ClinTrial Refer

Trial Acronym
TRANSFORM-1

Registration Number
NCT04472598

Scientific Title
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study Of Navitoclax In Combination With Ruxolitinib Versus Ruxolitinib In Subjects With Myelofibrosis (TRANSFORM-1)

Myelofibrosis is a type of bone marrow cancer that usually develops slowly and disrupts
body's normal production of blood cells. It causes bone marrow scarring, leading to severe
anemia that can cause weakness and fatigue. It can also cause a low number of blood-clotting
cells called platelets, which increases risk of bleeding. Myelofibrosis often causes an
enlarged spleen. The purpose of this study is to see if a combination of navitoclax and
ruxolitinib is more effective and safe in assessment of change in spleen volume when compared
to ruxolitinib in participants with myelofibrosis.

Navitoclax is an investigational drug for the treatment of myelofibrosis. Participants in
this study are divided into two groups, called treatment arms. Each group receives a
different treatment. Adult participants with a diagnosis of myelofibrosis will be enrolled.
Around 230 participants will be enrolled in approximately 130 sites worldwide.

Participants will receive oral navitoclax tablet with oral ruxolitinib tablet or oral
ruxolitinib tablet with oral placebo (no active drug) tablet and treatment may continue till
the participant cannot tolerate the study drug, or benefit is not achieved, or other reasons
which qualify for discontinuation of the study drug.

There may be a 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
tests, Magnetic Resonance Imaging (MRI), bone marrow tests, checking for side effects, and
completing questionnaires.

-  Documented diagnosis of Primary MyeloFibrosis (MF) or Secondary MF (post polycythemia vera [PPV] - MF or Post Essential Thrombocytopenia [PET] - MF) as defined by World Health Organization (WHO) classification.

    -  Must be able to complete the MF Symptom Assessment Form (MFSAF) v4.0 on at least 4 out of 7 days prior to randomization.
 --Must have at least 2 symptoms with a score >=3 or a total score of >=12, as measured by the MFSAF v4.0.

    -  Classified as intermediate-2, or high-Risk MF as defined by the Dynamic International Prognostic Scoring System Plus (DIPSS+).

    -  Has splenomegaly defined as spleen palpation measurement >= 5 centimeters (cm) below costal margin or spleen volume greater than or equal to 450 cubic cm as assessed centrally by Magnetic Resonance Imaging (MRI) or Computed Tomography (CT) scan.

    -  Ineligible for stem cell transplantation at time of study entry.

    -  Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.

-  Prior treatment with a Janus Kinase-2 (JAK-2) inhibitor.

    -  Prior treatment with a BH3-mimetic compound or bromodomain and extra-terminal motif (BET) inhibitor.

    -  Receiving medication that interferes with coagulation or platelet function except for low dose aspirin (up to 100 milligram daily) and low molecular weight heparin (LMWH) within 3 days prior to the first dose of study drug or during the study treatment period.

Trial Location Name

Address

Status

Alfred Health (Haematology)

The Alfred, Commercial Road, Melbourne VIC, Australia

Closed

Primary Contact : John Nguyen, john.nguyen@alfred.org.au , 03 9076 7135

Gosford Hospital (Haematology)

Holden St, Gosford NSW 2250, Australia

Closed

Primary Contact : Mark Lacey, Mark.Lacey@health.nsw.gov.au , 02 4320 3940

Liverpool Hospital (Haematology)

1 Campbell St, Liverpool NSW 2170, Australia

Closed

Primary Contact : Pinky Patel, pinky.patel@health.nsw.gov.au , 0474 279 274

Secondary Contact : Sharon Young, Sharon.young@health.nsw.gov.au

Peter MacCallum - Parkville Cancer Clinical Trials Unit (PCCTU) (Haematology)

Peter MacCallum Cancer Centre, Grattan Street, Melbourne VIC, Australia

Closed

Primary Contact : Enquiry Line Coordinator, clinicaltrials.enquiries@petermac.org , +613 8559 7456

St Vincents Hospital Sydney (Haematology)

St Vincent's Hospital Sydney, Victoria Street, Darlinghurst NSW, Australia

Closed

Primary Contact : Georgia McCaughan, georgia.mccaughan@svha.org.au , 02 9355 5656

AbbVie

Website URL

Link Name

Link URL

ANZCTR

http://www.anzctr.org.au/TrialSearch.aspx?searchTxt=NCT04472598

Clinicaltrials.gov

https://clinicaltrials.gov/ct2/show/NCT04472598

Secondary IDs
2020-000097-15M16-191

Study Type
Interventional

Intervention Code
Treatment - Drugs

Phase
Phase 3

Minimum Age
18

Maximum Age
100

Date Registered
09/11/2020

Date Closed
N/A

Status
Closed

Enrolment Type
By Specialist and/or Surgeon Referral

Mutation Status and Biomarker
Any or not stated

Is it a Cohort Trial?
No

Is this a Tele-Trial?
No

Can healthy volunteer participate?
No

Discipline

Health Condition

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