Nephroblastoma clinical trial and study

ISRCTN	ISRCTN16583459
DOI	https://doi.org/10.1186/ISRCTN16583459
Secondary identifying numbers	NTR58

Submission date: 04/08/2005
Registration date: 04/08/2005
Last edited: 29/10/2021

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Cancer

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr J de Kraker
Scientific

SIOP Nephroblastoma Trial and Study Office
Meibergdreef 9, room A3-273
Amsterdam
1105 AZ
Netherlands

Phone	+31 (0)20 5665697
Email	siop-wilms@amc.uva.nl

Study information

Study design	Randomised, double-blind, active controlled, parallel group trial
Primary study design	Interventional
Secondary study design	Randomised controlled trial
Study setting(s)	Not specified
Study type	Treatment
Scientific title	Nephroblastoma clinical trial and study
Study acronym	SIOP 2001
Study objectives	1. To continue a risk-adapted stratification of therapeutic intensity, incorporating response to pre-operative chemotherapy, in all children with Wilms tumour and other renal tumours of childhood 2. To test the treatment hypothesis that doxorubicin is not necessary in patients with intermediate risk tumours and local stage II or III by a multicentre prospective randomised trial 3. To determine prospectively the prognostic significance of specific histological subtypes following pre-operative chemotherapy, as specified in the protocol. In particular, the study aims to: confirm the adverse prognostic significance of the blastemal predominant subtypes and whether this can be offset by intensifying therapy and investigate the hypothesis that the epithelial and stromal-predominant subtypes have a favourable prognosis and investigate the prognostic significance of the percentage necrosis after pre-operative chemotherapy in relation to the type and amount of residual viable tumour. 4. To minimise acute and late toxicity without jeopardising event free and overall survival by reducing treatment for: patients with focal anaplasia, and patients with stage I, intermediate risk tumours 5. To determine prospectively the prognostic significance of tumour volume following pre-operative chemotherapy and its relation to histological subtype 6. To determine prospectively the prognostic significance of specimen weight at time of nephrectomy and its relation to histological subtype 7. To reduce the number of drug administrations, hospital visits and thereby costs in the preoperative phase
Ethics approval(s)	Ethics approval received from local ethics committee.
Health condition(s) or problem(s) studied	Nephroblastoma
Intervention	Establishing equivalence between two post-operative treatments. Trial arm with doxorubicin versus trial arm without doxorubicin.
Intervention type	Drug
Pharmaceutical study type(s)
Phase	Not Specified
Drug / device / biological / vaccine name(s)	Doxorubicin
Primary outcome measure	Event free survival (EFS).
Secondary outcome measures	No secondary outcome measures
Overall study start date	01/06/2002
Completion date	01/06/2009

Eligibility

Participant type(s)	Patient
Age group	Child
Lower age limit	6 Months
Upper age limit	18 Years
Sex	Both
Target number of participants	350
Total final enrolment	583
Key inclusion criteria	1. All localised disease nephroblastoma patients age more than 6 months or less than 18 years at time of diagnosis 2. Unilateral tumour with clinical and ultrasonic characteristics compatible with nephroblastoma or biopsy proven histological diagnosis 3. Written informed consent and national ethical committee approval 4. Stage II and III intermediate risk histology after pre-treatment according to protocol and after operation
Key exclusion criteria	1. All other kind of renal tumours of infancy 2. Patients without previous anti-tumour treatment
Date of first enrolment	01/06/2002
Date of final enrolment	01/06/2009

Locations

Countries of recruitment

Netherlands

Study participating centre

SIOP Nephroblastoma Trial and Study Office

Amsterdam
1105 AZ
Netherlands

Sponsor information

Academic Medical Centre (AMC) (The Netherlands)
University/education

Emma Kinderziekenhuis
Postbus 22660
Amsterdam
1105 AZ
Netherlands

Website	http://www.amc.uva.nl/
"ROR"	https://ror.org/03t4gr691

Funders

Funder type

Research organisation

The Foundation of Paediatric Cancer Research (Stichting Kindergeneeskundig Kankeronderzoek [SKK]) (The Netherlands)

No information available

German Cancer Aid (Deutsche Krebshilfe) (Germany)

No information available

The Swedish Childhood Cancer Foundation (Barncancerfonden) (Sweden)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan	Not provided at time of registration

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Plain English results		21/07/2015	29/10/2021	No	Yes

Editorial Notes

29/10/2021: The following changes have been made:
1. The Cancer Research UK lay results summary has been added.
2. The total final enrolment number has been added.