Condition category
Date applied
Date assigned
Last edited
Prospectively registered
Overall trial status
Recruitment status
No longer recruiting

Contact information



Primary contact

Miss Bridget Large


Contact details

School of Cancer Sciences
University of Birmingham
B15 2TT
United Kingdom

Additional identifiers

EudraCT number

2010-023135-42 number


Protocol/serial number


Study information

Scientific title

International randomized phase II trial of the combination of Vincristine and Irinotecan with or without Temozolomide (VI or VIT) in children and adults with refractory or relapsed rhabdomyosarcoma



Study hypothesis

This is an international open-label, multicenter, randomized phase II trial

Primary objective: To evaluate the efficacy of the combination of temozolomide with vincristine and irinotecan in children and adult patients with refractory or relapsed rhabdomyosarcoma as assessed by confirmed objective tumor response

Secondary objective: To evaluate the safety, tolerability and efficacy of VIT and VI alone as assessed by: duration of response, time to tumor progression, time to treatment failure, overall survival and adverse event profile.

More details can be found at

Ethics approval

South Central – Oxford A, South West REC Centre, 11/SC/0410; First MREC approval date 18/01/2012

Study design

Randomised; Interventional; Design type: Treatment

Primary study design


Secondary study design

Randomised controlled trial

Trial setting


Trial type


Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet


Topic: National Cancer Research Network; Subtopic: Sarcoma; Disease: Soft Tissue


Arm A - VI:
Day (D)1 and D8 Vincristine 1.5 mg/m2 (maximum 2mg) direct IV infusion
(0.05 mg/kg for patient ≤ 10 kg)
D1 to D5 Irinotecan 50 mg/m2/d, IV
1 cycle/ 21 days – maximum of 12 cycles

Arm B - VIT:
D1 to D5 Temozolomide 125 mg/m2/d, PO*
D1 and D8 Vincristine 1.5 mg/m2 (maximum 2mg) direct IV infusion
(0.05 mg/kg for patient ≤ 10 kg)
D1 to D5 Irinotecan 50 mg/m2/d, IV
1 cycle/ 21 days – maximum of 12 cycles
*The dose will be escalated to 150 mg/m2/day at cycle 2 for patients who do not experience > grade 3 toxicity of any kind

Follow Up Length: 60 month(s)

Intervention type



Phase II

Drug names

Vincristine, irinotecan and temozolomide

Primary outcome measures

Complete or partial tumour response; Timepoint(s): after the first 2 cycles of treatment which must be confirmed by a follow-up objective tumour assesment

Secondary outcome measures

1. Duration of response
2. time to tumour progression
3. Time to treatment failure
4. Overall survival and adverse event profile

Overall trial start date


Overall trial end date


Reason abandoned


Participant inclusion criteria

Tumor characteristics:
1. Histologically or cytologically confirmed diagnosis of rhabdomyosarcoma (new biopsy recommended)
2. Relapsed or refractory disease which has failed standard treatment approaches
3. Patients must have measurable disease defined as lesions that can be measured in three dimensions by medical imaging techniques such as computerised tomography (CT) or magnetic resonance imaging (MRI). Ascites, pleural fluid, bone marrow disease and lesions seen on Tc scintigraphy or positron emission tomography (PET) scan only are not considered measurable.

Patient characteristics:
1. Age > 6 months and < 50 years
2. Karnofsky performance status (PS) 70-100% (for patients > 12 years of age)
OR Lansky Play Score 70-100% (for patients = 12 years of age)
3. Life expectancy >= 12 weeks
4. Adequate bone marrow function :
4.1. Absolute neutrophil count >= 1000/mm3
4.2. Platelet count >= 100,000/mm3 (transfusion independent)
4.3. Hemoglobin >= 8.5 g/dL (transfusion allowed)
5. Adequate renal function
5.1. Serum creatinine < 1.5 X ULN for age
5.2. If serum creatinine > 1.5 ULN, creatinine clearance or radioisotope GFR) must be > 70 ml/min/1.73 m²
6. Adequate hepatic function :
6.1. Total bilirubin = 1.5 times upper limit of normal (ULN) for age, except if the patient is known to have Gilbert’s syndrome
6.2. ALT and AST < 2.5 X ULN for age
7. Negative pregnancy test in females with childbearing potential
8. Fertile patients must use effective contraception
9. No active > grade 2 diarrhea or uncontrolled infection
10. No other malignancy, including secondary malignancy
11. Patient affiliated with a health insurance system. Applicable for French patients only
12. Written informed consent of patient and/or parents/ guardians

Prior or concurrent therapy:
1. More than 3 weeks since prior radiation therapy to the site of any progressive lesion that will be identified as a target lesion to measure tumor response
2. At least 3 weeks since prior myelosuppressive therapy (6 weeks for nitrosourea, 2 weeks for vincristine, vinorelbine, vinblastine and lowdose cyclophosphamide)
3. No concurrent enzyme-inducing anticonvulsants (EIAC), including phenytoin, phenobarbital, or carbamazepine
4. No concurrent administration of any of the following : rifampicin, voriconazole, itraconazole, ketoconazole, aprepitant
5. No prior irinotecan or temozolomide administration
6. Prior administration of vincristine is allowed
7. Concurrent palliative radiation therapy to sites allowed except for the main measurable target lesion
8. Prior allo- or autologous SCT allowed; Upper Age Limit 50 years ; Lower Age Limit 6 months

Participant type


Age group




Target number of participants

Planned Sample Size: 80; UK Sample Size: 20

Participant exclusion criteria

1. Inclusion criteria failure
2. Concomitant anticancer treatment
3. Know hypersensitivity to any component of study drugs or ingredients
4. Pregnancy or breast feeding
5. Patients with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption
6. Neuromuscular disorders (e.g. Charcot-Marie Tooth disease)
7. Uncontrolled intercurrent illness or active infection
8. Unavailable for medical follow-up (geographic, social or mental reasons)

Recruitment start date


Recruitment end date



Countries of recruitment

France, Italy, Netherlands, Spain, United Kingdom

Trial participating centre

School of Cancer Sciences
B15 2TT
United Kingdom

Sponsor information


Centre Oscar Lambret (France)

Sponsor details

c/o : Anne-Sophie DEFACHELLES
3 rue Frédéric Combemale

Sponsor type




Funder type


Funder name

Cancer Research UK (CRUK) (UK)

Alternative name(s)


Funding Body Type

private sector organisation

Funding Body Subtype

other non-profit


United Kingdom

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes