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)
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1
CHU Angers
Angers, France
2
CHU Caen
Caen, France
3
CHU Lille
Lille, France
4
CHU Lyon
Lyon, France