Givosiran ( DrugBank: Givosiran )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
254ポルフィリン症20

254. ポルフィリン症


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
No.TrialIDDate_
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PhaseCountries
1JPRN-jRCT2071200074
02/02/202124/12/2020Expanded Access Protocol of Givosiran for Patients with Acute Hepatic PorphyriaExpanded Access Protocol of Givosiran for Patients with Acute Hepatic Porphyria (AHP) Acute hepatic porphyria (AHP)Patients will be administered givosiran 2.5 mg/kg as a subcutaneous (SC) injection once monthly.Fujii NorihisaNULLComplete>= 12age oldNot applicableBoth10N/AJapan
2EUCTR2017-002432-17-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
3EUCTR2017-002432-17-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
4NCT03505853
(ClinicalTrials.gov)
April 26, 201813/4/2018A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)A Drug-Drug Interaction Study to Investigate the Effect of Givosiran on the Pharmacokinetics (PK) of Midazolam, Caffeine, Losartan, Omeprazole, and Dextromethorphan in Patients With Acute Intermittent Porphyria (AIP) Who Are Asymptomatic High Excreters (ASHE)Acute Intermittent Porphyria (AIP);Acute Hepatic Porphyria (AHP);Porphyria, Acute Intermittent;Acute PorphyriaDrug: Givosiran;Drug: 5-probe cocktailAlnylam PharmaceuticalsNULLCompleted18 Years65 YearsAll10Phase 1Sweden
5EUCTR2017-002432-17-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
6EUCTR2017-002432-17-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
7EUCTR2017-002432-17-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
8EUCTR2017-002432-17-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
9EUCTR2017-002432-17-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
10EUCTR2017-002432-17-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
11EUCTR2016-002638-54-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
12EUCTR2017-002432-17-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
13EUCTR2017-002432-17-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
14EUCTR2017-002432-17-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
15NCT03338816
(ClinicalTrials.gov)
November 16, 20177/11/2017ENVISION: 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 PorphyriasAcute Hepatic Porphyria;Acute Intermittent Porphyria;Porphyria, Acute Intermittent;Acute Porphyria;Hereditary Coproporphyria (HCP);Variegate Porphyria (VP);ALA Dehydratase Deficient Porphyria (ADP)Drug: Givosiran;Drug: PlaceboAlnylam PharmaceuticalsNULLCompleted12 YearsN/AAll94Phase 3United States;Australia;Bulgaria;Canada;Denmark;Finland;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Spain;Sweden;Taiwan;United Kingdom;Belgium;Switzerland
16NCT02949830
(ClinicalTrials.gov)
October 201628/10/2016A 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-AS1Acute Intermittent PorphyriaDrug: givosiran (ALN-AS1)Alnylam PharmaceuticalsNULLCompleted18 YearsN/AAll16Phase 1/Phase 2United States;Sweden;United Kingdom
17EUCTR2016-002638-54-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
18NCT02452372
(ClinicalTrials.gov)
May 6, 201519/5/2015A 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 PorphyriaDrug: givosiran (ALN-AS1);Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNULLCompleted18 Years65 YearsAll40Phase 1United States;Sweden;United Kingdom
19EUCTR2017-002432-17-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
20EUCTR2017-002432-17-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