Rare DiseasesSTEP 2 0NCT05773651
Rare Tumours in Children and Adolescents (STEP)
An observational study of Rare Diseases, run by University Hospital Tuebingen at 1 site in Germany.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 day to 18 years
- Conditions the study is for
- Rare Diseases
- What is being tested?
- Data collection
- How many people
- 10,000 participants
- Where
- 1 siteGermany
- Study dates
- 11 Jan 2023to Jan 2055
- How long will I take part?
- 50 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children and adolescents with rare solid tumors from primary care clinic, university hospitals, specialty centers, ...
Who may be able to join
- 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)
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Germany1 site, 1 recruiting
- Recruiting
University Hospital Tübingen
Tübingen, 72076
Ines Brecht, PD Dr. med., +49 7071 29 ext. 81380, ines.brecht@med.uni-tuebingen.de
Michael Abele, Dr. med., +49 7071 29 ext. 61387, michael.abele@med.uni-tuebingen.de
About this study
Official title: 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.
Read the 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.
- Conditions
- Rare Diseases
- Being tested
- Data collection
- Keywords
- Epidemiological data collection, Rare tumors in children, Rare tumors in adolescents, Clinical data collection
- Medical subject headings
- Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Data collectionOther
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
| Study group | Role | What they receive |
|---|---|---|
| Rare tumor disease | Prospective epidemiological and clinical data collection of subjects with diagnosis of a rare solid tumor. Receives: Other: Data collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival
Period between study entry and failure of induction therapy, recurrence or death from any cause is measured.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2023
- Study start11 Jan 2023
- Primary completionJan 2050 (estimated)
- Study completionJan 2055 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rare DiseasesStudies of the same condition, open and under way.
- More trials run by University Hospital TuebingenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 10,000 participants (estimated)
- Target duration
- 50 Years
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ines Brecht, PD Dr. med., Study director, University Hospital TuebingenDominik Schneider, Prof. Dr. med., Study director, Clinic for Pediatric and Adolescent Medicine at Dortmund Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05773651
- Sponsor's protocol ID
- STEP 2.0
- Acronym
- STEP 2 0
- Status last verified
- Sep 2024
- First submitted
- 6 Mar 2023
- Last updated
- 1 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rare Tumours in Children and Adolescents (STEP 2.0) - Register for the Documentation of Rare Tumours in Children and Adolescents
- ClinicalTrials.gov ID
- NCT05773651
- Acronym / sponsor ID
- STEP 2 0 / STEP 2.0
- Conditions
- Rare Diseases
- Keywords
- Epidemiological data collection, Rare tumors in children, Rare tumors in adolescents, Clinical data collection
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10,000 participants (estimated)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Study start
- 11 Jan 2023 (actual)
- Primary completion
- Jan 2050 (estimated)
- Study completion
- Jan 2055 (estimated)
- First posted
- 17 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 50 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rare tumor disease | – | Other: Data collectionProspective epidemiological and clinical data collection of subjects with diagnosis of a rare solid tumor. |
Who may qualify
- Ages
- 1 day to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children and adolescents with rare solid tumors from primary care clinic, university hospitals, specialty centers, ...
Key inclusion criteria (4 listed)
- 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)
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Data collectionThe 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 | Other | Rare tumor disease |
Primary outcome
Event-free survival
Time frame: 5 years
Period between study entry and failure of induction therapy, recurrence or death from any cause is measured.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2024.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ines Brecht, PD Dr. med. study director
University Hospital Tuebingen
Dominik Schneider, Prof. Dr. med. study director
Clinic for Pediatric and Adolescent Medicine at Dortmund Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Rare Diseases, run by University Hospital Tuebingen at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ines Brecht, PD Dr. med. (+49 7071 29 ext. 81380, ines.brecht@med.uni-tuebingen.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-free survival (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2024.