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Trametinib for Myelomonocytic Leukemia

UT Southwestern/Simmons Cancer Center-Dallas, Dallas, TX
Myelomonocytic Leukemia+1 More ConditionsTrametinib - Drug
Eligibility
1 - 21
All Sexes
What conditions do you have?
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Study Summary

This trial looks at how well trametinib works in treating patients with JML who have relapsed or who don't respond to treatment. Trametinib may stop tumor cell growth by blocking enzymes needed for cell growth.

Eligible Conditions
  • Juvenile Myelomonocytic Leukemia
  • Neurofibromatosis Type 1

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 2

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Veliparib
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Trametinib
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Study Objectives

1 Primary · 2 Secondary · Reporting Duration: 12 cycles (1 cycle = 28 days)

Day 28
Objective response
Day 28
Incidence of adverse events
Mutant allele burden
Pharmacokinetic (PK) parameters of trametinib
Trametinib concentrations

Trial Safety

Phase-Based Safety

2 of 3
This is further along than 68% of similar trials

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1
Veliparib
1
Trametinib
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Side Effects for

Cohort D
47%Headache
47%Pyrexia
41%Diarrhoea
35%Myalgia
35%Constipation
35%Nausea
35%Vomiting
35%Chills
35%Arthralgia
29%Abdominal pain
29%Asthenia
24%Folliculitis
24%Gamma-glutamyltransferase increased
18%Weight decreased
18%Aphasia
18%Blood alkaline phosphatase increased
18%Decreased appetite
18%Rash
18%Oedema peripheral
18%Dry mouth
18%Fatigue
18%Aspartate aminotransferase increased
18%Alanine aminotransferase increased
18%Muscular weakness
18%Dizziness
18%Seizure
18%Cough
18%Dry skin
12%Epistaxis
12%Insomnia
12%Hypophosphataemia
12%Eyelid oedema
12%Ejection fraction decreased
12%Anaemia
12%Thrombocytopenia
12%Visual impairment
12%Urinary tract infection
12%Weight increased
12%Hypocalcaemia
12%Hypokalaemia
12%Back pain
12%Pain in extremity
12%Paraesthesia
12%Partial seizures
12%Alopecia
12%Pruritus
6%Generalised erythema
6%Papilloma
6%Bronchitis
6%Respiratory tract infection
6%Blood sodium increased
6%Visual field defect
6%Skin exfoliation
6%Skin striae
6%Lipase increased
6%Anxiety
6%Muscle spasms
6%Metrorrhagia
6%Nasal congestion
6%Rhinitis allergic
6%Blood potassium decreased
6%Dermatitis acneiform
6%Blood cholesterol increased
6%Tendonitis
6%Gastrointestinal haemorrhage
6%Neck pain
6%Abdominal pain upper
6%Acute kidney injury
6%Conjunctival irritation
6%Eye pruritus
6%Diplopia
6%Vision blurred
6%Vitreous detachment
6%Gastrooesophageal reflux disease
6%Pain
6%Xerosis
6%Cellulitis
6%Skin infection
6%Neutrophil count decreased
6%Dehydration
6%Musculoskeletal pain
6%Musculoskeletal chest pain
6%Motor dysfunction
6%Paresis
6%Sleep disorder
6%Erythema
6%Hot flush
6%Hypotension
6%Lymphoedema
6%Onychomycosis
6%Papule
6%Renal failure
6%Dry eye
6%Punctate keratitis
6%Visual acuity reduced
6%Abdominal pain lower
6%Faeces soft
6%Gastrointestinal disorder
6%Face oedema
6%Lung infection
6%Herpes zoster
6%Rash pustular
6%Hyperhidrosis
6%Ligament sprain
6%Amylase increased
6%Blood creatinine increased
6%Hypomagnesaemia
6%Hyponatraemia
6%Joint range of motion decreased
6%Intertrigo
6%Night sweats
6%Amnesia
6%Dysarthria
6%Pain of skin
6%Tonic clonic movements
6%Agitation
6%Confusional state
6%Dyspnoea
6%Productive cough
6%Aquagenic wrinkling of palms
6%Pruritus generalised
6%Skin lesion
6%Deep vein thrombosis
6%Presyncope
6%Temporal lobe epilepsy
This histogram enumerates side effects from a completed 2018 Phase 2 trial (NCT02039947) in the Cohort D ARM group. Side effects include: Headache with 47%, Pyrexia with 47%, Diarrhoea with 41%, Myalgia with 35%, Constipation with 35%.

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 10 Other Conditions
This treatment demonstrated efficacy for 10 other conditions.

Trial Design

1 Treatment Group

Treatment (trametinib)
1 of 1

Experimental Treatment

10 Total Participants · 1 Treatment Group

Primary Treatment: Trametinib · No Placebo Group · Phase 2

Treatment (trametinib)Experimental Group · 4 Interventions: Bone Marrow Aspiration and Biopsy, Computed Tomography, Magnetic Resonance Imaging, Trametinib · Intervention Types: Procedure, Procedure, Procedure, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Computed Tomography
2017
Completed Phase 2
~2710
Magnetic Resonance Imaging
2017
Completed Phase 3
~1180
Trametinib
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 12 cycles (1 cycle = 28 days)

Who is running the clinical trial?

Children's Oncology GroupNETWORK
451 Previous Clinical Trials
236,946 Total Patients Enrolled
National Cancer Institute (NCI)Lead Sponsor
13,275 Previous Clinical Trials
41,234,802 Total Patients Enrolled
Elliot StieglitzPrincipal InvestigatorChildren's Oncology Group

Eligibility Criteria

Age 1 - 21 · All Participants · 1 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have an enlarged spleen.
References

Frequently Asked Questions

Can people over the age of 20 participate in this research?

"This clinical trial is only for paediatric patients that fall in the 1 month to 21 year age range. Out of the 4,885 active clinical trials, 494 are for patients under 18 and 1291 are for senior citizens." - Anonymous Online Contributor

Unverified Answer

Could you tell me if this research is currently ongoing and recruiting participants?

"That is correct. The trial, which began recruiting on October 6th 2017, as indicated by clinicaltrials.gov, is still looking for 24 more patients to be admitted at 54 different sites." - Anonymous Online Contributor

Unverified Answer

When was the last time Trametinib received a thumbs up from the FDA?

"Trametinib's rating is a 2. Although there is some evidence affirming its safety, no studies have looked into this medication's effectiveness." - Anonymous Online Contributor

Unverified Answer

Are there other examples in the medical literature of Trametinib's efficacy?

"Trametinib was first used in 2012 during a clinical trial at University of Chicago Medical Center. 155 such studies have completed, with 96 more ongoing. Many of the active trials are based in Austin, Texas." - Anonymous Online Contributor

Unverified Answer

What is the total number of test subjects in this clinical trial?

"A total of 24 individuals are required for this clinical trial, all of which must meet the pre-specified inclusion criteria. The Dell Children's Medical Center of Central Texas in Austin and Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center in Houston are two locations where patients can take part in this study." - Anonymous Online Contributor

Unverified Answer

Where are the different sites for this trial taking place?

"In total, there are 57 sites where patients can enroll in this trial, including Dell Children's Medical Center of Central Texas in Austin, Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center in Houston, and Nemours Children's Hospital in Orlando." - Anonymous Online Contributor

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

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