Rare Tumours in Children and Adolescents (STEP) (STEP 2 0)

Rare Tumours in Children and Adolescents (STEP 2.0) - Register for the Documentation of Rare Tumours in Children and Adolescents

The aim of the STEP registry is to collect and evaluate experience and data on the diagnosis and treatment of rare childhood tumors in order to use the knowledge gained to improve the treatment prospects for our patients. The rarity of a disease should not be a disadvantage for the young patients.

Study Overview

• Status: Recruiting
• Conditions: Rare Diseases
• Intervention / Treatment: Other: Data collection

Detailed Description

The objective of the STEP registry is to optimise the diagnosis and treatment of patients with rare tumour diseases in childhood and adolescence. Therefore, a continuous prospective collection of clinical data on rare paediatric tumours is conducted to improve the understanding of these tumours. Beyond analysis of clinical data, further scientific research on the biological and molecular genetic characteristics of these tumours is performed. These data and a close collaboration with international partners, especially the European EXPeRT group, enable the improvement of treatment recommendations for these tumours along with establishment a global interdisciplinary network of rare tumour specialists.

• Study Type: Observational
• Enrollment (Estimated): 10000

Contacts and Locations

• Study Contact: Name: Ines Brecht, PD Dr. med.; Phone Number: 81380 +49 7071 29; Email: ines.brecht@med.uni-tuebingen.de
• Study Contact Backup: Name: Michael Abele, Dr. med.; Phone Number: 61837 +49 7071 29; Email: michael.abele@med.uni-tuebingen.de

Study Locations

• Germany
  • Tübingen, Germany, 72076
    • Recruiting
    • University Hospital Tübingen
    • Contact: Ines Brecht, PD Dr. med.; Phone Number: 81380 +49 7071 29; Email: ines.brecht@med.uni-tuebingen.de
    • Contact: Michael Abele, Dr. med.; Phone Number: 61387 +49 7071 29; Email: michael.abele@med.uni-tuebingen.de

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 day to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Children and adolescents with rare solid tumors from primary care clinic, university hospitals, specialty centers, ...

Description

Inclusion Criteria:

• Diagnosis of a rare solid tumor
• Age at diagnosis: Neonatal period to 18 years (In the case of young adults, registration in the database and/or referral to advisory contact persons within the framework of the competence network can take place upon request and after declaration of consent.)
• Information, education, written consent of the patient or the guardian
• Not recorded in any of the existing clinical studies/ registers of the German Society for Pediatric Oncology and Hematology (GPOH)

Exclusion Criteria:

• Registration of the tumor diagnosis in a prospective therapy study/ another clinical registry of the GPOH
• Lack of information, explanation and/or written consent of the patient or the legal guardian.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Rare tumor disease; Prospective epidemiological and clinical data collection of subjects with diagnosis of a rare solid tumor.	Other: Data collection; The data collection includes, among other things: Diagnosis of the rare tumor (pathological findings/ reference pathological findings), full name, birth date, gender, clinical registry inclusion and exclusion criteria met - yes / no, signed declaration of consent-yes / no, if yes: date of signature

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Event-free survival	Period between study entry and failure of induction therapy, recurrence or death from any cause is measured.	5 years

Collaborators and Investigators

• Sponsor: University Hospital Tuebingen
• Investigators: Study Director: Ines Brecht, PD Dr. med., University Hospital Tübingen; Study Director: Dominik Schneider, Prof. Dr. med., Clinic for Pediatric and Adolescent Medicine at Dortmund Hospital

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): January 11, 2023
• Primary Completion (Estimated): January 1, 2050
• Study Completion (Estimated): January 1, 2055

Study Registration Dates

• First Submitted: March 6, 2023
• First Submitted That Met QC Criteria: March 6, 2023
• First Posted (Actual): March 17, 2023

Study Record Updates

• Last Update Posted (Actual): October 1, 2024
• Last Update Submitted That Met QC Criteria: September 27, 2024
• Last Verified: September 1, 2024

More Information

Terms related to this study

• Keywords: Epidemiological data collection; Rare tumors in children; Rare tumors in adolescents; Clinical data collection
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Rare Diseases
• Other Study ID Numbers: STEP 2.0

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The STEP Register will provide data in a pseudonymised manner to national and international databases set up to optimize the diagnosis and treatment of rare tumors in children and adolescents
• IPD Sharing Time Frame: Data will become available after analysis and unlimited.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No