Clinical trials: Noonan Syndrome
Child Syndrome
Study of Metabolic Modifications in Children With Noonan Syndrome
Noonan syndrome (NS) is a rare genetic disease (incidence 1/2500 live births) characterized by the association of craniofacial manifestations, cardiopathies, short stature, and tumor predisposition.
- Trial name
- MetabNoonan (NCT02383316)
- Testing
- Oral Glucose tolerance test
- Where
- France
- Volunteers
- 20 people
- Sponsor
- University Hospital, Toulouse
Genetic Disorder, Noonan Syndrome
Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome
This trial is conducted in Asia. The aim of the trial is to investigate the long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome.
- Trial name
- NCT01927861
- Testing
- somatropin
- Where
- 27 sites in Japan
- Volunteers
- 51 people
- Sponsor
- Novo Nordisk A/S
Noonan Syndrome
French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
This is a non-interventional registry of children treated with Norditropin® for short stature due to Noonan Syndrome (NS). This study aims to provide data on long-term growth evolution and safety of Norditropin® as well as Health…
- Trial name
- NCT05308927
- Testing
- Norditropin
- Where
- 3 sites in France
- Volunteers
- 221 people
- Sponsor
- Novo Nordisk A/S
Danon Disease, Fabry Disease
Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
What is this study about? This research is focused on improving the care for people with rare heart muscle diseases, known as rare cardiomyopathies.
- Conditions
- Danon Disease, Fabry Disease, Cardiac Amyloidosis, Noonan Syndrome
- Trial name
- NCT07336394
- Where
- China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Chinese Academy of Medical Sciences, Fuwai Hospital
Noonan Syndrome, LEOPARD Syndrome
Hormonal Sensitivity in Patients With Noonan and LEOPARD Syndromes
Noonan and LEOPARD syndromes share, with variable severity, different clinical traits, notably craniofacial manifestations, cardiopathies, short stature, and juvenile cancers.
- Trial name
- NOLEO (NCT02486731)
- Where
- France
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Noonan Syndrome
Effect of MAXOMAT ® on the Growth of Small Children to NOONAN's Syndrome
1. Clinical Objective : To improve the growth of these children 2. Genetic objective : A study of the genetics of the syndrome
- Trial name
- NCT00452725
- Testing
- MAXOMAT ®, biosynthetic growth hormone
- Where
- France
- Volunteers
- 36 people
- Sponsor
- Sanofi
RASopathy, Costello Syndrome
Solid Tumors in RASopathies
RASopathies are a group of syndromes, caused by variants of genes involved in the regulation of the Ras/MAP/ERK pathway. This intracellular transduction pathway profoundly affects embryogenic development, organogenesis, synaptic…
- Conditions
- RASopathy, Costello Syndrome, Cardio-Facio-Cutaneous Syndrome, Noonan Syndrome
- Trial name
- 4218 (NCT05761314)
- Testing
- Molecular characterization of solid tumor in RASopathies
- Where
- Italy
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Observational Prospective Study on Patients Treated With Norditropin®
This observational study is conducted globally. The aim of the study is to investigate the effectiveness and safety of real-life treatment with Norditropin®.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Adult Growth Hormone Deficiency, Genetic Disorder
- Trial name
- NCT00960128
- Testing
- somatropin
- Where
- 23 sites in Argentina, Belgium and 21 more
- Volunteers
- 21,249 people
- Sponsor
- Novo Nordisk A/S
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 56 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
Noonan Syndrome
Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan Syndrome
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.
- Trial name
- RASTAT (NCT02713945)
- Testing
- Simvastatin
- Where
- 12 sites in France
- Volunteers
- 53 people
- Sponsor
- University Hospital, Toulouse
Cardiomegaly, Noonan Syndrome
MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
The goal of this study is to evaluate the effectiveness of trametinib treatment in patients with Hyperthropic cardiomyopathy and a genetic mutation in the RAS/MAPK pathway.
- Trial name
- MEKinRAS (NCT06555237)
- Testing
- Trametinib tablet, Standard therapy
- Where
- Poland
- Volunteers
- 40 people
- Sponsor
- Medical University of Warsaw
Genetic Disorder, Noonan Syndrome
Genetic Testing of Noonan Subjects Previously Treated With Norditropin®. An Extension to Trial GHNOO-1658
This trial is conducted in Europe. The aim of this trial is to obtain the PTPN11 mutation status and investigate the impact of the PTPN11 mutation status on the effect of somatropin (Norditropin®) by use of data obtained in the…
- Trial name
- NCT01529944
- Testing
- somatropin
- Where
- Sweden
- Volunteers
- 22 people
- Sponsor
- Novo Nordisk A/S
Noonan Syndrome
Registro Nazionale Sindrome Noonan / National Registry of Noonan Syndrome
This study will create a national registry (a secure online database) of people with Noonan syndrome in Italy. Noonan syndrome is a genetic condition. It can affect growth, the heart, and other parts of the body.
- Trial name
- MOMAGPNONA01 (NCT07881159)
- Where
- 35 sites in Italy
- Volunteers
- 400 people
- Sponsor
- Istituto Giannina Gaslini