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	NCT03554031 (ClinicalTrials.gov)	April 14, 2018	30/5/2018	A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome	A Single Arm, Multicenter Phase III Clinical Trial to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome	Prader-Willi Syndrome	Drug: Recombinant Human Growth Hormone (rhGH) Injection	GeneScience Pharmaceuticals Co., Ltd.	Children's Hospital of Fudan University;Tongji medical college huazhong university of science & technology;Beijing Children's Hospital;Peking University First Hospital;Peking Union Medical College Hospital;The Children's Hospital of Zhejiang University School of Medicine	Unknown status	1 Month	5 Years	All	30	Phase 3	China
2	EUCTR2017-002164-41-ES (EUCTR)	03/07/2017	21/06/2017	Study with adult patients with Prader-Willi syndrome, which evaluate the effect of growth hormone therapy on muscle tone and its relation with muscle strength and body composition assessed with imaging procedures.	Growth hormone therapy in adults with Prader-Willi syndrome: Effect on muscle tone assessed by functional magnetic resonance imaging (fMRI) and its relation to muscle strenght and body composition.	Patients with Prader-Willi Syndrome (SPW) with Growth hormone deficit. 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: Genotonorm Miniquick 0,2 mg INN or Proposed INN: RECOMBINANT HUMAN GROWTH HORMON Other descriptive name: RECOMBINANT HUMAN GROWTH HORMONE	Fundació Parc Taulí	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes		Phase 4	Spain

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

NCT03554031
(ClinicalTrials.gov)

April 14, 2018

30/5/2018

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome

A Single Arm, Multicenter Phase III Clinical Trial to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome

Drug: Recombinant Human Growth Hormone (rhGH) Injection

GeneScience Pharmaceuticals Co., Ltd.

Children's Hospital of Fudan University;Tongji medical college huazhong university of science & technology;Beijing Children's Hospital;Peking University First Hospital;Peking Union Medical College Hospital;The Children's Hospital of Zhejiang University School of Medicine

Unknown status

1 Month

5 Years

All

Phase 3

China

EUCTR2017-002164-41-ES
(EUCTR)

03/07/2017

21/06/2017

Study with adult patients with Prader-Willi syndrome, which evaluate the effect of growth hormone therapy on muscle tone and its relation with muscle strength and body composition assessed with imaging procedures.

Growth hormone therapy in adults with Prader-Willi syndrome: Effect on muscle tone assessed by functional magnetic resonance imaging (fMRI) and its relation to muscle strenght and body composition.

Patients with Prader-Willi Syndrome (SPW) with Growth hormone deficit.
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: Genotonorm Miniquick 0,2 mg
INN or Proposed INN: RECOMBINANT HUMAN GROWTH HORMON
Other descriptive name: RECOMBINANT HUMAN GROWTH HORMONE

Fundació Parc Taulí

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 4

Spain