DDrare: Database of Drug Development for Rare Diseases

254. ポルフィリン症
［臨床試験数：59，薬物数：52（DrugBank：17），標的遺伝子数：17，標的パスウェイ数：33］

Searched query = "Porphyria", "CEP", "Erythropoietic protoporphyria", "EPP", "X-linked dominant protoporphyria", "XLDP", "PCT", "HEP", "AIP", "ADP", "VP", "Hereditary coproporphyria", "HCP"
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 = 1639325-43-1; 5-probe cocktail; A novel synthetic, orally-administered, non-peptide small molecule, which acts as; ALN-60519; ALN-AS1; Afamelanotide; Afamelanotide Implant; CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid); CAS# in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid); COLESTYRAMINE; CUV1647; Chlorpromazine; Chlorpromazine Hydrochloride; Colestipol; Colestyramine; Concentration unit: mg milligram(s); Cysteine; Cysteine hydrochloride; Deferasirox; Dersimelagon; Exjade; Givosiran; Givosiran (ALN-AS1); Glucose; Harvoni; Heme arginate; Hemin; Hemin for injection; Hydroxychloroquine; Iron; Isoniazid; Luteinizing hormone; Luteinizing hormone-releasing factor; MT-7117; MT-7117 High Dose; MT-7117 Low Dose; MT-7117 high dose; MT-7117 low dose; Nle4-D-Phe7-alpha-MSH; Oral Iron; Other: Glucose; Panhematin; Porphobilinogen; Procedure: Phlebotomy; Questran; Recombinant human porphobilinogen deaminase (Porphozym); SCENESSE; Scenesse; Sterile Normal Saline (0.9% NaCl); Tin mesoporphyrin; [MT-7117]; [Nle4,D-Phe7]-a-melanocyte stimulating hormone or NDP-a-MSH

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	EUCTR2009-011018-51-GB (EUCTR)	15/06/2009	27/04/2009	A Phase III, Safety Extension Study in Patients withErythropoietic Protoporphyria (EPP) - Phase III EPP Safety Extension Study	A Phase III, Safety Extension Study in Patients withErythropoietic Protoporphyria (EPP) - Phase III EPP Safety Extension Study	Erythropoietic Protoporphyria (EPP) MedDRA version: 13.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]	Product Name: Afamelanotide Product Code: CUV1647 INN or Proposed INN: afamelanotide Other descriptive name: Trade Name: SCENESSE	Clinuvel Pharmaceuticals Limited	NULL	Not Recruiting			Female: yes Male: yes	15	Phase 3	Finland;United Kingdom;Netherlands;Ireland

No.

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Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

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sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2009-011018-51-GB
(EUCTR)

15/06/2009

27/04/2009

A Phase III, Safety Extension Study in Patients withErythropoietic Protoporphyria (EPP) - Phase III EPP Safety Extension Study

Erythropoietic Protoporphyria (EPP)
MedDRA version: 13.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Product Name: Afamelanotide
Product Code: CUV1647
INN or Proposed INN: afamelanotide
Other descriptive name: Trade Name: SCENESSE

Clinuvel Pharmaceuticals Limited

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Finland;United Kingdom;Netherlands;Ireland