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	NCT03989947 (ClinicalTrials.gov)	June 12, 2019	22/5/2019	An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia	A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia	Achondroplasia	Drug: Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111	BioMarin Pharmaceutical	NULL	Enrolling by invitation	15 Months	N/A	All	70	Phase 2	United States;Australia;Japan;United Kingdom

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

NCT03989947
(ClinicalTrials.gov)

June 12, 2019

22/5/2019

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia

Achondroplasia

Drug: Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111

BioMarin Pharmaceutical

NULL

Enrolling by invitation

15 Months

N/A

All

Phase 2

United States;Australia;Japan;United Kingdom