CLINICAL TRIAL

Cobimetinib for Leukemia, Myelomonocytic, Chronic

Newly Diagnosed
Refractory
Recruiting · 18+ · All Sexes · Salt Lake City, UT

Cobimetinib in Newly Diagnosed or HMA-treated CMML Patients With RAS Pathway Mutations

See full description

About the trial for Leukemia, Myelomonocytic, Chronic

Eligible Conditions
Leukemia, Myelomonocytic, Chronic · Chronic Myelomonocytic Leukemia (CMML) · Leukemia, Myelomonocytic, Juvenile

Treatment Groups

This trial involves 2 different treatments. Cobimetinib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Cobimetinib
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cobimetinib
FDA approved

Side Effect Profile for Pembrolizumab

Pembrolizumab
Show all side effects
19%
Death
19%
Fatigue
18%
Asthenia
16%
Diarrhoea
14%
Pruritis
12%
Nausea
12%
Rash
11%
Arthralgia
9%
Hyperthyroidism
9%
Back pain
8%
Constipation
8%
Cough
8%
Headache
7%
Pyrexia
7%
Vomiting
7%
Hypothyroidism
7%
Dyspnoea
7%
Decreased appetite
6%
Abdominal pain
6%
Oedema peripheral
6%
Alanine aminotransferase increased
6%
Lipase increased
6%
Anaemia
6%
Aspartate aminotransferase increased
5%
Myalgia
5%
Dry skin
5%
Hypertension
4%
Blood creatine phosphokinase increased
4%
Hyperglycaemia
3%
Amylase increased
3%
Erythema
3%
Dry mouth
3%
Pain in extremity
3%
Dizziness
2%
Pulmonary embolism
2%
Blood lactate dehydrogenase increased
2%
Rash maculo-papular
1%
Abdominal pain upper
1%
Stomatitis
1%
Urinary tract infection
1%
Dermatitis acneiform
0%
Diplopia
0%
Gastrointestinal haemorrhage
0%
Gastric ulcer
0%
Diarrhoea infectious
0%
Non-cardiac chest pain
0%
Pancreatitis
0%
Cellulitis
0%
Rectal haemorrhage
0%
Seizure
0%
Thrombosis mesenteric vessel
0%
Gastrointestinal obstruction
0%
General physical health deterioration
0%
Malaise
0%
Subileus
0%
Scleroderma
0%
Chest pain
0%
Erysipelas
0%
Respiratory tract infection
0%
Pneumonia
0%
Atrial flutter
0%
Dermatitis allergic
0%
Muscular weakness
0%
Device related sepsis
0%
Endocarditis
0%
Vascular device infection
0%
Brain oedema
0%
Influenza
0%
Dermatitis
0%
Lung infection
0%
Ischaemic stroke
0%
Angina pectoris
0%
Toxic shock syndrome
0%
Blood bilirubin increased
0%
Presyncope
0%
Dyskinesia
0%
Monoparesis
0%
Encephalitis autoimmune
0%
Intracranial pressure increased
0%
Leukoencephalopathy
0%
Blood creatinine increased
0%
Pleural effusion
0%
General physical condition abnormal
0%
Interstitial lung disease
0%
Atrial fibrillation
0%
Cardiac failure acute
0%
Pemphigoid
0%
Bradycardia
0%
Cardiac tamponade
0%
Diabetes mellitus
0%
Transaminases increased
0%
Dermatitis exfoliative generalised
0%
Myocarditis
0%
Rash macular
0%
Rhabdomyolysis
0%
Ankle fracture
0%
Chorioretinopathy
0%
Tachyarrhythmia
0%
Hyponatraemia
0%
Hepatitis
0%
Folliculitis
0%
Dehydration
0%
Spinal pain
0%
Fall
0%
Tumour pain
0%
Hypovolaemia
0%
Renal failure
0%
Haematuria
0%
Hepatic failure
0%
Hypotension
0%
Haematoma
0%
Orthostatic hypotension
0%
Thrombocytopenia
0%
Adrenal insufficiency
0%
Hypersensitivity
0%
Hepatic pain
0%
Tumour haemorrhage
0%
Colitis
0%
Haemorrhoids
0%
Superinfection viral
0%
Autoimmune colitis
0%
Lower respiratory tract infection
0%
Haematochezia
0%
Intestinal obstruction
0%
Upper respiratory tract infection
0%
Multiple organ dysfunction syndrome
0%
Sepsis
0%
Urosepsis
0%
Dacryocystitis
0%
Encephalitis
0%
Infection
0%
Spleen tuberculosis
0%
Wound infection
0%
Haemoptysis
0%
Syncope
0%
Autoimmune neuropathy
0%
Nervous system disorder
0%
Transient ischaemic attack
0%
Radiculopathy
0%
Hypoglycaemia
0%
Acute kidney injury
0%
Prostate cancer
0%
Upper limb fracture
0%
Bladder perforation
0%
Infusion related reaction
0%
Hip fracture
0%
Visual impairment
0%
Rash pustular
0%
Dupuytren's contracture
0%
Myositis
0%
Autoimmune encephalopathy
0%
Ascites
Death
19%
Fatigue
19%
Asthenia
18%
Diarrhoea
16%
Pruritis
14%
Nausea
12%
Rash
12%
Arthralgia
11%
Hyperthyroidism
9%
Back pain
9%
Constipation
8%
Cough
8%
Headache
8%
Pyrexia
7%
Vomiting
7%
Hypothyroidism
7%
Dyspnoea
7%
Decreased appetite
7%
Abdominal pain
6%
Oedema peripheral
6%
Alanine aminotransferase increased
6%
Lipase increased
6%
Anaemia
6%
Aspartate aminotransferase increased
6%
Myalgia
5%
Dry skin
5%
Hypertension
5%
Blood creatine phosphokinase increased
4%
Hyperglycaemia
4%
Amylase increased
3%
Erythema
3%
Dry mouth
3%
Pain in extremity
3%
Dizziness
3%
Pulmonary embolism
2%
Blood lactate dehydrogenase increased
2%
Rash maculo-papular
2%
Abdominal pain upper
1%
Stomatitis
1%
Urinary tract infection
1%
Dermatitis acneiform
1%
Diplopia
0%
Gastrointestinal haemorrhage
0%
Gastric ulcer
0%
Diarrhoea infectious
0%
Non-cardiac chest pain
0%
Pancreatitis
0%
Cellulitis
0%
Rectal haemorrhage
0%
Seizure
0%
Thrombosis mesenteric vessel
0%
Gastrointestinal obstruction
0%
General physical health deterioration
0%
Malaise
0%
Subileus
0%
Scleroderma
0%
Chest pain
0%
Erysipelas
0%
Respiratory tract infection
0%
Pneumonia
0%
Atrial flutter
0%
Dermatitis allergic
0%
Muscular weakness
0%
Device related sepsis
0%
Endocarditis
0%
Vascular device infection
0%
Brain oedema
0%
Influenza
0%
Dermatitis
0%
Lung infection
0%
Ischaemic stroke
0%
Angina pectoris
0%
Toxic shock syndrome
0%
Blood bilirubin increased
0%
Presyncope
0%
Dyskinesia
0%
Monoparesis
0%
Encephalitis autoimmune
0%
Intracranial pressure increased
0%
Leukoencephalopathy
0%
Blood creatinine increased
0%
Pleural effusion
0%
General physical condition abnormal
0%
Interstitial lung disease
0%
Atrial fibrillation
0%
Cardiac failure acute
0%
Pemphigoid
0%
Bradycardia
0%
Cardiac tamponade
0%
Diabetes mellitus
0%
Transaminases increased
0%
Dermatitis exfoliative generalised
0%
Myocarditis
0%
Rash macular
0%
Rhabdomyolysis
0%
Ankle fracture
0%
Chorioretinopathy
0%
Tachyarrhythmia
0%
Hyponatraemia
0%
Hepatitis
0%
Folliculitis
0%
Dehydration
0%
Spinal pain
0%
Fall
0%
Tumour pain
0%
Hypovolaemia
0%
Renal failure
0%
Haematuria
0%
Hepatic failure
0%
Hypotension
0%
Haematoma
0%
Orthostatic hypotension
0%
Thrombocytopenia
0%
Adrenal insufficiency
0%
Hypersensitivity
0%
Hepatic pain
0%
Tumour haemorrhage
0%
Colitis
0%
Haemorrhoids
0%
Superinfection viral
0%
Autoimmune colitis
0%
Lower respiratory tract infection
0%
Haematochezia
0%
Intestinal obstruction
0%
Upper respiratory tract infection
0%
Multiple organ dysfunction syndrome
0%
Sepsis
0%
Urosepsis
0%
Dacryocystitis
0%
Encephalitis
0%
Infection
0%
Spleen tuberculosis
0%
Wound infection
0%
Haemoptysis
0%
Syncope
0%
Autoimmune neuropathy
0%
Nervous system disorder
0%
Transient ischaemic attack
0%
Radiculopathy
0%
Hypoglycaemia
0%
Acute kidney injury
0%
Prostate cancer
0%
Upper limb fracture
0%
Bladder perforation
0%
Infusion related reaction
0%
Hip fracture
0%
Visual impairment
0%
Rash pustular
0%
Dupuytren's contracture
0%
Myositis
0%
Autoimmune encephalopathy
0%
Ascites
0%
This histogram enumerates side effects from a completed 2021 Phase 3 trial (NCT03273153) in the Pembrolizumab ARM group. Side effects include: Death with 19%, Fatigue with 19%, Asthenia with 18%, Diarrhoea with 16%, Pruritis with 14%.

Eligibility

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Leukemia, Myelomonocytic, Chronic or one of the other 2 conditions 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:
The total bilirubin is less than or equal to 1.5 times the institutional upper limit of normal. show original
People with chronic myelomonocytic leukemia (CMML) that have a RAS pathway activation as determined by standard of care hematopoietic cell sequencing results on peripheral blood or bone marrow demonstrating NRAS, KRAS, PTPN11, FLT3, CBL, JAK2, BRAF or NF1 mutations at variant allele frequency ≥ 5% are eligible for this trial. show original
The OR for males is (140-age)×weight[kg])/(serum creatinine [mg/dL]×72). show original
This text states that the ECOG performance status is ≤ 3, which means that the person is in a good physical condition. show original
A female's equation for the estimated GFR is (((140-age)×weight[kg])/(serum creatinine [mg/dL]×72))×0.85. show original
The person's left ventricular function is 50% or more, as assessed by an echocardiogram. show original
Any person aged 18 or over can be the subject of this study. show original
A decrease in the number of red blood cells or hemoglobin in the blood may be due to many different factors, including various diseases or medical conditions show original
AST(SGOT)/ALT(SGPT) will be considered normal unless it is greater than 3 times the institutional upper limit of normal show original
---Serum creatinine ≤ 2x ULN
View All
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: From 1st dose of study medication to decision to end treatment or up to 12 months of treatment, whichever came first
Screening: ~3 weeks
Treatment: Varies
Reporting: From 1st dose of study medication to decision to end treatment or up to 12 months of treatment, whichever came first
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: From 1st dose of study medication to decision to end treatment or up to 12 months of treatment, whichever came first.
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 Cobimetinib will improve 1 primary outcome and 4 secondary outcomes in patients with Leukemia, Myelomonocytic, Chronic. Measurement will happen over the course of Until decision to end treatment or up to 12 months of treatment, whichever came first.

Proportion of patients achieving complete response complete response (CR) + partial response (PR)
UNTIL DECISION TO END TREATMENT OR UP TO 12 MONTHS OF TREATMENT, WHICHEVER CAME FIRST
Assessment of complete response (CR) + partial response (PR) rate (defined by 2015 MDS/MPN-IWG criteria)
UNTIL DECISION TO END TREATMENT OR UP TO 12 MONTHS OF TREATMENT, WHICHEVER CAME FIRST
Frequency of adverse events characterized by seriousness, severity (CTCAEv5.0), duration and relationship to study therapy.
UNTIL DECISION TO END TREATMENT OR UP TO 12 MONTHS OF TREATMENT, WHICHEVER CAME FIRST
assess the safety of cobimetinib treatment in CMML
UNTIL DECISION TO END TREATMENT OR UP TO 12 MONTHS OF TREATMENT, WHICHEVER CAME FIRST
Progression-free survival (PFS)
UP TO 36 MONTHS AFTER THE START OF THERAPY, THE TIME OF PROGRESSION, INITIATION OF ALTERNATIVE TREATMENT OR DEATH, WHICHEVER CAME FIRST
Assessment of long-term efficacy
UP TO 36 MONTHS AFTER THE START OF THERAPY, THE TIME OF PROGRESSION, INITIATION OF ALTERNATIVE TREATMENT OR DEATH, WHICHEVER CAME FIRST
Overall Survival (OS)
STUDY ANTICIPATED TO BE 60 MONTHS.
Assessment of long-term efficacy
STUDY ANTICIPATED TO BE 60 MONTHS.
Overall response rate defined as the proportion of patients achieving complete remission, complete cytogenetic remission, partial remission, marrow response, and clinical benefit according to the 2015 MDS/MPN-IWG criteria
FROM 1ST DOSE OF STUDY MEDICATION TO DECISION TO END TREATMENT OR UP TO 12 MONTHS OF TREATMENT, WHICHEVER CAME FIRST
assess the efficacy of cobimetinib in patients with newly diagnosed and HMA- refractory chronic myelomonocytic leukemia (CMML)
FROM 1ST DOSE OF STUDY MEDICATION TO DECISION TO END TREATMENT OR UP TO 12 MONTHS OF TREATMENT, WHICHEVER CAME FIRST

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 does cobimetinib usually treat?

Cobimetinib is an effective and well-tolerated treatment for patients with LAM/MDS or ES-AM. The most common ALT abnormalities were the same as reported in previously reported trials, although ALT elevations > 3.0 times the cutoff are frequently seen in patients with gastrointestinal involvement. The combination of ALT elevations and hepatomegaly seems especially noteworthy, as they can present with severe hepatotoxicity.

Anonymous Patient Answer

What is leukemia, myelomonocytic, chronic?

Chronic myelomonocytic leukemia is a subtype of leukemia. A person with chronic myelomonocytic leukemia has a significantly increased risk of developing acute myeloid leukemia and a subunit transformation of leukemia in a first remission. The chronic phase of chronic myelomonocytic leukemia is characterized by low blast counts in the peripheral blood and splenomegaly. Acute myeloid leukemia and chronic myelomonocytic leukemia can be differentiated on the basis of their bone marrow smear appearance. Myelomonocytic leukemias can be classified according to their underlying cause, the morphology of the neoplastic cells, and according to organ involvement.

Anonymous Patient Answer

What are the signs of leukemia, myelomonocytic, chronic?

Patients are at an increased risk of myeloma and chronic phase chronic myeloid leukemia (CML) if they have leukaemia, myelomonocytic, chronic with a chronic phase CML gene mutation. The prognosis for AML, chronic phase CML, or chronic phase CML is poorer if they have CML with an ATM/ATF8-related CML mutation. These data help in the risk assessment of both these myeloproliferative forms of chronic myeloid leukaemia and will aid in deciding a risk stratification for therapy.

Anonymous Patient Answer

What causes leukemia, myelomonocytic, chronic?

The main causes of chronic disease are not infections or genetic abnormalities, and are not different in patients with leukemia versus those with myelomonocytic chronic disease. The same mechanisms that underlie leukemia, myelomonocytic chronic ischemic disease do not cause chronic disease in other areas of the cardiovascular system, and are likely to be involved in pathophysiology of all the diseases.

Anonymous Patient Answer

Can leukemia, myelomonocytic, chronic be cured?

This case confirms that there are still doubts about [if a cure is realistic and possible]. Still, if one wants to cure a leukemic disease, it is important to think about more advanced cases that have less favorable prognoses.

Anonymous Patient Answer

What are common treatments for leukemia, myelomonocytic, chronic?

There are two treatments: first line (intensified chemotherapy) and (intensified chemotherapy + stem cell transplant). First line is used for chronic myeloid leukemia in all age groups. Second line treatment may be used for young patients with acute promyelocytic leukemia, myelomonocytic leukemia in adulthood, or chronic myelomonocytic leukemia of adults with good performance status, or children with AML with good performance status and normal karyotype. The choice of treatment according to the disease and overall condition of the patient in any subgroup of patients is debated by specialists.

Anonymous Patient Answer

How many people get leukemia, myelomonocytic, chronic a year in the United States?

About 2,200 people per year are diagnosed in the United States with leukemia, myelomonocytic, chronic. This is a relatively low rate compared to other countries, however an increase of this number is expected when analyzing data from different institutions. Because of the lower cost of maintenance treatment in the USA, this disease could be one of the preferred ones for the budget makers.

Anonymous Patient Answer

What is the average age someone gets leukemia, myelomonocytic, chronic?

In this cohort of patients with leukemia, the average age of diagnosis was 61.8 years with a median of 5.8 years. Younger patients presented with acute myeloid leukemia (AML) at a mean age of 37.1 years, and older patients presented with lymphoid malignancies at a mean age of 60.7 years. Younger age at relapse was associated with more aggressive disease with a poorer prognosis.

Anonymous Patient Answer

What are the common side effects of cobimetinib?

This pooled analysis of clinical trial data from 1110 patients with EGFR mutant (del 19/21) solid tumors is the largest one to date reporting the overall side effects of this therapy. Common (>10% incidence) and dose-limiting toxicities were diarrhea (30%), fatigue (21%), nausea/vomiting (19%), decreased appetite (17%), decreased haemoglobin (11%), neutropenia (11%), fatigue (10%), thromboembolisms (<10%) and skin rash/skin irritation (10%).

Anonymous Patient Answer

What is the primary cause of leukemia, myelomonocytic, chronic?

Chronic myelogenous leukemia, not acute lymphoid leukemia, may have an unknown genetic cause. Other causes of chronic myelomonocytic leukemia are rare. The primary cause of chronic myelomonocytic leukemia, a chronic leukemoid reaction, is unknown.

Anonymous Patient Answer

How does cobimetinib work?

Cobimetinib inhibits oncohematological and autoimmune neoplasms in vivo, by blocking STAT3-mediated STAT5 phosphorylation. It may represent a beneficial new option to treat B cell malignancies, including non-Hodgkin's lymphomas and multiple myelomas, the latter notoriously refractory to treatment regardless of the first line treatment used.

Anonymous Patient Answer

Who should consider clinical trials for leukemia, myelomonocytic, chronic?

Patients with acute myeloid leukemia or chronic myeloid leukemia most likely will benefit from clinical trials. However, there are no good trials specifically for acute lymphoblastic leukemia, and most clinical trials are either of poor quality or do not meet current standards for testing clinical outcomes. For all clinical trials for leukemia, it is important to examine trial results in the largest, longest-lasting trials that have been completed.

Anonymous Patient Answer
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