NCT02713945: RASTAT

Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan Syndrome

Completed Phase 3 Last updated 11 May 2023

What this trial tests

Phase 3 trial testing Simvastatin in Noonan Syndrome in 53 participants. Completed in 3 March 2023.

Timeline

25 January 2017

Primary endpoint
3 March 2023

3 March 2023

Quick facts

Lead sponsor	University Hospital, Toulouse
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	53
Start date	25 January 2017
Primary completion	3 March 2023
Estimated completion	3 March 2023
Sites	12 locations across France

Drugs / interventions tested

• Simvastatin (simvastatin) — full drug profile →
• Placebo

Conditions studied

• Noonan Syndrome — all drugs for Noonan Syndrome →

Sponsor

University Hospital, Toulouse

Who can join

Adults 6 to 16, any sex, with Noonan Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study evaluate the efficacy of 3-hydroxy-3-methylglutaryl coenzyme A (HMG-COA) reductase inhibitors, also known as "statins" in the treatment of growth and skeletal abnormalities in children with Noonan syndrome. Half of patients will receive simvastatin while the other half will receive a placebo.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. The RASopathies: from pathogenetics to therapeutics.
   Hebron KE, Hernandez ER, Yohe ME. · · 2022 · cited 92× · PMID 35178568 · DOI 10.1242/dmm.049107
2. The molecular mechanisms of cardiac development and related diseases.
   Li Y, Du J, Deng S, Liu B, et al · · 2024 · cited 53× · PMID 39715759 · DOI 10.1038/s41392-024-02069-8
3. Novel therapeutic perspectives in Noonan syndrome and RASopathies.
   Saint-Laurent C, Mazeyrie L, Yart A, Edouard T. · · 2024 · cited 25× · PMID 37863846 · DOI 10.1007/s00431-023-05263-y
4. New prospectives on treatment opportunities in RASopathies.
   Gelb BD, Yohe ME, Wolf C, Andelfinger G. · · 2022 · cited 23× · PMID 36533679 · DOI 10.1002/ajmg.c.32024
5. Drosophila RASopathy models identify disease subtype differences and biomarkers of drug efficacy.
   Das TK, Gatto J, Mirmira R, Hourizadeh E, et al · · 2021 · cited 19× · PMID 33855281 · DOI 10.1016/j.isci.2021.102306
6. Modeling (not so) rare developmental disorders associated with mutations in the protein-tyrosine phosphatase SHP2.
   Solman M, Woutersen DTJ, den Hertog J. · · 2022 · cited 8× · PMID 36407105 · DOI 10.3389/fcell.2022.1046415
7. Lipid metabolism in homeostasis and disease.
   Li Z, Deng W, Yang L, Tang C, et al · · 2026 · cited 2× · PMID 41692800 · DOI 10.1038/s41392-025-02357-x
8. Exploring New Drug Repurposing Opportunities for MEK Inhibitors in RASopathies: A Comprehensive Review of Safety, Efficacy, and Future Perspectives of Trametinib and Selumetinib
   Gazzin A, Fornari F, Cardaropoli S, Carli D, et al · · 2024