254. ポルフィリン症 Porphyria Clinical trials / Disease details


臨床試験数 : 72 薬物数 : 53 - (DrugBank : 16) / 標的遺伝子数 : 19 - 標的パスウェイ数 : 35

  
15 trials found
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1EUCTR2017-002432-17-NL
(EUCTR)
21/06/201819/02/2018Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of
2EUCTR2017-002432-17-FI
(EUCTR)
21/05/201830/11/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of
3EUCTR2017-002432-17-PL
(EUCTR)
12/04/201816/01/2018Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Czechia;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Brazil;Belgium;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of
4EUCTR2017-002432-17-IT
(EUCTR)
19/02/201802/02/2021Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
ALNYLAM PHARMACEUTICALS, INC.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Czechia;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Brazil;Belgium;Denmark;Australia;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of
5EUCTR2017-002432-17-BG
(EUCTR)
19/02/201809/11/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 24.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of
6EUCTR2017-002432-17-DE
(EUCTR)
03/01/201804/10/2017ENVISION: 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 - ENVISION 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of
7EUCTR2017-002432-17-BE
(EUCTR)
29/12/201719/12/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of
8EUCTR2017-002432-17-SE
(EUCTR)
21/12/201707/11/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of
9EUCTR2016-002638-54-SE
(EUCTR)
20/12/201709/08/2016Long-term Safety and Clinical Activity of ALN-AS1 in Patients with Acute Intermittent Porphyria (AIP), a rare metabolic disorder.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 (AIP)
MedDRA version: 20.0;Level: LLT;Classification code 10000818;Term: Acute intermittent porphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
24Phase 1;Phase 2United States;United Kingdom;Sweden
10EUCTR2017-002432-17-DK
(EUCTR)
18/12/201725/10/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Denmark;Australia;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of
11EUCTR2017-002432-17-ES
(EUCTR)
11/12/201717/10/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of
12EUCTR2017-002432-17-GB
(EUCTR)
22/11/201726/09/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;Italy;United Kingdom;Switzerland;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of;Sweden
13EUCTR2016-002638-54-GB
(EUCTR)
30/08/201618/08/2016Long-term Safety and Clinical Activity of ALN-AS1 in Patients with Acute Intermittent Porphyria (AIP), a rare metabolic disorder.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 (AIP)
MedDRA version: 20.0;Level: LLT;Classification code 10000818;Term: Acute intermittent porphyria;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS# in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
24 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom;Sweden
14EUCTR2017-002432-17-CZ
(EUCTR)
02/11/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;Italy;Switzerland;United Kingdom;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of;Sweden
15EUCTR2017-002432-17-FR
(EUCTR)
15/01/2018Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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 - ENVISION Acute Hepatic Porphyrias (AHP)
MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
INN or Proposed INN: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of