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ISRCTN45535982
Alternativer Eintrag: NCT04625907

An overarching study for children and adults with frontline and relapsed rhabdomyosarcoma

Datenbasis: WHO (Import vom 07.11.2024)
Geändert: 10.07.2024, 01:00
Krankheitskategorie:

Health conditions (Datenquelle: WHO)

Rhabdomyosarcoma
Cancer

Interventions (Datenquelle: WHO)

FaR-RMS is an over-arching international study for patients with newly diagnosed and relapsed RMS including multiarm, multi-stage questions involving chemotherapy and radiotherapy, with no upper age limit.

Study Entry
FaR-RMS includes a study entry point where all patients with RMS may give consent for the analysis of their biological samples and tumour pathology, alongside the collection of very basic patient characteristics, a treatment summary, and follow-up data for events.

Risk Group Allocation
Patient disease status and risk group allocation will determine which randomisations they are eligible to enter. The risk group assignment is based on analyses performed on outcome data from the recent EpSSG-RMS2005 trial.

Treatment Questions (randomisation/Phase 1b registration)
Patients may be entered into more than one treatment questions following study entry dependent on their disease status. Treatment questions may be available for patients with VHR, HR and Standard Risk (SR) disease. Separate consent is required for study entry and for each trial question. Newly diagnosed patients should, where possible, be entered into the FaR-RMS study at the time of initial diagnosis prior to receiving any chemotherapy. However, newly diagnosed patients can enter at the point of radiotherapy or maintenance, and those with relapsed disease can enter the study at the point of relapse even if not previously entered at initial diagnosis.

Consent
For patients who appear to meet the criteria for participation in the study they and their parents/guardian (if applicable) will be approached by a Consultant or a delegated member of the research team working on this study. This trial will be explained to them and they will be given a copy of the age-specific Patient/Parent Information She

Inclusion/Exclusion Criteria (Datenquelle: WHO)

Gender: Both
Inclusion criteria: Inclusion criteria for study entry:
1. Histologically confirmed diagnosis of RMS (except pleomorphic RMS)
2. Written informed consent from the patient and/or the parent/legal guardian

Inclusion criteria for all randomisations and registrations:
1. 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
2. Written informed consent from the patient and/or the parent/legal guardian
3. Medically fit to receive treatment

Frontline chemotherapy specific inclusion:
1. Entered into the FaR-RMS study at diagnosis
2. No prior treatment for RMS other than surgery
3. Documented negative pregnancy test for female patients of childbearing potential
4. 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

Phase 1b specific inclusion:
1. VHR disease
2. Age >12 months and =25 years
3. Adequate hepatic function: ALT or AST < 2.5 X ULN for age
4. Adequate renal function: estimated or measured creatinine clearance =60 ml/min/1.73 m2
5. Absolute neutrophil count =1.0x 10^9/L
6. Platelets = 80 x 10^9/L

CT1a specific inclusion:
1. VHR disease
2. Age = 6 months
3. Available for randomisation =60 days after diagnostic biopsy/surgery
4. Fractional Shortening = 28%
5. Absolute neutrophil count =1.0x 10^9/L (except in patients with documented bone marrow disease)
6. Platelets = 80 x 109/L (except in patients with documented bone marrow disease)

CT1b specific inclusion:
1. HR disease
2. Age = 6 months
3. Available for randomisation =60 days after diagnostic biopsy/surgery
4. Absolute neutrophil count =1.0x 10^9/L
5. Platelets = 80 x 10^9/L

Radiotherapy inclusion:
1. Entered into the FaR-RMS study (at diagnosis or prior to radiotherapy randomisation)
2. VHR, HR and SR disease
3. = 2 years of age
4. Receiving frontline induction treatment as part of the FaR-RMS trial or with a IVA/IVADo based chemotherapy regimen. Note that patients for whom ifosfamide has been replaced with cyclophosphamide will be eligible
5. Documented negative pregnancy test for female patients of childbearing potential

RT1a and RT1b specific inclusion:
1. Primary tumour deemed resectable (predicted R0/ R1 resection feasible) after 3 cycles of induction chemotherapy (6 cycles for metastatic disease)
2. Adjuvant radiotherapy required in addition to surgical resection (local decision)
3. 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 and RT1c specific inclusion:
1. Higher Local Failure Risk (HLFR) based on presence of either of the following criteria:
2. Unfavourable site
3. Age = 18yrs

RT1c specific inclusion:
1. Primary radiotherapy indicated (local decision)
2. 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 specific inclusion:
1. Available for randomisation after cycle 6 and before the start of cycle 9 of induction chemotherapy
2. Unfavourable metastatic disease, defined as Modified Oberlin Prognostic Score 2-4

Maintenance specific Inclusion:
1. Received frontline induction chemotherapy as part of the FaR-RMS trial or with a IVA/IVADo based chemotherapy regi
Exclusion criteria: Phase 1b specific exclusion:
1. Weight <10kg
2. Active > grade 2 diarrhoea
3. Prior allo- or autologous Stem Cell Transplant
4. Uncontrolled inter-current illness or active infection
5. Pre-existing medical condition precluding treatment
6. Known hypersensitivity to any of the treatments or excipients
7. Second malignancy
8. Pregnant or breastfeeding women
9. Urinary outflow obstruction that cannot be relieved prior to starting treatment
10. Active inflammation of the urinary bladder (cystitis)

CT1a and CT1b specific exclusion:
1. Active > grade 2 diarrhoea
2. Prior allo- or autologous Stem Cell Transplant
3. Uncontrolled inter-current illness or active infection
4. Pre-existing medical condition precluding treatment
5. Known hypersensitivity to any of the treatments or excipients
6. Second malignancy
7. Pregnant or breastfeeding women
8. Urinary outflow obstruction that cannot be relieved prior to starting treatment
9. Active inflammation of the urinary bladder (cystitis)

Radiotherapy specific exclusion
1. Prior allo- or autologous Stem Cell Transplant
2. Second malignancy
3. Pregnant or breastfeeding women
4. Receiving radiotherapy as brachytherapy

CT2a and CT2b specific exclusion:
1. Prior allo- or autologous Stem Cell Transplant
2. Uncontrolled inter current illness or active infection
3. Second malignancy
4. Pregnant or breastfeeding women
5. Urinary outflow obstruction that cannot be relieved prior to starting treatment
6. Active inflammation of the urinary bladder (cystitis)

CT3 specific exclusion:
1. Active > grade 2 diarrhoea
2. Prior allo- or autologous Stem Cell Transplant
3. Uncontrolled inter-current illness or active infection
4. Pre-existing medical condition precluding treatment
5. Known hypersensitivity to any of the treatments or excipients
6. Second malignancy
7. Pregnant or breastfeeding women

Weitere Angaben zur Studie im WHO-Primärregister

https://www.isrctn.com/ISRCTN45535982

Weitere Angaben zur Studie aus der Datenbank der WHO (ICTRP)

https://trialsearch.who.int/Trial2.aspx?TrialID=ISRCTN45535982
Weitere Informationen zur Studie

Datum der Studienregistrierung

11.06.2019

Einschluss der ersten teilnehmenden Person

31.07.2020

Rekrutierungsstatus

Ongoing

Wissenschaftlicher Titel (Datenquelle: WHO)

FaR-RMS: An overarching study for children and adults with Frontline and Relapsed RhabdoMyoSarcoma

Studientyp (Datenquelle: WHO)

Interventional

Design der Studie (Datenquelle: WHO)

Randomized; Interventional; Design type: Treatment, Radiotherapy (Treatment)

Phase (Datenquelle: WHO)

Not Applicable

Primäre Endpunkte (Datenquelle: WHO)

1. Event-free survival (EFS) ? primary outcome measure for CT1A, CT1B, CT2A, CT2B, RT2, CT3. Time is measured as time from each registration/randomisation to first failure event (Relapse or progression of existing disease/occurrence of disease at new sites, death from any cause without disease progression, second malignant neoplasm) or time to last follow-up date if no events
2. Local failure free survival (LFFS) ? primary outcome measure for RT1A, RT1B, RT1C, PET Substudy. Defined as time from randomisation to first local failure event (relapse or progression of tumour at the primary site at any time even if there has been a prior concurrent local, regional or distant failure), or time to last follow-up date if no events.

Sekundäre Endpunkte (Datenquelle: WHO)

1. Event-free survival (EFS) ? secondary outcome measure for RT1A, RT1B, RT1C, All patients, PET Substudy. Time is measured as time from each registration/randomisation to first failure event (Relapse or progression of existing disease/ occurrence of disease at new sites, Death from any cause without disease progression, Second malignant neoplasm) or time to last follow-up date if no events.
2. Overall survival (OS) ? secondary outcome measure for CT1A, CT1B, CT2A, CT2B, RT1A, RT1B, RT1C, RT2, CT3, All patients. Time is measured as time from each registration/randomisation to death from any cause, or time to last follow-up date if no death.
3. Loco-regional failure-free survival (LRFFS) ? secondary outcome measure for RT1A, RT1B, RT1C, RT2. Defined as time from randomisation to first local or regional failure event. (relapse or progression of tumour at the primary site at any time even if there has been a prior concurrent local, regional or distant failure or relapse or progression of tumour at regional lymph nodes at any time even if there has been a prior distant failure) or time to last follow-up date if no events.
4. Toxicity ? secondary outcome measure for CT1A, CT1B, CT2A, CT2B, Phase 1b, CT3. Measured using Common Terminology Criteria for Adverse Events (CTCAE v 4). Toxicity is measured during cycles 1,2,3,4,5,6,7,8 and 9 of frontline chemotherapy and phase 1b, cycles 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24 of those randomised to receive additional cycles in CT2A, and cycles 7,8,9,10,11 and 12 of those randomised to receive additional cycles in CT2B.
5. Acute wound complications and post-operative complications ? secondary outcome measure for RT1A, RT1B. Measured using specific grade 3 and above complications according to CTCAE v 4 and Clavien Dindo scale from day of surgery to 120 days post surgery. This information is collected 120 days post surgery
6. Acute post-radiotherapy complications ? secondary outcome measure for RT1A, RT1B, RT1C, RT2. Measured using any grade 3 and above event according to CTCAE v 4 from completion of radiotherapy to 120 days post completion of radiotherapy. This information is collected 120 days post completion of radiotherapy.
7. Late local therapy complications ? secondary outcome measure for RT1A, RT1B, RT1C. Measured using specific grade 3 and above events according to CTCAE and Clavien-Dindo scale from 120 days from date of last local therapy until end of follow up (minimum three years). Follow up is as per local practise, but we are likely to collect this information at follow up year 1,2,3 and then as many years afterwards as they wish to supply.
8. Health-related quality of life (HRQoL) ? secondary outcome measure for RT1A, RT2. Assessed using PedsQL for the paediatric population (under 18 years) and EORTC QLQ-C30 for patients 18 years of age and over. This will be assessed for eligible patients at the following timepoints: at the start of radiotherapy, at completion of radiotherapy, 3 months following the end of radiotherapy, 24 months following radiotherapy.
9. Response (R) ? secondary outcome measure for Phase 1b, CT1A, CT1B, CT3. Defined as complete (CR) or partial response (PR) and is clinically defined. Patients who are not assessable for response ? e.g. because of early stopping of treatment or death ? will be assumed to be non-responders. Response will be assessed after course 2 and 6 for the newly diagnosed chemotherapy for very high risk and high risk randomisations; it will be assessed after course 2 and 4 for the relapse randomisation.
10. Duration of response ? secondary outcome measure for CT3. Defined as time from the date of first response (as defined above) to date of relapse, progression or death from any cause.
11. Best response (BR) ? secondary outcome measure for CT3. Assessed throughout the treatment for the relapse randomisation.
12. Recommended phase 2 dose (RP2D) ? secondary outcome measure for Phase 1b. Based on tolerability, where tolerability is evaluated through the occurrence of dose-limiting toxicity (DLT). DLTs will be defined in the relevant protocol section for each Phase Ib study. DLTs are collected up to 21 days after the start of cycle 2 for Phase 1b.
13. Maximum tolerated dose (MTD) ? secondary outcome measure for Phase 1b. Defined as the dose level at which no or one participant experiences a DLT when at least two of three to six participants experience a DLT at the next highest dose. DLTs are collected up to 21 days after the start of cycle 2 for Phase 1b.
14. PET response ? secondary outcome measure for Phase 1b. Assessed by PERCIST criteria after 3 cycles of chemotherapy.

Kontakt für Auskünfte (Datenquelle: WHO)

Cancer Research UK; Grant Codes: C18599/A22043

Ergebnisse der Studie (Datenquelle: WHO)

Zusammenfassung der Ergebnisse

noch keine Angaben verfügbar

Link zu den Ergebnissen im Primärregister

noch keine Angaben verfügbar

Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten

Other
1. Protocol will be available 2. Peer reviewed scientific journals 3. Internal report 4. Conference presentation 5. Publication on website 6. Submission to regulatory authorities

Studiendurchführungsorte

Durchführungsländer (Datenquelle: WHO)

Australia, Austria, Belgium, Canada, Czech Republic, England, Finland, France, Germany, Greece, Ireland, Israel, Italy, Netherlands, New Zealand, Northern Ireland, Norway, Portugal, Scotland, Slovakia, Slovenia, Spain, Switzerland, United Kingdom, Wales

Kontakt für weitere Auskünfte zur Studie

Kontakt für wissenschaftliche Auskünfte (Datenquelle: WHO)

Louise
Moeller
Senior Trial CoordinatorUniversity of BirminghamCRCTU, Vincent DriveEdgbaston
+44 (0)121 414 2996
farrms@trials.bham.ac.uk

Weitere Studienidentifikationsnummern

Secondary ID (Datenquelle: WHO)

Alternativer Eintrag: NCT04625907
2018-000515-24
42490
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