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	EUCTR2019-002954-21-GB (EUCTR)	31/03/2020	13/05/2020	A study to evaluate Infigratinib in children with Achondroplasia	Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2 - PROPEL 2	Achondroplasia in Children MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: Infigratinib phosphate Product Code: BGJ398 (also known as BBP-831) INN or Proposed INN: INFIGRATINIB	QED Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	78	Phase 2	United States;France;Canada;Spain;Australia;United Kingdom
2	NCT04265651 (ClinicalTrials.gov)	March 10, 2020	29/1/2020	Study of Infigratinib in Children With Achondroplasia	Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2	Achondroplasia	Drug: Infigratinib 0.016 mg/kg;Drug: Infigratinib 0.032 mg/kg;Drug: Infigratinib 0.064 mg/kg;Drug: Infigratinib 0.128 mg/kg	QED Therapeutics, Inc.	NULL	Recruiting	3 Years	11 Years	All	60	Phase 2	Australia;Spain;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

EUCTR2019-002954-21-GB
(EUCTR)

31/03/2020

13/05/2020

A study to evaluate Infigratinib in children with Achondroplasia

Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2 - PROPEL 2

Achondroplasia in Children
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: Infigratinib phosphate
Product Code: BGJ398 (also known as BBP-831)
INN or Proposed INN: INFIGRATINIB

QED Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;France;Canada;Spain;Australia;United Kingdom

NCT04265651
(ClinicalTrials.gov)

March 10, 2020

29/1/2020

Study of Infigratinib in Children With Achondroplasia

Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2

Achondroplasia

Drug: Infigratinib 0.016 mg/kg;Drug: Infigratinib 0.032 mg/kg;Drug: Infigratinib 0.064 mg/kg;Drug: Infigratinib 0.128 mg/kg

QED Therapeutics, Inc.

NULL

Recruiting

3 Years

11 Years

All

Phase 2

Australia;Spain;United Kingdom