lives of the study treatment, whichever is greater
Peripheral blood blasts count of > 10%.
Received a monoclonal antibody (Ab) or immunoglobulin-based agent within 1 year of screening, or has documented severe hypersensitivity reactions/immunogenicity (IG) to a prior biologic
Splenic irradiation within 6 months prior to the first dose of study drug
Received blood platelet transfusion within 28 days prior to first dose of study treatment.

Other protocol-defined Inclusion/Exclusion criteria may apply.

Study Locations

Australia
Novartis Investigative Site
Recruiting
Adelaide, 5000
South Australia
Australia
Novartis Investigative Site
Recruiting
Melbourne, 3000
Victoria
Australia
Novartis Investigative Site
Recruiting
Melbourne, 3004
Victoria
Australia
Belgium
Novartis Investigative Site
Recruiting
Leuven, 3000
-
Belgium
Denmark
Novartis Investigative Site
Recruiting
Copenhagen, 2100
-
Denmark
Germany
Novartis Investigative Site
Recruiting
Mannheim, 68305
Baden-Wuerttemberg
Germany
Novartis Investigative Site
Recruiting
Chemnitz, 09113
-
Germany
Novartis Investigative Site
Recruiting
Freiburg, 79106
-
Germany
Novartis Investigative Site
Recruiting
Jena, 07740
-
Germany
Novartis Investigative Site
Recruiting
Stuttgart, 70176
-
Germany
Hungary
Novartis Investigative Site
Recruiting
Budapest, 1083
HUN
Hungary
Italy
Novartis Investigative Site
Recruiting
Firenze, 50134
FI
Italy
Netherlands
Novartis Investigative Site
Recruiting
Amsterdam, 1081 HV
-
Netherlands
Spain
Novartis Investigative Site
Recruiting
Malaga, 29010
Andalucia
Spain
Novartis Investigative Site
Recruiting
Alicante, 03010
Comunidad Valenciana
Spain
Novartis Investigative Site
Recruiting
Valencia, 46010
Comunidad Valenciana
Spain
Novartis Investigative Site
Recruiting
Las Palmas de Gran Canaria, 35010
-
Spain
Novartis Investigative Site
Recruiting
Salamanca, 37007
-
Spain
Sweden
Novartis Investigative Site
Recruiting
Stockholm, 14186
-
Sweden
Switzerland
Novartis Investigative Site
Recruiting
St. Gallen, 9007
-
Switzerland
Novartis Investigative Site
Recruiting
Zurich, 8091
-
Switzerland
United Kingdom
Novartis Investigative Site
Recruiting
London, SE1 9RT
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Name: 
Novartis Pharmaceuticals
Phone: 
Email: 

Have a question?

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