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Histone Deacetylase Inhibitor

Mocetinostat + Vinorelbine for Rhabdomyosarcoma

Phase 1
Recruiting
Led By Noah C. Federman, MD
Research Sponsored by Jonsson Comprehensive Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Platelets (PLT) >= 100,000/mm^3 (>= 100 x 10^9/L) (transfusion independent, defined as not receiving platelet transfusions within a 7 day period prior to screening)
Have histologically or cytological confirmed diagnosis of rhabdomyosarcoma with locally advanced/unresectable, metastatic, refractory or relapsed disease who have failed standard therapy and for whom no known curative therapy exists
Must not have
History of prolonged corrected QT (QTc) interval (e.g., repeated demonstration of a QTc interval > 450 milliseconds, unless associated with the use of medications known to prolong the QTc interval). QTc will be calculated using the Bazett formula (RR interval = 60/heart rate; QTI corrected = QT interval/sqr[RRinterval])
Incomplete recovery from any surgery (other than central venous catheter or port placement) prior to treatment
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing the side effects and best dose of mocetinostat when given with vinorelbine to treat kids, teens, and young adults with rhabdomyosarcoma that has spread and is not responding to treatment or has come back.

Who is the study for?
This trial is for children, adolescents, and young adults with rhabdomyosarcoma that's advanced or returned after treatment. Participants need to have a certain level of hemoglobin, be able to follow the study plan, and give informed consent (with guardian consent if under 18). They should not be pregnant or breastfeeding and must agree to contraception measures.
What is being tested?
The trial tests mocetinostat combined with vinorelbine in treating rhabdomyosarcoma. Mocetinostat may block enzymes needed for tumor growth while vinorelbine could stop cell division. The goal is to see if this combination works better than vinorelbine alone.
What are the potential side effects?
Possible side effects include reactions at the infusion site, fatigue, nausea, low blood counts increasing infection risk, liver issues, heart problems like changes in heartbeat rhythm or function. Side effects can vary from person to person.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My platelet count is above 100,000 and I haven't had a transfusion in the last week.
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My rhabdomyosarcoma is advanced, cannot be surgically removed, or has returned, and standard treatments have failed.
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I am 17 or older and can do most daily activities, or I am under 16 and mostly active.
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I am over 18 years old for the first group, or over 12 for later groups.
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My liver function tests are within the required range.
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My kidney function, measured by creatinine levels, is within the normal range.
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All side effects from my previous cancer treatments, except for hair loss, have mostly gone away.
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My hemoglobin level is above 9.0 g/dL, transfusions included.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My heart's electrical cycle (QTc interval) is longer than usual.
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I have not fully recovered from recent surgery, except for procedures to place a central venous catheter or port.
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I haven't had heart issues, strokes, clots, or needed heart rhythm drugs in the last 6 months.
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I do not have any active infections, including hepatitis and HIV.
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I have brain metastases that are causing symptoms.
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I am not taking medication that affects my heart's rhythm.
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I am currently breastfeeding.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To describe any dose-limiting toxicity (DLT)
To determine the maximum tolerated dose (MTD) or highest protocol defined doses (in the absence of exceeding the MTD)
Secondary study objectives
Area under the Plasma Concentration versus Time Curve (AUC) of mocetinostat
Clearance (CL) of mocetinostat
Disease Control (DC)
+7 more

Side effects data

From 2016 Phase 2 trial • 20 Patients • NCT02303262
60%
Fatigue
40%
Anemia
40%
Nausea
40%
Neutrophil count decreased
30%
Anorexia
30%
Platelet count decreased
30%
Dyspnea
25%
Vomiting
20%
Dysgeusia
20%
Insomnia
20%
Diarrhea
20%
White blood cell decreased
15%
Constipation
10%
Hypertension
10%
Lymphocyte count decreased
10%
Hypoalbuminemia
10%
Alkaline phosphatase increased
10%
Decreased appetite
10%
Chills
10%
Pain
10%
Dry mouth
10%
Hypokalemia
10%
Dizziness
10%
Acid reflux
10%
Back pain
10%
Dehydration
10%
Headache
10%
Fever
5%
Malaise
5%
Peripheral sensory neuropathy
5%
Achy
5%
Facial acne
5%
Sciatic pain
5%
Pericardial tamponade
5%
Hydronephrosis
5%
Fracture
5%
Nasal irritation
5%
Thigh pain
5%
Pericarditis
5%
GGT increased
5%
QTCF elevated
5%
Erythematous neck rash
5%
Myalgia
5%
Hypotension
5%
Absolute neutrophil count decreased
5%
Night sweats
5%
Right sided chest pain
5%
Neck pain
5%
Arthralgia
5%
Elevated LFTs
5%
Neck stiffness
5%
Bruising
5%
Laryngeal inflammation
5%
Palpitations
5%
Pruritic rash
5%
Memory impairment
5%
Powerport soreness
5%
Hypocalcemia
5%
Pulmonary embolism
5%
Cord compression
5%
Calf tenderness/burning
5%
Pharyngitis
5%
Muscle cramps
5%
Fungal infection
5%
Hyponatremia
5%
Alanine Aminotransferase increased
5%
Cough
5%
Creatinine increased
5%
Flatulence
5%
Burping
5%
Dry skin
5%
Generalized muscle weakness
5%
Ejection fraction decreased
5%
Paresthesia
5%
Sore nostril
5%
Hypophosphatemia
5%
Weight loss
5%
Bronchopulmonary hemorrhage
5%
Failure to thrive
5%
Febrile neutropenia
5%
Mucositis
5%
Neutropenia
5%
Non-cardiac chest pain
5%
Pericardial effusion
5%
Pneumonia
5%
Syncope
5%
Upper respiratory infection
5%
Urinary tract infection
5%
Sharp left sided pain
5%
Shoulder pain
5%
Sore throat
5%
Stomatitis
5%
Throat irritation
5%
Thrush
5%
Ventricular arrhythmia
5%
Vertigo
5%
Voice alteration
100%
80%
60%
40%
20%
0%
Study treatment Arm
Mocetinostat and Gemcitabine

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment (vinorelbine, mocetinostat)Experimental Treatment2 Interventions
Participants receive mocetinostat in combination with vinorelbine
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vinorelbine
2013
Completed Phase 4
~2190
Mocetinostat
2013
Completed Phase 2
~60

Find a Location

Who is running the clinical trial?

Jonsson Comprehensive Cancer CenterLead Sponsor
370 Previous Clinical Trials
35,089 Total Patients Enrolled
1 Trials studying Rhabdomyosarcoma
14 Patients Enrolled for Rhabdomyosarcoma
Mirati Therapeutics Inc.Industry Sponsor
70 Previous Clinical Trials
8,091 Total Patients Enrolled
Phase One FoundationOTHER
4 Previous Clinical Trials
265 Total Patients Enrolled
Noah C. Federman, MDPrincipal InvestigatorUniversity of California at Los Angeles

Media Library

Mocetinostat (Histone Deacetylase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04299113 — Phase 1
Rhabdomyosarcoma Research Study Groups: Treatment (vinorelbine, mocetinostat)
Rhabdomyosarcoma Clinical Trial 2023: Mocetinostat Highlights & Side Effects. Trial Name: NCT04299113 — Phase 1
Mocetinostat (Histone Deacetylase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04299113 — Phase 1
~3 spots leftby May 2025