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 195. Noonan syndrome
 [ 10 clinical trials,    11 drugs(DrugBank: 4 drugs),    2 target genes / 9 target pathways

Searched query = "Noonan syndrome"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03435627February 26, 201827 May 2019Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)Genetic Disorder;Noonan SyndromeDrug: SomatropinNovo Nordisk A/SRecruiting3 YearsN/AAll60N/AJapan
2EUCTR2016-005022-10-DE13/02/201823 July 2018Improvement of synaptic plasticity and cognitive function in RAS pathway disordersImprovement of synaptic plasticity and cognitive function in RAS pathway disordersNoonan Syndrom and Neurofibromatosis Type 1
MedDRA version: 20.0 Level: PT Classification code 10029748 Term: Noonan syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: PT Classification code 10029268 Term: Neurofibromatosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Not possible to specify
Product Name: Lovastatin
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Lovastatin
CAS Number: 75330-75-5
Other descriptive name: LOVASTATIN
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use
Product Name: Lamotrigin
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Lamotrigin
CAS Number: 84057-84-1
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use
Technische Universität München Fakultät für MedizinAuthorisedFemale: yes
Male: yes
28Phase 2Germany
3NCT02713945January 25, 201722 July 2019Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan SyndromeTreatment With HMG-COA Reductase Inhibitor (Simvastatin) of Growth and Bone Abnormalities in Children With Noonan Syndrome: A Phase III Randomised, Double Blind, Placebo-controlled Therapeutic TrialNoonan SyndromeDrug: Simvastatin;Drug: PlaceboUniversity Hospital, ToulouseRecruiting6 Years16 YearsAll62Phase 3France
4EUCTR2016-000647-14-FR23/05/201610 September 2018Treatment with simvastatin of growth and bone abnormalities in children with Noonan syndromeTreatment with HMG-COA reductase inhibitor (simvastatin) of growth and bone abnormalities in children with Noonan syndrome : a phase III randomised, double-blind, placebo-controlled therapeutic trial - RASTATNoonan syndrome;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]Trade Name: Simvastatin
Product Name: Simvastatin
Pharmaceutical Form: Film-coated tablet
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use
University Hospital ToulouseAuthorisedFemale: yes
Male: yes
62Phase 3France
5NCT01927861August 19, 201320 August 2018Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan SyndromeA 52-week, Multi-centre, Randomised, Double-blind, Parallel-group, no Treatment Controlled (Open-label) Trial Investigating the Efficacy and Safety of Two Doses of NN-220 in Short Stature With Noonan SyndromeGenetic Disorder;Noonan SyndromeDrug: somatropinNovo Nordisk A/SNot recruiting3 Years10 YearsAll51Phase 3Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2011-003392-10-GB23/01/201219 March 2012A study to look at the effect of the study drug on heart muscle thickness, the amount of drug that ends up in the blood, and the safety and tolerability of study drug in Noonan syndrome patients.An open label study to assess safety, tolerability, pharmacokinetics and pharmacodynamics of MEK162 in Noonan syndrome hypertrophic cardiomyopathy.Noonan syndrome hypertrophic cardiomyopathy
MedDRA version: 14.1 Level: PT Classification code 10029748 Term: Noonan syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1 Level: PT Classification code 10020871 Term: Hypertrophic cardiomyopathy System Organ Class: 10007541 - Cardiac disorders ;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Code: MEK162
Pharmaceutical Form: Film-coated tablet
CAS Number: 606143-89-9
Current Sponsor code: MEK162
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 15-
Novartis Pharma Services AGAuthorisedFemale: yes
Male: yes
22United Kingdom;United States
7NCT01529944September 200819 February 2015Genetic Testing of Noonan Subjects Previously Treated With Norditropin®. An Extension to Trial GHNOO-1658Genetic Testing of Noonan Subjects Previously Treated With Norditropin® in the GHNOO-1658 TrialGenetic Disorder;Noonan SyndromeDrug: somatropinNovo Nordisk A/SNot recruitingN/AN/ABoth22Phase 3Sweden
8NCT00351221May 200619 February 2015Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan SyndromeA Phase 2, Open-Label, Multicenter, Clinical Trial to Evaluate the Pharmacokinetics, Safety and Efficacy of Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 in Children With Growth Failure Due to Noonan SyndromeNoonan SyndromeDrug: rhIGF-1/rhIGFBP-3Insmed IncorporatedNot recruiting2 Years16 YearsBoth24Phase 2United States
9EUCTR2005-000042-37-SE11/07/200519 March 2012Norditropin Treatment in Subjects with Noonan Syndrome. Effects on Linear Growth and Final Height - Data Collection and Follow-up VisitNorditropin Treatment in Subjects with Noonan Syndrome. Effects on Linear Growth and Final Height - Data Collection and Follow-up VisitNoonan syndrome associated short stature
MedDRA version: 7.1 Level: PT Classification code 10029748
Trade Name: Norditropin 24 IU
Product Name: Norditropin 24 IU
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Somatropin
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 24-
Trade Name: Norditropin SimpleXx
Product Name: Norditropin SimpleXx
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Somatropin
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 3,3-
INN or Proposed INN: Somatropin
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 6,7-
INN or Proposed INN: Somatropin
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Novo Nordisk A/SNot RecruitingFemale: yes
Male: yes
Sweden
10NCT00452725October 199719 February 2015Effect of MAXOMAT ® on the Growth of Small Children to NOONAN's SyndromeEffect of the Growth Hormone MAXOMAT ® on the Growth of Small Children and Adolescents (<-2 SD) Due to NOONAN's SyndromeNoonan SyndromeDrug: MAXOMAT ®, biosynthetic growth hormoneSanofiNot recruiting3 Years15 YearsBoth36Phase 3France

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