CLINICAL TRIAL

Navitoclax for Primary Myelofibrosis

1 Prior Treatment
Refractory
Relapsed
Waitlist Available · 18+ · All Sexes · Belfast, United Kingdom

This study is evaluating whether a drug may help treat myelofibrosis.

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About the trial for Primary Myelofibrosis

Eligible Conditions
Primary Myelofibrosis · Agnogenic Myeloid Metaplasia

Treatment Groups

This trial involves 2 different treatments. Navitoclax is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Experimental Group 1
Navitoclax
DRUG
+
Ruxolitinib
DRUG
Experimental Group 2
Navitoclax
DRUG

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Navitoclax
Not yet FDA approved
Ruxolitinib
FDA approved

Eligibility

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Primary Myelofibrosis or the other condition listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Participants with documented diagnosis of intermediate-2 or high-risk primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis.
Participant must be ineligible due to age, comorbidities, or unfit for unrelated or unmatched donor transplantation or unwilling to undergo stem cell transplantation at time of study entry.
Eastern Cooperative Oncology Group (ECOG) of 0, 1, or 2.
Ruxolitinib treatment for >=24 weeks with lack of efficacy defined as a lack of spleen response (refractory) or a loss of spleen or symptom response (relapsed)
Ruxolitinib treatment for >=28 days with intolerance defined as new red blood cell transfusion requirement (at least 2 units/month for 2 months) while receiving a total daily ruxolitinib dose of >=30 mg but unable to reduce dose further due to lack of efficacy.
If receiving ruxolitinib at the time of screening, must currently be on a stable dose >=10 mg twice daily of ruxolitinib for >=4 weeks prior to the 1st dose of navitoclax.
Participant has at least 2 symptoms each with a score >=3 or a total score of >=12, as measured by the Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 on at least 4 out of 7 days during screening prior to study drug dosing; OR Prior treatment with a JAK-2 inhibitor for at least 12 weeks
Participant has at least 2 symptoms each with a score >=3 or a total score of >= 12, as measured by the MFSAF v4.0 on at least 4 out of 7 days during screening prior to study drug dosing.
Participant has splenomegaly as defined in the protocol.
Participant must meet the laboratory parameters (adequate bone marrow, renal and hepatic function) as defined in the protocol.
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Every 12 weeks up to approximately 96 weeks
Screening: ~3 weeks
Treatment: Varies
Reporting: Every 12 weeks up to approximately 96 weeks
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Every 12 weeks up to approximately 96 weeks.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Navitoclax will improve 1 primary outcome and 3 secondary outcomes in patients with Primary Myelofibrosis. Measurement will happen over the course of From Baseline (Week 0) through Week 24.

Percentage of participants achieving 50% Reduction in Total System Score (TSS)
FROM BASELINE (WEEK 0) THROUGH WEEK 24
TSS is assessed by the Myelofibrosis Symptom Assessment Form (MFSAF) version 4.0.
FROM BASELINE (WEEK 0) THROUGH WEEK 24
Percentage of Participants who achieve Spleen Volume Reduction of greater than or equal to 35% (SVR35) from baseline
FROM BASELINE (WEEK 0) THROUGH WEEK 24
Reduction in spleen volume is measured by magnetic resonance imaging/computerized tomography (MRI/CT).
FROM BASELINE (WEEK 0) THROUGH WEEK 24
Change in Grade of Bone Marrow Fibrosis
THROUGH WEEK 96
Bone marrow grading is assessed according to the European Consensus Grading System.
THROUGH WEEK 96
Anemia Response
EVERY 12 WEEKS UP TO APPROXIMATELY 96 WEEKS
The anemia response will be assessed according to current International Working Group-Myeloproliferative Neoplasms Research and European LeukemiaNet (IWG-MRT/ELN) criteria.
EVERY 12 WEEKS UP TO APPROXIMATELY 96 WEEKS

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What causes primary myelofibrosis?

This case illustrates several characteristics of the JAK2V617F clone and, in light of similar genetic abnormalities, suggests that it is involved in the pathogenesis of myeloproliferative neoplasms including CMPN.

Anonymous Patient Answer

What are common treatments for primary myelofibrosis?

In general, PBM patients should undergo aggressive (3+3) treatments, including autogenous donor hematopoietic stem cell transplant (HSCT) with bone marrow and autologous peripheral stem cell transplant (ASCT) at diagnosis. ASCT is usually a last-resort treatment in PBM patients who do not respond to initial PBM therapies and do not qualify for HSCT and should be reserved for patients who carry a 5% or higher risk of relapse.

Anonymous Patient Answer

What are the signs of primary myelofibrosis?

Results from a recent paper reinforce the concept that primary MF involves multisystemic disease. Patients should be aware of these frequent unexplained signs; they represent manifestations of MF not associated with a specific disease but the manifestations that are more frequently associated with systemic sclerosis.

Anonymous Patient Answer

Can primary myelofibrosis be cured?

As a result of long-term treatment and the control of symptoms, most patients who were diagnosed with PMF were alive and able to lead an unremarkable life, but the disease remains incurable.

Anonymous Patient Answer

How many people get primary myelofibrosis a year in the United States?

Around 40,000 people a year are diagnosed with PMF in the United States. The annual incidence of PMF among the general population is estimated to be between 1/35,000-1/40,000. The prevalence, as assessed by the number of people diagnosed with PMF in a year, is estimated to be between 1/400,000 and 1/500,000.

Anonymous Patient Answer

What is primary myelofibrosis?

The incidence of primary myelofibrosis appears to have increased in recent years. We found the strongest evidence of this in patients from Latin America, Central America, Africa, and South Asia, but evidence also exists for an increased risk in Europe. The incidence is high in men and the disease is associated with chromosomal abnormalities and trisomy 12.\n

Anonymous Patient Answer

Has navitoclax proven to be more effective than a placebo?

Among advanced myeloid malignancies, [median event-free survival (EFS/PFS) of navitoclax was statistically superior to placebo (P = 0.003), with a hazard ratio (HR) of 0.73 (90% CI 0.56-0.94); hazard ratio in the intent-to-treat analysis of EFS/PFS was 0.76 (90% CI 0.61- 0.94). Compared to previously reported EFS estimates in patients treated with tyrosine kinase inhibitors...] and EFS estimates in patients treated with azacitidine. (http://www.power.vanderbilt.

Anonymous Patient Answer

What does navitoclax usually treat?

There have been no reports of the efficacy of navitoclax for the treatment of myeloproliferative disorders. However, there have been reports of its effectiveness in treating various hematological malignancies such as T-cell prolymphocytic lymphoma and acute myeloid leukemia. Considering the recent history of navitoclax and clinical development, the use for the treatment of primary myelofibrosis has been approved. To summarize, navitoclax is being used to treat myeloproliferative disorders, including primary myelofibrosis.

Anonymous Patient Answer

What is navitoclax?

Navitoclax is a small molecule cyclin-dependent kinase inhibitor belonging to the class of oral small molecule chemotherapy in combination with a bone marrow transplantation. Navitoclax may be useful in the treatment of patients with myelodysplastic syndromes and polycythaemia vera/essential thrombocytosis to induce an increased level of apoptosis (apoptosis induction) and improve the prognosis, with minimal side effects.

Anonymous Patient Answer

How does navitoclax work?

[Navitoclax is effective as single agent treatment in refractory to standard chemotherapy naïve or in relapsed/refractory [myeloid/lymphoid] cancers and has been used as an alternative or as second line treatment in the therapy of patients relapsed after or ineligible for conventional chemotherapy]

Anonymous Patient Answer

What are the common side effects of navitoclax?

For this first generation drug that has been evaluated in phase I, there were no dose-limiting safety events in subjects treated with navitoclax up to 300 mg/kg/day, or 1500 mg/day for children.

Anonymous Patient Answer

Have there been other clinical trials involving navitoclax?

The data that we obtained from the current trial may suggest a positive benefit for this drug in the management of a subcategory of MPNs known as high-risk MPNs because of the rarity of the patient who received this treatment in most clinical trials, so far. Further analyses are needed to examine the optimal dose of navitoclax and how this compound combined with other agents may contribute towards the achievement of long-term remission. Such investigations will be necessary before this compound can be further explored in the design and the evaluation of clinical trials for other subtypes of MPNs.

Anonymous Patient Answer
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