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	EUCTR2013-004137-32-FR (EUCTR)		18/06/2015	A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia	A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia	achondroplasia MedDRA version: 18.0;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: TBD Other descriptive name: MODIFIED RHCNP	BioMarin Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	24	Phase 2	United States;France;Australia;United Kingdom

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EUCTR2013-004137-32-FR
(EUCTR)

18/06/2015

A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia

A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia

achondroplasia
MedDRA version: 18.0;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: TBD
Other descriptive name: MODIFIED RHCNP

BioMarin Pharmaceutical Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;France;Australia;United Kingdom