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RhabdomyosarcomaPhase 1NCT04299113

Mocetinostat With Vinorelbine in Children, Adolescents & Young Adults With Refractory and/or Recurrent Rhabdomyosarcoma

A Phase 1 study testing Vinorelbine and Mocetinostat in people with Rhabdomyosarcoma, run by Jonsson Comprehensive Cancer Center at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

This phase I trial studies the side effects and best dose of mocetinostat when given together with vinorelbine to see how well it works in treating children, adolescents, and young adults with rhabdomyosarcoma that has spread to nearby tissues or lymph nodes and cannot be removed by surgery (locally advanced… Read more about this study

Who is it for?
Ages 13 years and older
Conditions the study is for
Rhabdomyosarcoma
What is being tested?
Vinorelbine and Mocetinostat
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
14 participants
Where
1 siteUnited States
Study dates
14 May 2020to 30 Dec 2027
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 13 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Willing and able to provide written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent. For subjects < 18 years of age, their parents or legal guardians must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines
  • 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
  • Measurable disease according to RECIST version 1.1
  • Prior cancer therapy: Subjects may have received any number of prior therapy regimens. In the investigator's opinion, subjects must have tolerated prior cytotoxic therapies well and have adequate bone marrow reserve. At the time of treatment initiation, at least 3 weeks must have elapsed after prior cytotoxic chemotherapy. At least 7 days must have elapsed since completion of any prior non-cytotoxic cancer therapy and any associated AEs must have resolved
  • Prior radiotherapy is allowed if >= 2 weeks have elapsed for local palliative radiation therapy (XRT) (small port); >= 6 months must have elapsed if prior total body irradiation, craniospinal XRT or if > 50% radiation of the pelvis; > 6 weeks must have elapsed if other substantial bone marrow radiation (defined per principal investigator's [PI's] discretion). Subjects who have received brain irradiation must have completed whole brain radiotherapy and/or gamma knife at least 4 weeks prior to enrollment
  • Subjects with controlled asymptomatic central nervous system (CNS) involvement are allowed in absence of therapy with anticonvulsants. Subjects not requiring steroids or requiring steroids at a stable dose (=< 4 mg/day dexamethasone or equivalent) for at least 2 weeks are eligible
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  • Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (version 4.03) grade < 1 or to the baseline laboratory values as defined below
  • Eastern Cooperative Oncology Group (ECOG) performance status (PS) =< 2 in subjects >= 17 years old; or Karnofsky/Lansky > 50 in subjects < 16 years old
  • Subjects age > 18 years for first cohort. Subjects must be > 12 years old for the second and subsequent cohorts
  • Life expectancy of at least 3 months
  • Absolute neutrophil count (ANC) >= 1000/mm^3 (>= 1.0 x 10^9/L)
  • 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)
  • Hemoglobin > 9.0 g/dL (transfusions are allowed)
  • Serum creatinine =< 1.5 x upper limit of normal (ULN) or creatinine clearance > 60 mL/min
  • Total serum bilirubin =< 1.5 x ULN; =< 5 x ULN if Gilbert's syndrome
  • Liver transaminases (aspartate aminotransferase [AST]/alanine aminotransferase [ALT]) =< 2.5 x ULN; =< 5 x ULN if liver metastases are present
  • Pregnancy test if female of child-bearing potential negative within 7 days of starting treatment
  • Cardiac ejection fraction > 50% or shortening fraction > 28% by echocardiography (ECHO) or multigated acquisition scan (MUGA)
  • Females of child-bearing potential must have a negative pregnancy test during screening and be neither breastfeeding nor intending to become pregnant during study participation. Females of childbearing potential must agree to avoid pregnancy during the study and commit to abstinence from heterosexual intercourse or agree to use two methods of birth control (one highly effective method and one additional effective method) at least 4 weeks before the start of protocol therapy, for the duration of study participation, and for 6 months after the last dose of mocetinostat
  • Males with partner(s) of childbearing potential must take appropriate precautions to avoid fathering a child from the screening period until 90 days after receiving the last dose of mocetinostat. They must commit to abstinence from heterosexual intercourse or agree to use appropriate barrier contraception
  • Prior to enrollment of females or males of reproductive potential, the investigator must document confirmation of the subject's understanding of the possible teratogenic effects of mocetinostat
  • Willingness and ability to comply with scheduled visits, treatment plan, laboratory tests and other study procedures

Who cannot join

  • Current participation in another therapeutic clinical trial
  • Symptomatic brain metastases
  • History of previous cancer (non RMS), except squamous cell or basal-cell carcinoma of the skin or any in situ carcinoma that has been completely resected, which required therapy within the previous 3 years. Other low grade cancers can be reviewed and allowed at the discretion of the PI
  • Incomplete recovery from any surgery (other than central venous catheter or port placement) prior to treatment
  • Any of the following in the past 6 months: pericarditis, pericardial effusion, symptomatic congestive heart failure, cerebrovascular accident or transient ischemic attack, pulmonary embolism, deep vein thrombosis, symptomatic bradycardia, requirement for anti-arrhythmic medication
  • 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])
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  • History of additional risk factors for torsade de pointes (e.g., heart failure, family history of long QT syndrome)
  • Use of concomitant medications that increase or possibly increase the risk to prolong the QTc interval and/or induce torsades de pointes ventricular arrhythmia
  • Females who are breastfeeding/lactating
  • Known active infections (e.g., bacterial, fungal, viral including hepatitis and human immunodeficiency virus [HIV] positivity)
  • Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study or compromise protocol objectives in the opinion of the investigator and/or the sponsor

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Rebecca Phelan

    Los Angeles, California, 90095

Other trials in United States

About this study

Official title: A Phase I Dose Escalation/Expansion Clinical Trial of Mocetinostat in Combination With Vinorelbine in Children, Adolescents and Young Adults With Refractory and/or Recurrent Rhabdomyosarcoma (RMS)

This phase I trial studies the side effects and best dose of mocetinostat when given together with vinorelbine to see how well it works in treating children, adolescents, and young adults with rhabdomyosarcoma that has spread to nearby tissues or lymph nodes and cannot be removed by surgery (locally advanced unresectable) or has spread to other places in the body (metastatic), and does not respond to treatment (refractory) or has come back (relapsed). Mocetinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as vinorelbine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving mocetinostat and vinorelbine may work better in treating children, adolescents, and young adults with rhabdomyosarcoma compared to vinorelbine alone.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine the first cycle dose-limiting toxicities (DLTs), maximum tolerated dose (MTD), and a biologically effective and recommended phase 2 dose (RP2D) of mocetinostat administered orally three times per week for a total of 9 doses per 21 day cycle given in combination with vinorelbine on days 1, 8, 15 of 21 day cycles in subjects with refractory or recurrent rhabdomyosarcoma (RMS). (Phase 1 Dose Escalation) II. To determine the progression-free survival (PFS), defined as time from first dose of vinorelbine to tumor progression or death due to any cause, at the RP2D of mocetinostat administered orally three times per week starting on day 3 for a total of 9 doses per 21 day cycle given in combination with vinorelbine on days 1, 8, 15 of a 21 day cycle in subjects with refractory or recurrent RMS. (Expansion Cohort)

SECONDARY OBJECTIVES:

  1. Safety profile of mocetinostat in combination with vinorelbine as characterized by adverse event (AE) type, severity, timing and relationship to study drugs, as well as laboratory abnormalities in the first and subsequent treatment cycles. (Phase 1 Dose Escalation) II. Pharmacokinetics (PK) of mocetinostat in plasma. (Phase 1 Dose Escalation) III. Clinical benefit rate (CBR = complete response [CR] + partial response [PR] and stable disease [SD]) of mocetinostat + vinorelbine in metastatic/refractory/unresectable RMS. (Phase 1 Dose Escalation) IV. Antitumor activity of mocetinostat + vinorelbine in refractory/recurrent RMS as measured by overall response rate (ORR), duration of response (DOR), disease control (DC), duration of disease control, as well as progression-free survival (PFS). (Phase 1 Dose Escalation) V. Pharmacodynamics of mocetinostat on molecular targets in surrogate tissue. (Phase 1 Dose Escalation and Expansion Cohort) VI. Exploratory biomarker development to enable prediction of drug toxicity, tumor response and the mechanism(s) of acquired study drug resistance. (Phase 1 Dose Escalation and Expansion Cohort) VII. Obtain RMS tissue biological samples pre-treatment and at progression to assess for differences in gene expression by next gen (generation) sequencing and ribonucleic acid (RNA) sequencing (seq). (Phase 1 Dose Escalation and Expansion Cohort) VIII. Antitumor activity of mocetinostat + vinorelbine in metastatic/refractory RMS as measured by overall response rate (ORR) and duration of response (DOR), disease control (DC), duration of disease control, as well as progression-free survival (PFS) according to Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1. (Expansion Cohort) IX. Safety and tolerability of mocetinostat and vinorelbine as characterized by adverse event type, severity, timing and relationship to study drug, as well as laboratory abnormalities. (Expansion Cohort)

OUTLINE: This is a phase I, dose-escalation study of mocetinostat followed by additional studies.

Being tested
Vinorelbine, Mocetinostat
Keywords
Locally Advanced Rhabdomyosarcoma, Recurrent Rhabdomyosarcoma, Refractory Rhabdomyosarcoma, Unresectable Rhabdomyosarcoma
Medical subject headings
Rhabdomyosarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VinorelbineDrug

    Also called 3'',4''-Didehydro-4''-deoxy-C''-norvincaleukoblastine, 5''-Nor-Anhydrovinblastine, 71486-22-1, Dihydroxydeoxynorvinkaleukoblastine, nor-5''-Anhydrovinblastine, vinorelbine, VINORELBINE

    Given IV

  • MocetinostatDrug

    Also called 726169-73-9, Benzamide, N-(2-aminophenyl)-4-[[[4-(3-pyridinyl)-2-pyrimidinyl]amino]methyl]-, MG-0103, MGCD0103, MGCD0103, N-(2-aminophenyl)-4-((4-pyridin-3-ylpyrimidin-2-ylamino)methyl)benzamide

    Given PO

Study groups in NCT04299113
Study groupRoleWhat they receive
Treatment (vinorelbine, mocetinostat)ExperimentalParticipants receive mocetinostat in combination with vinorelbine
Receives: Drug: Vinorelbine; Drug: Mocetinostat

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To describe any dose-limiting toxicity (DLT)

    Percentage of subjects with dose-limiting toxicities (DLTs) as assessed by NCI CTCAE (Version 4.03)

    Measured: 1 year

  2. To determine the maximum tolerated dose (MTD) or highest protocol defined doses (in the absence of exceeding the MTD)

    The MTD is the highest dose associated with first-cycle DLT in < 33% of subjects

    Measured: 1 year

Secondary outcomes (10)

  1. To determine the Recommended Phase 2 Dose (RP2D) for mocetinostat in combination with vinorelbine

    The RP2D may be determined by the MTD or optimal target inhibition with an acceptable safety profile

    Measured: 1 year

  2. Incidence of Adverse Events (AEs) as assessed by NCI CTCAE (Version 4.03)

    Assess incidence of all AEs by NCI CTCAE (Version 4.03) grades 1-5

    Measured: 1 year

  3. Objective Tumor Response

    Measured using RECIST, Version 1.1

    Measured: 2 years

  4. Progression Free Survival (PFS)

    Estimated using Kaplan-Meier methodology

    Measured: 2 years

  5. Disease Control (DC)

    Proportion of subjects with a confirmed Complete Response (CR), Partial Response (PR), or Stable Disease (SD) according to RECIST v1.1

    Measured: 2 years

  6. Duration of Response (DOR)

    Measured from the first date a response is identified (either CR or PR) until the date of disease progression

    Measured: 2 years

  7. Area under the Plasma Concentration versus Time Curve (AUC) of mocetinostat

    Continuous variables will be summarized with means, standard deviations, medians, minimums, and maximums

    Measured: 2 years

  8. Clearance (CL) of mocetinostat

    Continuous variables will be summarized with means, standard deviations, medians, minimums, and maximums

    Measured: 2 years

  9. Half-Life [T1/2] of mocetinostat

    Continuous variables will be summarized with means, standard deviations, medians, minimums, and maximums

    Measured: 2 years

  10. Volume of Distribution (Vd) of mocetinostat

    Continuous variables will be summarized with means, standard deviations, medians, minimums, and maximums

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Mar 2020
  2. Study start14 May 2020
  3. Primary completion30 Dec 2026 (estimated)
  4. Study completion30 Dec 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
14 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04299113
Sponsor's protocol ID
19-000353
Other IDs
NCI-2019-08263
Status last verified
Jul 2026
First submitted
10 Feb 2020
Last updated
17 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Vinorelbine and Mocetinostat in people with Rhabdomyosarcoma, run by Jonsson Comprehensive Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 22 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 17 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To describe any dose-limiting toxicity (DLT) (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04299113.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jul 2026.

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