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	NCT03338816 (ClinicalTrials.gov)	November 16, 2017	7/11/2017	ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)	ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias	Acute Hepatic Porphyria;Acute Intermittent Porphyria;Porphyria, Acute Intermittent;Acute Porphyria;Hereditary Coproporphyria (HCP);Variegate Porphyria (VP);ALA Dehydratase Deficient Porphyria (ADP)	Drug: Givosiran;Drug: Placebo	Alnylam Pharmaceuticals	NULL	Active, not recruiting	12 Years	N/A	All	94	Phase 3	United States;Australia;Bulgaria;Canada;Denmark;Finland;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Spain;Sweden;Taiwan;United Kingdom;Belgium;Switzerland
2	NCT02949830 (ClinicalTrials.gov)	October 2016	28/10/2016	A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)	A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1	Acute Intermittent Porphyria	Drug: givosiran (ALN-AS1)	Alnylam Pharmaceuticals	NULL	Active, not recruiting	18 Years	N/A	All	17	Phase 1;Phase 2	United States;Sweden;United Kingdom
3	NCT02452372 (ClinicalTrials.gov)	May 6, 2015	19/5/2015	A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)	A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)	Acute Intermittent Porphyria	Drug: givosiran (ALN-AS1);Drug: Sterile Normal Saline (0.9% NaCl)	Alnylam Pharmaceuticals	NULL	Completed	18 Years	65 Years	All	40	Phase 1	United States;Sweden;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

NCT03338816
(ClinicalTrials.gov)

November 16, 2017

7/11/2017

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias

Acute Hepatic Porphyria;Acute Intermittent Porphyria;Porphyria, Acute Intermittent;Acute Porphyria;Hereditary Coproporphyria (HCP);Variegate Porphyria (VP);ALA Dehydratase Deficient Porphyria (ADP)

Drug: Givosiran;Drug: Placebo

Alnylam Pharmaceuticals

NULL

Active, not recruiting

12 Years

N/A

All

Phase 3

United States;Australia;Bulgaria;Canada;Denmark;Finland;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Spain;Sweden;Taiwan;United Kingdom;Belgium;Switzerland

NCT02949830
(ClinicalTrials.gov)

October 2016

28/10/2016

A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1

Acute Intermittent Porphyria

Drug: givosiran (ALN-AS1)

Alnylam Pharmaceuticals

NULL

Active, not recruiting

18 Years

N/A

All

Phase 1;Phase 2

United States;Sweden;United Kingdom

NCT02452372
(ClinicalTrials.gov)

May 6, 2015

19/5/2015

A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)

Acute Intermittent Porphyria

Drug: givosiran (ALN-AS1);Drug: Sterile Normal Saline (0.9% NaCl)

Alnylam Pharmaceuticals

NULL

Completed

18 Years

65 Years

All

Phase 1

United States;Sweden;United Kingdom