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AzTrials

RhabdomyosarcomaPhase 2NCT04388839

Evolutionary Therapy for Rhabdomyosarcoma

A Phase 2 study testing Vincristine, Cyclophosphamide and Vinorelbine in people with Rhabdomyosarcoma, run by H. Lee Moffitt Cancer Center and Research Institute at 18 sites in United States.

Active, not recruiting

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

Official record
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Trial summary

This clinical trial will evaluate 4 different strategies of chemotherapy schedules in newly diagnosed participants with metastatic Fusion Positive (alveolar) Rhabdomyosarcoma. The participant and their physician will choose from: Arm A) a first strike therapy, Arm B) a first strike-second strike (maintenance) therapy… Read more about this study

Who is it for?
Any age
Conditions the study is for
Rhabdomyosarcoma
What is being tested?
Vincristine, Cyclophosphamide and Vinorelbine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
18 sitesUnited States
Study dates
29 Dec 2020to Aug 2027
Phase
Phase 2
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 Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Participants must have a new histologic diagnosis of rhabdomyosarcoma
  • Participants must have FISH, PCR or other molecular confirmation of PAX/FOXO1 fusion per institutional standards
  • Participants must have sufficient tissue (up to 10 unstained FFPE) for correlative testing
  • All participants must have distant metastatic disease; either biopsy positive or PET avid extranodal or distant nodal lesions determined by the investigator to be metastatic disease. Patients with a single distant metastatic site that has been excised prior to study entry are eligible
  • No prior systemic chemotherapy
  • Participants enrolled to Arm B, maintenance, must be able to take oral cyclophosphamide. Note: enteral administration of cyclophosphamide is allowable.
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  • Males and females of reproductive potential may not participate unless they have agreed to the use of, at minimum, two methods of contraception during and after treatment or abstinence.
  • Women of childbearing potential should adhere to contraception for a period of 4 months after completion of systematic chemotherapy administration
  • Men who are sexually active with women of child bearing potential should adhere to contraception for a period of 4 months after completion of systematic chemotherapy administration
  • All patients and/or their parents or legal guardians must have the ability to understand and the willingness to sign a written informed consent or assent document.

Who cannot join

  • Participants with regional lymph nodes as the only site of disease are not eligible. Distant nodal sites alone are eligible
  • Participants who are receiving any other investigational agents for rhabdomyosarcoma are ineligible
  • Participants must not be receiving any additional medicines being given for the specific purpose of treating cancer. Alternative medications including, but not limited to cannabis based products would not be a reason for exclusion
  • Participants are ineligible if they have uncontrolled intercurrent illness including, but not limited to:
  • ongoing or active infection not expected to resolve with current antibiotic plan
  • cardiac arrhythmia
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  • psychiatric illness/social situations that would limit compliance with study requirements
  • Patients who are pregnant or breastfeeding are not eligible because there is no available information regarding human fetal or teratogenic toxicities. Females of childbearing potential must have a negative serum or urine pregnancy test within 24 hours of starting protocol therapy.
  • Participants who are considered unable to comply with the safety monitoring requirements of the study are not eligible

Trial locations and contacts

18 sites in 1 country.

18 sites in 1 country

United States18 sites

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045

  • University of Alabama at Birmingham Comprehensive Cancer Center

    Birmingham, Alabama, 35294

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14263

  • University of North Carolina Lineberger Comprehensive Cancer Center

    Chapel Hill, North Carolina, 27599

  • Carolinas Medical Center, Levine Cancer Institute

    Charlotte, North Carolina, 28204

  • Cleveland Clinic

    Cleveland, Ohio, 44106

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Duke Children's Hospital

    Durham, North Carolina, 27710

  • University of Florida

    Gainesville, Florida, 32610

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106

  • MD Anderson

    Houston, Texas, 77030

  • University of Miami - Sylvester Comprehensive Cancer Center

    Miami, Florida, 33136

  • Vanderbilt - Ingram Cancer Center

    Nashville, Tennessee, 37232

  • Primary Children's Medical Center/Utah

    Salt Lake City, Utah, 84113

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701

  • Moffitt Cancer Center

    Tampa, Florida, 33612

  • Montefiore Medical Cancer Center

    The Bronx, New York, 10467

Other trials in United States

About this study

Official title: Evolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma

This clinical trial will evaluate 4 different strategies of chemotherapy schedules in newly diagnosed participants with metastatic Fusion Positive (alveolar) Rhabdomyosarcoma. The participant and their physician will choose from: Arm A) a first strike therapy, Arm B) a first strike-second strike (maintenance) therapy, Arm C) an adaptively timed therapy, and Arm D) conventional chemotherapy.

Being tested
Vincristine, Cyclophosphamide, Vinorelbine, Actinomycin D, Cyclophosphamide Pill
Keywords
Soft Tissue Cancer, Skeletal Muscle Tissue Cancer, Sarcoma
Medical subject headings
Rhabdomyosarcoma, Sarcoma

What participants receive

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

  • VincristineDrug

    IV push over 1 minute with dosing ranging from 0.24mg up to 1.5mg

  • CyclophosphamideDrug

    IV over 60 minutes with dosing ranging from 220mg to 1200mg

  • VinorelbineDrug

    Also called Navelbine

    IV push over 6-10 minutes with dosing ranging from 4mg-25mg

  • Actinomycin DDrug

    Also called Cosmegen

    Actinomycin D should not be given with radiation. Will be administered through IV over 3-5 minutes with dosing ranging from 0.025mg-0.045mg

  • Cyclophosphamide PillDrug

    Based on Body Surface Area (BSA) round to nearest 25mg

Study groups in NCT04388839
Study groupRoleWhat they receive
Arm A - First StrikeExperimentalParticipants will receive 42 weeks of conventional doses of vinorelbine, actinomycin D and cyclophosphamide
Receives: Drug: Cyclophosphamide; Drug: Vinorelbine; Drug: Actinomycin D
Arm B - Second Strike - MaintenanceExperimentalParticipants will receive conventional doses of Vincristine/Actinomycin D/Cyclophosphamide (VAC) until complete response (CR) for 12-42 weeks and then switch to up to 2 years of vinorelbine/oral cyclophoshamide
Receives: Drug: Vincristine; Drug: Cyclophosphamide; Drug: Vinorelbine; Drug: Actinomycin D; Drug: Cyclophosphamide Pill
Arm C - Adaptive TherapyExperimentalTherapy with VAC that starts and stops based on response, adaptive timing of therapy, with a prolonged time to progression rather than complete remission goal
Receives: Drug: Vincristine; Drug: Cyclophosphamide; Drug: Actinomycin D
Arm - D Conventional TherapyActive comparatorParticipants will receive a chemotherapy combination based on published trials. An example would be 42 weeks of VAC but may also include irinotecan, doxorubicin, ifosfamide, etoposide.
Receives: Drug: Vincristine; Drug: Cyclophosphamide; Drug: Actinomycin D

What is being measured

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

  1. First Strike Event Free Survival

    Participants who choose the first strike treatment will have event free survival assessed at 3 years after initiating treatment. Event free survival is defined as time from treatment initiation to event which includes (1) any recurrence (local or regional, or distant) and (2) death due to any cause.

    Measured: Baseline to 3 years

  2. Second Strike Event Free Survival

    Participants who choose the second strike treatment will have event free survival assessed at 3 years after initiating treatment. Event free survival is defined as time from treatment initiation to event which includes (1) any recurrence (local or regional, or distant) and (2) death due to any cause

    Measured: Baseline to 3 years

  3. Adaptive Therapy Event Free Survival

    Participants who choose the adaptive therapy will have event free survival assessed at 3 years after initiating treatment. Event free survival is defined as time from treatment initiation to event which includes (1) progression that does not respond to additional VAC dosing and (2) death due to any cause

    Measured: Baseline to 3 years

Secondary outcomes (2)

  1. Overall Survival

    The time to event endpoint of overall survival is defined as the duration of time from diagnosis to death or last follow-up, where event would be death from any cause

    Measured: 5 years

  2. Treatment-related adverse events of a certain grade or higher

    Number of participants with treatment-related adverse events of a certain grade or higher and hematological/biochemical toxicities based on laboratory measurements as assessed by CTCAE v5.0

    Measured: Baseline to 5 years

Timeline

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

  1. First posted14 May 2020
  2. Study start29 Dec 2020
  3. Primary completionAug 2026 (estimated)
  4. Study completionAug 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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04388839
Sponsor's protocol ID
MCC-20339
Status last verified
Nov 2025
First submitted
11 May 2020
Last updated
26 Nov 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Vincristine, Cyclophosphamide and Vinorelbine in people with Rhabdomyosarcoma, run by H. Lee Moffitt Cancer Center and Research Institute at 18 sites 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 10 inclusion and 9 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 26 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

First Strike Event Free Survival (time frame: Baseline to 3 years). The study lists 3 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 NCT04388839.

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 26 Nov 2025.

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