Rare Diseases

All Conditions

Crouzon Syndrome

Jackson-Weiss Syndrome

Pfeiffer Syndrome

* Data collection from the medical file
* Quality of life questionnaire (QLQC30) at inclusion, at each change of treatment and then every 6 months

Data collection and quality of life questionnaire

Julien Bollard

Alexandra BIETTE

Andreas LEITNER

Thomas BRODOWICZ

Jana Halámková

Ninna Aggerholm

Loic CHAIGNEAU

Antoine ITALIANO

Alice HERVIEU

Nicolas PENEL

Valérie LE BRUN LY

Alexandra Biette

Jean-Yves BLAY, Pr

Marie Eve GARCIA

Esma SAADA-BOUZID

Sarah WATSON

Jean-Philippe SPANO

Pascaline BOUDOU-ROUQUETTE

Jean-Pierre LOTZ

Cécile GUILLEMET

Thibaud VALENTIN

Manfred WELSLAU

Jalid SEHOULI

Mathias HANEL

Jorg TROJAN

Haifa Kathrin Al-Ali

Marino Venerito

Bernd KASPER

Volker HEINEMANN

Hans-Heinrich WOLF

Constantin Roder

Volker KUNZMANN

Maria Abbondenza PANTALEO

Toni IBRAHIM

Marica EOLI

Alice BERGAMINI

Mario GIULIANO

Riccardo SOFFIETTI

Ellen KAPITEIJN

Iwona LUGOWSKA

Mojca UNK

Javier MARTIN-BROTO

Robin JONES

John BRIDGEWATER

This registry will make it possible to describe real life management of patients with rare actionable fusions and to better understand these cancers. In addition of clinical data from the medical files, a quality of life questionnaire (QLQ-C30) will be complete at inclusion, at each new treatment and then every 6 months. The patients will be followed for a period of at least 2 years after the inclusion.

This TRacKING registry is a European collaborative tool to improve the management of patients with actionable fusions, by sharing of data from rare tumor indications.

1. Administrative opening of the center
2. Routine identification of a patients harboring a rare actionable fusion
3. Patient's inclusion

   * Signature of written informed consent,
   * Declaration by the physician to the coordinating center (using the "Physician declaration" form)
   * Completion of the QLQ-C30 questionnaire (at inclusion, at each new treatment and then every 6 months).
4. Collection of retrospective and prospective data into the e-CRF by a local CRA using patients' medical files.

Real Word European Registry of NTRK Fusions and Other Rare Actionable Fusions (TRacKING)

To describe the survival rate of patients with solid cancers harboring a NTRK fusion or other rare actionable fusion in real-life practice, according to overall survival (OS)

Treatment scheme : number of line and type of treatments

To evaluate patient-reported outcomes concerning individual QoL according to QLQ-C30 (EORTC Quality of Life questionnaires)

using QLQC30 (EORTC Quality of Life questionnaires)

Using Common Terminology Criteria for Adverse Events (CTCAE) V5.0.

Centre Leon Berard

Amino Acids

All Drugs and Chemicals

Tyrosine

Informed consent (IC) before start of any treatment after diagnosis of NTRK fusion-positive cancer. All data after diagnosis of NTRK fusion-positive cancer are collected prospectively.

IC after start of any treatment after diagnosis of NTRK fusion-positive cancer. Data after study inclusion are collected prospectively and retrospectively.

Patients deceased prior to study inclusion (no IC required). All data are collected retrospectively.

Benjamin Kasenda, PD Dr. Dr.

The objective of this registry is to analyze treatment reality and outcome of patients with locally advanced or metastatic solid tumors harboring a fusion of NTRK1, NTRK2 or NTRK3

The REALTRK registry will provide data on treatment reality of patients with locally advanced or metastatic solid tumors harboring NTRK1, NTRK2 or NTRK3 gene fusions, thereby generating real world evidence. It will identify factors that influence treatment decisions after receiving the diagnosis of a NTRK (Neurotrophic Tyrosine Receptor Kinase) fusion-positive cancer. All treatment lines administered before and after the molecular diagnosis of a NTRK fusion-positive cancer, irrespective of the type of antineoplastic treatment, will be documented. Data will be assessed at least 36 months per patient (i.e. until 36 months after inclusion of the last patient in the study). This approach will allow a description of TRK fusion protein-targeted therapies and other therapy strategies regarding effectiveness and disease-related symptomology within the limitations of non-randomized studies in terms of comparative analyses. Intra-individual and inter-individual comparisons (for the latter, provided that a sufficient number of patients with a NTRK fusion-positive cancer are not treated with a TRK inhibitor) could be performed.

The associated biomarker profiling module of the REALTRK registry will aim to set up a decentralized biobank for future research on molecular alterations.

Registry for Molecular Testing, Treatment and Outcome of Patients With Solid Tumors Harboring a NTRK Gene Fusion

Registry for Molecular Testing, Treatment and Outcome of Patients With Locally Advanced or Metastatic Solid Tumors Harboring a Fusion of NTRK1, NTRK2 or NTRK3

Proportion of patients with CR or PR as best response

Descriptive summary of demographics, patient and disease characteristics

Description of test methods used for diagnosis of a NTRK fusion-positive cancer and results thereof

Description of physician-reported factors affecting decision to test for NTRK fusion and treatment decision making after diagnosis of NTRK gene fusion

Description of all treatment lines given to the patient after diagnosis of NTRK gene fusion including: Type of treatment (Systemic TRK inhibitor treatments or Non-TRK inhibitor treatments), treatment duration, dosing, treatment modifications and reasons thereof, reasons for end of treatment.

Treatment-emergent AEs (i.e., AEs which occurred during a specific TRK inhibitor treatment and in the respective survival FU) will be calculated per patient (absolute and relative frequencies) and case-based (absolute frequencies). The occurrence of any (S)AE will be displayed overall and per CTCAE grade. Adverse drug reactions (ADRs) and AESIs will be displayed accordingly.

Incidence of AEs (MedDRA Preferred Term (PT) by System Organ Class (SOC)) will be calculated accordingly for each type of AE/ADR.

Description of physician-reported evaluation of TRK inhibitor therapy

Description of courses of disease-related symptoms (weight loss, ECOG) after diagnosis of NTRK gene fusion (Only for patients of inclusion group I)

Proportion of patients with CR, PR or SD as best response

Time from start of treatment to the first objective tumor response (e.g., tumor shrinkage of ≥30%) observed for patients who achieved a CR or PR

Time from documentation of tumor response to disease progression or death from any cause

Time from start of treatment until disease progression or death

Time from start of treatment until death of any cause

ratio of PFS of the first treatment line with a TRK inhibitor to time to progression (TTP) in the preceding treatment line without a TRK inhibitor

Time from start of treatment until PD or death

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Real Word European Registry of NTRK Fusions and Other Rare Actionable Fusions (TRacKING)	NCT04921553	Cancer Cancer Metastat... NTRK Gene Fusio... NTRK Family Gen... ATIC-ALK Fusion... ALK Fusion Prot... BCR-FGFR1 Fusio... ROS1 Gene Trans... COL1A1-PDGFB Fu... RET Gene Transl... Gene Fusion ROS Gene Transl... BRAF Gene Rearr... FGFR2 Gene Tran... FGFR3 Gene Tran... NTRK1 Gene Tran...	Data collection...	18 Years -	Centre Leon Berard
Real Word European Registry of NTRK Fusions and Other Rare Actionable Fusions (TRacKING)	NCT04921553	Cancer Cancer Metastat... NTRK Gene Fusio... NTRK Family Gen... ATIC-ALK Fusion... ALK Fusion Prot... BCR-FGFR1 Fusio... ROS1 Gene Trans... COL1A1-PDGFB Fu... RET Gene Transl... Gene Fusion ROS Gene Transl... BRAF Gene Rearr... FGFR2 Gene Tran... FGFR3 Gene Tran... NTRK1 Gene Tran...	Data collection...	18 Years -	Centre Leon Berard
Registry for Molecular Testing, Treatment and Outcome of Patients With Solid Tumors Harboring a NTRK Gene Fusion	NCT04557813	NTRK Family Gen...		18 Years -	iOMEDICO AG
Registry for Molecular Testing, Treatment and Outcome of Patients With Solid Tumors Harboring a NTRK Gene Fusion	NCT04557813	NTRK Family Gen...		18 Years -	iOMEDICO AG

Spots Global Cancer Trial Database for ntrk family gene mutation