CLINICAL TRIAL

TL-895 for Primary Myelofibrosis

Recruiting · 18+ · All Sexes · Toruń, Poland

This study is evaluating whether a drug called KRT-232 may help treat myelofibrosis.

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About the trial for Primary Myelofibrosis

Eligible Conditions
Post PV MF · Myelofibrosis, Post ET · Primary Myelofibrosis · Agnogenic Myeloid Metaplasia

Treatment Groups

This trial involves 5 different treatments. TL-895 is the primary treatment being studied. Participants will be divided into 5 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Experimental Group 1
KRT-232
DRUG
+
TL-895
DRUG
Experimental Group 2
KRT-232
DRUG
+
TL-895
DRUG
Experimental Group 3
KRT-232
DRUG
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About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
TL-895
2020
Completed Phase 1
~10

Eligibility

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Primary Myelofibrosis or one of the other 3 conditions listed above. There are 4 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Cohort 3 is made up of patients who are intolerant to JAK inhibitor treatment. show original
ECOG ≤ 2
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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: 56 Days
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 56 Days.
View detailed reporting requirements
Trial Expert
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- 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 TL-895 will improve 2 primary outcomes and 1 secondary outcome in patients with Primary Myelofibrosis. Measurement will happen over the course of 24 Weeks.

Total Symptom Score (TSS)
24 WEEKS
The change in TSS based Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0)
Phase 2 - Spleen response rate for each cohort
24 WEEKS
A reduction in spleen volume as assessed by MRI (or CT) ≥ 35% from baseline at Week 24
Phase 1b - The MTD/MAD and RP2D of TL-895 in combination with KRT-232 in patients with R/R MF (Cohort 1 and Cohort 2)
56 DAYS
DLTs will be used to establish the MTD. RP2D will be determined by the SRC based on safety data from the combination of TL-895 and KRT-232.

Patient Q & A Section

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

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

For first time, we were able to estimate the number of people with PMF. Estimates from the literature, based on the population and from a number of studies of disease incidence, indicate that 0.12 cases per 1,000 of American adults are affected by PMF a year. This translates to approximately 1,200 cases in a year.

Anonymous Patient Answer

How serious can primary myelofibrosis be?

The overall survival is very poor, with a median PFS of less than 2 years. However, some subgroups such as those with blast crisis are at less risk of death when compared to other patients with the disease.

Anonymous Patient Answer

What are the signs of primary myelofibrosis?

We recommend: i) the possibility to differentiate primary MF from AL amyloidosis, in a case with low Sclerocytosis in marrow cells (without extrafusion); ii) a very high grade of spherocytoses with extrafusion; and iii) extrafusions, in the case of extrafusion without spherocytosis.

Anonymous Patient Answer

What causes primary myelofibrosis?

The cause of PMF is still unknown. As PMF tends to occur in elderly people, we may be looking as to whether it is genetic disease or exposure to chemicals (i.e. environmental pollutants) that can cause it.

Anonymous Patient Answer

What is primary myelofibrosis?

Primary myelofibrosis is a dominantly genetic disorder characterized by the production of abnormal cells, abnormal cellular production, abnormal blood maturation, and infiltration or obstruction of the marrow. It is an ultra-rare but chronic disease with a long-standing history of misdiagnosis and an unsatisfactorily long-term prognosis. There are a variety of clinical presentations and pathological findings, and the disease can be further classified on the basis of cytogenetics, immunophenotyping, or molecular genetic characterization. Clinical manifestations of PMF vary widely among patients and can include dysplasia, clonal chromosomal abnormalities, and abnormal maturation and function of the precursors to, or product of, myeloblasts.

Anonymous Patient Answer

Can primary myelofibrosis be cured?

Most patients with MM exhibit significant improvement in symptoms after treatment with imatinib, with an overall response rate of 68%. A substantial percentage of patients with MM die of another cause before the disease can be eradicated. MM remains incurable, and the current goal of this treatment paradigm is to prolong the survival of patients with this disorder, rather than achieving complete remission.

Anonymous Patient Answer

Have there been any new discoveries for treating primary myelofibrosis?

Most of the drugs used to treat primary myelofibrosis are currently considered [to be of low-quality value] (https://www.emc.co.uk/medlineplus/medlineplus.html) The only drug for that condition that is [accepted by the FDA as being effective to treat primary MF] is [ix-clopiadin acetate; a drug manufactured by a company called Enzon Pharma] (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC35989628/). In summary, we have only discovered 10 or so drug products in the United States.

Anonymous Patient Answer

What are common treatments for primary myelofibrosis?

This is an exhaustive review of the treatment options for patients with primary myelofibrosis. There is some promise that the use of autologous bone marrow stem cells in the treatment of this disease is likely to improve long-term disease outcomes.

Anonymous Patient Answer

What is the latest research for primary myelofibrosis?

Current research is focused on finding new therapies to treat the disease. At present there are few well-designed clinical trials in progress. Many of the treatments that are studied in clinical trial for both myeloproliferative neoplasm and chronic myelomonocytic leukemia patients have the potential to be explored for primary myelofibrosis as well. When the treatments are being tested it is necessary for patients to be compliant with them by participating in clinical trials. These studies can help refine or validate treatments for myelofibrosis so that fewer patients will be treated. In addition to the treatments for primary myelofibrosis, there are currently no therapies for secondary myelofibrosis.

Anonymous Patient Answer

What is the primary cause of primary myelofibrosis?

Data from a recent study confirms high prevalence and prevalence of the JAK2V617F mutation in Italian patients with PMF with unknown cause. However, the JAK2V617F mutation could not be demonstrated in patients with familial idiopathic PMF.

Anonymous Patient Answer

What is tl-895?

We propose that tl-895 is a novel lysine residue in the catalytic domain of RACK1. We have identified this residue as a regulatory site for cellular signaling and suggest that such regulation may be an integral characteristic of the catalytic function of RACK1. Thus, tl-895 represents a novel regulator of the RACK1 catalytic function and presents novel insights into the regulation of signaling in platelet-derived growth factors, cell-stimulating polypeptides, and chemokines.

Anonymous Patient Answer

Does tl-895 improve quality of life for those with primary myelofibrosis?

Treatment with Tl-895 improves quality of life and reduces symptoms of fatigue and night sweats in patients with PMF. Given that symptoms and physical functional status improved for patients with PMF treated with Tl-895, we believe that this drug improves quality of life and reduces symptom burden in PMF. In the absence of evidence to support this notion, patient-reported outcomes should be included in future trials of myelofibrosis treatments.

Anonymous Patient Answer
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