Status:

RECRUITING

Hematological Anomalies in Children With Rasopathy

Lead Sponsor:

Assistance Publique - Hôpitaux de Paris

Conditions:

RAS Mutation

Eligibility:

All Genders

Up to 15 years

Brief Summary

During childhood, patients with RASopathies (Noonan syndrome and related diseases) can harbor various hematological anomalies ranging from isolated monocytosis, myelemia, thrombocytopenia or splenomeg...

Eligibility Criteria

Inclusion

  • Age \< 16 years
  • Patient newly diagnosed with genetically confirmed rasopathy : Noonan syndrome, type 1 neurofibromatosis, Noonan syndrome with multiple lentigines, CBL syndrome, Costello syndrome, cardiofaciocutaneous syndrome or Legius syndrome i.e. with a germline mutation of one of these genes: PTPN11, SOS1, NRAS, RAF1, BRAF, SHOC2, MEK1, MEK2, CBL, NF1, SPRED1, KRAS, HRAS, NF1, SHOC2, LZTR1, SOS2, RIT1, RASA2, RRAS, PPP1CB, or a new gene of interest published during the recruitment period
  • No history of hematological malignancy
  • Written informed consent obtained from the parents
  • Health insurance

Exclusion

  • History of malignant hematological pathology

Key Trial Info

Start Date :

November 11 2020

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

November 1 2029

Estimated Enrollment :

300 Patients enrolled

Trial Details

Trial ID

NCT04286360

Start Date

November 11 2020

End Date

November 1 2029

Last Update

June 4 2024

Active Locations (14)

Enter a location and click search to find clinical trials sorted by distance.

Page 1 of 4 (14 locations)

1

CHU Angers

Angers, France

2

CHU Caen

Caen, France

3

CHU Lille

Lille, France

4

CHU Lyon

Lyon, France