DDrare: Database of Drug Development for Rare Diseases

193. プラダー・ウィリ症候群
［臨床試験数：95，薬物数：104（DrugBank：27），標的遺伝子数：50，標的パスウェイ数：63］

Searched query = "Prader-Willi syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 1.2 mg ZGN-440 for injectable suspension; 1.8 mg ZGN-440 for injectable suspension; ACOMPLIA 20 mg film-coated tablets; ACTH; AZP-531; Acetylcysteine; Acomplia; Air; Anastrozole; B. lactis B94; Beloranib; Byetta; CBDV; CBDV Compound; Cannabidiol; Cannabidiol Oral Solution; Carbetocin; Chloride; Choline; Coenzyme Q10; D.3.2 Product code where applicable13:; DCCR; DIAZOXIDE CHOLINE; Diazoxide; Diazoxide choline; Diazoxide choline controlled-release tablet; EU/1/06/362/003: 5µg (1 pen); Eutropin; Exenatide; FE 992097; Fluimucil; Fluimucil (acetylcysteine); GENOTONORM; GENOTROPIN (Somatropin) is a growth hormone treatment. It is an exact copy of the natural growth hormone that our bodies make. The main difference is that GENOTROPIN is man-made.; GLWL-01; Genotonorm; Genotonorm Miniquick 0,2 mg; Genotropin; Growth hormone; Growth hormone (Genotonorm® or Omnitrope®); Guanfacine; Guanfacine extended release (GXR); Human growth hormone; Humatrope 6 mg/12 mg/24 mg injektiokuiva-aine ja liuotin, liuosta varten; INN Not yet proposed; Intranasal Oxytocin (IN-OXT); Intranasal carbetocin Dose 1; Intranasal carbetocin Dose 2; Intranasal oxytocin; LIRAGLUTIDE; Liraglutide; Livoletide; METOPROLOL SUCCINATE; Medical Air vs Oxygen; Medical air; Metoprolol; Metoprololsuccinat Orion 25mg; Metoprololsuccinat ”Orion” 25mg; Metyrapone; Norditropin SimpleXx; Nutropin AQ; OMNITROPE; OT; OXYTOCIN; Octreotide; Omnitrope; Other: Control; Other: Low dose (1 mcg) ACTH stimulation test; Other: Overnight metyrapone test; Oxygen; Oxytocin; Oxytocin (OXT) continuous; Physiological serum (Sodium chloride); Pitolisant; Pitolisant oral tablets; Probiotic; Procedure: DEXA, blood tests, H.G.P.O, osseous age.; Procedure: biopsy; R05CB01; RECOMBINANT HUMAN GROWTH HORMON; RECOMBINANT HUMAN GROWTH HORMONE; RM-493; Recombinant Human Growth Hormone (rhGH) Injection; Recombinant Somatropin; Recombinant human growth hormone; RhGH; Rimonabant; SOMATROPIN; Sandostatin LAR; Saxenda; Sodium chloride; Somatropin; Subcutaneous Beloranib in Suspension; Syntocinon; Syntocinon nasal spray; Syntocinon®/- Spray; TESOFENSINE; Tesofensine; Tesofensine/Metoprolol; Topiramate; ZGN-440; ZGN-440 for Injectable Suspension; ZGN-440 sterile diluent; [AZP-531]

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2019-000735-61-NL (EUCTR)		04/08/2020	Treatment with N-acetylcysteine for skin picking in children and young adults with PWS	N- acetylcysteine treatment for skin picking in children and young adults with PWS: a randomized, controlled, cross-over trial.	Prader-Willi syndrome MedDRA version: 20.0;Level: PT;Classification code 10036476;Term: Prader-Willi syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Hormonal diseases [C19]	Trade Name: Fluimucil (acetylcysteine) Product Name: Fluimucil Product Code: R05CB01	Dutch Growth Research Foundation	NULL	NA			Female: yes Male: yes	35	Phase 2;Phase 3	Netherlands

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EUCTR2019-000735-61-NL
(EUCTR)

04/08/2020

Treatment with N-acetylcysteine for skin picking in children and young adults with PWS

N- acetylcysteine treatment for skin picking in children and young adults with PWS: a randomized, controlled, cross-over trial.

Prader-Willi syndrome
MedDRA version: 20.0;Level: PT;Classification code 10036476;Term: Prader-Willi syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Trade Name: Fluimucil (acetylcysteine)
Product Name: Fluimucil
Product Code: R05CB01

Dutch Growth Research Foundation

NULL

Female: yes
Male: yes

Phase 2;Phase 3

Netherlands