DDrare: Database of Drug Development for Rare Diseases

272. 進行性骨化性線維異形成症
［臨床試験数：23，薬物数：21（DrugBank：4），標的遺伝子数：10，標的パスウェイ数：87］

Searched query = "Fibrodysplasia ossificans progressiva", "FOP"
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 = AZD0530; AZD0530 Difumarate; Garetosmab; PALOVAROTENE; PALOVAROTENE - 1.5mg; PALOVAROTENE - 10 mg; PALOVAROTENE - 1mg; PALOVAROTENE - 2 mg; PALOVAROTENE - 2.5 mg; PALOVAROTENE - 3 mg; PALOVAROTENE - 4 mg; PALOVAROTENE - 5 mg; Palovarotene; Palovarotene dose level 1; Palovarotene dose level 2; Palovarotene dose level 3; Palovarotene dose level 4; Perhexiline; REGN2477; SARACATINIB DIFUMARATE; Saracatinib

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	NCT04307953 (ClinicalTrials.gov)	August 5, 2020	11/3/2020	Saracatinib Trial TO Prevent FOP	Saracatinib Trial TO Prevent FOP	Fibrodysplasia Ossificans Progressiva	Drug: AZD0530 Difumarate;Drug: Matching placebo	VU University Medical Center	Royal National Orthopaedic Hospital NHS Trust;Klinikum Garmisch-Patenkirchen;University of Oxford;Brigham and Women's Hospital;AstraZeneca;Innovative Medicines Initiative	Recruiting	18 Years	65 Years	All	20	Phase 2	Germany;Netherlands;United Kingdom
2	EUCTR2019-003324-20-NL (EUCTR)	08/01/2020	16/10/2019	A clinical trial to judge the safety, tolerability and effects on abnormal bone formation of reseach medication AZD 0530 (sarcatinib) in patients with FOP	Saracatinib trial TO Prevent FOP - STOPFOP	Fibrodyplasia Ossificans Progressiva (FOP) MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: Saracatinib Product Code: AZD0530 INN or Proposed INN: Saracatinib Other descriptive name: SARACATINIB DIFUMARATE	VU University Medical Center	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	16	Phase 2	Netherlands

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

NCT04307953
(ClinicalTrials.gov)

August 5, 2020

11/3/2020

Saracatinib Trial TO Prevent FOP

Fibrodysplasia Ossificans Progressiva

Drug: AZD0530 Difumarate;Drug: Matching placebo

VU University Medical Center

Royal National Orthopaedic Hospital NHS Trust;Klinikum Garmisch-Patenkirchen;University of Oxford;Brigham and Women's Hospital;AstraZeneca;Innovative Medicines Initiative

Recruiting

18 Years

65 Years

All

Phase 2

Germany;Netherlands;United Kingdom

EUCTR2019-003324-20-NL
(EUCTR)

08/01/2020

16/10/2019

A clinical trial to judge the safety, tolerability and effects on abnormal bone formation of reseach medication AZD 0530 (sarcatinib) in patients with FOP

Saracatinib trial TO Prevent FOP - STOPFOP

Fibrodyplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: Saracatinib
Product Code: AZD0530
INN or Proposed INN: Saracatinib
Other descriptive name: SARACATINIB DIFUMARATE

VU University Medical Center

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Netherlands