Recruiting PHASE1, PHASE2 Rhabdomyosarcoma

New treatment option for Rhabdomyosarcoma

Official title FaR-RMS: An Overarching Study for Children and Adults With Frontline and Relapsed RhabdoMyoSarcoma

ClinicalTrials.gov ID: NCT04625907

What this study is testing

What is Irinotecan?

Irinotecan is an investigational medicine, being studied as a potential treatment for rhabdomyosarcoma.

Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.

What it's testing: FaR-RMS is an over-arching study for children and adults with newly diagnosed and relapsed rhabdomyosarcoma (RMS)

Phase 2: a mid-size study of how well it works

A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.

Who can take part

Adults

You may be able to join if

for study entry - Mandatory at first point of study entry
Histologically confirmed diagnosis of RMS (except pleomorphic RMS)
Written informed consent from the patient and/or the parent/legal guardian Phase 1b Dose Finding - IRIVA Inclusion
Entered in to the FaR-RMS study at diagnosis
Very High Risk disease

See the full eligibility criteria

Who can join

for study entry - Mandatory at first point of study entry
Histologically confirmed diagnosis of RMS (except pleomorphic RMS)
Written informed consent from the patient and/or the parent/legal guardian Phase 1b Dose Finding - IRIVA Inclusion
Entered in to the FaR-RMS study at diagnosis
Very High Risk disease
Age \>12 months and ≤25 years
No prior treatment for RMS other than surgery
Medically fit to receive treatment
Adequate hepatic function:
Total bilirubin ≤ 1.5 times upper limit of normal (ULN) for age, unless the patient is known to have Gilbert's syndrome
ALT or AST \< 2.5 X ULN for age
Absolute neutrophil count ≥1.0x 109/L
Platelets ≥ 80 x 109/L
Adequate renal function: estimated or measured creatinine clearance ≥60 ml/min/1.73 m2
Documented negative pregnancy test for female patients of childbearing potential
Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
Written informed consent from the patient and/or the parent/legal guardian Exclusion
Weight \<10kg
Active \> grade 2 diarrhoea
Prior allo- or autologous Stem Cell Transplant
Uncontrolled inter-current illness or active infection
Pre-existing medical condition precluding treatment
Urinary outflow obstruction that cannot be relieved prior to starting treatment
Active inflammation of the urinary bladder (cystitis)
Known hypersensitivity to any of the treatments or excipients
Second malignancy
Pregnant or breastfeeding women Frontline chemotherapy randomisation Very High Risk - CT1a Inclusion
Entered in to the FaR-RMS study at diagnosis
Very High Risk disease
Age ≥ 6 months
Available for randomisation ≤60 days after diagnostic biopsy/surgery
No prior treatment for RMS other than surgery
Medically fit to receive treatment
Adequate hepatic function : a. Total bilirubin ≤ 1.5 times upper limit of normal (ULN) for age, unless the patient is known to have Gilbert's syndrome
Absolute neutrophil count ≥1.0x 109/L (except in patients with documented bone marrow disease)
Platelets ≥ 80 x 109/L (except in patients with documented bone marrow disease)
Fractional Shortening ≥ 28%
Documented negative pregnancy test for female patients of childbearing potential
Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
Written informed consent from the patient and/or the parent/legal guardian Exclusion
Active \> grade 2 diarrhoea
Prior allo- or autologous Stem Cell Transplant
Uncontrolled inter-current illness or active infection
Pre-existing medical condition precluding treatment
Urinary outflow obstruction that cannot be relieved prior to starting treatment
Active inflammation of the urinary bladder (cystitis)
Known hypersensitivity to any of the treatments or excipients
Second malignancy
Pregnant or breastfeeding women Frontline chemotherapy randomisation High Risk - CT1b Inclusion
Entered in to the FaR-RMS study at diagnosis
High Risk disease
Age ≥ 6 months
Available for randomisation ≤60 days after diagnostic biopsy/surgery
No prior treatment for RMS other than surgery
Medically fit to receive treatment
Adequate hepatic function : a. Total bilirubin ≤ 1.5 times upper limit of normal (ULN) for age, except if the patient is known to have Gilbert's syndrome
Absolute neutrophil count ≥1.0x 109/L
Platelets ≥ 80 x 109/L
Documented negative pregnancy test for female patients of childbearing potential
Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
Written informed consent from the patient and/or the parent/legal guardian Exclusion
Active \> grade 2 diarrhoea
Prior allo- or autologous Stem Cell Transplant
Uncontrolled inter-current illness or active infection
Pre-existing medical condition precluding treatment
Urinary outflow obstruction that cannot be relieved prior to starting treatment
Active inflammation of the urinary bladder (cystitis)
Known hypersensitivity to any of the treatments or excipients
Second malignancy
Pregnant or breastfeeding women Frontline Radiotherapy Note: eligible patients may enter multiple radiotherapy randomisations. Radiotherapy Inclusion - for all radiotherapy randomisations
Entered in to the FaR-RMS study (at diagnosis or prior to radiotherapy randomisation)
Very High Risk, High Risk and Standard Risk disease
≥ 2 years of age
Receiving frontline induction treatment as part of the FaR-RMS trial or with a IVA/IVADo based chemotherapy regimen patients for whom. Note that patients for whom ifosfamide has been replaced with cyclophosphamide will...
Patient assessed as medically fit to receive the radiotherapy
Documented negative pregnancy test for female patients of childbearing potential
Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
Written informed consent from the patient and/or the parent/legal guardian Radiotherapy Exclusion - for all radiotherapy randomisations
Prior allo- or autologous Stem Cell Transplant
Second malignancy
Pregnant or breastfeeding women
Receiving radiotherapy as brachytherapy RT1a Specific Inclusion
Primary tumour deemed resectable (predicted R0/ R1 resection feasible) after 3 cycles of induction chemotherapy (6 cycles for metastatic disease)
Adjuvant radiotherapy required in addition to surgical resection (local decision).
Available for randomisation after cycle 3 and prior to the start of cycle 6 of induction chemotherapy for localised disease, or after cycle 6 and prior to the start of cycle 9 for metastatic disease RT1b Specific...
Primary tumour deemed resectable (predicted R0/R1 resection) after 3 cycles of induction chemotherapy (6 cycles for metastatic disease).
Adjuvant radiotherapy required in addition to surgical resection (local decision)
Higher Local Failure Risk (HLFR) based on presence of either of the following criteria:
Unfavourable site
Age ≥ 18yrs
Available for randomisation after cycle 3 and prior to the start of cycle 6 of induction chemotherapy for localised disease, or after cycle 6 and prior to the start of cycle 9 for metastatic disease RT1c Specific...
Primary radiotherapy indicated (local decision)
Higher Local Failure Risk (HLFR) based on either of the following criteria:
Unfavourable site
Age ≥ 18yrs
Available for randomisation after cycle 3 and prior to the start of cycle 6 of induction chemotherapy for localised disease, or after cycle 6 and prior to the start of cycle 9 for metastatic disease RT2
Available for randomisation after cycle 6 and before the start of cycle 9 of induction chemotherapy.
Unfavourable metastatic disease, defined as Modified Oberlin Prognostic Score 2-4
Note: Definition of metastatic lesions for RT2 eligibility Modified Oberlin Prognostic Score (1 point for each adverse factor):
Age ≥10y
Extremity, Other, Unidentified Primary Site
Bone and/ or Bone Marrow involvement
≥3 metastatic sites Unfavourable metastatic disease: 2- 4 adverse factors Favourable metastatic disease: 0-1 adverse factors Maintenance chemotherapy (Very High Risk) - CT2a Inclusion Randomisation must take place...
Entered in to the FaR-RMS study (at diagnosis or at any subsequent time point)
Very High Risk disease
Received frontline induction chemotherapy as part of the FaR-RMS trial or with a IVA/IVADo based chemotherapy regimen a. Patients for whom ifosfamide has been replaced with cyclophosphamide will be eligible
Completed 11 cycles of VnC maintenance treatment (either oral or IV regimens)
No evidence of progressive disease
Absence of severe vincristine neuropathy - i.e requiring discontinuation of vincristine treatment)
Medically fit to continue to receive treatment
Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
Written informed consent from the patient and/or the parent/legal guardian Exclusion
Prior allo- or autologous Stem Cell Transplant
Uncontrolled intercurrent illness or active infection
Urinary outflow obstruction that cannot be relieved prior to starting treatment
Active inflammation of the urinary bladder (cystitis)
Second malignancy
Pregnant or breastfeeding women Maintenance chemotherapy (High Risk) - CT2b Randomisation must take place during the 6th cycle of maintenance chemotherapy. Inclusion
Entered in to the FaR-RMS study (at diagnosis or at any subsequent time point)
High Risk disease
Received frontline induction chemotherapy as part of the FaR-RMS trial or with a IVA based chemotherapy regimen. Note that patients for whom ifosfamide has been replaced with cyclophosphamide will be eligible
Completed 5 cycles of VnC maintenance treatment
No evidence of progressive disease
Absence of severe vincristine neuropathy i.e. requiring discontinuation of vincristine treatment
Medically fit to continue to receive treatment
Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
Written informed consent from the patient and/or the parent/legal guardian Exclusion
Prior allo- or autologous Stem Cell Transplant
Uncontrolled inter current illness or active infection
Urinary outflow obstruction that cannot be relieved prior to starting treatment
Active inflammation of the urinary bladder (cystitis)
Second malignancy
Pregnant or breastfeeding women CT3 Relapsed Chemotherapy Inclusion:
Entered in to the FaR-RMS study (at diagnosis or at any subsequent time point)
First or subsequent relapse of histologically verified RMS
Age ≥ 6 months
Measurable or evaluable disease
No cytotoxic chemotherapy or other investigational medicinal product (IMP) within previous three weeks: within two weeks for vinorelbine and cyclophosphamide maintenance chemotherapy
Medically fit to receive trial treatment
Documented negative pregnancy test for female patients of childbearing potential within 7 days of planned randomisation
Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
Written informed consent from the patient and/or the parent/legal guardian Exclusion:
Progression during frontline therapy without previous response (=Refractory to first line treatment)
Prior regorafenib or temozolomide
Active \> grade 1 diarrhoea
ALT or AST \>3.0 x upper limit normal (ULN)
Bilirubin, Total \>1.5 x ULN; total bilirubin is allowed up to 3 x ULN if Gilbert's syndrome is documented
Patients with unstable angina or new onset angina (within 3 months of planned date of randomisation), recent myocardial infarction (within 6 months of randomisation) and those with cardiac failure New York Heart...
Uncontrolled hypertension \> 95th centile for age and gender
Prior allo- or autologous Stem Cell Transplant
Uncontrolled inter-current illness or active infection
Pre-existing medical condition precluding treatment
Known hypersensitivity to any of the treatments or excipients
Second malignancy
Pregnant or breastfeeding women

The study team makes the final eligibility decision.

Where it's taking place

Brisbane, Australia
Camperdown, Australia
Clayton, Australia
Melbourne, Australia
New Lambton Heights, Australia
Perth, Australia
Sydney, Australia
Westmead, Australia
Woolloongabba, Australia
Linz, Austria
Vienna, Austria
Brussels, Belgium
Ghent, Belgium
Leuven, Belgium
Liège, Belgium
Brno, Czechia
Aarhus, Denmark
Copenhagen, Denmark
Angers, France
Besançon, France

+ 81 more site(s).

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, not specified. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Brisbane, Australia; Camperdown, Australia; Clayton, Australia; Melbourne, Australia; New Lambton Heights, Australia; Perth, Australia and 95 more location(s). Enter your location above to see the nearest site and check your eligibility.

Explore other conditions

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.