DDrare: Database of Drug Development for Rare Diseases

276. 軟骨無形成症
［臨床試験数：37，薬物数：29（DrugBank：6），標的遺伝子数：4，標的パスウェイ数：25］

Searched query = "Achondroplasia"
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 = Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111; BGJ398 (also known as BBP-831); BMN 111; C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER; INFIGRATINIB; Infigratinib; Infigratinib 0.016 mg/kg; Infigratinib 0.032 mg/kg; Infigratinib 0.064 mg/kg; Infigratinib 0.128 mg/kg; Infigratinib phosphate; MODIFIED RHCNP; Modified recombinant human C-type natriuretic peptide; Modified recombinant human c-type; Natriuretic peptide; Normal Saline; PF-07256472; Phosphate; Placebo; Polyethylene glycol; Recifercept; Recifercept (proposed INN); Somatropin; TA-46; TBD; TransCon CNP; TransCon CNP 3.9 mg CNP-38/vial; Vorsoritide; Vosoritide

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	EUCTR2015-003836-11-ES (EUCTR)	19/04/2017	10/03/2017	A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia	A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. -	achondroplasia MedDRA version: 19.1;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]	Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vorsoritide Other descriptive name: MODIFIED RHCNP	BioMarin Pharmaceutical Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110	Phase 3	France;United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom

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agemin

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EUCTR2015-003836-11-ES
(EUCTR)

19/04/2017

10/03/2017

A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. -

achondroplasia
MedDRA version: 19.1;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]

Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vorsoritide
Other descriptive name: MODIFIED RHCNP

BioMarin Pharmaceutical Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

110

Phase 3

France;United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom