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	EUCTR2020-001189-13-DK (EUCTR)	22/12/2020	07/10/2020	Phase 2 study of safety, tolerability, PK and efficacy of recifercept in achondroplasia	A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA	Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]	Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46	Pfizer Inc., 235 East 42nd Street, New York, NY 10017	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	63	Phase 2	Portugal;United States;Spain;Belgium;Australia;Denmark;United Kingdom;Italy;Japan
2	NCT04638153 (ClinicalTrials.gov)	December 2, 2020	15/9/2020	A Study Of Safety, Tolerability And Effectiveness Of Reciferecept In Children With Achondroplasia	A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA	Achondroplasia	Biological: Recifercept	Pfizer	NULL	Recruiting	3 Months	10 Years	All	63	Phase 2	Belgium;Spain
3	EUCTR2020-001189-13-PT (EUCTR)		29/09/2020	Phase 2 study of safety, tolerability, PK and efficacy of recifercept in achondroplasia	A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA	Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]	Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46	Pfizer Inc.	NULL	NA			Female: yes Male: yes	63	Phase 2	United States;Portugal;Canada;Spain;Belgium;Denmark;Australia;United Kingdom;Japan;Italy

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

EUCTR2020-001189-13-DK
(EUCTR)

22/12/2020

07/10/2020

Phase 2 study of safety, tolerability, PK and efficacy of recifercept in achondroplasia

A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA

Achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]

Product Name: Recifercept (proposed INN)
Product Code: PF-07256472
INN or Proposed INN: Recifercept (proposed INN)
Other descriptive name: TA-46

Pfizer Inc., 235 East 42nd Street, New York, NY 10017

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Portugal;United States;Spain;Belgium;Australia;Denmark;United Kingdom;Italy;Japan

NCT04638153
(ClinicalTrials.gov)

December 2, 2020

15/9/2020

A Study Of Safety, Tolerability And Effectiveness Of Reciferecept In Children With Achondroplasia

A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA

Achondroplasia

Biological: Recifercept

Pfizer

NULL

Recruiting

3 Months

10 Years

All

Phase 2

Belgium;Spain

EUCTR2020-001189-13-PT
(EUCTR)

29/09/2020

Phase 2 study of safety, tolerability, PK and efficacy of recifercept in achondroplasia

A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA

Product Name: Recifercept (proposed INN)
Product Code: PF-07256472
INN or Proposed INN: Recifercept (proposed INN)
Other descriptive name: TA-46

Pfizer Inc.

NULL

Female: yes
Male: yes

Phase 2

United States;Portugal;Canada;Spain;Belgium;Denmark;Australia;United Kingdom;Japan;Italy