DDrare: Database of Drug Development for Rare Diseases

166. 弾性線維性仮性黄色腫
［臨床試験数：11，薬物数：23（DrugBank：6），標的遺伝子数：5，標的パスウェイ数：26］

Searched query = "Pseudoxanthoma elasticum", "PXE"
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 = Aflibercept; CAS No.: 7758-16-9; Disodium Pyrophosphate; Eylea; Intravitreal injection ranibizumab; LANSOPRAZOLE; Lansolprazole; Lansoprazole; Lansoprazole 30mg; Lucentis; Magnesium; Magnesium Oxide; Magnesium oxide; Pyrophosphate; RANIBIZUMAB; RFB002; RFB002, rhuFab V2; RFB002A; Ranibizumab; Ranibizumab injection (0.5 mg); RhuFab V2; SODIUM ACID PYROPHOSPHATE; SODIUM ACID PYROPHOSPHATE SAPP ROR 28 FOOD GRADE

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	NCT04441671 (ClinicalTrials.gov)	December 8, 2020	20/1/2020	Oral Pyrophosphate Absorption in PXE Disease	Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)	Pseudoxanthoma Elasticum	Drug: Disodium Pyrophosphate	Tampere University Hospital	UMC Utrecht;Hungarian Academy of Sciences	Not yet recruiting	18 Years	80 Years	All	12	Phase 2	NULL
2	NCT04660461 (ClinicalTrials.gov)	February 4, 2020	3/12/2020	Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)	Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)	Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)	Drug: Lansoprazole 30mg;Drug: Placebo	Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud	NULL	Recruiting	18 Years	N/A	All	20	Phase 4	Spain
3	EUCTR2016-004021-16-ES (EUCTR)	31/10/2018	10/03/2017	Response to oral lansoprazole in patients with Pseudoxanthoma Elasticum	Response to oral lansoprazole in inorganic pyrophosphate levels in patients with Grönblad-Stranberg disease (Pseudoxanthoma Elasticum) - FIM-PXE-2016-01	Grönblad-Stranberg disease (Pseudoxanthoma Elasticum) MedDRA version: 19.1;Level: PT;Classification code 10037150;Term: Pseudoxanthoma elasticum;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]	Trade Name: Lansoprazole INN or Proposed INN: Lansolprazole Other descriptive name: LANSOPRAZOLE	Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud (FIMABIS)	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 2	Spain
4	EUCTR2018-001492-20-FI (EUCTR)	20/06/2018	11/06/2018	Pyrophosphate therapy in PXE disease	Pseudoxanthoma elasticum – supplementation therapy of pyrophosphate deficiency	Pseudoxanthoma elasticum;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: SODIUM ACID PYROPHOSPHATE Product Name: SODIUM ACID PYROPHOSPHATE SAPP ROR 28 FOOD GRADE Product Code: CAS No.: 7758-16-9	Tampere university hospital	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	100	Phase 2;Phase 3	Finland

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

NCT04441671
(ClinicalTrials.gov)

December 8, 2020

20/1/2020

Oral Pyrophosphate Absorption in PXE Disease

Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)

Pseudoxanthoma Elasticum

Drug: Disodium Pyrophosphate

Tampere University Hospital

UMC Utrecht;Hungarian Academy of Sciences

Not yet recruiting

18 Years

80 Years

All

Phase 2

NULL

NCT04660461
(ClinicalTrials.gov)

February 4, 2020

3/12/2020

Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)

Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)

Drug: Lansoprazole 30mg;Drug: Placebo

Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud

NULL

Recruiting

18 Years

N/A

All

Phase 4

Spain

EUCTR2016-004021-16-ES
(EUCTR)

31/10/2018

10/03/2017

Response to oral lansoprazole in patients with Pseudoxanthoma Elasticum

Response to oral lansoprazole in inorganic pyrophosphate levels in patients with Grönblad-Stranberg disease (Pseudoxanthoma Elasticum) - FIM-PXE-2016-01

Grönblad-Stranberg disease (Pseudoxanthoma Elasticum)
MedDRA version: 19.1;Level: PT;Classification code 10037150;Term: Pseudoxanthoma elasticum;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Trade Name: Lansoprazole
INN or Proposed INN: Lansolprazole
Other descriptive name: LANSOPRAZOLE

Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud (FIMABIS)

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Spain

EUCTR2018-001492-20-FI
(EUCTR)

20/06/2018

11/06/2018

Pyrophosphate therapy in PXE disease

Pseudoxanthoma elasticum – supplementation therapy of pyrophosphate deficiency

Pseudoxanthoma elasticum;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: SODIUM ACID PYROPHOSPHATE
Product Name: SODIUM ACID PYROPHOSPHATE SAPP ROR 28 FOOD GRADE
Product Code: CAS No.: 7758-16-9

Tampere university hospital

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

100

Phase 2;Phase 3

Finland