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
| 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 | JPRN-jRCT2071200074 | 02/02/2021 | 24/12/2020 | Expanded Access Protocol of Givosiran for Patients with Acute Hepatic Porphyria | Expanded 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 Norihisa | NULL | Complete | >= 12age old | Not applicable | Both | 10 | N/A | Japan |
| 2 | EUCTR2017-002432-17-NL (EUCTR) | 21/06/2018 | 19/02/2018 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United 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 | ||
| 3 | EUCTR2017-002432-17-FI (EUCTR) | 21/05/2018 | 30/11/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 4 | NCT03505853 (ClinicalTrials.gov) | April 26, 2018 | 13/4/2018 | A 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 Porphyria | Drug: Givosiran;Drug: 5-probe cocktail | Alnylam Pharmaceuticals | NULL | Completed | 18 Years | 65 Years | All | 10 | Phase 1 | Sweden |
| 5 | EUCTR2017-002432-17-PL (EUCTR) | 12/04/2018 | 16/01/2018 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United 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 | ||
| 6 | EUCTR2017-002432-17-IT (EUCTR) | 19/02/2018 | 02/02/2021 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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 | ALNYLAM PHARMACEUTICALS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United 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 | ||
| 7 | EUCTR2017-002432-17-BG (EUCTR) | 19/02/2018 | 09/11/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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: 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 8 | EUCTR2017-002432-17-DE (EUCTR) | 03/01/2018 | 04/10/2017 | 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 | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United 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 | ||
| 9 | EUCTR2017-002432-17-BE (EUCTR) | 29/12/2017 | 19/12/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United 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 | ||
| 10 | EUCTR2017-002432-17-SE (EUCTR) | 21/12/2017 | 07/11/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 11 | EUCTR2016-002638-54-SE (EUCTR) | 20/12/2017 | 09/08/2016 | Long-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. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 1;Phase 2 | United States;United Kingdom;Sweden | ||
| 12 | EUCTR2017-002432-17-DK (EUCTR) | 18/12/2017 | 25/10/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Denmark;Australia;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 13 | EUCTR2017-002432-17-ES (EUCTR) | 11/12/2017 | 17/10/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United 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 | ||
| 14 | EUCTR2017-002432-17-GB (EUCTR) | 22/11/2017 | 26/09/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 74 | Phase 3 | United 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 | ||
| 15 | 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 | Completed | 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 |
| 16 | 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 | Completed | 18 Years | N/A | All | 16 | Phase 1/Phase 2 | United States;Sweden;United Kingdom |
| 17 | EUCTR2016-002638-54-GB (EUCTR) | 30/08/2016 | 18/08/2016 | Long-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. | NULL | Authorised-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): no | United States;United Kingdom;Sweden | ||
| 18 | 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 |
| 19 | EUCTR2017-002432-17-CZ (EUCTR) | 02/11/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United 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 | |||
| 20 | EUCTR2017-002432-17-FR (EUCTR) | 15/01/2018 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | 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. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United 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 |