Analyzing blood abnormalities in children with RAS mutations

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study focuses on children diagnosed with RASopathies, such as Noonan syndrome, to investigate various hematological anomalies that may arise. It aims to analyze peripheral blood cell counts and smears at diagnosis and one year later, tracking the incidence, age of occurrence, and evolution of these abnormalities. The study includes biobanking for patients under three years of age to facilitate further research. The findings will help establish guidelines for screening and follow-up care in affected children.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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 Criteria:

* History of malignant hematological pathology

Where it's running

• CHU Angers
  Angers, France Recruiting
• CHU Caen
  Caen, France Recruiting
• CHU Lille
  Lille, France Recruiting
• CHU Lyon
  Lyon, France Recruiting
• CHU Marseille - Hôpital de la Timone
  Marseille, France Recruiting
• CHU Montpellier
  Montpellier, France Recruiting

Study contacts

• Study coordinator: Marion STRULLU, MD
• Email: marion.strullu@aphp.fr
• Phone: 187891611

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• BG-85738 alone or with other anticancer drugs in RAS-mutant advanced or metastatic solid tumors

  This study tests BG-85738, alone or with other cancer drugs, in adults whose solid tumors have RAS mutations and have progressed.

  Recruiting at 3 sites Phase 1 BeOne Medicines · South Brisbane, Queensland and 2 other locations · NCT07746141

• AN4035 in advanced cancers with RAS mutations and high CEACAM5 expression

  This first-in-human trial tests AN4035 to see if it is safe, tolerable, and possibly active in people with RAS-mutated, CEACAM5-positive advanced solid tumors, including when given with cetuximab.

  Recruiting at 2 sites Phase 1 Adlai Nortye Biopharma Co., Ltd. · Perth and 1 other locations · NCT07686445

• GFH276 with Cetuximab or chemotherapy in RAS-mutated solid tumors and PDAC

  This study tests whether GFH276 combined with cetuximab or standard chemotherapy can help adults with advanced solid tumors or pancreatic cancer that carry a RAS mutation.

  Recruiting at 10 sites Phase 1/2 Genfleet Therapeutics (Shanghai) Inc. · Concord, New South Wales and 16 other locations · NCT07678593

• Oral AN9025 for advanced solid tumors with RAS mutations

  This trial tests whether the oral drug AN9025 is safe and tolerable for adults with advanced solid tumors that have RAS mutations.

  Recruiting at 2 sites Phase 1 Adlai Nortye Biopharma Co., Ltd. · Orlando, Florida and 2 other locations · NCT07252479

• TNG462 combined with RAS inhibitors or chemotherapy for MTAP‑loss pancreatic and RAS‑mutant lung cancer

  This trial will test whether taking oral TNG462 together with RAS inhibitors or standard chemotherapy is safe and helps people with MTAP‑loss pancreatic cancer or RAS‑mutant non‑small cell lung cancer.

  Recruiting at 18 sites Phase 1/2 Tango Therapeutics, Inc. · Scottsdale, Arizona and 17 other locations · NCT06922591

• Combination treatment for metastatic colorectal cancer

  This study is testing a combination of three drugs to see if it can help patients with certain types of metastatic colorectal cancer who have previously responded well to similar treatments.

  Recruiting Phase 2 Beijing Hospital · Beijing, Beijing Municipality · NCT06321081

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.