Clinical trials: Noonan Syndrome
Solid Tumor
Dose-Esc/Exp RMC4630 & Cobi in Relapsed/Refractory Solid Tumors & RMC4630& Osi in EGFR+ Locally Adv/Meta NSCLC
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of RMC-4630 and cobimetinib in adult participants with relapsed/refractory solid tumors with specific…
- Trial name
- NCT03989115
- Testing
- RMC-4630, Cobimetinib, Drug: Osimertinib
- Where
- 24 sites in United States, South Korea
- Volunteers
- 113 people
- Sponsor
- Revolution Medicines, Inc.
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
Non-Small Cell Lung Cancer
BR790 in Combination With Anlotinib in Adult Subjects With Advanced Non-Small Cell Lung Cancer
This is a Phase Ⅰ/Ⅱa, multi-center, open-label study, aiming to evaluate the safety, tolerability, pharmacokinetic (PK), and efficacy of BR790 in combination with anlotinib in adult participants with advanced NSCLC.
- Trial name
- NCT05715398
- Testing
- BR790+anlotinib
- Volunteers
- 90 people
- Sponsor
- Shanghai Gopherwood Biotech Co., Ltd.
Solid Tumor, Unspecified, Adult
RLY-1971 in Subjects With Advanced or Metastatic Solid Tumors
This study is a multi-center, open-label, dose escalation and expansion study of RLY-1971 in subjects with advanced or metastatic solid tumors.
- Trial name
- NCT04252339
- Testing
- RLY-1971
- Where
- 6 sites in United States
- Volunteers
- 56 people
- Sponsor
- Hoffmann-La Roche
Genetic Disorder, Noonan Syndrome
Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as…
- Trial name
- NCT03435627
- Testing
- Somatropin
- Where
- 22 sites in Japan
- Volunteers
- 71 people
- Sponsor
- Novo Nordisk A/S
Solid Tumors
Dose Escalation of RMC-4630 Monotherapy in Relapsed/Refractory Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of escalating doses of RMC-4630 monotherapy in adult participants with relapsed/refractory solid tumors…
- Trial name
- NCT03634982
- Testing
- RMC-4630
- Where
- 12 sites in United States
- Volunteers
- 133 people
- Sponsor
- Revolution Medicines, Inc.
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
Solid Tumor and Hematologic Malignancies
MEK162 for Patients With RAS/RAF/MEK Activated Tumors
The purpose of this signal seeking study is to determine whether treatment with MEK162 demonstrates sufficient efficacy in select pathway-activated solid tumors and/or hematologic malignancies to warrant further study
- Trial name
- SIGNATURE (NCT01885195)
- Testing
- MEK162
- Where
- 68 sites in United States
- Volunteers
- 110 people
- Sponsor
- Pfizer
Cardiomegaly
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of MEK162 in Noonan Syndrome Hypertrophic Cardiomyopathy
The purpose of the study is to determine whether the ability of MEK162 to antagonize MEK activation in NS HCM patients, who usually have upstream mutations in the Ras-Raf-Mek-Erk pathway that lead to MEK activation, would be…
- Trial name
- NCT01556568
- Testing
- MEK162
- Where
- 2 sites in United States, United Kingdom
- Sponsor
- Array Biopharma, now a wholly owned subsidiary of Pfizer
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
ALK Fusion Protein Expression, BRAF wt Allele
Capmatinib, Ceritinib, Regorafenib, or Entrectinib in Treating Patients With BRAF/NRAS Wild-Type Stage III-IV Melanoma
This phase II trial studies how well capmatinib, ceritinib, regorafenib, or entrectinib work in treating patients with BRAF/NRAS wild-type stage III-IV melanoma.
- Conditions
- ALK Fusion Protein Expression, BRAF wt Allele, Invasive Skin Melanoma, MET Fusion Gene Positive
- Trial name
- NCT02587650
- Testing
- Capmatinib, Ceritinib, Entrectinib
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of California, San Francisco
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
Genetic Disorder, Noonan Syndrome
Somatropin Effect on Linear Growth and Final Height in Subjects With Noonan Syndrome
This trial is conducted in Europe. The aim of this trial is to evaluate the effect of somatropin (Norditropin®) on final height in children with Noonan syndrome having being treated for up to 10 years with somatropin…
- Trial name
- NCT01529840
- Testing
- somatropin
- Where
- Sweden
- Volunteers
- 24 people
- Sponsor
- Novo Nordisk A/S
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
Noonan Syndrome
Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
The trial will investigate the treatment of growth failure in children with Noonan syndrome. Abnormalities in the growth hormone (GH) - insulin-like growth factor-I (IGF-I) axis resulting in low IGF-I levels have been suggested…
- Trial name
- NCT00351221
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Insmed Incorporated